← Back to Search

Other

DYN101 for Centronuclear Myopathy (Unite-CNM Trial)

Phase 1 & 2
Waitlist Available
Led By N.C. Voermans, MD, PhD
Research Sponsored by Dynacure
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline until study termination, up to 28 months
Awards & highlights

Unite-CNM Trial Summary

This trial is testing a new medicine called DYN101 for safety, tolerability, and preliminary efficacy in patients with a disease called Centronuclear myopathy (CNM). There is currently no available treatment for this disease. The trial will consist of a Single dose treatment part, a Multiple dose treatment part, and a Multiple dose extension part. All subjects will participate in the Single dose treatment part, unless they withdraw. During this time, multiple tests will be performed to better understand how the drug is distributed and then later removed from the body and whether there are any signs of an effect.

Eligible Conditions
  • Centronuclear Myopathy

Unite-CNM Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline until study termination, up to 28 months
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline until study termination, up to 28 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number of Participants With Drug-related Treatment Emergent Adverse Events (TEAEs)

Unite-CNM Trial Design

3Treatment groups
Experimental Treatment
Group I: cohort 3Experimental Treatment1 Intervention
DYN101 in a high dose (9 mg/kg), (unless the IDMC advises otherwise). In each cohort, there will be 3-4 subjects with a mutation in DNM2 (subcohort a) and 2-3 subjects with a mutation in MTM1 (subcohort b).
Group II: cohort 2Experimental Treatment1 Intervention
DYN101 in a middle dose (4.5 mg/kg), (unless the IDMC advises otherwise). In each cohort, there will be 3-4 subjects with a mutation in DNM2 (subcohort a) and 2-3 subjects with a mutation in MTM1 (subcohort b).
Group III: cohort 1Experimental Treatment1 Intervention
DYN101 in a low dose (1.5 mg/kg), (unless the independent data monitoring committee [IDMC] advises otherwise). In each cohort, there will be 3-4 subjects with a mutation in DNM2 (subcohort a) and 2-3 subjects with a mutation in MTM1 (subcohort b).

Find a Location

Who is running the clinical trial?

DynacureLead Sponsor
3 Previous Clinical Trials
60 Total Patients Enrolled
3 Trials studying Centronuclear Myopathy
60 Patients Enrolled for Centronuclear Myopathy
Chris Freitag, MDStudy DirectorDynacure
N.C. Voermans, MD, PhDPrincipal InvestigatorRadboud University Medical Center

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
clinicaltrials.govStudy
Early Phase Human Drug Trial to Investigate Dynamin 101 (DYN101) in Patients ≥ 16 Years With Centronuclear Myopathies
2020. "Early Phase Human Drug Trial to Investigate Dynamin 101 (DYN101) in Patients ≥ 16 Years With Centronuclear Myopathies". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04033159.
~3 spots leftby Apr 2025
Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top