A Phase 2 interventional study of Placebo and BCX9930 monotherapy in Paroxysmal Nocturnal Hemoglobinuria (PNH), sponsored by BioCryst Pharmaceuticals. Terminated at 5 sites in 3 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-10-29.
Sponsored by BioCryst Pharmaceuticals · Phase 2, Interventional, and Treatment
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.
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.
191 studies on the registry are indexed under Hemoglobinuria, Paroxysmal; 49 are open to participants now.
This study's enrollment of 12 is below the median of 33 across 150 interventional studies indexed under Hemoglobinuria, Paroxysmal.
Browse Hemoglobinuria, Paroxysmal studies →BioCryst Pharmaceuticals is the lead sponsor of 54 studies on the registry; 1 is open to participants now.
Of its 10 completed or terminated interventional studies of FDA-regulated products, 8 (80%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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 twice daily (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.
Drug: BCX9930 monotherapy
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 duration on Placebo in Part 1 was 12 weeks. The maximum treatment duration on BCX9930 was 281 days.
Drug: Placebo · Drug: BCX9930 monotherapy
Administered orally twice daily
Administered orally twice daily
Part 1: Change From Baseline in Hemoglobin at Week 12
Time frame: Baseline, Week 12
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
Part 1: Number of Units of pRBCs Transfused
Time frame: From Week 4 to Week 12
Part 1: Percent Change From Baseline in Lactate Dehydrogenase
Time frame: Baseline, Week 12
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
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
Part 1: Number of Participants With Hemoglobin ≥ 12 g/dL
Time frame: At Week 12
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
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
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
Part 1: Change From Baseline in Absolute Reticulocyte Count (ARC)
Time frame: Baseline, Week 12
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
Part 1: Change From Baseline in Haptoglobin
Time frame: Baseline, Week 12
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
Part 1: Change From Baseline in Total Bilirubin
Time frame: Baseline, Week 12
Part 1: Change From Baseline in Aspartate Aminotransferase (AST)
Time frame: Baseline, Week 12
The study was conducted in Malaysia, South Africa, and Korea.
| Milestone | BCX9930/BCX9930 | Placebo/BCX9930 |
|---|---|---|
| Started | 10 | 2 |
| Completed | 8 | 1 |
| Not completed | 2 | 1 |
| Withdrew: Withdrawal by subject | 0 | 1 |
| Withdrew: Pregnancy | 1 | 0 |
| Withdrew: Adverse event | 1 | 0 |
| Milestone | BCX9930/BCX9930 | Placebo/BCX9930 |
|---|---|---|
| Started | 8 | 1 |
| Completed | 4 | 1 |
| Not completed | 4 | 0 |
| Withdrew: Miscellaneous | 4 | 0 |
| grams per deciliter (g/dL) | BCX9930 | Placebo |
|---|---|---|
| Baseline | 8.49 ± 1.556 | 9.48 ± 1.721 |
| Change at Week 12 | 2.23 ± 2.146 | -1.23 ± 0.330 |
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.
| Participants | BCX9930 | Placebo |
|---|---|---|
| Part 1: Number of Participants Who Were Transfusion-free | 8 | 1 |
| units of pRBCs | BCX9930 | Placebo |
|---|---|---|
| Part 1: Number of Units of pRBCs Transfused | 1 | 0 |
| percent change | BCX9930 | Placebo |
|---|---|---|
| Part 1: Percent Change From Baseline in Lactate Dehydrogenase | -69.8 ± 26.00 | 9.1 ± 45.47 |
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.
| Scores on a scale | BCX9930 | Placebo |
|---|---|---|
| Baseline | 34.3 ± 13.47 | 13.0 ± 8.49 |
| Change at Week 12 | -2.3 ± 13.48 | -2.0 ± 0.00 |
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.
| percent reduction | BCX9930 | Placebo |
|---|---|---|
| Part 1: Percentage (%) Reduction in the Rate of pRBC Units Transfused | 89 | 100 |
| Participants | BCX9930 | Placebo |
|---|---|---|
| Part 1: Number of Participants With Hemoglobin ≥ 12 g/dL | 2 | 0 |
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.
| Participants | BCX9930 | Placebo |
|---|---|---|
| Part 1: Number of Participants Achieving Hemoglobin Stabilization | 8 | 2 |
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.
| % of PNH-RBC within total RBC population | BCX9930 | Placebo |
|---|---|---|
| Baseline | 50.16 ± 30.386 | 33.72 ± 3.765 |
| Change at Week 12 | 30.76 ± 25.925 | -2.15 ± 0.032 |
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.
| ratio | BCX9930 | Placebo |
|---|---|---|
| Baseline | 0.6425 ± 0.28016 | 0.6860 |
| Change at Week 12 | 0.2905 ± 0.26628 | -0.3052 |
| 10^9 cells/microliter (μL) | BCX9930 | Placebo |
|---|---|---|
| Baseline | 200.01 ± 81.094 | 261.47 ± 13.529 |
| Change at Week 12 | -111.01 ± 80.799 | -21.43 |
Number of participants with ARC in the normal range (50 - 100 x 10\^9 cells/L) were reported.
| Participants | BCX9930 | Placebo |
|---|---|---|
| Part 1: Number of Participants With ARC in the Normal Range | 5 | 0 |
| grams per liter (g/L) | BCX9930 | Placebo |
|---|---|---|
| Baseline | 0.182 ± 0.0711 | 0.195 ± 0.1485 |
| Change at Week 12 | 0.493 ± 0.6948 | 0.000 ± 0.0000 |
Number of participants with haptoglobin ≥ LLN Reference Range (≥0.3 g/L) were reported.
| Participants | BCX9930 | Placebo |
|---|---|---|
| Part 1: Number of Participants With Haptoglobin ≥ Lower Limit of Normal (LLN) Reference Range | 5 | 1 |
| milligram per deciliter (mg/dL) | BCX9930 | Placebo |
|---|---|---|
| Change at Week 12 | -0.97 ± 1.706 | 0.80 ± 0.283 |
| Baseline | 1.66 ± 1.944 | 3.45 ± 2.475 |
| Units per liter (U/L) | BCX9930 | Placebo |
|---|---|---|
| Baseline | 107.43 ± 71.295 | 83.60 ± 6.505 |
| Change at Week 12 | -68.83 ± 58.333 | 13.00 ± 36.487 |
Collected over From Day 1 up to 408 days. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| BCX9930/BCX9930 | 0/10 (0%) | 5/10 (50%) | 10/10 (100%) |
| Placebo | 0/2 (0%) | 1/2 (50%) | 2/2 (100%) |
| BCX9930 After Placebo | 0/1 (0%) | 1/1 (100%) | 1/1 (100%) |
| Event | BCX9930/BCX9930 | Placebo | BCX9930 After Placebo |
|---|---|---|---|
| Enterovirus infectionInfections and infestations | 0/10 | 0/2 | 1/1 |
| Budd-Chiari syndromeHepatobiliary disorders | 0/10 | 1/2 | 0/1 |
| InfluenzaInfections and infestations | 1/10 | 0/2 | 0/1 |
| Pneumonia fungalInfections and infestations | 1/10 | 0/2 | 0/1 |
| TuberculosisInfections and infestations | 1/10 | 0/2 | 0/1 |
| Urinary tract infectionInfections and infestations | 1/10 | 0/2 | 0/1 |
| Breakthrough haemolysisBlood and lymphatic system disorders | 1/10 | 0/2 | 0/1 |
| Intervertebral disc protrusionMusculoskeletal and connective tissue disorders | 1/10 | 0/2 | 0/1 |
| Renal failureRenal and urinary disorders | 1/10 | 0/2 | 0/1 |
| Event | BCX9930/BCX9930 | Placebo | BCX9930 After Placebo |
|---|---|---|---|
| DiarrhoeaGastrointestinal disorders | 3/10 | 0/2 | 1/1 |
| AlopeciaGastrointestinal disorders | 0/10 | 0/2 | 1/1 |
| HyperhidrosisSkin and subcutaneous tissue disorders | 0/10 | 0/2 | 1/1 |
| Upper respiratory tract infectionInfections and infestations | 4/10 | 0/2 | 1/1 |
| ConjunctivitisInfections and infestations | 0/10 | 0/2 | 1/1 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 3/10 | 0/2 | 1/1 |
| Musculoskeletal stiffnessMusculoskeletal and connective tissue disorders | 0/10 | 0/2 | 1/1 |
| NeutropeniaBlood and lymphatic system disorders | 0/10 | 0/2 | 1/1 |
| HeadacheNervous system disorders | 4/10 | 0/2 | 1/1 |
| Weight increasedInvestigations | 0/10 | 0/2 | 1/1 |
The safety population included all participants who received at least 1 dose of study drug, whether placebo or BCX9930. Therefore, BCX9930 group included participants who received BCX9930 throughout the study and placebo group included data for participants who received placebo initially and then switched to BCX9930.
| Age, Continuous(Years) | BCX9930/BCX9930 | Placebo/BCX9930 | Total |
|---|---|---|---|
| Mean | 37.8 ± 12.70 | 37.0 ± 9.90 | 37.7 ± 11.87 |
| Sex: Female, Male(Participants) | BCX9930/BCX9930 | Placebo/BCX9930 | Total |
|---|---|---|---|
| Female | 6 | 2 | 8 |
| Male | 4 | 0 | 4 |
| Ethnicity (NIH/OMB)(Participants) | BCX9930/BCX9930 | Placebo/BCX9930 | Total |
|---|---|---|---|
| Hispanic or Latino | 0 | 0 | 0 |
| Not Hispanic or Latino | 10 | 2 | 12 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | BCX9930/BCX9930 | Placebo/BCX9930 | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 3 | 2 | 5 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 7 | 0 | 7 |
| White | 0 | 0 | 0 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 0 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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Studies of the drug tested here: Pelecopan