A Retrospective Study to Characterize Participants With Propionic Acidemia

Part of paid clinical trials in Stanford, California.

Sponsor
ModernaTX, Inc.
Study ID
NCT05769621
Status
Recruiting
Apply to this trial

Conditions

  • Propionic Acidemia

Eligibility Criteria

Sex
ALL
Age
2 Years - N/A
Healthy Volunteers
Not accepted

Study Details

This is a non-interventional, observational, global, multicenter, study describing participant characteristics, clinical outcomes, and event rates in participants with propionic acidemia (PA).

Key Dates

Start date
Jun 16, 2023
Status verified
Feb 2026
Primary completion
Sep 30, 2026
Completion
Sep 30, 2026

Study Design

Enrollment
60 participants (estimated)

Arms

  • Arm: PA Participants
    Participants with PA who meet all eligibility criteria for medical record abstraction.

Primary Outcome Measure

Number of Annualized Clinical Event Committee (CEC)-adjudicated Metabolic Decompensation Events (MDEs) [ Time Frame: Up to 10 years ]

Central Contacts

Locations (6)

FacilityCityStateZIPSite coordinators
University of Stanford Medical CenterStanfordCalifornia94305-2200-
Johns Hopkins UniversityBaltimoreMaryland21287-0010-
University of Michigan HospitalAnn ArborMichigan48109-5000-
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104-
Texas Children's HospitalHoustonTexas77030-2614-
Seattle Children's HospitalSeattleWashington98105-3901-

Find similar trials in Stanford, CA

By research site
University of Stanford Medical Center· Stanford, CAJohns Hopkins University· Baltimore, MDUniversity of Michigan Hospital· Ann Arbor, MIChildren's Hospital of Philadelphia· Philadelphia, PATexas Children's Hospital· Houston, TXSeattle Children's Hospital· Seattle, WA

Related Studies