A Prospective Natural History and Outcome Measure Discovery Study of Charcot-Marie-Tooth Disease, Type 4J

Part of paid clinical trials in San Francisco, California.

Sponsor
Elpida Therapeutics SPC
Study ID
NCT06151600
Status
Recruiting
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Conditions

  • Neuro-Degenerative Disease
  • Neuromuscular Diseases
  • Peripheral Neuropathy

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Study Details

This is a multicenter, longitudinal, prospective observational natural history study of subjects with a molecularly confirmed diagnosis of CMT4J. The study will enroll 20 subjects of any age into a uniform protocol for follow-up and evaluations. Subject visits will occur every 12 months + 4 weeks for up to 2 years.

Key Dates

Start date
Jul 29, 2024
Status verified
Jan 2026
Primary completion
Mar 1, 2031
Completion
Mar 1, 2032

Study Design

Enrollment
20 participants (estimated)

Primary Outcome Measure

Study objective [ Time Frame: 2 years ]

Central Contacts

Locations (3)

FacilityCityStateZIPSite coordinators
Stanford UniversitySan FranciscoCalifornia94305
Rachel Thomas, RN
[email protected]
Keith Gottlieb, PhD
[email protected]
John Day, MD,PhD (PRINCIPAL_INVESTIGATOR)
University of IowaIowa CityIowa52242
Rachel Thomas, RN
[email protected]
Keith Gottlieb, PhD
[email protected]
Michael Shy, MD (PRINCIPAL_INVESTIGATOR)
University of Texas SouthwesternDallasTexas75390
Rachel Thomas, RN
[email protected]
Keith Gottlieb, PhD
[email protected]
Susan Iannaccone, MD (PRINCIPAL_INVESTIGATOR)

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By research site
Stanford University· San Francisco, CAUniversity of Iowa· Iowa City, IAUniversity of Texas Southwestern· Dallas, TX

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