Nomacopan (rVA576) in Transplant Associated Thrombotic Microangiopathy

• Status: Recruiting
• Trial end date: 2024-06-01
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: AKARI Therapeutics

Criteria

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 100 days of their first 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 Coomb's test.

3. Patients who do not receive nomacopan within 14 days of the initial diagnosis of TMA.

4. Patients having an active systemic or organ system bacterial or fungal infection or progressive severe infection

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

8. A positive ADAMTS13 test (<10%),