Clinical trial NCT06244082: Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping
Official title: A Phase 2 Open-label Study to Evaluate the Pharmacodynamics and Long-Term Safety and Tolerability of AOC 1044 Administered Intravenously to DMD Participants With Mutations Amenable to Exon 44 Skipping
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: DMD
Duchenne Muscular Dystrophy
Duchenne
Exon 44
Keywords: EXPLORE44
EXPLORE44-OLE
EXPLORE44 OLE
Avidity Biosciences
Avidity
AOC 1044
AOC 1044-CS1
AOC 1044-CS2
AOC
Lead sponsor: Avidity Biosciences, Inc.
Sponsor class: INDUSTRY
First posted: 2024-02-06
Enrollment: 39
Sex: MALE
Minimum age (years): 7
Maximum age (years): 27
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: AOC 1044-CS2 (EXPLORE44-OLE) is an Open-label Study to Evaluate the Long-Term Safety and Tolerability of AOC 1044 Administered Intravenously to DMD Participants with Mutations Amenable to Exon 44 Skipping.

Detailed description: AOC 1044-CS2 (EXPLORE44-OLE) is an open label, extension study to Part B of AOC 1044-CS1 (EXPLORE44). AOC 1044-CS2 is designed to evaluate the long-term safety, tolerability, pharmacokinetics, and exploratory efficacy of AOC 1044.

All participants who enroll in AOC 1044-CS2 will receive active treatment. The treatment period is 2 years with IV dosing every 6 weeks.

Once participants have completed active treatment, they will be followed through a 3-month safety follow-up period. The sponsor may extend active treatment beyond 2 years at a future timepoint.

Eligibility criteria: Key Inclusion Criteria

Rollover Participants:

* Satisfactorily completed AOC 1044-CS1 (EXPLORE44) as determined by the Investigator and Sponsor
* No significant tolerability issues with AOC 1044

De novo Participants:

* 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

Rollover Participants:

* Presence of any new condition or worsening of existing condition that could affect a participant\&#39;s safety or ability to comply with study procedures

De novo Participants:

* Serum hemoglobin \&lt; 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

Primary outcomes:
measure: Incidence of Treatment Emergent Adverse Events (TEAEs)
timeFrame: Through study completion (approximately 2 years)
Secondary outcomes:
measure: Change from baseline in serum creatine kinase concentration at Study Weeks 24, 48, and 102
timeFrame: Through study completion (approximately 2 years)
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: Arkansas Children&amp;#39;s Hospital
state: Arkansas
city: La Jolla
country: United States
facility: University of California, San Diego, Rady&#39;s Children&#39;s Hospital
state: California
city: Sacramento
country: United States
facility: UC Davis Medical Center
state: California
city: San Carlos
country: United States
facility: Lucille Packard Children&amp;#39;s Hospital at Stanford
state: California
city: Atlanta
country: United States
facility: Rare Disease Research - Atlanta
state: Georgia
city: Worcester
country: United States
facility: University of Massachusetts Medical School
state: Massachusetts
city: St. Paul
country: United States
facility: Gillette Children&#39;s Specialty Healthcare
state: Minnesota
city: Hillsborough
country: United States
facility: Rare Disease Research
state: North Carolina
city: Columbus
country: United States
facility: Abigail Wexner Research Institute at Nationwide Children&#39;s Hospital
state: Ohio
city: Denton
country: United States
facility: Neurology Rare Disease Center
state: Texas
Study officials:
affiliation: Avidity Biosciences, Inc.
name: Carmen Castrillo, MD
role: STUDY_DIRECTOR
References: