A Randomized, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-101 Administered to Participants with Myotonic Dystrophy Type 1

Myotonic Dystrophy Type 1

MAD Cohorts: Number and proportion of participants with treatment-emergent adverse events (TEAEs), treatment-emergent serious adverse events (TESAEs), TEAEs considered related to study drug, and TEAEs leading to discontinuation from study drug and discontinuation from the study., Dose Expansion Cohort: Change from baseline in CASI in skeletal muscle tissue at Week 13

Change from baseline in CASI in skeletal muscle tissue Change from baseline in DMPK RNA expression in muscle tissue, MAD Cohorts Change from baseline in hand grip relaxation time by a dynamometer Change from baseline in myotonia as measured by vHOT Change from baseline in Quantitative Myometry Testing (QMT) Change from baseline in 10-meter walk/run test (10-MWRT) Change from baseline in stair-ascend/descend test Change from baseline in 5 times sit to stand (5×STS) Change from baseline in 9-Hole Peg Test (9-HPT), Dose Expansion Cohort Change from baseline in myotonia as measured by vHOT (middle finger) at Week 25 Change from baseline in 10-MWRT at Week 25 Change from baseline in QMT subtotal of four muscle groups (percent predicted of handgrip strength, ankle dorsiflexion, knee flexion, and knee extension) at Week 25 Change from baseline in 5×STS at Week 25 Change from baseline in percent predicted hand grip strength at Week 25 Change from baseline in MDHI total score at Week 25, MAD Cohorts Plasma endpoints: Max observed plasma drug concentration Time to max observed plasma drug concentration Area under the plasma-drug concentration-time curve from time 0 to the last quantifiable concentration AUC extrapolated to time infinity Apparent terminal phase elimination rate constant and elimination half-life Plasma clearance Volume of distribution at the terminal phase, if appropriate and at steady state, if appropriate Muscle tissue endpoint: Tissue ASO concentration, MAD Cohorts Incidence of antidrug antibodies (ADAs)

No related papers found yet.

France, Germany, Italy, Netherlands