Can genes predict liver risk in kids with rare disorder?
NCT ID NCT01862211
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study looks at genetic differences in children with alpha-1 antitrypsin deficiency, a rare inherited condition that can cause liver disease. Researchers will compare gene patterns between children who develop liver problems and those who do not, using blood samples from about 400 participants. The goal is to find genetic markers that could help predict which children are at highest risk, allowing for earlier and more tailored care.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
What this could lead to
If successful, this study could help identify which children with alpha-1 antitrypsin deficiency are at higher risk for severe liver disease, enabling earlier monitoring and personalized care.
What could go wrong
This is an observational genetic study, not a treatment trial. It may not find clear genetic markers, and any findings would need further validation before changing clinical practice.
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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Locations
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AP-HP - Kremlin Bicêtre
Le Kremlin-Bicêtre, France
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AP-HP Hôpital Necker
Paris, France
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CH Saint Nazaire
Saint-Nazaire, France
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CHG Le HAVRE
Le Havre, France
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CHU Estaing
Clermont-Ferrand, France
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CHU d'Amiens - Hopital Nord
Amiens, France
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CHU de BESANCON
Besançon, France
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Centre de Pédiatrie Gatien de Clocheville
Tours, France
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Hopital Hautepierre
Strasbourg, France
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Hopital de la Timone
Marseille, France
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Hôpital Anne de Bretagne
Rennes, France
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Hôpital Brabois Enfants
Nancy, France
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Hôpital Charles Nicolle
Rouen, France
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Hôpital Couple Enfant
La Tronche, France
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Hôpital Femme Mère Enfant de Lyon
Bron, France
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Hôpital Jeanne de Flandre
Lille, France
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Hôpital Mère Enfant
Nantes, France
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Hôpital Nord
Saint-Etienne, France
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Hôpital Pellegrin
Bordeaux, France
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Hôpital des Enfants
Toulouse, France