There are no available treatments aside from supportive care for patients with Centronuclear myopathy (CNM). This trial will assess the safety and tolerability as well as pharmacokinetics (PK), pharmacodynamics (PD) and preliminary efficacy of DYN101 in participants 2 to 17 years of age with CNM caused by mutations in DNM2 or MTM1.The trial will consist of a pre-screening consent, a screening period, a run-in period (if applicable), and a Part 1 of 12 weeks with weekly infusion of DYN101 to evaluate safety and tolerability as well as PK, PD and preliminary efficacy. The dose level may need adjustment based on the Part 1 results of the current study and available data from the Unite-CNM study (DYN101-C101, NCT04033159). If a dose adjustment is needed, Part 2 will be conducted in the same participants and the newly selected dose level will be used to assess whether efficacy is seen after an additional 12 weeks of treatment. As this trial is investigational, there is no defined, expected benefit for subjects who participate in this trial except a better knowledge of their disease.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
DYN101, is a constrained ethyl gapmer ASO directed against human DNM2 pre-mRNA
I-Motion Institute - Trousseau Hospital
Paris, France
Universitätsklinikum Essen
Essen, Germany
Incidence of drug-related Treatment Emergent Adverse Events (TEAEs)
Time frame: Baseline until Week 12
Measurement of DYN101 concentration in plasma
Time frame: Week 1 Day 1: before the start of infusion (baseline), immediately after the end of infusion, and at 1, 3, 7, 23, 71 hours after the end of infusion. Week 13 Day 1: predose and 3 hours after the end of infusion
Maximum plasma drug concentration (Cmax) for DYN101
Time frame: Week 1 Day 1: before the start of infusion (baseline), immediately after the end of infusion, and at 1, 3, 7, 23, 71 hours after the end of infusion. Week 13 Day 1: predose and 3 hours after the end of infusion
Area under the Plasma Concentration versus Time Curve (AUC) of DYN101
Time frame: Week 1 Day 1: before the start of infusion (baseline), immediately after the end of infusion, and at 1, 3, 7, 23, 71 hours after the end of infusion. Week 13 Day 1: predose and 3 hours after the end of infusion
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