Duchenne Muscular DystrophyPhase 1NCT00451074
Six Month Study of Gentamicin in Duchenne Muscular Dystrophy With Stop Codons
A Phase 1 study testing Gentamicin infusions twice a week for six months in people with Duchenne Muscular Dystrophy, run by Nationwide Children's Hospital at 3 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 5 to 20 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Gentamicin infusions twice a week for six months
- Type of treatment
- Injection or infusion
- How many people
- 12 participants
- Where
- 3 sitesUnited States
- Study dates
- Mar 2007to Jul 2009
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 20 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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)
Trial locations and contacts
3 sites in 1 country.
United States3 sites
The Research Institute at Nationwide Children's Hospital
Columbus, Ohio, 43205-2696
University of Kansas
Kansas City, Kansas, 66160-0001
Neuromuscular Research Institute - Scottsdale Healthcare Hopsital at Shea
Scottsdale, Arizona, 85258
About this study
Official title: A Six Month Randomized, Clinical Trial of Gentamicin in Duchenne Muscular Dystrophy Subjects With Stop Codon Mutations
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.
Read the detailed description
The primary purpose of this second cohort is to see if the IV Medication, gentamicin, is safe to give twice a week for six months to boys with Duchenne muscular dystrophy (DMD). Secondarily, we want to know if gentamicin can help strengthen the muscles of boys with DMD who have a particular type of genetic mutation known as a stop codon. The gentamicin is thought to allow for "read-through" of this type of mutation which would allow for the production of dystrophin, a protein which is lacking in boys with DMD.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Gentamicin infusions twice a week for six months
- Keywords
- Stop codon mutations
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Gentamicin infusions twice a week for six monthsDrug
Gentamicin infusions twice a week
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 6 months
Secondary outcomes (2)
Determine if gentamicin given over six months improves muscle strength.
Measured: 6 months
Determine if gentamicin given over six months increases dystrophin binding at the muscle membrane.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Mar 2007 (estimated)
- Study startMar 2007
- Primary completionJul 2009
- Study completionJul 2009
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- More trials run by Nationwide Children's HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nationwide Children's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- National Institutes of Health (NIH), National Institute of Neurological Disorders and Stroke (NINDS)
- Responsible party
- Principal investigator, Jerry R. Mendell, Nationwide Children's Hospital
- Investigators
- Jerry R. Mendell, M.D., Principal investigator, The Research Institute at Nationwide Children's Hospital/ Nationwide Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00451074
- Sponsor's protocol ID
- NS043186
- Other IDs
- 7R01NS043186
- Status last verified
- Mar 2012
- First submitted
- 21 Mar 2007
- Last updated
- 23 Mar 2012
- Results posted
- No
- Data monitoring committee
- Yes
Publications
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.
PubMed 21179598Malik 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.
PubMed 20517938
Study at a glance
From the registry record, last updated by the sponsor on 23 Mar 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Six Month Randomized, Clinical Trial of Gentamicin in Duchenne Muscular Dystrophy Subjects With Stop Codon Mutations
- ClinicalTrials.gov ID
- NCT00451074
- Acronym / sponsor ID
- NS043186
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Stop codon mutations
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Nationwide Children's HospitalUniversity, hospital or other
- Collaborators
- National Institutes of Health (NIH), National Institute of Neurological Disorders and Stroke (NINDS)
- Study start
- Mar 2007
- Primary completion
- Jul 2009 (actual)
- Study completion
- Jul 2009 (actual)
- First posted
- 23 Mar 2007 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
The primary purpose of this second cohort is to see if the IV Medication, gentamicin, is safe to give twice a week for six months to boys with Duchenne muscular dystrophy (DMD). Secondarily, we want to know if gentamicin can help strengthen the muscles of boys with DMD who have a particular type of genetic mutation known as a stop codon. The gentamicin is thought to allow for "read-through" of this type of mutation which would allow for the production of dystrophin, a protein which is lacking in boys with DMD.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 5 to 20 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Gentamicin infusions twice a week for six monthsGentamicin infusions twice a week | Drug | – |
Primary outcome
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
2 secondary outcomes
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Mar 2012.
- Results publication: PubMed 21179598Malik 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.
- Results publication: PubMed 20517938Malik 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.
- Center for Gene Therapy, The Research Institute at Nationwide Children's HospitalLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jerry R. Mendell, M.D. principal investigator
The Research Institute at Nationwide Children's Hospital/ Nationwide Children's Hospital
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Gentamicin infusions twice a week for six months in people with Duchenne Muscular Dystrophy, run by Nationwide Children's Hospital at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 Mar 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00451074, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Mar 2012.