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Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients (MARINA-OLE)

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ClinicalTrials.gov Identifier: NCT05479981
Recruitment Status : Active, not recruiting
First Posted : July 29, 2022
Last Update Posted : July 21, 2023
Sponsor:
Information provided by (Responsible Party):
Avidity Biosciences, Inc.

Study Description
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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

Condition or disease Intervention/treatment Phase
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 Drug: Placebo Drug: AOC 1001 Phase 2

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.

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 37 participants
Allocation: Non-Randomized
Intervention 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.
Primary Purpose: Treatment
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
Actual Study Start Date : August 4, 2022
Estimated Primary Completion Date : June 2025
Estimated Study Completion Date : June 2025

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Arms and Interventions
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Arm Intervention/treatment
Experimental: AOC 1001
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.
 Drug: AOC 1001
AOC 1001 will be administered by intravenous (IV) infusion.
Experimental: AOC 1001 (with Placebo at Day 43)
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.
 Drug: Placebo
Placebo will be administered by intravenous (IV) infusion.
Other Name: Saline

Drug: AOC 1001
AOC 1001 will be administered by intravenous (IV) infusion.


Outcome Measures
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Primary Outcome Measures :
  1. Number and severity of treatment-emergent adverse events (TEAEs) [ Time Frame: Through study completion, up to Day 729 ]

Secondary Outcome Measures :
  1. Plasma pharmacokinetic (PK) parameters [ Time Frame: Through study completion, up to Day 729 ]
    Maximum and trough plasma concentration

  2. AOC 1001 levels in muscle tissue [ Time Frame: Through Day 183 ]
  3. Change and percentage change from baseline in DMPK mRNA knockdown [ Time Frame: Through Day 183 ]
  4. Change and percentage change from baseline in Spliceopathy [ Time Frame: Through Day 183 ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years to 66 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05479981


Locations
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United States, California
University of California Los Angeles
Los Angeles, California, United States, 90095
Stanford University
Palo Alto, California, United States, 94304
United States, Colorado
University of Colorado
Denver, Colorado, United States, 80045
United States, Florida
University of Florida
Gainesville, Florida, United States, 32608
United States, Kansas
Kansas University Medical Center
Kansas City, Kansas, United States, 66205
United States, New York
University of Rochester Medical Center
Rochester, New York, United States, 14642
United States, Ohio
Ohio State University
Columbus, Ohio, United States, 43221
United States, Virginia
Virginia Commonwealth University
Richmond, Virginia, United States, 23298
Sponsors and Collaborators
Avidity Biosciences, Inc.
Investigators
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Study Director: Li Tai, MD Avidity Biosciences, Inc.
More Information
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Responsible Party: Avidity Biosciences, Inc.
ClinicalTrials.gov Identifier: NCT05479981    
Other Study ID Numbers: AOC 1001-CS2
First Posted: July 29, 2022    Key Record Dates
Last Update Posted: July 21, 2023
Last Verified: July 2023

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Avidity Biosciences, Inc.:
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
Additional relevant MeSH terms:
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Muscular Dystrophies
Musculoskeletal Diseases
Myotonic Dystrophy
Muscular Diseases
Myotonic Disorders
Muscular Disorders, Atrophic
 Nervous System Diseases
Neurodegenerative Diseases
Neuromuscular Diseases
Heredodegenerative Disorders, Nervous System
Genetic Diseases, Inborn