Back to resultsExtension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients (MARINA-OLE)
Primary Purpose
DM1, Muscular Dystrophies, Myotonic Dystrophy
Status
Active
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Placebo
AOC 1001
Sponsored by
About this trial
This is an interventional treatment trial for DM1 focused on measuring DM1, Myotonic Dystrophy 1, Myotonic Dystrophy Type 1 (DM1), Myotonic Dystrophy, DM, Dystrophy Myotonic, Myotonic Disorders, Steinert Disease, MARINA-OLE, MARINA, Avidity Biosciences, Avidity, AOC 1001, Myotonic Muscular Dystrophy
Eligibility Criteria
Key Inclusion Criteria:
- Completion of AOC 1001-CS1 (MARINA) study with satisfactory compliance and no significant tolerability issues
Key Exclusion Criteria:
- Pregnancy, intent to become pregnant, or active breastfeeding
- Unwilling or unable to continue to comply with contraceptive requirements
- Any new conditions or worsening of existing conditions that in the opinion of the investigator or sponsor would make the participant unsuitable for the study or could interfere with participation or completion of the study
Sites / Locations
- University of California Los Angeles
- Stanford University
- University of Colorado
- University of Florida
- Kansas University Medical Center
- University of Rochester Medical Center
- Ohio State University
- Virginia Commonwealth University
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
AOC 1001
AOC 1001 (with Placebo at Day 43)
Arm Description
AOC 1001 will be administered quarterly. On Day 43 patients will receive an additional dose. Treatment assignment will be based on treatment received in AOC 1001-CS1. If participant did not receive AOC 1001 on Day 43 in AOC 1001-CS1, participant will receive AOC 1001 treatment on Day 43 in AOC 1001-CS2.
AOC 1001 will be administered quarterly. On Day 43 patients will receive an additional dose. Treatment assignment will be based on treatment received in AOC 1001-CS1. If participant received AOC 1001 on Day 43 in AOC 1001-CS1, participant will receive blinded placebo treatment on Day 43 in AOC 1001-CS2.
Outcomes
Primary Outcome Measures
Number and severity of treatment-emergent adverse events (TEAEs)
Secondary Outcome Measures
Plasma pharmacokinetic (PK) parameters
Maximum and trough plasma concentration
AOC 1001 levels in muscle tissue
Change and percentage change from baseline in DMPK mRNA knockdown
Change and percentage change from baseline in Spliceopathy
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05479981
Brief Title
Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients
Acronym
MARINA-OLE
Official Title
A Phase 2 Extension Study to Evaluate the Long-Term Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients
Study Type
Interventional
2. Study Status
Record Verification Date
July 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
August 4, 2022 (Actual)
Primary Completion Date
June 2025 (Anticipated)
Study Completion Date
June 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Avidity Biosciences, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
AOC 1001-CS2 (MARINA-OLE) is a Phase 2 extension of the AOC 1001-CS1 (MARINA) study to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients
Detailed Description
This study will continue to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of AOC 1001 in participants that enrolled in the randomized, placebo-controlled, First-In-Human Phase 1/2 AOC 1001-CS1 (MARINA) clinical study.
Participants from AOC 1001-CS1 are eligible to enroll in AOC 1001-CS2 if they have satisfactorily completed AOC 1001-CS1.
The total duration of active treatment in AOC 1001-CS2 is approximately 24 months. Once participants have completed active treatment, they will be followed through a 9-month safety follow-up period. The sponsor may extend active treatment beyond 24 months at a future timepoint.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
DM1, Muscular Dystrophies, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonic Disorders, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, Neurodegenerative Diseases
Keywords
DM1, Myotonic Dystrophy 1, Myotonic Dystrophy Type 1 (DM1), Myotonic Dystrophy, DM, Dystrophy Myotonic, Myotonic Disorders, Steinert Disease, MARINA-OLE, MARINA, Avidity Biosciences, Avidity, AOC 1001, Myotonic Muscular Dystrophy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Masking Description
Participants will receive one blinded treatment at Day 43. Treatment assignment on this day will be based on treatment received in AOC 1001-CS1 so that participants who did not receive active drug on Day 43 in AOC 1001-CS1 will received active drug on Day 43 in AOC 1001-CS2.
Allocation
Non-Randomized
Enrollment
37 (Actual)
8. Arms, Groups, and Interventions
Arm Title
AOC 1001
Arm Type
Experimental
Arm Description
AOC 1001 will be administered quarterly. On Day 43 patients will receive an additional dose. Treatment assignment will be based on treatment received in AOC 1001-CS1. If participant did not receive AOC 1001 on Day 43 in AOC 1001-CS1, participant will receive AOC 1001 treatment on Day 43 in AOC 1001-CS2.
Arm Title
AOC 1001 (with Placebo at Day 43)
Arm Type
Experimental
Arm Description
AOC 1001 will be administered quarterly. On Day 43 patients will receive an additional dose. Treatment assignment will be based on treatment received in AOC 1001-CS1. If participant received AOC 1001 on Day 43 in AOC 1001-CS1, participant will receive blinded placebo treatment on Day 43 in AOC 1001-CS2.
Intervention Type
Drug
Intervention Name(s)
Placebo
Other Intervention Name(s)
Saline
Intervention Description
Placebo will be administered by intravenous (IV) infusion.
Intervention Type
Drug
Intervention Name(s)
AOC 1001
Intervention Description
AOC 1001 will be administered by intravenous (IV) infusion.
Primary Outcome Measure Information:
Title
Number and severity of treatment-emergent adverse events (TEAEs)
Time Frame
Through study completion, up to Day 729
Secondary Outcome Measure Information:
Title
Plasma pharmacokinetic (PK) parameters
Description
Maximum and trough plasma concentration
Time Frame
Through study completion, up to Day 729
Title
AOC 1001 levels in muscle tissue
Time Frame
Through Day 183
Title
Change and percentage change from baseline in DMPK mRNA knockdown
Time Frame
Through Day 183
Title
Change and percentage change from baseline in Spliceopathy
Time Frame
Through Day 183
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
66 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria:
Completion of AOC 1001-CS1 (MARINA) study with satisfactory compliance and no significant tolerability issues
Key Exclusion Criteria:
Pregnancy, intent to become pregnant, or active breastfeeding
Unwilling or unable to continue to comply with contraceptive requirements
Any new conditions or worsening of existing conditions that in the opinion of the investigator or sponsor would make the participant unsuitable for the study or could interfere with participation or completion of the study
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Li Tai, MD
Organizational Affiliation
Avidity Biosciences, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
University of California Los Angeles
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
Stanford University
City
Palo Alto
State/Province
California
ZIP/Postal Code
94304
Country
United States
Facility Name
University of Colorado
City
Denver
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
University of Florida
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32608
Country
United States
Facility Name
Kansas University Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66205
Country
United States
Facility Name
University of Rochester Medical Center
City
Rochester
State/Province
New York
ZIP/Postal Code
14642
Country
United States
Facility Name
Ohio State University
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43221
Country
United States
Facility Name
Virginia Commonwealth University
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23298
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients
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