Nomacopan (rVA576) in Transplant Associated Thrombotic Microangiopathy

Akari Therapeutics

Status and phase: Terminated; Phase 3

Conditions: Thrombotic Microangiopathies

Treatments: Drug: nomacopan (rVA576)

Study type: Interventional

Funder types: Industry

Identifiers

NCT04784455

AK901

Details and patient eligibility

About

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

Full 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 efficacy

Enrollment: 10 patients

Sex: All

Ages: 6 months to 18 years old

Volunteers: No Healthy Volunteers

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 graft-versus-host disease (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10%) returned from their screening assessment, the patient should be withdrawn from the study

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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10 participants in 1 patient group

nomacopan (rVA576)

Experimental group

Description:

The study population will consist of paediatric patients who have undergone allogeneic or autologous haematopoietic stem cell transplantation (HSCT) and develop transplant-associated thrombotic microangiopathy (HSCT-TMA) within a year of HSCT

Treatment:

Drug: nomacopan (rVA576)