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Complement Inhibitor

Nomacopan for Transplant-Associated Thrombotic Microangiopathy

Phase 3
Waitlist Available
Research Sponsored by AKARI Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Clinical or histological diagnosis of TMA
Aged ≥ 0.5 and < 18 years at the time of diagnosis of TMA
Must not have
Received eculizumab or any other complement blocker therapy at any time
Patients weighing less than 5 kg
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 28 weeks
Awards & highlights
Pivotal Trial
No Placebo-Only Group

Summary

This trial is testing nomacopan, a medication that helps reduce inflammation and prevent blood clots. It targets children who have developed a serious blood vessel condition after a stem cell transplant. The study aims to see if nomacopan is safe and effective for these patients.

Who is the study for?
This trial is for children and teenagers under 18 who've had a stem cell transplant within the last 100 days and are diagnosed with Thrombotic Microangiopathy (TMA). They must not be allergic to the drug being tested, weigh at least 5 kg, have no severe infections or acute GVHD grade 4, haven't used complement blocker therapy before, and don't have a positive ADAMTS13 test.
What is being tested?
The study tests nomacopan (rVA576) in young patients with TMA following a stem cell transplant. It's conducted across multiple centers to see how well this treatment works for these patients. The participants will receive nomacopan after their diagnosis of TMA.
What are the potential side effects?
While specific side effects of nomacopan in this context aren't listed here, common side effects may include reactions at the injection site, potential allergic responses due to hypersensitivity to ingredients, or complications related to immune system interactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have been diagnosed with TMA.
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I was diagnosed with TMA before turning 18 but after 6 months old.
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I have had a stem cell transplant.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have taken eculizumab or similar medications before.
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I weigh less than 5 kg.
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I have the most severe form of acute graft-versus-host disease.
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I do not have any severe or worsening infections.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~28 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and 28 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
RBC 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
Secondary study objectives
Normalisation of lab parameters
Platelet transfusion independence
Renal Function Improvement
+1 more

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: nomacopan (rVA576)Experimental Treatment1 Intervention
The study population will consist of paediatric patients who have undergone allogeneic or autologous HSCT and develop HSCT-TMA within a year of HSCT

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Thrombotic Microangiopathy (TMA) involves the formation of clots in small blood vessels, leading to organ damage. Common treatments target the complement system, a part of the immune system that, when overactivated, contributes to TMA. Nomacopan, for example, inhibits Complement C5, preventing the formation of the membrane attack complex that damages endothelial cells, and Leukotriene B4, reducing inflammation. These mechanisms are crucial as they help to prevent further vascular injury and inflammation, thereby mitigating the progression of TMA and improving patient outcomes.
MASP-1 of the complement system enhances clot formation in a microvascular whole blood flow model.

Find a Location

Who is running the clinical trial?

AKARI TherapeuticsLead Sponsor
7 Previous Clinical Trials
52 Total Patients Enrolled

Media Library

nomacopan (rVA576) (Complement Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT04784455 — Phase 3
Thrombotic Microangiopathy Research Study Groups: nomacopan (rVA576)
Thrombotic Microangiopathy Clinical Trial 2023: nomacopan (rVA576) Highlights & Side Effects. Trial Name: NCT04784455 — Phase 3
nomacopan (rVA576) (Complement Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04784455 — Phase 3
~2 spots leftby Dec 2025