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Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping (EXPLORE44)

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ClinicalTrials.gov Identifier: NCT05670730
Recruitment Status : Recruiting
First Posted : January 4, 2023
Last Update Posted : September 28, 2023
Sponsor:
Information provided by (Responsible Party):
Avidity Biosciences, Inc.

Study Description
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Brief Summary:

AOC 1044-CS1 (EXPLORE44) is a Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of single and multiple ascending doses of AOC 1044 in healthy adult volunteers and participants with DMD mutations amenable to exon 44 skipping.

Part A is a single dose design with multiple cohorts (dose levels) in healthy adult volunteers.

Part B is a multiple-ascending dose design with 3 cohorts (dose levels) in participants with Duchenne.


Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Exon 44 Drug: AOC 1044 Drug: Placebo Phase 1 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 64 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Intervention Model Description:

AOC 1044-CS1 (EXPLORE44) is a 2-part study:

Part A: 5 cohorts with single ascending doses conducted in healthy adult volunteers

Part B: 3 cohorts with multiple ascending doses in participants with DMD mutations amenable to exon 44 skipping
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 1/2, Randomized, Placebo-controlled, Double-blind, Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamic Effects of Single and Multiple Ascending Doses of AOC 1044 Administered Intravenously to Healthy Adult Volunteers and Participants With DMD Mutations Amenable to Exon 44 Skipping
Actual Study Start Date : November 9, 2022
Estimated Primary Completion Date : March 2025
Estimated Study Completion Date : March 2025

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: AOC 1044-CS1 Part A - Single Dose Levels 1-5
AOC 1044 will be administered once.
 Drug: AOC 1044
AOC 1044 will be administered via intravenous (IV) infusion
Placebo Comparator: AOC 1044-CS1 Part A - Single Dose: Placebo
Placebo will be administered once.
 Drug: Placebo
Placebo will be administered via intravenous (IV) infusion.
Other Name: Saline
Experimental: AOC 1044-CS1 Part B - Multiple Ascending Dose Levels 1-3
AOC 1044 will be administered three times.
 Drug: AOC 1044
AOC 1044 will be administered via intravenous (IV) infusion
Placebo Comparator: AOC 1044-CS1 Part B - Multiple Ascending Dose: Placebo
Placebo will be administered three times.
 Drug: Placebo
Placebo will be administered via intravenous (IV) infusion.
Other Name: Saline


Outcome Measures
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Primary Outcome Measures :
  1. Incidence of treatment-emergent adverse events (TEAEs) [ Time Frame: Through study completion, up to Day 85 (Part A) or Day 169 (Part B) ]

Secondary Outcome Measures :
  1. Plasma pharmacokinetic (PK) parameters [ Time Frame: Through Week 8 (Part A); Through Week 12 (Part B) ]
    Maximum plasma concentration (Cmax) of AOC 1044

  2. Plasma pharmacokinetic (PK) parameters [ Time Frame: Through Week 8 (Part A); Through Week 12 (Part B) ]
    Terminal half-life (T1/2) of AOC 1044

  3. Plasma pharmacokinetic (PK) parameters [ Time Frame: Through Week 8 (Part A); Through Week 12 (Part B) ]
    Area under the concentration-time curve (AUC) of AOC 1044

  4. PMO44 levels in skeletal muscle tissue [ Time Frame: Through Week 4 (Part A); Through Week 16 (Part B) ]
  5. Urine pharmacokinetic parameters [ Time Frame: Day 1-2 (0-24 hours after first dose) (Part A); Day 1-2 (0-24 hours after first dose) (Part B) ]
    Fraction of PMO44 excreted in urine

  6. Change from baseline in exon skipping as measured in skeletal muscle (Part B only) [ Time Frame: Baseline, Week 16 ]
  7. Absolute change from baseline in dystrophin protein level in skeletal muscle (Part B only) [ Time Frame: Baseline, Week 16 ]
  8. Percentage change from baseline in dystrophin protein level in skeletal muscle (Part B only) [ Time Frame: Baseline, Week 16 ]

Eligibility Criteria
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Ages Eligible for Study:   7 Years to 55 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   Yes
Criteria

Part A:

Key Inclusion Criteria:

  • Aged 18 to 55 years, inclusive, at the time of informed consent
  • Body mass index (BMI) of 18.5 to 32.0 kg/m2

Key Exclusion Criteria:

  • Clinically significant abnormalities in laboratory results, ECGs, or vitals
  • Current or recent use of prescription or nonprescription drugs
  • Positive drug/alcohol test at Screening or Day -1
  • Elevated blood pressure (BP) >130/80 mmHg at Screening
  • Participation in a clinical study in which an investigational product was received within 1 month of screening or 5 half-lives of the investigational product
  • Blood or plasma donation within 16 weeks of planned AOC 1044 administration Note: Other protocol defined Inclusion/Exclusion criteria may apply

Part B:

Key Inclusion Criteria:

  • Aged 7 to 27 years, inclusive, at the time of informed consent
  • Clinical diagnosis of DMD or clear onset of DMD symptoms at or before the age of 6 years
  • Confirmation of DMD gene mutation amenable to exon 44 skipping
  • Weight ≥ 23 kg

  • Ambulatory or non-ambulatory

    • Ambulatory participants: LVEF ≥50% and FVC≥50%
    • Non-ambulatory participants: LVEF ≥45% and FVC≥40%
  • PUL 2.0 entry item A ≥3
  • If on corticosteroids, stable dose for 30 days before screening and throughout the study

Key Exclusion Criteria:

  • Biceps brachii muscles unsuitable for biopsy
  • Serum hemoglobin < lower limit of normal
  • Uncontrolled hypertension or diabetes
  • Prior treatment with any cell or gene therapy
  • Prior treatment with another exon 44 skipping agent within 6 months prior to informed consent
  • Recently treated with an investigational drug
  • History of multiple drug allergies
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): NCT05670730


Contacts
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Contact: Avidity Biosciences, Inc. 858-771-7038 medinfo@aviditybio.com

Locations
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United States, California
Stanford University Recruiting
Palo Alto, California, United States, 94304
Contact: Rabia Farooquee         
United States, Georgia
Rare Disease Research - Atlanta Recruiting
Atlanta, Georgia, United States, 30329
Contact: Brenda Almaras         
United States, Texas
Worldwide Clinical Trials (Part A only) Recruiting
San Antonio, Texas, United States, 78217
Sponsors and Collaborators
Avidity Biosciences, Inc.
Investigators
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Study Director: Mark Stahl, MD, PhD Avidity Biosciences, Inc.
More Information
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Responsible Party: Avidity Biosciences, Inc.
ClinicalTrials.gov Identifier: NCT05670730    
Other Study ID Numbers: AOC 1044-CS1
First Posted: January 4, 2023    Key Record Dates
Last Update Posted: September 28, 2023
Last Verified: September 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.:
EXPLORE44
Avidity Biosciences
Avidity
AOC 1044
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
 Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked