Development and Validation of a Disability Severity Index... | NCT01455623

Studies
Development and Validation of a Disability Severity Index for CMT

NCT01455623

Sponsor

University of Iowa

Status

Completed

Last Update Posted

Mar 26, 2014Estimated

Enrollment

291Actual

Phase

Not provided

Conditions

Charcot Marie Tooth Disease

Interventions

Not provided

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Mar 2014

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jun 2011

Primary Completion Date

Mar 2014Actual

Completion Date

Mar 2014Actual

First Submitted Date

Oct 17, 2011

First Submission Date that Met QC Criteria

Oct 19, 2011

First Posted Date

Oct 20, 2011Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Mar 25, 2014

Last Update Posted Date

Mar 26, 2014Estimated

Name	Class
National Institute of Neurological Disorders and Stroke (NINDS)	NIH
Muscular Dystrophy Association	OTHER

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief 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 (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.

Detailed Description

Not provided

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

291Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
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.

Interventions

Not provided

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Compare Patient and Healthcare Provider impressions of what constitutes mild, moderate and severe impairment in CMT		1 year

Secondary Outcomes

Measure	Description	Time Frame
Develop a Disability Severity Index including items with high patient-physician concordance		1 year

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

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.

Exclusion Criteria:

Does not read or speak English.

Accepts Healthy Volunteers

Yes

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

8 Years

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Two cohorts:

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.
Healthcare Providers working in the field of CMT.

Sampling Method

Non-Probability Sample

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Sindhu Ramchandren, MD	Wayne State University	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
University of Iowa		Iowa City	Iowa	52242	United States

Condition Browse Module

MeSH Terms

ID	Term
D002607	Charcot-Marie-Tooth Disease

Ancestor Terms

ID	Term
D015417	Hereditary Sensory and Motor Neuropathy
D009421	Nervous System Malformations
D009422	Nervous System Diseases
D020271	Heredodegenerative Disorders, Nervous System

Browse Leaves

Not provided

Browse Branches

Not provided