DystrophinopathyPhase 1NCT01388764
Safety, Tolerability and Effects of L-Arginine in Boys With Dystrophinopathy on Corticosteroids
A Phase 1 study testing L-arginine in people with Dystrophinopathy and Duchenne Muscular Dystrophy, run by Massachusetts General Hospital at 1 site in United States.
Trial summary
The purpose of the study is to assess the safety, tolerability, and effects of L-Arginine on muscles in boys with dystrophinopathy on corticosteroids. Specifically, to see if L-arginine reduces muscle signal abnormalities on MRI done pre and post 30 days of L-arginine administration. Read more about this study
- Who is it for?
- Ages 7 to 11 years, men only
- Conditions the study is for
- Dystrophinopathy, Duchenne Muscular Dystrophy, Becker's Muscular Dystrophy
- What is being tested?
- L-arginine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2012to May 2012
- 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 7 to 11 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Confirmation of diagnosis of dystrophinopathy, documented by clinical exam and dystrophin DNA mutation analysis
- Ambulatory male subjects between the ages of 7-11 years
- Stable dosage of corticosteroids for 3 months prior to entry (Screening/Baseline Day 0) and during treatment period
- Able to follow instructions and give assent
- Able to complete nonsedated MR
Who cannot join
- Presence of metallic orthopedic hardware in the lower extremity that could affect MRI/MRS measurements
- Routine MRI exclusion criteria such as the presence of a pacemaker, cochlear implant, or cerebral aneurysm clip
- Subjects not capable of cooperating during MR examination
- Known hypersensitivity to L-arginine
- Exposure to another investigational agent, investigational supplements, growth hormone within 3 months prior to entry (Screening/Baseline Day 0) or during treatment period
- Subjects must not be taking L-arginine for at least 4 weeks prior to entry (Day 0)
- Subjects who are non-ambulatory or with daytime ventilatory dependence
Trial locations and contacts
1 site in 1 country.
United States1 site
Massachusetts General Hospital
Boston, Massachusetts, 02114
About this study
Official title: Pilot Study: To Assess the Safety, Tolerability and Effects of L-Arginine on Muscles in Boys With Dystrophinopathy on Corticosteroids
The purpose of the study is to assess the safety, tolerability, and effects of L-Arginine on muscles in boys with dystrophinopathy on corticosteroids. Specifically, to see if L-arginine reduces muscle signal abnormalities on MRI done pre and post 30 days of L-arginine administration.
Read the detailed description
Dystrophinopathy is a muscular dystrophy (includes Duchenne or Becker's Muscular Dystrophy) that can be a lethal muscle disorder resulting from defects in the gene for dystrophin, a structural protein required to maintain muscle integrity. Absence of functional dystrophin leaves the muscle membrane vulnerable to damage during contraction. This damage can be exacerbated by an inflammatory response leading to myofiber necrosis.
L-arginine is a widely available dietary supplement amino acid postulated to affect dystrophinopathy in several favorable ways: upregulation of utrophin, vasodilation in muscle via nitric oxide, enhanced synthesis of creatine, increase levels of growth hormone.
We hypothesize that administration of L-arginine may increase levels of creatine and growth hormone and in turn reduce the extent of myofiber damage in our patients with dystrophinopathy
- Being tested
- L-arginine
- Keywords
- L-arginine supplement study
- 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.
L-arginineDrug
Subjects will receive oral L-Arginine (0.3 grams/kg/day, divided 2 times per day, not to exceed 14 grams/day)
| Study group | Role | What they receive |
|---|---|---|
| L-arginine | Experimental | Not described Receives: Drug: L-arginine |
What is being measured
The primary outcome is the main question the study is designed to answer.
MRI/MRS of calf muscle
MRI/MRS will be performed of the calf muscle in all subjects (N=8) to assess muscle signal abnormalities on MRI and creatine levels on MRS, done at the start of the study (Day 0) and at the end of the study (Day 30), after 30 days of L-arginine administration.
Measured: Day 0 and Day 30
Secondary outcomes (3)
Blood tests
We will obtain safety labs [complete blood count (CBC) and comprehensive metabolic panel (CMP)] from all subjects (N =8), at day 0 and day 30, after 30 days of oral L-argninine administration.
Measured: Day 0 and Day 30
Assessment of muscle strength and function
Measurements of upper and lower extremity strength will be performed using a hand-held dynamometer. Functional tests will also be performed which include time to walk specified distances and time to climb stairs.
Measured: Day 0 and Day 30
Pulmonary function tests
Subjects will have pulmonary function studies to assess forced vital capacity
Measured: Day 0 and Day 30
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jul 2011 (estimated)
- Study startJan 2012
- Primary completionMay 2012
- Study completionMay 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for DystrophinopathyStudies of the same condition, open and under way.
- More trials run by Massachusetts General 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
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Massachusetts General Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Namita Goyal, Massachusetts General Hospital
- Investigators
- Namita Goyal, MD, Principal investigator, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01388764
- Sponsor's protocol ID
- 2011D001591
- Status last verified
- Jun 2012
- First submitted
- 5 Jul 2011
- Last updated
- 3 Jul 2012
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Jul 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study: To Assess the Safety, Tolerability and Effects of L-Arginine on Muscles in Boys With Dystrophinopathy on Corticosteroids
- ClinicalTrials.gov ID
- NCT01388764
- Acronym / sponsor ID
- 2011D001591
- Keywords
- L-arginine supplement study
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Massachusetts General HospitalUniversity, hospital or other
- Study start
- Jan 2012
- Primary completion
- May 2012 (actual)
- Study completion
- May 2012 (actual)
- First posted
- 7 Jul 2011 (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 the study is to assess the safety, tolerability, and effects of L-Arginine on muscles in boys with dystrophinopathy on corticosteroids. Specifically, to see if L-arginine reduces muscle signal abnormalities on MRI done pre and post 30 days of L-arginine administration.
Detailed description
Dystrophinopathy is a muscular dystrophy (includes Duchenne or Becker's Muscular Dystrophy) that can be a lethal muscle disorder resulting from defects in the gene for dystrophin, a structural protein required to maintain muscle integrity. Absence of functional dystrophin leaves the muscle membrane vulnerable to damage during contraction. This damage can be exacerbated by an inflammatory response leading to myofiber necrosis.
L-arginine is a widely available dietary supplement amino acid postulated to affect dystrophinopathy in several favorable ways: upregulation of utrophin, vasodilation in muscle via nitric oxide, enhanced synthesis of creatine, increase levels of growth hormone.
We hypothesize that administration of L-arginine may increase levels of creatine and growth hormone and in turn reduce the extent of myofiber damage in our patients with dystrophinopathy
Study design
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| L-arginine | Experimental | Drug: L-arginine |
Who may qualify
- Ages
- 7 to 11 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Confirmation of diagnosis of dystrophinopathy, documented by clinical exam and dystrophin DNA mutation analysis
- Ambulatory male subjects between the ages of 7-11 years
- Stable dosage of corticosteroids for 3 months prior to entry (Screening/Baseline Day 0) and during treatment period
- Able to follow instructions and give assent
- Able to complete nonsedated MR
Key exclusion criteria (7 listed)
- Presence of metallic orthopedic hardware in the lower extremity that could affect MRI/MRS measurements
- Routine MRI exclusion criteria such as the presence of a pacemaker, cochlear implant, or cerebral aneurysm clip
- Subjects not capable of cooperating during MR examination
- Known hypersensitivity to L-arginine
- Exposure to another investigational agent, investigational supplements, growth hormone within 3 months prior to entry (Screening/Baseline Day 0) or during treatment period
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 |
|---|---|---|
| L-arginineSubjects will receive oral L-Arginine (0.3 grams/kg/day, divided 2 times per day, not to exceed 14 grams/day) | Drug | L-arginine |
Primary outcome
MRI/MRS of calf muscle
Time frame: Day 0 and Day 30
MRI/MRS will be performed of the calf muscle in all subjects (N=8) to assess muscle signal abnormalities on MRI and creatine levels on MRS, done at the start of the study (Day 0) and at the end of the study (Day 30), after 30 days of L-arginine administration.
3 secondary outcomes
Blood tests
Time frame: Day 0 and Day 30
Assessment of muscle strength and function
Time frame: Day 0 and Day 30
Pulmonary function tests
Time frame: Day 0 and Day 30
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 3 Jul 2012.
Related trials for Dystrophinopathy
RecruitingObservational study
Duchenne Electronic Health Record Study
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Registry for Duchenne and Becker Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy
Recruiting
VirtualPark_Pediatric
Dystrophinopathy, Muscular Dystrophies
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The Duchenne Registry
Duchenne Muscular Dystrophy, Becker Muscular Dystrophy
RecruitingObservational study
DMD Gene Variants and Cardiac Dysfunction in Young Males With Dystrophinopathies
Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy
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.
Namita Goyal, MD principal investigator
Massachusetts General Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing L-arginine in people with Dystrophinopathy and Duchenne Muscular Dystrophy, run by Massachusetts General Hospital 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 5 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 3 Jul 2012. 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 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?
MRI/MRS of calf muscle (time frame: Day 0 and Day 30). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 3 Jul 2012.