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

StemCells, Inc

Status and phase: Withdrawn; Phase 1

Conditions: Neuronal Ceroid Lipofuscinosis

Treatments: Biological: HuCNS-SC

Study type: Interventional

Funder types: Industry

Identifiers

NCT01238315

CL-N03-NCL

Details and patient eligibility

About

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.

Full 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.

Sex: All

Ages: 6 months to 6 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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0 participants in 1 patient group

HuCNS-SC

Other group

Treatment:

Biological: HuCNS-SC