An observational study in Batten Disease, Neuronal Ceroid Lipofuscinosis CLN6 and Neuronal Ceroid Lipofuscinosis CLN3, sponsored by Emily de los Reyes. Terminated at 1 site in United States. Per ClinicalTrials.gov, last updated 2025-07-30.
Sponsored by Emily de los Reyes · Observational
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Among 38 studies in Neuronal Ceroid-Lipofuscinoses with a site in United States — 10 open to participants now, 18 under way, none of them phase 3. Neuronal Ceroid-Lipofuscinoses · United States.
Among the 38 studies in Neuronal Ceroid-Lipofuscinoses with a site in United States, this one is the 16th largest by enrollment of the 18 observational ones that give a count, is one of 3 that started in 2021, is one of 18 observational ones, and was the 30th registered.
49 studies on the registry are indexed under Neuronal Ceroid-Lipofuscinoses; 13 are open to participants now.
Browse Neuronal Ceroid-Lipofuscinoses studies →10 of the 38 studies in Neuronal Ceroid-Lipofuscinoses with a site in United States are open now. Recruiting first, the newest first:
New York Stem Cell Foundation Research Institute · Sites in United States
Ages 30 days+
National Human Genome Research Institute (NHGRI) · Sites in United States
Ages 18–100 · Healthy volunteers
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) · Sites in United States
Ages 1 wk–100 yrs
Joanne Kurtzberg, MD · Sites in United States
Ages 1 wk–22 yrs
Foundation Fighting Blindness · Sites in United States
Healthy volunteers
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 11 Neuronal Ceroid-Lipofuscinoses studies recruiting now, 7 (64%) set no upper age limit and 2 (18%) stop at 75 or younger; 10 take children under 18; 2 take healthy volunteers.
This study: all sexes · no healthy volunteers.
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38 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 16 · Phase 2 12 · Phase 3 1 · Phase 4 1
First in 1995; this study’s year is marked. 3 started in 2021.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 10 is below the median of 57 across 23 observational studies indexed under Neuronal Ceroid-Lipofuscinoses.
Median enrollment 11 across the 20 interventional studies, 65 across the 18 observational. Of the 19 that completed or were terminated, 8 (42%) have results posted.
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9 of 38 have an industry lead sponsor. The 10 most frequent:
Emily de los Reyes is the lead sponsor of 5 studies on the registry; 1 is open to participants now.
Emily de los Reyes has 4 studies filed under Neuronal Ceroid-Lipofuscinoses — fewer than ten, so there is no comparison to draw.
5 of the studies in Neuronal Ceroid-Lipofuscinoses with a site in United States that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT04098211Children's Hospital of Orange CountyLongitudinal Assessment of Atypical Tripeptidyl Peptidase 1 Enzyme Deficiency Patients | 2026-12 |
| NCT04273243Emily de los ReyesLong-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer | 2026-12 |
| NCT03285425Emily de los ReyesNatural History of Neuronal Ceroid Lipofuscinosis, Batten's CLN6 Diseae | 2026-12 |
| NCT07582484The Charlotte and Gwenyth Gray FoundationGene Therapy Trial for CLN6 Batten Disease | 2027-08 |
| NCT02254863Joanne Kurtzberg, MDUCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells | 2027-10 |
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Among the 38 studies in Neuronal Ceroid-Lipofuscinoses with a site in United States, from their registry updates:
Nothing recorded among them since 2026-09-25.
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This is a multicenter, international, study to assess the natural history data from people with Batten disease by collecting both retrospective and prospective information about the motor, behavioral and functional capabilities of patients. The study initially has 2 cohorts. Cohort 1 (n ≈ 75) includes subjects with CLN6 Batten disease. Cohort 2 (n ≈ 120) includes subjects with juvenile CLN3 Batten disease. Additional cohorts for other Batten disease subtypes may be added in the future.
Subjects diagnosed with CLN3 or CLN6 and their parents/legal guardians are eligible to take part in this study.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Assess the change over time in rating as determined using the Unified Batten Disease Rating Scale (UBDRS).
The UBDRS is a clinical ratings instrument used specifically to assess motor, seizure, behavioral and functional capabilities.
Time frame: 10 years
Assess the change over time in rating as determined using the Hamburg Scale.
The Hamburg scale is an established tool to capture the rate of decline or regression.
Time frame: 10 years
Assess the change over time in cognitive function using Mullen Scales of Early Learning (for children up to 60 months old).
Time frame: 10 years
Assess the change over time in cognitive function using WPPSI-IV (for children up to 7 years 7 months old).
Time frame: 10 years
Assess the change over time in cognitive function using WISC-V (for patients older than 6 years old).
Time frame: 10 years
Characterize the age of onset of disease, including the timing of both the loss of capacities and the emergence of disease-related signs and symptoms
Time frame: 10 years
Ran from Apr 2021 to primary completion in Mar 2022, 10 months.
How these figures are worked out, and which studies are left out of them.
This study is terminated, as verified in Jul 2025. You cannot join it, but the record below documents what was studied.
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Neuronal Ceroid-Lipofuscinoses →
Emily de los Reyes