CLN6Phase 1/2NCT07582484
Gene Therapy Trial for CLN6 Batten Disease
A Phase 1/2 study testing scAAV9.CB.CLN6 (dose: 1.5E14 vector genomes) in people with CLN6 and Batten Disease, run by The Charlotte and Gwenyth Gray Foundation at 1 site in United States.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 4 months and older
- Conditions the study is for
- CLN6, Batten Disease, Batten's Disease
- What is being tested?
- scAAV9.CB.CLN6 (dose: 1.5E14 vector genomes)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2026to Aug 2028
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 months and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Diagnosis of CLN6
- At least 4 months old
Who cannot join
- Presence of another inherited neurologic disease
- Prior stem cell transplantation
- Prior gene transfer, gene editing, or viral vector therapy
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site
University of California, San Diego - Rady Children's
La Jolla, California, 92093
Olivia Kim-McManus, MD, 858-576-1700 ext. x224550, okimmcmanus@health.ucsd.edu
About this study
Official title: Phase 1/2b Gene Transfer Clinical Trial for Variant Late Infantile Neuronal Ceroid Lipofuscinosis (CLN6 Batten Disease), Delivering the CLN6 Gene by Self-Complementary AAV9
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
- Conditions
- CLN6, Batten Disease, Batten's Disease, Neuronal Ceroid Lipofuscinosis CLN6, Neuronal Ceroid Lipofuscinosis
- Being tested
- scAAV9.CB.CLN6 (dose: 1.5E14 vector genomes)
- Keywords
- CLN6, CLN6 Batten, Batten, Batten's, Neuronal Ceroid Lipofuscinosis
- Medical subject headings
- Neuronal Ceroid-Lipofuscinoses
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
scAAV9.CB.CLN6 (dose: 1.5E14 vector genomes)Drug
self-complementary adeno-associated viral vector, serotype 9 (scAAV9), which contains the human CLN6 gene under the control of a hybrid CMV/CB promoter
| Study group | Role | What they receive |
|---|---|---|
| Treatment group | Experimental | Not described Receives: Drug: scAAV9.CB.CLN6 (dose: 1.5E14 vector genomes) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: From informed consent through Month 24
Secondary outcomes (2)
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.
Measured: 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.
Measured: Baseline, Day 28, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 May 2026
- Study startAug 2026 (estimated)
- Primary completionAug 2027 (estimated)
- Study completionAug 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for CLN6Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- The Charlotte and Gwenyth Gray Foundation
- Sponsor type
- University, hospital or other
- Collaborators
- University of California, San Diego
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07582484
- Sponsor's protocol ID
- CGGF-CLN6-001
- Status last verified
- May 2026
- First submitted
- 20 Apr 2026
- Last updated
- 14 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 14 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1/2b Gene Transfer Clinical Trial for Variant Late Infantile Neuronal Ceroid Lipofuscinosis (CLN6 Batten Disease), Delivering the CLN6 Gene by Self-Complementary AAV9
- ClinicalTrials.gov ID
- NCT07582484
- Acronym / sponsor ID
- CGGF-CLN6-001
- Conditions
- CLN6, Batten Disease, Batten's Disease, Neuronal Ceroid Lipofuscinosis CLN6, Neuronal Ceroid Lipofuscinosis
- Keywords
- CLN6, CLN6 Batten, Batten, Batten's, Neuronal Ceroid Lipofuscinosis
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- The Charlotte and Gwenyth Gray FoundationUniversity, hospital or other
- Collaborators
- University of California, San Diego
- Study start
- Aug 2026 (estimated)
- Primary completion
- Aug 2027 (estimated)
- Study completion
- Aug 2028 (estimated)
- First posted
- 13 May 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment group | Experimental | Drug: scAAV9.CB.CLN6 (dose: 1.5E14 vector genomes) |
Who may qualify
- Ages
- 4 months and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Diagnosis of CLN6
- At least 4 months old
Key exclusion criteria (3 listed)
- Presence of another inherited neurologic disease
- Prior stem cell transplantation
- Prior gene transfer, gene editing, or viral vector therapy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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 | Drug | Treatment group |
Primary outcome
Number of Participants With Treatment-Emergent Adverse Events as Assessed by CTCAE Version 5.0
Time frame: From informed consent through Month 24
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.
2 secondary outcomes
Change From Baseline in Hamburg Rating Scale Score
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
Time frame: Baseline, Day 28, Month 3, Month 6, Month 9, Month 12, Month 18, and Month 24
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 May 2026.
- Individual participant dataSharing planned: Undecided
Related trials for CLN6
Active, not recruitingObservational study
Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer
CLN6, Batten Disease
RecruitingObservational study
Clinical and Neuropsychological Investigations in Batten Disease
Neuronal Ceroid Lipofuscinosis, Neuronal Ceroid Lipofuscinosis CLN1
Active, not recruitingObservational study
Natural History of Neuronal Ceroid Lipofuscinosis, Batten's CLN6 Diseae
Batten Disease, CLN6
RecruitingObservational study
Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD Database
Neuronal Ceroid Lipofuscinosis, Batten Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing scAAV9.CB.CLN6 (dose: 1.5E14 vector genomes) in people with CLN6 and Batten Disease, run by The Charlotte and Gwenyth Gray Foundation at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 14 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Tiffany M Sepp (617-710-0770, tiffany@vanguardclinical.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Treatment-Emergent Adverse Events as Assessed by CTCAE Version 5.0 (time frame: From informed consent through Month 24). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 May 2026.