Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy
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ClinicalTrials.gov Identifier: NCT02354781 |
Recruitment Status :
Completed
First Posted : February 3, 2015
Results First Posted : April 15, 2020
Last Update Posted : April 15, 2020
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Sponsor:
Jerry R. Mendell
Collaborators:
Duchenne Alliance
Milo Therapeutics
Information provided by (Responsible Party):
Jerry R. Mendell, Nationwide Children's Hospital
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Study Type | Interventional |
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Study Design | Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Other |
Condition |
Duchenne Muscular Dystrophy |
Intervention |
Biological: rAAV1.CMV.huFollistin344 |
Enrollment | 3 |
Participant Flow
Recruitment Details | |
Pre-assignment Details |
Arm/Group Title | 2.4E12 vg/kg CMV.huFollistatin344 |
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Participants enrolled will receive a single dose of 2.4E12 vg/kg (1.2E12vg/kg/limb) of rAAV1.CMV.huFollistatin344 delivered to the lower limbs |
Period Title: Overall Study | |
Started | 3 |
Completed | 3 |
Not Completed | 0 |
Baseline Characteristics
Arm/Group Title | Dose Group 1 | |
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Participants enrolled will receive a single dose of 2.4E12 vg/kg (1.2E12vg/kg/limb) of rAAV1.CMV.huFollistatin344 delivered to the lower limbs | |
Overall Number of Baseline Participants | 3 | |
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The Safety Population included all subjects who received one dose of 2.4E12 vg/kg (1.2E12vg/kg/limb) of rAAV1.CMV.huFollistatin344. The Safety Population is the primary analysis population for safety assessments
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Age, Categorical
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 3 participants | |
<=18 years |
3 100.0%
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Between 18 and 65 years |
0 0.0%
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>=65 years |
0 0.0%
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Sex: Female, Male
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 3 participants | |
Female |
0 0.0%
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Male |
3 100.0%
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Ethnicity (NIH/OMB)
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 3 participants | |
Hispanic or Latino |
0 0.0%
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Not Hispanic or Latino |
3 100.0%
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Unknown or Not Reported |
0 0.0%
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Race (NIH/OMB)
Measure Type: Count of Participants Unit of measure: Participants |
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Number Analyzed | 3 participants | |
American Indian or Alaska Native |
0 0.0%
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Asian |
0 0.0%
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Native Hawaiian or Other Pacific Islander |
0 0.0%
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Black or African American |
0 0.0%
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White |
3 100.0%
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More than one race |
0 0.0%
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Unknown or Not Reported |
0 0.0%
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Region of Enrollment
Measure Type: Number Unit of measure: Participants |
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United States | Number Analyzed | 3 participants |
3 |
Outcome Measures
Adverse Events
Limitations and Caveats
[Not Specified]
More Information
Results Point of Contact
Name/Title: | Dr. Jerry Mendell |
Organization: | Nationwide Children's Hospital |
Phone: | 1-614-722-4877 |
EMail: | Jerry.Mendell@nationwidechildrens.org |
Publications:
Responsible Party: | Jerry R. Mendell, Nationwide Children's Hospital |
ClinicalTrials.gov Identifier: | NCT02354781 |
Other Study ID Numbers: |
14-00630 |
First Submitted: | January 26, 2015 |
First Posted: | February 3, 2015 |
Results First Submitted: | January 24, 2020 |
Results First Posted: | April 15, 2020 |
Last Update Posted: | April 15, 2020 |