Condition (MeSH)
Hereditary Central Nervous System Demyelinating Diseases
ClinicalTrials.gov lists 124 trials for Hereditary Central Nervous System Demyelinating Diseases or a narrower condition beneath it. Shire has the most among industry and government lead sponsors, with 8.
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Trials per phase (log scale)
1 more section with rows
4 of the 124 trials are tagged with Hereditary Central Nervous System Demyelinating Diseases itself; the rest are tagged with a narrower condition beneath it.
When did Hereditary Central Nervous System Demyelinating Diseases trials start? (26)
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. 3 trials are 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
- Phase 4
- Not applicable
- Observational
Top of scale: 9
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) | 1 |
| 2025 | 2 |
| 2024 | 4 |
| 2023 | 8 |
| 2022 | 6 |
| 2021 | 8 |
| 2020 | 4 |
| 2019 | 6 |
| 2018 | 9 |
| 2017 | 5 |
Showing 10 of 26 earlier years.
No forecast is made for this condition: it averages 4.0 trial starts a year over the five years to September, under the 5 a year the method needs.
Prediction, not a registry value: median of 36 months from start to primary completion across 5 trials still running at the 2026-10-09 snapshot; registered median of 38 months across 63 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Hereditary Central Nervous System Demyelinating Diseases trials in? (9)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 10 |
| Phase 1/2 | 15 |
| Phase 2 | 17 |
| Phase 2/3 | 5 |
| Phase 3 | 6 |
| Phase 4 | 1 |
| Not applicable | 20 |
| Observational | 43 |
| Expanded access | 7 |
How many Hereditary Central Nervous System Demyelinating Diseases trials are recruiting? (12)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 5 |
| Recruiting | 21 |
| Enrolling by invitation | 2 |
| Active, not recruiting | 8 |
| Available | 1 |
Showing 5 of 12 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Hereditary Central Nervous System Demyelinating Diseases trials? (7)
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 |
|---|---|
| Shire | 8 |
| Genetix Biotherapeutics Inc. | 5 |
| Ionis Pharmaceuticals, Inc. | 4 |
| Orchard Therapeutics | 3 |
| Talaris Therapeutics Inc. | 3 |
Showing 5 of 7 sponsors.
Which drugs are being tested for Hereditary Central Nervous System Demyelinating Diseases? (6)
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 |
|---|---|
| Recombinant human Arylsulfatase A (rhASA) | 3 |
| Melphalan | 3 |
| CLOFARABINE | 2 |
| Cytoxan | 2 |
| zilganersen | 2 |
Showing 5 of 6 drugs.
Interventions as named in the registry (9)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Alemtuzumab s.c. | 5 |
| Cyclosporine 0.05% | 3 |
| Mofetil Mycophenolate | 3 |
| Stem Cell Transplantation | 3 |
| Hematopoietic Stem Cell Transplantation | 2 |
Showing 5 of 9 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 Hereditary Central Nervous System Demyelinating Diseases 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 |
|---|---|---|
| Death | 61 | 3 |
| Pyrexia | 27 | 6 |
| Seizure | 15 | 3 |
| Febrile neutropenia | 12 | 2 |
| Auto recovery | 11 | 2 |
Showing 5 of 10 adverse events.
Which publications cover Hereditary Central Nervous System Demyelinating Diseases? (11)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| yippee like 3 (ypel3) is a novel gene required for myelinating and perineurial glia development. | PLoS genetics | 2020 | Yes |
| Lesion evolution and neurodegeneration in RVCL-S: A monogenic microvasculopathy. | Neurology | 2020 | Yes |
| 5,10-methenyltetrahydrofolate synthetase deficiency causes a neurometabolic disorder associated with microcephaly, epilepsy, and cerebral hypomyelination. | Molecular genetics and metabolism | 2018 | Yes |
| Severe neurodegeneration, progressive cerebral volume loss and diffuse hypomyelination associated with a homozygous frameshift mutation in CSTB. | European journal of human genetics : EJHG | 2017 | Yes |
| A clinical approach to the diagnosis of patients with leukodystrophies and genetic leukoencephelopathies. | Molecular genetics and metabolism | 2015 | Yes |
Showing 5 of 11 publications.
Sub-conditions in MeSH (6)
MeSH descendants one level down with at least one trial, most trials first.
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: prevalence and rare-disease identity.