NCT05148299

A Study of Pegcetacoplan for Patients With Transplant-associated Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplantation

An Open-label, Single-arm, Multicenter Pilot Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy and Safety of Pegcetacoplan in Patients With Transplant-associated Thrombotic Microangiopathy (TA-TMA) After Hematopoietic Stem Cell Transplantation (HSCT)

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: Swedish Orphan Biovitrum · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of the study was to assess pharmacokinetics (PK), pharmacodynamics (PD), efficacy, and safety of pegcetacoplan in patients with TA-TMA after HSCT.

Detailed description

This was a pilot study, and the sample size was based on practical rather than statistical aspects. A total of 12 patients were to be included and treated in the study. With 12 patients included, it was estimated that 9 patients would complete at least 4 weeks of treatment, which is deemed sufficient to characterize the PK of pegcetacoplan in patients with TA-TMA to an appropriate precision. In addition, 12 patients would provide a 72 % probability to observe a response rate of at least 8 responders of the 12 patients recruited (assuming the true response rate is 70 %).

Conditions

• Transplant-Associated Thrombotic Microangiopathy

Interventions

Type	Name	Description
DRUG	Pegcetacoplan	20-cc glass vials-1080 mg

Timeline

• Start date: 2022-02-01
• Primary completion: 2024-12-08
• Completion: 2024-12-08
• First posted: 2021-12-08
• Last updated: 2025-11-28
• Results posted: 2025-11-28

Locations

14 sites across 5 countries: United States, France, Greece, Italy, Spain

Regulatory

• FDA-regulated drug study