This is a phase 1/2a double-blinded, placebo-controlled, dose-escalating study to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of single and multiple ascending doses of SRP-1003 compared to placebo in male and female participants with type 1 myotonic dystrophy (DM1). Participants who have provided written informed consent and met all protocol eligibility requirements will be randomized to receive single (Part 1) or multiple (Part 2) doses of SRP-1003 or placebo.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
78
SRP-1003 by IV infusion
0.9% sodium chloride (NaCl) calculated volume to match active treatment by IV infusion
SRP-1003 by SC injection(s)
Number of Participants with Treatment-emergent Adverse Events Over Time Through End of Study (EOS)
Time frame: Single-dose phase (Part 1): Up to Day 90 (EOS); multiple-dose phase (Part 2): Up to Day 180 (EOS)
PK of SRP-1003: Maximum Observed Plasma Concentration (Cmax)
Time frame: Single-dose phase (Part 1): Up 24 hours post-dose; multiple-dose phase (Part 2): Through 24 hours post first and second dose
PK of SRP-1003: Area Under the Plasma Concentration Versus Time Curve from Zero to 24 Hours (AUC0-24)
Time frame: Single-dose phase (Part 1): Up 24 hours post-dose; multiple-dose phase (Part 2): Through 24 hours post first and second dose
PK of SRP-1003: Area Under the Plasma Concentration Versus Time Curve from Zero to the Last Quantifiable Plasma Concentration (AUClast)
Time frame: Single-dose phase (Part 1): Up 24 hours post-dose; multiple-dose phase (Part 2): Through 24 hours post first and second dose
PK of SRP-1003: Area Under the Plasma Concentration Versus Time Curve from Zero to Infinity (AUCinf)
Time frame: Single-dose phase (Part 1): Up 24 hours post-dose; multiple-dose phase (Part 2): Through 24 hours post first and second dose
Change from Baseline at Day 120 for Video Hand Opening Time (vHOT)
Time frame: (Part 2): Baseline, Day 120
Change from Baseline Over Time for the Timed Up and Go Test (TUG) Assessment
Time frame: (Part 2): Baseline through EOS (up to 180 days)
Change from Baseline Over Time for the 10-Meter Walk/Run Test (10MWT) Assessment
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0.9% NaCl calculated volume to match active treatment by SC injection(s)
Liverpool Hospital
Liverpool, New South Wales, Australia
RECRUITINGSunshine Coast University Hospital
Birtinya, Queensland, Australia
RECRUITINGRoyal Brisbane And Women's Hospital
Herston, Queensland, Australia
RECRUITINGAlfred Health
Melbourne, Victoria, Australia
RECRUITINGNucleus Network Brisbane Clinic
Herston, Australia
RECRUITINGMater Hospital Brisbane
South Brisbane, Australia
RECRUITINGUniversitair Ziekenhuis Gent
Ghent, Belgium
RECRUITINGUZ Leuven
Leuven, Belgium
RECRUITINGUniversity Of Calgary
Calgary, Canada
RECRUITINGUniversity Of Alberta
Edmonton, Canada
RECRUITING...and 25 more locations
Time frame: (Part 2): Baseline through EOS (up to 180 days)
Change from Baseline Over Time for the Hand-held Quantitative Dynamometry Assessment
Time frame: (Part 2): Baseline through EOS (up to 180 days)
Change from Baseline Over Time for the Video Hand Opening Time (vHOT) Assessment
Time frame: (Part 2): Baseline through EOS (up to 180 days)
Change from Baseline Over Time for the Myotonic Dystrophy Type 1 Activity and Participation Scale (DM1-Activ-C) Assessment
Time frame: (Part 2): Baseline through EOS (up to 180 days)
Change from Baseline Over Time for the Myotonic Dystrophy Health Index (MDHI) Assessment
Time frame: (Part 2): Baseline through EOS (up to 180 days)