Study of HuCNS-SC Cells in Patients With Infantile or Late Infantile Neuronal Ceroid Lipofuscinosis (NCL)

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00337636
• Date of registration: 13/06/2006
• Prospective Registration: No
• Primary sponsor: StemCells, Inc.
• Public title: Study of HuCNS-SC Cells in Patients With Infantile or Late Infantile Neuronal Ceroid Lipofuscinosis (NCL)
• Scientific title: A Phase I Study of the Safety and Preliminary Effectiveness of Human CNS Stem Cells (HuCNS-SC) in Patients With Neuronal Ceroid Lipofuscinosis Caused by Palmitoyl Protein Thioesterase 1 (PPT1) or Tripeptidyl Peptidase 1 (TPP-I) Deficiency
• Date of first enrolment: May 2006
• Target sample size: 6
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00337636
• Study type: Interventional
• Study design: Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Robert Steiner, MD
• Affiliation: Oregon Health and Science University

Key inclusion & exclusion criteria

Inclusion Criteria:

Patients MAY be eligible to participate in this research study if they:

- Are age 18 months to 12 years old

- Have a clinical diagnosis of infantile neuronal ceroid lipofuscinosis (INCL) or late
infantile neuronal ceroid lipofuscinosis (LINCL)

- Have a mutation of the CLN1 or CLN2 gene

- Have severe cognitive, communication, behavior and language impairment

Exclusion Criteria:

Patients may not be eligible to participate in this research study if they:

- Have cognitive, communication, behavior and language function less than that of a 1
year old

- Have previously received an organ, tissue or bone marrow transplantation

- Have previously participated in any gene or cell therapy study

- Have infection with hepatitis virus, Cytomegalovirus, Epstein Barr Virus, or Human
Immunodeficiency Virus (HIV)

- Have a current or prior cancer

- Have a bleeding disorder

- Are unable to have an MRI scan



Age minimum: 18 Months
Age maximum: 12 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Neuronal Ceroid Lipofuscinosis

Intervention(s)

Drug: Medication to suppress the immune system

Procedure: Surgery to implant human CNS stem cells (HuCNS-SC)

Primary Outcome(s)

Safety [Time Frame: one year post transpant]

Secondary Outcome(s)

Preliminary efficacy [Time Frame: one year post transplant]

Secondary ID(s)

CL-N001-05

Source(s) of Monetary Support

Please refer to primary and secondary sponsors