DYNE-251 for Muscular Dystrophy, Duchenne

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Rare Disease Research, LLC, Atlanta, GA
Muscular Dystrophy, Duchenne+2 More
DYNE-251 - Drug
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

1 of 3

Other trials for Muscular Dystrophy, Duchenne

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

1 of 3

Other trials for Muscular Dystrophy, Duchenne

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: Drug
Placebo-Controlled MAD Period - DYNE-251
Drug
Experimental Group · 1 Intervention: DYNE-251 · Intervention Types: Drug
Open-Label and Long-Term Extension Period - DYNE-251
Drug
Experimental Group · 1 Intervention: DYNE-251 · Intervention Types: Drug
Treatment
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
Photo of Atlanta 1Photo of Atlanta 2Photo of Atlanta 3
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 preview

Michael Gill - B. Sc.

First Published: October 9th, 2021

Last Reviewed: August 12th, 2022

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.