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DYNE-251

Phase 1

Duchenne Muscular Dystrophy (DMD) | Small molecule | Neurology |Dyne Therapeutics, Inc.|Last Updated: Aug 11, 2026

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindCONTROLLED
Total Trials1
Total Enrollment86

FDA Designations

ORPHAN_DRUGBREAKTHROUGH_THERAPYFAST_TRACKRMATACCELERATED_APPROVALRARE_PEDIATRIC_DISEASE

Clinical trial landscape

DYNE-251 · 1 trial · 1 indication

Phase 1 1
NCT05524883Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 SkippingDuchenne Muscular Dystrophy (DMD)
ACTIVE NOT_RECRUITING86 Analytics
PHASE1ACTIVE NOT_RECRUITING
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
Duchenne Muscular Dystrophy (DMD)
NCT0552488386
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Study Endpoints

Primary Endpoints

Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Through study completion, up to Week 337
Change From Baseline in Dystrophin Protein Levels in Muscle Tissue at Week 25
Baseline, Week 25

Secondary Endpoints

Change From Baseline in Muscle Tissue Exon 51 Skipping Levels at Week 25 For Participants Dosed at Q4W or Q8W Interval With a Second Biopsy Performed at Week 25
Baseline, Week 25
Change From Baseline in Muscle Tissue Percent Dystrophin-Positive Fiber (PDPF) at Week 25 For Participants Dosed at Q4W or Q8W Interval With a Second Biopsy Performed at Week 25
Baseline, Week 25
Change From Baseline in Blood Creatine Kinase (CK) Levels up to Week 337 For Participants Dosed at Q4W or Q8W Interval With a Second Biopsy Performed at Week 25
Baseline, up to Week 337
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Placebo-Controlled MAD Period - DYNE-251EXPERIMENTALDYNE-251 will be administered once every 4 weeks (Q4W) or once every 8 weeks (Q8W) over 24 weeks.
Placebo-Controlled MAD Period - PlaceboEXPERIMENTALPlacebo will be administered Q4W or Q8W over 24 weeks.
Open-Label and Long-Term Extension Period - DYNE-251EXPERIMENTALDYNE-251 will be administered Q4W or Q8W for up to 288 weeks after participants complete the Placebo-Controlled MAD Period of the study.

Interventions

NameTypeDescription
DYNE-251DRUGAdministered by IV infusion
PlaceboDRUGAdministered by IV infusion
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Eligibility Criteria

Age Range4 Years to 16 Years
SexMALE
Healthy VolunteersNo
Study Sites30

Inclusion Criteria: * Age 4 to 16 years inclusive, at the time of informed consent/assent. * Male with a confirmed diagnosis of DMD and with a mutation in the dystrophin gene characterized by exon deletion amenable to exon 51 skipping. * Upper extremity muscle group that is amenable to muscle biops...

Countries:United StatesAustraliaBelgiumCanadaIrelandItalySouth KoreaSpainUnited Kingdom
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Recent Changes (Last 90 Days)

MEDIUMAug 11, 2026NCT05524883primaryCompletionDate: changed
MEDIUMAug 11, 2026NCT05524883primaryCompletionDate: changed

Frequently asked questions about DYNE-251

What is DYNE-251 used for in Duchenne Muscular Dystrophy?

DYNE-251 is an investigational small molecule being developed for the treatment of Duchenne Muscular Dystrophy (DMD) in patients amenable to exon 51 skipping. It is currently in Phase 1 clinical development and has not been approved by the FDA. The drug is being studied in a clinical trial enrolling male participants aged 4 years and older.

Who makes DYNE-251?

DYNE-251 is being developed by Dyne Therapeutics, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol DYN. The company is conducting a Phase 1 clinical trial of DYNE-251 in patients with Duchenne Muscular Dystrophy amenable to exon 51 skipping.

What phase is DYNE-251 in?

DYNE-251 is in Phase 1 clinical development. It is an investigational drug and has not received FDA approval. The ongoing Phase 1 trial is active but not recruiting participants, and it is designed to evaluate the safety, tolerability, pharmacodynamics, efficacy, and pharmacokinetics of DYNE-251 in Duchenne Muscular Dystrophy patients.

What clinical trials is DYNE-251 in?

DYNE-251 is being studied in one clinical trial with the identifier NCT05524883. This Phase 1 study is titled 'Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping.' The trial is active but not recruiting, with an enrollment of 86 participants across multiple countries.

What FDA designations has DYNE-251 received?

DYNE-251 has received several FDA designations, including Orphan Drug, Breakthrough Therapy, Fast Track, Regenerative Medicine Advanced Therapy (RMAT), Accelerated Approval, and Rare Pediatric Disease designations. These designations are intended to expedite the development and review of the drug for Duchenne Muscular Dystrophy.

Is DYNE-251 the same as any other drug?

DYNE-251 is the primary name for this investigational drug. No alternative names have been reported for this asset. It is being developed specifically for Duchenne Muscular Dystrophy patients amenable to exon 51 skipping, and it is currently in Phase 1 clinical trials.