Multicentre Study of Nomacopan (rVA576) in Paediatric Haematopoietic Stem-Cell Transplant Associated Thrombotic Microangiopathy

AK901

Multicentre Study of nomacopan in Paediatric Haematopoietic Stem-Cell Transplant Associated Thrombotic Microangiopathy

2021-02-01

2025-06-01

2025-12-01

Recruiting

Early phase I

50

Inclusion Criteria: 1. Aged ≥ 0.5 and \< 18 years at the time of diagnosis of TMA. 2. Undergone allogeneic or autologous HSCT. 3. TMA diagnosis within a year of their allogeneic or autologous HSCT. 4. Clinical or histological diagnosis of TMA 5. Provision of written informed consent. 6. Provision of informed assent Exclusion Criteria: 1. Patients weighing less than 5 kg. 2. Patients with a positive direct Coombs' test. 3. Patients who do not receive nomacopan within 21 days of the initial diagnosis of TMA. 4. Patients having an active systemic or organ system bacterial or fungal infection or progressive severe infection at the time of diagnosis of TMA 5. Grade 4 Acute GVHD 6. Received eculizumab or any other complement blocker therapy at any time. 7. Known hypersensitivity to the active ingredient or excipients If an enrolled patient has a positive ADAMTS13 test (\<10%) returned from their screening assessment, the patient should be withdrawn from the study

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': 'Open-label, uncontrolled, multi-centre two-part study. Part A: dose algorithm, safety and efficacy Part B: safety and efficacy', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 50, 'type': 'ESTIMATED'}}

2024-02-08