PIONEER: A Phase 2 Clinical Trial for Adolescents with PNH
Research type
Research Study
Full title
An Open-Label, Single-Arm, Phase 2 Study to Evaluate the Safety, Pharmacokinetics, and Biologic Activity of Pegcetacoplan in Pediatric Patients with Paroxysmal Nocturnal Hemoglobinuria
IRAS ID
1003723
Contact name
Mara Furlow
Contact email
Sponsor organisation
Apellis Pharmaceuticals, Inc.
Eudract number
2020-001350-21
Research summary
Paroxysmal Nocturnal Hemoglobinuria (PNH) a rare disorder that causes the red blood cells to break down too early resulting in low hemoglobin levels (anemia). PNH patients may continue to suffer from complications such as blood clots, bleeding, or anemia even after receiving standard treatment such as transfusions. This research study is being conducted is to learn more about the safety of the investigational drug, pegcetacoplan (peg-set-a-koé-plan), and its ability to reduce anemia and the need for repeated blood transfusions in adolescents (12-17 year-olds) with PNH. This study will also evaluate how the body processes pegcetacoplan. Approximately 12 boys and girls around the globe will be asked if they want to take part in this study. All of them will be 12-17 years old. Suitable participants will receive pegcetacoplan for up to 5 months (20 weeks) and will be enrolled in the study for up to 4 years.
REC name
London - Westminster Research Ethics Committee
REC reference
21/LO/0655
Date of REC Opinion
9 Nov 2021
REC opinion
Further Information Favourable Opinion