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

Neuronal Ceroid Lipofuscinosis Clinical Trial

Official 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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00337636
• Other study ID #: CL-N001-05
• Status: Completed
• Phase: Phase 1
• First received: June 13, 2006
• Last updated: January 13, 2015
• Start date: May 2006
• Est. completion date: September 2009

Study information

• Verified date: January 2015
• Source: StemCells, Inc.
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

Patients with infantile or late infantile NCL have either a reduced amount of, or are missing, the palmitoyl protein thioesterase 1 (PPT1) enzyme or the tripeptidyl peptidase 1 (TPP-I) enzyme. Human central nervous system stem cells (HuCNS-SC) are an investigational product derived from human brain cells. HuCNS-SC have been shown to survive and migrate within the brains of mice. When grown in the laboratory, HuCNS-SC have been shown to produce the PPT1 and TPP-I enzymes. In mice missing the PPT1 enzyme, HuCNS-SC have been shown to increase the amount of this enzyme in the brain, to reduce the amount of abnormal storage material in the brain, and to prevent the death of some neurons (a type of cell) in the brain.

Participation in this study will involve screening assessments, surgery to implant HuCNS-SC, medication to suppress the immune system, and a series of follow-up assessments. The length of time from the start of screening through to the last follow-up visit will be approximately 13 months, with frequent visits to the study center during this time. After completion of this study, patients will be monitored for an additional 4 years under a separate long term follow-up protocol.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 6
• Est. completion date: September 2009
• Est. primary completion date: February 2009
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Months to 12 Years

Eligibility

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

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Neuronal Ceroid Lipofuscinosis
• Neuronal Ceroid-Lipofuscinoses

Intervention

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

Drug:
• Medication to suppress the immune system
Immunosuppression for 12 months post transplant

Locations

Country	Name	City	State
United States	Oregon Health and Science University	Portland	Oregon

Sponsors (1)

Lead Sponsor	Collaborator
StemCells, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety		one year post transpant	Yes
Secondary	Preliminary efficacy		one year post transplant	No

External Links

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