Status:
TERMINATED
Nomacopan (rVA576) in Transplant Associated Thrombotic Microangiopathy
Lead Sponsor:
AKARI Therapeutics
Conditions:
Thrombotic Microangiopathies
Eligibility:
All Genders
6-18 years
Phase:
PHASE3
Brief Summary
Multicentre Study of nomacopan in Paediatric Haematopoietic Stem-Cell Transplant Associated Thrombotic Microangiopathy
Detailed Description
This is an open-label, multi-centre study of two-parts, Part A and B, includes 24 weeks of treatment, safety follow up after 30 days. Part A: dose algorithm, safety and efficacy Part B: safety and e...
Eligibility Criteria
Inclusion
- Aged ≥ 0.5 and \< 18 years at the time of diagnosis of TMA.
- Undergone allogeneic or autologous HSCT.
- TMA diagnosis within a year of their allogeneic or autologous HSCT.
- Clinical or histological diagnosis of TMA
- Provision of written informed consent.
- Provision of informed assent
Exclusion
- Patients weighing less than 5 kg.
- Patients with a positive direct Coombs' test.
- Patients who do not receive nomacopan within 21 days of the initial diagnosis of TMA.
- Patients having an active systemic or organ system bacterial or fungal infection or progressive severe infection at the time of diagnosis of TMA
- Grade 4 Acute graft-versus-host disease (GVHD)
- Received eculizumab or any other complement blocker therapy at any time.
- 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10%) returned from their screening assessment, the patient should be withdrawn from the study
Key Trial Info
Start Date :
February 1 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 15 2024
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT04784455
Start Date
February 1 2021
End Date
May 15 2024
Last Update
June 5 2025
Active Locations (9)
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1
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
2
Stanford Children's Hospital
Palo Alto, California, United States, 94304
3
Duke University Medical Center, Children's Health Center
Durham, North Carolina, United States, 27710
4
Children's Hospitall of Philadelphia
Philadelphia, Pennsylvania, United States, 19104