A Study of Pegcetacoplan for Patients With Transplant-associated Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplantation
NCT ID: NCT05148299
Last Updated: 2025-11-28
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE2
12 participants
INTERVENTIONAL
2022-02-01
2024-12-08
Brief Summary
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Detailed Description
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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
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Pegcetacoplan
sterile solution in stoppered glass vial given as infusion, for a total treatment duration of 12 to 16 weeks.
Pegcetacoplan
20-cc glass vials-1080 mg
Interventions
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Pegcetacoplan
20-cc glass vials-1080 mg
Eligibility Criteria
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Inclusion Criteria
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.
3. Refrain from donating sperm.
8. 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.
Exclusion Criteria
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).
11. Pregnancy or breastfeeding.
12. Positive human immunodeficiency virus antibody at screening or documented in pre-HSCT medical record.
13. Hepatitis C virus detectable by polymerase chain reaction at screening or documented in pre-HSCT medical record.
14. 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.
15. Known or suspected hereditary fructose intolerance.
16. Hypersensitivity to pegcetacoplan or any of its excipients.
17. Inability to cooperate with study procedures or any condition that, in the opinion of the investigator, could increase the patient's risk by participating in the study or confound the outcome of the study.
18 Years
ALL
No
Sponsors
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Apellis Pharmaceuticals, Inc.
INDUSTRY
Swedish Orphan Biovitrum
INDUSTRY
Responsible Party
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Principal Investigators
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Study physician
Role: STUDY_DIRECTOR
Swedish Orphan Biovitrum AB
Locations
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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
ASST Monza - S. Gerardo Hospital
Monza, , Italy
United Hospitals Villa Sofia Cervello
Palermo, , Italy
University Polyclinic Foundation "Agostino Gemelli" - IRCCS
Roma, , Italy
University Hospital Puerta de Hierro Majadahonda
Madrid, , Spain
Countries
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Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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2023-510443-37-00
Identifier Type: CTIS
Identifier Source: secondary_id
2021-003157-27
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
Sobi.PEGCET-201
Identifier Type: -
Identifier Source: org_study_id
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