Becker Muscular DystrophyNCT01539772
Becker Muscular Dystrophy - A Natural History Study to Predict Efficacy of Exon Skipping
An observational study of Becker Muscular Dystrophy, run by Cooperative International Neuromuscular Research Group at 16 sites in 4 countries.
Trial summary
This is a multi-center natural history study that will be conducted at participating centers in the Cooperative International Neuromuscular Research Group (CINRG). Following a baseline evaluation, participants will have three follow-up visits over a three-year period. Read more about this study
- Who is it for?
- Ages 4 years and older, men only
- Conditions the study is for
- Becker Muscular Dystrophy
- How many people
- 85 participants
- Where
- 16 sitesin 4 countries
- Study dates
- Apr 2012to Aug 2018
- 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 4 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Study population: BMD participants over 4 years of age with in-frame deletions in the dystrophin gene.
Who may be able to join
- Male
- Age 4 or older
- Diagnosis of BMD with an in-frame deletion in the dystrophin gene, where the boundaries of the mutations are confirmed.
Who cannot join
- Investigator assessment of inability to comply with protocol
Trial locations and contacts
16 sites in 4 countries.
16 sites in 4 countries
United States13 sites
Children's Healthcare of Atlanta
Atlanta, Georgia
Carolinas Medical Center
Charlotte, North Carolina
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois
UT Southwestern Medical Center
Dallas, Texas, 75390-9036
Duke Children's Hospital and Health Center
Durham, North Carolina
University of Florida
Gainesville, Florida
Penn State Hershey Medical Center
Hershey, Pennsylvania, 17033
University of Tennessee
Memphis, Tennessee
University of Minnesota
Minneapolis, Minnesota
University of Pittsburgh
Pittsburgh, Pennsylvania
University of California Davis
Sacramento, California
Washington University
St Louis, Missouri
Children's National Health System
Washington D.C., District of Columbia
United Kingdom1 site
Institute of Genetic Medicine - Newcastle University
Newcastle upon Tyne
About this study
Official title: PITT0112: Becker Muscular Dystrophy - A Natural History Study to Predict Efficacy of Exon Skipping
This is a multi-center natural history study that will be conducted at participating centers in the Cooperative International Neuromuscular Research Group (CINRG). Following a baseline evaluation, participants will have three follow-up visits over a three-year period. The investigators will characterize the Becker muscular dystrophy phenotype, and correlate specific abnormal dystrophin proteins with the range of clinical outcomes.
Read the detailed description
We will utilize the Cooperative International Neuromuscular Research group (CINRG) network to collect cohorts of Becker muscular dystrophy (BMD) patients with in-frame deletions in the dystrophin gene. We will collect clinical data across multiple body systems and correlate these findings to the high-resolution deletion break-point mapping performed from the tissue samples. We will investigate the observed variability to deepen our understanding of molecular mechanisms relevant to the optimization of exon skipping therapeutic approaches.
- Conditions
- Becker 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 |
|---|---|---|
| Becker | BMD participants over 4 years of age with in-frame deletions in the dystrophin gene. . |
What is being measured
The primary outcome is the main question the study is designed to answer.
Strength and function
Measured: Annual
Quality of life
These questionnaires include:
- Pediatric Quality of Life Inventory (PedsQL)
- Pediatrics and Adult Neuromuscular module Quality of Life (NeuroQOL)
Measured: Annual
Medical history assessment - ambulation status, medication history, hospitalizations, surgeries, nutrition, fractures, and cardiac tests
Measured: Annual
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Feb 2012 (estimated)
- Study startApr 2012
- Primary completionAug 2018 (estimated)
- Study completionAug 2018 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Becker 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 85 participants (actual)
Sponsor and investigators
- Lead sponsor
- Cooperative International Neuromuscular Research Group
- Sponsor type
- Research network
- Responsible party
- Sponsor
- Investigators
- Paula R Clemens, MD, Study chair, University of Pittsburgh
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01539772
- Sponsor's protocol ID
- PITT0112
- Status last verified
- Jun 2018
- First submitted
- 22 Feb 2012
- Last updated
- 15 Jun 2018
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 15 Jun 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- PITT0112: Becker Muscular Dystrophy - A Natural History Study to Predict Efficacy of Exon Skipping
- ClinicalTrials.gov ID
- NCT01539772
- Acronym / sponsor ID
- PITT0112
- Conditions
- Becker Muscular Dystrophy
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 85 participants (actual)
- Lead sponsor
- Cooperative International Neuromuscular Research GroupResearch network
- Study start
- Apr 2012
- Primary completion
- Aug 2018 (estimated)
- Study completion
- Aug 2018 (estimated)
- First posted
- 28 Feb 2012 (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 is a multi-center natural history study that will be conducted at participating centers in the Cooperative International Neuromuscular Research Group (CINRG). Following a baseline evaluation, participants will have three follow-up visits over a three-year period. The investigators will characterize the Becker muscular dystrophy phenotype, and correlate specific abnormal dystrophin proteins with the range of clinical outcomes.
Detailed description
We will utilize the Cooperative International Neuromuscular Research group (CINRG) network to collect cohorts of Becker muscular dystrophy (BMD) patients with in-frame deletions in the dystrophin gene. We will collect clinical data across multiple body systems and correlate these findings to the high-resolution deletion break-point mapping performed from the tissue samples. We will investigate the observed variability to deepen our understanding of molecular mechanisms relevant to the optimization of exon skipping therapeutic approaches.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Becker | – | –BMD participants over 4 years of age with in-frame deletions in the dystrophin gene. . |
Who may qualify
- Ages
- 4 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- BMD participants over 4 years of age with in-frame deletions in the dystrophin gene.
Key inclusion criteria (3 listed)
- Male
- Age 4 or older
- Diagnosis of BMD with an in-frame deletion in the dystrophin gene, where the boundaries of the mutations are confirmed.
Key exclusion criteria (1 listed)
- Investigator assessment of inability to comply with protocol
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: Annual
Quality of life
Time frame: Annual
These questionnaires include: * Pediatric Quality of Life Inventory (PedsQL) * Pediatrics and Adult Neuromuscular module Quality of Life (NeuroQOL)
Medical history assessment - ambulation status, medication history, hospitalizations, surgeries, nutrition, fractures, and cardiac tests
Time frame: Annual
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Jun 2018.
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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.
Paula R Clemens, MD study chair
University of Pittsburgh
16 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Becker Muscular Dystrophy, run by Cooperative International Neuromuscular Research Group at 16 sites in 4 countries.
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 3 inclusion and 1 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: status not confirmed, as of the sponsor's last update on 15 Jun 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in United States, Canada, Italy, United Kingdom. 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: Annual). 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?
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 15 Jun 2018.