Gene Therapy for Children With CLN3 Batten Disease

Running, not enrolling · Phase 1/Phase 2

Conditions studied: CLN3, Batten Disease

In brief

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.

Key facts

Study ID: NCT03770572
Run by: Neela Therapeutics
People needed: 7
Starts: 2018-11-13
Expected to finish: 2029-09-30
Last updated by the study team: 2026-06-10

Who can join

Age: 3 and older, up to 10. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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

You may not qualify if…

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
Presence of immunologic disease
Has received stem cell or bone marrow transplantation
Has received any form of organ transplant
History of or current chemotherapy, radiotherapy, or other immunosuppression therapy within the past 30 days (corticosteroid treatment may be permitted at the discretion of the investigator)
Current use of cannabinoids and any by-products
Contraindications for intrathecal administration of the product or lumbar puncture (for collection of CSF), such as bleeding disorders or other medical conditions (eg, spina bifida, meningitis, or clotting abnormalities)
Contraindications for MRI scans (eg, cardiac pacemaker, metal fragment or chip in the eye, aneurysm clip in the brain)
Poorly controlled seizures - intractable epilepsy
Episode of generalized motor status epilepticus within 4 weeks before the Gene Transfer visit
History of corneal or intraocular surgery
Severe infection (eg, upper respiratory tract infection, pneumonia, pyelonephritis, or meningitis) within 4 weeks before the Gene Transfer visit (Enrollment may be postponed.)
Has received any investigational medication within 30 days before the infusion of study drug
Has a medical condition or extenuating circumstance that, in the opinion of the investigator, might compromise the subject's ability to comply with the protocol required testing or procedures or compromise the subject's wellbeing, safety, or clinical interpretability
Pregnancy at screening or Day 0. Any female subject judged by the investigator to be of childbearing potential will be tested for pregnancy.
Family does not want to disclose subject's study participation with primary care physician and other medical providers

Where it is running

Nationwide Children's Hospital — Columbus, Ohio, United States

Full record on ClinicalTrials.gov

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