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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 days 90 (cohort 2), 120 (cohort 1) and both cohorts at day 180, months 12, 18 and 24
Awards & highlights
No Placebo-Only Group

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 weeks
Treatment ~ Varies
Follow Up ~days 90 (cohort 2), 120 (cohort 1) and both cohorts at day 180, months 12, 18 and 24
This trial's timeline: 3 weeks for screening, Varies for treatment, and days 90 (cohort 2), 120 (cohort 1) and both cohorts at day 180, months 12, 18 and 24 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Secondary study objectives
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).
Other study objectives
Score of Muscle Function Using the The North Star Ambulatory Assessment (NSAA).

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

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
~0 spots leftby Nov 2025