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

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Brief Title

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

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

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.
    

Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Number and severity of treatment-emergent adverse events (TEAEs)

Secondary Outcome

 Plasma pharmacokinetic (PK) parameters

Condition

DM1

Intervention

Placebo

Study Arms / Comparison Groups

 AOC 1001
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.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

44

Start Date

July 2022

Completion Date

April 2025

Primary Completion Date

April 2025

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
      

Gender

All

Ages

18 Years - 66 Years

Accepts Healthy Volunteers

No

Contacts

Li Tai, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT05479981

Organization ID

AOC 1001-CS2


Responsible Party

Sponsor

Study Sponsor

Avidity Biosciences, Inc.


Study Sponsor

Li Tai, MD, Study Director, Avidity Biosciences, Inc.


Verification Date

July 2022