DYNE-251 for Muscular Dystrophy, Duchenne
Phase-Based Progress Estimates
Effectiveness
Safety
Rare Disease Research, LLC, Atlanta, GA
Muscular Dystrophy, Duchenne+2 More
DYNE-251 - DrugEligibility
< 18
Male
What conditions do you have?
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Eligibility
< 18
Male
What conditions do you have?
Select
Study Summary
The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) doses of DYNE-251 in participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The study consists of 3 periods: a multiple-ascending dose (MAD) / placebo-controlled period (24 weeks), an open-label period (24 weeks) and a long-term extension period (96 weeks).
Eligible Conditions
- Muscular Dystrophy, Duchenne
Treatment Effectiveness
Effectiveness Progress
Study Objectives
2 Primary · 19 Secondary · Reporting Duration: Baseline, up to Week 145
Baseline, Week 25
Change From Baseline in Dystrophin Protein Levels in Muscle Tissue at Week 25
Change From Baseline in Muscle Tissue Exon 51 Skipping Levels at Week 25
Change From Baseline in Muscle Tissue Percent Dystrophin-Positive Fiber (PDPF) at Week 25
Week 145
Change From Baseline in 10-Meter Run/Walk (10-MRW) Time in Ambulatory Participants up to Week 145
Change From Baseline in Blood Creatine Kinase (CK) Levels up to Week 145
Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score in Ambulatory Participants up to Week 145
Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) up to Week 145
Change From Baseline in Performance Upper Limb (PUL) Scale Version 2.0 Score up to Week 145
Change From Baseline in Time to Rise From Floor in Ambulatory Participants up to Week 145
Week 145
AUC Extrapolated to Infinity of DYNE-251 in Plasma (AUC∞)
Apparent Terminal Elimination Half-Life of DYNE-251 in Plasma (t½)
Apparent Terminal Elimination Rate Constant of DYNE-251 in Plasma (λz)
Area Under the Concentration-Time Curve From Hour 0 to the Last Measurable Concentration of DYNE-251 in Plasma (AUC0-tlast)
Clearance (CL) of DYNE-251 in Plasma
Maximum Observed Drug Concentration of DYNE-251 in Plasma (Cmax)
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Percentage of Participants With Antidrug Antibodies (ADAs)
Time to Maximum Concentration of DYNE-251 in Plasma (tmax)
Tissue Phosphorodiamidate Morpholino Oligomer (PMO) Concentration of DYNE-251 in Muscle Tissue
Volume of Distribution at Steady State of DYNE-251 in Plasma (Vss)
Volume of Distribution at the Terminal Phase of DYNE-251 in Plasma (Vz)
Trial Safety
Safety Progress
Trial Design
3 Treatment Groups
Placebo-Controlled MAD Period - Placebo
1 of 3
Placebo-Controlled MAD Period - DYNE-251
1 of 3
Open-Label and Long-Term Extension Period - DYNE-251
1 of 3
Experimental Treatment
46 Total Participants · 3 Treatment Groups
Primary Treatment: DYNE-251 · Has Placebo Group · Phase 1 & 2
Placebo-Controlled MAD Period - Placebo
Drug
Experimental Group · 1 Intervention: Placebo · Intervention Types: DrugPlacebo-Controlled MAD Period - DYNE-251
Drug
Experimental Group · 1 Intervention: DYNE-251 · Intervention Types: DrugOpen-Label and Long-Term Extension Period - DYNE-251
Drug
Experimental Group · 1 Intervention: DYNE-251 · Intervention Types: DrugTreatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Placebo
1995
Completed Phase 3
~2670
Trial Logistics
Trial Timeline
Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: baseline, up to week 145
Closest Location: Rare Disease Research, LLC · Atlanta, GA
2020First Recorded Clinical Trial
14 TrialsResearching Muscular Dystrophy, Duchenne
12 CompletedClinical Trials
Who is running the clinical trial?
Dyne TherapeuticsLead Sponsor
1 Previous Clinical Trials
64 Total Patients Enrolled
Eligibility Criteria
Age < 18 · Male Participants · 7 Total Inclusion Criteria
Mark “yes” if the following statements are true for you:You have a medical condition that is amenable to muscle biopsy.
About The Reviewer
Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.
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