Paroxysmal Nocturnal Hemoglobinuria (PNH)
Conditions
Keywords
BCX9930, factor D inhibitor, proximal complement inhibitor, oral therapy, paroxysmal nocturnal hemoglobinuria
Brief summary
The purpose of this study was to determine the efficacy and safety of BCX9930 monotherapy for the treatment of adult participants with PNH not currently receiving complement inhibitor therapy.
Detailed description
This was a randomized, placebo-controlled, double-blind, parallel-group, 2-part study. Parts 1 and 2 was to be conducted in the same participants. Part 1 of the study was designed to evaluate the efficacy, safety, and tolerability of treatment with oral BCX9930 monotherapy for 12 weeks versus placebo in participants with PNH who were not currently receiving treatment with complement inhibitor therapy. Participants were randomized to receive BCX9930 or placebo under double blind conditions for the 12-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 open-label 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 placebo in Part 1 discontinued that therapy at the Week 12 visit and received BCX9930 in Part 2.
Interventions
DRUGPlacebo
Administered orally twice daily
DRUGBCX9930 monotherapy
Administered orally twice daily
Sponsors
BioCryst Pharmaceuticals
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
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 * No complement inhibitor therapy for ≥ 12 months prior to screening * Contraindication to or no access to approved (C3 or C5) complement inhibitor therapies * Documentation of current vaccinations against Neisseria meningitidis and Streptococcus pneumoniae or willingness to start vaccination series * At screening: PNH clone of ≥ 10%, hemoglobin ≤ 10.5 g/dL and lactate dehydrogenase ≥ 2 × upper limit of normal
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 12 | Baseline, Week 12 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Part 1: Number of Units of pRBCs Transfused | From Week 4 to Week 12 | — |
| Part 1: Percent Change From Baseline in Lactate Dehydrogenase | Baseline, Week 12 | — |
| Part 1: Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score | Baseline, Week 12 | 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: Percentage (%) Reduction in the Rate of pRBC Units Transfused | Prestudy (12 months prior to screening) and from Week 4 to Week 12 | The rate of pRBC units transfused from Week 4 to Week 12 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 g/dL | At Week 12 | — |
| Part 1: Number of Participants Achieving Hemoglobin Stabilization | From Week 4 to Week 12 | 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 12. |
| Part 1: Change From Baseline in Total Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Clone Size | Baseline, Week 12 | 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: Number of Participants Who Were Transfusion-free | From Week 4 to Week 12 | 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 12, 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: Change From Baseline in Absolute Reticulocyte Count (ARC) | Baseline, Week 12 | — |
| Part 1: Number of Participants With ARC in the Normal Range | Week 12 | Number of participants with ARC in the normal range (50 - 100 x 10\^9 cells/L) were reported. |
| Part 1: Change From Baseline in Haptoglobin | Baseline, Week 12 | — |
| Part 1: Number of Participants With Haptoglobin ≥ Lower Limit of Normal (LLN) Reference Range | Week 12 | Number of participants with haptoglobin ≥ LLN Reference Range (≥0.3 g/L) were reported. |
| Part 1: Change From Baseline in Total Bilirubin | Baseline, Week 12 | — |
| Part 1: Change From Baseline in Aspartate Aminotransferase (AST) | Baseline, Week 12 | — |
| Part 1: Change From Baseline in Ratio of Total PNH RBC Clone Size to PNH White Blood Cell (WBC) Clone Size | Baseline, Week 12 | The total PNH RBC clone size refers to the percentage of PNH-affected (i.e, 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. |
Countries
Malaysia, South Africa, South Korea
Participant flow
Recruitment details
The study was conducted in Malaysia, South Africa, and Korea.
Pre-assignment details
A total 12 participants were randomized and treated.
Participants by arm
| Arm | Count |
|---|---|
| BCX9930/BCX9930 Participants received BCX9930 monotherapy in double blind manner (Part 1) for 12 weeks, then in an open-label manner for the remainder of the study (Part 2). After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants on blinded study treatment were switched to open-label BCX9930 prior to Week 12. Initially participants were to receive BCX9930 500 mg BID orally. 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 in Part 1 was 12 weeks. The overall maximum duration on BCX9930 was 378 days. | 10 |
| Placebo/BCX9930 Participants received BCX9930 matching placebo in double blind manner for 12 weeks (Part 1). After the sponsor decided to halt enrolment in the study permanently and terminate the study, participants switched to open-label BCX9930 monotherapy (Part 2) prior to Week 12, if earlier. The maximum treatment duration in Part 1 was 12 weeks. The maximum duration on Placebo in Part 1 was 12 weeks. The maximum treatment duration on BCX9930 was 281 days. | 2 |
| Total | 12 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Part 1:DB Phase (up to 12 Weeks) | Adverse Event | 1 | 0 |
| Part 1:DB Phase (up to 12 Weeks) | Pregnancy | 1 | 0 |
| Part 1:DB Phase (up to 12 Weeks) | Withdrawal by Subject | 0 | 1 |
| Part 2:Open-label Phase (up to 52 Weeks) | Miscellaneous | 4 | 0 |
Baseline characteristics
| Characteristic | Placebo/BCX9930 | Total | BCX9930/BCX9930 |
|---|---|---|---|
| Age, Continuous | 37.0 Years STANDARD_DEVIATION 9.9 | 37.7 Years STANDARD_DEVIATION 11.87 | 37.8 Years STANDARD_DEVIATION 12.7 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants | 0 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 2 Participants | 12 Participants | 10 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 | 2 Participants | 5 Participants | 3 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 7 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 | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 0 Participants | 0 Participants | 0 Participants |
| Sex: Female, Male Female | 2 Participants | 8 Participants | 6 Participants |
| Sex: Female, Male Male | 0 Participants | 4 Participants | 4 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 10 | 0 / 2 | 0 / 1 |
| other Total, other adverse events | 10 / 10 | 2 / 2 | 1 / 1 |
| serious Total, serious adverse events | 5 / 10 | 1 / 2 | 1 / 1 |
Outcome results
Primary
Part 1: Change From Baseline in Hemoglobin at Week 12
Time frame: Baseline, Week 12
Population: Participants in the All Subjects as Treated (ASaT) population (all participants who received at least 1 dose of study drug and had a post baseline laboratory assessment) in Part 1 were analyzed.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| BCX9930 | Part 1: Change From Baseline in Hemoglobin at Week 12 | Baseline | 8.49 grams per deciliter (g/dL) | Standard Deviation 1.556 |
| BCX9930 | Part 1: Change From Baseline in Hemoglobin at Week 12 | Change at Week 12 | 2.23 grams per deciliter (g/dL) | Standard Deviation 2.146 |
| Placebo | Part 1: Change From Baseline in Hemoglobin at Week 12 | Baseline | 9.48 grams per deciliter (g/dL) | Standard Deviation 1.721 |
| Placebo | Part 1: Change From Baseline in Hemoglobin at Week 12 | Change at Week 12 | -1.23 grams per deciliter (g/dL) | Standard Deviation 0.33 |
Secondary
Part 1: Change From Baseline in Absolute Reticulocyte Count (ARC)
Time frame: Baseline, Week 12
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 | 200.01 10^9 cells/microliter (μL) | Standard Deviation 81.094 |
| BCX9930 | Part 1: Change From Baseline in Absolute Reticulocyte Count (ARC) | Change at Week 12 | -111.01 10^9 cells/microliter (μL) | Standard Deviation 80.799 |
| Placebo | Part 1: Change From Baseline in Absolute Reticulocyte Count (ARC) | Baseline | 261.47 10^9 cells/microliter (μL) | Standard Deviation 13.529 |
| Placebo | Part 1: Change From Baseline in Absolute Reticulocyte Count (ARC) | Change at Week 12 | -21.43 10^9 cells/microliter (μL) | — |
Secondary
Part 1: Change From Baseline in Aspartate Aminotransferase (AST)
Time frame: Baseline, Week 12
Population: Participants in the ASaT population in Part 1 were analyzed.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| BCX9930 | Part 1: Change From Baseline in Aspartate Aminotransferase (AST) | Baseline | 107.43 Units per liter (U/L) | Standard Deviation 71.295 |
| BCX9930 | Part 1: Change From Baseline in Aspartate Aminotransferase (AST) | Change at Week 12 | -68.83 Units per liter (U/L) | Standard Deviation 58.333 |
| Placebo | Part 1: Change From Baseline in Aspartate Aminotransferase (AST) | Baseline | 83.60 Units per liter (U/L) | Standard Deviation 6.505 |
| Placebo | Part 1: Change From Baseline in Aspartate Aminotransferase (AST) | Change at Week 12 | 13.00 Units per liter (U/L) | Standard Deviation 36.487 |
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 12
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 | 34.3 Scores on a scale | Standard Deviation 13.47 |
| BCX9930 | Part 1: Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score | Change at Week 12 | -2.3 Scores on a scale | Standard Deviation 13.48 |
| Placebo | Part 1: Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score | Baseline | 13.0 Scores on a scale | Standard Deviation 8.49 |
| Placebo | Part 1: Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score | Change at Week 12 | -2.0 Scores on a scale | Standard Deviation 0 |
Secondary
Part 1: Change From Baseline in Haptoglobin
Time frame: Baseline, Week 12
Population: Participants in the ASaT population in Part 1 were analyzed.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| BCX9930 | Part 1: Change From Baseline in Haptoglobin | Baseline | 0.182 grams per liter (g/L) | Standard Deviation 0.0711 |
| BCX9930 | Part 1: Change From Baseline in Haptoglobin | Change at Week 12 | 0.493 grams per liter (g/L) | Standard Deviation 0.6948 |
| Placebo | Part 1: Change From Baseline in Haptoglobin | Baseline | 0.195 grams per liter (g/L) | Standard Deviation 0.1485 |
| Placebo | Part 1: Change From Baseline in Haptoglobin | Change at Week 12 | 0.000 grams per liter (g/L) | Standard Deviation 0 |
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 (i.e, 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 12
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 | 0.6425 ratio | Standard Deviation 0.28016 |
| 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 12 | 0.2905 ratio | Standard Deviation 0.26628 |
| Placebo | Part 1: Change From Baseline in Ratio of Total PNH RBC Clone Size to PNH White Blood Cell (WBC) Clone Size | Baseline | 0.6860 ratio | — |
| Placebo | Part 1: Change From Baseline in Ratio of Total PNH RBC Clone Size to PNH White Blood Cell (WBC) Clone Size | Change at Week 12 | -0.3052 ratio | — |
Secondary
Part 1: Change From Baseline in Total Bilirubin
Time frame: Baseline, Week 12
Population: Participants in the ASaT population in Part 1 were analyzed.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| BCX9930 | Part 1: Change From Baseline in Total Bilirubin | Change at Week 12 | -0.97 milligram per deciliter (mg/dL) | Standard Deviation 1.706 |
| BCX9930 | Part 1: Change From Baseline in Total Bilirubin | Baseline | 1.66 milligram per deciliter (mg/dL) | Standard Deviation 1.944 |
| Placebo | Part 1: Change From Baseline in Total Bilirubin | Change at Week 12 | 0.80 milligram per deciliter (mg/dL) | Standard Deviation 0.283 |
| Placebo | Part 1: Change From Baseline in Total Bilirubin | Baseline | 3.45 milligram per deciliter (mg/dL) | Standard Deviation 2.475 |
Secondary
Part 1: Change From Baseline in Total Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (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 12
Population: Participants in the ASaT population Part 1 were analyzed.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| BCX9930 | Part 1: Change From Baseline in Total Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Clone Size | Baseline | 50.16 % of PNH-RBC within total RBC population | Standard Deviation 30.386 |
| BCX9930 | Part 1: Change From Baseline in Total Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Clone Size | Change at Week 12 | 30.76 % of PNH-RBC within total RBC population | Standard Deviation 25.925 |
| Placebo | Part 1: Change From Baseline in Total Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Clone Size | Baseline | 33.72 % of PNH-RBC within total RBC population | Standard Deviation 3.765 |
| Placebo | Part 1: Change From Baseline in Total Paroxysmal Nocturnal Hemoglobinuria (PNH) Red Blood Cell (RBC) Clone Size | Change at Week 12 | -2.15 % of PNH-RBC within total RBC population | Standard Deviation 0.032 |
Secondary
Part 1: Number of Participants Achieving 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 12.
Time frame: From Week 4 to Week 12
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 Achieving Hemoglobin Stabilization | 8 Participants |
| Placebo | Part 1: Number of Participants Achieving Hemoglobin Stabilization | 2 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 12, 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 12
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 | 8 Participants |
| Placebo | Part 1: Number of Participants Who Were Transfusion-free | 1 Participants |
Secondary
Part 1: Number of Participants With ARC in the Normal Range
Number of participants with ARC in the normal range (50 - 100 x 10\^9 cells/L) were reported.
Time frame: Week 12
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 | 5 Participants |
| Placebo | Part 1: Number of Participants With ARC in the Normal Range | 0 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 12
Population: Participants in the ASaT population in Part 1 were analyzed.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| BCX9930 | Part 1: Number of Participants With Haptoglobin ≥ Lower Limit of Normal (LLN) Reference Range | 5 Participants |
| Placebo | Part 1: Number of Participants With Haptoglobin ≥ Lower Limit of Normal (LLN) Reference Range | 1 Participants |
Secondary
Part 1: Number of Participants With Hemoglobin ≥ 12 g/dL
Time frame: At Week 12
Population: Participants in the ASaT population in Part 1 were analyzed.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| BCX9930 | Part 1: Number of Participants With Hemoglobin ≥ 12 g/dL | 2 Participants |
| Placebo | Part 1: Number of Participants With Hemoglobin ≥ 12 g/dL | 0 Participants |
Secondary
Part 1: Number of Units of pRBCs Transfused
Time frame: From Week 4 to Week 12
Population: Participants in the ASaT population in Part 1 were analyzed.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| BCX9930 | Part 1: Number of Units of pRBCs Transfused | 1 units of pRBCs |
| Placebo | 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 12 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 12
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 | 89 percent reduction |
| Placebo | Part 1: Percentage (%) Reduction in the Rate of pRBC Units Transfused | 100 percent reduction |
Secondary
Part 1: Percent Change From Baseline in Lactate Dehydrogenase
Time frame: Baseline, Week 12
Population: Participants in the ASaT population in Part 1 were analyzed.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| BCX9930 | Part 1: Percent Change From Baseline in Lactate Dehydrogenase | -69.8 percent change | Standard Deviation 26 |
| Placebo | Part 1: Percent Change From Baseline in Lactate Dehydrogenase | 9.1 percent change | Standard Deviation 45.47 |