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 range3 Years – 10 Years
SexALL
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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
✕. Abnormal laboratory values considered clinically significant
✕
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.