A Phase 1/2 interventional study of scAAV9.CB.CLN6 (dose: 1.5E14 vector genomes) in CLN6, Batten Disease and Batten's Disease, sponsored by The Charlotte and Gwenyth Gray Foundation. Not yet recruiting at 1 site in United States. Open to participants aged 4 Months and older. Per ClinicalTrials.gov, last updated 2026-05-14.
Sponsored by The Charlotte and Gwenyth Gray Foundation · Phase 1/2, Interventional, and Treatment
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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 9th largest by planned enrollment of the 20 interventional ones that give a count, is the only one that started in 2026, is one of 9 in phase 1/2, reaches primary completion later than 5 of the 17 other live ones, and was the 38th 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: 4 Months and older · 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. 1 started in 2026.
By drug class, each counting the studies that test any of its medicines:
This study's planned enrollment of 12 is close to the median of 13 across 22 interventional 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:
This is the only study on the registry with The Charlotte and Gwenyth Gray Foundation as lead sponsor.
The Charlotte and Gwenyth Gray Foundation has 1 study 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 |
Add all 5 to your calendar (.ics) → With an account.
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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The goal of this clinical trial is to learn if a gene therapy called scAAV9.CB.CLN6 can treat children with CLN6 Batten disease (variant late infantile neuronal ceroid lipofuscinosis). The main questions it aims to answer are if he gene therapy safe and well tolerated, and if the gene therapy help slow disease progression or improve symptoms.
Participants will:
Receive a single dose of the gene therapy through an injection into the fluid around the spinal cord (intrathecal administration) Have regular study visits over 2 years for safety checks and assessments of disease progression Be followed for an additional 3 years in a long-term follow-up study
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Exclusion Criteria:
Drug: scAAV9.CB.CLN6 (dose: 1.5E14 vector genomes)
self-complementary adeno-associated viral vector, serotype 9 (scAAV9), which contains the human CLN6 gene under the control of a hybrid CMV/CB promoter
Number of Participants With Treatment-Emergent Adverse Events as Assessed by CTCAE Version 5.0
Treatment-emergent adverse events, including serious adverse events, hospitalizations, deaths, clinically significant laboratory abnormalities, and other clinically significant safety findings will be recorded from informed consent through Month 24. Adverse events will be assessed for intensity using CTCAE version 5.0 and for relationship to scAAV9.CB.CLN6. The number and percentage of participants with treatment-emergent adverse events will be summarized.
Time frame: From informed consent through Month 24
Change From Baseline in Hamburg Rating Scale Score
The Hamburg Rating Scale will be used to assess disease status and function in participants with CLN6 disease. Change from baseline in Hamburg Rating Scale score will be summarized at scheduled post-baseline visits.
Time frame: Baseline, Day 28, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24
Change From Baseline in Weill-Cornell Late Infantile Neuronal Ceroid Lipofuscinosis Scale Score
The Weill-Cornell Late Infantile Neuronal Ceroid Lipofuscinosis Scale will be used to assess disease status and function in participants with CLN6 disease. Change from baseline in Weill-Cornell Scale score will be summarized at scheduled post-baseline visits.
Time frame: Baseline, Day 28, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24
Plan to share: Undecided
No publications or documents are linked to this record.
Start is estimated for Aug 2026; primary completion for Aug 2027, 12 months later.
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This study is not yet recruiting, as verified in May 2026. You cannot join it, but the record below documents what was studied.
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Neuronal Ceroid-Lipofuscinoses →