Recruiting

Observational Study

Sponsor:

Hereditary Neuropathy Foundation

Code:

NCT05902351

Conditions

Charcot-Marie-Tooth Disease

Charcot-Marie-Tooth

Charcot-Marie-Tooth Disease, Type IA

Charcot-Marie-Tooth Disease Type 2A

Charcot-Marie-Tooth Disease Type 2

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Study Details

Brief summary:

The goal of this Natural History Study for Charcot-Marie-Tooth is to acquire, record, and analyze patient-reported data and associated genetic reports, Electronic Health Records (EHRs) and clinical notes to identify the burden, diagnostic journey, and prevalence of disease that will aid scientists in their work toward finding a cure.

Participants will be asked to complete a Natural History Survey.

Conditions

Charcot-Marie-Tooth Disease

Charcot-Marie-Tooth

Charcot-Marie-Tooth Disease, Type IA

Charcot-Marie-Tooth Disease Type 2A

Charcot-Marie-Tooth Disease Type 2

Study ID

NCT05902351

Start date

Nov 1, 2013

Status verified date

Sep, 2024

Completion date

Dec 31, 2029

Anticipated

Primary completion date

Dec 31, 2029

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0+

Healthy Volunteers: Not accepted

Inclusion Criteria:

Patients will be made aware of the study by HNF and others (referenced above) and invited to participate. Once patients have reviewed and signed electronically the informed consent document, it is attached to their file.

All affected individuals with CMT/IN are eligible to participate in GRIN with proper informed consent.

Children, adolescents and adults with either a confirmed diagnosis or suspected to have CMT/IN are eligible with parent and/or guardian consent.

Individuals that have been clinically diagnosed through family history and/or standard clinical testing (e.g. neuro exam, EMG, NCS) and/or genetically tested or suspected to have CMT/IN (note: many mutations have not been identified yet) are eligible.

Exclusion Criteria:

People that do not have Charcot-Marie-Tooth or other Inherited Neuropathies

Study Design

Enrollment

10000 participants

Anticipated

Interventions and Outcome Measures

Primary outcome measure

  • Identify the type of CMT [ Time Frame: 156 weeks ]
  • Disease Symptoms [ Time Frame: 156 weeks ]
  • Impact of symptoms on Activities of Daily Living [ Time Frame: 156 weeks ]
  • Associated Comorbidities [ Time Frame: 156 weeks ]

Central Contacts and Locations

Central contacts

Allison Moore

212-722-8396allison@hnf-cure.org

Joy Aldrich

212-722-8396JoyAldrich@hnf-cure.org

Locations

Hereditary Neuropathy Foundation

Recruiting

New York, New York, United States, 10128

Contacts

Allison Moore

212-722-8396allison@hnf-cure.org

Joy Aldrich

2127228396joyaldrich@hnf-cure.org

More Information

Sponsor

Hereditary Neuropathy Foundation

Last update posted

Oct 1, 2024

Last verified

Sep, 2024

Keywords

  • Inherited Neuropathies
  • Peripheral Neuropathy
  • Charcot-Marie-Tooth
  • Charcot-Marie-Tooth Disease
  • CMT

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Hereditary Neuropathy Foundation on 2024-10-01.