Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) … (NCT02725580) | Clinical Trial Compass
CompletedPhase 1/2
Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) Disease
United States13 participantsStarted 2016-05
Plain-language summary
This is a phase 1/2, open-label, single dose study to evaluate the safety and efficacy of AT-GTX-501 delivered intrathecally into the lumbar spinal cord region of participants with mild to moderate variant late infantile neuronal ceroid lipofuscinosis associated with mutation(s) in the CLN6 gene (vLINCL6 disease).
Who can participate
Age range1 Year
SexALL
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Inclusion criteria
✓. Diagnosis of vLINCL6 disease determined by genotype available at screening
✓. A score of ≥ 3 on the quantitative clinical assessment of the Hamburg motor-language aggregate scale at screening
✓. Aged ≥ 1 year
✓. Ambulatory or able to walk with assistance
Exclusion criteria
✕. Presence of another inherited neurologic disease, for example, other forms of Batten disease (also known as NCL) or seizures unrelated to vLINCL6 disease (participants with febrile seizures may be eligible at discretion of the investigator.)
✕. Presence of another neurological illness that may have caused cognitive decline (for example, trauma, meningitis, hemorrhage) before screening
✕. Active viral infection (includes human immunodeficiency virus or serology positive for hepatitis B or C)
✕. Has received stem cell or bone marrow transplantation for vLINCL6 disease
✕. Contraindications for intrathecal administration of the product or lumbar puncture, such as bleeding disorders or other medical conditions (for example, spina bifida, meningitis, or clotting abnormalities)
✕. Contraindications for magnetic resonance imaging scans (for example, cardiac pacemaker, metal fragment or chip in the eye, aneurysm clip in the brain)
✕. Episode of generalized motor status epilepticus within 4 weeks before the gene transfer visit (Visit 2)
What they're measuring
1
Incidence And Severity Of Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
✕. Severe infection (for example, pneumonia, pyelonephritis, or meningitis) within 4 weeks before the gene transfer visit (Visit 2) (Enrollment may be postponed.)