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

NCT05148299 | Completed Phase 2 Results FDA Drug

• 3 other identifiers: Sobi.PEGCET-2012023-510443-37-002021-003157-27
• Study Type: interventional
• Target: 12
• Locations: 5 countries
• Sites: 14

Brief 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.

Conditions

Transplant-Associated Thrombotic Microangiopathy

Keywords

TA-TMA

Outcome Measures

Primary Outcomes (4)

• Pegcetacoplan Pharmacokinetic (PK) Parameter Area Under the Curve Limited to the End of Dosing Interval (AUC0-tau)

  Area under the concentration-time curve limited to the end of the dosing interval. The samples included in the calculation of AUC0-tau were collected at the following times: on dosing Days 1, 3 and 5, PK samples were taken up to 30 minutes pre-dose and at 15 minutes (± 5 min), 30 minutes (± 5 min), 1 hour (± 10 min), 4 hours (± 10 min), 8 hours (± 30 min), and 24 hours (± 30 min) post-dose as well as on Day 8 pre-dose.

  Week 1

• Pegcetacoplan PK Parameter Maximal Serum Concentration (Cmax)

  Maximum observed serum concentration. Determined using the samples collected post-dose on dosing Days 1, 3 and 5.

  Week 1

• Pegcetacoplan PK Parameter Time to Cmax (Tmax)

  Time of maximum measured serum concentration. Determined using the samples collected post-dose on dosing Days 1, 3 and 5.

  Week 1

• Pegcetacoplan PK Parameter Observed Serum Concentration Pre-dose (Ctrough)

  Observed serum concentration pre-dose. From Day 8 (Week 1) and onwards, PK samples were taken pre-dose at each visit.

  Week 1 up to Week 14

Secondary Outcomes (18)

• Absolute Levels and Change From Baseline in sC5b-9

  Week 24

• Absolute Levels and Change From Baseline in C3a

  Week 24

• Absolute Levels and Change From Baseline in C3

  Week 24

• Absolute Levels and Change From Baseline in Bb

  Week 24

• Absolute Levels and Change From Baseline in C4a

  Week 24

Other Outcomes (17)

• Number of Participants With Treatment-emergent Adverse Events

  From treatment start to end of study, up to 6 months (8 weeks since last dose of IMP)

• Change From Baseline in Platelets

  Week 24

• Change From Baseline in Hemoglobin

  Week 24

Study Arms (1)

Pegcetacoplan

EXPERIMENTAL

sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.

Drug: Pegcetacoplan

Interventions

Pegcetacoplan DRUG

20-cc glass vials-1080 mg

Pegcetacoplan

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male and female patients aged ≥ 18 years at the time of informed consent form (ICF) signature.
• Received allogeneic HSCT.
• Diagnosis of TA-TMA established, as per laboratory markers indicating TMA.
• Have a diagnosis of TA-TMA that persists despite initial management of any triggering condition.
• Have random urine protein/creatinine ratio (rUPCR) ≥ 1 mg/mg.
• 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.
• Men must agree to the following for the duration of the study and 8 weeks after their last dose of IMP:
• Avoid fathering a child.
• Use protocol-defined methods of contraception.
• Refrain from donating sperm.
• Patient and/or legally authorized representative must be capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF.

You may not qualify if:

• Positive direct Coombs test.
• Known familial or acquired ADAMTS13 deficiency.
• Known Shiga toxin-related hemolytic uremic syndrome.
• Known bone marrow or graft failure.
• Diagnosis of disseminated intravascular coagulation.
• Diagnosis of veno-occlusive disease (VOD).
• Active GI bleeding (hematemesis or hematochezia) at baseline.
• Body weight \< 30 kg and \> 100 kg.
• Uncontrolled systemic bacterial or fungal infection, presence or suspicion of sepsis.
• Previously or currently treated with a complement inhibitor (approved or investigational).
• Pregnancy or breastfeeding.
• Positive human immunodeficiency virus antibody at screening or documented in pre-HSCT medical record.
• Hepatitis C virus detectable by polymerase chain reaction at screening or documented in pre-HSCT medical record.
• Chronic inactive hepatitis B virus with viral loads \> 1000 IU/mL (\> 5000 copies/mL) at screening or documented in pre-HSCT medical record. Eligible patients who are chronic active carriers (≤ 1000 IU/mL) must receive prophylactic antiviral treatment (e.g., entecavir, tenofovir, lamivudine) according to local country guidelines.
• Known or suspected hereditary fructose intolerance.

Sponsors & Collaborators

• Swedish Orphan Biovitrum: lead
• Apellis Pharmaceuticals, Inc.: collaborator

Study Sites (14)

City of Hope

Duarte, California, 91010, United States

Location

Mayo Clinic - Rochester

Rochester, Minnesota, 55905, United States

Location

Saint-Louis Hospital

Paris, Paris, 75010, France

Location

Archet 1 hospital, Department of Clinical Hematology

Nice, 06200, France

Location

CHU de Saint-Etienne

Saint-Priest-en-Jarez, 42270, France

Location

University General Hospital "Attikon"

Athens, Chaidari, 12462, Greece

Location

General Hospital of Athens "Evangelismos"

Athens, 10676, Greece

Location

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

Thessaloniki, 57010, Greece

Location

Hospital San Giuseppe Moscati

Avellino, 83100, Italy

Location

Big Metropolitan Hospital Niguarda Regional Health Authority

Milan, 20162, Italy

Location

ASST Monza - S. Gerardo Hospital

Monza, 20900, Italy

Location

United Hospitals Villa Sofia Cervello

Palermo, 90146, Italy

Location

University Polyclinic Foundation "Agostino Gemelli" - IRCCS

Roma, 00168, Italy

Location

University Hospital Puerta de Hierro Majadahonda

Madrid, 28222, Spain

Location

MeSH Terms

Interventions

pegcetacoplan

Limitations and Caveats

The small sample size limits the generalizability of the findings.

Results Point of Contact

• Title: Medical Director
• Organization: Swedish Orphan Biovitrum AB

medical.info@sobi.com

+4686972000

Study Officials

• Study physician

  Swedish Orphan Biovitrum AB

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: open label, single arm, multicenter

Study Record Dates

• First Submitted: October 21, 2021
• First Posted: December 8, 2021
• Study Start: February 1, 2022
• Primary Completion: December 8, 2024
• Study Completion: December 8, 2024
• Last Updated: November 28, 2025
• Results First Posted: November 28, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

FR (3); GR (3); ES (1); US (2); IT (5)