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.
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