Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping
NCT ID: NCT05670730
Last Updated: 2025-07-14
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE1/PHASE2
70 participants
INTERVENTIONAL
2022-11-09
2024-11-25
Brief Summary
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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.
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
SEQUENTIAL
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
TREATMENT
QUADRUPLE
Study Groups
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AOC 1044-CS1 Part A - Single Dose Levels 1-5
AOC 1044 will be administered once.
AOC 1044
AOC 1044 will be administered via intravenous (IV) infusion
AOC 1044-CS1 Part A - Single Dose: Placebo
Placebo will be administered once.
Placebo
Placebo will be administered via intravenous (IV) infusion.
AOC 1044-CS1 Part B - Multiple Ascending Dose Levels 1-3
AOC 1044 will be administered three times.
AOC 1044
AOC 1044 will be administered via intravenous (IV) infusion
AOC 1044-CS1 Part B - Multiple Ascending Dose: Placebo
Placebo will be administered three times.
Placebo
Placebo will be administered via intravenous (IV) infusion.
Interventions
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AOC 1044
AOC 1044 will be administered via intravenous (IV) infusion
Placebo
Placebo will be administered via intravenous (IV) infusion.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Body mass index (BMI) of 18.5 to 32.0 kg/m2
* 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
Exclusion Criteria
* 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
Part B:
* 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
7 Years
55 Years
MALE
Yes
Sponsors
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Avidity Biosciences, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Carmen Castrillo, MD
Role: STUDY_DIRECTOR
Avidity Biosciences, Inc.
Locations
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Arkansas Children's
Little Rock, Arkansas, United States
UCSD
La Jolla, California, United States
Stanford University
Palo Alto, California, United States
Rare Disease Research - Atlanta
Atlanta, Georgia, United States
University of Iowa
Iowa City, Iowa, United States
University of Massachusetts Chan Medical School
Worcester, Massachusetts, United States
Gillette Children's
Saint Paul, Minnesota, United States
Rare Disease Research NC
Hillsborough, North Carolina, United States
Abigail Research Institute at Nationwide Children's Hospital
Columbus, Ohio, United States
Worldwide Clinical Trials (Part A only)
San Antonio, Texas, United States
Countries
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Other Identifiers
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AOC 1044-CS1
Identifier Type: -
Identifier Source: org_study_id
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