Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients
NCT ID: NCT05027269
Last Updated: 2024-02-05
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE1/PHASE2
38 participants
INTERVENTIONAL
2021-10-28
2023-02-14
Brief Summary
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Part A is a single dose design with 1 cohort (dose level). In Part A, the patient duration is 6 months as the treatment period is 1 day followed by a 6 month follow-up period.
Part B is a multiple-ascending dose design with 2 cohorts (dose levels). In Part B, the patient duration is 6 months as the treatment period is 3 months followed by a 3 month follow-up period.
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
SEQUENTIAL
TREATMENT
QUADRUPLE
Study Groups
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Part A Single Dose: AOC 1001 Dose Level 1
AOC 1001 will be administered once.
AOC 1001
AOC 1001 will be administered by intravenous (IV) infusion.
Part A Single Dose: Placebo
Saline will be administered once.
Placebo
Placebo will be administered by intravenous (IV) infusion.
Part B Multiple Ascending Dose: AOC 1001 Dose Levels 2 & 3
AOC 1001 will be administered three times.
AOC 1001
AOC 1001 will be administered by intravenous (IV) infusion.
Part B Multiple Ascending Dose: Placebo
Saline will be administered three times.
Placebo
Placebo will be administered by intravenous (IV) infusion.
Interventions
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AOC 1001
AOC 1001 will be administered by intravenous (IV) infusion.
Placebo
Placebo will be administered by intravenous (IV) infusion.
Eligibility Criteria
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Inclusion Criteria
* Clinician assessed signs of DM1
* Ability to walk independently (orthoses and ankle braces allowed) for at least 10 meters at screening
Exclusion Criteria
* BMI \> 35 kg/m2
* Uncontrolled hypertension
* Congenital DM1
* History of tibialis anterior (TA) biopsy within 3 months of Day 1 or planning to undergo TA biopsies during study period
* Recently treated with an investigational drug
* Treatment with anti-myotonic medication within 14 days of Day 1
18 Years
65 Years
ALL
No
Sponsors
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Avidity Biosciences, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Li Tai, MD
Role: STUDY_DIRECTOR
Avidity Biosciences, Inc.
Locations
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University of California Los Angeles
Los Angeles, California, United States
Stanford University
Palo Alto, California, United States
University of Colorado
Denver, Colorado, United States
University of Florida
Gainesville, Florida, United States
Kansas University Medical Center
Kansas City, Kansas, United States
University of Rochester Medical Center
Rochester, New York, United States
Ohio State University
Columbus, Ohio, United States
Virginia Commonwealth University
Richmond, Virginia, United States
Countries
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Related Links
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Related Info
Other Identifiers
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AOC 1001-CS1
Identifier Type: -
Identifier Source: org_study_id
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