Condition (MeSH)
Charcot-Marie-Tooth Disease
ClinicalTrials.gov lists 111 trials for Charcot-Marie-Tooth Disease or a narrower condition beneath it.
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Trials per phase (log scale)
111 of the 111 trials are tagged with Charcot-Marie-Tooth Disease itself; the rest are tagged with a narrower condition beneath it.
When did Charcot-Marie-Tooth Disease trials start? (20)
Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year. 1 trial is registered to start in 2027 or later, and not listed here.
Trials over time, by phase
- Phase 1
- Phase 1/2
- Phase 2
- Phase 2/3
- Phase 3
- Not applicable
- Observational
Top of scale: 15
Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.
| Year | Trials started |
|---|---|
| 2026 (partial year) | 9 |
| 2025 | 15 |
| 2024 | 10 |
| 2023 | 8 |
| 2022 | 6 |
| 2021 | 6 |
| 2020 | 6 |
| 2019 | 5 |
| 2018 | 5 |
| 2017 | 7 |
Showing 10 of 20 earlier years.
Forecast of the full-year count, planned starts included, not rows of this table: about 11 trials starting in 2026 and about 13 in 2027, the average of the 24 months through September, counted as registered and carried forward. The rows of this table count trials registered so far with a start date in the year, planned starts included; the forecast looks ahead to the count for the whole year, so it is not the same measure. In backtests on conditions of this size, forecasts made part-way through the year were typically off by about 28 percent and ran about 20 percent low against the count later registered; forecasts made before the year began were off by about 39 percent and ran about 37 percent low against the count later registered. These figures rest on a few years of history (target years from 2018 to 2025, varying by figure), place each condition as it is mapped today rather than as it was then, and score against a count for the newest year that was still being revised. Only condition series were scored.
Prediction, not a registry value: median of 31 months from start to primary completion across 14 trials still running at the 2026-10-09 snapshot; registered median of 18 months across 55 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Charcot-Marie-Tooth Disease trials in? (7)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 3 |
| Phase 1/2 | 4 |
| Phase 2 | 10 |
| Phase 2/3 | 2 |
| Phase 3 | 3 |
| Not applicable | 42 |
| Observational | 47 |
How many Charcot-Marie-Tooth Disease trials are recruiting? (7)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 4 |
| Recruiting | 31 |
| Active, not recruiting | 4 |
| Completed | 50 |
| Terminated | 6 |
Showing 5 of 7 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Charcot-Marie-Tooth Disease trials? (3)
Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.
| Sponsor | Trials |
|---|---|
| Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA | 2 |
| ENCell | 2 |
| NMD Pharma A/S | 2 |
Which drugs are being tested for Charcot-Marie-Tooth Disease? (2)
Shown in this order: most trials first. One row per drug, matched to a single molecule under all its names, ranked by number of trials.
| Drug | Trials |
|---|---|
| ACE-083 | 2 |
| Vitamin C | 2 |
Interventions as named in the registry (13)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| EN001 | 2 |
| EDK060, dose C | 1 |
| Low Level Laser Therapy | 1 |
| MD1003 | 1 |
| CLZ-2002 | 1 |
Showing 5 of 13 interventions.
These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.
Which serious adverse events do Charcot-Marie-Tooth Disease trials report? (10)
Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.
| Adverse event | Subjects affected | Trials |
|---|---|---|
| Sleep apnoea syndrome | 2 | 1 |
| Suicide attempt | 2 | 2 |
| Appendicitis | 1 | 1 |
| Arthrolysis | 1 | 1 |
| Autoimmune encephalopathy | 1 | 1 |
Showing 5 of 10 adverse events.
Which publications cover Charcot-Marie-Tooth Disease? (102)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Postural balance and visual dependence in patients with demyelinating neuropathies differ between acquired and hereditary etiologies. | Revue neurologique | 2025 | Yes |
| ATP1A1-linked diseases require a malfunctioning protein product from one allele. | Biochimica et biophysica acta. Molecular cell research | 2024 | Yes |
| Multicenter Validation of the Charcot-Marie-Tooth Functional Outcome Measure. | Neurology | 2024 | Yes |
| Monoallelic variation in DHX9, the gene encoding the DExH-box helicase DHX9, underlies neurodevelopment disorders and Charcot-Marie-Tooth disease. | American journal of human genetics | 2023 | Yes |
| A medical odyssey of a 72-year-old man with Charcot-Marie-Tooth disease type 2 newly diagnosed with biallelic variants in SORD gene causing sorbitol dehydrogenase deficiency. | American journal of medical genetics. Part A | 2023 | Yes |
Showing 5 of 102 publications.
Prevalence (33)
Shown in this order: jurisdiction, then disease name. Orphanet observation for the mapped ORPHA concept; the mapping relation column says whether that concept is exactly this condition or broader or narrower.
| Orphanet disease | Type | Class | Geography | Jurisdiction | Mapping relation |
|---|---|---|---|---|---|
| Charcot-Marie-Tooth disease/Hereditary motor and sensory neuropathy | Point prevalence | 1-5 / 10 000 | United Kingdom | GB | Exact |
| Charcot-Marie-Tooth disease/Hereditary motor and sensory neuropathy | Point prevalence | 1-5 / 10 000 | United Kingdom | GB | Unspecified |
| Charcot-Marie-Tooth disease/Hereditary motor and sensory neuropathy | Point prevalence | 1-5 / 10 000 | United Kingdom | GB | Exact |
| Charcot-Marie-Tooth disease/Hereditary motor and sensory neuropathy | Point prevalence | 1-5 / 10 000 | Europe | Not stated | Exact |
| Charcot-Marie-Tooth disease/Hereditary motor and sensory neuropathy | Point prevalence | 1-5 / 10 000 | Sweden | Not stated | Exact |
Showing 5 of 33 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Charcot-Marie-Tooth disease/Hereditary motor and sensory neuropathy | 166 | Exact | Orphanet Rare Disease Ontology (ORDO) |
| Charcot-Marie-Tooth disease/Hereditary motor and sensory neuropathy | 166 | Exact | Orphadata Science knowledge files, Product 1 |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: sub-conditions in mesh.