New drug livmarli tracked for Long-Term safety in kids with rare liver disorders
NCT ID NCT07290257
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study follows 230 children with Alagille syndrome or progressive familial intrahepatic cholestasis (PFIC) who are taking Livmarli, a drug to reduce bile buildup and itching. Researchers will monitor side effects, liver function, and long-term outcomes like need for transplant. The goal is to see if Livmarli is safe and effective over years of use.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Livmarli (maralixibat)
What this could lead to
If successful, this could confirm Livmarli as a safe long-term treatment to manage symptoms and slow liver damage in these rare childhood diseases.
What could go wrong
This is a low-intervention safety study, not a cure trial. It may show side effects or limited benefit, and results may not apply to all patients.
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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2nd Paediatric Clinic P &A Kyriakou Children's Hospital
NOT_YET_RECRUITINGAthens, 11527, Greece
Contact
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AO Ospedale PAPA GIOVANNI XXIII
RECRUITINGBergamo, Lombardy, 24126, Italy
Contact Email: •••••@•••••
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Bicetre University Hospital
RECRUITINGLe Kremlin-Bicêtre, Île-de-France Region, 94270, France
Contact Phone: •••-•••-•••• Email: •••••@•••••
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CHU de Toulouse - Hôpital des Enfants
RECRUITINGToulouse, Occitanie, 31049, France
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Charite Berlin
RECRUITINGBerlin, 13353, Germany
Contact Email: •••••@•••••
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Children's University Hospital Essen
RECRUITINGEssen, 45149, Germany
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Cliniques Universitaires Saint Luc (UCLouvain)
RECRUITINGBrussels, 1200, Belgium
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Hospices Civils de Lyon - Hopital Femme Mère Enfant
RECRUITINGBron, Auvergne-Rhône-Alpes, 69500, France
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Hospital Universitairo Vall D'Hebron
RECRUITINGBarcelona, Catalonia, 08035, Spain
Contact Email: •••••@•••••
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Hospital Universitario La Paz
RECRUITINGMadrid, 28046, Spain
Contact Email: •••••@•••••
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Istituto mediterraneo trapianti - ISMETT
RECRUITINGPalermo, Sicily, 90127, Italy
Contact Email: •••••@•••••
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Unidade Local de Saude de Coimbra Paediatric Department Avenida Afonso Romăo
NOT_YET_RECRUITINGCoimbra, 3000-602, Portugal
Contact
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University Hospital Gent (UZ Gent)
RECRUITINGGhent, 9000, Belgium
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University Hospital Hamburg-Eppendorf
RECRUITINGHamburg, 20246, Germany
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University Medical Center Groningen (UMCG)
RECRUITINGGroningen, 9713GZ, Netherlands
Contact Phone: •••-•••-•••• Email: •••••@•••••