Paroxysmal Nocturnal Hemoglobinuria
Conditions
Keywords
PNH, Paroxysmal, Hemoglobinuria, ACH-0144471, ALXN2040, Danicopan
Brief summary
The purpose of this study is to evaluate the long-term safety and efficacy of ACH-0144471 in participants with paroxysmal nocturnal hemoglobinuria (PNH) who have demonstrated clinical benefit from ACH-0144471 in Study ACH471-100. This study is designed to include up to 12 participants.
Interventions
DRUGACH-0144471
ACH-0144471 will be administered to all participants enrolled in the study.
Sponsors
Achillion, a wholly owned subsidiary of Alexion
Alexion Pharmaceuticals, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Study designed to include up to 12 participants who completed treatment in Study ACH471-100 and demonstrated clinical benefit from ACH-0144471 with no significant safety or tolerability concerns. * Negative pregnancy test for females prior to dosing and throughout the study.
Exclusion criteria
* Have developed any clinically relevant co-morbidities while participating in Study ACH471-100 that would make the participant inappropriate for the continuation of treatment with ACH-0144471, in the opinion of the Investigator. * Have developed any clinically significant laboratory abnormalities while participating in Study ACH471-100 that, in the opinion of the Investigator, would make the participant inappropriate for the study or put the participant at undue risk. * Females who are pregnant, nursing, or planning to become pregnant during the study or within 90 days of study drug administration or participants with a female partner who is pregnant, nursing, or planning to become pregnant during the study or within 90 days of study drug administration.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Grade 3 and Grade 4 Adverse Events (AEs), And AEs Leading To Discontinuation | Baseline up to 4.5 years | An AE was as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), important medical event or reaction. The intensity of an AE was graded according to the Common Terminology Criteria for Adverse Events (CTCAE) Adverse Event Severity Grading Table. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. |
| Change From Baseline in LDH Level at Week 25 | Baseline, Week 25 | Change from Baseline = Serum LDH levels at Week 25 - Baseline Serum LDH levels. Baseline was the baseline value from the primary Study ACH471-100. |
| Change From Baseline in Hgb Level in the Absence of RBC Transfusion at Week 25 | Baseline, Week 25 | Change from Baseline = Hgb levels at Week 25 - Baseline Hgb levels. Baseline was the baseline value from the primary Study ACH471-100. |
| Change From Baseline in Reticulocyte Counts at Week 25 | Baseline, Week 25 | Change from Baseline = reticulocyte count at Week 25 - Baseline reticulocyte count. Baseline was the baseline value from the primary Study ACH471-100. |
| Number of RBC Units Transfused | Baseline up to Week 169 | — |
| Number of RBC Transfusion Instances | Baseline up to Week 169 | — |
| Change From Baseline in PNH Clone Size at Week 25 | Baseline, Week 25 | The PNH clone size refers to the percentage of PNH-affected cells versus normal cells within the total cell population. Change from Baseline = PNH clone size at Week 25 - Baseline PNH clone size. Baseline was the baseline value from the primary Study ACH471-100. |
| Change From Baseline in AP Complement Functional Activity at Week 25 | Baseline, Week 25 | Serum AP functional activity was measured by the Wieslab functional immunoassay method. Change from Baseline = Serum AP functional activity at Week 25 - Baseline Serum AP functional activity. Baseline was the baseline value from the primary Study ACH471-100. |
| Change From Baseline in Free Hgb at Week 25 | Baseline, Week 25 | Change from Baseline = free Hgb at Week 25 - Baseline free Hgb. Baseline was the baseline value from the primary Study ACH471-100. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Hgb Level in the Absence of RBC Transfusion at Weeks 49 and 169 | Baseline, Weeks 49 and 169 | Change from Baseline = Hgb levels at specified postbaseline visit - Baseline Hgb levels. Baseline was the baseline value from the primary Study ACH471-100. |
| Change From Baseline in Reticulocyte Counts at Weeks 49 and 169 | Baseline, Weeks 49 and 169 | Change from Baseline = reticulocyte count at specified postbaseline visit - Baseline reticulocyte count. Baseline was the baseline value from the primary Study ACH471-100. |
| Change From Baseline in PNH Clone Size at Weeks 49 and 73 | Baseline, Weeks 49 and 73 | The PNH clone size refers to the percentage of PNH-affected cells versus normal cells within the total cell population. Change from Baseline = PNH clone size at specified postbaseline visit - Baseline PNH clone size. Baseline was the baseline value from the primary Study ACH471-100. |
| Change From Baseline in AP Complement Functional Activity at Weeks 49 and 145 | Baseline, Weeks 49 and 145 | Serum AP functional activity was measured by the Wieslab functional immunoassay method. Change from Baseline = Serum AP functional activity at specified postbaseline visit - Baseline Serum AP functional activity. Baseline was the baseline value from the primary Study ACH471-100. |
| Change From Baseline in Free Hgb at Weeks 49 and 169 | Baseline, Weeks 49 and 169 | Change from Baseline = free Hgb at specified postbaseline visit - Baseline free Hgb. Baseline was the baseline value from the primary Study ACH471-100. |
| Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score at Weeks 21, 41, and 153 | Baseline, Weeks 21, 41, and 153 | The FACIT-Fatigue scale is a collection of quality of life questionnaires pertaining to the management of fatigue symptoms due to a chronic illness. The FACIT-Fatigue is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function over the preceding 7 days. Participants score each item on a 5-point scale: 0 (Not at all) to 4 (Very much). Total scores range from 0 to 52, with higher score indicating better quality of life. |
| Change From Baseline in European Organisation for Research and Treatment of Cancer, Quality of Life Questionnaire-Core 30 Scale (EORTC-QLQ-C30): Global Health Status/Qol Score at Weeks 21, 41, and 153 | Baseline, Weeks 21, 41, and 153 | EORTC-QLQ-C30 is comprised of 30 questions. First 28 questions used 4-point scale (1=not at all,2=a little,3=quite a bit,4=very much) for evaluating 5 functional scales (physical, role, emotional, cognitive, social), 3 symptom scales (fatigue, nausea/vomiting, pain) & other single items. For each item, high score = high level of symptomatology/problem. Last 2 questions represented participant's assessment of overall health (global health status) and quality of life, coded on 7-point scale (1=very poor to 7=excellent). Answers were converted into grading scale, with total score between 0 and 100. A high score represented a favourable outcome with a best quality of life for participant. |
| Change From Baseline in LDH Level at Weeks 49 and 169 | Baseline, Weeks 49 and 169 | Change from Baseline = Serum LDH levels at specified postbaseline visit - Baseline Serum LDH levels. Baseline was the baseline value from the primary Study ACH471-100. |
Countries
Italy, New Zealand, South Korea
Participant flow
Pre-assignment details
Participants who demonstrated a clinical benefit from danicopan in the primary Study ACH471-100 (NCT03053102), were eligible for long-term treatment with danicopan in this extension study ACH471-103.
Participants by arm
| Arm | Count |
|---|---|
| Danicopan Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \[mg\] 3 times daily \[TID\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision. | 8 |
| Total | 8 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Missing follow-up visit | 1 |
| Overall Study | Withdrawal by Subject | 1 |
Baseline characteristics
| Characteristic | Danicopan |
|---|---|
| Age, Continuous | 37.51 years STANDARD_DEVIATION 14.752 |
| Alternative Pathway (AP) Complement Functional Activity | 66.70 percentage of activity STANDARD_DEVIATION 12.732 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 8 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Free Hgb | 36.08 mg/deciliter (dL) STANDARD_DEVIATION 42.417 |
| Hemoglobin (Hgb) Level in the Absence of Red Blood Cell (RBC) Transfusion | 96.50 grams (g)/liter (L) STANDARD_DEVIATION 18.055 |
| Lactate Dehydrogenase (LDH) Level | 1403.13 units (U)/liter (L) STANDARD_DEVIATION 602.824 |
| Paroxysmal Nocturnal Hemoglobinuria (PNH) Clone Size | 39.67 percentage of the total cell population STANDARD_DEVIATION 29.696 |
| Race/Ethnicity, Customized Race Asian | 1 Participants |
| Race/Ethnicity, Customized Race Other | 1 Participants |
| Race/Ethnicity, Customized Race White | 6 Participants |
| Reticulocyte Counts | 0.15 10^12 cells/L STANDARD_DEVIATION 0.078 |
| Sex: Female, Male Female | 4 Participants |
| Sex: Female, Male Male | 4 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 8 |
| other Total, other adverse events | 8 / 8 |
| serious Total, serious adverse events | 3 / 8 |
Outcome results
Primary
Change From Baseline in AP Complement Functional Activity at Week 25
Serum AP functional activity was measured by the Wieslab functional immunoassay method. Change from Baseline = Serum AP functional activity at Week 25 - Baseline Serum AP functional activity. Baseline was the baseline value from the primary Study ACH471-100.
Time frame: Baseline, Week 25
Population: Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for Serum AP functional activity.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Danicopan | Change From Baseline in AP Complement Functional Activity at Week 25 | -46.36 percentage of activity | Standard Deviation 25.316 |
Primary
Change From Baseline in Free Hgb at Week 25
Change from Baseline = free Hgb at Week 25 - Baseline free Hgb. Baseline was the baseline value from the primary Study ACH471-100.
Time frame: Baseline, Week 25
Population: Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for free Hgb.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Danicopan | Change From Baseline in Free Hgb at Week 25 | 59.83 mg/dL | Standard Deviation 66.414 |
Primary
Change From Baseline in Hgb Level in the Absence of RBC Transfusion at Week 25
Change from Baseline = Hgb levels at Week 25 - Baseline Hgb levels. Baseline was the baseline value from the primary Study ACH471-100.
Time frame: Baseline, Week 25
Population: Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for Hgb level.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Danicopan | Change From Baseline in Hgb Level in the Absence of RBC Transfusion at Week 25 | 24.67 g/L | Standard Deviation 18.052 |
Primary
Change From Baseline in LDH Level at Week 25
Change from Baseline = Serum LDH levels at Week 25 - Baseline Serum LDH levels. Baseline was the baseline value from the primary Study ACH471-100.
Time frame: Baseline, Week 25
Population: Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the post baseline assessments for LDH level.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Danicopan | Change From Baseline in LDH Level at Week 25 | -683.29 U/L | Standard Deviation 845.175 |
Primary
Change From Baseline in PNH Clone Size at Week 25
The PNH clone size refers to the percentage of PNH-affected cells versus normal cells within the total cell population. Change from Baseline = PNH clone size at Week 25 - Baseline PNH clone size. Baseline was the baseline value from the primary Study ACH471-100.
Time frame: Baseline, Week 25
Population: Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for PNH clone size.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Danicopan | Change From Baseline in PNH Clone Size at Week 25 | 22.00 percentage of the total cell population | Standard Deviation 5.831 |
Primary
Change From Baseline in Reticulocyte Counts at Week 25
Change from Baseline = reticulocyte count at Week 25 - Baseline reticulocyte count. Baseline was the baseline value from the primary Study ACH471-100.
Time frame: Baseline, Week 25
Population: Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for reticulocyte count.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Danicopan | Change From Baseline in Reticulocyte Counts at Week 25 | -0.07 10^12 cells/L | Standard Deviation 0.063 |
Primary
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Grade 3 and Grade 4 Adverse Events (AEs), And AEs Leading To Discontinuation
An AE was as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), important medical event or reaction. The intensity of an AE was graded according to the Common Terminology Criteria for Adverse Events (CTCAE) Adverse Event Severity Grading Table. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
Time frame: Baseline up to 4.5 years
Population: Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Danicopan | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Grade 3 and Grade 4 Adverse Events (AEs), And AEs Leading To Discontinuation | Any TEAEs | 8 participants |
| Danicopan | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Grade 3 and Grade 4 Adverse Events (AEs), And AEs Leading To Discontinuation | SAEs | 3 participants |
| Danicopan | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Grade 3 and Grade 4 Adverse Events (AEs), And AEs Leading To Discontinuation | TEAE Grade 3 | 2 participants |
| Danicopan | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Grade 3 and Grade 4 Adverse Events (AEs), And AEs Leading To Discontinuation | TEAE Grade 4 | 0 participants |
| Danicopan | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Grade 3 and Grade 4 Adverse Events (AEs), And AEs Leading To Discontinuation | AE leading to discontinuation | 0 participants |
Primary
Number of RBC Transfusion Instances
Time frame: Baseline up to Week 169
Population: Full analysis set included all enrolled and treated participants.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Danicopan | Number of RBC Transfusion Instances | 3.4 RBC transfusion instances | Standard Deviation 8 |
Primary
Number of RBC Units Transfused
Time frame: Baseline up to Week 169
Population: Full analysis set included all enrolled and treated participants.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Danicopan | Number of RBC Units Transfused | 6.5 RBC units | Standard Deviation 16.83 |
Secondary
Change From Baseline in AP Complement Functional Activity at Weeks 49 and 145
Serum AP functional activity was measured by the Wieslab functional immunoassay method. Change from Baseline = Serum AP functional activity at specified postbaseline visit - Baseline Serum AP functional activity. Baseline was the baseline value from the primary Study ACH471-100.
Time frame: Baseline, Weeks 49 and 145
Population: Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for Serum AP functional activity. 'Number analyzed' = participants evaluable at specified timepoint.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Danicopan | Change From Baseline in AP Complement Functional Activity at Weeks 49 and 145 | Change at Week 49 | -59.80 percentage of activity | Standard Deviation 14.217 |
| Danicopan | Change From Baseline in AP Complement Functional Activity at Weeks 49 and 145 | Change at Week 145 | -52.43 percentage of activity | Standard Deviation 2.779 |
Secondary
Change From Baseline in European Organisation for Research and Treatment of Cancer, Quality of Life Questionnaire-Core 30 Scale (EORTC-QLQ-C30): Global Health Status/Qol Score at Weeks 21, 41, and 153
EORTC-QLQ-C30 is comprised of 30 questions. First 28 questions used 4-point scale (1=not at all,2=a little,3=quite a bit,4=very much) for evaluating 5 functional scales (physical, role, emotional, cognitive, social), 3 symptom scales (fatigue, nausea/vomiting, pain) & other single items. For each item, high score = high level of symptomatology/problem. Last 2 questions represented participant's assessment of overall health (global health status) and quality of life, coded on 7-point scale (1=very poor to 7=excellent). Answers were converted into grading scale, with total score between 0 and 100. A high score represented a favourable outcome with a best quality of life for participant.
Time frame: Baseline, Weeks 21, 41, and 153
Population: Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for EORTC-QLQ-C30 (Global Health Status/Qol) score. 'Number analyzed' = participants evaluable at specified timepoint.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Danicopan | Change From Baseline in European Organisation for Research and Treatment of Cancer, Quality of Life Questionnaire-Core 30 Scale (EORTC-QLQ-C30): Global Health Status/Qol Score at Weeks 21, 41, and 153 | Change at Week 41 | 11.90 units on a scale | Standard Deviation 29.603 |
| Danicopan | Change From Baseline in European Organisation for Research and Treatment of Cancer, Quality of Life Questionnaire-Core 30 Scale (EORTC-QLQ-C30): Global Health Status/Qol Score at Weeks 21, 41, and 153 | Change at Week 21 | 16.67 units on a scale | Standard Deviation 25.973 |
| Danicopan | Change From Baseline in European Organisation for Research and Treatment of Cancer, Quality of Life Questionnaire-Core 30 Scale (EORTC-QLQ-C30): Global Health Status/Qol Score at Weeks 21, 41, and 153 | Change at Week 153 | 4.17 units on a scale | Standard Deviation 17.678 |
Secondary
Change From Baseline in Free Hgb at Weeks 49 and 169
Change from Baseline = free Hgb at specified postbaseline visit - Baseline free Hgb. Baseline was the baseline value from the primary Study ACH471-100.
Time frame: Baseline, Weeks 49 and 169
Population: Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for free Hgb. 'Number analyzed' = participants evaluable at specified timepoint.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Danicopan | Change From Baseline in Free Hgb at Weeks 49 and 169 | Change at Week 169 | -30.75 mg/dL | Standard Deviation 54.942 |
| Danicopan | Change From Baseline in Free Hgb at Weeks 49 and 169 | Change at Week 49 | 105.92 mg/dL | Standard Deviation 92.308 |
Secondary
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score at Weeks 21, 41, and 153
The FACIT-Fatigue scale is a collection of quality of life questionnaires pertaining to the management of fatigue symptoms due to a chronic illness. The FACIT-Fatigue is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function over the preceding 7 days. Participants score each item on a 5-point scale: 0 (Not at all) to 4 (Very much). Total scores range from 0 to 52, with higher score indicating better quality of life.
Time frame: Baseline, Weeks 21, 41, and 153
Population: Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for FACIT-Fatigue scale score. 'Number analyzed' = participants evaluable at specified timepoint.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Danicopan | Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score at Weeks 21, 41, and 153 | Change at Week 21 | 8.4 units on a scale | Standard Deviation 11.75 |
| Danicopan | Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score at Weeks 21, 41, and 153 | Change at Week 41 | 9.1 units on a scale | Standard Deviation 13.37 |
| Danicopan | Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score at Weeks 21, 41, and 153 | Change at Week 153 | 3.0 units on a scale | Standard Deviation 0 |
Secondary
Change From Baseline in Hgb Level in the Absence of RBC Transfusion at Weeks 49 and 169
Change from Baseline = Hgb levels at specified postbaseline visit - Baseline Hgb levels. Baseline was the baseline value from the primary Study ACH471-100.
Time frame: Baseline, Weeks 49 and 169
Population: Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for Hgb level. 'Number analyzed' = participants evaluable at specified timepoint.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Danicopan | Change From Baseline in Hgb Level in the Absence of RBC Transfusion at Weeks 49 and 169 | Change at Week 169 | 54.50 g/L | Standard Deviation 14.849 |
| Danicopan | Change From Baseline in Hgb Level in the Absence of RBC Transfusion at Weeks 49 and 169 | Change at Week 49 | 21.40 g/L | Standard Deviation 18.995 |
Secondary
Change From Baseline in LDH Level at Weeks 49 and 169
Change from Baseline = Serum LDH levels at specified postbaseline visit - Baseline Serum LDH levels. Baseline was the baseline value from the primary Study ACH471-100.
Time frame: Baseline, Weeks 49 and 169
Population: Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for LDH level. 'Number analyzed' = participants evaluable at specified timepoint.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Danicopan | Change From Baseline in LDH Level at Weeks 49 and 169 | Change at Week 49 | -862.50 U/L | Standard Deviation 568.302 |
| Danicopan | Change From Baseline in LDH Level at Weeks 49 and 169 | Change at Week 169 | -1388.00 U/L | Standard Deviation 562.857 |
Secondary
Change From Baseline in PNH Clone Size at Weeks 49 and 73
The PNH clone size refers to the percentage of PNH-affected cells versus normal cells within the total cell population. Change from Baseline = PNH clone size at specified postbaseline visit - Baseline PNH clone size. Baseline was the baseline value from the primary Study ACH471-100.
Time frame: Baseline, Weeks 49 and 73
Population: Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for PNH clone size. 'Number analyzed' = participants evaluable at specified timepoint.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Danicopan | Change From Baseline in PNH Clone Size at Weeks 49 and 73 | Change at Week 49 | 30.83 percentage of the total cell population | Standard Deviation 11.531 |
| Danicopan | Change From Baseline in PNH Clone Size at Weeks 49 and 73 | Change at Week 73 | 32.00 percentage of the total cell population | Standard Deviation 19.17 |
Secondary
Change From Baseline in Reticulocyte Counts at Weeks 49 and 169
Change from Baseline = reticulocyte count at specified postbaseline visit - Baseline reticulocyte count. Baseline was the baseline value from the primary Study ACH471-100.
Time frame: Baseline, Weeks 49 and 169
Population: Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for reticulocyte count. 'Number analyzed' = participants evaluable at specified timepoint.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Danicopan | Change From Baseline in Reticulocyte Counts at Weeks 49 and 169 | Change at Week 49 | -0.05 10^12 cells/L | Standard Deviation 0.065 |
| Danicopan | Change From Baseline in Reticulocyte Counts at Weeks 49 and 169 | Change at Week 169 | -0.12 10^12 cells/L | Standard Deviation 0.049 |