CLN3 / Batten Disease (neuronal ceroid lipofuscinoses) - Healing Genes

CLN3 / Batten Disease (neuronal ceroid lipofuscinoses)

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Gene Transfer Study of AAV9-CLN3 for Treatment NCL Type 3

Phase I/IIa Gene Transfer Clinical Trial for Juvenile Neuronal Ceroid Lipofuscinosis, Delivering the CLN3 Gene by Self-Complementary AAV9

Phase 1 / 2


Nationwide Children’s Hospital researchers in Columbus, OH, seek patients with Batten disease, also known as ceroid lipofuscinoses, neuronal, type 3 (CLN-3), for a study of an investigatory therapy. This gene therapy will attempt to transfer the functional CLN3 gene to the patient within a viral package. The viral package is delivered in a single intrathecal (spinal cord) injection, and the patients will be followed for 3 years before a long-term follow up period of 5 years.


  • Be 3 to 10 years of age
  • Have CLN3 diagnosis, confirmed by the presence of a mutation in the CLN3 gene by DNA test
  • Meet physical impairment criteria


  1. Screening to confirm acceptance to the study.
  2. A single intrathecal dose of the investigative drug will be delivered intrathecally at 1 of 2 possible dosage levels.
  3. Follow up to assess motor, seizure, behavioral and functional capabilities for 3 years, then enter a long-term follow up period of 5 years.

The trial will take place at the Nationwide Children’s Hospital in Columbus, OH. Map.
Lisa Moffitt, RN  |  614-722-2650  |  [email protected]
Nationwide Children’s Hospital
Amicus Therapeutics
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