A PHASE 1/PHASE 2 OPEN-LABEL SINGLE ARM STUDY WITH DOSE ESCALATION (PART A), AND DOSE EXPANSION (PART B) PARTS TO EVALUATE THE SAFETY, TOLERABILITY, AND EFFICACY OF SAR446268, AN ADENO-ASSOCIATED VIRAL VECTOR-MEDIATED GENE THERAPY IN PARTICIPANTS 10 TO 50 YEARS OLD WITH NON-CONGENITAL MYOTONIC DYSTROPHY TYPE 1

Registry ID

DFI17808

Source registry

HEALTH_CANADA

Status

Ongoing

Phase

PHASE2

Study type

INTERVENTIONAL

Sponsor

SANOFI-AVENTIS CANADA INC

Start date

2025-10-30

Last update

2025-07-11

Conditions

• CONGENITAL MYOTONIC DYSTROPHY

Related clinical trials

• A 52-WEEK, OPEN-LABEL STUDY TO EVALUATE THE LONG-TERM SAFETY AND EFFICACY OF TIDEGLUSIB FOR THE TREATMENT OF CONGENITAL DM1 (REACH CDM X)

View on source registry