Charcot-Marie-Tooth DiseaseNCT05902351
Natural History Study for Charcot Marie Tooth Disease
An observational study of Charcot-Marie-Tooth Disease and Charcot-Marie-Tooth, run by Hereditary Neuropathy Foundation at 1 site in United States.
Trial 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… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Charcot-Marie-Tooth Disease, Charcot-Marie-Tooth, Charcot-Marie-Tooth Disease, Type IA
- How many people
- 10,000 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Nov 2013to 31 Dec 2029
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: This study is open to anyone that has Charcot-Marie-Tooth Disease or other Inherited Neuropathies.
Who may be able to join
- 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.
Who cannot join
- People that do not have Charcot-Marie-Tooth or other Inherited Neuropathies
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Hereditary Neuropathy Foundation
New York, New York, 10128
Allison Moore, 2127228396, allison@hnf-cure.org
Joy Aldrich, 2127228396, joyaldrich@hnf-cure.org
About this study
Official title: Global Registry for Inherited Neuropathies Natural History Study for Charcot Marie Tooth Disease
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, Charcot-Marie-Tooth Disease, Type 2C
- Keywords
- Inherited Neuropathies, Peripheral Neuropathy, Charcot-Marie-Tooth, Charcot-Marie-Tooth Disease, CMT
- Medical subject headings
- Charcot-Marie-Tooth Disease, Charcot-Marie-Tooth disease, Type 2A, Charcot-Marie-Tooth disease, Type 2C, Charcot-Marie-Tooth disease, Type 2B2, Charcot-Marie-Tooth Disease, Axonal, Type 2a1, Charcot-Marie-Tooth disease, Type 4B1, Limb-girdle muscular dystrophy, type 1B, Disease
What is being measured
The primary outcome is the main question the study is designed to answer.
Identify the type of CMT
Patient-Reported Outcomes depending on individual experience I.e. Genetic testing, clinical observation, EMG, family history.
Measured: 156 weeks
Disease Symptoms
Patient-Reported Observations
Measured: 156 weeks
Impact of symptoms on Activities of Daily Living
Patient-Reported Observations
Measured: 156 weeks
Associated Comorbidities
Patient-Reported Observations
Measured: 156 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jun 2023
- Study start1 Nov 2013
- Primary completion31 Dec 2029 (estimated)
- Study completion31 Dec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Charcot-Marie-Tooth DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 10,000 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hereditary Neuropathy Foundation
- Sponsor type
- Other government
- Responsible party
- Sponsor
- Investigators
- Allison Moore, Principal investigator, Hereditary Neuropathy Foundation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05902351
- Sponsor's protocol ID
- GRIN1001
- Status last verified
- Sep 2026
- First submitted
- 2 May 2023
- Last updated
- 17 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 17 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Global Registry for Inherited Neuropathies Natural History Study for Charcot Marie Tooth Disease
- ClinicalTrials.gov ID
- NCT05902351
- Acronym / sponsor ID
- GRIN1001
- 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, Charcot-Marie-Tooth Disease, Type 2C
- Keywords
- Inherited Neuropathies, Peripheral Neuropathy, Charcot-Marie-Tooth, Charcot-Marie-Tooth Disease, CMT
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 10,000 participants (estimated)
- Lead sponsor
- Hereditary Neuropathy FoundationOther government
- Study start
- 1 Nov 2013 (actual)
- Primary completion
- 31 Dec 2029 (estimated)
- Study completion
- 31 Dec 2029 (estimated)
- First posted
- 15 Jun 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- This study is open to anyone that has Charcot-Marie-Tooth Disease or other Inherited Neuropathies.
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (1 listed)
- People that do not have Charcot-Marie-Tooth or other Inherited Neuropathies
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Sep 2026.
- Landing page and entry for patients to participate in the Global Registry for Inherited NeuropathiesLink provided by the sponsor.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Allison Moore principal investigator
Hereditary Neuropathy Foundation
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Charcot-Marie-Tooth Disease and Charcot-Marie-Tooth, run by Hereditary Neuropathy Foundation at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 17 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Allison Moore (212-722-8396, allison@hnf-cure.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Identify the type of CMT (time frame: 156 weeks). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Sep 2026.