PIONEER: A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) | Aplastic Anemia and MDS International Foundation (AAMDSIF) Return to top.

PIONEER: A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Trial Sponsor

Apellis Pharmaceuticals

Contact Info

Apellis Clinical Trial Information

Start Date: February 4, 2021 End Date: October 1, 2024 Identifier: NCT04901936

If you are interested in learning more about your possible participation in this clinical trial, please complete the form. Your information will be forwarded directly to the sponsoring company.


The purpose of this open-label study is to evaluate the safety, effectiveness, and biologic activity (how the investigational medication is processed by the body) of pegcetacoplan in 12-17 year-olds who have paroxysmal nocturnal hemoglobinuria (PNH).

The study will consist of a 4-week screening period followed by a 16-week treatment period. Participants switching from a C5 inhibitor will have an additional 4-week run-in period between the screening and treatment periods. At the completion of the study treatment period, participants will either enter a long-term extension period or a 2-month follow-up period.

All eligible study participants will receive pegcetacoplan, administered via subcutaneous infusion twice a week at home. The subcutaneous infusion requires two small needles to be inserted into the fatty layer of tissue under the skin and the investigational medication will flow into the body. Study participants and/or caregivers will be trained on home administration of pegcetacoplan.

Bone Marrow Disease(s): 
Associated Drug(s): 
Phase 2
Age Group: 
12 years to 17 years
Accepts Healthy Volunteers: 
Details on 
Inclusion Criteria: 
  • 12-17 years old at the time of screening
  • Weigh at least 20 kg (approximately 44 lbs)
  • Have been diagnosed with PNH, confirmed by high-sensitivity flow cytometry (granulocyte or monocyte clone >10%)
    • Not being treated with an approved complement inhibitor (eculizumab or ravulizumab) prior to start of pegcetacoplan dosing, AND have hemolytic anemia. Hemolytic anemia is defined as hemoglobin (Hb) less than the lower limit of normal (Hb < LLN) and LDH >1.5 times the upper limit of normal (ULN);
    • Currently receiving treatment with an approved complement inhibitor (eculizumab or ravulizumab) AND have evidence of ongoing anemia. Ongoing anemia is defined as Hb < LLN and ARC > ULN
  • Have a platelet count >75,000/mm3 and an absolute neutrophil count >1000/mm3
Exclusion Criteria: 
  • Are an adult, 18 years of age or older, with PNH
  • Known or suspected hereditary fructose intolerance (HFI)
  • History of hereditary complement deficiency, bone marrow transplant, or meningococcal disease (meningitis, bacteremia or septicemia)
  • Females who are pregnant or breastfeeding

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