TerminatedPhase 3

Nomacopan (rVA576) in Transplant Associated Thrombotic Microangiopathy

Stopped: The early termination of this study is a business decision following a portfolio reprioritization plan. The decision is not related to any Efficacy, Safety or Clinical concerns regarding Nomacopan/rVA576

United States, Poland10 participantsStarted 2021-02-01

Plain-language summary

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

Who can participate

Age range6 Months – 18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. 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 criteria

✕. 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

What they're measuring

Number of Participants NOT Requiring a Red Blood Bell Transfusion (Transfusion Independence) for 28 Days or More OR Number of Participants With a Urine Protein Creatinine Ratio Value of ≤ 2 mg/mg Maintained for 28 Days or More.

Timeframe: 24 weeks

Trial details

NCT IDNCT04784455

SponsorAKARI Therapeutics

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2024-05-15

Results submitted2025-04-30

View on ClinicalTrials.gov More Thrombotic Microangiopathies trials