All > Muscular Dystrophy

Cohort 2 for Duchenne Muscular Dystrophy

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Arkansas Children's Hospital, Little Rock, ARDuchenne Muscular DystrophyEDG-5506 Dose 3 - Drug
Eligibility
4 - 9
Male
What conditions do you have?
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Study Summary

This trial is studying safety, how a drug is absorbed and how it affects biomarkers in kids with Duchenne muscular dystrophy. It will be done in two parts.

Eligible Conditions
  • Duchenne Muscular Dystrophy

Treatment Effectiveness

Effectiveness Progress

1 of 3

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Study Objectives

2 Primary · 20 Secondary · Reporting Duration: 12 months

12 months
Incidence of abnormal clinical chemistry test results
Incidence of abnormal coagulation test results
Incidence of abnormal hematology test results
Incidence of abnormal urinalysis test results
Number of adverse events during treatment with EDG-5506 or placebo
Number of participants with changes in ECG PR interval
Number of participants with changes in ECG QRS interval
Number of participants with changes in ECG QT interval
Number of participants with changes in ECG QTc interval
Number of participants with changes in FVC
Number of participants with changes in clinical chemistry
Number of participants with changes in coagulation
Number of participants with changes in hematology
Number of participants with changes in neurological examination
Number of participants with changes in physical examination
Number of participants with changes in urinalysis
Number of participants with changes in vital signs
Pharmacokinetics as measured by steady state plasma concentration
Severity of adverse events during treatment with EDG-5506 or placebo
12 weeks
Change from Baseline in fast skeletal muscle troponin I
Change from Baseline in serum creatinine kinase
Change from Baseline in serum myoglobin

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Similar Trials

1
Part A - AME
2
Tadalafil
2
Tideglusib

Trial Design

3 Treatment Groups

Cohort 2
1 of 3
Cohort 3
1 of 3
Cohort 1
1 of 3

Experimental Treatment

27 Total Participants · 3 Treatment Groups

Primary Treatment: Cohort 2 · Has Placebo Group · Phase 2

Cohort 2Experimental Group · 2 Interventions: Placebo, EDG-5506 Dose 2 · Intervention Types: Drug, Drug
Cohort 3Experimental Group · 2 Interventions: Placebo, EDG-5506 Dose 3 · Intervention Types: Drug, Drug
Cohort 1Experimental Group · 2 Interventions: Placebo, EDG-5506 Dose 1 · Intervention Types: Drug, Drug
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Placebo
1995
Completed Phase 3
~2640

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 12 months

Who is running the clinical trial?

Edgewise Therapeutics, Inc.Lead Sponsor
7 Previous Clinical Trials
416 Total Patients Enrolled
Sam Collins, MBBS, PhDStudy ChairEdgewise Therapeutics, Inc.
4 Previous Clinical Trials
103 Total Patients Enrolled

Eligibility Criteria

Age 4 - 9 · Male Participants · 4 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
References

Frequently Asked Questions

Does this research involve volunteers who are below the age of twenty?

"This trial seeks patients ranging from 4 to 9 years old." - Anonymous Online Contributor

Unverified Answer

How safe is Cohort 2 for human participants?

"The safety of Cohort 2 scored a 2 on our scale, as there is evidence that it is safe to use but no data yet demonstrating its efficacy." - Anonymous Online Contributor

Unverified Answer

Are there any open positions available to participants in this investigation?

"The public records on clinicaltrials.gov confirm that this particular medical trial is actively seeking participants, with the first posting having been made on October 1st 2022 and its most recent update occurring in late-October of the same year." - Anonymous Online Contributor

Unverified Answer

What goals is this trial aiming to accomplish?

"The main aim of the trial, which will be monitored over 12 months, is to assess severity of any adverse events resulting from EDG-5506 and placebo. Additionally, secondary objectives for this experiment include observing changes in neurological examinations among all participants as well as alterations in fast skeletal muscle troponin I levels and coagulation records across all patients." - Anonymous Online Contributor

Unverified Answer

Which individuals would be ideal candidates for this clinical trial?

"To qualify for this trial, participants must meet the criteria of having muscular dystrophy (Duchenne) and should be between 4 to 9 years old. The study is currently searching for 27 enrollees." - Anonymous Online Contributor

Unverified Answer

What is the upper limit of participants in this medical research study?

"Indeed, the official data posted on clinicaltrials.gov attests to this experiment's active recruitment process. It was first listed on October 1st 2022 and recently updated on October 24th of the same year. The trial aims to enrol 27 patients from a single site." - 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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