nomacopan (rVA576) for Thrombotic Microangiopathies

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
Thrombotic Microangiopathies+1 More
nomacopan (rVA576) - Drug
Eligibility
< 65
All Sexes
What conditions do you have?
Select

Study Summary

This trial is testing a new medication to treat a complication from stem cell transplants in children.

Eligible Conditions
  • Thrombotic Microangiopathies

Treatment Effectiveness

Effectiveness Progress

2 of 3
This is further along than 85% of similar trials

Study Objectives

2 Primary · 4 Secondary · Reporting Duration: 28 weeks

24 weeks
Normalisation of lab parameters
Platelet transfusion independence
RBC transfusion independence for ≥ 28 days immediately prior to any scheduled clinical visit up to Week 24
Renal Function Improvement
Urine protein creatinine ratio ≤ 2 mg/mg
28 weeks
Safety and tolerability of nomacopan

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Trial Design

1 Treatment Group

nomacopan (rVA576)
1 of 1

Experimental Treatment

50 Total Participants · 1 Treatment Group

Primary Treatment: nomacopan (rVA576) · No Placebo Group · Phase 3

nomacopan (rVA576)
Drug
Experimental Group · 1 Intervention: nomacopan (rVA576) · Intervention Types: Drug

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 28 weeks

Who is running the clinical trial?

AKARI TherapeuticsLead Sponsor
7 Previous Clinical Trials
57 Total Patients Enrolled

Eligibility Criteria

Age < 65 · All Participants · 6 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
The study will require provision of written informed consent.
The study found that the median time to TMA diagnosis was 100 days for those who had an allogeneic HSCT and 85 days for those who had an autologous HSCT.
A person who is at least 0.5 years old and younger than 18 years old when they are diagnosed with TMA.
Undergone allogeneic or autologous HSCT

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 13th, 2021

Last Reviewed: November 24th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.