Can a daily pill help kids with rare liver disease avoid transplant?
NCT ID NCT06850038
First seen Jun 27, 2026 · Last updated Jun 27, 2026
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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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
Phone: •••-•••-•••• 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
NOT_YET_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