This trial is no longer recruiting

Recruitment for this study has ended, so applications are closed. You can still read the trial details, or browse similar trials that are currently recruiting.

Not recruiting

Observational Study

Sponsor:

University of Iowa

Code:

NCT01203085

Conditions

Charcot Marie Tooth Disease

Eligibility Criteria

Sex: All

Age: 0 - 21

Healthy Volunteers: Not accepted

Study Details

Brief summary:

The primary goal of this project is to develop and test a Charcot Marie Tooth disease (CMT) Pediatric Scale for use in evaluation in natural history CMT study.

Conditions

Charcot Marie Tooth Disease

Study ID

NCT01203085

Start date

Apr, 2010

Status verified date

May, 2024

Completion date

Dec, 2024

Anticipated

Primary completion date

Dec, 2024

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0 - 21

Healthy Volunteers: Not accepted

Inclusion Criteria:

All patients MUST be seen in person at one of the participating centers for enrollment in this study.

  • Children (< 21 years of age)
  • Known or probable inherited neuropathies classified as CMT1, CMT2, or CMT4

Exclusion Criteria:

  • Known diagnoses of acquired neuropathy including toxic (e. g. medication related neuropathies); metabolic (e.g. diabetic), immune mediated or inflammatory \[acute inflammatory demyelinating polyradiculoneuropathy (AIDP) or chronic inflammatory demyelinating polyneuropathy (CIDP)\] polyneuropathies; neuropathy related to leukodystrophy, congenital muscular dystrophy; and patients with severe general medical conditions.
  • Entirely normal conduction velocities of upper and lower limbs as this suggests that the subject may not have a neuropathy.

Study Design

Enrollment

500 participants

Anticipated

Interventions and Outcome Measures

Arms

Pediatric patients

All patients 21 years of age and under who are enrolled in the 6601 study and have undergone the pediatric scale tests.

Primary outcome measure

  • CMT Peds Scale Part 1: Symptoms [ Time Frame: 1 year ]
  • CMT Peds Score Part 2: Foot and Ankle Involvement [ Time Frame: 1 year ]
  • CMT Peds Scale Part 3: Hand dexterity [ Time Frame: 1 year ]
  • CMT Peds Scale Part 4: Hand strength [ Time Frame: 1 year ]
  • CMT Peds Scale Part 5: Foot Strength [ Time Frame: 1 year ]
  • CMT Peds Score Part 6: Sensation [ Time Frame: 1 year ]
  • CMT Peds Scale Part 7: Balance [ Time Frame: 1 year ]
  • CMT Peds Scale Part 8: Motor Function [ Time Frame: 1 year ]

Central Contacts and Locations

Central contacts

Shawna M Feely, MS

319-384-6362Shawna-Feely@uiowa.edu

Tiffany Grider, MS

319-384-6362Tiffany-Grider@uiowa.edu

Locations

Stanford University

Recruiting

Palo Alto, California, United States, 94305

Contacts

Carly Siskind, MS, LCGC

650-721-5588csiskind@stanfordmed.org

Principal Investigator:

John Day, MD

University of Connecticut/Connecticut Children's Medical Center

Recruiting

Hartford, Connecticut, United States, 06106

Contacts

Jennifer Twatchtman-Bassett, BA

860-837-6512jtwatchtman@connecticutchildrens.org

Principal Investigator:

Gyula Acsadi, MD

Nemours Children's Clinic

Recruiting

Orlando, Florida, United States, 32827

Contacts

Taye Akinbe

407-650-7151taye.akinbi@nemours.org

Principal Investigator:

Omer Abdul Hamid, MD

University of Iowa

Recruiting

Iowa City, Iowa, United States, 52242

Contacts

Nicole M Kressin, MSN, RN

319-384-6362nicole-kressin@uiowa.edu

Principal Investigator:

Michael E Shy, MD

University of Michigan

Recruiting

Ann Arbor, Michigan, United States, 48109

Contacts

Queen Bolden

gmbolden@med.umich.edu

Principal Investigator:

Brett McCray, MD, PhD

University of Rochester

Recruiting

Rochester, New York, United States, 14642

Contacts

Janet Sowden

585-275-1267janet_sowden@urmc.rochester.edu

Principal Investigator:

David Herrmann, MD

Children's Hospital of Philadelphia

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Contacts

Sabrina Yum, MD

215-590-1719YUMS@email.chop.edu

Principal Investigator:

Sabrina Yum, MD

More Information

Sponsor

University of Iowa

Last update posted

May 10, 2024

Last verified

May, 2024

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-25. This information was provided to ClinicalTrials.gov by University of Iowa on 2024-05-10. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.