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Gene Therapy
rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy
Phase 1 & 2
Waitlist Available
Led By Kevin Flanigan, MD
Research Sponsored by Kevin Flanigan
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 2 years
Awards & highlights
Summary
This trial will test a new gene therapy for Duchenne Muscular Dystrophy using a modified intravascular limb infusion procedure to deliver the vector to each lower limb of the subject.
Eligible Conditions
- Duchenne Muscular Dystrophy
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 2 years
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~2 years
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Number of Unanticipated Grade III or Higher Treatment-Related Toxicities
Secondary outcome measures
Expression of GALGT2 as Demonstrated by Immunofluorescent Staining With Anti-CT Epitope Antibodies or WFA Lectin in Muscle Biopsy Sections at 120 Days Post Injection (Cohort 1) and 90 Days Post-injection (Cohort 2).
GALGT2 Protein Expression Quantified by Western Blot and Assessed by Densitometry in Muscle Biopsy Tissue at 120 Days Post-injection (Cohort 1) and 90 Days Post-injection (Cohort 2)
Other outcome measures
Number of Meters Walked During the 6 Minute Walk Test
Score of Muscle Function Using the The North Star Ambulatory Assessment (NSAA).
Strength of the Bilateral Knee Flexors and Extensors During the Maximal Voluntary Isometric Strength Test.
+1 moreTrial Design
2Treatment groups
Experimental Treatment
Group I: Cohort 2 (Dose Escalation) rAAVrh74.MCK.GALGT2Experimental Treatment1 Intervention
N=3 [5 x E13 vg/kg per leg, delivered bilaterally (total 1.0 x E14 vg/kg)]
Group II: Cohort 1 (Minimal Efficacious Dose) rAAVrh74.MCK.GALGT2Experimental Treatment1 Intervention
N = 3 [2.5 x E13 vg/kg per leg, delivered bilaterally (total 5.0 x E13 vg/kg)]
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Who is running the clinical trial?
Kevin FlaniganLead Sponsor
3 Previous Clinical Trials
5 Total Patients Enrolled
Kevin Flanigan, MDPrincipal InvestigatorNationwide Children's Hospital
4 Previous Clinical Trials
30 Total Patients Enrolled
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Eligibility Criteria:
This trial includes the following eligibility criteria:- You have been diagnosed with an autoimmune disease or are currently receiving treatment for one.You can straighten your knee completely without any difficulty or help.You are currently receiving or are able to receive treatment with eteplirsen.You are able to follow instructions and participate in muscle testing.You are able to walk at least 350 meters during a test called the 6-minute walk test.You currently have a viral infection that is showing noticeable symptoms.You have symptoms or signs of a heart condition called cardiomyopathy.You have difficulty breathing when doing physical activities, swelling in your legs, feeling short of breath when lying down, or abnormal sounds in your lungs.
Research Study Groups:
This trial has the following groups:- Group 1: Cohort 2 (Dose Escalation) rAAVrh74.MCK.GALGT2
- Group 2: Cohort 1 (Minimal Efficacious Dose) rAAVrh74.MCK.GALGT2
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
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.
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