Nomacopan (rVA576) in Transplant Associated Thrombotic Microangiopathy

February 7, 2024 updated by: AKARI Therapeutics

Multicentre Study of Nomacopan (rVA576) in Paediatric Haematopoietic Stem-Cell Transplant Associated Thrombotic Microangiopathy

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

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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 efficacy

Study Type

Interventional

Enrollment (Estimated)

50

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Poland
      • Wrocław, Poland, 50556
        • Recruiting
        • Uniwersytecki Szpital Kliniczny im. Jana Mikulicza Radeckiego we Wroclawiu
        • Contact:
        • Principal Investigator:
          • Krzysztof Kalwak
  • United Kingdom
      • London, United Kingdom, WC1N3JH
        • Recruiting
        • Great Ormond Street Hospital (GOSH)
        • Contact:
        • Principal Investigator:
          • Robert Chiesa
      • Manchester, United Kingdom, M139WL
        • Recruiting
        • Royal Manchester Children's Hospital
        • Contact:
        • Principal Investigator:
          • Robert Wynn
  • United States
    • California
      • Los Angeles, California, United States, 90027
      • Palo Alto, California, United States, 94304
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Recruiting
        • Duke University Medical Center, Children's Health Center
        • Contact:
        • Principal Investigator:
          • Vinod Prasad
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • Children's Hospitall of Philadelphia
        • Contact:
        • Principal Investigator:
          • Timothy Olson

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

10 months to 16 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

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 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: nomacopan (rVA576)
The study population will consist of paediatric patients who have undergone allogeneic or autologous HSCT and develop HSCT-TMA within a year of HSCT
Drug: nomacopan (rVA576)
The study population will consist of paediatric patients who have undergone allogeneic or autologous HSCT and develop HSCT-TMA within a year of HSCT

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
RBC transfusion independence for ≥ 28 days immediately prior to any scheduled clinical visit up to Week 24
Time Frame: 24 weeks
Transfusion independence is defined as no RBC or platelet 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 primary efficacy endpoints.
24 weeks
Urine protein creatinine ratio ≤ 2 mg/mg
Time Frame: 24 weeks
Urine protein creatinine ratio ≤ 2 mg/mg
24 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Renal Function Improvement
Time Frame: 24 weeks
Percentage of patients who achieve the primary endpoint of urine protein creatinine ratio ≤ 2 mg/mg (the nephrotic threshold) for ≥ 28 days
24 weeks
Platelet transfusion independence
Time Frame: 24 weeks
Platelet transfusion independence for ≥ 28 days within the 24 week timeframe
24 weeks
Normalisation of lab parameters
Time Frame: 24 weeks
Lactate dehydrogenase (LDH) ≤ULN
24 weeks
Normalisation of lab parameters
Time Frame: 24 weeks
Normalization of haptoglobin
24 weeks
Safety and tolerability of nomacopan
Time Frame: 28 weeks
New or worsening AEs after dosing of investigational product will be recorded in the eCRF.
28 weeks
Safety and tolerability of nomacopan
Time Frame: 28 weeks
Listings of subjects who have an SAE.
28 weeks
Safety and tolerability of nomacopan
Time Frame: 28 weeks
Listings of subjects who discontinue from the study due to an AE.
28 weeks
Safety and tolerability of nomacopan
Time Frame: 28 weeks
Occurrence of significant laboratory abnormalities will be summarized.
28 weeks
Normalisation of lab parameters
Time Frame: 24 weeks
Plasma sC5b-9 ≤ ULN
24 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

AKARI Therapeutics

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

February 1, 2021

Primary Completion (Estimated)

June 1, 2025

Study Completion (Estimated)

December 1, 2025

Study Registration Dates

First Submitted

November 19, 2020

First Submitted That Met QC Criteria

March 4, 2021

First Posted (Actual)

March 5, 2021

Study Record Updates

Last Update Posted (Actual)

February 8, 2024

Last Update Submitted That Met QC Criteria

February 7, 2024

Last Verified

February 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AK901

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Thrombotic Microangiopathies

Clinical Trials on nomacopan (rVA576)

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Norway

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe