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

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

Status

Withdrawn

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01238315

Enrollment

Registered

2010-11-10

Start date

2010-11-30

Completion date

2011-04-30

Last updated

2015-01-15

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Neuronal Ceroid Lipofuscinosis

Keywords

Batten's disease, INCL, LINCL, Neuronal Ceroid Lipofuscinosis, NCL

Brief 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.

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.

Interventions

BIOLOGICALHuCNS-SC

Surgery to implant human CNS stem cells

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

6 Months to 6 Years

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

Design outcomes

Primary

Measure	Time frame	Description
number of participants with adverse events.	one year following transplantation	Safety will be measured by number of adverse events and changes in neurological status.

Secondary

Measure	Time frame
Number of subjects with improvement in neurological, neuropsychological development, MRI and MRS	one year following transplantation

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026