Clinical Research Alert: Phase 1 Study of Gene Therapy in Individuals with Myotonic Dystrophy Type 1

Researchers at Sanofi are seeking individuals with non-congenital myotonic dystrophy type 1 (DM1) to participate in a phase 1/2 clinical trial to evaluate the safety of an investigational gene therapy (SAR446268) to treat DM1. DM1 is caused by a defect in the DMPK gene that leads to the production of abnormally long DMPK RNA transcripts, which interfere with protein synthesis and disrupt normal cellular functions. SAR446268 uses an adeno-associated viral (AAV) vector to deliver specifically engineered microRNA (miRNA) molecules into muscle cells in order to target and suppress the toxic DMPK RNA responsible for causing DM1. The current study will examine the safety and tolerability of SAR446268 treatment and its effects on DMPK RNA levels in people with the disease.

The study

This is a phase 1/2 open-label study with dose escalation (part A) and dose expansion (part B). This means that all participants will receive SAR446268 treatment. In part A, participants will receive different doses to determine the optimal therapeutic dose. In part B, additional participants will receive the optimal therapeutic dose as determined by part A. The total duration of the study for participants will be 104 weeks. This will include 22 doctor visits scheduled over two years, with 15 of the visits occurring in the first 3 months.

The drug will be administered by intravenous (in the vein) infusion. The effects will be evaluated using a number of tests and procedures including but not limited to: vHOT (video hand opening time), 10MWRT (10m Walk Run Test), dynamometer, needle biopsy, vector shedding, and routine safety labs and tests (blood, urine, ultrasound of heart, EKG and Holter monitor).

Study criteria

To be eligible for part A of the research study, individuals must meet the following inclusion criteria:

• Must be 18 to 50 years of age, inclusive
• Have non-congenital onset DM1
• Have a pacemaker and/or implantable cardioverter-defibrillator (ICD)

Individuals may not be eligible to participate if they are affected by another condition or receiving another treatment that might interfere with the ability to undergo safe testing.

Please visit this link for the full listing of inclusion and exclusion criteria.

Interested in participating?

Travel, food, and lodging support is available for eligible participants.

To learn more about the study or inquire about participation, contact the closest participating hospital using Clinical Trials.gov or Sanofi Studies.