The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment basivarsen (DYNE-101) for the treatment of myotonic dystrophy 1 (DM1).
The study consists of three periods: a Screening period (up to 8 weeks), Placebo-Controlled Period (48 weeks) and a Long-Term Extension Period (24 weeks). An Independent Data Monitoring Committee (IDMC) comprised of members independent and external to the Sponsor will review safety and tolerability data of this study at regular intervals.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
150
Administered by IV infusion
Administered by IV infusion
Rare Disease Research, LLC
Atlanta, Georgia, United States
RECRUITINGRoy Blunt NextGen Precision Health Institute
Columbia, Missouri, United States
RECRUITINGRare Disease Research, LLC
Hillsborough, North Carolina, United States
5 Times Sit-To-Stand (5×STS) Time
Time frame: Baseline, Week 49
Video Hand Opening Time (vHOT) [Middle Finger]
Time frame: Baseline, Week 49
Quantitative Muscle Testing (QMT) Total
Time frame: Baseline, Week 49
Clinician Global Impression of Change (CGI-C)
Time frame: Week 49
10-Meter Walk/Run Test (10-MWRT) Velocity (m/s)
Time frame: Baseline, Week 49
Patient Global Impression of Change (PGI-C)
Time frame: Week 49
DM1-ACTIV^C Total Score
Time frame: Baseline, Week 49
Myotonic Dystrophy Health Index (MDHI) Total
Time frame: Baseline, Week 49
Patient Global Impression of Severity (PGI-S)
Time frame: Baseline, Week 49
Clinician Global Impression of Severity (CGI-S)
Time frame: Baseline, Week 49
9-Hole Peg Test (9-HPT) Time
Time frame: Baseline, Week 49
Myotonic Dystrophy Health Index (MDHI) Subscale Scores
Time frame: Baseline, Week 49
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
The University of Texas Health Science Center at San Antonio
San Antonio, Texas, United States
RECRUITINGNational Hospital Organization Osaka Toneyama Medical Center
Toyonaka-Shi, Osaka, Japan
RECRUITINGYamaguchi University Hospital
Ube-Shi, Yamaguchi, Japan
RECRUITINGMaximum Observed Plasma Drug Concentration (Cmax) of DYNE-101
Time frame: Pre-dose, and at multiple time points up to Week 73
Time to Maximum Concentration (tmax) of DYNE-101
Time frame: Pre-dose, and at multiple time points up to Week 73
Area Under the Concentration-Time Curve (AUC) from Hour 0 to the Last Measurable Concentration (AUC0-tlast) of DYNE-101
Time frame: Pre-dose, and at multiple time points up to Week 73
Area Under the Concentration-Time Curve Extrapolated to Infinity (AUC0-∞) of DYNE-101
Time frame: Pre-dose, and at multiple time points up to Week 73
Apparent Terminal Elimination Rate Constant (λZ) of DYNE-101
Time frame: Pre-dose, and at multiple time points up to Week 73
Apparent Terminal Elimination Half-Life (t½) of DYNE-101
Time frame: Pre-dose, and at multiple time points up to Week 73
Plasma clearance (CL) of DYNE-101
Time frame: Pre-dose, and at multiple time points up to Week 73
Volume of Distribution at the Terminal Phase (Vz), if Appropriate of DYNE-101
Time frame: Pre-dose, and at multiple time points up to Week 73
Volume of Distribution at Steady State (Vss), if Appropriate of DYNE-101
Time frame: Pre-dose, and at multiple time points up to Week 73
Number of Participants With Antidrug Antibodies (ADAs)
Time frame: Up to Week 73