Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.GALGT2
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ClinicalTrials.gov Identifier: NCT02704325 |
Recruitment Status :
Withdrawn
First Posted : March 10, 2016
Last Update Posted : February 6, 2018
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Condition or disease | Intervention/treatment | Phase |
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Duchenne Muscular Dystrophy | Biological: rAAVrh74.MCK.GALGT2 Other: PLACEBO (Saline) | Phase 1 Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 0 participants |
Allocation: | Non-Randomized |
Intervention Model: | Single Group Assignment |
Intervention Model Description: | This is a dose escalation trial that will begin with the minimal efficacious dose as determined by preclinical studies and approved by the FDA. During the course of the trial, if safety is shown the dose will be escalated according to the clinical protocol. |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | Phase I Gene Transfer Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MCK.GALGT2 |
Estimated Study Start Date : | April 2016 |
Estimated Primary Completion Date : | July 2018 |
Estimated Study Completion Date : | February 2020 |

Arm | Intervention/treatment |
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Experimental: GALGT2 Viral Vector
Dose: 1E12 vg (total dose) (n=3) of rAAVrh74.MCK.GALGT2 vs Placebo (Saline). All participants will receive rAAVrh74.MCK.GALGT2 in the right or the left EDB muscle and receive Saline in the opposite EDB muscles. The investigator will not know which side will receive rAAVrh74.MCK.GALGT2 vs Saline until after the trial is over. At the end of the trial the investigator will be unblinded.
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Biological: rAAVrh74.MCK.GALGT2
Direct intramuscular injection of rAAVrh74.MCK.GALGT2 transferred to the extensor digitorum brevis muscle (EDB) of one foot and the other side receiving saline alone. |
Experimental: Saline
Dose: 1E12 vg (total dose) (n=3) of rAAVrh74.MCK.GALGT2 vs Placebo (Saline). All participants will receive rAAVrh74.MCK.GALGT2 in the right or the left EDB muscle and receive Saline in the opposite EDB muscles. The investigator will not know which side will receive rAAVrh74.MCK.GALGT2 vs Saline until after the trial is over. At the end of the trial the investigator will be unblinded.
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Other: PLACEBO (Saline)
Direct intramuscular injection of rAAVrh74.MCK.GALGT2 transferred to the extensor digitorum brevis muscle (EDB) of one foot and the OTHER side receiving saline alone. |
- Treatment related toxicities [ Time Frame: 2 years ]Based on the development of unacceptable toxicity defined as the occurrence of any one Grade III or higher treatment-related toxicities.
- Expression of GALGT2 demonstrated with anti-CT epitope antibodies. [ Time Frame: 6 or 12 weeks ]
- GALGT2 protein expression quantified by western blot and assessed by densitometry [ Time Frame: 6 or 12 weeks ]
- Transduction efficiency measured by qPCR of the GALGT transgene from muscle, and expressed as vector genomes normalized to a genomic single-copy control. [ Time Frame: 6 or 12 weeks ]
- Number of fibers containing central nuclei compared between muscles by paired t-tests [ Time Frame: 6 or 12 weeks ]
- Dystrophin expression demonstrated with antibodies to N-terminal, C-terminal, and rod domains [ Time Frame: 6 or 12 weeks ]
- Utrophin expression [ Time Frame: 6 or 12 weeks ]
- Leukocyte markers including CD45, CD3, CD4, CD8, and MAC 387 [ Time Frame: 6 or 12 weeks ]
- Muscle will be examined for histological appearance [ Time Frame: 6 or 12 weeks ]
- Antibodies to rAAVrh74 along with PBMC ELISpots to both rAAVrh74 capsid and GALGT protein will be evaluated at different time points during the study [ Time Frame: 6 or 12 weeks ]

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Ages Eligible for Study: | 9 Years and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Nonambulant subjects, age 9 or older
- Confirmed mutation in the DMD gene using a clinically accepted technique that completely defines the mutation
- A magnetic resonance image of the EDB showing preservation of sufficient muscle mass to permit transfection
- Males of any ethnic group will be eligible
- Ability to cooperate with all study procedures
- Willingness of sexually active subjects with reproductive capacity to practice reliable method of contraception (If appropriate).
- Stable dose of corticosteroid therapy (including either prednisone or deflazacort and their generic forms) for 12 weeks prior to gene transfer
Exclusion Criteria:
- Active viral infection based on clinical observations.
- The presence of a DMD mutation without weakness or loss of function
- Symptoms or signs of cardiomyopathy, including:
- Dyspnea on exertion, pedal edema, shortness of breath upon lying flat, or rales at the base of the lungs
- Echocardiogram with ejection fraction below 40%
- Serological evidence of HIV infection, or Hepatitis A, B or C infection
- Diagnosis of (or ongoing treatment for) an autoimmune disease
- Persistent leukopenia or leukocytosis (WBC ≤ 3.5 K/µL or ≥ 20.0 K/µL) or an absolute neutrophil count < 1.5K/µL
- Concomitant illness or requirement for chronic drug treatment that in the opinion of the PI creates unnecessary risks for gene transfer
- Subjects with rAAVrh74 binding antibody titers ≥ 1:400 as determined by ELISA immunoassay
- Presence of circulating anti-Sda antibodies as determined by study approved laboratory.
- Abnormal laboratory values in the clinically significant range, based upon normal values in the Nationwide Children's Hospital Laboratory

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02704325
United States, Ohio | |
Nationwide Children's Hospital | |
Columbus, Ohio, United States, 43205 |
Principal Investigator: | Kevin Flanigan, MD | Nationwide Children's Hospital |
Responsible Party: | Kevin Flanigan, Professor of Pediatrics, Nationwide Children's Hospital |
ClinicalTrials.gov Identifier: | NCT02704325 |
Other Study ID Numbers: |
GALGT2 for DMD |
First Posted: | March 10, 2016 Key Record Dates |
Last Update Posted: | February 6, 2018 |
Last Verified: | February 2018 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
DMD Duchenne Muscular Dystrophy |
Muscular Dystrophies Muscular Dystrophy, Duchenne Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases |
Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked |