Actively Recruiting
A Retrospective Cohort Study to Characterize Propionic Acidemia Patients
Led by ModernaTX, Inc. · Updated on 2026-02-05
60
Participants Needed
29
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
This research aims to describe the characteristics, clinical outcomes, and event rates in participants with propionic acidemia (PA), a rare metabolic disorder. It is a non-interventional, observational, global, multicenter study that collects data retrospectively from medical records to better understand PA over time. Data will be gathered by reviewing medical records from various study sites, including hospitals, clinics, and academic centers. Participants included will be those diagnosed with PA confirmed by genetic testing, with records documenting specific metabolic events. The study will cover up to 10 years of medical history and events. Participants' involvement consists of allowing researchers to review their medical records to collect information about metabolic decompensation events, hospitalizations, and urgent healthcare visits related to PA. Researchers will analyze the number and severity of these events, along with hospitalization data, to understand PA's clinical impact. The study does not involve direct treatment or interventions and focuses on data collection and analysis.
CONDITIONS
Brief Title
A Retrospective Study to Characterize Participants With Propionic Acidemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Confirmed diagnosis of propionic acidemia by molecular genetic testing of PCCA and/or PCCB mutations
- Participant provided informed consent and assent, if applicable
- Medical records with sufficient data for event adjudication available from birth or January 1, 2015, whichever is later
- Experienced at least one metabolic decompensation event in the 24 months before the index date or at least three such events within any 12-month period since birth or January 1, 2015
- Confirmed age of disease onset (early or late, with early defined as neonatal period)
- Participants meeting censoring criteria may enroll if they have at least 2 years of data before censoring and meet all inclusion criteria based on available data
You will not qualify if you...
- Participation in any clinical study of an investigational agent
- Received gene therapy treatment
- Confirmed organ transplantation
- Investigator unable to obtain relevant clinical information
- Death
- Prior or ongoing participation in clinical study mRNA-3927-P101 (NCT04159103) or mRNA-3927-P101-EXT (NCT05130437)
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person or remote)
Duration - Up to 10 years of historical data
Participants' medical records are retrospectively reviewed to collect data relevant to propionic acidemia and clinical events.
No visits; data collection from existing medical records
Trial Site Locations
Total: 29 locations
1
University of Stanford Medical Center
Stanford, California, United States, 94305-2200
Not Yet Recruiting
2
Johns Hopkins University
Baltimore, Maryland, United States, 21287-0010
Actively Recruiting
3
University of Michigan Hospital
Ann Arbor, Michigan, United States, 48109-5000
Active, Not Recruiting
4
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
5
Texas Children's Hospital
Houston, Texas, United States, 77030-2614
Terminated
6
Seattle Children's Hospital
Seattle, Washington, United States, 98105-3901
Actively Recruiting
7
Royal Children's Hospital Melbourne
Parkville, Victoria, Australia, 3052
Withdrawn
8
Stollery Children's Hospital University of Alberta
Edmonton, Alberta, Canada, T6G 2B7
Withdrawn
9
CHU Toulouse
Toulouse, Haute-Garonne, France, 31300
Actively Recruiting
10
CHRU Nancy Hôpital des Enfants
Vandœuvre-lès-Nancy, Meurthe-et-Moselle, France, 54511
Withdrawn
11
Hôpital Necker - Enfants Malades
Paris, Paris, France, 75743
Actively Recruiting
12
CHU de Marseille - Hôpital de la Timone
Marseille, France, 13386
Actively Recruiting
13
Azienda Ospedaliera Universitaria Federico II
Naples, Campania, Italy, 80131
Actively Recruiting
14
ASST di Monza - Azienda Ospedaliera San Gerardo
Monza, Monza A Brianza, Italy, 20900
Active, Not Recruiting
15
Azienda Ospedaliero Universitaria A Meyer - INCIPIT - PIN
Florence, Tuscany, Italy, 50139
Actively Recruiting
16
Fujita Health University Hospital
Toyoake, Aichi-ken, Japan, 470-1192
Not Yet Recruiting
17
The Jikei University School of Medicine
Minato, Tokyo, Japan, 105-8471
Not Yet Recruiting
18
Erasmus MC
Rotterdam, South Holland, Netherlands, 3015 GD
Not Yet Recruiting
19
Hospital Sant Joan de Deu
Esplugues de Llobregat, Barcelona, Spain, 8950
Actively Recruiting
20
Hospital Universitario Marques de Valdecilla
Santander, Cantabria, Spain, 39008
Actively Recruiting
21
Hospital Universitario Cruces
Barakaldo, Spain, 48903
Actively Recruiting
22
Hospital Universitario Raymon y Cajal
Madrid, Spain, 28034
Actively Recruiting
23
Hospital Universitario 12 de Octubre
Madrid, Spain, 28041
Active, Not Recruiting
24
Hospital Universitario Virgen del Rocio - PPDS
Seville, Spain, 41013
Actively Recruiting
25
Hospital Universitari I Politecnic La Fe de Valencia
Valencia, Spain, 46026
Actively Recruiting
26
Victoria Hospital
Kirkcaldy, Fife, United Kingdom, KY2 5AH
Withdrawn
27
St Mary's Hospital - PPDS
Manchester, Lancashire, United Kingdom
Active, Not Recruiting
28
Great Ormond Street Hospital
City of London, London, United Kingdom
Active, Not Recruiting
29
University Hospital Birmingham
Birmingham, United Kingdom, B29 6JD
Actively Recruiting
Research Team
M
Moderna WeCare Team
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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