NCT05741346

Long-term Safety of BCX9930 in Participants With Paroxysmal Nocturnal Hemoglobinuria

Terminated Phase 2 Results posted Last updated 24 October 2025

What this trial tests

Phase 2 trial testing BCX9930 in Paroxysmal Nocturnal Hemoglobinuria in 28 participants. Terminated before completion.

Timeline

18 January 2023

Primary endpoint
31 January 2025

31 January 2025

Quick facts

Lead sponsor	BioCryst Pharmaceuticals
Phase	Phase 2
Status	Terminated
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	28
Start date	18 January 2023
Primary completion	31 January 2025
Estimated completion	31 January 2025
Sites	11 locations across France, South Africa, Malaysia, United Kingdom, Hungary, South Korea, Spain

Drugs / interventions tested

• BCX9930 — full drug profile →

Conditions studied

• Paroxysmal Nocturnal Hemoglobinuria — all drugs for Paroxysmal Nocturnal Hemoglobinuria →

Sponsor

BioCryst Pharmaceuticals — full company profile →

Who can join

18 and older, any sex, with Paroxysmal Nocturnal Hemoglobinuria. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Number of Participants With Treatment-emergent Events (TEAEs) Primary · From Day 1 up to 30 days after last dose (up to approximately 100 weeks)

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 di

Group	Value	95% CI
BCX9930	27

Adverse events — posted to ClinicalTrials.gov

Time frame: From Day 1 up to 30 days after last dose (up to approximately 100 weeks). Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

BCX9930

Serious: 16/28 (57%)

Deaths: 0/28

Serious adverse events (15 terms)

Reaction	System	BCX9930
Haemolysis	Blood and lymphatic system disorders	—
Acute kidney injury	Renal and urinary disorders	—
Intravascular haemolysis	Blood and lymphatic system disorders	—
Anaemia	Blood and lymphatic system disorders	—
Blood lactate dehydrogenase increased	Investigations	—
C-reactive protein increased	Investigations	—
Hypotension	Vascular disorders	—
Thrombophlebitis	Vascular disorders	—
Diarrhoea	Gastrointestinal disorders	—
Hypoxia	Respiratory, thoracic and mediastinal disorders	—
Pulmonary embolism	Respiratory, thoracic and mediastinal disorders	—
Tubulointerstitial nephritis	Renal and urinary disorders	—
Escherichia urinary tract infection	Infections and infestations	—
Urinary tract infection	Infections and infestations	—
Pneumonia	Infections and infestations	—

Other adverse events (88 terms — click to expand)

Reaction	System	BCX9930
Haemolysis	Blood and lymphatic system disorders	—
Upper respiratory tract infection	Infections and infestations	—
Fatigue	General disorders	—
Headache	Nervous system disorders	—
Diarrhoea	Gastrointestinal disorders	—
Nausea	Gastrointestinal disorders	—
Acute kidney injury	Renal and urinary disorders	—
Back pain	Musculoskeletal and connective tissue disorders	—
Pain	General disorders	—
Pruritus	Skin and subcutaneous tissue disorders	—
Haemoglobinuria	Renal and urinary disorders	—
Pain in extremity	Musculoskeletal and connective tissue disorders	—
Pyrexia	General disorders	—
Cough	Respiratory, thoracic and mediastinal disorders	—
Insomnia	Psychiatric disorders	—
Anaemia	Blood and lymphatic system disorders	—
Abdominal pain	Gastrointestinal disorders	—
Toothache	Gastrointestinal disorders	—
Vomiting	Gastrointestinal disorders	—
Haematuria	Renal and urinary disorders	—
Nasopharyngitis	Infections and infestations	—
Urinary tract infection	Infections and infestations	—
Squamous cell carcinoma of skin	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	—
Thrombophlebitis	Vascular disorders	—
Asthenia	General disorders	—
Inflammation	General disorders	—
Breast mass	Reproductive system and breast disorders	—
Vaginal haemorrhage	Reproductive system and breast disorders	—
Erectile dysfunction	Reproductive system and breast disorders	—
Dyspnoea	Respiratory, thoracic and mediastinal disorders	—
Oropharyngeal pain	Respiratory, thoracic and mediastinal disorders	—
Respiratory disorder	Respiratory, thoracic and mediastinal disorders	—
Epistaxis	Respiratory, thoracic and mediastinal disorders	—
Pulmonary embolism	Respiratory, thoracic and mediastinal disorders	—
Major depression	Psychiatric disorders	—
Blood cholesterol increased	Investigations	—
Blood lactate dehydrogenase increased	Investigations	—
Heart rate increased	Investigations	—
Lung diffusion test decreased	Investigations	—
SARS-CoV-2 test positive	Investigations	—

Most-reported serious reactions: Haemolysis, Acute kidney injury, Intravascular haemolysis, Anaemia, Blood lactate dehydrogenase increased, C-reactive protein increased, Hypotension, Thrombophlebitis.

Data from ClinicalTrials.gov NCT05741346 adverse events section.

Sponsor's own description

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.

Publications & conference data

No peer-reviewed publications indexed yet for this trial.