Phase 1/2 Study to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients

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Brief Title

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

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

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 3 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.
    


Study Phase

Phase 1/Phase 2

Study Type

Interventional


Primary Outcome

Frequency of treatment emergent adverse events (TEAEs)

Secondary Outcome

 Plasma pharmacokinetic (PK) parameters

Condition

DM1

Intervention

AOC 1001

Study Arms / Comparison Groups

 Part A Single Dose: AOC 1001 Dose Level 1
Description:  AOC 1001 will be administered once.

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

October 28, 2021

Completion Date

September 2023

Primary Completion Date

September 2023

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.
      

Gender

All

Ages

18 Years - 65 Years

Accepts Healthy Volunteers

No

Contacts

Li Tai, MD, 858-771-7038, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT05027269

Organization ID

AOC 1001-CS1


Responsible Party

Sponsor

Study Sponsor

Avidity Biosciences, Inc.


Study Sponsor

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


Verification Date

June 2022