Iptacopan study for children with PNH
Treatments studied
Part of Blood & lymphatic clinical trials.
This trial tests an oral medication, iptacopan, for children with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood condition. It aims to see how well the drug works in kids who have not tried anti-C5 therapy or who are switching from it.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Child is 2 to under 18 years old
- Diagnosed with PNH confirmed by a special blood test showing at least 10% affected red and white blood cells
- Weighs at least 35 kg (about 77 pounds) for the first group
- If taking anti-C5 therapy: stable dose for at least 6 months before joining
- If not taking anti-C5 therapy: low hemoglobin (less than 10 g/dL) and high LDH (a marker of red blood cell breakdown) at screening
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.
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.
This study looks at how well iptacopan works in everyday medical practice for people with paroxysmal nocturnal hemoglobinuria (PNH) being treated in Italy. It gathers information from routine care to understand real-world effectiveness.
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 program gives access to the drug iptacopan for patients with serious conditions who have no other options. It is not a standard clinical trial but a special way to get the drug when all approved treatments have failed or are not suitable.
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 study looks at how well iptacopan works for people with PNH (a condition where red blood cells break down too quickly). It is for adults who have never used certain treatments or who have been on other similar medicines.
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