Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2021-006776-17 | EudraCT Number |
Not provided
Not provided
Not provided
Sponsor decision
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| BCX9930 | Experimental | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| BCX9930 | Drug | Taken orally at 400 mg BID |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | From Day 1 up to 30 days after last dose (up to approximately 100 weeks) |
Not provided
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Phillip Scheinberg, MD, PhD | Beneficência Portuguesa de São Paulo | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Investigative Site | Paris | France | ||||
| Investigative Site |
Not provided
Not provided
Not provided
Not provided
Not provided
A total of 28 participants were enrolled. The participants were enrolled from previous studies BCX9930-201 (2020-000501-93), BCX9930-202 (2020-004438-39), or BCX9930-203 (2020-004403-14).
The study was conducted in France, Hungary, Malaysia, South Africa, South Korea, Spain, and the United Kingdom.
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | BCX9930 | 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 milligram (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. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | BCX9930 | 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 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. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | Safety Population was defined as all participants who received at least 1 dose. | Posted | Count of Participants | Participants | From Day 1 up to 30 days after last dose (up to approximately 100 weeks) |
From Day 1 up to 30 days after last dose (up to approximately 100 weeks)
Safety Population was defined as all participants who received at least 1 dose of study intervention.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | BCX9930 | 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 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. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Blood lactate dehydrogenase increased | Investigations | MedDRA 24.0 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Squamous cell carcinoma of skin | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 24.0 | Systematic Assessment |
The sponsor decided to prematurely terminate the study due to business reasons. The decision to stop the development of BCX9930 was not due to safety reasons or due to lack of efficacy.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | BioCryst Pharmaceuticals Inc. | +001 919859 1302 | clinicaltrials@biocryst.com |
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jul 27, 2023 | Oct 10, 2025 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Sep 23, 2024 | Oct 10, 2025 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D006457 | Hemoglobinuria, Paroxysmal |
| ID | Term |
|---|---|
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Budapest |
| Hungary |
| Investigative Site | Ampang | Malaysia |
| Investigative Site | Bloemfontein | South Africa |
| Investigative Site | Cape Town | South Africa |
| Investigative Site | Pretoria | South Africa |
| Investigative Site | Daejeon | South Korea |
| Investigative Site | Barcelona | Spain |
| Investigative Site | Valencia | Spain |
| Investigative Site | Leeds | United Kingdom |
| Investigative Site | London | United Kingdom |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| ID |
|---|
| Title |
|---|
| Description |
|---|
| OG000 | BCX9930 | 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 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. |
|
|
| 0 |
| 28 |
| 16 |
| 28 |
| 27 |
| 28 |
| C-reactive protein increased | Investigations | MedDRA 24.0 | Systematic Assessment |
|
| Hypotension | Vascular disorders | MedDRA 24.0 | Systematic Assessment |
|
| Thrombophlebitis | Vascular disorders | MedDRA 24.0 | Systematic Assessment |
|
| Haemolysis | Blood and lymphatic system disorders | MedDRA 24.0 | Systematic Assessment |
|
| Anaemia | Blood and lymphatic system disorders | MedDRA 24.0 | Systematic Assessment |
|
| Intravascular haemolysis | Blood and lymphatic system disorders | MedDRA 24.0 | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
|
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
|
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
|
| Acute kidney injury | Renal and urinary disorders | MedDRA 24.0 | Systematic Assessment |
|
| Tubulointerstitial nephritis | Renal and urinary disorders | MedDRA 24.0 | Systematic Assessment |
|
| Escherichia urinary tract infection | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
|
| Urinary tract infection | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
|
| Pneumonia | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
|
| Thrombophlebitis | Vascular disorders | MedDRA 24.0 | Systematic Assessment |
|
| Fatigue | General disorders | MedDRA 24.0 | Systematic Assessment |
|
| Pain | General disorders | MedDRA 24.0 | Systematic Assessment |
|
| Pyrexia | General disorders | MedDRA 24.0 | Systematic Assessment |
|
| Asthenia | General disorders | MedDRA 24.0 | Systematic Assessment |
|
| Inflammation | General disorders | MedDRA 24.0 | Systematic Assessment |
|
| Breast mass | Reproductive system and breast disorders | MedDRA 24.0 | Systematic Assessment |
|
| Vaginal haemorrhage | Reproductive system and breast disorders | MedDRA 24.0 | Systematic Assessment |
|
| Erectile dysfunction | Reproductive system and breast disorders | MedDRA 24.0 | Systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
|
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
|
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
|
| Respiratory disorder | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
|
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
|
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
|
| Insomnia | Psychiatric disorders | MedDRA 24.0 | Systematic Assessment |
|
| Major depression | Psychiatric disorders | MedDRA 24.0 | Systematic Assessment |
|
| Blood cholesterol increased | Investigations | MedDRA 24.0 | Systematic Assessment |
|
| Blood lactate dehydrogenase increased | Investigations | MedDRA 24.0 | Systematic Assessment |
|
| Heart rate increased | Investigations | MedDRA 24.0 | Systematic Assessment |
|
| Lung diffusion test decreased | Investigations | MedDRA 24.0 | Systematic Assessment |
|
| SARS-CoV-2 test positive | Investigations | MedDRA 24.0 | Systematic Assessment |
|
| Epicondylitis | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
|
| Overdose | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
|
| Skin laceration | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
|
| Tachycardia | Cardiac disorders | MedDRA 24.0 | Systematic Assessment |
|
| Headache | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
|
| Dizziness | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
|
| Hypersomnia | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
|
| Multiple sclerosis | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
|
| Radiculopathy | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
|
| Haemolysis | Blood and lymphatic system disorders | MedDRA 24.0 | Systematic Assessment |
|
| Anaemia | Blood and lymphatic system disorders | MedDRA 24.0 | Systematic Assessment |
|
| Splenomegaly | Blood and lymphatic system disorders | MedDRA 24.0 | Systematic Assessment |
|
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA 24.0 | Systematic Assessment |
|
| Vertigo | Ear and labyrinth disorders | MedDRA 24.0 | Systematic Assessment |
|
| Diarrhoea | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
|
| Nausea | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
|
| Abdominal pain | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
|
| Toothache | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
|
| Vomiting | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
|
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
|
| Dyspepsia | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
|
| Glossodynia | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
|
| Dysphagia | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
|
| Oesophageal spasm | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
|
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
|
| Acne | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
|
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
|
| Rash pruritic | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
|
| Skin lesion | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
|
| Rash | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
|
| Acute kidney injury | Renal and urinary disorders | MedDRA 24.0 | Systematic Assessment |
|
| Haemoglobinuria | Renal and urinary disorders | MedDRA 24.0 | Systematic Assessment |
|
| Haematuria | Renal and urinary disorders | MedDRA 24.0 | Systematic Assessment |
|
| Chronic kidney disease | Renal and urinary disorders | MedDRA 24.0 | Systematic Assessment |
|
| Micturition urgency | Renal and urinary disorders | MedDRA 24.0 | Systematic Assessment |
|
| Pollakiuria | Renal and urinary disorders | MedDRA 24.0 | Systematic Assessment |
|
| Sterile pyuria | Renal and urinary disorders | MedDRA 24.0 | Systematic Assessment |
|
| Urinary tract pain | Renal and urinary disorders | MedDRA 24.0 | Systematic Assessment |
|
| Dysuria | Renal and urinary disorders | MedDRA 24.0 | Systematic Assessment |
|
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
|
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
|
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
|
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
|
| Gouty arthritis | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
|
| Muscle fatigue | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
|
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
|
| Urinary tract infection | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
|
| Acute sinusitis | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
|
| COVID-19 | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
|
| Candida infection | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
|
| Folliculitis | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
|
| Herpes simplex reactivation | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
|
| Influenza | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
|
| Periorbital cellulitis | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
|
| Pulmonary tuberculosis | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
|
| Rhinovirus infection | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
|
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
|
| Gout | Metabolism and nutrition disorders | MedDRA 24.0 | Systematic Assessment |
|
| Hypercalcaemia | Metabolism and nutrition disorders | MedDRA 24.0 | Systematic Assessment |
|
| Hyperuricaemia | Metabolism and nutrition disorders | MedDRA 24.0 | Systematic Assessment |
|
| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA 24.0 | Systematic Assessment |
|
| Hypomagnesaemia | Metabolism and nutrition disorders | MedDRA 24.0 | Systematic Assessment |
|
Not provided
Not provided
| D009190 |
| Myelodysplastic Syndromes |
| D001855 | Bone Marrow Diseases |