A Study to Assess the Long-term Safety and Clinical Activity of mRNA-3927 in Participants Previously Enrolled in the mRNA-3927-P101 Study
Start Date
11/9/2021
Completion Date
12/4/2031
Summary
The main purpose of this study is to evaluate the long-term safety of mRNA-3927 administered to participants with propionic acidemia (PA) who have previously participated in Study mRNA-3927-P101 (NCT04159103).
Detailed Description
The study will assess long-term safety of mRNA-3927. Participants with PA who were previously enrolled and completed the end-of-treatment (EOT)/early termination (ET) visit of the mRNA-3927-P101 study will have the option to enroll into this extension study provided all eligibility criteria have been met. The study will include 2 periods: 1) Treatment Period and 2) Follow-up Period (90 days after the EOT visit). All participants will enter the study receiving mRNA-3927 at the same dose and at the same dosing interval last received in the mRNA-3927-P101 study.
Eligibility Criteria
Age Range: 1 years to No maximum
Interventions
mRNA-3927
Conditions
Locations
Ronald Reagan UCLA Medical Center
Los Angeles, California 90095
United States
University of Stanford Medical Center
Palo Alto, California 94304-1503
United States
University of Michigan Hospitals
Ann Arbor, Michigan 48109
United States
Icahn School of Medicine at Mount Sinai
New York, New York 10029
United States
Duke University Medical System (Duke Health)
Durham, North Carolina 27710
United States
The Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania 19104
United States
Texas Children's Hospital
Houston, Texas 77030
United States
Hospital For Sick Children
Toronto, Ontario M5G 1X8
Canada
AP-HM- Hôpital de La Timone
Marseille, 13005
France
Hôpital Necker - Enfants Malades
Paris, 75019
France
Fujita Health University Hospital
Toyoake-shi, Akita 470-1192
Japan
Tohoku University Hospital
Sendai, Miyagi 980-8574
Japan
Erasmus MC -Dr. Molewaterplein 40
Rotterdam, South Holland 3015 GD
Netherlands
Universitair Medisch Centrum Utrecht - PPDS
Utrecht, 3584 CX
Netherlands
King Faisal Specialist Hospital & Research Centre
Riyadh, Ar Riya 11211
Saudi Arabia
King Abdullah Children's Specialist Hospital
Riyadh, Ar Riya 14611
Saudi Arabia
Hospital Universitario Cruces
Barakaldo, Vizcaya 48903
Spain
Hospital Universitario 12 de Octubre
Madrid, 28041
Spain
Willink Biochemical Genetics Unit - Manchester
Manchester, England M13 9WL
United Kingdom
University Hospital Birmingham NHS Foundation Trust
Birmingham, West Midlands B15 2TH
United Kingdom
Birmingham Women's and Children's NHS Foundation Trust
Birmingham, West Midlands B4 6NH
United Kingdom
Great Ormond Street Hospital for Children NHS Foundation Trust
London, WC1N 3JH
United Kingdom