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Gene Therapy Trial Report

Summary

Follistatin Gene Transfer to Patients With Becker Muscular Dystrophy and Sporadic Inclusion Body Myositis


NCTID NCT01519349 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Becker Muscular Dystrophy, Sporadic Inclusion Body Myositis
Disease Ontology Term DOID:9883; DOID:3429
Compound Name RAAV1.CMV.huFollistatin344
Sponsor Nationwide Children's Hospital
Funder Type Other
Recruitment Status
Completed
Enrollment Count 15 (ACTUAL)
Results Posted Not Available

Therapy Information


Target Gene/Variant FST (FS344)
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Overexpression of protective allele/gene
Route of Administration Intramuscular
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV1
Dose 1 2E11 vg/kg (single leg)
Dose 2 3E11 vg/kg/quad
Dose 3 6E11 vg/kg/quad

Study Record Dates


Current Stage Phase1
Submit Date 2012-01-23
Completion Date 2017-10
Last Update 2023-10-02

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: * All subjects \[sIBM and BMD must be ambulatory and have identifiable atrophy of the quadriceps muscle with muscle weakness ≥2 standard deviations below predicted using quantitative muscle testing (maximum voluntary isometric strength testing), and difficulty getting out of chairs, climbing stairs, and getting up from the floor. * sIBM patients include males and post-menopause females of any ethnic or racial group. Diagnosis of sIBM is based on previously published criteria that include distribution of weakness (knee extensor weakness, finger flexor weakness) and histological presence of inflammation and vacuolar myopathy. Patients with inflammation, vacuolar changes and intracellular amyloid deposits or 15/18nm filaments fulfill criteria irrespective of clinical features. * BMD patients include adult males (\>18yo) of any ethnic or racial group with proven mutation of dystrophin gene and continued ambulation after age 15 years old. * Ability to cooperate for muscle testing * Deficit in muscle strength greater than 2 standard deviation below age expectations * Willingness of sexually active subjects with reproductive capacity (only male population) to practice reliable method of contraception until two negative sperm samples are obtained post gene transfer Exclusion Criteria: * Active viral infection * History or evidence of active infection with hepatitis C, hepatitis A or B, or HIV * Patients with any other cause of muscle weakness based on medical history and screening physical exam including: myopathy (other dystrophies, polymyositis, and dermatomyositis), neuropathy (from any cause), myasthenia gravis, and weakness related to degenerative joint disease of the spine. * Ongoing immunosuppressive therapy or immunosuppressive therapy within 3 months of starting the trial (e.g. corticosteroids, cyclosporine, tacrolimus, methotrexate, cyclophosphamide, intravenous immunoglobulin, rituximab) * Concomitant illness or requirement for chronic drug treatment that in the opinion of the PI creates unnecessary risks for gene transfer. Patients taking any of the following drugs will be excluded: drugs for treatment of myopathy or neuropathy or agents used to treat diabetes mellitus * Knee or ankle contractures preventing proper muscle strength testing * Patients with AAV1 neutralizing antibody titers ≥ 1:1600 as determined by ELISA immunoassay * Patients with history of angina and patients with past history of myocardial infarction in the past 6 months
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations United States

Regulatory Information


Has US IND True
FDA Designations
Recent Updates Study drug was well tolerated, was not tested further

Resources/Links