NCT04784455

Nomacopan (rVA576) in Transplant Associated Thrombotic Microangiopathy

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

• Status: Terminated
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: AKARI Therapeutics · Industry
• Sex: All
• Age: 6 Months – 18 Years
• Healthy volunteers: Not accepted

Summary

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

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

Conditions

• Thrombotic Microangiopathies

Interventions

Type	Name	Description
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

Timeline

• Start date: 2021-02-01
• Primary completion: 2024-05-15
• Completion: 2024-05-15
• First posted: 2021-03-05
• Last updated: 2025-06-05
• Results posted: 2025-05-15

Locations

9 sites across 3 countries: United States, Poland, United Kingdom

Regulatory

• FDA-regulated drug study