New drug hopes to tame rare Gut-Heart disease in toddlers
NCT ID NCT07142343
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tests a drug called pozelimab in children aged 1 to 5 who have CHAPLE disease, a very rare genetic condition that causes severe stomach and heart problems. The main goal is to see if the drug is safe and how it affects the body. Only 5 children will take part, and the drug works by blocking a part of the immune system to control symptoms.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- pozelimab (a lab-made antibody that blocks a part of the immune system called C5)
- What this could lead to
- If it works, this could provide a treatment option for young children with CHAPLE disease, helping control life-threatening gastrointestinal and cardiovascular symptoms.
- What could go wrong
- This is a very small, early-phase study with only 5 children, so results may not apply to everyone. The drug blocks part of the immune system, which could increase infection risk.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Marmara University Hospital
RECRUITINGIstanbul, 10 34899, Turkey (Türkiye)