Condition (MeSH)
Neuronal Ceroid-Lipofuscinoses
ClinicalTrials.gov lists 49 trials for Neuronal Ceroid-Lipofuscinoses or a narrower condition beneath it. BioMarin Pharmaceutical has the most among industry and government lead sponsors, with 4.
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Trials per phase (log scale)
49 of the 49 trials are tagged with Neuronal Ceroid-Lipofuscinoses itself; the rest are tagged with a narrower condition beneath it.
When did Neuronal Ceroid-Lipofuscinoses trials start? (22)
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.
Trials over time, by phase
- Phase 1
- Phase 1/2
- Phase 2
- Phase 2/3
- Phase 4
- Not applicable
- Observational
Top of scale: 6
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) | 2 |
| 2024 | 1 |
| 2023 | 2 |
| 2022 | 5 |
| 2021 | 6 |
| 2020 | 5 |
| 2019 | 1 |
| 2018 | 2 |
| 2017 | 2 |
| 2016 | 2 |
Showing 10 of 22 earlier years.
No forecast is made for this condition: it averages 2.0 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 53 months across 24 trials with a primary completion typed Actual. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Neuronal Ceroid-Lipofuscinoses trials in? (8)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 7 |
| Phase 1/2 | 10 |
| Phase 2 | 2 |
| Phase 2/3 | 1 |
| Phase 3 | 1 |
| Phase 4 | 1 |
| Not applicable | 2 |
| Observational | 25 |
How many Neuronal Ceroid-Lipofuscinoses trials are recruiting? (8)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 1 |
| Recruiting | 11 |
| Enrolling by invitation | 1 |
| Active, not recruiting | 8 |
| Completed | 20 |
Showing 5 of 8 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Neuronal Ceroid-Lipofuscinoses trials? (4)
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 |
|---|---|
| BioMarin Pharmaceutical | 4 |
| Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | 3 |
| REGENXBIO Inc. | 2 |
| StemCells, Inc. | 2 |
Which drugs are being tested for Neuronal Ceroid-Lipofuscinoses? (1)
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 |
|---|---|
| BMN 190 | 4 |
Interventions as named in the registry (14)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Natural history | 2 |
| Hematopoetic Stem Cell Transplantation | 1 |
| Azathioprine Pill | 1 |
| AAV2CUhCLN2 (3x10^12 particle units) | 1 |
| Human Placental Derived Stem Cell | 1 |
Showing 5 of 14 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 Neuronal Ceroid-Lipofuscinoses 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 | 54 | 1 |
| Hypersensitivity | 16 | 4 |
| Device end of service | 13 | 1 |
| Pyrexia | 10 | 4 |
| Auto recovery | 9 | 1 |
Showing 5 of 10 adverse events.
Which publications cover Neuronal Ceroid-Lipofuscinoses? (50)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Safety and efficacy of cerliponase alfa in children with neuronal ceroid lipofuscinosis type 2 (CLN2 disease): an open-label extension study. | The Lancet. Neurology | 2024 | Yes |
| Exploring concurrent validity of the CLN2 Clinical Rating Scale: Comparison to PedsQL using cerliponase alfa clinical trial data. | PloS one | 2024 | Yes |
| Twenty-Year Survival Analysis of Adeno-Associated Virus Vector Serotype 2-Mediated Gene Therapy to the Central Nervous System for CLN2 Disease. | Human gene therapy | 2024 | Yes |
| Assessment of Safety and Biodistribution of AAVrh.10hCLN2 Following Intracisternal Administration in Nonhuman Primates for the Treatment of CLN2 Batten Disease. | Human gene therapy | 2023 | Yes |
| Ongoing retinal degeneration despite intraventricular enzyme replacement therapy with cerliponase alfa in late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2 disease). | The British journal of ophthalmology | 2023 | Yes |
Showing 5 of 50 publications.
Prevalence (12)
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 |
|---|---|---|---|---|---|
| Neuronal ceroid lipofuscinosis | Prevalence at birth | 1-9 / 100 000 | Norway | Not stated | Exact |
| Neuronal ceroid lipofuscinosis | Annual incidence | 1-9 / 1 000 000 | Italy | Not stated | Exact |
| Neuronal ceroid lipofuscinosis | Point prevalence | 1-9 / 1 000 000 | Italy | Not stated | Exact |
| Neuronal ceroid lipofuscinosis | Point prevalence | 1-9 / 100 000 | Europe | Not stated | Exact |
| Neuronal ceroid lipofuscinosis | Point prevalence | Unknown | Worldwide | Not stated | Exact |
Showing 5 of 12 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Neuronal ceroid lipofuscinosis | 216 | Exact | Orphanet Rare Disease Ontology (ORDO) |
| Neuronal ceroid lipofuscinosis | 216 | 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.