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

Primary Purpose

DM1, Myotonic Dystrophy 1, Myotonic Dystrophy

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
AOC 1001
Placebo
Sponsored by
Avidity Biosciences, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

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, Avidity Biosciences, Avidity, AOC 1001, Myotonic Muscular Dystrophy

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

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 3

Arm 4

Arm Type

Experimental

Placebo Comparator

Experimental

Placebo Comparator

Arm Label

Part A Single Dose: AOC 1001 Dose Level 1

Part A Single Dose: Placebo

Part B Multiple Ascending Dose: AOC 1001 Dose Levels 2 & 3

Part B Multiple Ascending Dose: Placebo

Arm Description

AOC 1001 will be administered once.

Saline will be administered once.

AOC 1001 will be administered three times.

Saline will be administered three times.

Outcomes

Primary Outcome Measures

Frequency of treatment emergent adverse events (TEAEs)

Secondary Outcome Measures

Plasma pharmacokinetic (PK) parameters
Maximum plasma concentration (Cmax)
Plasma pharmacokinetic (PK) parameters
Time to maximum plasma concentration (Tmax)
Plasma pharmacokinetic (PK) parameters
Terminal Half-life (t1/2)
Plasma pharmacokinetic (PK) parameters
Area Under the Concentration-time Curve (AUC)
Urine pharmacokinetic (PK) parameters
fraction excreted (fe) in urine
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

First Posted
August 24, 2021
Last Updated
June 23, 2023
Sponsor
Avidity Biosciences, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT05027269
Brief Title
Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients
Acronym
MARINA
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
Study Type
Interventional

2. Study Status

Record Verification Date
June 2023
Overall Recruitment Status
Completed
Study Start Date
October 28, 2021 (Actual)
Primary Completion Date
February 14, 2023 (Actual)
Study Completion Date
February 14, 2023 (Actual)

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-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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
DM1, Myotonic Dystrophy 1, Myotonic Dystrophy, Myotonic Dystrophy Type 1 (DM1), Dystrophy Myotonic, Myotonic Disorders, Steinert Disease, Myotonic Muscular Dystrophy
Keywords
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

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Sequential Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
38 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Part A Single Dose: AOC 1001 Dose Level 1
Arm Type
Experimental
Arm Description
AOC 1001 will be administered once.
Arm Title
Part A Single Dose: Placebo
Arm Type
Placebo Comparator
Arm Description
Saline will be administered once.
Arm Title
Part B Multiple Ascending Dose: AOC 1001 Dose Levels 2 & 3
Arm Type
Experimental
Arm Description
AOC 1001 will be administered three times.
Arm Title
Part B Multiple Ascending Dose: Placebo
Arm Type
Placebo Comparator
Arm Description
Saline will be administered three times.
Intervention Type
Drug
Intervention Name(s)
AOC 1001
Intervention Description
AOC 1001 will be administered by intravenous (IV) infusion.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo will be administered by intravenous (IV) infusion.
Primary Outcome Measure Information:
Title
Frequency of treatment emergent adverse events (TEAEs)
Time Frame
Through study completion, up to Day 183
Secondary Outcome Measure Information:
Title
Plasma pharmacokinetic (PK) parameters
Description
Maximum plasma concentration (Cmax)
Time Frame
Through study completion, up to Day 183
Title
Plasma pharmacokinetic (PK) parameters
Description
Time to maximum plasma concentration (Tmax)
Time Frame
Through study completion, up to Day 183
Title
Plasma pharmacokinetic (PK) parameters
Description
Terminal Half-life (t1/2)
Time Frame
Through study completion, up to Day 183
Title
Plasma pharmacokinetic (PK) parameters
Description
Area Under the Concentration-time Curve (AUC)
Time Frame
Through study completion, up to Day 183
Title
Urine pharmacokinetic (PK) parameters
Description
fraction excreted (fe) in urine
Time Frame
Through study completion, up to Day 183
Title
AOC 1001 levels in muscle tissue
Time Frame
Through study completion, up to Day 183
Title
Change and percentage change from baseline in DMPK mRNA knockdown
Time Frame
Through study completion, up to Day 183
Title
Change and percentage change from baseline in Spliceopathy
Time Frame
Through study completion, up to Day 183

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility 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.
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

Plan to Share IPD
No
Links:
URL
https://www.aviditybiosciences.com/
Description
Related Info

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

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