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zeleciment basivarsen

Phase 3

Myotonic Dystrophy Type 1 (DM1) | Small molecule | Rare Disease |Dyne Therapeutics, Inc.|Last Updated: Sep 3, 2026

Target and mechanism

ModalitySmall molecule

Also known as zeleciment basivarsen (DYNE-101)

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment150

FDA Designations

No designations recorded

Clinical trial landscape

zeleciment basivarsen · 1 trial · 5 indications

Phase 3 1
NCT07486934Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1Myotonic Dystrophy Type 1 (DM1)
RECRUITING150 Analytics
PHASE3RECRUITING
Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1
Myotonic Dystrophy Type 1 (DM1)Unlock trial analytics

Study Endpoints

Primary Endpoints

5 Times Sit-To-Stand (5×STS) Time
Baseline, Week 49

Secondary Endpoints

Video Hand Opening Time (vHOT) [Middle Finger]
Baseline, Week 49
Quantitative Muscle Testing (QMT) Total
Baseline, Week 49
Clinician Global Impression of Change (CGI-C)
Week 49
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Placebo-Controlled Period: DYNE-101EXPERIMENTALParticipants will be randomized to receive DYNE-101, once every 8 weeks (Q8W) for up to 48 weeks.
Placebo-Controlled Period: PlaceboPLACEBO_COMPARATORParticipants will be randomized to receive DYNE-101 matching placebo, Q8W for up to 48 weeks.
Long-Term Extension Period: DYNE-101EXPERIMENTALParticipants who receive DYNE-101 in Placebo-Controlled Period will continue to received DYNE-101, Q8W for up to 24 weeks. Participants who received placebo in Placebo-Controlled Period will receive DYNE-101, Q8W for up to 24 weeks.

Interventions

NameTypeDescription
zeleciment basivarsen (DYNE-101)DRUGAdministered by IV infusion
PlaceboDRUGAdministered by IV infusion
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Eligibility Criteria

Age Range16 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites37

Inclusion Criteria: * Diagnosis of DM1 confirmed by molecular genetics with trinucleotide repeat size greater than (\>) 100. Historical results from clinical testing are acceptable. * Able to walk 10 meters and complete 5 times sit to stand independently (inserts or supports that don't go above the...

Countries:United StatesAustraliaBelgiumDenmarkFranceGermanyItalyJapanNetherlandsSpainUnited Kingdom
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Recent Changes (Last 90 Days)

LOWSep 3, 2026NCT07486934lastUpdatePostDate: changed
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Frequently asked questions about zeleciment basivarsen

What is zeleciment basivarsen used for?

Zeleciment basivarsen is an investigational drug being studied for the treatment of Myotonic Dystrophy Type 1 (DM1), a rare genetic disorder. It is currently in Phase 3 clinical development and has not been approved by regulatory authorities.

Who makes zeleciment basivarsen?

Zeleciment basivarsen is being developed by Dyne Therapeutics, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol DYN. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with Myotonic Dystrophy Type 1.

What phase is zeleciment basivarsen in?

Zeleciment basivarsen is currently in Phase 3 clinical development. It is an investigational drug, meaning it has not yet been approved by regulatory agencies. The ongoing Phase 3 trial is designed to assess its efficacy, safety, and tolerability in participants with Myotonic Dystrophy Type 1.

What clinical trials is zeleciment basivarsen in?

Zeleciment basivarsen is being evaluated in a Phase 3 clinical trial with the identifier NCT07486934. This randomized, double-blind, placebo-controlled study is recruiting approximately 150 participants with Myotonic Dystrophy Type 1 across multiple countries, including the United States, Australia, and several European nations.

Is zeleciment basivarsen the same as DYNE-101?

Yes, zeleciment basivarsen is also known as DYNE-101. Both names refer to the same investigational drug being developed by Dyne Therapeutics for the treatment of Myotonic Dystrophy Type 1. The drug is currently in Phase 3 clinical trials.