New mRNA treatment for rare metabolic disease moves to Long-Term safety check
NCT ID NCT05130437
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study looks at the long-term safety of an experimental medicine called mRNA-3927 for people with propionic acidemia, a rare genetic disorder that prevents the body from breaking down certain proteins and fats. About 50 participants who were in an earlier study will continue receiving the treatment. The goal is to see if it remains safe and helps control the disease over time.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for PROPIONIC ACIDEMIA are added.
By submitting, you agree to our Terms of use
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
-
AP-HM- Hôpital de La Timone
NOT_YET_RECRUITINGMarseille, 13005, France
-
Birmingham Women's and Children's NHS Foundation Trust
COMPLETEDBirmingham, West Midlands, B4 6NH, United Kingdom
-
Duke University Medical System (Duke Health)
RECRUITINGDurham, North Carolina, 27710, United States
-
Erasmus MC -Dr. Molewaterplein 40
NOT_YET_RECRUITINGRotterdam, South Holland, 3015 GD, Netherlands
-
Fujita Health University Hospital
RECRUITINGToyoake-shi, Akita, 470-1192, Japan
-
Great Ormond Street Hospital for Children NHS Foundation Trust
RECRUITINGLondon, WC1N 3JH, United Kingdom
-
Hospital For Sick Children
RECRUITINGToronto, Ontario, M5G 1X8, Canada
-
Hospital Universitario 12 de Octubre
NOT_YET_RECRUITINGMadrid, 28041, Spain
-
Hospital Universitario Cruces
NOT_YET_RECRUITINGBarakaldo, Vizcaya, 48903, Spain
-
Hôpital Necker - Enfants Malades
NOT_YET_RECRUITINGParis, 75019, France
-
Icahn School of Medicine at Mount Sinai
RECRUITINGNew York, New York, 10029, United States
-
King Abdullah Children's Specialist Hospital
NOT_YET_RECRUITINGRiyadh, Ar Riya, 14611, Saudi Arabia
-
King Faisal Specialist Hospital & Research Centre
NOT_YET_RECRUITINGRiyadh, Ar Riya, 11211, Saudi Arabia
-
Ronald Reagan UCLA Medical Center
NOT_YET_RECRUITINGLos Angeles, California, 90095, United States
-
Texas Children's Hospital
RECRUITINGHouston, Texas, 77030, United States
-
The Children's Hospital of Philadelphia (CHOP)
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
-
Tohoku University Hospital
RECRUITINGSendai, Miyagi, 980-8574, Japan
-
Universitair Medisch Centrum Utrecht - PPDS
NOT_YET_RECRUITINGUtrecht, 3584 CX, Netherlands
-
University Hospital Birmingham NHS Foundation Trust
RECRUITINGBirmingham, West Midlands, B15 2TH, United Kingdom
-
University of Michigan Hospitals
RECRUITINGAnn Arbor, Michigan, 48109, United States
-
University of Stanford Medical Center
RECRUITINGPalo Alto, California, 94304-1503, United States
-
Willink Biochemical Genetics Unit - Manchester
RECRUITINGManchester, England, M13 9WL, United Kingdom
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Newborn screening study aims to catch rare diseases at birth
- Experimental gene therapy targets rare metabolic disease in toddlers
- Medical records reveal clues to rare genetic disease in amish and mennonite populations
- Scientists launch Largest-Ever study to unravel rare metabolic disease
- MRNA therapy takes on rare metabolic disease
- No treatment, just data: new study tracks rare metabolic disease