Safety and Efficacy Study of HuCNS-SC in Subjects With Neuronal Ceroid Lipofuscinosis

This study has been withdrawn prior to enrollment.
(Lack of timely patient accrual)
Information provided by (Responsible Party):
StemCells, Inc. Identifier:
First received: November 5, 2010
Last updated: January 13, 2015
Last verified: January 2015
The purpose of this Phase Ib study is to determine if "Human Central Nervous System Stem Cells"(HuCNS-SC) is safe to be transplanted in subjects with infantile and late infantile neuronal ceroid lipofuscinosis. The study will also measure post-transplantation disease progression.

Condition Intervention Phase
Neuronal Ceroid Lipofuscinosis
Biological: HuCNS-SC
Phase 1

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase Ib Study of the Safety and Preliminary Efficacy of Allogeneic Intracerebral Human Central Nervous System Stem Cell Transplantation in Subjects With Non-Refractory Infantile and Late Infantile Neuronal Ceroid Lipofuscinosis

Resource links provided by NLM:

Further study details as provided by StemCells, Inc.:

Primary Outcome Measures:
  • number of participants with adverse events. [ Time Frame: one year following transplantation ] [ Designated as safety issue: Yes ]
    Safety will be measured by number of adverse events and changes in neurological status.

Secondary Outcome Measures:
  • Number of subjects with improvement in neurological, neuropsychological development, MRI and MRS [ Time Frame: one year following transplantation ] [ Designated as safety issue: No ]

Enrollment: 0
Study Start Date: November 2010
Study Completion Date: April 2011
Primary Completion Date: April 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
HuCNS-SC Biological: HuCNS-SC
Surgery to implant human CNS stem cells
Other Names:
  • human neural stem cells
  • stem cells
  • neural stem cells

Detailed Description:
Participation in this study will involve screening assessments, surgery to implant HuCNS-SC, medication to suppress immune system and a series of follow-up assessments.

Ages Eligible for Study:   6 Months to 6 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Age 6 months to 6 years
  • Male or female
  • Clinical diagnosis of Infantile neuronal ceroid lipofuscinosis or late infantile neuronal ceroid lipofuscinosis
  • CLN1 or CLN2 mutation

Exclusion Criteria:

  • Previously received an organ, tissue or bone marrow transplantation
  • Previously participated in any gene or cell therapy study
  • Infection with hepatitis virus, cytomegalovirus, Epstein-Barr virus, or Human Immunodeficiency Virus (HIV)
  • Current or prior cancer
  • Bleeding disorder
  • Unable to have an MRI scan
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01238315

United States, Oregon
Oregon Health and Science University
Portland, Oregon, United States, 97239
Sponsors and Collaborators
StemCells, Inc.
Principal Investigator: Nathan Selden, MD, PhD Oregon Health and Science University
  More Information

Additional Information:
Responsible Party: StemCells, Inc. Identifier: NCT01238315     History of Changes
Other Study ID Numbers: CL-N03-NCL 
Study First Received: November 5, 2010
Last Updated: January 13, 2015
Health Authority: United States: Food and Drug Administration

Keywords provided by StemCells, Inc.:
Batten's disease
Neuronal Ceroid Lipofuscinosis

Additional relevant MeSH terms:
Neuronal Ceroid-Lipofuscinoses
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Lipid Metabolism Disorders
Lipid Metabolism, Inborn Errors
Metabolic Diseases
Metabolism, Inborn Errors
Nervous System Diseases
Neurodegenerative Diseases processed this record on May 25, 2016