No treatment, just data: new study tracks rare metabolic disease

NCT ID NCT05769621

Recruiting now Knowledge-focused Sponsor: ModernaTX, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study is not testing any new drug or treatment. Instead, researchers will look back at medical records of 60 people with propionic acidemia, a rare genetic disorder that affects how the body breaks down certain proteins and fats. The goal is to better understand the disease, including how often patients have serious metabolic episodes and need hospital care. This information could help design future studies and improve care.

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Conditions

The condition(s) this trial relates to.

propionic acidemia

As listed by the trial registrant

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

PROPIONIC ACIDEMIA

Contacts and locations

Locations

ASST di Monza - Azienda Ospedaliera San Gerardo
ACTIVE_NOT_RECRUITING

Monza, Monza A Brianza, 20900, Italy
Azienda Ospedaliera Universitaria Federico II
RECRUITING

Naples, Campania, 80131, Italy
Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN
RECRUITING

Florence, Tuscany, 50139, Italy
CHRU Nancy Hôpital des Enfants
WITHDRAWN

Vandœuvre-lès-Nancy, Meurthe-et-Moselle, 54511, France
CHU Toulouse
RECRUITING

Toulouse, Haute-Garonne, 31300, France
CHU de Marseille - Hôpital de la Timone
RECRUITING

Marseille, 13386, France
Children's Hospital of Philadelphia
RECRUITING

Philadelphia, Pennsylvania, 19104, United States
Erasmus MC
NOT_YET_RECRUITING

Rotterdam, South Holland, 3015 GD, Netherlands
Fujita Health University Hospital
NOT_YET_RECRUITING

Toyoake, Aichi-ken, 470-1192, Japan
Great Ormond Street Hospital
ACTIVE_NOT_RECRUITING

City of London, London, United Kingdom
Hospital Sant Joan de Deu
RECRUITING

Esplugues de Llobregat, Barcelona, 8950, Spain
Hospital Universitari I Politecnic La Fe de Valencia
RECRUITING

Valencia, 46026, Spain
Hospital Universitario 12 de Octubre
ACTIVE_NOT_RECRUITING

Madrid, 28041, Spain
Hospital Universitario Cruces
RECRUITING

Barakaldo, 48903, Spain
Hospital Universitario Marques de Valdecilla
RECRUITING

Santander, Cantabria, 39008, Spain
Hospital Universitario Raymon y Cajal
RECRUITING

Madrid, 28034, Spain
Hospital Universitario Virgen del Rocio - PPDS
RECRUITING

Seville, 41013, Spain
Hôpital Necker - Enfants Malades
RECRUITING

Paris, Paris, 75743, France
Johns Hopkins University
RECRUITING

Baltimore, Maryland, 21287-0010, United States
Royal Children's Hospital Melbourne
WITHDRAWN

Parkville, Victoria, 3052, Australia
Seattle Children's Hospital
RECRUITING

Seattle, Washington, 98105-3901, United States
St Mary's Hospital - PPDS
ACTIVE_NOT_RECRUITING

Manchester, Lancashire, United Kingdom
Stollery Children's Hospital University of Alberta
WITHDRAWN

Edmonton, Alberta, T6G 2B7, Canada
Texas Children's Hospital
TERMINATED

Houston, Texas, 77030-2614, United States
The Jikei University School of Medicine
NOT_YET_RECRUITING

Minato, Tokyo, 105-8471, Japan
University Hospital Birmingham
RECRUITING

Birmingham, B29 6JD, United Kingdom
University of Michigan Hospital
ACTIVE_NOT_RECRUITING

Ann Arbor, Michigan, 48109-5000, United States
University of Stanford Medical Center
NOT_YET_RECRUITING

Stanford, California, 94305-2200, United States
Victoria Hospital
WITHDRAWN

Kirkcaldy, Fife, KY2 5AH, United Kingdom

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