A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping
Pivotal Trial
This treatment is in the last trial phase before FDA approval.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
Ready to participate?
Review the details below, then apply to join this clinical trial.
At a Glance
Age
7 – 16
Sex
Male
Trial phase
Phase 3
Study type
Interventional
Purpose
Treatment
Participants needed
70 (estimated)
Sponsor
Avidity Biosciences, Inc. · Industry
Who this trial is looking for
This trial is looking for boys aged 7 to 16 with Duchenne Muscular Dystrophy (DMD) who have a specific gene mutation. Participants will receive an intravenous treatment and be monitored for health and safety.
Are You a Good Fit for This Trial?
Must be takingCorticosteroids
You may be able to join if
I am a male between 7 to 16 years old
I have been diagnosed with Duchenne Muscular Dystrophy (DMD)
I have a genetic test confirming a specific gene mutation
I can walk without assistance
I have been on stable steroid treatment for at least 6 months
I have completed required assessments before starting
You may not be able to join if
I have had previous cell or gene therapy
I have taken another oligonucleotide in the last 6 months
My lab test results are not within the required range
I am not on a stable growth hormone program for at least 1 month
I am not on a stable testosterone program for at least 1 month
I am not on a stable givinostat program for at least 6 months
Summarized in plain language from this trial's official eligibility criteria.
The full criteria are further down this page — only the research team can
confirm whether you qualify.
Think this trial could be right for you?
Answer a few quick questions to see if you may meet the eligibility requirements.
A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1044 for the treatment of Duchenne Muscular Dystrophy (DMD) with Gene Mutations Amenable to Exon 44 Skipping
Trial Locations
Loading…
Loading trial locations…
Facility
City
State
Country
Status
Eligibility Criteria
Key Inclusion Criteria:
* Ambulatory males with clinical and genetic diagnosis of DMD
* Acceptable genetic test confirming dystrophin gene mutation amenable to exon 44 skipping
* 7 to 16 years of age at time of consent
* TTR and NSAA assessment completed within the protocol specified parameters at …
Key Inclusion Criteria:
* Ambulatory males with clinical and genetic diagnosis of DMD
* Acceptable genetic test confirming dystrophin gene mutation amenable to exon 44 skipping
* 7 to 16 years of age at time of consent
* TTR and NSAA assessment completed within the protocol specified parameters at Screening
* On a stable regimen of corticosteroids (including Vamolorone) for at least 6 months prior to Day 1. Steroid regimen must be anticipated to remain stable.
Key Exclusion Criteria:
* Previous treatment cell or gene therapy.
* Treatment with another oligonucleotide within 6 months of informed consent (not including COVID-19 RNA vaccines).
* Lab values outside of the protocol specified range at Screening
* If on any of the following treatments (growth hormone, testosterone or givinostat), participants must be on a stable regimen and must plan to maintain it for the duration of the study. Participants will be excluded if regimen stability prior to informed consent is as follows:
* Less than 1 month, for growth hormone and/or testosterone
* Less than 6 months for givinostat
Picking one helps us show the most relevant trials first.
Your results are loading in the background — you can
change this any time from the results page.
Set your location to continue
Find My Trials uses your location to surface clinical trials near you.
Add your city or zip code to your profile and try again.
