Study of Human Central Nervous System (CNS) Stem Cells Transplantation in Pelizaeus-Merzbacher Disease (PMD) Subjects

StemCells, Inc

Status and phase: Completed; Phase 1

Conditions: Pelizaeus-Merzbacher Disease

Treatments: Biological: HuCNS-SC cells

Study type: Interventional

Funder types: Industry

Identifiers

NCT01005004

CL-N01-PMD

Details and patient eligibility

About

The purpose of this study is to determine the safety and preliminary effectiveness of human central nervous system stem cells (HuCNS-SC®) transplantation in patients with Connatal Pelizaeus-Merzbacher Disease (PMD).

Full description

Enrolled subjects will be transplanted with HuCNS-SC cells into the brain and will receive immunosuppression for nine months. The study observation period is for one year after transplant surgery. Thereafter, subjects will be enrolled in a long-term observational follow-up study for four years.

Enrollment: 4 patients

Sex: Male

Ages: 6 months to 5 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Confirmed clinical diagnosis of connatal PMD
• Molecular genetic confirmation of mutation in the proteolipid protein 1 (PLP1) gene
• MRI consistent with PMD as interpreted by a qualified neuroradiologist

Exclusion criteria

• Other significant congenital brain abnormality not related to PMD
• Previous participation in gene transfer or cell transplant trial
• Presence of neurological signs and symptoms not consistent with PMD
• Current or prior malignancy
• Prior organ, tissue or bone marrow transplant

Trial design

4 participants in 1 patient group

HuCNS-SC cells

Experimental group

Description:

Intracerebral implantation of HuCNS-SC via direct injection during surgery

Treatment:

Biological: HuCNS-SC cells