NCT04901936 A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
| NCT ID | NCT04901936 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | Apellis Pharmaceuticals, Inc. |
| Condition | Paroxysmal Nocturnal Hemoglobinuria (PNH) |
| Study Type | INTERVENTIONAL |
| Enrollment | 12 participants |
| Start Date | 2021-02-04 |
| Primary Completion | 2028-12 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 12 participants in total. It began in 2021-02-04 with a primary completion date of 2028-12.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
The purpose of this study is to evaluate the safety, effectiveness, and biological activity (how the investigational medication is processed by the body) of pegcetacoplan in 12-17 year-olds (adolescents) who have paroxysmal nocturnal hemoglobinuria (PNH).
Eligibility Criteria
Inclusion Criteria: * Are 12-17 years old at the time of screening * Weigh at least 20 kg (approx. 44 lbs) * Have the diagnosis of PNH, confirmed by high-sensitivity flow cytometry (granulocyte or monocyte clone \>10%) * EITHER: * 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); OR * 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
Contact & Investigator
Apellis Clinical Trial Information Line
✉ clinicaltrials@apellis.com📞 1-833-284-6361 (833-CT Info-1)
Frequently Asked Questions
Who can join the NCT04901936 clinical trial?
This trial is open to participants of all sexes, aged 12 Years or older, up to 17 Years, studying Paroxysmal Nocturnal Hemoglobinuria (PNH). Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT04901936 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT04901936 currently recruiting?
Yes, NCT04901936 is actively recruiting participants. Contact the research team at clinicaltrials@apellis.com for enrollment information.
Where is the NCT04901936 trial being conducted?
This trial is being conducted at Atlanta, United States, Prague, Czechia, Paris, France, Ampang, Malaysia and 8 additional locations.
Who is sponsoring the NCT04901936 clinical trial?
NCT04901936 is sponsored by Apellis Pharmaceuticals, Inc.. The trial plans to enroll 12 participants.