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 Read more

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.

alpha 1-antitrypsin deficiency Digestive System Diseases

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • AP-HP - Kremlin Bicêtre

    Le Kremlin-Bicêtre, France

  • AP-HP Hôpital Necker

    Paris, France

  • CH Saint Nazaire

    Saint-Nazaire, France

  • CHG Le HAVRE

    Le Havre, France

  • CHU Estaing

    Clermont-Ferrand, France

  • CHU d'Amiens - Hopital Nord

    Amiens, France

  • CHU de BESANCON

    Besançon, France

  • Centre de Pédiatrie Gatien de Clocheville

    Tours, France

  • Hopital Hautepierre

    Strasbourg, France

  • Hopital de la Timone

    Marseille, France

  • Hôpital Anne de Bretagne

    Rennes, France

  • Hôpital Brabois Enfants

    Nancy, France

  • Hôpital Charles Nicolle

    Rouen, France

  • Hôpital Couple Enfant

    La Tronche, France

  • Hôpital Femme Mère Enfant de Lyon

    Bron, France

  • Hôpital Jeanne de Flandre

    Lille, France

  • Hôpital Mère Enfant

    Nantes, France

  • Hôpital Nord

    Saint-Etienne, France

  • Hôpital Pellegrin

    Bordeaux, France

  • Hôpital des Enfants

    Toulouse, France