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A Study of Pegcetacoplan for Patients With Transplant-associated Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplantation | Clinical Trials | Clareo Health

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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)

NCT05148299•Swedish Orphan Biovitrum

View on ClinicalTrials.gov

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 %).

Eligibility

Age

18 - No limit years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. Male and female patients aged ≥ 18 years at the time of informed consent form (ICF) signature.
•2. Received allogeneic HSCT.
•3. Diagnosis of TA-TMA established, as per laboratory markers indicating TMA.
•4. Have a diagnosis of TA-TMA that persists despite initial management of any triggering condition.
•5. Have random urine protein/creatinine ratio (rUPCR) ≥ 1 mg/mg.
•6. Women of childbearing potential, defined as any women who have experienced menarche and who are NOT permanently sterile or postmenopausal, must have a negative serum pregnancy test at screening and agree to use protocol-defined methods of contraception for the duration of the study and 8 weeks after their last investigational medicinal product (IMP) dose.
•Note: Postmenopausal is defined as having had 12 consecutive months with no menses without an alternative medical cause.
•7. Men must agree to the following for the duration of the study and 8 weeks after their last dose of IMP:
•1. Avoid fathering a child.
•2. Use protocol-defined methods of contraception.
+2 more criteria

Exclusion Criteria

•1. Positive direct Coombs test.
•2. Known familial or acquired ADAMTS13 deficiency.
•3. Known Shiga toxin-related hemolytic uremic syndrome.
•4. Known bone marrow or graft failure.
•5. Diagnosis of disseminated intravascular coagulation.
•6. Diagnosis of veno-occlusive disease (VOD).
•7. Active GI bleeding (hematemesis or hematochezia) at baseline.
•8. Body weight \< 30 kg and \> 100 kg.
•9. Uncontrolled systemic bacterial or fungal infection, presence or suspicion of sepsis.
•10. Previously or currently treated with a complement inhibitor (approved or investigational).
+7 more criteria

Locations (14)

City of Hope

Duarte, California, United States

Mayo Clinic - Rochester

Rochester, Minnesota, United States

Saint-Louis Hospital

Paris, Paris, France

Archet 1 hospital, Department of Clinical Hematology

Nice, France

CHU de Saint-Etienne

Saint-Priest-en-Jarez, France

University General Hospital "Attikon"

Athens, Chaidari, Greece

General Hospital of Athens "Evangelismos"

Athens, Greece

General Hospital of Thessaloniki "G. Papanikolaou", Hematology Department - BMT Unit

Thessaloniki, Greece

Hospital San Giuseppe Moscati

Avellino, Italy

Big Metropolitan Hospital Niguarda Regional Health Authority

Milan, Italy

Key Dates

Start Date

February 1, 2022

Primary Completion Date

December 8, 2024

Completion Date

December 8, 2024

Last Updated

November 28, 2025

Enrollment

ACTUAL participants

Conditions

Transplant-Associated Thrombotic Microangiopathy

Interventions

Pegcetacoplan

DRUG