Paroxysmal Nocturnal Hemoglobinuria (PNH)
Conditions
Keywords
BCX9930, factor D inhibitor, proximal complement inhibitor, oral therapy, paroxysmal nocturnal hemoglobinuria, inadequate response to C5 inhibitor, C5 inhibitor, eculizumab, ravulizumab
Brief summary
The purpose of this study was to determine the efficacy and safety of BCX9930 monotherapy for the treatment of PNH compared to continued C5 inhibitor therapy in adult PNH participants with residual anemia despite treatment with a C5 inhibitor.
Detailed description
This was a randomized, active comparator-controlled, open-label, parallel-group, 2-part study. Parts 1 and 2 were conducted in the same participants. Part 1 of the study was designed to evaluate the efficacy, safety, and tolerability of oral BCX9930 monotherapy for 24 weeks versus continuing C5 inhibitor therapy in participants with PNH with inadequate response to their current C5 inhibitor therapy. Participants were randomized to either discontinue C5 inhibitor therapy and start BCX9930 monotherapy or to continue C5 inhibitor therapy for the 24-week randomized treatment period. The primary efficacy and safety analyses were based on Part 1. Part 2 of the study was designed to evaluate the long-term safety, tolerability, and effectiveness of BCX9930 monotherapy when administered through Week 52. All participants in Part 2 received BCX9930. Participants who were randomized to BCX9930 monotherapy in Part 1 continued to receive BCX9930 in Part 2. Participants who were randomized to C5 inhibitor therapy in Part 1 discontinued that therapy at the Week 24 visit and received BCX9930 in Part 2.
Interventions
DRUGEculizumab
Administered by intravenous infusion per current dose regimen
DRUGRavulizumab
Administered by intravenous infusion per current dose regimen
DRUGBCX9930
Administered orally twice daily
Sponsors
BioCryst Pharmaceuticals
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Male or female, aged ≥ 18 years old * Body weight ≥ 40 kg * Documented diagnosis of PNH * Currently being treated with a stable C5 inhibitor regimen * Documentation of current vaccinations against Neisseria meningitidis and Streptococcus pneumoniae or willing to start vaccination series * At screening: PNH clone size of ≥ 10% and hemoglobin ≤ 10.5 g/dL
Exclusion criteria
* Known history of or existing diagnosis of hereditary complement deficiency * History of hematopoietic cell transplant or solid organ transplant or anticipated candidate for transplantation * Myocardial infarction or cerebrovascular accident within 30 days prior to screening, or current and uncontrolled clinically significant cardiovascular or cerebrovascular condition * History of malignancy within 5 years prior to the screening visit * Active bacterial, viral, or fungal infection or any other serious infection within 14 days prior to screening * Treatment with anti-thymocyte globulin within 180 days prior to screening * Initiation of treatment with an erythrocyte or platelet growth factor, or danazol within 28 days prior to screening * Receiving iron supplementation with an unstable dose in the 28 days prior to screening
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Part 1: Change From Baseline in Hemoglobin at Week 24 | Baseline, Week 24 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Part 1: Number of Participants Who Were Transfusion-free | From Week 4 to Week 24 | The number of participants who did not receive any transfusions (packed red blood cells \[pRBCs\] or whole blood) during the period of interest were reported. Participants who were transfusion free were defined for each treatment group as the number of participants who did not receive any transfusions (pRBCs or whole blood) during the period of interest from the start to the end, inclusive, divided by the total number of participants in that treatment group at the start of the period of interest. Participants who (1) discontinued treatment prior to Week 24, or (2) did not receive a transfusion during the period of interest despite recording a hemoglobin (Hb) value ≤ 9 g/dL with symptoms assessed by the investigator as warranting transfusion or a Hb value ≤ 7 g/dL regardless of symptoms were not considered transfusion free. |
| Part 1: Number of Units of pRBCs Transfused | From Week 4 to Week 24 | — |
| Part 1: Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score | Baseline, Week 24 | 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. |
| Part 1: Percent Change From Baseline in Lactate Dehydrogenase | Baseline, Week 24 | — |
| Part 1: Percentage (%) Reduction in the Rate of pRBC Units Transfused | Prestudy (12 months prior to screening) and from Week 4 to Week 24 | The rate of pRBC units transfused from Week 4 to Week 24 was calculated and compared to the rate of pRBC units transfused prestudy during the 12 months prior to screening. The percent reduction in rate of pRBC units transfused was the percent difference in rate relative to the prestudy rate, calculated as: (current rate - prestudy rate)/prestudy rate \* 100%. Total rate among all participants was evaluated here. Rate of pRBC units transfusion was defined as the percentage of participants who received pRBC transfusions. |
| Part 1: Number of Participants With Hemoglobin ≥ 12 Grams Per Deciliter (g/dL) | Week 24 | — |
| Part 1: Number of Participants Who Achieved Hemoglobin Stabilization | From Week 4 to Week 24 | Hemoglobin stabilization was defined as the participants who avoided 2 g/dL or greater decrease in hemoglobin in the absence of transfusion from Week 4 to Week 24. |
| Part 1: Change From Baseline in Complement Component 3 (C3)-Opsonized Red Blood Cells (RBCs) | Baseline, Week 24 | Red blood cells opsonized by C3 were to be assessed by flow cytometry . |
| Part 1: Change From Baseline in Total Paroxysmal Nocturnal Hemoglobinuria (PNH) RBC Clone Size | Baseline, Week 24 | The total PNH RBC clone size refers to the percentage of PNH affected (ie, Type 2 and 3) RBC cells within the total RBC population. |
| Part 1: Change From Baseline in Ratio of Total PNH RBC Clone Size to PNH White Blood Cell (WBC) Clone Size | Baseline, Week 24 | The total PNH RBC clone size refers to the percentage of PNH affected (ie, Type 2 and 3) RBCs within the total RBC population. The PNH WBC clone size refers to the percentage of PNH-affected WBCs within the total WBC population. The ratio of total PNH RBC clone size to PNH WBC clone size = ratio of percent total PNH RBCs / percent PNH WBCs. |
| Part 1: Change From Baseline in Absolute Reticulocyte Count (ARC) | Baseline, Week 24 | — |
| Part 1: Number of Participants With ARC in the Normal Range | Week 24 | Number of participants with ARC in the normal range (0.03 - 0.12 10\^6 cells/uL) were reported. |
| Part 1: Change From Baseline in Haptoglobin | Baseline, Week 24 | — |
| Part 1: Number of Participants With Haptoglobin ≥ Lower Limit of Normal (LLN) Reference Range | Week 24 | Number of Participants With Haptoglobin ≥ LLN Reference Range (≥0.3 g/L) were reported. |
| Part 1: Change From Baseline in Total Bilirubin | Baseline, Week 24 | — |
Countries
France, Hungary, Italy, Spain, United Kingdom
Participant flow
Recruitment details
A total 12 participants were randomized and treated.
Pre-assignment details
The study was conducted in France, Hungary, Italy, Spain and United Kingdom.
Participants by arm
| Arm | Count |
|---|---|
| BCX9930/BCX9930 Participants were randomized to receive BCX9930 during Part 1 and continued to receive the same during Part 2. Per protocol amendment, participants who previously received 500 mg BID and remained on study treatment were dose adjusted to 400 mg BID. For all newly enrolled participants, at the initiation of BCX9930 dosing, participants were administered a dose of 200 mg BID for the first 14 days of treatment before increasing to 400 mg BID. The maximum treatment duration on BCX9930 in Part 1 was 24 weeks. The overall maximum treatment duration on BCX9930 was 377 days. | 8 |
| C5-INH/BCX9930 Participants randomized to this group continued the existing C5-INH therapy during Part 1. After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants entered Part 2 where they switched to open-label BCX9930 monotherapy prior to Week 24, if earlier. The maximum treatment duration on C5-INH in Part 1 was 24 weeks. The maximum treatment duration on BCX9930 was 197 days. | 4 |
| Total | 12 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Part 1 (up to 24 Weeks) | Adverse Event | 1 | 0 |
| Part 1 (up to 24 Weeks) | Withdrawal by Subject | 0 | 1 |
| Part 2 (up to 52 Weeks) | Adverse Event | 1 | 0 |
| Part 2 (up to 52 Weeks) | Miscellaneous | 3 | 1 |
Baseline characteristics
| Characteristic | BCX9930/BCX9930 | C5-INH/BCX9930 | Total |
|---|---|---|---|
| Age, Continuous | 58.8 years STANDARD_DEVIATION 17.66 | 48.5 years STANDARD_DEVIATION 14.25 | 55.3 years STANDARD_DEVIATION 16.71 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants | 0 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 5 Participants | 2 Participants | 7 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 3 Participants | 2 Participants | 5 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 1 Participants | 1 Participants |
| Race (NIH/OMB) Black or African American | 2 Participants | 0 Participants | 2 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 | 1 Participants | 1 Participants | 2 Participants |
| Race (NIH/OMB) White | 5 Participants | 2 Participants | 7 Participants |
| Sex: Female, Male Female | 4 Participants | 3 Participants | 7 Participants |
| Sex: Female, Male Male | 4 Participants | 1 Participants | 5 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 8 | 0 / 4 | 0 / 3 |
| other Total, other adverse events | 7 / 8 | 3 / 4 | 3 / 3 |
| serious Total, serious adverse events | 2 / 8 | 0 / 4 | 1 / 3 |
Outcome results
Primary
Part 1: Change From Baseline in Hemoglobin at Week 24
Time frame: Baseline, Week 24
Population: Participants in the all subjects as treated (ASaT) population in Part 1 with available data were analyzed.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| BCX9930 | Part 1: Change From Baseline in Hemoglobin at Week 24 | Baseline | 9.12 grams per deciliter (g/dL) | Standard Deviation 1.096 |
| BCX9930 | Part 1: Change From Baseline in Hemoglobin at Week 24 | Change at Week 24 | 3.48 grams per deciliter (g/dL) | Standard Deviation 0.674 |
| C5-INH | Part 1: Change From Baseline in Hemoglobin at Week 24 | Baseline | 9.13 grams per deciliter (g/dL) | Standard Deviation 0.847 |
| C5-INH | Part 1: Change From Baseline in Hemoglobin at Week 24 | Change at Week 24 | 0.72 grams per deciliter (g/dL) | Standard Deviation 0.884 |
Secondary
Part 1: Change From Baseline in Absolute Reticulocyte Count (ARC)
Time frame: Baseline, Week 24
Population: Participants in the ASaT population in Part 1 with available data were analyzed.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| BCX9930 | Part 1: Change From Baseline in Absolute Reticulocyte Count (ARC) | Baseline | 0.2933 10^6 cells/microliter (μL) | Standard Deviation 0.10453 |
| BCX9930 | Part 1: Change From Baseline in Absolute Reticulocyte Count (ARC) | Change at Week 24 | -0.2142 10^6 cells/microliter (μL) | Standard Deviation 0.05384 |
| C5-INH | Part 1: Change From Baseline in Absolute Reticulocyte Count (ARC) | Baseline | 0.3129 10^6 cells/microliter (μL) | Standard Deviation 0.16201 |
| C5-INH | Part 1: Change From Baseline in Absolute Reticulocyte Count (ARC) | Change at Week 24 | -0.0462 10^6 cells/microliter (μL) | Standard Deviation 0.01823 |
Secondary
Part 1: Change From Baseline in Complement Component 3 (C3)-Opsonized Red Blood Cells (RBCs)
Red blood cells opsonized by C3 were to be assessed by flow cytometry .
Time frame: Baseline, Week 24
Population: No data was collected for this endpoint.
Secondary
Part 1: Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale 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, Week 24
Population: Participants in the ASaT population in Part 1 with available data were analyzed.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| BCX9930 | Part 1: Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score | Baseline | 36.3 Scores on a scale | Standard Deviation 9.69 |
| BCX9930 | Part 1: Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score | Change at Week 24 | -0.3 Scores on a scale | Standard Deviation 12.86 |
| C5-INH | Part 1: Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score | Baseline | 40.0 Scores on a scale | Standard Deviation 8.49 |
| C5-INH | Part 1: Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score | Change at Week 24 | 1.0 Scores on a scale | — |
Secondary
Part 1: Change From Baseline in Haptoglobin
Time frame: Baseline, Week 24
Population: Participants in the ASaT population in Part 1 with available data were analyzed.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| BCX9930 | Part 1: Change From Baseline in Haptoglobin | Baseline | 0.126 grams per liter (g/L) | Standard Deviation 0.0732 |
| BCX9930 | Part 1: Change From Baseline in Haptoglobin | Change at Week 24 | -0.024 grams per liter (g/L) | Standard Deviation 0.0409 |
| C5-INH | Part 1: Change From Baseline in Haptoglobin | Baseline | 0.105 grams per liter (g/L) | Standard Deviation 0.0268 |
| C5-INH | Part 1: Change From Baseline in Haptoglobin | Change at Week 24 | -0.015 grams per liter (g/L) | Standard Deviation 0.0268 |
Secondary
Part 1: Change From Baseline in Ratio of Total PNH RBC Clone Size to PNH White Blood Cell (WBC) Clone Size
The total PNH RBC clone size refers to the percentage of PNH affected (ie, Type 2 and 3) RBCs within the total RBC population. The PNH WBC clone size refers to the percentage of PNH-affected WBCs within the total WBC population. The ratio of total PNH RBC clone size to PNH WBC clone size = ratio of percent total PNH RBCs / percent PNH WBCs.
Time frame: Baseline, Week 24
Population: Participants in the ASaT population in Part 1 with available data were analyzed.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| BCX9930 | Part 1: Change From Baseline in Ratio of Total PNH RBC Clone Size to PNH White Blood Cell (WBC) Clone Size | Baseline | 0.8128 ratio | Standard Deviation 0.16528 |
| BCX9930 | Part 1: Change From Baseline in Ratio of Total PNH RBC Clone Size to PNH White Blood Cell (WBC) Clone Size | Change at Week 24 | 0.2336 ratio | Standard Deviation 0.11595 |
| C5-INH | Part 1: Change From Baseline in Ratio of Total PNH RBC Clone Size to PNH White Blood Cell (WBC) Clone Size | Change at Week 24 | -0.0668 ratio | Standard Deviation 0.09462 |
| C5-INH | Part 1: Change From Baseline in Ratio of Total PNH RBC Clone Size to PNH White Blood Cell (WBC) Clone Size | Baseline | 0.9059 ratio | Standard Deviation 0.12113 |
Secondary
Part 1: Change From Baseline in Total Bilirubin
Time frame: Baseline, Week 24
Population: Participants in the ASaT population in Part 1 with available data were analyzed.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| BCX9930 | Part 1: Change From Baseline in Total Bilirubin | Baseline | 3.44 milligram per deciliter (mg/dL) | Standard Deviation 3.679 |
| BCX9930 | Part 1: Change From Baseline in Total Bilirubin | Change at Week 24 | -2.66 milligram per deciliter (mg/dL) | Standard Deviation 3.672 |
| C5-INH | Part 1: Change From Baseline in Total Bilirubin | Baseline | 1.10 milligram per deciliter (mg/dL) | Standard Deviation 0.424 |
| C5-INH | Part 1: Change From Baseline in Total Bilirubin | Change at Week 24 | -0.02 milligram per deciliter (mg/dL) | Standard Deviation 0.25 |
Secondary
Part 1: Change From Baseline in Total Paroxysmal Nocturnal Hemoglobinuria (PNH) RBC Clone Size
The total PNH RBC clone size refers to the percentage of PNH affected (ie, Type 2 and 3) RBC cells within the total RBC population.
Time frame: Baseline, Week 24
Population: Participants in the ASaT population in Part 1 with available data were analyzed.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| BCX9930 | Part 1: Change From Baseline in Total Paroxysmal Nocturnal Hemoglobinuria (PNH) RBC Clone Size | Baseline | 80.51 % of PNH-RBC within total RBC population | Standard Deviation 16.94 |
| BCX9930 | Part 1: Change From Baseline in Total Paroxysmal Nocturnal Hemoglobinuria (PNH) RBC Clone Size | Change at Week 24 | 23.18 % of PNH-RBC within total RBC population | Standard Deviation 11.81 |
| C5-INH | Part 1: Change From Baseline in Total Paroxysmal Nocturnal Hemoglobinuria (PNH) RBC Clone Size | Baseline | 83.40 % of PNH-RBC within total RBC population | Standard Deviation 14.34 |
| C5-INH | Part 1: Change From Baseline in Total Paroxysmal Nocturnal Hemoglobinuria (PNH) RBC Clone Size | Change at Week 24 | -6.34 % of PNH-RBC within total RBC population | Standard Deviation 8.986 |
Secondary
Part 1: Number of Participants Who Achieved Hemoglobin Stabilization
Hemoglobin stabilization was defined as the participants who avoided 2 g/dL or greater decrease in hemoglobin in the absence of transfusion from Week 4 to Week 24.
Time frame: From Week 4 to Week 24
Population: Participants in the ASaT population in Part 1 with available data were analyzed.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| BCX9930 | Part 1: Number of Participants Who Achieved Hemoglobin Stabilization | 5 Participants |
| C5-INH | Part 1: Number of Participants Who Achieved Hemoglobin Stabilization | 4 Participants |
Secondary
Part 1: Number of Participants Who Were Transfusion-free
The number of participants who did not receive any transfusions (packed red blood cells \[pRBCs\] or whole blood) during the period of interest were reported. Participants who were transfusion free were defined for each treatment group as the number of participants who did not receive any transfusions (pRBCs or whole blood) during the period of interest from the start to the end, inclusive, divided by the total number of participants in that treatment group at the start of the period of interest. Participants who (1) discontinued treatment prior to Week 24, or (2) did not receive a transfusion during the period of interest despite recording a hemoglobin (Hb) value ≤ 9 g/dL with symptoms assessed by the investigator as warranting transfusion or a Hb value ≤ 7 g/dL regardless of symptoms were not considered transfusion free.
Time frame: From Week 4 to Week 24
Population: Participants in the ASaT population in Part 1 were analyzed.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| BCX9930 | Part 1: Number of Participants Who Were Transfusion-free | 7 Participants |
| C5-INH | Part 1: Number of Participants Who Were Transfusion-free | 4 Participants |
Secondary
Part 1: Number of Participants With ARC in the Normal Range
Number of participants with ARC in the normal range (0.03 - 0.12 10\^6 cells/uL) were reported.
Time frame: Week 24
Population: Participants in the ASaT population in Part 1 with available data were analyzed.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| BCX9930 | Part 1: Number of Participants With ARC in the Normal Range | 4 Participants |
| C5-INH | Part 1: Number of Participants With ARC in the Normal Range | 1 Participants |
Secondary
Part 1: Number of Participants With Haptoglobin ≥ Lower Limit of Normal (LLN) Reference Range
Number of Participants With Haptoglobin ≥ LLN Reference Range (≥0.3 g/L) were reported.
Time frame: Week 24
Population: Participants in the ASaT population in Part 1 with available data were analyzed.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| BCX9930 | Part 1: Number of Participants With Haptoglobin ≥ Lower Limit of Normal (LLN) Reference Range | 0 Participants |
| C5-INH | Part 1: Number of Participants With Haptoglobin ≥ Lower Limit of Normal (LLN) Reference Range | 0 Participants |
Secondary
Part 1: Number of Participants With Hemoglobin ≥ 12 Grams Per Deciliter (g/dL)
Time frame: Week 24
Population: Participants in the ASaT population in Part 1 with available data were analyzed.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| BCX9930 | Part 1: Number of Participants With Hemoglobin ≥ 12 Grams Per Deciliter (g/dL) | 6 Participants |
| C5-INH | Part 1: Number of Participants With Hemoglobin ≥ 12 Grams Per Deciliter (g/dL) | 0 Participants |
Secondary
Part 1: Number of Units of pRBCs Transfused
Time frame: From Week 4 to Week 24
Population: Participants in the ASaT population in Part 1 were analyzed.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| BCX9930 | Part 1: Number of Units of pRBCs Transfused | 0 units of pRBCs |
| C5-INH | Part 1: Number of Units of pRBCs Transfused | 0 units of pRBCs |
Secondary
Part 1: Percentage (%) Reduction in the Rate of pRBC Units Transfused
The rate of pRBC units transfused from Week 4 to Week 24 was calculated and compared to the rate of pRBC units transfused prestudy during the 12 months prior to screening. The percent reduction in rate of pRBC units transfused was the percent difference in rate relative to the prestudy rate, calculated as: (current rate - prestudy rate)/prestudy rate \* 100%. Total rate among all participants was evaluated here. Rate of pRBC units transfusion was defined as the percentage of participants who received pRBC transfusions.
Time frame: Prestudy (12 months prior to screening) and from Week 4 to Week 24
Population: Participants in the ASaT population in Part 1 were analyzed.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| BCX9930 | Part 1: Percentage (%) Reduction in the Rate of pRBC Units Transfused | 100 percent reduction |
| C5-INH | Part 1: Percentage (%) Reduction in the Rate of pRBC Units Transfused | NA percent reduction |
Secondary
Part 1: Percent Change From Baseline in Lactate Dehydrogenase
Time frame: Baseline, Week 24
Population: Participants in the ASaT population in Part 1 with available data were analyzed.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| BCX9930 | Part 1: Percent Change From Baseline in Lactate Dehydrogenase | 24.3 percent change | Standard Deviation 53.07 |
| C5-INH | Part 1: Percent Change From Baseline in Lactate Dehydrogenase | -21.0 percent change | Standard Deviation 4.03 |