New study tracks health crises in rare metabolic disorder

NCT ID NCT05769621

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

First seen Sep 30, 2025 · Last updated Apr 25, 2026 · Updated 25 times

Summary

This study is for people with propionic acidemia, a rare genetic condition that affects how the body breaks down certain proteins and fats. Researchers will look at medical records from about 60 participants to count how often serious metabolic events, hospital stays, and urgent care visits happen. No new treatments or drugs are given; the goal is to better understand the disease and its impact.

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.

Get updates

Get notified about this study

Our safety recommendation!

By submitting, you agree to our Terms of use

Contacts and locations

Show contact details

Study contacts

Contact

Phone: •••-•••-•••• Email: •••••@•••••

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WITHDRAWN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

Conditions

Explore the condition pages connected to this study.

PROPIONIC ACIDEMIA