Gene Therapy for Children With CLN3 Batten Disease (NCT03770572) | Clinical Trial Compass
Active — Not RecruitingPhase 1/2
Gene Therapy for Children With CLN3 Batten Disease
United States7 participantsStarted 2018-11-13
Plain-language summary
This is a phase 1/2, open-label, single dose, dose-escalation clinical trial to evaluate the safety and efficacy of CLN-301 (previous NCH Code: scAAV9.P546.CLN3) delivered intrathecally into the lumbar spinal cord region of subjects with CLN3 Batten disease.
Who can participate
Age range
3 Years – 10 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Diagnosis of CLN3 Batten disease determined by genotype available at screening by a College of American Pathologists/Clinical Laboratory Improvement Amendments (CAP/CLIA)-certified laboratory (or a non-US laboratory with an equivalent national accreditation/certification)
. Aged ≥ 3 to \< 11 years
. UBDRS physical impairment score of ≤ 7
. Able to walk independently at least 50 feet
Exclusion criteria
. Presence of another inherited neurologic or metabolic disease, eg, other forms of Batten disease (also known as neuronal ceroid lipofuscinosis; NCL) or seizures unrelated to CLN3 Batten disease (subjects with febrile seizures may be eligible at the discretion of the investigator)
. Presence of another neurological illness that may have caused cognitive decline (eg, trauma, meningitis, hemorrhage) before screening
. Active viral infection (includes HIV or serology positive for hepatitis B or C)
. Subjects with 2 consecutive aminotransaminase liver tests \> 3 times the upper limit of normal or \> 1.5 times the upper limit of normal if taking valproic acid at Visit 1 (screening/baseline)
. Subjects with anti-AAV9 antibody titers \> 1:400 as determined by ELISA (enzyme-linked immunosorbent assay) binding immunoassay
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Safety evaluation based on the development of dose-limiting toxicity (DLT).
Timeframe: 36 Months
2
Efficacy: Change in rating as determined using the Unified Batten Disease Rating Scale (UBDRS) rating scale.