Muscular Dystrophy, DuchennePhase 2/3NCT00016653
Creatine and Glutamine in Steroid-Naive Duchenne Muscular Dystrophy
A Phase 2/3 study testing Creatine Monohydrate and Glutamine in people with Muscular Dystrophy, Duchenne, run by Cooperative International Neuromuscular Research Group at 1 site in United States.
Trial summary
This study will help to determine the effectiveness of glutamine and creatine as a possible therapy for DMD. Boys with DMD who are enrolled in this trial will be randomly chosen to receive creatine monohydrate or glutamine or an inactive placebo orally for six months. Read more about this study
- Who is it for?
- Ages 5 to 9 years, men only
- Conditions the study is for
- Muscular Dystrophy, Duchenne
- What is being tested?
- Creatine Monohydrate and Glutamine
- How many people
- 48 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2000to Dec 2006
- Phase
- Phase 2/3
- 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 9 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Aged 5 - 9 years old
- Able to walk without assistance
- Diagnosis of DMD confirmed by one of the following:
- a) Positive X-linked family history; or
- b) Dystrophin immunofluorescence and/or immunoblot, which shows complete dystrophin deficiency, and clinical picture consistent with DMD; or
- c) Gene deletion test positive in the central rod domain (exons 25 - 60) of dystrophin, where reading frame can be predicted as 'out-of-frame', and clinical picture consistent with DMD.
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- Glucocorticosteroid-naive (i.e. has not been treated with prednisone or deflazacort within 1 year before the study began), or has been involved in other therapeutic research protocol within the last year
- Forced Vital Capacity (a lung function test) > 50% of predicted value
- Evidence of muscle weakness by MRC score or clinical functional evaluation
- MRC (manual muscle test) score variability no greater than 10% between screening visits 1 and 2
Who cannot join
- Failure to achieve any of the criteria listed above
- Symptomatic DMD carrier
- Symptomatic cardiomyopathy or ventricular arrhythmias
- Previous (6 months or less) or current use of glutamine or creatine (for DMD or any other indication)
- Use of carnitine, other amino acids, coenzyme Q10, or any herbal medicines within the last month
- History of significant concomitant illness or significant impairment of renal or hepatic function
- Evidence of allergy to chocolate or milk solids (substances will be delivered in a powdered hot cocoa mixture)
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's National Medical Center
Washington D.C., District of Columbia, 20010
About this study
Official title: A Multicenter Randomized Placebo-controlled Double-blind Study to Assess Efficacy and Safety of Glutamine and Creatine Monohydrate in Duchenne Muscular Dystrophy
This study will help to determine the effectiveness of glutamine and creatine as a possible therapy for DMD. Boys with DMD who are enrolled in this trial will be randomly chosen to receive creatine monohydrate or glutamine or an inactive placebo orally for six months. Once a month during the six-month treatment period, the study participants will have their muscle strength evaluated using manual and computerized testing methods. This study will be conducted at several CINRG Centers throughout the U.S., Belgium, Israel and Puerto Rico. This study is supported by the Muscular Dystrophy Association.
Read the detailed description
PURPOSE OF THE STUDY The purpose of this study is to see if children with Duchenne muscular dystrophy (DMD) who are given glutamine (a nutritional supplement) or creatine monohydrate (a nutritional supplement) have changes in strength compared to children who are given no medicinal treatment. Children who are chosen to be in this research study will be diagnosed with DMD, be between the ages of 5 and 10 years old, and will not have participated in any other DMD clinical trials within the last year, and will not have been on medications that disqualify them from participation in this trial. All children in this clinical trial are entered at the discretion of the doctors in charge of the study. During the first and second visits, children who fulfill the criteria for participation in the clinical trial will be determined. We expect 50 subjects to participate at CINRG centers worldwide.
PROCEDURES
Children will be randomized (a procedure like flipping a coin) into one of three groups: treated with glutamine, treated with creatine monohydrate, or untreated (placebo: a sugar pill with no medication). Neither parents, children, nor the doctors treating the children will know the group each child has been assigned to. Because of the study design, there is a possibility that children will receive no treatment (placebo group) during the clinical trial. During the trial children must take one supplement mixed with water in the morning and supplements mixed with water each night.
Nine (9) clinic visits are required to complete this clinical trial. During visits to the clinic, each child will be evaluated by members of the research team to determine the child?s strength. Strength will be measured in different ways: manual testing, quantitative testing, and the usual measures used during visits to the neurologist (child will be asked to rise from the floor, walk across room, etc.). Manual testing is the way strength is usually measured by your child?s doctor. The quantitative muscle strength test (QMT) is a mechanical way to measure strength: the child sits/lays on an examining table and is asked to pull/push a strap. This test will determine the strength of different muscle groups in kilograms of force. An electrocardiogram (ECG, a heart test) will be performed during screening visit 2 and month 6 and blood (a small amount equal to about 2 to 3 tablespoons) will be drawn on screening visit 2 and months 1, 3 and 6. Urine tests will be done at screening visit 2 nd months 1, 3 and 6 to monitor kidney function.
- Conditions
- Muscular Dystrophy, Duchenne
- Being tested
- Creatine Monohydrate, Glutamine
- Keywords
- Duchenne, muscular, dystrophy, treatment
- 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.
Creatine MonohydrateDrug
GlutamineDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 May 2001 (estimated)
- Study startJun 2000
- Study completionDec 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Muscular Dystrophy, DuchenneStudies of the same condition, open and under way.
- 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Masking
- Double blind
- Enrolment
- 48 participants
Sponsor and investigators
- Lead sponsor
- Cooperative International Neuromuscular Research Group
- Sponsor type
- Research network
- Investigators
- Diana M Escolar, MD, Study director, Cooperative International Neuromuscular Research GroupGunnar Buyse, MD, PhD, Study director, Cooperative International Neuromuscular Research Group
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00016653
- Sponsor's protocol ID
- CNMC0599
- Status last verified
- Oct 2011
- First submitted
- 21 May 2001
- Last updated
- 27 Oct 2011
- Results posted
- No
Publications
Escolar DM, Buyse G, Henricson E, Leshner R, Florence J, Mayhew J, Tesi-Rocha C, Gorni K, Pasquali L, Patel KM, McCarter R, Huang J, Mayhew T, Bertorini T, Carlo J, Connolly AM, Clemens PR, Goemans N, Iannaccone ST, Igarashi M, Nevo Y, Pestronk A, Subramony SH, Vedanarayanan VV, Wessel H; CINRG Group. CINRG randomized controlled trial of creatine and glutamine in Duchenne muscular dystrophy. Ann Neurol. 2005 Jul;58(1):151-5. doi: 10.1002/ana.20523.
PubMed 15984021
Study at a glance
From the registry record, last updated by the sponsor on 27 Oct 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter Randomized Placebo-controlled Double-blind Study to Assess Efficacy and Safety of Glutamine and Creatine Monohydrate in Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT00016653
- Acronym / sponsor ID
- CNMC0599
- Conditions
- Muscular Dystrophy, Duchenne
- Keywords
- Duchenne, muscular, dystrophy, treatment
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 48 participants
- Lead sponsor
- Cooperative International Neuromuscular Research GroupResearch network
- Study start
- Jun 2000
- Study completion
- Dec 2006
- First posted
- 23 May 2001 (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.
This study will help to determine the effectiveness of glutamine and creatine as a possible therapy for DMD. Boys with DMD who are enrolled in this trial will be randomly chosen to receive creatine monohydrate or glutamine or an inactive placebo orally for six months. Once a month during the six-month treatment period, the study participants will have their muscle strength evaluated using manual and computerized testing methods. This study will be conducted at several CINRG Centers throughout the U.S., Belgium, Israel and Puerto Rico. This study is supported by the Muscular Dystrophy Association.
Detailed description
PURPOSE OF THE STUDY The purpose of this study is to see if children with Duchenne muscular dystrophy (DMD) who are given glutamine (a nutritional supplement) or creatine monohydrate (a nutritional supplement) have changes in strength compared to children who are given no medicinal treatment. Children who are chosen to be in this research study will be diagnosed with DMD, be between the ages of 5 and 10 years old, and will not have participated in any other DMD clinical trials within the last year, and will not have been on medications that disqualify them from participation in this trial. All children in this clinical trial are entered at the discretion of the doctors in charge of the study. During the first and second visits, children who fulfill the criteria for participation in the clinical trial will be determined. We expect 50 subjects to participate at CINRG centers worldwide.
PROCEDURES
Children will be randomized (a procedure like flipping a coin) into one of three groups: treated with glutamine, treated with creatine monohydrate, or untreated (placebo: a sugar pill with no medication). Neither parents, children, nor the doctors treating the children will know the group each child has been assigned to. Because of the study design, there is a possibility that children will receive no treatment (placebo group) during the clinical trial. During the trial children must take one supplement mixed with water in the morning and supplements mixed with water each night.
Nine (9) clinic visits are required to complete this clinical trial. During visits to the clinic, each child will be evaluated by members of the research team to determine the child?s strength. Strength will be measured in different ways: manual testing, quantitative testing, and the usual measures used during visits to the neurologist (child will be asked to rise from the floor, walk across room, etc.). Manual testing is the way strength is usually measured by your child?s doctor. The quantitative muscle strength test (QMT) is a mechanical way to measure strength: the child sits/lays on an examining table and is asked to pull/push a strap. This test will determine the strength of different muscle groups in kilograms of force. An electrocardiogram (ECG, a heart test) will be performed during screening visit 2 and month 6 and blood (a small amount equal to about 2 to 3 tablespoons) will be drawn on screening visit 2 and months 1, 3 and 6. Urine tests will be done at screening visit 2 nd months 1, 3 and 6 to monitor kidney function.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Masking
- Double blind
Who may qualify
- Ages
- 5 to 9 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (10 listed)
- Aged 5 - 9 years old
- Able to walk without assistance
- Diagnosis of DMD confirmed by one of the following:
- a) Positive X-linked family history; or
- b) Dystrophin immunofluorescence and/or immunoblot, which shows complete dystrophin deficiency, and clinical picture consistent with DMD; or
Key exclusion criteria (7 listed)
- Failure to achieve any of the criteria listed above
- Symptomatic DMD carrier
- Symptomatic cardiomyopathy or ventricular arrhythmias
- Previous (6 months or less) or current use of glutamine or creatine (for DMD or any other indication)
- Use of carnitine, other amino acids, coenzyme Q10, or any herbal medicines within the last month
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 |
|---|---|---|
| Creatine Monohydrate | Drug | – |
| Glutamine | Drug | – |
Primary outcome
Not reported in the source record.
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 27 Oct 2011.
- Results publication: PubMed 15984021Escolar DM, Buyse G, Henricson E, Leshner R, Florence J, Mayhew J, Tesi-Rocha C, Gorni K, Pasquali L, Patel KM, McCarter R, Huang J, Mayhew T, Bertorini T, Carlo J, Connolly AM, Clemens PR, Goemans N, Iannaccone ST, Igarashi M, Nevo Y, Pestronk A, Subramony SH, Vedanarayanan VV, Wessel H; CINRG Group. CINRG randomized controlled trial of creatine and glutamine in Duchenne muscular dystrophy. Ann Neurol. 2005 Jul;58(1):151-5. doi: 10.1002/ana.20523.
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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.
Diana M Escolar, MD study director
Cooperative International Neuromuscular Research Group
Gunnar Buyse, MD, PhD study director
Cooperative International Neuromuscular Research Group
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Creatine Monohydrate and Glutamine in people with Muscular Dystrophy, Duchenne, run by Cooperative International Neuromuscular Research Group at 1 site 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 10 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 27 Oct 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00016653, which also lists 1 linked publication.
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 27 Oct 2011.