Nomacopan (rVA576) in Transplant Associated Thrombotic Microangiopathy

NCT04784455 | Terminated Phase 3 Results FDA Drug

• 1 other identifier: AK901
• Study Type: interventional
• Target: 10
• Locations: 3 countries
• Sites: 9

Brief Summary

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

Conditions

Thrombotic Microangiopathies

Outcome Measures

Primary Outcomes (1)

• 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.

  Red blood cell transfusion independence for ≥28 days immediately prior to any scheduled clinical visit up to Week 24 or Urine protein creatinine ratio ≤ 2 mg/mg maintained over ≥ 28 days immediately prior to any scheduled clinical visit up to week 24 Transfusion independence is defined as no RBC transfusion attributable to, or required to manage, thrombotic microangiopathy (TMA). Transfusions required for causes other than TMA will not be considered within the evaluation of the efficacy endpoints.

  24 weeks

Secondary Outcomes (4)

• Number of Participants With a Normalised sC5b-9 Value (Where sC5b-9 is the Same Value as the Upper Limit of Normal or Less)

  24 weeks

• Number of Participants With a Normalised Lactate Dehydrogenase (LDH) Value (Where LDH is the Same Value as the Upper Limit of Normal or Less)

  24 weeks

• Normalisation of Lab Parameters

  24 weeks

• Number of Participants Not Requiring a Platelet Transfusion (Transfusion Independence) for 28 Days or More.

  24 weeks

Study Arms (1)

nomacopan (rVA576)

EXPERIMENTAL

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

Drug: nomacopan (rVA576)

Interventions

nomacopan (rVA576) DRUG

The study population will consist of paediatric patients who have undergone allogeneic or autologous HSCT and develop HSCT-TMA within a year of HSCT

nomacopan (rVA576)

Eligibility Criteria

• Age: 6 Months - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• AKARI Therapeutics: lead

Study Sites (9)

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

Stanford Children's Hospital

Palo Alto, California, 94304, United States

Location

Duke University Medical Center, Children's Health Center

Durham, North Carolina, 27710, United States

Location

Children's Hospitall of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Uniwersytecki Szpital Kliniczny im. Jana Mikulicza Radeckiego we Wroclawiu

Wroclaw, 50556, Poland

Location

The Royal Marsden NHS Foundation Trust

London, SM25PT, United Kingdom

Location

St. Georges University Hospital

London, SW170QT, United Kingdom

Location

Great Ormond Street Hospital (GOSH)

London, WC1N3JH, United Kingdom

Location

Royal Manchester Children's Hospital

Manchester, M139WL, United Kingdom

Location

MeSH Terms

Conditions

Thrombotic Microangiopathies

Condition Hierarchy (Ancestors)

Thrombocytopenia; Blood Platelet Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Cytopenia

Results Point of Contact

• Title: Chief Scientific Officer
• Organization: Akari Therapeutics

miles.nunn@akaritx.com

+44 208 004 0261

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Open-label, uncontrolled, multi-centre two-part study. Part A: dose algorithm, safety and efficacy Part B: safety and efficacy

Study Record Dates

• First Submitted: November 19, 2020
• First Posted: March 5, 2021
• Study Start: February 1, 2021
• Primary Completion: May 15, 2024
• Study Completion: May 15, 2024
• Last Updated: June 5, 2025
• Results First Posted: May 15, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

GB (4); PL (1); US (4)