- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05027269
Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients (MARINA)
A Randomized, Double-Blind, Placebo-Controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple-Doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients
AOC 1001-CS1 is a randomized, double-blind, placebo-controlled, Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients (MARINA).
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.
Study Overview
Status
Conditions
Intervention / Treatment
Study Type
Enrollment (Actual)
Phase
- Phase 2
- Phase 1
Contacts and Locations
Study Locations
-
-
California
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Los Angeles, California, United States, 90095
- University of California Los Angeles
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Palo Alto, California, United States, 94304
- Stanford University
-
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Colorado
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Denver, Colorado, United States, 80045
- University of Colorado
-
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Florida
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Gainesville, Florida, United States, 32608
- University of Florida
-
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Kansas
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Kansas City, Kansas, United States, 66205
- Kansas University Medical Center
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New York
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Rochester, New York, United States, 14642
- University of Rochester Medical Center
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Ohio
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Columbus, Ohio, United States, 43221
- Ohio State University
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Virginia
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Richmond, Virginia, United States, 23298
- Virginia Commonwealth University
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-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
Key Inclusion Criteria:
- Genetic diagnosis of DM1 (CTG repeat length ≥ 100)
- Clinician assessed signs of DM1
- Ability to walk independently (orthoses and ankle braces allowed) for at least 10 meters at screening
Key Exclusion Criteria:
- Diabetes that is not adequately controlled
- 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
Note: Other protocol defined Inclusion/Exclusion criteria may apply.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Sequential Assignment
- Masking: Quadruple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Part A Single Dose: AOC 1001 Dose Level 1
AOC 1001 will be administered once.
|
AOC 1001 will be administered by intravenous (IV) infusion.
|
Placebo Comparator: Part A Single Dose: Placebo
Saline will be administered once.
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Placebo will be administered by intravenous (IV) infusion.
|
Placebo Comparator: Part B Multiple Ascending Dose: Placebo
Saline will be administered three times.
|
Placebo will be administered by intravenous (IV) infusion.
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Experimental: Part B Multiple Ascending Dose: AOC 1001 Dose Levels 2 & 3
AOC 1001 will be administered three times.
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AOC 1001 will be administered by intravenous (IV) infusion.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Frequency of treatment emergent adverse events (TEAEs)
Time Frame: Through study completion, up to Day 183
|
Through study completion, up to Day 183
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Plasma pharmacokinetic (PK) parameters
Time Frame: Through study completion, up to Day 183
|
Maximum plasma concentration (Cmax)
|
Through study completion, up to Day 183
|
Plasma pharmacokinetic (PK) parameters
Time Frame: Through study completion, up to Day 183
|
Time to maximum plasma concentration (Tmax)
|
Through study completion, up to Day 183
|
Plasma pharmacokinetic (PK) parameters
Time Frame: Through study completion, up to Day 183
|
Terminal Half-life (t1/2)
|
Through study completion, up to Day 183
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Plasma pharmacokinetic (PK) parameters
Time Frame: Through study completion, up to Day 183
|
Area Under the Concentration-time Curve (AUC)
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Through study completion, up to Day 183
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Urine pharmacokinetic (PK) parameters
Time Frame: Through study completion, up to Day 183
|
fraction excreted (fe) in urine
|
Through study completion, up to Day 183
|
AOC 1001 levels in muscle tissue
Time Frame: Through study completion, up to Day 183
|
Through study completion, up to Day 183
|
|
Change and percentage change from baseline in DMPK mRNA knockdown
Time Frame: Through study completion, up to Day 183
|
Through study completion, up to Day 183
|
|
Change and percentage change from baseline in Spliceopathy
Time Frame: Through study completion, up to Day 183
|
Through study completion, up to Day 183
|
Collaborators and Investigators
Sponsor
Investigators
- Study Director: Li Tai, MD, Avidity Biosciences, Inc.
Publications and helpful links
Helpful Links
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- AOC 1001-CS1
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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