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

• Register: ClinicalTrials.gov
• Last refreshed on: 9 October 2023
• Main ID: NCT01519349
• Date of registration: 23/01/2012
• Prospective Registration: No
• Primary sponsor: Nationwide Children's Hospital
• Public title: Follistatin Gene Transfer to Patients With Becker Muscular Dystrophy and Sporadic Inclusion Body Myositis
• Scientific title: Phase I Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Becker Muscular Dystrophy and Sporadic Inclusion Body Myositis.
• Date of first enrolment: January 2012
• Target sample size: 15
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT01519349
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Single Group Assignment. Primary purpose: Other. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Jerry R Mendell, M.D.
• Affiliation: Nationwide Children's Hospital

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Sporadic Inclusion Body Myositis

Becker Muscular Dystrophy

Intervention(s)

Biological: rAAV1.CMV.huFollistatin344

Primary Outcome(s)

Safety [Time Frame: 2 years]

Secondary Outcome(s)

Muscle Function and Strength Testing [Time Frame: 2 years]

Secondary ID(s)

NCH-696110

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Parent Project Muscular Dystrophy