Charcot Marie Tooth Disease6604NCT01455623
Development and Validation of a Disability Severity Index for CMT
An observational study of Charcot Marie Tooth Disease, run by University of Iowa at 1 site in United States.
Trial summary
It is necessary for outcome measures to accurately reflect the state of health of a person in order for clinical trials to show benefit. The most commonly used outcome measure for Charcot Marie Tooth Disease (CMT) is the CMT Neuropathy Score, which uses cutoffs of points designated as mild (0-10 points), moderate… Read more about this study
- Who is it for?
- Ages 8 years and olderHealthy volunteers can take part
- Conditions the study is for
- Charcot Marie Tooth Disease
- How many people
- 291 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2011to Mar 2014
- 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 8 years and older
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: Two cohorts: 1. Patients self identified as having CMT and have chosen to be a part of the Inherited Neuropathies Consortium Contact Registry, housed by the Rare Disease Clinical Research Network. 2. Healthcare Providers working in the field of CMT.
Who may be able to join
- patients with CMT ages 8-100,
- health care professionals who take care of patients with CMT.
- Eligible patients will be patients that have joined the INC RDCRN Contact Registry. These will be patients or guardians with known or probable inherited neuropathies.
- Eligible health care professionals who participate in the care of patients with inherited neuropathies (physicians, genetic counselors, physical and occupational therapists etc.) attending the 4th International CMT Consortium to be held in Potomac Maryland June 29-July 1, 2011.
Who cannot join
- Does not read or speak English.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Development and Validation of a Disability Severity Index for Charcot Marie Tooth Disease
It is necessary for outcome measures to accurately reflect the state of health of a person in order for clinical trials to show benefit. The most commonly used outcome measure for Charcot Marie Tooth Disease (CMT) is the CMT Neuropathy Score, which uses cutoffs of points designated as mild (0-10 points), moderate (11-20) or severe (21-36). These terms are arbitrary. This study is looking to base mild, moderate, and severe on what both people affected with CMT and those who provide for people with CMT consider appropriate.
- Conditions
- Charcot Marie Tooth Disease
- Keywords
- CMT
- Medical subject headings
- Charcot-Marie-Tooth Disease
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 |
|---|---|---|
| Health Care Provider | A person working within the field of CMT. | |
| Patient with CMT | Any person of any age self-identifying as having CMT and belonging to the Inherited Neuropathies Consortium Contact Registry hosted by the Rare Disease Clinical Research Network. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Compare Patient and Healthcare Provider impressions of what constitutes mild, moderate and severe impairment in CMT
Measured: 1 year
Secondary outcomes (1)
Develop a Disability Severity Index including items with high patient-physician concordance
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Oct 2011 (estimated)
- Study startJun 2011
- Primary completionMar 2014
- Study completionMar 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Charcot Marie Tooth DiseaseStudies of the same condition, open and under way.
- More trials run by University of IowaThe 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 291 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Iowa
- Sponsor type
- University, hospital or other
- Collaborators
- National Institute of Neurological Disorders and Stroke (NINDS), Muscular Dystrophy Association
- Responsible party
- Principal investigator, Michael Shy, University of Iowa
- Investigators
- Sindhu Ramchandren, MD, Principal investigator, Wayne State University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01455623
- Sponsor's protocol ID
- INC-6604
- Acronym
- 6604
- Status last verified
- Mar 2014
- First submitted
- 17 Oct 2011
- Last updated
- 26 Mar 2014
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Mar 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Development and Validation of a Disability Severity Index for Charcot Marie Tooth Disease
- ClinicalTrials.gov ID
- NCT01455623
- Acronym / sponsor ID
- 6604 / INC-6604
- Conditions
- Charcot Marie Tooth Disease
- Keywords
- CMT
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 291 participants (actual)
- Lead sponsor
- University of IowaUniversity, hospital or other
- Collaborators
- National Institute of Neurological Disorders and Stroke (NINDS), Muscular Dystrophy Association
- Study start
- Jun 2011
- Primary completion
- Mar 2014 (actual)
- Study completion
- Mar 2014 (actual)
- First posted
- 20 Oct 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.
It is necessary for outcome measures to accurately reflect the state of health of a person in order for clinical trials to show benefit. The most commonly used outcome measure for Charcot Marie Tooth Disease (CMT) is the CMT Neuropathy Score, which uses cutoffs of points designated as mild (0-10 points), moderate (11-20) or severe (21-36). These terms are arbitrary. This study is looking to base mild, moderate, and severe on what both people affected with CMT and those who provide for people with CMT consider appropriate.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Health Care Provider | – | –A person working within the field of CMT. |
| Patient with CMT | – | –Any person of any age self-identifying as having CMT and belonging to the Inherited Neuropathies Consortium Contact Registry hosted by the Rare Disease Clinical Research Network. |
Who may qualify
- Ages
- 8 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Two cohorts: 1. Patients self identified as having CMT and have chosen to be a part of the Inherited Neuropathies Consortium Contact Registry, housed by the Rare Disease Clinical Research Network. 2. Healthcare Providers working in the field of CMT.
Key inclusion criteria (4 listed)
- patients with CMT ages 8-100,
- health care professionals who take care of patients with CMT.
Key exclusion criteria (1 listed)
- Does not read or speak English.
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
Compare Patient and Healthcare Provider impressions of what constitutes mild, moderate and severe impairment in CMT
Time frame: 1 year
1 secondary outcome
Develop a Disability Severity Index including items with high patient-physician concordance
Time frame: 1 year
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 26 Mar 2014.
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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.
Sindhu Ramchandren, MD principal investigator
Wayne State University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Charcot Marie Tooth Disease, run by University of Iowa 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 4 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: finished, as of the sponsor's last update on 26 Mar 2014. 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.
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?
Compare Patient and Healthcare Provider impressions of what constitutes mild, moderate and severe impairment in CMT (time frame: 1 year). 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 26 Mar 2014.