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

StemCells, Inc.

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Conditions

Neuronal Ceroid Lipofuscinosis

Interventions

HuCNS-SCBIOLOGICAL

Surgery to implant human CNS stem cells

Eligibility

Sex: ALLMin age: 6 MonthsMax age: 6 Years

Medical Language ↔ Plain English

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

Locations (1)

Oregon Health and Science University

Portland, Oregon, United States

Outcomes

Primary Outcomes

number of participants with adverse events.

Safety will be measured by number of adverse events and changes in neurological status.

Time frame: one year following transplantation

Secondary Outcomes

Number of subjects with improvement in neurological, neuropsychological development, MRI and MRS

Time frame: one year following transplantation

Data from ClinicalTrials.gov

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