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Exon Skipping Agent
AOC 1044 for Duchenne Muscular Dystrophy (EXPLORE44 Trial)
Phase 1 & 2
Recruiting
Research Sponsored by Avidity Biosciences, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up day 1-2 (0-24 hours after first dose) (part a); day 1-2 (0-24 hours after first dose) (part b)
Awards & highlights
EXPLORE44 Trial Summary
This trial studies a new drug to treat Duchenne muscular dystrophy. It tests safety, how well it works and how it affects the body in healthy adults and people with Duchenne.
Who is the study for?
This trial is for healthy adults and those with Duchenne Muscular Dystrophy (DMD) who can benefit from exon 44 skipping. Participants must be aged 18-55 years with a BMI of 18.5 to 32 kg/m2.Check my eligibility
What is being tested?
The study tests AOC 1044, which could potentially treat DMD by skipping exon 44 in the gene mutation. It's given in single or multiple doses to see how safe it is and how the body responds.See study design
What are the potential side effects?
Possible side effects are not detailed here but will likely include reactions at the injection site, potential liver enzyme changes, and other symptoms related to altering muscle protein production.
EXPLORE44 Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ day 1-2 (0-24 hours after first dose) (part a); day 1-2 (0-24 hours after first dose) (part b)
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~day 1-2 (0-24 hours after first dose) (part a); day 1-2 (0-24 hours after first dose) (part b)
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Incidence of treatment-emergent adverse events (TEAEs)
Secondary outcome measures
Absolute change from baseline in dystrophin protein level in skeletal muscle (Part B only)
Change from baseline in exon skipping as measured in skeletal muscle (Part B only)
PMO44 levels in skeletal muscle tissue
+3 moreEXPLORE44 Trial Design
4Treatment groups
Experimental Treatment
Placebo Group
Group I: AOC 1044-CS1 Part B - Multiple Ascending Dose Levels 1-3Experimental Treatment1 Intervention
AOC 1044 will be administered three times.
Group II: AOC 1044-CS1 Part A - Single Dose Levels 1-5Experimental Treatment1 Intervention
AOC 1044 will be administered once.
Group III: AOC 1044-CS1 Part A - Single Dose: PlaceboPlacebo Group1 Intervention
Placebo will be administered once.
Group IV: AOC 1044-CS1 Part B - Multiple Ascending Dose: PlaceboPlacebo Group1 Intervention
Placebo will be administered three times.
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Logistics
Participation is compensated
You will be compensated for participating in this trial.
Who is running the clinical trial?
Avidity Biosciences, Inc.Lead Sponsor
5 Previous Clinical Trials
621 Total Patients Enrolled
Mark Stahl, MD, PhDStudy DirectorAvidity Biosciences, Inc.
1 Previous Clinical Trials
24 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have previously undergone cell or gene therapy.My biceps muscles cannot be biopsied.I weigh at least 23 kg.I was diagnosed with DMD or showed symptoms by age 6.I am currently taking or have recently taken prescription or over-the-counter drugs.My DMD gene mutation can be treated by skipping exon 44.I haven't taken any exon 44 skipping drugs in the last 6 months.I am between 7 and 27 years old.I am between 18 and 55 years old.I have not donated blood or plasma within the last 16 weeks.I have uncontrolled high blood pressure or diabetes.
Research Study Groups:
This trial has the following groups:- Group 1: AOC 1044-CS1 Part B - Multiple Ascending Dose Levels 1-3
- Group 2: AOC 1044-CS1 Part A - Single Dose: Placebo
- Group 3: AOC 1044-CS1 Part A - Single Dose Levels 1-5
- Group 4: AOC 1044-CS1 Part B - Multiple Ascending Dose: Placebo
Awards:
This trial has 0 awards, including:Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Can individuals aged 55 and above participate in this trial?
"For this clinical trial, candidates must be aged over 7 and under 45 for consideration."
Answered by AI
Could I potentially qualify for participation in this experiment?
"This trial seeks 64 participants who are between 7 and 45 years old, suffering from Duchenne or Becker Muscular dystrophy."
Answered by AI
Is this research experiment currently registering participants?
"The trial is still enrolling participants, as evidenced by the recent update to its posting on clinicaltrials.gov dated January 2nd 2023. Initially posted November 9th 2022, it has been open for recruitment since that time."
Answered by AI
What is the criterion for admission to this experiment?
"Affirmative. The clinical trial, which was initially posted on November 9th 2022 and has since been updated as of January 2nd 2023, is actively recruiting participants according to the data hosted on clinicaltrials.gov. Approximately 64 patients must be enrolled from 1 medical centre."
Answered by AI
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