The Long-Term Safety and Efficacy Follow-Up Study of Subjects Who Completed the Phase I Clinical Trial of Neurostem®-AD
The purpose of the study is to determine the long-term safety and exploratory efficacy of NEUROSTEM®-AD, administered via an open brain surgery to subjects with dementia of the Alzheimer's type, who were eligible for and enrolled in the earlier part of the phase I. Aside from the subjects who completed the earlier part of the Phase I, 3 additional subjects with comparable demographics and disease characteristics as the treatment group will be enrolled into a control group, followed-up for 3 months, and compared for various disease progression indicators with the treatment group.
The hypothesis is that NEUROSTEM®-AD is safe and effective in the treatment of dementia of the Alzheimer's type.
Central Nervous System Diseases
Nervous System Diseases
Delirium, Dementia, Amnestic, Cognitive Disorders
|Study Design:||Observational Model: Case Control
Time Perspective: Prospective
|Official Title:||The Long-Term Safety and Efficacy Follow-up Study of Subjects Who Completed the Phase I Clinical Trial of Neurostem®-AD|
- Safety [ Time Frame: upto 24 months post-op ] [ Designated as safety issue: Yes ]Incidence rate ot adverse events (vital signs, physical examination, mixed lymphocyte reaction, and laboratory tests)
- Efficacy [ Time Frame: upto 24 months post-op ] [ Designated as safety issue: No ]
Primary Efficacy Variable:
ADAS-cog response rate, ADAS-cog response is defined as when ADAS-cog score at the end of the study is not worse than the Baseline score.
Secondary Efficacy Variables:
- Changes in Seoul Instrumental Activities of Daily Living (S-IADL)
- Changes in Mini Mental State Examination Korean verson (K-MMSE)
- Changes in Caregiver-administered Neuropsuchiatric Inventory
- Changes in Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-Cog)
- Changes in CMRglc: regional cerebral metabolic rate for glucose (FDG-PET)
|Study Start Date:||March 2012|
|Estimated Study Completion Date:||September 2013|
|Estimated Primary Completion Date:||September 2013 (Final data collection date for primary outcome measure)|
A single administration of human umbilical cord blood-derived mesenchymal stem cells through a brain surgery
DOSE A - 250,000 cells per entry site, 3 million cells per brain; DOSE B - 500,000 cells per entry site, 6 million cells per brain
NEUROSTEM®-AD was administered to eligible subjects in the early part of the Phase I clinical study. In this follow-up study, no intervention will be performed.
Other Name: human umbilical cord blood-derived mesenchymal stem cells (hUCB-MSCs)
A group of subjects with comparable demographics (age and gender) and disease characteristics [Clinical Dementia Rating Scale Sum of Boxes (CDR-SOB)] as the NEUROSTEM®-AD-treated group, but did not receive the treatment with NEUROSTEM®-AD and were continued on conventional therapy. Restrictions in the concurrent use of drug therapy are as follows:
Patients are, in principle, permitted to continue the drug therapy they were on prior to the enrollment, for the treatment of concurrent illnesses other than Dementia, such as hypertension, diabetes mellitus, or hyperlipidemia.
For drugs used in the treatment of dementia, behavior-modifying drugs can be added to the pharmacological regimen of a subject during the course of the study. However, adding a new cognitive enhancer, such as donepezil, memantine, galantamine, rivastigmine, is not permitted while dose adjustment is permitted given that the drug had been in use prior to the initiation of the study.
This is a long-term follow up study of the earlier part of the phase I, during which the safe and effective dose(safety) of NEUROSTEM®-AD was determined for implantation into the brains of subjects with Dementia of the Alzheimer's type. Subjects with Dementia of the Alzheimer's type, who signed the informed consent form and meet the eligibility criteria, were implanted with a single dose of NEUROSTEM®-AD, hUBC-MSCs, into the brain. The subjects were hospitalized for 5 to 10 days following the surgical implantation and were observed for acute adverse events: Gradient echo MRI within the the 24 hours post-op, vital signs, clinical laboratory tests, chest x-rays within Day 2. On Day 14, DLT was assessed, and the subjects were followed up on the safety and disease progression of dementia (of the Alzheimer's type) for 12 weeks post-implantation.
In this part of the study, the subjects described above will be followed-up for upto Month 24, and 3 additional subjects with comparable demographics and disease characteristics as the treatment group (refer to Inclusion/Exclusion Criteria) will be enrolled as a control group, followed up for 3 months and compared with the treatment group for various indicators of the disease progression.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01696591
|Contact: Duk-Lyul Na, MD, PhDfirstname.lastname@example.org|
|Korea, Republic of|
|Samsung Medical Center||Recruiting|
|Seoul, Korea, Republic of, 135-710|
|Contact: Duk L. Na, MD, PhD +82-2-3410-3594 email@example.com|
|Principal Investigator:||Duk L. Na, MD,PhD||Samsung Medical Center|