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.
Similar studies
Other trials that look related to this one.
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This study tests an oral medication (MY008211A) designed to reduce red blood cell destruction in people with paroxysmal nocturnal hemoglobinuria (PNH). The trial aims to see if the drug is safe and effective in improving anemia and lowering markers of disease activity.
This trial tests an experimental RNA drug (ALN-CFB) for people with PNH who still have low red blood cell counts despite taking a C5 inhibitor. The drug aims to block a protein (complement factor B) to improve anemia.
This trial tests a new drug called lanoracopan for the blood disease PNH. It aims to see if lanoracopan can safely control PNH symptoms and improve blood counts in patients who haven't responded well to other treatments.
This study looks at how well pegcetacoplan works and how safe it is for people with two rare kidney diseases, called C3G and IC-MPGN. It will help doctors understand how the medicine helps patients in real life, outside of a strict clinical trial.
This trial tests a new pill (MY008211A) for people with paroxysmal nocturnal hemoglobinuria (PNH) who still have low red blood cell counts (anemia) even after taking anti-C5 antibody therapy. The goal is to see if this new drug can raise hemoglobin levels and improve energy.
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