Development of Charcot Marie Tooth Disease (CMT) Pediatric Scale for Children With CMT

(INC-6603 Trial)

Recruiting at10 trial locations

Overseen by Michael E Shy, MD

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Academic

Recruiting

Sponsor: University of Iowa

No Placebo Group

Trial Summary

What is the purpose of this trial?

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.

Research Team

Michael E Shy, MD

Principal Investigator

University of Iowa

Eligibility Criteria

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

Treatment Details

Interventions

• N/A (N/A)

Who Is Running the Clinical Trial?

University of Iowa

Lead Sponsor

Trials

486

Recruited

934,000+

Dr. Afable

University of Iowa

Chief Medical Officer since 2005

MD from Loyola Stritch School of Medicine, MPH from the University of Illinois

Joel Harris

University of Iowa

Chief Executive Officer since 2023

B.A. in Economics from the University of Iowa, Post-baccalaureate Certificate in Narrative Communications from Northwestern University

National Institute of Neurological Disorders and Stroke (NINDS)

Collaborator

Trials

1,403

Recruited

655,000+

Jordan Gladman

National Institute of Neurological Disorders and Stroke (NINDS)

Chief Medical Officer

MD from Harvard Medical School

Walter J. Koroshetz

National Institute of Neurological Disorders and Stroke (NINDS)

Chief Executive Officer since 2007

MD from the University of Chicago

University of Rochester

Collaborator

Trials

883

Recruited

555,000+

Kevin Koch

University of Rochester

Chief Executive Officer since 2020

PhD in Organic Chemistry from the University of Rochester

Brian Druker

University of Rochester

Chief Medical Officer since 2015

MD from Harvard Medical School

Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta

Collaborator

Trials

84

Recruited

317,000+

Prof. Giuseppe Turchetti

Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta

Chief Executive Officer since 2024

PhD in Economics, University of Pisa

Dr. Lorenzo Maggi

Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta

Chief Medical Officer since 2022

MD, specialization in Neurology

Nemours Children's Clinic

Collaborator

Trials

128

Recruited

18,000+

R. Lawrence Moss

Nemours Children's Clinic

Chief Executive Officer since 2018

MD, FACS, FAAP

Matthew Davis

Nemours Children's Clinic

Chief Medical Officer since 2023

MD, MAPP

University College London Hospitals

Collaborator

Trials

204

Recruited

1,221,000+

Dr. Jonathan Broomberg

University College London Hospitals

Chief Medical Officer

MD from University of Witwatersrand

David Probert

University College London Hospitals

Chief Executive Officer since 2021

MBA from UCL Global Business School for Health

Children's Hospital of Philadelphia

Collaborator

Trials

749

Recruited

11,400,000+

Joseph W. St. Geme III

Children's Hospital of Philadelphia

Chief Medical Officer since 2021

MD, PhD, MPH

Madeline Bell

Children's Hospital of Philadelphia

Chief Executive Officer since 2015

BSc in Nursing from Villanova University, MSc in Organizational Dynamics from the University of Pennsylvania

Muscular Dystrophy Association

Collaborator

Trials

38

Recruited

60,800+

Sydney Children's Hospitals Network

Collaborator

Trials

37

Recruited

43,300+