This is a phase 1/2a double-blinded, placebo-controlled, dose-escalating study to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of single and multiple ascending doses of ARO-DM1 compared to placebo in male and female subjects 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 ARO-DM1 or placebo.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
78
ARO-DM1 by intravenous (IV) infusion
0.9% NaCl calculated volume to match active treatment by IV infusion
ARO-DM1 by subcutaneous (SC) injection(s)
Research Site
Liverpool, New South Wales, Australia
RECRUITINGResearch Site
Birtinya, Queensland, Australia
RECRUITINGResearch Site
Number of Participants with Treatment -Emergent Adverse Events (TEAEs) 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)
Pharmacokinetics (PK) of ARO-DM1: 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 ARO-DM1: 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 ARO-DM1: 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 ARO-DM1: 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
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
0.9% NaCl calculated volume to match active treatment by SC injection(s)
Herston, Queensland, Australia
Research Site
Melbourne, Victoria, Australia
RECRUITINGResearch Site
Christchurch, New Zealand
RECRUITINGResearch Site
Taichung, Taiwan
RECRUITINGResearch Site
Taipei, Taiwan
RECRUITINGResearch Site
Taipei, Taiwan
RECRUITINGResearch Site
Bangkok, Bangkok, Thailand
RECRUITINGResearch Site
Hat Yai, Changwat Songkhla, Thailand
RECRUITING...and 1 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)