Six Month Study of Gentamicin in Duchenne Muscular Dystrophy With Stop Codons

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00451074
• Date of registration: 21/03/2007
• Prospective Registration: No
• Primary sponsor: Nationwide Children's Hospital
• Public title: Six Month Study of Gentamicin in Duchenne Muscular Dystrophy With Stop Codons
• Scientific title: A Six Month Randomized, Clinical Trial of Gentamicin in Duchenne Muscular Dystrophy Subjects With Stop Codon Mutations
• Date of first enrolment: March 2007
• Target sample size: 12
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00451074
• Study type: Interventional
• Study design: Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Jerry R. Mendell, M.D.
• Affiliation: The Research Institute at Nationwide Children's Hospital/ Nationwide Children's Hospital

Key inclusion & exclusion criteria

Inclusion Criteria:

- Age 5-20 years

- Duchenne muscular dystrophy documented by written report of stop codon mutation
analysis of the dystrophin gene.

- Subject is capable of cooperating for efficacy and safety testing

- Absent dystrophin on muscle biopsy

- Subjects may be untreated, taking prednisone or comparable corticosteroids

- Subjects taking corticosteroids must be on the same dose for at least 3 months (90
days) prior to the start of the study.

Exclusion Criteria:

- Known allergy to any aminoglycoside or sulfate compounds

- Current use of potential nephrotoxic or ototoxic drug

- Current use of corticosteroids has not been stable for 3 months (90) days

- Known mutation at nucleotide 1555 in 12S rRNA gene of mitochondrial DNA (predisposes
to aminoglycoside hearing loss and commercially available via Athena Diagnostics
Lab). This DNA testing (Hearing susceptibility test) will be made available through
funding from this grant.

- Inability to hear within the range of 0 to 25 dB in any hearing frequency by pure
tone audiometry

- Cystatin C equal to or > 1.4mg/L

- Other medical condition that would impede the conduct of study (e.g., congestive
heart failure)



Age minimum: 5 Years
Age maximum: 20 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Duchenne Muscular Dystrophy

Intervention(s)

Drug: Gentamicin infusions twice a week for six months

Primary Outcome(s)

In this phase 1 clinical trial, safety will be measured via gentamicin trough levels, audiology, and renal function tests. These lab tests will remain in the normal range while infusing gentamicin twice a week for 6 month. [Time Frame: 6 months]

Secondary Outcome(s)

Determine if gentamicin given over six months improves muscle strength. [Time Frame: 6 months]

Determine if gentamicin given over six months increases dystrophin binding at the muscle membrane. [Time Frame: 6 months]

Secondary ID(s)

7R01NS043186

NS043186

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

National Institutes of Health (NIH)

National Institute of Neurological Disorders and Stroke (NINDS)