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Gene Transfer Clinical Trial to Deliver rAAVrh74.MCK.GALGT2 for Duchenne Muscular Dystrophy

NCT03333590 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 2

Last updated 2025-03-13

Study results available
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Summary

The proposed clinical trial study of rAAVrh74.MCK.GALGT2 for duchenne muscular dystrophy (DMD) patients. There will be a modified intravascular limb infusion (ILI) procedure that will be used to sequentially deliver vector to each whole lower limb of DMD subjects via a major lower limb artery.

Conditions

Interventions

BIOLOGICAL

rAAVrh74.MCK.GALGT2

Adeno-associated virus serotype rh74 carrying the GALGT2 gene under the control of a MCK promoter (rAAVrh74.MCK.GALGT2) will be delivered one time to each of the lower limbs through the femoral artery using an intravascular limb infusion technique (ILI)

Sponsors & Collaborators

  • Kevin Flanigan

    lead OTHER

Principal Investigators

  • Kevin Flanigan, MD · Nationwide Children's Hospital

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
4 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-11-06
Primary Completion
2020-11-04
Completion
2023-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03333590 on ClinicalTrials.gov