A Phase 2 interventional study of BCX9930 in Paroxysmal Nocturnal Hemoglobinuria, sponsored by BioCryst Pharmaceuticals. Terminated at 11 sites in 7 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-10-24.
Sponsored by BioCryst Pharmaceuticals · Phase 2, Interventional, and Treatment
This study was designed to provide continued access to BCX9930 for participants with Paroxysmal Nocturnal Hemoglobinuria (PNH) who had benefited from treatment with BCX9930 in another BioCryst-sponsored study for PNH who, in the opinion of the investigator, would benefit from continued treatment with BCX9930; who did not have access to other effective treatment options; and to monitor the safety of BCX9930 in participants continuing to receive BCX9930 for the treatment of PNH.
This was an open-label, non-randomized study to evaluate the long-term safety of BCX9930 in participants with PNH. PNH is an acquired, rare, serious, and potentially life-threatening disorder characterized by destruction of red blood cells (RBCs) resulting from uncontrolled activity of complement.
The participants in this study had previously benefited from BCX9930 in BioCryst studies BCX9930-201, BCX9930-202, and BCX9930-203. As the development program was not being discontinued for safety reasons or due to a lack of efficacy, and the preliminary data available from the ongoing clinical studies in PNH did not change the current safety or efficacy profile for BCX9930, BioCryst remained committed to providing BCX9930 to those participants who had participated in the ongoing studies who were currently receiving benefit from BCX9930 and wished to continue treatment.
This study was used to meet that commitment by allowing for continued access to treatment with BCX9930 for any clinical study participant with PNH currently receiving treatment with BCX9930. As the development of BCX9930 was terminated, treatment was provided for a maximum of 96 weeks, as long as the investigator believed it was in the participant's best interest to continue treatment, or until the participant had access to alternative therapy for PNH, whichever came first.
188 studies on the registry are indexed under Hemoglobinuria, Paroxysmal; 48 are open to participants now.
This study's enrollment of 28 is below the median of 34 across 147 interventional studies indexed under Hemoglobinuria, Paroxysmal.
Browse Hemoglobinuria, Paroxysmal studies →BioCryst Pharmaceuticals is the lead sponsor of 54 studies on the registry; 2 are 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.
Exclusion Criteria:
Participants who had completed at least 12 weeks of treatment with BCX9930 in studies BCX9930-201, BCX9930-202, or BCX9930-203, and in the opinion of the investigator, had benefited from treatment with BCX9930 and were expected to continue benefiting from BCX9930, with no other effective treatment options, continued to receive BCX9930 tablets at a dose of 400 mg twice daily (BID) for up to 96 weeks. For participants who were permanently discontinuing BCX9930, in the absence of alternative complement inhibitor therapy, and if medically appropriate, the dose of BCX9930 was tapered based on investigator medical judgement.
Drug: BCX9930
Taken orally at 400 mg BID
Number of Participants With Treatment-emergent Events (TEAEs)
An adverse event (AE) is any untoward medical occurrence in a clinical study participant. No causal relationship with study intervention or with the clinical study itself is implied. An AE could be an unfavorable and unintended sign, symptom (including an abnormal laboratory finding), syndrome, or illness that developed or worsened during the clinical study. A serious adverse event (SAE) is defined as any untoward medical occurrence that results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, or other medically important event. An AE is considered treatment emergent if its start date was on or after the date of first dose of study treatment in Study 205 or if the AE was ongoing from the prior study. TEAEs included both serious TEAEs and non-serious TEAEs.
Time frame: From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
The study was conducted in France, Hungary, Malaysia, South Africa, South Korea, Spain, and the United Kingdom.
| Milestone | BCX9930 |
|---|---|
| Started | 28 |
| Completed | 0 |
| Not completed | 28 |
| Withdrew: Withdrawn due to termination of study | 28 |
An adverse event (AE) is any untoward medical occurrence in a clinical study participant. No causal relationship with study intervention or with the clinical study itself is implied. An AE could be an unfavorable and unintended sign, symptom (including an abnormal laboratory finding), syndrome, or illness that developed or worsened during the clinical study. A serious adverse event (SAE) is defined as any untoward medical occurrence that results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly/birth defect, or other medically important event. An AE is considered treatment emergent if its start date was on or after the date of first dose of study treatment in Study 205 or if the AE was ongoing from the prior study. TEAEs included both serious TEAEs and non-serious TEAEs.
| Participants | BCX9930 |
|---|---|
| Number of Participants With Treatment-emergent Events (TEAEs) | 27 |
Collected over From Day 1 up to 30 days after last dose (up to approximately 100 weeks). Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| BCX9930 | 0/28 (0%) | 16/28 (57.1%) | 27/28 (96.4%) |
| Event | BCX9930 |
|---|---|
| HaemolysisBlood and lymphatic system disorders | 10/28 |
| Acute kidney injuryRenal and urinary disorders | 5/28 |
| Intravascular haemolysisBlood and lymphatic system disorders | 3/28 |
| AnaemiaBlood and lymphatic system disorders | 2/28 |
| Blood lactate dehydrogenase increasedInvestigations | 1/28 |
| C-reactive protein increasedInvestigations | 1/28 |
| HypotensionVascular disorders | 1/28 |
| ThrombophlebitisVascular disorders | 1/28 |
| DiarrhoeaGastrointestinal disorders | 1/28 |
| HypoxiaRespiratory, thoracic and mediastinal disorders | 1/28 |
| Event | BCX9930 |
|---|---|
| HaemolysisBlood and lymphatic system disorders | 9/28 |
| Upper respiratory tract infectionInfections and infestations | 6/28 |
| FatigueGeneral disorders | 5/28 |
| HeadacheNervous system disorders | 5/28 |
| DiarrhoeaGastrointestinal disorders | 4/28 |
| NauseaGastrointestinal disorders | 4/28 |
| Acute kidney injuryRenal and urinary disorders | 4/28 |
| Back painMusculoskeletal and connective tissue disorders | 4/28 |
| PainGeneral disorders | 3/28 |
| PruritusSkin and subcutaneous tissue disorders | 3/28 |
| Age, Continuous(years) | BCX9930 |
|---|---|
| Mean | 41.1 ± 14.51 |
| Sex: Female, Male(Participants) | BCX9930 |
|---|---|
| Female | 13 |
| Male | 15 |
| Race/Ethnicity, Customized(Participants) | BCX9930 |
|---|---|
| Ethnicity: Hispanic or Latino | 0 |
| Ethnicity: Not Hispanic or Latino | 25 |
| Ethnicity: Not Reported | 1 |
| Ethnicity: Unknown | 2 |
| Race: American Indian or Alaska Native | 0 |
| Race: Asian | 5 |
| Race: Native Hawaiian or Other Pacific Islander | 0 |
| Race: Black or African American | 13 |
| Race: White | 9 |
| Race: Other | 1 |
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