Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1

Part of paid clinical trials in Stanford, California.

Sponsor
Dyne Therapeutics
Study ID
NCT05481879
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

  • Myotonic Dystrophy Type 1 (DM1)

Eligibility Criteria

Sex
ALL
Age
18 Years - 65 Years
Healthy Volunteers
Not accepted

Interventions

  • DYNE-101 — DRUG
    Administered by IV infusion
  • Placebo — DRUG
    Administered by IV infusion

Study Details

The primary purpose of the study is to evaluate the safety and tolerability of multiple intravenous (IV) doses of DYNE-101 administered to participants with Myotonic Dystrophy Type 1 (DM1). The study consists of 4 periods: A Screening Period (up to 8 weeks), a Placebo-Controlled Period (24 weeks), a Treatment Period (24 weeks) and a Long-Term Extension (LTE) Period (168 weeks) in both multiple-ascending dose (MAD) and dose expansion cohorts.

Key Dates

Start date
Sep 5, 2022
Status verified
Aug 2025
Primary completion
Jul 31, 2029
Completion
Jul 31, 2029

Study Design

Enrollment
116 participants (estimated)
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: MAD Cohort: Placebo-Controlled Period: DYNE-101
    Participants will be randomized to receive ascending doses of DYNE-101, once every 4 weeks (Q4W) or once every 8 weeks (Q8W) for up to 24 weeks.
  • Placebo Comparator: MAD Cohort: Placebo-Controlled Period: Placebo
    Participants will be randomized to receive DYNE-101 matching placebo, Q4W or Q8W for up to 24 weeks.
  • Experimental: MAD Cohort: Treatment Period: DYNE-101
    Participants who receive DYNE-101 in Placebo-Controlled Period will continue to receive DYNE-101, Q4W or Q8W for up to 24 weeks. Participants who receive placebo in Placebo-Controlled Period will receive DYNE-101, Q4W or Q8W for up to 24 weeks.
  • Experimental: MAD Cohort: Long-Term Extension Period: DYNE-101
    Participants will receive DYNE-101, Q4W or Q8W for up to 168 weeks.
  • Experimental: Dose Expansion Cohort: Placebo-Controlled Period: DYNE-101
    Participants will receive DYNE-101, Q8W for up to 24 weeks.
  • Experimental: Dose Expansion Cohort: Placebo-Controlled Period: Placebo
    Participants will receive DYNE-101 matching placebo, Q8W for up to 24 weeks.
  • Experimental: Dose Expansion Cohort: Treatment Period: DYNE-101
    Participants who receive DYNE-101 in Placebo-Controlled Period will continue to receive DYNE-101, Q8W for up to 24 weeks. Participants who receive placebo in Placebo-Controlled Period will receive DYNE-101, Q8W for up to 24 weeks.
  • Experimental: Dose Expansion Cohort: Long-Term Extension Period: DYNE-101
    Participants will receive DYNE-101, Q8W for up to 168 weeks.

Primary Outcome Measure

MAD Cohorts: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) [ Time Frame: Through study completion, up to Week 217 ]

Central Contacts

Locations (8)

FacilityCityStateZIPSite coordinators
Stanford UniversityStanfordCalifornia94305
Lidia Choniawko
[email protected]
Dr. John Day, MD, PhD (PRINCIPAL_INVESTIGATOR)
University of Florida College of MedicineGainesvilleFlorida32610
Debbye Landers
[email protected]
Dr. Sub Subramony, MD (PRINCIPAL_INVESTIGATOR)
Indiana University School of MedicineIndianapolisIndiana46202
Patti Hogan, BSN RN, CCRA
[email protected]
Laurie Gutmann, MD (PRINCIPAL_INVESTIGATOR)
University of IowaIowa CityIowa52242
Heena Olade, RN, MSN
[email protected]
Andrea Swenson, MD (PRINCIPAL_INVESTIGATOR)
Washington University in St. LouisSt LouisMissouri63110
Natalie Goedeker
[email protected]
Dr. Craig Zaidman, MD (PRINCIPAL_INVESTIGATOR)
University of Rochester Medical CenterRochesterNew York14642
Dr. Joahnna Hamel, MD (PRINCIPAL_INVESTIGATOR)
Neurology Rare Disease CenterDentonTexas76208
Isabella Herman
[email protected]
Diana Castro, MD (PRINCIPAL_INVESTIGATOR)
Virginia Commonwealth University (VCU)RichmondVirginia23298
Jodie Howell
[email protected]
Anarosa Rezeq
[email protected]
Nicholas Johnson, M.D., M.Sci., FAAN (PRINCIPAL_INVESTIGATOR)

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