Duchenne Muscular DystrophyNCT00468832
Longitudinal Study of the Natural History of Duchenne Muscular Dystrophy (DMD)
An observational study of Duchenne Muscular Dystrophy, run by Cooperative International Neuromuscular Research Group at 21 sites in 8 countries.
Trial summary
The purpose of this study is to establish the largest long-term assessment of people with Duchenne muscular dystrophy (DMD). In this study, the investigators associated with the Cooperative International Neuromuscular Research Group CINRG) will take a detailed look (for a minimum of eight years) at DMD participant's… Read more about this study
- Who is it for?
- Ages 2 to 30 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- Duchenne Muscular Dystrophy
- How many people
- 551 participants
- Where
- 21 sitesin 8 countries
- Study dates
- Dec 2005to Dec 2019
- Phase
- Observational
- 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 2 to 30 years
- Men only
- Healthy volunteers accepted
- Child, Adult
Study population: The incidence of DMD is considered equal across racial and ethnic groups. At the start of the CINRG program, it was assumed by investigators that the aggregate subject populations of the participating US sites should closely mirror the racial and ethnic distribution of the US population. DMD is an X-linked recessive disease affecting only males. However, female carriers of the disease can be symptomatic due to skewed X-inactivation, a secondary genetic event. We have opted to study the most commonly affected population, males, to ensure subject homogeneity. As such, there are no outreach programs planned for women in this study.
DMD Subject Inclusion Criteria
- Affected subjects must be male and between the ages of 2 and 30
- Affected subjects between the ages of 2 and 4 must have a diagnosis of DMD confirmed by at least one the following OR have an older male sibling that meet at least one of the following criteria:
- Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin deficiency, and clinical picture consistent with typical Duchenne dystrophy OR
- Gene deletion test positive (missing one or more exons) 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 typical Duchenne dystrophy.
- Complete dystrophin gene sequencing showing an alteration (point mutation, duplication, other) that is expected to preclude production of the dystrophin protein(i.e. nonsense mutation, deletion/duplication leading to a downstream stop codon),with a typical clinical picture of DMD.
- Affected subjects between the ages of 5 and 30 must either fulfill the above criteria OR show evidence of a dystrophinopathy and clinical picture consistent with Duchenne Muscular Dystrophy
- Participants who have documented clinical symptoms referable to a dystrophinopathy and direct support of the diagnosis by either (1) a positive DNA analysis for dystrophin mutation, (2) a muscle biopsy demonstrating abnormal dystrophin, or (3) an elevated CK (>5X normal), and X-linked pedigree and an affected family member who meets either criterion (1) or (2) as described above.
NOTE: Determination of the appropriate clinical symptoms consistent with DMD will generally be the responsibility of the clinician. At a minimum this will include progressive loss of function, with additional consideration for other clinical features such as a characteristic gait, a positive Gower sign and calf pseudohypertrophy. When immunostaining of muscle biopsy is used to determining case definition, the clinical reviewer (site PI) should confirm that appropriate testing has ruled out a secondary deficiency of dystrophin. Affected subjects that do not exhibit the above symptoms consistent with DMD should be excluded.
- Muscle weakness prevalent by 5 years of age
- Non-affected adult subjects must be Parent(s) or legal guardian(s) of an eligible affected subject.
DMD Serum Biomarker Inclusion Criteria
- Participants must meet eligibility criteria for the DMD phenotyping portion of this study
- For the GC-treatment response cohort, participants must initiate GC treatment within the first year of study participation (i.e. between their first study visit and their one year follow-up visit)
DMD Subject Exclusion Criteria
For those subjects that confirm DMD diagnosis through a clinical picture consistent with DMD
- Steroid-naïve subjects ambulating past the 13th birthday
- Steroid users ambulating past the 16th birthday
- Subjects/families who are unwilling or unable to comply with the protocol study procedures or visits
Controls Subject Inclusion Criteria
- Male sex
- Age 6-30 years
- Able to comply with functional testing instructions
Control Serum Biomarker Inclusion criteria
- Participants must be male
- Participants must be free of DMD, other neuromuscular disease, or other significant concomitant illness
- Participant must be free of glucocorticoid therapy
Control Subject Exclusion Criteria
- Musculoskeletal disease
- Musculoskeletal injury within 6 months of enrollment
- Other concomitant illness that precludes functional testing in the judgment of the investigator or clinical evaluator
- Completion of enrollment for age cohort
Trial locations and contacts
21 sites in 8 countries.
21 sites in 8 countries
United States11 sites
Carolinas Medical Center
Charlotte, North Carolina, 28207
Lurie Children's Hospial
Chicago, Illinois, 60614
Duke Children's Hospital
Durham, North Carolina, 27710
University of of Florida
Gainesville, Florida, 32610
University of Tennessee
Memphis, Tennessee, 38104
University of Minnesota
Minneapolis, Minnesota, 55455
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15213
Children's Hospital of Virginia
Richmond, Virginia, 23220
University of California, Davis
Sacramento, California, 95817
Washington University, St. Louis
St Louis, Missouri, 63110
Children's National Medical Center
Washington D.C., District of Columbia, 20010
Canada3 sites
Alberta Children's Hospital
Calgary, Alberta, T2T 5C7
University of Alberta
Edmonton, Alberta, T6G 2J3
Holland Bloorview Kids Rehab Hospital
Toronto, Ontario, M4G 1R8
Australia2 sites
Royal Children's Hospital
Melbourne, Victoria, 3052
The Children's Hospital at Westmead
Sydney, New South Wales, 2145
About this study
Official title: Longitudinal Study of the Relationship Between Impairment, Activity Limitation, Participation and Quality of Life in Persons With Confirmed Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to establish the largest long-term assessment of people with Duchenne muscular dystrophy (DMD). In this study, the investigators associated with the Cooperative International Neuromuscular Research Group CINRG) will take a detailed look (for a minimum of eight years) at DMD participant's physical abilities, the medical problems they experience, and how they use health care services. Physical abilities will be compared to a group of healthy controls.
The second purpose of this study is to find out whether small, normal differences in the genetic makeup of people with DMD (called "single nucleotide polymorphisms" or "SNPs") affect how their disease progresses and relates to muscle strength/size and steroid response.
Read more
The third purpose of this study is to study genetic variations associated with DMD.
The final purpose of this study is to determine whether certain biomarkers are present in people with DMD and not in healthy controls.
Read the detailed description
Phenotyping Study Aims
Aim 1: Longitudinally assess body function and body structure (impairment) through the measurement of anthropometrics, muscle strength and pulmonary function in subjects with DMD through the multicenter CINRG network.
Aim 2: Longitudinally assess activity limitations in subjects with DMD through CINRG with timed motor performance, burden of care, and functional status.
Aim 3: Longitudinally assess secondary conditions in subjects with DMD, and relative risks of developing those conditions based on exposure to preventive interventions.
Aim 4: Longitudinally assess participation, life satisfaction, service utilization and health-related quality of life in subjects with DMD.
Aim 5: Determine appropriate outcome measurements for impairment, activities (activity limitations), participation and quality of life to determine the effect of prednisone and other therapeutic interventions on these factors.
Aim 6: Using the most robust impairment, activity, participation and quality of life outcome measures, determine the sample size, power and statistical methods for the analysis of the effect size for future planned randomized-controlled rehabilitation interventions in DMD.
Aim 7: Examine the associations between interventions and incidence and severity of secondary conditions, achievement of disease milestones and measures of motor function and mobility.
Aim 8: To assess the validity and responsiveness of novel clinical outcome measures in DMD, including the 6-minute walk test (6MWT), the 9-hole peg test (9-HPT) Egen Klassification Scale(EK), the North Star Ambulatory Assessment (NSAA), and quantitative key and pinch grip strength testing.
Aim 9: To assess the reliability, validity and responsiveness of novel patient-reported outcome(PRO) measures in DMD, including the NeuroQOL and PedsQL Neuromuscular Module.
Aim 10: To assess the clinical meaningfulness of novel objective clinical outcome measures by assessing their ability to predict milestones of loss of ability to stand from supine, to stand from a seated position, to climb stairs, to ambulate independently and to raise a hand to the mouth.
AIM 11: To determine the impact of development and growth on outcome measure performance,we will assess physical function on a group of healthy, typically developing male children and adults between 6 and 30 years of age for outcome measures of motor function and strength including the9-HPT, 6MWT, NSAA, timed function tests and quantitative muscle strength (QMT). Test results from this cohort will be used to develop initial percent predicted for age values for these assessments.
SNP Genotyping Study Aim
Our goal of the proposed study is to define polygenic modifiers of disease progression, and also response to treatment with glucocorticoids (prednisone and deflazacort). The most common type of human genetic variation is the single-nucleotide polymorphism (SNP, a base position at which two alternative bases occur at an appreciable frequency (>1%) in the population. SNPs are 90% of variation in the human genome. SNPs occur on the average of 1 per 1000 to 2000 bp throughout the 3.2 billion bp of the human genome while coding region SNPs (cSNPs) occur on the average of 1 per 346 bp.
Genome-wide Association Study Aim
Our goal of the proposed study is to isolate genomic DNA and find possible correlations of clinical phenotypes with gene loci associated with mild vs. severe clinical presentation, progression, or response to steroids.
Serum Biomarkers Study Aim
Our goal is to discover and validate sensitive and specific serum biomarkers for DMD.
- Conditions
- Duchenne Muscular Dystrophy
- 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.
| Study group | Role | What they receive |
|---|---|---|
| Ongoing Duchenne Muscular Dystrophy (DMD) Cohort | 340 patients currently enrolled participants with DMD. | |
| New Young Duchenne Muscular Dystrophy (DMD) Cohort | Additional 100 confirmed DMD participants aged 4-7 years old to be recruited. | |
| Typically Developing Control Cohort | Up to 370 typically developing male children and adults aged 6-30 years old to be recruited. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Strength and function
These assessments include:
- Quantitative muscle testing
- Manual muscle testing
- Pulmonary function testing
- Functional evaluations (nine hole peg, six minute walk, North Star ambulatory assessment, Brooke and Vignos scales, Egen Klassification (EK) scale, and range of motion).
Measured: Collected at yearly visits
Quality of life
These questionnaires include:
- Pediatric Quality of Life Inventory (PedsQL)
- Pediatric Orthopaedic Functional Health Questionnaire of the Pediatric Orthopaedic Society of North America (POSNA)
- World Health Organization Quality of Life Assessment - Brief (WHO QOL Brief)
- Pediatrics and Adult Neuromuscular module Quality of Life (NeuroQOL)
- Review of Systems
Measured: Collected at yearly visits
Medical history assessment
Outcomes on ambulation status, medication history, hospitalizations, surgeries, nutrition, fractures, and cardiac tests.
Measured: Collected at yearly visits
Secondary outcomes (1)
Biomarkers and genetic modifiers
Genotyping and Serum Biomarkers include blood/saliva collection for:
- Blood collection for Genome Wide Association Study (GWAS) analysis (one time sample, any visit)
- Blood or saliva collection for SNP sample (one time sample, any visit)
- Blood collection for biomarker analysis (collected at each visit)
Measured: Collected either once at any visit or each visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 May 2007 (estimated)
- Study startDec 2005
- Primary completionDec 2019 (estimated)
- Study completionDec 2019 (estimated)
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.
- 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
- Observational
- Observational model
- Case-control
- Time perspective
- Prospective
- Enrolment
- 551 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cooperative International Neuromuscular Research Group
- Sponsor type
- Research network
- Collaborators
- U.S. Department of Education, National Institutes of Health (NIH), United States Department of Defense, Parent Project Muscular Dystrophy
- Responsible party
- Sponsor
- Investigators
- Craig McDonald, MD, Study chair, University of California, Davis
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00468832
- Sponsor's protocol ID
- UCD0305
- Status last verified
- Apr 2016
- First submitted
- 1 May 2007
- Last updated
- 21 Apr 2016
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Thangarajh M, Spurney CF, Gordish-Dressman H, Clemens PR, Hoffman EP, McDonald CM, Henricson EK; CINRG Investigators. Neurodevelopmental Needs in Young Boys with Duchenne Muscular Dystrophy (DMD): Observations from the Cooperative International Neuromuscular Research Group (CINRG) DMD Natural History Study (DNHS). PLoS Curr. 2018 Oct 17;10:ecurrents.md.4cdeb6970e54034db2bc3dfa54b4d987. doi: 10.1371/currents.md.4cdeb6970e54034db2bc3dfa54b4d987.
PubMed 30443431McDonald CM, Henricson EK, Abresch RT, Duong T, Joyce NC, Hu F, Clemens PR, Hoffman EP, Cnaan A, Gordish-Dressman H; CINRG Investigators. Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study. Lancet. 2018 Feb 3;391(10119):451-461. doi: 10.1016/S0140-6736(17)32160-8. Epub 2017 Nov 22.
PubMed 29174484
Study at a glance
From the registry record, last updated by the sponsor on 21 Apr 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Longitudinal Study of the Relationship Between Impairment, Activity Limitation, Participation and Quality of Life in Persons With Confirmed Duchenne Muscular Dystrophy (DMD)
- ClinicalTrials.gov ID
- NCT00468832
- Acronym / sponsor ID
- UCD0305
- Conditions
- Duchenne Muscular Dystrophy
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 551 participants (actual)
- Lead sponsor
- Cooperative International Neuromuscular Research GroupResearch network
- Collaborators
- U.S. Department of Education, National Institutes of Health (NIH), United States Department of Defense, Parent Project Muscular Dystrophy
- Study start
- Dec 2005
- Primary completion
- Dec 2019 (estimated)
- Study completion
- Dec 2019 (estimated)
- First posted
- 3 May 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 establish the largest long-term assessment of people with Duchenne muscular dystrophy (DMD). In this study, the investigators associated with the Cooperative International Neuromuscular Research Group CINRG) will take a detailed look (for a minimum of eight years) at DMD participant's physical abilities, the medical problems they experience, and how they use health care services. Physical abilities will be compared to a group of healthy controls.
The second purpose of this study is to find out whether small, normal differences in the genetic makeup of people with DMD (called "single nucleotide polymorphisms" or "SNPs") affect how their disease progresses and relates to muscle strength/size and steroid response.
The third purpose of this study is to study genetic variations associated with DMD.
The final purpose of this study is to determine whether certain biomarkers are present in people with DMD and not in healthy controls.
Detailed description
Phenotyping Study Aims
Aim 1: Longitudinally assess body function and body structure (impairment) through the measurement of anthropometrics, muscle strength and pulmonary function in subjects with DMD through the multicenter CINRG network.
Aim 2: Longitudinally assess activity limitations in subjects with DMD through CINRG with timed motor performance, burden of care, and functional status.
Aim 3: Longitudinally assess secondary conditions in subjects with DMD, and relative risks of developing those conditions based on exposure to preventive interventions.
Aim 4: Longitudinally assess participation, life satisfaction, service utilization and health-related quality of life in subjects with DMD.
Aim 5: Determine appropriate outcome measurements for impairment, activities (activity limitations), participation and quality of life to determine the effect of prednisone and other therapeutic interventions on these factors.
Aim 6: Using the most robust impairment, activity, participation and quality of life outcome measures, determine the sample size, power and statistical methods for the analysis of the effect size for future planned randomized-controlled rehabilitation interventions in DMD.
Aim 7: Examine the associations between interventions and incidence and severity of secondary conditions, achievement of disease milestones and measures of motor function and mobility.
Aim 8: To assess the validity and responsiveness of novel clinical outcome measures in DMD, including the 6-minute walk test (6MWT), the 9-hole peg test (9-HPT) Egen Klassification Scale(EK), the North Star Ambulatory Assessment (NSAA), and quantitative key and pinch grip strength testing.
Aim 9: To assess the reliability, validity and responsiveness of novel patient-reported outcome(PRO) measures in DMD, including the NeuroQOL and PedsQL Neuromuscular Module.
Aim 10: To assess the clinical meaningfulness of novel objective clinical outcome measures by assessing their ability to predict milestones of loss of ability to stand from supine, to stand from a seated position, to climb stairs, to ambulate independently and to raise a hand to the mouth.
AIM 11: To determine the impact of development and growth on outcome measure performance,we will assess physical function on a group of healthy, typically developing male children and adults between 6 and 30 years of age for outcome measures of motor function and strength including the9-HPT, 6MWT, NSAA, timed function tests and quantitative muscle strength (QMT). Test results from this cohort will be used to develop initial percent predicted for age values for these assessments.
SNP Genotyping Study Aim
Our goal of the proposed study is to define polygenic modifiers of disease progression, and also response to treatment with glucocorticoids (prednisone and deflazacort). The most common type of human genetic variation is the single-nucleotide polymorphism (SNP, a base position at which two alternative bases occur at an appreciable frequency (>1%) in the population. SNPs are 90% of variation in the human genome. SNPs occur on the average of 1 per 1000 to 2000 bp throughout the 3.2 billion bp of the human genome while coding region SNPs (cSNPs) occur on the average of 1 per 346 bp.
Genome-wide Association Study Aim
Our goal of the proposed study is to isolate genomic DNA and find possible correlations of clinical phenotypes with gene loci associated with mild vs. severe clinical presentation, progression, or response to steroids.
Serum Biomarkers Study Aim
Our goal is to discover and validate sensitive and specific serum biomarkers for DMD.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Ongoing Duchenne Muscular Dystrophy (DMD) Cohort | – | –340 patients currently enrolled participants with DMD. |
| New Young Duchenne Muscular Dystrophy (DMD) Cohort | – | –Additional 100 confirmed DMD participants aged 4-7 years old to be recruited. |
| Typically Developing Control Cohort | – | –Up to 370 typically developing male children and adults aged 6-30 years old to be recruited. |
Who may qualify
- Ages
- 2 to 30 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
- Study population
- The incidence of DMD is considered equal across racial and ethnic groups. At the start of the CINRG program, it was assumed by investigators that the aggregate subject populations of the participating US sites should closely mirror the racial and ethnic distribution of the US population. DMD is an X-linked recessive disease affecting only males. However, female carriers of the disease can be symptomatic due to skewed X-inactivation, a secondary genetic event. We have opted to study the most commonly affected population, males, to ensure subject homogeneity. As such, there are no outreach programs planned for women in this study.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Strength and function
Time frame: Collected at yearly visits
These assessments include: * Quantitative muscle testing * Manual muscle testing * Pulmonary function testing * Functional evaluations (nine hole peg, six minute walk, North Star ambulatory assessment, Brooke and Vignos scales, Egen Klassification (EK) scale, and range of motion).
Quality of life
Time frame: Collected at yearly visits
These questionnaires include: * Pediatric Quality of Life Inventory (PedsQL) * Pediatric Orthopaedic Functional Health Questionnaire of the Pediatric Orthopaedic Society of North America (POSNA) * World Health Organization Quality of Life Assessment - Brief (WHO QOL Brief) * Pediatrics and Adult Neuromuscular module Quality of Life (NeuroQOL) * Review of Systems
Medical history assessment
Time frame: Collected at yearly visits
Outcomes on ambulation status, medication history, hospitalizations, surgeries, nutrition, fractures, and cardiac tests.
1 secondary outcome
Biomarkers and genetic modifiers
Time frame: Collected either once at any visit or each visit
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Apr 2016.
- Derived from this study: PubMed 30443431Thangarajh M, Spurney CF, Gordish-Dressman H, Clemens PR, Hoffman EP, McDonald CM, Henricson EK; CINRG Investigators. Neurodevelopmental Needs in Young Boys with Duchenne Muscular Dystrophy (DMD): Observations from the Cooperative International Neuromuscular Research Group (CINRG) DMD Natural History Study (DNHS). PLoS Curr. 2018 Oct 17;10:ecurrents.md.4cdeb6970e54034db2bc3dfa54b4d987. doi: 10.1371/currents.md.4cdeb6970e54034db2bc3dfa54b4d987.
- Derived from this study: PubMed 29174484McDonald CM, Henricson EK, Abresch RT, Duong T, Joyce NC, Hu F, Clemens PR, Hoffman EP, Cnaan A, Gordish-Dressman H; CINRG Investigators. Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study. Lancet. 2018 Feb 3;391(10119):451-461. doi: 10.1016/S0140-6736(17)32160-8. Epub 2017 Nov 22.
- CINRG WebsiteLink 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.
Craig McDonald, MD study chair
University of California, Davis
21 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Duchenne Muscular Dystrophy, run by Cooperative International Neuromuscular Research Group at 21 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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: status not confirmed, as of the sponsor's last update on 21 Apr 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 21 sites in United States, Canada, Australia, Argentina, India and 3 more countries. 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.
Are results available for this study?
Results are not available in the source record.
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?
Strength and function (time frame: Collected at yearly visits). The study lists 3 primary outcomes in all. 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 NCT00468832, 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 21 Apr 2016.