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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
Ability to extend the knee fully against gravity
Preserved ambulation with ability to walk ≥ 350 meters during the 6MWT
Screening 3 weeks
Treatment Varies
Follow Up 2 years
Awards & highlights

Study 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

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
You can straighten your knee completely without any difficulty or help.
You are able to walk at least 350 meters during a test called the 6-minute walk test.
You are able to follow instructions and participate in muscle testing.


Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~2 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and 2 years for reporting.

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 more

Trial 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)]

Find a Location

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

Media Library

rAAVrh74.MCK.GALGT2 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03333590 — Phase 1 & 2
Duchenne Muscular Dystrophy Research Study Groups: Cohort 2 (Dose Escalation) rAAVrh74.MCK.GALGT2, Cohort 1 (Minimal Efficacious Dose) rAAVrh74.MCK.GALGT2
Duchenne Muscular Dystrophy Clinical Trial 2023: rAAVrh74.MCK.GALGT2 Highlights & Side Effects. Trial Name: NCT03333590 — Phase 1 & 2
rAAVrh74.MCK.GALGT2 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03333590 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are individuals being accepted currently for this research study?

"According to clinicaltrials.gov, no new patients are being accepted into this trial since its last edit on July 26th 2022. Nonetheless, there are 86 other studies actively recruiting right now which may be of interest to potential participants."

Answered by AI
Recent research and studies
~0 spots leftby Apr 2025