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Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients (MARINA)

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ClinicalTrials.gov Identifier: NCT05027269
Recruitment Status : Completed
First Posted : August 30, 2021
Last Update Posted : February 5, 2024
Sponsor:
Information provided by (Responsible Party):
Avidity Biosciences, Inc.

Study Description
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Brief Summary:

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.


Condition or disease Intervention/treatment Phase
DM1 Myotonic Dystrophy 1 Myotonic Dystrophy Myotonic Dystrophy Type 1 (DM1) Dystrophy Myotonic Myotonic Disorders Steinert Disease Myotonic Muscular Dystrophy Drug: AOC 1001 Drug: Placebo Phase 1 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 38 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : October 28, 2021
Actual Primary Completion Date : February 14, 2023
Actual Study Completion Date : February 14, 2023

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Part A Single Dose: AOC 1001 Dose Level 1
AOC 1001 will be administered once.
 Drug: AOC 1001
AOC 1001 will be administered by intravenous (IV) infusion.
Placebo Comparator: Part A Single Dose: Placebo
Saline will be administered once.
 Drug: Placebo
Placebo will be administered by intravenous (IV) infusion.
Experimental: Part B Multiple Ascending Dose: AOC 1001 Dose Levels 2 & 3
AOC 1001 will be administered three times.
 Drug: AOC 1001
AOC 1001 will be administered by intravenous (IV) infusion.
Placebo Comparator: Part B Multiple Ascending Dose: Placebo
Saline will be administered three times.
 Drug: Placebo
Placebo will be administered by intravenous (IV) infusion.


Outcome Measures
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Primary Outcome Measures :
  1. Frequency of treatment emergent adverse events (TEAEs) [ Time Frame: Through study completion, up to Day 183 ]

Secondary Outcome Measures :
  1. Plasma pharmacokinetic (PK) parameters [ Time Frame: Through study completion, up to Day 183 ]
    Maximum plasma concentration (Cmax)

  2. Plasma pharmacokinetic (PK) parameters [ Time Frame: Through study completion, up to Day 183 ]
    Time to maximum plasma concentration (Tmax)

  3. Plasma pharmacokinetic (PK) parameters [ Time Frame: Through study completion, up to Day 183 ]
    Terminal Half-life (t1/2)

  4. Plasma pharmacokinetic (PK) parameters [ Time Frame: Through study completion, up to Day 183 ]
    Area Under the Concentration-time Curve (AUC)

  5. Urine pharmacokinetic (PK) parameters [ Time Frame: Through study completion, up to Day 183 ]
    fraction excreted (fe) in urine

  6. AOC 1001 levels in muscle tissue [ Time Frame: Through study completion, up to Day 183 ]
  7. Change and percentage change from baseline in DMPK mRNA knockdown [ Time Frame: Through study completion, up to Day 183 ]
  8. Change and percentage change from baseline in Spliceopathy [ Time Frame: Through study completion, up to Day 183 ]

Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

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): NCT05027269


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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Additional Information:

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Responsible Party: Avidity Biosciences, Inc.
ClinicalTrials.gov Identifier: NCT05027269    
Other Study ID Numbers: AOC 1001-CS1
First Posted: August 30, 2021    Key Record Dates
Last Update Posted: February 5, 2024
Last Verified: February 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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
Avidity Biosciences
Avidity
AOC 1001
Myotonic Muscular Dystrophy
Additional relevant MeSH terms:
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Muscular Dystrophies
Myotonic Dystrophy
Myotonic Disorders
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
 Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases