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Small Molecule
EDG-5506 for Becker Muscular Dystrophy
Phase 2
Recruiting
Research Sponsored by Edgewise Therapeutics, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Able to complete the 100-meter timed test in < 150 seconds.
Adults (aged 18 to 50 years, inclusive) with a documented dystrophin mutation and phenotype consistent with Becker muscular dystrophy
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 12 months (canyon cohorts 1, 2, 4, 5), 18 months (grand canyon cohort 6)
Awards & highlights
Study Summary
This trial is testing a new drug to see if it's safe and effective for people with Becker muscular dystrophy.
Who is the study for?
This trial is for adolescents (12-17) and adults (18-50) with Becker muscular dystrophy confirmed by genetic testing. Participants must be able to complete a 100-meter walk within certain times, perform specific physical assessments, and have been ambulatory beyond certain ages with or without steroids.Check my eligibility
What is being tested?
The GRAND CANYON study tests the safety and effectiveness of EDG-5506 at different doses (5 mg, 10 mg, 12.5 mg) compared to a placebo in treating Becker muscular dystrophy. It's randomized and double-blind, meaning neither participants nor researchers know who gets the drug or placebo.See study design
What are the potential side effects?
While specific side effects of EDG-5506 are not listed here, common side effects in trials like this may include gastrointestinal symptoms, fatigue, muscle pain or cramps, headaches, and potential allergic reactions.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I can walk 100 meters in less than 150 seconds.
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I am between 18 and 50 years old with a confirmed diagnosis of Becker muscular dystrophy.
Select...
I was able to walk after 16 without steroids and after 18 with steroids.
Select...
I am a teenager with a confirmed genetic mutation for Becker muscular dystrophy.
Select...
I can do a specific physical test and score between 5 and 32.
Select...
I am 18-50 years old with Becker muscular dystrophy, and I could walk after 16 without steroids or after 18 with them.
Select...
I can complete a physical ability test with a specific score range.
Select...
I can walk 100 meters in less than 150 seconds.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 12 months (canyon cohorts 1, 2, 4, 5), 18 months (grand canyon cohort 6)
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~12 months (canyon cohorts 1, 2, 4, 5), 18 months (grand canyon cohort 6)
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Change from Baseline in serum Creatine Kinase
Number of adverse events in those treated with EDG-5506 or placebo
Severity of adverse events in those treated with EDG-5506 or placebo
Secondary outcome measures
Change from Baseline in 100-meter timed test
Change from Baseline in growth as assessed by height centile on World Health Organization growth charts
Change from Baseline in stride velocity (95th percentile)
+6 moreTrial Design
5Treatment groups
Experimental Treatment
Group I: Adult Cohort 6Experimental Treatment2 Interventions
Drug: EDG-5506 Drug: Placebo
Group II: Adult Cohort 2Experimental Treatment2 Interventions
Drug: EDG-5506 Drug: Placebo
Group III: Adult Cohort 1Experimental Treatment2 Interventions
Drug: EDG-5506 Drug: Placebo
Group IV: Adolescent Cohort 5Experimental Treatment2 Interventions
Drug: EDG-5506 Drug: Placebo
Group V: Adolescent Cohort 4Experimental Treatment2 Interventions
Drug: EDG-5506 Drug: Placebo
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Placebo
1995
Completed Phase 3
~2670
Find a Location
Who is running the clinical trial?
SysnavIndustry Sponsor
8 Previous Clinical Trials
415 Total Patients Enrolled
Medpace, Inc.Industry Sponsor
94 Previous Clinical Trials
29,445 Total Patients Enrolled
ImagingNMDUNKNOWN
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I can complete a mobility test with a score between 10 and 32.I can walk 100 meters in less than 200 seconds, with or without help from a device.I can do a specific physical test and score between 5 and 32.I can walk 100 meters in less than 150 seconds.I am between 18 and 50 years old with a confirmed diagnosis of Becker muscular dystrophy.I was able to walk after 16 without steroids and after 18 with steroids.I am a teenager with a confirmed genetic mutation for Becker muscular dystrophy.My lung function is severely reduced or I need help breathing during the day.I am 18-50 years old with Becker muscular dystrophy, and I could walk after 16 without steroids or after 18 with them.I have taken oral steroids for bone marrow disease in the last 6 months.I can complete a physical ability test with a specific score range.Your heart's pumping ability is less than 40% as shown in a heart ultrasound.I can walk 100 meters in less than 150 seconds.
Research Study Groups:
This trial has the following groups:- Group 1: Adult Cohort 6
- Group 2: Adolescent Cohort 5
- Group 3: Adult Cohort 1
- Group 4: Adult Cohort 2
- Group 5: Adolescent Cohort 4
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.
Becker Muscular Dystrophy Patient Testimony for trial: Trial Name: NCT05291091 — Phase 2
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
What particular objectives is this clinical trial attempting to achieve?
"This 12-month medical trial will be primarily evaluating the severity of adverse effects in those treated with EDG-5506 or placebo. Additional objectives include assessing changes from baseline in 10m walking/running test, North Star Ambulatory Assessment scale, and 100m timed tests for adult participants."
Answered by AI
Is this clinical trial currently recruiting participants?
"Affirmative. Data hosted on clinicaltrials.gov suggests that recruitment for this trial is ongoing, with the first post appearing on July 6th 2022 and updates as recent as November 2nd 2022. The study seeks to enrol 66 patients from 9 separate medical facilities."
Answered by AI
Are there any other centers conducting this trial within the same state?
"This clinical trial is actively recruiting patients from 9 different locations across the United States, including Washington University School of Medicine in Saint Louis, Kennedy Krieger Institute in Baltimore, and University Kansas Medical Center in Kansas City."
Answered by AI
Are there opportunities to participate in this medical experiment?
"Those with Becker Muscular dystrophy and between the ages of 12 to 50 can apply for this trial. The total amount of available slots is 66 patients."
Answered by AI
What is the current participant enrollment for this trial?
"In order to commence the trial, Medpace, Inc. requires 66 participants who satisfy all study entry criteria. The medical research organisation will be collecting data from two sites based in Saint Louis and Baltimore respectively."
Answered by AI
Has the first dosage of EDG-5506 received its authorization from the Food and Drug Administration?
"The safety of EDG-5506 Dose 1 was evaluated and found to be a 2, as Phase 2 trials typically lack evidence related to efficacy but do feature some data pertaining to safety."
Answered by AI
Is the research team actively seeking elderly participants for this trial?
"To participate in this research, potential patients must be aged 12 to 50. Person younger than 18 and older then 65 are not eligible for the trial; however they may qualify for 60 and 49 other trials respectively."
Answered by AI
Who else is applying?
What site did they apply to?
University of Massachusetts Memorial Medical Center
What portion of applicants met pre-screening criteria?
Met criteria
Did not meet criteria
How many prior treatments have patients received?
0
1
Why did patients apply to this trial?
My son DMD treatment 14years age. Hey! My name is Dawid and I’m from Poland. When I was like 15 I realised that something was wrong with my legs and body because there was a lot of problems with stand up and run directly. Sport lesson was difficult and I couldn’t compare with my friends. When doctors told me that I have BMD I was treated with mexiletine but there’s no possibility to buy it in Poland so please if you can help me some how please I’m begging you to help me.
PatientReceived 2+ prior treatments
How responsive is this trial?
Typically responds via
Email
Average response time
- < 2 Days
Most responsive sites:
- University of Massachusetts Memorial Medical Center: < 48 hours
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