NCT00451074

Brief Summary

The purpose of this study is to determine the safety of giving intravenous (IV) gentamicin to boys with Duchenne muscular dystrophy who have stop codon mutations.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
12

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Mar 2007

Typical duration for phase_1

Geographic Reach
1 country

3 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

March 1, 2007

Completed
20 days until next milestone

First Submitted

Initial submission to the registry

March 21, 2007

Completed
2 days until next milestone

First Posted

Study publicly available on registry

March 23, 2007

Completed
2.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2009

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2009

Completed
Last Updated

March 23, 2012

Status Verified

March 1, 2012

Enrollment Period

2.3 years

First QC Date

March 21, 2007

Last Update Submit

March 22, 2012

Conditions

Duchenne Muscular Dystrophy

Keywords

Stop codon mutations

Outcome Measures

Primary Outcomes (1)

  • 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.

    6 months

Secondary Outcomes (2)

  • Determine if gentamicin given over six months improves muscle strength.

    6 months

  • Determine if gentamicin given over six months increases dystrophin binding at the muscle membrane.

    6 months

Interventions

Gentamicin infusions twice a week for six monthsDRUG

Gentamicin infusions twice a week

Eligibility Criteria

Age5 Years - 20 Years
Sexmale
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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.

You may not qualify if:

  • 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)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

Neuromuscular Research Institute - Scottsdale Healthcare Hopsital at Shea

Scottsdale, Arizona, 85258, United States

Location

University of Kansas

Kansas City, Kansas, 66160-0001, United States

Location

The Research Institute at Nationwide Children's Hospital

Columbus, Ohio, 43205-2696, United States

Location

Related Publications (2)

  • Malik V, Rodino-Klapac LR, Viollet L, Mendell JR. Aminoglycoside-induced mutation suppression (stop codon readthrough) as a therapeutic strategy for Duchenne muscular dystrophy. Ther Adv Neurol Disord. 2010 Nov;3(6):379-89. doi: 10.1177/1756285610388693.

    PMID: 21179598RESULT

  • Malik V, Rodino-Klapac LR, Viollet L, Wall C, King W, Al-Dahhak R, Lewis S, Shilling CJ, Kota J, Serrano-Munuera C, Hayes J, Mahan JD, Campbell KJ, Banwell B, Dasouki M, Watts V, Sivakumar K, Bien-Willner R, Flanigan KM, Sahenk Z, Barohn RJ, Walker CM, Mendell JR. Gentamicin-induced readthrough of stop codons in Duchenne muscular dystrophy. Ann Neurol. 2010 Jun;67(6):771-80. doi: 10.1002/ana.22024.

    PMID: 20517938RESULT

Related Links

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Jerry R. Mendell, M.D.

    The Research Institute at Nationwide Children's Hospital/ Nationwide Children's Hospital

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Director, Center for Gene Therapy

Study Record Dates

First Submitted

March 21, 2007

First Posted

March 23, 2007

Study Start

March 1, 2007

Primary Completion

July 1, 2009

Study Completion

July 1, 2009

Last Updated

March 23, 2012

Record last verified: 2012-03

Locations

US(3)