Gene therapy trial hopes to tackle muscle disease
NCT ID NCT06844214
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a gene therapy called SAR446268 for people aged 10 to 55 with myotonic dystrophy type 1. The therapy is given once through an IV and aims to reduce harmful DMPK RNA and improve muscle function. The trial has two parts: first, finding the safest dose in a small group, then testing that dose in more participants. It will last about two years per person.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
SAR446268 (gene therapy given by IV infusion)
What this could lead to
If successful, this could lead to a treatment that slows or improves muscle function in people with myotonic dystrophy type 1.
What could go wrong
This is an early phase 1/2 trial with only 32 participants, so safety and effectiveness are not yet proven. Gene therapies can have unexpected side effects, and the long-term risks are unknown.
Disclaimer
Read more
Show less
This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for MYOTONIC DYSTROPHY TYPE 1 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
-
Columbia University Medical Center - Neurological Institute, 710 W. 168th, 2nd floor, suite 204 - Site Number : 8400003
RECRUITINGNew York, New York, 10032, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Hospital Italiano de Buenos Aires, Juan Domingo Peron 4190 - Site Number: 0320001
RECRUITINGBuenos Aires, 1181, Argentina
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
-
Investigational Site Number : 0360001
RECRUITINGBrisbane, Queensland, 4029, Australia
-
Investigational Site Number : 3760002
RECRUITINGRamat Gan, 5262100, Israel
-
Investigational Site Number : 8260002
RECRUITINGNewcastle upon Tyne, NE7 7DN, United Kingdom
-
The Montreal Neurological Institute and Hospital, 3801 rue University - Site Number: 1240001
RECRUITINGMontreal, Quebec, H3A 2B4, Canada
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
-
University of Florida, 2004 Mowry Road - Site Number: 8400005
RECRUITINGGainesville, Florida, 32601, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
University of South Florida - Neuromuscular Research, 13330 USF Laurel Drive - Site Number: 8400001
RECRUITINGTampa, Florida, 33612, United States
Contact
Contact Email: •••••@•••••
-
Virginia Commonwealth University Medical Center- Site Number : 8400006
RECRUITINGRichmond, Virginia, 23219, United States