A Long-Term Safety Study of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)

A Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Status

Terminated

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT04702568

Enrollment

Registered

2021-01-11

Start date

2020-12-18

Completion date

2023-10-04

Last updated

2025-04-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Paroxysmal Nocturnal Hemoglobinuria, PNH

Keywords

BCX9930, Factor D inhibitor, PNH, paroxysmal Nocturnal Hemoglobinuria, oral treatment

Brief summary

This study was designed to evaluate the long-term safety of daily oral treatment with BCX9930 in participants who had participated in a previous BCX9930 trial for PNH and showed a benefit of treatment as determined by the Investigator. The study allowed continued access to BCX9930 for enrolled participants. The study also evaluated the long-term effectiveness and impact on quality of life and general well-being of BCX9930 treatment, and the participant's satisfaction with the medication.

Interventions

DRUGBCX9930

BCX9930 for oral administration

DRUGEculizumab

Administered at stable dose at the time of study entry

DRUGRavulizumab

Administered at stable dose at the time of study entry

Study design

Allocation

NON_RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

Inclusion criteria

* Male or non-pregnant, non-lactating female participants * Successfully participated in a previous BCX9930 study of PNH and experienced improvement in their PNH

Exclusion criteria

* Apart from a diagnosis of PNH, any clinically significant medical or psychiatric condition or medical history, other than those associated with PNH disease, that, in the opinion of the Investigator or Sponsor, would interfere with the participant's ability to participate in the study or participation would increase the risk for that participant * Pregnant, planning to become pregnant, or having been pregnant within 90 days of Day 1, or lactating Note: Other protocol-defined Inclusion/

Design outcomes

Primary

Measure	Time frame	Description
Number of Participants With Treatment Emergent Adverse Events (TEAEs)	From first dose of study drug up to 3 weeks after last dose (Week 147)	An AE was defined as any untoward medical occurrence associated with the use of an intervention in humans, whether or not considered intervention-related in a participant or clinical investigation participant who administered a pharmaceutical product regardless of its causal relationship to the study treatment. An AE was considered treatment-emergent if its start date and time was on or after the date and time of first on-study dose of study drug.

Secondary

Measure	Time frame	Description
Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Baseline, Weeks 24, 48, 72, 96, and 120	Data was reported for number of participants with clinical PNH symptom of Dyspnea. The severity of clinical PNH symptom of Dyspnea was graded as none, mild, moderate and severe based solely on investigator's discretion.
Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Baseline, Weeks 24, 48, 72, 96, and 120	Data was reported for number of participants with clinical PNH symptom of Chest pain/discomfort. The severity of clinical PNH symptom of chest pain/discomfort was graded as none, mild, moderate and severe based solely on investigator's discretion.
Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Baseline, Weeks 24, 48, 72, 96, and 120	Data was reported for number of participants with clinical PNH symptom of difficulty swallowing. The severity of clinical PNH symptom of difficulty swallowing was graded as none, mild, moderate and severe based solely on investigator's discretion.
Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Baseline, Weeks 24, 48, 72, 96, and 120	Data was reported for number of participants with clinical PNH symptom of abdominal pain. The severity of clinical PNH symptom of abdominal pain was graded as none, mild, moderate and severe based solely on investigator's discretion.
Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Baseline, Weeks 24, 48, 72, 96, and 120	Data was reported for number of participants with clinical PNH symptom of headache. The severity of clinical PNH symptom of headache was graded as none, mild, moderate and severe based solely on investigator's discretion.
Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Baseline, Weeks 24, 48, 72, 96, and 120	Data was reported for number of participants with clinical PNH symptom of erectile dysfunction. The severity of clinical PNH symptom of erectile dysfunction was graded as none, mild, moderate and severe based solely on investigator's discretion.
Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Baseline, Weeks 24, 48, 72, 96, and 120	Data was reported for number of participants with clinical PNH symptom of Hemoglobinuria.The severity of clinical PNH symptom of hemoglobinuria was graded as none, mild, moderate and severe based solely on investigator's discretion.
Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Baseline, Weeks 24, 48, 72, 96, and 120	Data was reported for number of participants with clinical PNH symptom of fatigue. The severity of clinical PNH symptom of fatigue was graded as none, mild, moderate and severe based solely on investigator's discretion.
Change From Baseline in Lactate Dehydrogenase (LDH)	Baseline, Weeks, 24, 48, 72, 96, 120, and 144	—
Change From Baseline in Hemoglobin	Baseline, Weeks 24, 48, 72, 96, 120, and 144	—
Change From Baseline in Haptoglobin	Baseline, Weeks 24, 48, 72, 96, and 120	—
Change From Baseline in Reticulocytes	Baseline, Weeks 24, 48, 72, 96, and 120	—
Number of Participants With Blood Transfusions or Thromboses	From first dose of study drug up to 3 weeks after last dose (Week 147)	Data was reported for number of participants for whom blood transfusion was required or who experienced the thrombosis events.
Number of Blood Transfusions	From first dose of study drug up to 3 weeks after last dose (Week 147)	Number of blood transfusions were reported.
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Total Score	Baseline, Weeks 24, 48, 72, and 96	The FACIT-Fatigue scale questionnaire was used to determine the level of fatigue experienced by participants. This questionnaire was a 13-item measure that assessed self-reported fatigue and its impact upon daily activities and function. Item scores ranged from 0 (not at all) to 4 (very much), and the total score ranged from 0 to 52, with higher scores indicating greater quality of life.
Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Baseline, Weeks 24, 48, 72, 96, and 120	Data was reported for number of participants with clinical PNH symptom of jaundice. The severity of clinical PNH symptom of jaundice was graded as none, mild, moderate and severe based solely on investigator's discretion.

Countries

Austria, South Africa, United Kingdom

Participant flow

Recruitment details

Participants who had participated in previous BCX9930 trials (BCX9930-101 \[NCT04330534\], BCX9930-202 \[NCT05116774\], or BCX9930-203 \[NCT05116787\]) for Paroxysmal Nocturnal Hemoglobinuria (PNH) and showed a benefit of treatment as determined by the investigator were enrolled in this long-term safety trial.

Participants by arm

Arm	Count
C5-INH Naïve Group This group included participants who, prior to enrolling in their previous BCX9930 study, were either naïve to eculizumab or ravulizumab treatment, or naive to treatment with any complement inhibitor therapy (or had received no treatment in the prior 12 months), and with anemia due to ongoing intravascular hemolysis. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.	12
C5-INH Inadequate Response Group This group included participants who, prior to enrolling in their previous BCX9930 study, were receiving stable treatment with eculizumab or ravulizumab and had an inadequate response to that therapy (ie, residual anemia and/or ongoing need for transfusion). Depending on the prior study, participants may have continued the C5 inhibitor, with BCX9930 provided as an add-on therapy. Participants were to commence treatment at the same BCX9930 dose level and regimen last administered to that individual in the prior study. Per last protocol amendment, all participants received BCX9930 400 mg orally BID. Treatment duration was up to 144 weeks.	7
Total	19

Withdrawals & dropouts

Period	Reason	FG000	FG001
Overall Study	Bone marrow transplant	1	0
Overall Study	Pregnancy	1	0
Overall Study	Transitioned to the BCX9930-205 roll-over study	10	4
Overall Study	Withdrawal by Subject	0	2
Overall Study	Withdrawn due to Investigator decision	0	1

Baseline characteristics

Characteristic	C5-INH Inadequate Response Group	Total	C5-INH Naïve Group
Age, Continuous	44.6 years STANDARD_DEVIATION 18.59	34.9 years STANDARD_DEVIATION 14.5	29.3 years STANDARD_DEVIATION 7.82
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants	0 Participants	0 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	7 Participants	19 Participants	12 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) Asian	1 Participants	2 Participants	1 Participants
Race (NIH/OMB) Black or African American	2 Participants	9 Participants	7 Participants
Race (NIH/OMB) More than one race	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants	0 Participants	0 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants	1 Participants	1 Participants
Race (NIH/OMB) White	4 Participants	7 Participants	3 Participants
Sex: Female, Male Female	5 Participants	8 Participants	3 Participants
Sex: Female, Male Male	2 Participants	11 Participants	9 Participants

Adverse events

Event type	EG000 affected / at risk	EG001 affected / at risk
deaths Total, all-cause mortality	0 / 12	0 / 7
other Total, other adverse events	12 / 12	7 / 7
serious Total, serious adverse events	6 / 12	3 / 7

Outcome results

Primary

Number of Participants With Treatment Emergent Adverse Events (TEAEs)

An AE was defined as any untoward medical occurrence associated with the use of an intervention in humans, whether or not considered intervention-related in a participant or clinical investigation participant who administered a pharmaceutical product regardless of its causal relationship to the study treatment. An AE was considered treatment-emergent if its start date and time was on or after the date and time of first on-study dose of study drug.

Time frame: From first dose of study drug up to 3 weeks after last dose (Week 147)

Population: The safety population included all participants who received at least 1 capsule or tablet of study drug.

Arm	Measure	Value (COUNT_OF_PARTICIPANTS)
C5-INH Naïve Group	Number of Participants With Treatment Emergent Adverse Events (TEAEs)	12 Participants
C5-INH Inadequate Response Group	Number of Participants With Treatment Emergent Adverse Events (TEAEs)	7 Participants

Secondary

Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Total Score

The FACIT-Fatigue scale questionnaire was used to determine the level of fatigue experienced by participants. This questionnaire was a 13-item measure that assessed self-reported fatigue and its impact upon daily activities and function. Item scores ranged from 0 (not at all) to 4 (very much), and the total score ranged from 0 to 52, with higher scores indicating greater quality of life.

Time frame: Baseline, Weeks 24, 48, 72, and 96

Population: Participants in the mITT population with available data were analyzed.

Arm	Measure	Group	Value (MEAN)	Dispersion
C5-INH Naïve Group	Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Total Score	Change at Week 24	0.09 Scores on a scale	Standard Deviation 4.11
C5-INH Naïve Group	Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Total Score	Change at Week 72	-0.38 Scores on a scale	Standard Deviation 2.875
C5-INH Naïve Group	Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Total Score	Change at Week 48	-0.63 Scores on a scale	Standard Deviation 2.387
C5-INH Naïve Group	Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Total Score	Change at Week 96	-1.13 Scores on a scale	Standard Deviation 4.357
C5-INH Naïve Group	Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Total Score	Baseline	43.82 Scores on a scale	Standard Deviation 9.4
C5-INH Inadequate Response Group	Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Total Score	Change at Week 96	-18.00 Scores on a scale	Standard Deviation 27.185
C5-INH Inadequate Response Group	Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Total Score	Baseline	40.14 Scores on a scale	Standard Deviation 13.533
C5-INH Inadequate Response Group	Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Total Score	Change at Week 24	-3.36 Scores on a scale	Standard Deviation 8.066
C5-INH Inadequate Response Group	Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Total Score	Change at Week 48	2.33 Scores on a scale	Standard Deviation 3.215
C5-INH Inadequate Response Group	Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Total Score	Change at Week 72	-4.64 Scores on a scale	Standard Deviation 12.016

Secondary

Change From Baseline in Haptoglobin

Time frame: Baseline, Weeks 24, 48, 72, 96, and 120

Population: Participants in the mITT population with available data were analyzed.

Arm	Measure	Group	Value (MEAN)	Dispersion
C5-INH Naïve Group	Change From Baseline in Haptoglobin	Baseline	0.325 grams per liter (g/L)	Standard Deviation 0.2832
C5-INH Naïve Group	Change From Baseline in Haptoglobin	Change at Week 24	0.000 grams per liter (g/L)	Standard Deviation 0.0447
C5-INH Naïve Group	Change From Baseline in Haptoglobin	Change at Week 48	0.213 grams per liter (g/L)	Standard Deviation 0.1126
C5-INH Naïve Group	Change From Baseline in Haptoglobin	Change at Week 72	0.025 grams per liter (g/L)	Standard Deviation 0.1165
C5-INH Naïve Group	Change From Baseline in Haptoglobin	Change at Week 96	-0.013 grams per liter (g/L)	Standard Deviation 0.0354
C5-INH Naïve Group	Change From Baseline in Haptoglobin	Change at Week 120	-0.013 grams per liter (g/L)	Standard Deviation 0.0354
C5-INH Inadequate Response Group	Change From Baseline in Haptoglobin	Baseline	0.216 grams per liter (g/L)	Standard Deviation 0.135
C5-INH Inadequate Response Group	Change From Baseline in Haptoglobin	Change at Week 72	-0.210 grams per liter (g/L)	Standard Deviation 0.1414
C5-INH Inadequate Response Group	Change From Baseline in Haptoglobin	Change at Week 24	-0.082 grams per liter (g/L)	Standard Deviation 0.0844
C5-INH Inadequate Response Group	Change From Baseline in Haptoglobin	Change at Week 120	-0.100 grams per liter (g/L)	—
C5-INH Inadequate Response Group	Change From Baseline in Haptoglobin	Change at Week 96	0.195 grams per liter (g/L)	Standard Deviation 0.4313

Secondary

Change From Baseline in Hemoglobin

Time frame: Baseline, Weeks 24, 48, 72, 96, 120, and 144

Population: Participants in the mITT population with available data were analyzed.

Arm	Measure	Group	Value (MEAN)	Dispersion
C5-INH Naïve Group	Change From Baseline in Hemoglobin	Change at Week 144	-2.70 grams per deciliter (g/dl)	—
C5-INH Naïve Group	Change From Baseline in Hemoglobin	Change at Week 24	0.75 grams per deciliter (g/dl)	Standard Deviation 1.159
C5-INH Naïve Group	Change From Baseline in Hemoglobin	Change at Week 48	-0.61 grams per deciliter (g/dl)	Standard Deviation 1.974
C5-INH Naïve Group	Change From Baseline in Hemoglobin	Change at Week 72	-0.64 grams per deciliter (g/dl)	Standard Deviation 2.327
C5-INH Naïve Group	Change From Baseline in Hemoglobin	Change at Week 96	-0.33 grams per deciliter (g/dl)	Standard Deviation 1.914
C5-INH Naïve Group	Change From Baseline in Hemoglobin	Change at Week 120	0.38 grams per deciliter (g/dl)	Standard Deviation 1.555
C5-INH Naïve Group	Change From Baseline in Hemoglobin	Baseline	11.88 grams per deciliter (g/dl)	Standard Deviation 1.688
C5-INH Inadequate Response Group	Change From Baseline in Hemoglobin	Baseline	11.53 grams per deciliter (g/dl)	Standard Deviation 1.803
C5-INH Inadequate Response Group	Change From Baseline in Hemoglobin	Change at Week 72	-0.93 grams per deciliter (g/dl)	Standard Deviation 1.484
C5-INH Inadequate Response Group	Change From Baseline in Hemoglobin	Change at Week 24	-1.32 grams per deciliter (g/dl)	Standard Deviation 2.578
C5-INH Inadequate Response Group	Change From Baseline in Hemoglobin	Change at Week 120	-0.47 grams per deciliter (g/dl)	Standard Deviation 2.098
C5-INH Inadequate Response Group	Change From Baseline in Hemoglobin	Change at Week 48	-0.95 grams per deciliter (g/dl)	Standard Deviation 2.333
C5-INH Inadequate Response Group	Change From Baseline in Hemoglobin	Change at Week 96	-0.70 grams per deciliter (g/dl)	Standard Deviation 2.687

Secondary

Change From Baseline in Lactate Dehydrogenase (LDH)

Time frame: Baseline, Weeks, 24, 48, 72, 96, 120, and 144

Population: Participants in the mITT population with available data were analyzed.

Arm	Measure	Group	Value (MEAN)	Dispersion
C5-INH Naïve Group	Change From Baseline in Lactate Dehydrogenase (LDH)	Change at Week 144	-59.0 Units per liter (U/L)	—
C5-INH Naïve Group	Change From Baseline in Lactate Dehydrogenase (LDH)	Change at Week 24	-46.7 Units per liter (U/L)	Standard Deviation 154.55
C5-INH Naïve Group	Change From Baseline in Lactate Dehydrogenase (LDH)	Change at Week 48	-62.5 Units per liter (U/L)	Standard Deviation 80.97
C5-INH Naïve Group	Change From Baseline in Lactate Dehydrogenase (LDH)	Change at Week 72	-6.1 Units per liter (U/L)	Standard Deviation 238.41
C5-INH Naïve Group	Change From Baseline in Lactate Dehydrogenase (LDH)	Change at Week 96	237.4 Units per liter (U/L)	Standard Deviation 347.65
C5-INH Naïve Group	Change From Baseline in Lactate Dehydrogenase (LDH)	Change at Week 120	195.4 Units per liter (U/L)	Standard Deviation 355.03
C5-INH Naïve Group	Change From Baseline in Lactate Dehydrogenase (LDH)	Baseline	475.6 Units per liter (U/L)	Standard Deviation 210.98
C5-INH Inadequate Response Group	Change From Baseline in Lactate Dehydrogenase (LDH)	Baseline	248.3 Units per liter (U/L)	Standard Deviation 125.71
C5-INH Inadequate Response Group	Change From Baseline in Lactate Dehydrogenase (LDH)	Change at Week 72	163.5 Units per liter (U/L)	Standard Deviation 118.09
C5-INH Inadequate Response Group	Change From Baseline in Lactate Dehydrogenase (LDH)	Change at Week 24	38.6 Units per liter (U/L)	Standard Deviation 45.51
C5-INH Inadequate Response Group	Change From Baseline in Lactate Dehydrogenase (LDH)	Change at Week 120	31.0 Units per liter (U/L)	Standard Deviation 125.87
C5-INH Inadequate Response Group	Change From Baseline in Lactate Dehydrogenase (LDH)	Change at Week 48	38.5 Units per liter (U/L)	Standard Deviation 65.76
C5-INH Inadequate Response Group	Change From Baseline in Lactate Dehydrogenase (LDH)	Change at Week 96	-4.0 Units per liter (U/L)	Standard Deviation 134.35

Secondary

Change From Baseline in Reticulocytes

Time frame: Baseline, Weeks 24, 48, 72, 96, and 120

Population: Participants in the mITT population with available data were analyzed

Arm	Measure	Group	Value (MEAN)	Dispersion
C5-INH Naïve Group	Change From Baseline in Reticulocytes	Baseline	0.1066 10^6 cells per microliter (μL)	Standard Deviation 0.02363
C5-INH Naïve Group	Change From Baseline in Reticulocytes	Change at Week 24	0.0164 10^6 cells per microliter (μL)	Standard Deviation 0.02339
C5-INH Naïve Group	Change From Baseline in Reticulocytes	Change at Week 48	0.0048 10^6 cells per microliter (μL)	Standard Deviation 0.03518
C5-INH Naïve Group	Change From Baseline in Reticulocytes	Change at Week 72	0.0044 10^6 cells per microliter (μL)	Standard Deviation 0.03643
C5-INH Naïve Group	Change From Baseline in Reticulocytes	Change at Week 96	0.0409 10^6 cells per microliter (μL)	Standard Deviation 0.03805
C5-INH Naïve Group	Change From Baseline in Reticulocytes	Change at Week 120	0.0173 10^6 cells per microliter (μL)	Standard Deviation 0.03303
C5-INH Inadequate Response Group	Change From Baseline in Reticulocytes	Change at Week 96	0.0607 10^6 cells per microliter (μL)	Standard Deviation 0.03585
C5-INH Inadequate Response Group	Change From Baseline in Reticulocytes	Baseline	0.0889 10^6 cells per microliter (μL)	Standard Deviation 0.03939
C5-INH Inadequate Response Group	Change From Baseline in Reticulocytes	Change at Week 72	0.0463 10^6 cells per microliter (μL)	Standard Deviation 0.03008
C5-INH Inadequate Response Group	Change From Baseline in Reticulocytes	Change at Week 24	0.0913 10^6 cells per microliter (μL)	Standard Deviation 0.09144
C5-INH Inadequate Response Group	Change From Baseline in Reticulocytes	Change at Week 120	0.0653 10^6 cells per microliter (μL)	Standard Deviation 0.0238
C5-INH Inadequate Response Group	Change From Baseline in Reticulocytes	Change at Week 48	0.0507 10^6 cells per microliter (μL)	Standard Deviation 0.03161

Secondary

Number of Blood Transfusions

Number of blood transfusions were reported.

Time frame: From first dose of study drug up to 3 weeks after last dose (Week 147)

Population: Participants from mITT population who required blood transfusions.

Arm	Measure	Value (NUMBER)
C5-INH Naïve Group	Number of Blood Transfusions	13 Number of blood transfusions
C5-INH Inadequate Response Group	Number of Blood Transfusions	25 Number of blood transfusions

Secondary

Number of Participants With Blood Transfusions or Thromboses

Data was reported for number of participants for whom blood transfusion was required or who experienced the thrombosis events.

Time frame: From first dose of study drug up to 3 weeks after last dose (Week 147)

Population: Participants in the mITT population with available data were analyzed.

Arm	Measure	Group	Value (COUNT_OF_PARTICIPANTS)
C5-INH Naïve Group	Number of Participants With Blood Transfusions or Thromboses	Blood transfusions	3 Participants
C5-INH Naïve Group	Number of Participants With Blood Transfusions or Thromboses	Thromboses	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Blood Transfusions or Thromboses	Blood transfusions	4 Participants
C5-INH Inadequate Response Group	Number of Participants With Blood Transfusions or Thromboses	Thromboses	0 Participants

Secondary

Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain

Data was reported for number of participants with clinical PNH symptom of abdominal pain. The severity of clinical PNH symptom of abdominal pain was graded as none, mild, moderate and severe based solely on investigator's discretion.

Time frame: Baseline, Weeks 24, 48, 72, 96, and 120

Population: Participants in the mITT population with available data were analyzed.

Arm	Measure	Group	Value (COUNT_OF_PARTICIPANTS)
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 24-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 96-mild	1 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Baseline-moderate	1 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 96-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 48-none	6 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 96-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 48-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 120-none	8 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 24-none	9 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 120-mild	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 72-none	7 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 120-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Baseline-mild	1 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 72-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 24-moderate	1 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 24-mild	2 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 48-mild	1 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 72-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Baseline-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 48-severe	1 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 96-none	7 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 72-mild	1 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 120-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Baseline-none	10 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 120-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Baseline-none	7 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Baseline-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Baseline-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Baseline-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 24-none	6 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 24-moderate	1 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 24-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 48-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 72-mild	1 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 72-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 72-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 96-none	2 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 96-mild	1 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 96-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 96-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 120-none	3 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 120-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 120-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 24-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 48-none	2 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 48-mild	1 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 48-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain	Week 72-none	2 Participants

Secondary

Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort

Data was reported for number of participants with clinical PNH symptom of Chest pain/discomfort. The severity of clinical PNH symptom of chest pain/discomfort was graded as none, mild, moderate and severe based solely on investigator's discretion.

Time frame: Baseline, Weeks 24, 48, 72, 96, and 120

Population: Participants in the mITT population with available data were analyzed.

Arm	Measure	Group	Value (COUNT_OF_PARTICIPANTS)
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 120-mild	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Baseline-mild	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Baseline-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 24-none	12 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 24-mild	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 24-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 24-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 48-none	7 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 48-mild	1 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 48-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 48-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 72-none	7 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 72-mild	1 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 72-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 72-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 96-none	7 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 96-mild	1 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 96-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 96-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 120-none	8 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 120-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 120-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Baseline-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Baseline-none	12 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 120-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Baseline-none	7 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 72-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Baseline-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 96-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Baseline-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 72-moderate	1 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 24-none	7 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Baseline-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 24-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 72-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 24-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 120-none	3 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 24-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 96-none	3 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 48-none	2 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 120-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 48-mild	1 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 96-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 48-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 120-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 48-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 96-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort	Week 72-none	2 Participants

Secondary

Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing

Data was reported for number of participants with clinical PNH symptom of difficulty swallowing. The severity of clinical PNH symptom of difficulty swallowing was graded as none, mild, moderate and severe based solely on investigator's discretion.

Time frame: Baseline, Weeks 24, 48, 72, 96, and 120

Population: Participants in the mITT population with available data were analyzed.

Arm	Measure	Group	Value (COUNT_OF_PARTICIPANTS)
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 96-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Baseline-mild	1 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Baseline-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Baseline-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 24-none	12 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 24-mild	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 24-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 24-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 48-none	6 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 48-mild	2 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 48-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 48-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 72-none	7 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 72-mild	1 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 72-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 72-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 96-none	7 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 96-mild	1 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 120-none	8 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 120-mild	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 120-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 120-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 96-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Baseline-none	11 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 96-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Baseline-none	6 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 72-none	2 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Baseline-mild	1 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 120-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Baseline-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 72-mild	1 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Baseline-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 120-none	3 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 24-none	7 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 72-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 24-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 96-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 24-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 72-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 24-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 120-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 48-none	2 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 96-none	2 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 48-mild	1 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 120-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 48-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 96-mild	1 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing	Week 48-severe	0 Participants

Secondary

Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea

Data was reported for number of participants with clinical PNH symptom of Dyspnea. The severity of clinical PNH symptom of Dyspnea was graded as none, mild, moderate and severe based solely on investigator's discretion.

Time frame: Baseline, Weeks 24, 48, 72, 96, and 120

Population: Participants in the mITT population with available data were analyzed.

Arm	Measure	Group	Value (COUNT_OF_PARTICIPANTS)
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 120-none	8 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Baseline-mild	1 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Baseline-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Baseline-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 24-none	11 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 24-mild	1 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 24-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 24-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 48-none	6 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 48-mild	2 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 48-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 48-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 72-none	7 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 72-mild	1 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 72-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 96-none	7 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 96-mild	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 96-moderate	1 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Baseline-none	11 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 120-mild	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 120-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 120-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 72-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 96-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 120-none	2 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Baseline-none	6 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 72-none	2 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Baseline-mild	1 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 72-mild	1 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Baseline-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 120-mild	1 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Baseline-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 72-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 24-none	7 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 96-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 24-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 96-none	2 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 24-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 120-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 24-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 96-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 48-none	2 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 72-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 48-mild	1 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 96-moderate	1 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 48-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 120-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea	Week 48-severe	0 Participants

Secondary

Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction

Data was reported for number of participants with clinical PNH symptom of erectile dysfunction. The severity of clinical PNH symptom of erectile dysfunction was graded as none, mild, moderate and severe based solely on investigator's discretion.

Time frame: Baseline, Weeks 24, 48, 72, 96, and 120

Population: Participants in the mITT population with available data were analyzed.

Arm	Measure	Group	Value (COUNT_OF_PARTICIPANTS)
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Baseline-none	9 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Baseline-mild	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Baseline-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Baseline-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 24-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 24-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 48-none	7 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 48-mild	1 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 48-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 48-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 72-none	7 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 72-mild	1 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 72-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 72-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 96-none	7 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 96-mild	1 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 96-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 96-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 120-none	7 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 120-mild	1 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 120-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 24-none	9 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 24-mild	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 120-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 72-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Baseline-none	2 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 96-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Baseline-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 72-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Baseline-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 120-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Baseline-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 24-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 72-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 24-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 120-none	1 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 24-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 96-none	2 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 48-none	1 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 120-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 48-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 96-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 48-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 120-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 48-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 96-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 72-none	1 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction	Week 24-none	2 Participants

Secondary

Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue

Data was reported for number of participants with clinical PNH symptom of fatigue. The severity of clinical PNH symptom of fatigue was graded as none, mild, moderate and severe based solely on investigator's discretion.

Time frame: Baseline, Weeks 24, 48, 72, 96, and 120

Population: The mITT (modified intent-to-treat) population included all participants who received at least 1 capsule or tablet of study drug and had post baseline assessment of PNH symptoms and/or laboratory data. Participants with available data at each visit were included.

Arm	Measure	Group	Value (COUNT_OF_PARTICIPANTS)
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 24-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Baseline-mild	3 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Baseline-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Baseline-severe	1 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 24-mild	2 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 24-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Baseline-none	8 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 48-mild	2 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 48-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 72-none	7 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 72-mild	1 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 72-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 96-mild	2 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 96-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 96-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 120-none	8 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 120-mild	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 120-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 120-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 24-none	10 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 48-none	6 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 48-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 72-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 96-none	6 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 48-moderate	1 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Baseline-none	4 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 96-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Baseline-mild	2 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 120-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Baseline-moderate	1 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 96-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 24-none	3 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Baseline-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 24-mild	3 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 96-severe	1 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 24-moderate	1 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 72-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 24-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 120-none	2 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 48-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 72-mild	2 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 48-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 120-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 72-none	1 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 48-none	2 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 72-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 120-moderate	1 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue	Week 96-none	2 Participants

Secondary

Number of Participants With Clinical PNH Symptom Based on Severity: Headache

Data was reported for number of participants with clinical PNH symptom of headache. The severity of clinical PNH symptom of headache was graded as none, mild, moderate and severe based solely on investigator's discretion.

Time frame: Baseline, Weeks 24, 48, 72, 96, and 120

Population: Participants in the mITT population with available data were analyzed.

Arm	Measure	Group	Value (COUNT_OF_PARTICIPANTS)
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 96-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 24-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Baseline-none	10 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 24-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 120-mild	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 48-none	7 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Baseline-mild	2 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 48-mild	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 96-mild	1 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 48-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Baseline-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 72-none	8 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 72-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 120-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 48-moderate	1 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Baseline-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 120-none	8 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 72-mild	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 24-none	11 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 72-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 120-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 96-none	7 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 24-mild	1 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 96-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 48-none	2 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 96-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 96-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 120-none	2 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 120-mild	1 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 120-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 120-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Baseline-none	7 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Baseline-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Baseline-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Baseline-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 24-none	6 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 24-mild	1 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 24-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 24-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 96-none	3 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 48-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 48-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 72-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 48-moderate	1 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 72-none	3 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 72-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 72-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Headache	Week 96-moderate	0 Participants

Secondary

Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria

Data was reported for number of participants with clinical PNH symptom of Hemoglobinuria.The severity of clinical PNH symptom of hemoglobinuria was graded as none, mild, moderate and severe based solely on investigator's discretion.

Time frame: Baseline, Weeks 24, 48, 72, 96, and 120

Population: Participants in the mITT population with available data were analyzed.

Arm	Measure	Group	Value (COUNT_OF_PARTICIPANTS)
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 96-missing severity	2 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Baseline-mild	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Baseline-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Baseline-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Baseline-missing severity	2 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 24-mild	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 24-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 24-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 24-missing severity	1 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 48-none	6 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 48-moderate	1 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 48-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 48-missing severity	1 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 72-none	7 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 72-mild	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 72-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 72-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 72-missing severity	1 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 96-none	6 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 96-mild	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 96-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 96-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Baseline-none	10 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 120-mild	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 120-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 120-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 24-none	11 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 48-mild	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 120-none	7 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 120-missing severity	1 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 72-mild	1 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Baseline-none	7 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 120-missing severity	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Baseline-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 72-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Baseline-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 96-missing severity	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Baseline-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 72-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Baseline-missing severity	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 24-none	5 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 120-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 24-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 72-missing severity	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 24-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 120-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 24-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 96-none	3 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 24-missing severity	2 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 120-none	3 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 48-none	3 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 96-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 48-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 120-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 48-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 96-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 48-missing severity	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 48-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 72-none	2 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria	Week 96-severe	0 Participants

Secondary

Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice

Data was reported for number of participants with clinical PNH symptom of jaundice. The severity of clinical PNH symptom of jaundice was graded as none, mild, moderate and severe based solely on investigator's discretion.

Time frame: Baseline, Weeks 24, 48, 72, 96, and 120

Population: Participants in the population with available data were analyzed.

Arm	Measure	Group	Value (COUNT_OF_PARTICIPANTS)
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Baseline-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 72-none	8 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 24-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 72-mild	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Baseline-missing severity	1 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 72-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 24-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 96-none	8 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Baseline-mild	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 96-mild	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 48-none	8 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 96-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 24-none	12 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 96-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 48-mild	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 120-none	8 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Baseline-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 120-mild	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 48-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 120-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 24-mild	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 120-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 48-severe	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 72-moderate	0 Participants
C5-INH Naïve Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Baseline-none	11 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 72-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Baseline-none	7 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Baseline-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Baseline-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Baseline-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Baseline-missing severity	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 24-none	7 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 24-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 24-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 24-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 48-none	3 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 48-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 48-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 48-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 72-none	3 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 72-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 72-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 96-none	3 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 96-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 96-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 96-severe	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 120-none	3 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 120-mild	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 120-moderate	0 Participants
C5-INH Inadequate Response Group	Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice	Week 120-severe	0 Participants

Source: ClinicalTrials.gov · Data processed: Feb 12, 2026

A Long-Term Safety Study of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Conditions

Keywords

Brief summary

Related papers

Interventions

Sponsors

Study design

Eligibility

Inclusion criteria

Exclusion criteria

Design outcomes

Primary

Secondary

Countries

Participant flow

Recruitment details

Participants by arm

Withdrawals & dropouts

Baseline characteristics

Adverse events

Outcome results

Number of Participants With Treatment Emergent Adverse Events (TEAEs)

Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) - Fatigue Scale Total Score

Change From Baseline in Haptoglobin

Change From Baseline in Hemoglobin

Change From Baseline in Lactate Dehydrogenase (LDH)

Change From Baseline in Reticulocytes

Number of Blood Transfusions

Number of Participants With Blood Transfusions or Thromboses

Number of Participants With Clinical PNH Symptom Based on Severity: Abdominal Pain

Number of Participants With Clinical PNH Symptom Based on Severity: Chest Pain/Discomfort

Number of Participants With Clinical PNH Symptom Based on Severity: Difficulty Swallowing

Number of Participants With Clinical PNH Symptom Based on Severity: Dyspnea

Number of Participants With Clinical PNH Symptom Based on Severity: Erectile Dysfunction

Number of Participants With Clinical PNH Symptom Based on Severity: Fatigue

Number of Participants With Clinical PNH Symptom Based on Severity: Headache

Number of Participants With Clinical PNH Symptom Based on Severity: Hemoglobinuria

Number of Participants With Clinical PNH Symptom Based on Severity: Jaundice