DystrophinopathiesNCT02146586
Clinical Evaluator Outcomes Reliability Study
An observational study of Dystrophinopathies, run by Cooperative International Neuromuscular Research Group.
Trial summary
This study outlines structured Clinical Evaluator (CE) testing techniques that are proposed to be implemented across all sites participating in the Cooperative International Neuromuscular Research Group (CINRG) research studies. Read more about this study
- Who is it for?
- Ages 6 years and older, men only
- Conditions the study is for
- Dystrophinopathies
- How many people
- 55 participants
- Where
- No sites listed yet
- Study dates
- May 2014to Dec 2016
- 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 6 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Study population: Dystrophinopathies (Duchenne Muscular Dystrophy and Becker Muscular Dystrophy)
Dystrophinopathy Participant Inclusion Criteria:
- Site confirmed genetic diagnosis of a dystrophinopathy
- Aged 6 years and older
- Able to transfer to testing table
- Able to walk 10 meters without an assistive device
Dystrophinopathy Participant Exclusion Criteria:
- Investigator assessment of inability to comply with protocol assessments
- Participants who have attention deficient disorder or other cognitive conditions that may limit attention span to perform protocol assessments
About this study
Official title: Cooperative International Neuromuscular Research Group (CINRG) Clinical Evaluator Outcomes Reliability Study
This study outlines structured Clinical Evaluator (CE) testing techniques that are proposed to be implemented across all sites participating in the Cooperative International Neuromuscular Research Group (CINRG) research studies. The study will determine if the selected techniques are reliable and reproducible across the CINRG network by evaluating the reliability and reproducibility of the measures between CEs
- Conditions
- Dystrophinopathies
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 |
|---|---|---|
| Dystrophinopathies | Not described | |
| Gold Standard Clinical Evaluators (GS-CEs) | Not described | |
| Sites Clinical Evaluators (CEs) | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Clinical Evaluator Assessments
The following assessments are conducted by certified physical therapists: Anthropometrics, goniometry, Brooke upper extremity and Vignos lower extremity scales, Performance Upper Limb Scale (PULS), North Star Ambulatory Assessment (NSAA), Six Minute Walk Test (6MWT), time to rise from floor, time to stand from a chair, time to climb four stairs, time to walk/run 10 meters, 9 Hole Peg Test (9HPT), Quantitative Muscle Tests (QMT), Manual Muscle testing (MMT), Force Vital Capacity (FVC), Force Expiratory Volume (FEV1), Maximum Inspiratory Pressure (MIP), Maximum Expiratory Pressure (MEP), Peak Expiratory Flow Rate (PEFR) and Peak Cough Flow (PCF).
The reliability of these commonly used measurements is fundamental to clinical research, our ability to have confidence in the data we collect and our ability to draw rational conclusions from the data.
Measured: One time visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 May 2014 (estimated)
- Study startMay 2014
- Primary completionDec 2016 (estimated)
- Study completionDec 2016 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for DystrophinopathiesStudies 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
- 55 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Cooperative International Neuromuscular Research Group
- Sponsor type
- Research network
- Responsible party
- Sponsor
- Investigators
- Lauren Morgenroth, MS, CGC, Principal investigator, Children's National Research InstituteTina Duong, MPT, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02146586
- Sponsor's protocol ID
- CNMC0713
- Status last verified
- Feb 2016
- First submitted
- 14 May 2014
- Last updated
- 2 Feb 2016
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 2 Feb 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Cooperative International Neuromuscular Research Group (CINRG) Clinical Evaluator Outcomes Reliability Study
- ClinicalTrials.gov ID
- NCT02146586
- Acronym / sponsor ID
- CNMC0713
- Conditions
- Dystrophinopathies
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 55 participants (estimated)
- Lead sponsor
- Cooperative International Neuromuscular Research GroupResearch network
- Study start
- May 2014
- Primary completion
- Dec 2016 (estimated)
- Study completion
- Dec 2016 (estimated)
- First posted
- 26 May 2014 (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 outlines structured Clinical Evaluator (CE) testing techniques that are proposed to be implemented across all sites participating in the Cooperative International Neuromuscular Research Group (CINRG) research studies. The study will determine if the selected techniques are reliable and reproducible across the CINRG network by evaluating the reliability and reproducibility of the measures between CEs
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Dystrophinopathies | – | – |
| Gold Standard Clinical Evaluators (GS-CEs) | – | – |
| Sites Clinical Evaluators (CEs) | – | – |
Who may qualify
- Ages
- 6 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Dystrophinopathies (Duchenne Muscular Dystrophy and Becker Muscular Dystrophy)
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
Clinical Evaluator Assessments
Time frame: One time visit
The following assessments are conducted by certified physical therapists: Anthropometrics, goniometry, Brooke upper extremity and Vignos lower extremity scales, Performance Upper Limb Scale (PULS), North Star Ambulatory Assessment (NSAA), Six Minute Walk Test (6MWT), time to rise from floor, time to stand from a chair, time to climb four stairs, time to walk/run 10 meters, 9 Hole Peg Test (9HPT), Quantitative Muscle Tests (QMT), Manual Muscle testing (MMT), Force Vital Capacity (FVC), Force Expiratory Volume (FEV1), Maximum Inspiratory Pressure (MIP), Maximum Expiratory Pressure (MEP), Peak Expiratory Flow Rate (PEFR) and Peak Cough Flow (PCF). The reliability of these commonly used measurements is fundamental to clinical research, our ability to have confidence in the data we collect and our ability to draw rational conclusions from the data.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Feb 2016.
Related trials for Dystrophinopathies
RecruitingObservational study
Decoding Central Defects in Dystrophinopathies From Diagnostic to Remediation
Dystrophinopathies
RecruitingObservational study
DMD Gene Variants and Cardiac Dysfunction in Young Males With Dystrophinopathies
Duchenne Muscular Dystrophy (DMD), Becker Muscular Dystrophy
Not yet recruitingObservational study
Social Cognition in Dystrophinopathies and Neurodevelopmental Disorders
Duchenne / Becker Muscular Dystrophy, Osteogenesis Imperfecta (OI)
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.
Lauren Morgenroth, MS, CGC principal investigator
Children's National Research Institute
Tina Duong, MPT principal investigator
Stanford University
Questions about this trial
What is this clinical trial studying?
An observational study of Dystrophinopathies, run by Cooperative International Neuromuscular Research Group.
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 2 Feb 2016. Status can change between updates, so confirm with the study contact before making plans.
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?
Clinical Evaluator Assessments (time frame: One time visit). 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 2 Feb 2016.