A Retrospective Study to Characterize Participants With Propionic Acidemia and Methylmalonic Acidemia
- Conditions
- Methylmalonic AcidemiaPropionic Acidemia
- Registration Number
- NCT05769621
- Lead Sponsor
- ModernaTX, Inc.
- Brief Summary
This is a non-interventional, global, multicenter, retrospective cohort study describing participant characteristics, clinical outcomes, and event rates in participants with propionic acidemia (PA) or Methylmalonic acidemia (MMA).
- Detailed Description
This study will abstract participants data from their medical records from approximately 2015-2025. All data will be abstracted retrospectively in 6-month increments.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 200
Not provided
Not provided
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Number of Annualized Metabolic Decompensation Events (MDEs) Up to 10 years
- Secondary Outcome Measures
Name Time Method Number of Participants with MDE Symptoms Up to 10 years MDE symptoms may include persistent vomiting, anorexia/failure to feed, lethargy, change in behavior or level of consciousness or increased seizure activity.
Number of Annualized Hospitalizations Related to PA or MMA Up to 10 years Number of Annualized Urgent Healthcare Visits Related to PA or MMA Up to 10 years Number of Participants who Received Medication During and Within ± 3 Days of an MDE Up to 10 years Medications for standard long-term and acute management of PA or MMA, which may include, but are not limited to, anesthesia, antibiotics/antifungals, anticoagulation medications, antiepileptics, biotin or cobalamin supplementation, hydration treatment, PA treatment, MMA treatment, pain medications, and transfusion related medications.
Number of Participants Requiring Dietary Management Up to 10 years Dietary management includes the following:
* Prescribed protein intake during metabolic stability and during MDEs
* Sick-day diet as available.Annualized MDE Duration Up to 10 years Annualized Duration of Hospitalizations Related to PA or MMA Up to 10 years Number of Participants with MDE as Assessed by a Pre-Specified Severity Rating Scale Up to 10 years Change from Baseline in Biomarkers Up to 10 years
Trial Locations
- Locations (26)
University of Stanford Medical Center
🇺🇸Stanford, California, United States
Johns Hopkins University
🇺🇸Lutherville, Maryland, United States
University of Michigan Hospital
🇺🇸Ann Arbor, Michigan, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Texas Children's Hospital
🇺🇸Houston, Texas, United States
Seattle Children's Hospital
🇺🇸Seattle, Washington, United States
Royal Children's Hospital Melbourne
🇦🇺Melbourne, Victoria, Australia
Stollery Children's Hospital University of Alberta
🇨🇦Edmonton, Alberta, Canada
CHRU Nancy Hôpital des Enfants
🇫🇷Vandœuvre-lès-Nancy, Meurthe-et-Moselle, France
Hôpital Necker - Enfants Malades
🇫🇷Paris Cedex 15, Paris, France
CHU de Marseille - Hôpital de la Timone
🇫🇷Marseille, France
CHU Toulouse
🇫🇷Toulouse, France
Azienda Ospedale Pediatrico Bambino Gesu - INCIPIT - PIN
🇮🇹Roma, Lazio, Italy
ASST di Monza - Azienda Ospedaliera San Gerardo
🇮🇹Monza, Monza A Brianza, Italy
Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN
🇮🇹Firenze, Toscana, Italy
Erasmus MC
🇳🇱Rotterdam, Netherlands
Hospital Universitario Marques de Valdecilla
🇪🇸Santander, Cantabria, Spain
Hospital Universitario Cruces
🇪🇸Barakaldo, Spain
Hospital Sant Joan de Deu
🇪🇸Barcelona, Spain
Hospital Universitario Raymon y Cajal
🇪🇸Madrid, Spain
Hospital Universitario 12 de Octubre
🇪🇸Madrid, Spain
Hospital Universitario Virgen del Rocio - PPDS
🇪🇸Sevilla, Spain
Hospital Universitari I Politecnic La Fe de Valencia
🇪🇸Valencia, Spain
Victoria Hospital
🇬🇧Kirkcaldy, Fife, United Kingdom
St Mary's Hospital - PPDS
🇬🇧Manchester, Lancashire, United Kingdom
Great Ormond Street Hospital
🇬🇧City of London, London, United Kingdom