Researchers track rare disease PMM2-CDG to unlock clues for future treatments

NCT ID NCT03173300

First seen Jun 27, 2026 · Last updated Jul 09, 2026 · Updated 2 times

Summary

This study is gathering medical information from 120 people with PMM2-CDG, a rare genetic disorder. Researchers will track growth, organ function, and development over time. The goal is to better understand the disease and help design future treatments. No new drugs are being tested.

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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

Locations

  • Centro Hospitalar do Porto

    Porto, Portugal

  • Children's Hospital of Philadelphia (CHOP)

    Philadelphia, Pennsylvania, 19104, United States

  • General University Hospital in Prague

    Prague, Czechia

  • Hospital Sant Joan de Déu

    Barcelona, Spain

  • Mayo Clinic College of Medicine

    Rochester, Minnesota, 55905, United States

  • Mother and Child Institute (Instytut Matki i Dziecka)

    Warsaw, Poland

  • Necker Enfants-Malades Hospital

    Paris, France

  • Radboud University Nejmegen Medical Center

    Nijmegen, Netherlands

  • Seattle Children's Hospital

    Seattle, Washington, 98105, United States

  • University Hospital Leuven

    Leuven, Belgium, Belgium

  • University Hospital of Catania

    Catania, Italy

More trials for these conditions

Other studies related to the condition(s) this trial covers.