AOC 1044 for Duchenne Muscular Dystrophy
(EXPLORE44OLE Trial)
Trial Summary
Do I have to stop taking my current medications for the trial?
The trial protocol does not specify if you need to stop taking your current medications. However, if you are on corticosteroids, you must be on a stable dose for 30 days before screening and throughout the study.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications, but if you are on corticosteroids, you must be on a stable dose for 30 days before screening and throughout the study.
What data supports the idea that AOC 1044 for Duchenne Muscular Dystrophy is an effective treatment?
The available research does not provide specific data on AOC 1044 for Duchenne Muscular Dystrophy. Instead, it discusses other treatments like drisapersen and golodirsen, which are used for similar purposes. These treatments work by helping the body produce a partially functional version of a protein called dystrophin, which is missing in people with Duchenne Muscular Dystrophy. While these studies show some promise in increasing dystrophin production, they do not provide direct evidence for the effectiveness of AOC 1044.12345
What data supports the effectiveness of the drug AOC 1044 for Duchenne Muscular Dystrophy?
Research on similar treatments, like drisapersen and eteplirsen, shows that exon-skipping drugs can help produce partially functional dystrophin, which is important for muscle health in Duchenne Muscular Dystrophy patients. These studies suggest that drugs like AOC 1044, which may work in a similar way, could potentially be effective.12345
What safety data exists for AOC 1044 (Delpacibart zotadirsen) for Duchenne Muscular Dystrophy?
The provided research does not contain any safety data for AOC 1044, Delpacibart zotadirsen, del-zota, or AOC-1044. The studies mentioned focus on the safety and efficacy of mometasone furoate and beclomethasone dipropionate for asthma treatment, which are unrelated to AOC 1044 or Duchenne Muscular Dystrophy.678910
Is the drug AOC 1044 a promising treatment for Duchenne Muscular Dystrophy?
Yes, AOC 1044 is a promising treatment for Duchenne Muscular Dystrophy. It uses a method called exon skipping, which helps produce a shorter but functional version of the dystrophin protein. This approach has shown potential in improving muscle function and strength, making it one of the most promising treatments for this condition.1112131415
What makes the drug AOC 1044 unique for treating Duchenne Muscular Dystrophy?
AOC 1044 is unique because it uses antisense oligonucleotide (AO) technology to skip specific exons in the dystrophin gene, potentially restoring the production of a functional dystrophin protein. This approach is part of a novel exon-skipping therapy that targets the genetic root of Duchenne Muscular Dystrophy, offering a personalized treatment option based on individual genetic mutations.1112131415
What is the purpose of this trial?
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.
Research Team
Carmen Castrillo, MD
Principal Investigator
Avidity Biosciences, Inc.
Eligibility Criteria
This trial is for individuals with Duchenne Muscular Dystrophy (DMD) who have specific genetic mutations that can be treated by skipping exon 44. Participants must have completed a prior study, EXPLORE44, without significant issues tolerating the treatment.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive AOC 1044 intravenously every 6 or 8 weeks for approximately 2 years
Follow-up
Participants are monitored for safety and effectiveness after treatment
Open-label extension
Participants may continue to receive treatment beyond 2 years if the sponsor extends the study
Treatment Details
Interventions
- AOC 1044
Find a Clinic Near You
Who Is Running the Clinical Trial?
Avidity Biosciences, Inc.
Lead Sponsor