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AOC 1044-CS1 Part A - Single Dose Levels 1-5 for Duchenne Muscular Dystrophy

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Worldwide Clinical Trials (Part A only), San Antonio, TXDuchenne Muscular Dystrophy+1 MoreAOC 1044 - Drug
Eligibility
7 - 45
Male
What conditions do you have?
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Study 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.

Eligible Conditions
  • Duchenne Muscular Dystrophy
  • Exon 44

Treatment Effectiveness

Effectiveness Progress

1 of 3

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1
Cohort 4 - 7 mg single-dose
1
Tadalafil
1
Vamorolone

Study Objectives

1 Primary · 6 Secondary · Reporting Duration: Day 1-2 (0-24 hours after first dose) (Part A); Day 1-2 (0-24 hours after first dose) (Part B)

Baseline, Week 16
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)
Percentage change from baseline in dystrophin protein level in skeletal muscle (Part B only)
Day 1
Urine pharmacokinetic parameters
Week 16
PMO44 levels in skeletal muscle tissue
Week 12
Plasma pharmacokinetic (PK) parameters
Day 169
Incidence of treatment-emergent adverse events (TEAEs)

Trial Safety

Safety Progress

1 of 3

Similar Trials

1
Cohort 4 - 7 mg single-dose
2
Tadalafil
2
Vamorolone

Trial Design

4 Treatment Groups

AOC 1044-CS1 Part A - Single Dose Levels 1-5
1 of 4
AOC 1044-CS1 Part B - Multiple Ascending Dose Levels 1-3
1 of 4
AOC 1044-CS1 Part A - Single Dose: Placebo
1 of 4
AOC 1044-CS1 Part B - Multiple Ascending Dose: Placebo
1 of 4

Experimental Treatment

Non-Treatment Group

64 Total Participants · 4 Treatment Groups

Primary Treatment: AOC 1044-CS1 Part A - Single Dose Levels 1-5 · Has Placebo Group · Phase 1 & 2

AOC 1044-CS1 Part A - Single Dose Levels 1-5
Drug
Experimental Group · 1 Intervention: AOC 1044 · Intervention Types: Drug
AOC 1044-CS1 Part B - Multiple Ascending Dose Levels 1-3
Drug
Experimental Group · 1 Intervention: AOC 1044 · Intervention Types: Drug
AOC 1044-CS1 Part A - Single Dose: Placebo
Drug
PlaceboComparator Group · 1 Intervention: Placebo · Intervention Types: Drug
AOC 1044-CS1 Part B - Multiple Ascending Dose: Placebo
Drug
PlaceboComparator Group · 1 Intervention: Placebo · Intervention Types: Drug

Trial Logistics

Logistics

Participation is compensated

You will be compensated for participating in this trial.

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: day 1-2 (0-24 hours after first dose) (part a); day 1-2 (0-24 hours after first dose) (part b)

Who is running the clinical trial?

Avidity Biosciences, Inc.Lead Sponsor
4 Previous Clinical Trials
610 Total Patients Enrolled
Mark Stahl, MD, PhDStudy DirectorAvidity Biosciences, Inc.

Eligibility Criteria

Age 7 - 45 · Male Participants · 11 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You are able to walk or use a wheelchair.
Your body mass index (BMI) falls between 18.5 and 32.0 kg/m2.
You have been diagnosed with DMD or started experiencing symptoms before the age of 6.
References

Frequently Asked Questions

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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

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