Can a daily pill help kids with rare liver disease avoid transplant?
NCT ID NCT06850038
First seen Jun 27, 2026 · Last updated Aug 04, 2026 · Updated 3 times
Summary
This study follows 30 people with Alagille syndrome who are taking odevixibat (Bylvay) in their daily lives. The goal is to see if the drug helps them avoid serious surgeries like liver transplant or bile duct repair over the long term. Researchers will also track weight, height, and safety. Because it's an observational registry, it reflects real-world use but cannot prove the drug causes the outcomes.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- odevixibat (Bylvay)
- What this could lead to
- If this works, it could show that odevixibat helps children with Alagille syndrome avoid or delay the need for liver transplant or bile duct surgery.
- What could go wrong
- This is a small, observational registry (30 people) with no placebo group, so results may not prove the drug works better than no treatment. It also only follows people already taking the drug, which can bias outcomes.
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
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Study contacts
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Contact
Email: •••••@•••••
Locations
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Children's Hospital Los Angeles
RECRUITINGLos Angeles, California, 90027, United States
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Children's Medical Center Of Dallas
RECRUITINGDallas, Texas, 75246, United States
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Columbia University Medical Center - Morgan Stanley Children's Hospital of NewYork - Presbyterian (MS-CHONY)
RECRUITINGNew York, New York, 10032, United States
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Le Bonheur Children's Hospital
RECRUITINGMemphis, Tennessee, 38105, United States
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NYU Langone - NYU Grossman School of Medicine
RECRUITINGNew York, New York, 10016, United States
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Primary Children's Hospital (PCH) - Intermountain Healthcare/University of Utah
NOT_YET_RECRUITINGSalt Lake City, Utah, 84113, United States
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Proactive El Paso,LLC
RECRUITINGEl Paso, Texas, 79902, United States
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SSM Health Cardinal Glennon Children's Hospital
RECRUITINGSt Louis, Missouri, 63104, United States
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The Children's Mercy Hospital
RECRUITINGKansas City, Missouri, 64108, United States
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UCSF Benioff Children's Hospital
RECRUITINGSan Francisco, California, 94158, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- Can a common cholesterol drug shrink skin lumps in kids with rare liver disease?
- New drug livmarli tracked for Long-Term safety in kids with rare liver disorders
- Liver drug maralixibat tested for Long-Term safety in rare disease patients
- New hope for kids with rare liver disease: long-term drug study launched