NCT01238315

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.

Trial Health

30
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Timeline
Completed

Started Nov 2010

Shorter than P25 for phase_1

Geographic Reach
1 country

1 active site

Status
withdrawn

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

November 1, 2010

Completed
4 days until next milestone

First Submitted

Initial submission to the registry

November 5, 2010

Completed
5 days until next milestone

First Posted

Study publicly available on registry

November 10, 2010

Completed
5 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2011

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2011

Completed
Last Updated

January 15, 2015

Status Verified

January 1, 2015

Enrollment Period

5 months

First QC Date

November 5, 2010

Last Update Submit

January 13, 2015

Conditions

Neuronal Ceroid Lipofuscinosis

Keywords

Batten's diseaseINCLLINCLNeuronal Ceroid LipofuscinosisNCL

Outcome Measures

Primary Outcomes (1)

  • number of participants with adverse events.

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

    one year following transplantation

Secondary Outcomes (1)

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

    one year following transplantation

Study Arms (1)

HuCNS-SC

OTHER
Biological: HuCNS-SC

Interventions

HuCNS-SCBIOLOGICAL

Surgery to implant human CNS stem cells

Also known as: human neural stem cells, stem cells, neural stem cells
HuCNS-SC

Eligibility Criteria

Age6 Months - 6 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Oregon Health and Science University

Portland, Oregon, 97239, United States

Location

Related Links

MeSH Terms

Conditions

Neuronal Ceroid-Lipofuscinoses

Condition Hierarchy (Ancestors)

Heredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesNervous System DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLipidosesLipid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsLipid Metabolism DisordersMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Nathan Selden, MD, PhD

    Oregon Health and Science University

    PRINCIPAL INVESTIGATOR
0

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 5, 2010

First Posted

November 10, 2010

Study Start

November 1, 2010

Primary Completion

April 1, 2011

Study Completion

April 1, 2011

Last Updated

January 15, 2015

Record last verified: 2015-01

Locations

US(1)