Natural History Study for Charcot Marie Tooth Disease
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT05902351 |
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Recruitment Status :
Recruiting
First Posted : June 15, 2023
Last Update Posted : June 15, 2023
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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.
| Condition or disease |
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| 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 Charcot-Marie-Tooth Disease, Type 2C Charcot-Marie-Tooth Disease Type 2A2B Charcot-Marie-Tooth Disease Type 2B2 Charcot-Marie-Tooth Disease Type 2A1 Charcot-Marie-Tooth Disease Type 4B1 Charcot-Marie-Tooth Disease, Type IB Charcot-Marie-Tooth Disease Type 2B1 Charcot-Marie-Tooth Disease Type 2U (Diagnosis) Charcot-Marie-Tooth Disease Type 4A Charcot-Marie-Tooth Disease, Type 4A, Axonal Form Charcot-Marie-Tooth Disease Type 2A2A Charcot-Marie-Tooth Disease Type 2S (Disorder) Charcot-Marie-Tooth Disease and Deafness Charcot-Marie-Tooth Disease Type 4B2 Charcot-Marie-Tooth Disease Type 4H Charcot-Marie-Tooth Disease Type 1F Charcot-Marie-Tooth Disease Type 4C Charcot-Marie-Tooth Disease Type 4E Charcot-Marie-Tooth Disease Type 1D Charcot-Marie-Tooth Disease Type 2Q (Diagnosis) Charcot-Marie-Tooth Disease Type 2A2 Charcot-Marie-Tooth Disease Type 2N (Diagnosis) Charcot-Marie-Tooth Disease Type 2B5 Charcot-Marie-Tooth Disease Type 2D Charcot-Marie-Tooth Disease Type 4D Charcot-Marie-Tooth Disease Type 2K Charcot-Marie-Tooth Disease Type 2L (Diagnosis) Charcot-Marie-Tooth Disease Type 2T Charcot-Marie-Tooth Disease Type 2I Charcot-Marie-Tooth Disease Type 2J Charcot-Marie-Tooth Disease Type 2E Charcot-Marie-Tooth Disease Type 2G Charcot-Marie-Tooth Disease Type 1C Charcot-Marie-Tooth Disease Type 2R Charcot-Marie-Tooth Disease Type 2O (Diagnosis) Charcot-Marie-Tooth Disease Type 2M Charcot-Marie-Tooth Disease Type 2P Charcot-Marie-Tooth Disease Type 2Y Charcot-Marie-Tooth Disease Type 4F (Diagnosis) Charcot-Marie-Tooth Disease Type 4B3 Charcot-Marie-Tooth Disease Type 2H HNPP X-Linked Charcot-Marie-Tooth Disease |
| Study Type : | Observational |
| Estimated Enrollment : | 10000 participants |
| Observational Model: | Cohort |
| Time Perspective: | Cross-Sectional |
| Official Title: | Global Registry for Inherited Neuropathies Natural History Study for Charcot Marie Tooth Disease |
| Actual Study Start Date : | November 1, 2013 |
| Estimated Primary Completion Date : | December 31, 2029 |
| Estimated Study Completion Date : | December 31, 2029 |
- Identify the type of CMT [ Time Frame: 156 weeks ]Patient-Reported Outcomes depending on individual experience I.e. Genetic testing, clinical observation, EMG, family history.
- Disease Symptoms [ Time Frame: 156 weeks ]Patient-Reported Observations
- Impact of symptoms on Activities of Daily Living [ Time Frame: 156 weeks ]Patient-Reported Observations
- Associated Comorbidities [ Time Frame: 156 weeks ]Patient-Reported Observations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | Child, Adult, Older Adult |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Non-Probability Sample |
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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05902351
| Contact: Allison Moore | 212-722-8396 | allison@hnf-cure.org | |
| Contact: Joy Aldrich | 212-722-8396 | JoyAldrich@hnf-cure.org |
| United States, New York | |
| Hereditary Neuropathy Foundation | Recruiting |
| New York, New York, United States, 10128 | |
| Contact: Allison Moore 212-722-8396 allison@hnf-cure.org | |
| Contact: Joy Aldrich 2127228396 joyaldrich@hnf-cure.org | |
| Principal Investigator: | Allison Moore | Hereditary Neuropathy Foundation |
| Responsible Party: | Hereditary Neuropathy Foundation |
| ClinicalTrials.gov Identifier: | NCT05902351 |
| Other Study ID Numbers: |
GRIN1001 |
| First Posted: | June 15, 2023 Key Record Dates |
| Last Update Posted: | June 15, 2023 |
| Last Verified: | June 2023 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | Undecided |
| Studies a U.S. FDA-regulated Drug Product: | No |
| Studies a U.S. FDA-regulated Device Product: | No |
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Inherited Neuropathies Peripheral Neuropathy Charcot-Marie-Tooth Charcot-Marie-Tooth Disease CMT |
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Tooth Diseases Deafness Charcot-Marie-Tooth Disease Nerve Compression Syndromes Hereditary Sensory and Motor Neuropathy Refsum Disease Disease Pathologic Processes Hearing Loss Hearing Disorders Ear Diseases Otorhinolaryngologic Diseases Sensation Disorders Neurologic Manifestations Nervous System Diseases |
Stomatognathic Diseases Nervous System Malformations Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases Polyneuropathies Peripheral Nervous System Diseases Neuromuscular Diseases Congenital Abnormalities Genetic Diseases, Inborn Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Metabolism, Inborn Errors Peroxisomal Disorders |