WithdrawnPhase 1

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

Stopped: Lack of timely patient accrual

United States0Started 2010-11

Plain-language summary

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.

Who can participate

Age range6 Months – 6 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

number of participants with adverse events.

Timeframe: one year following transplantation

Trial details

NCT IDNCT01238315

SponsorStemCells, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2011-04

View on ClinicalTrials.gov More Neuronal Ceroid Lipofuscinosis trials