Study of pegcetacoplan for kids with PNH blood problems
Treatments studied
Part of Blood & lymphatic clinical trials.
This trial tests pegcetacoplan in children (ages 12–17) with paroxysmal nocturnal hemoglobinuria (PNH), a condition that can cause ongoing blood cell breakdown. The goal is to see if the medicine can improve anemia related to PNH.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are 12–17 years old at the screening visit
- You weigh at least 20 kg (about 44 lbs)
- You have PNH confirmed by a specialized blood test (flow cytometry) showing the PNH cell change
- Your lab results show anemia: low hemoglobin (Hb below normal) plus signs of active blood breakdown or ongoing anemia
- Your blood counts meet minimums: platelets above 75,000 and neutrophils above 1,000
- You are not pregnant or breastfeeding
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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