Status:
COMPLETED
A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH
Lead Sponsor:
Apellis Pharmaceuticals, Inc.
Conditions:
Paroxysmal Nocturnal Hemoglobinuria
Eligibility:
All Genders
18+ years
Phase:
PHASE3
Brief Summary
Evaluation of the Efficacy and Safety of Pegcetacoplan in Patients with Paroxysmal Nocturnal Hemoglobinuria .
Eligibility Criteria
Inclusion
- Be at least 18 years old (inclusive).
- Have LDH ≥1.5 x ULN at the screening visit.
- Have PNH diagnosis, confirmed by high sensitivity flow cytometry (granulocyte or monocyte clone \>10%).
- Have Hb less than the lower limit of normal (LLN) at the screening visit.
- Have ferritin greater than/equal to the LLN, or total iron binding capacity (TIBC) less than/equal to ULN at the screening visit, based on central laboratory reference ranges. If a subject is receiving iron supplements at screening, the Investigator must ensure that the subject's dose has been stable for 4 weeks prior to screening, and it must be maintained throughout the study. Subjects not receiving iron at screening must not start iron supplementation during the course of the study.
- Body mass index (BMI) ≤ 35 kg/m2 at the screening visit.
- Have a platelet count of \>50,000/mm3 at the screening visit.
- Have an absolute neutrophil count \>500/mm3 at the screening visit.
Exclusion
- Treatment with any complement inhibitor (eg, eculizumab) within 3 months prior to screening.
- Hereditary complement deficiency.
- History of bone marrow transplantation.
- Concomitant use of any of the following medications is prohibited if not on a stable regimen for the time period indicated below prior to screening:
- Erythropoietin or immunosuppressants for at least 8 weeks
- Systemic corticosteroids for at least 4 weeks
- Vitamin K antagonists (eg, warfarin) with a stable international normalized ratio (INR) for at least 4 weeks
- Iron supplements, vitamin B12, or folic acid for at least 4 weeks
- Low-molecular-weight heparin for at least 4 weeks
Key Trial Info
Start Date :
August 27 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 23 2021
Estimated Enrollment :
53 Patients enrolled
Trial Details
Trial ID
NCT04085601
Start Date
August 27 2019
End Date
June 23 2021
Last Update
October 21 2022
Active Locations (30)
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1
Julian Coronel Medical Center
Cali, Colombia
2
Research Center of the Colombian Clinical Life Cancer Foundation
Medellín, Colombia
3
Queen Mary Hospital
Hong Kong, Hong Kong
4
Princess Margaret Hospital
Kwai Chung, Hong Kong