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NCT05481879 · ClinicalTrials.gov registry record · Phase 1
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1
A Phase 1 study of Myotonic Dystrophy Type 1 (DM1), sponsored by Dyne Therapeutics.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 116
- Enrollment target
- 17
- Study locations
NCT05481879 is a Phase 1 study of Myotonic Dystrophy Type 1 (DM1) that is actively recruiting participants, run by Dyne Therapeutics. The registered enrollment target is 116 participants. The trial reports 17 study locations across 7 states.
The verdict
NCT05481879, a Phase 1 study of Myotonic Dystrophy Type 1 (DM1), is actively recruiting participants, sponsored by Dyne Therapeutics.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 116 participants
- Enrollment target
- 17
- Study locations
Study Summary
The primary purpose of the study is to evaluate the safety and tolerability of multiple intravenous (IV) doses of DYNE-101 administered to participants with Myotonic Dystrophy Type 1 (DM1). The study consists of 4 periods: A Screening Period (up to 8 weeks), a Placebo-Controlled Period (24 weeks), a Treatment Period (24 weeks) and a Long-Term Extension (LTE) Period (168 weeks) in both multiple-ascending dose (MAD) and dose expansion cohorts.
Conditions Studied
Interventions
- DRUG Placebo
- DRUG DYNE-101
Study Locations (17)
Other
- CHU de Nantes - Nantes
- Institut de Myologie - Paris
- Charité - Universitätsmedizin Berlin - Berlin
- Ludwig Maximilians University, Munich - Friedrich Baur Institut - Munich
- Centro Clinico Nemo - Milan
- Fondazione Policlinico Universitario A Gemelli-Rome - Rome
- Radboud Medical Center - Nijmegen
- NZCR Auckland - Auckland
- University College London Hospitals - London
- John Walton Muscular Dystrophy Research Centre - Newcastle upon Tyne
- Salford Royal Hospital - Salford
Indiana
- Indiana University School of Medicine - Indianapolis
Iowa
- University of Iowa - Iowa City
New York
- University of Rochester Medical Center - Rochester
Texas
- Neurology Rare Disease Center - Denton
Virginia
- Virginia Commonwealth University (VCU) - Richmond
Victoria
- St. Vincent's Hospital - Fitzroy
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 116 participants |
| Start Date | 2022-09-05 |
| Est. Completion | 2029-07 |
| Phase | Phase 1 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05481879
The ClinicalTrials.gov registry entry for NCT05481879 describes a study currently listed as recruiting, categorized as Phase 1. The registered enrollment target is 116 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Dyne Therapeutics, which has 4 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Myotonic Dystrophy Type 1 (DM1) appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT05481879 reports 17 study locations spanning 7 distinct geographic areas - top geographies include Other, Indiana, Iowa.
Frequently Asked Questions
What is clinical trial NCT05481879 about?
NCT05481879 is a clinical study titled "Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1". The primary purpose of the study is to evaluate the safety and tolerability of multiple intravenous (IV) doses of DYNE-101 administered to participants with Myotonic Dystrophy Type 1 (DM1). The study consists of 4 periods: A Screening Period (up to 8 weeks), a Placebo-Controlled Period (24 weeks), ...
What is the current status of trial NCT05481879?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 116 participants. The study started on 2022-09-05. Estimated completion is 2029-07.
What conditions does trial NCT05481879 study?
This clinical trial studies the following conditions: Myotonic Dystrophy Type 1 (DM1).
What interventions are being tested in trial NCT05481879?
The interventions under investigation include: Placebo (DRUG), DYNE-101 (DRUG).
Who is sponsoring clinical trial NCT05481879?
This trial is sponsored by Dyne Therapeutics, which has 4 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05481879 being conducted?
This trial has 17 study locations across Indiana, Iowa, New York, Texas, Virginia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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