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Exon Skipping Agent

DYNE-251 for Duchenne Muscular Dystrophy (DELIVER Trial)

Phase 1 & 2
Recruiting
Research Sponsored by Dyne Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Ambulatory or non-ambulatory. A non-ambulatory participant must have been non-ambulatory for <2 years before enrolment
Male with a confirmed DMD mutation in the dystrophin gene characterized by exon deletion amenable to exon 51 skipping
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline, up to week 145
Awards & highlights

DELIVER Trial Summary

This trial is testing a new drug to treat Duchenne Muscular Dystrophy. The drug will be given intravenously to participants and the safety, tolerability, and effectiveness of the drug will be monitored over the course of 24 weeks.

Who is the study for?
Boys aged 4-16 with Duchenne muscular dystrophy (DMD) that can be treated by skipping exon 51, able to undergo muscle biopsy. They must have been on stable glucocorticoids for at least 12 weeks and have a healthy heart function. Non-ambulatory participants should not have lost mobility for more than two years.Check my eligibility
What is being tested?
The trial is testing DYNE-251's effects on safety, tolerability, and muscle protein levels in DMD patients compared to a placebo over multiple stages: an initial controlled phase of 24 weeks, followed by an open-label period of the same duration and then a long-term extension for up to 96 weeks.See study design
What are the potential side effects?
While specific side effects are not listed here, common concerns may include reactions at the infusion site, potential kidney or liver issues due to medication processing, allergic reactions, or general symptoms like fatigue or fever.

DELIVER Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have been unable to walk for less than 2 years.
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I am a male with a specific genetic change in my dystrophin gene that can be treated by targeting exon 51.
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I am between 4 and 16 years old.
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A muscle in my arm can be safely biopsied.
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I can lift my arms above my head without help.

DELIVER Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline, up to week 145
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, up to week 145 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Change From Baseline in Dystrophin Protein Levels in Muscle Tissue at Week 25
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Secondary outcome measures
Apparent Terminal Elimination Half-Life of DYNE-251 in Plasma (t½)
Apparent Terminal Phase Elimination Rate Constant of DYNE-251 in Plasma (λz)
Area Under the Plasma Drug Concentration Versus Time Curve From Time 0 (Dosing) Extrapolated to Time Infinity of DYNE-251 (AUC∞)
+21 more

DELIVER Trial Design

3Treatment groups
Experimental Treatment
Group I: Placebo-Controlled MAD Period - PlaceboExperimental Treatment1 Intervention
Placebo will be administered Q4W or Q8W over 24 weeks.
Group II: Placebo-Controlled MAD Period - DYNE-251Experimental Treatment1 Intervention
DYNE-251 will be administered once every 4 weeks (Q4W) or once every 8 weeks (Q8W) over 24 weeks.
Group III: Open-Label and Long-Term Extension Period - DYNE-251Experimental Treatment1 Intervention
DYNE-251 will be administered Q4W or Q8W for up to 96 weeks after participants complete the Placebo-Controlled MAD Period of the study.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Placebo
1995
Completed Phase 3
~2670

Find a Location

Who is running the clinical trial?

Dyne TherapeuticsLead Sponsor
1 Previous Clinical Trials
72 Total Patients Enrolled

Media Library

DYNE-251 (Exon Skipping Agent) Clinical Trial Eligibility Overview. Trial Name: NCT05524883 — Phase 1 & 2
Duchenne Muscular Dystrophy Research Study Groups: Placebo-Controlled MAD Period - DYNE-251, Placebo-Controlled MAD Period - Placebo, Open-Label and Long-Term Extension Period - DYNE-251
Duchenne Muscular Dystrophy Clinical Trial 2023: DYNE-251 Highlights & Side Effects. Trial Name: NCT05524883 — Phase 1 & 2
DYNE-251 (Exon Skipping Agent) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05524883 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Does the eligibility criteria for this trial encompass individuals aged 45 and older?

"This trial is seeking participants that are in the 4 to 16 year old demographic."

Answered by AI

Are there any current opportunities to partake in this experiment?

"That is correct. According to the latest information uploaded on clinicaltrials.gov, this trial - which was first shared on August 12th 2022- is actively searching for 46 patients across 3 medical centres."

Answered by AI

Who can qualify for participation in this trial?

"This clinical trial is recruiting 46 boys aged 4 to 16 with Duchenne muscular dystrophy. In addition, they must have an upper extremity Brooke Upper Extremity Scale score of 1 or 2, be ambulatory or non-ambulatory for less than two years prior to enrolment, and receiving a stable dosage of glucocorticoids for at least 12 weeks before treatment begins as well as having a left ventricular ejection fraction measured by echocardiogram or cardiac magnetic resonance imaging over 50%."

Answered by AI

What is the current recruitment number for participants of this trial?

"Affirmative. According to clinicaltrials.gov, this medical investigation began on August 12th 2022 and is presently enrolling 46 patients between 3 sites. The listing was most recently updated on October 21st 2022."

Answered by AI
~53 spots leftby Nov 2026