Back to resultsThe Safety and The Efficacy Evaluation of NEUROSTEM®-AD in Patients With Alzheimer's Disease
Primary Purpose
Dementia of the Alzheimer's Type
Status
Completed
Phase
Phase 1
Locations
Korea, Republic of
Study Type
Interventional
Intervention
Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells
Sponsored by
About this trial
This is an interventional treatment trial for Dementia of the Alzheimer's Type focused on measuring Alzheimer, Mesenchymal Stem Cells, Umbilical Cord Blood
Eligibility Criteria
Inclusion Criteria:
- Korean men and women who are age 50 or older
- Dementia as determined by DSM-IV criteria
- Probable alzheimer's disease as determined by NINCDS-ADRDA criteria
- K-MMSE score in the range of 10 to 24
- Positive result of PIB-PET imaging (SUV > 1.5,when comparing the result for the cerebellum with the result for the frontal lobe)
- Voluntarily participating subject who sign the consent form
Exclusion Criteria:
- Subject with psychological diseases (i.e. depression, schizophrenia, bipolar disorder, etc)
- Subject with dementia caused by other than Alzheimer's disease (i.e. infection of central nervous system, Creutzfeld-Jacob disease, severe head trauma, Pick's disease, Huntington's disease, and Parkinson's disease)
- Subject with vascular dementia as determined by the clinical criteria of DSM IV and the imaging criteria of Erkinjuntii
- Subject with severe white matter hyperintensities (WMH); Severe WMH is defined that length of the deep white matter is 25 mm or longer and length of the periventricular capping/banding is 10 mm or longer.
- Subject who have had stroke in 3 months.
- Subject with liver disease (two times higher than normal range of ALT/AST)
- Subject with severe kidney failure (1.5mg/dL of serum creatinine or more)
- Pregnant women or lactating women
- Hemoglobin < 9.5g/dL for men, < 9.0 g/dL for women; Total WBC count < 3000/mm3; Total bilirubin ≥ 3 mg/dL
- Subject who is suspect to have active lung diseases, based on check X-ray result from Visit 1
- Women of childbearing age who reject to practice contraception
- Subject who have been excluded in the subject selection process for this study before
- A platelet count < 150,000/mm3; PT ≥ 1.5; INR or aPTT ≥ 1.5 X control
- Subject with cancer
- History of alcohol or drug abuse
- Subject who cannot undergo MRI, CT, or PET screening
- Subject who cannot undergo anesthesia or stereotactic brain injection
- Subject who is determined inappropriate by the investigators
Sites / Locations
- Samsung Medical Center
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
NEUROSTEM®-AD
Arm Description
Outcomes
Primary Outcome Measures
Number of participants with Adverse event
Number of participants with adverse event, number of participants with normal range of vital signs, mixed lymphocyte reaction, and laboratory examination
Secondary Outcome Measures
Changes from the baseline in ADAS-cog at 12 weeks post-dose
Changes from the baseline in ADAS-cog, S-IADL, K-MMSE, CGA-NPI, ADAS-Cog, serum transthyretin, amyloid beta and tau in cerebrospinal fluid, PIB-PET and FDG-PET at 12 weeks post-dose.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01297218
Brief Title
The Safety and The Efficacy Evaluation of NEUROSTEM®-AD in Patients With Alzheimer's Disease
Official Title
Open-Label, Single-Center, Phase 1 Clinical Trial to Evaluate the Safety and the Efficacy of NEUROTSTEM®-AD in Patients With Dementia of the Alzheimer's Type
Study Type
Interventional
2. Study Status
Record Verification Date
April 2012
Overall Recruitment Status
Completed
Study Start Date
February 2011 (undefined)
Primary Completion Date
September 2011 (Actual)
Study Completion Date
December 2011 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Medipost Co Ltd.
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The primary purpose of this study is to evaluate the safety and the tolerability of NEUROSTEM®-AD (Human Umbilical Cord Blood Derived Mesenchymal Stem Cells) and to assess the maximum tolerated dose (MTD). This study is also to investigate the efficacy of this study drug in patients with dementia of Alzheimer's type.
Detailed Description
Most of the treatments for Alzheimer disease are chemical drug that is designed to temporarily increase acetylcholine, based on the cholinergic hypothesis. These drugs can improve the symptoms but is not able to inhibit the disease progression. New drugs from the disease have been developed but they have not been successful yet.
Mesenchymal stem cells (MSC) are capable of differentiating into various tissues. Due to the characteristics of the cells it has been widely investigated in tissue regeneration. In addition, the paracrine effect of MSC in microenvironment has been recently reported. MSC has been developed as an immunomodulation cell therapy product because it has been known that it does not cause immunological rejection in allo- and xeno-transplantation. Clinical studies showed that umbilical cord blood-derived MSC is immunologically stable and not toxic.
This study is to evaluate the safety and the tolerability of NEUROSTEM®-AD (Human Umbilical Cord Blood Derived Mesenchymal Stem Cells) and to assess the maximum tolerated dose (MTD). This study is also to investigate the efficacy of this study drug in patients with dementia of Alzheimer's type.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Dementia of the Alzheimer's Type
Keywords
Alzheimer, Mesenchymal Stem Cells, Umbilical Cord Blood
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
9 (Actual)
8. Arms, Groups, and Interventions
Arm Title
NEUROSTEM®-AD
Arm Type
Experimental
Intervention Type
Biological
Intervention Name(s)
Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells
Other Intervention Name(s)
NEUROSTEM®-AD
Intervention Description
DOSE A - 250,000 cells per 5 uL per 1 entry site, 3 million cells per brain DOSE B - 500,000 cells per 5 uL per 1 entry site, 6 million cells per brain
Primary Outcome Measure Information:
Title
Number of participants with Adverse event
Description
Number of participants with adverse event, number of participants with normal range of vital signs, mixed lymphocyte reaction, and laboratory examination
Time Frame
12 weeks from post-administration
Secondary Outcome Measure Information:
Title
Changes from the baseline in ADAS-cog at 12 weeks post-dose
Description
Changes from the baseline in ADAS-cog, S-IADL, K-MMSE, CGA-NPI, ADAS-Cog, serum transthyretin, amyloid beta and tau in cerebrospinal fluid, PIB-PET and FDG-PET at 12 weeks post-dose.
Time Frame
12 weeks from post-administration
10. Eligibility
Sex
All
Minimum Age & Unit of Time
50 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Korean men and women who are age 50 or older
Dementia as determined by DSM-IV criteria
Probable alzheimer's disease as determined by NINCDS-ADRDA criteria
K-MMSE score in the range of 10 to 24
Positive result of PIB-PET imaging (SUV > 1.5,when comparing the result for the cerebellum with the result for the frontal lobe)
Voluntarily participating subject who sign the consent form
Exclusion Criteria:
Subject with psychological diseases (i.e. depression, schizophrenia, bipolar disorder, etc)
Subject with dementia caused by other than Alzheimer's disease (i.e. infection of central nervous system, Creutzfeld-Jacob disease, severe head trauma, Pick's disease, Huntington's disease, and Parkinson's disease)
Subject with vascular dementia as determined by the clinical criteria of DSM IV and the imaging criteria of Erkinjuntii
Subject with severe white matter hyperintensities (WMH); Severe WMH is defined that length of the deep white matter is 25 mm or longer and length of the periventricular capping/banding is 10 mm or longer.
Subject who have had stroke in 3 months.
Subject with liver disease (two times higher than normal range of ALT/AST)
Subject with severe kidney failure (1.5mg/dL of serum creatinine or more)
Pregnant women or lactating women
Hemoglobin < 9.5g/dL for men, < 9.0 g/dL for women; Total WBC count < 3000/mm3; Total bilirubin ≥ 3 mg/dL
Subject who is suspect to have active lung diseases, based on check X-ray result from Visit 1
Women of childbearing age who reject to practice contraception
Subject who have been excluded in the subject selection process for this study before
A platelet count < 150,000/mm3; PT ≥ 1.5; INR or aPTT ≥ 1.5 X control
Subject with cancer
History of alcohol or drug abuse
Subject who cannot undergo MRI, CT, or PET screening
Subject who cannot undergo anesthesia or stereotactic brain injection
Subject who is determined inappropriate by the investigators
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Duk L. Na, M.D.
Organizational Affiliation
Samsung Medical Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
Samsung Medical Center
City
Seoul
Country
Korea, Republic of
12. IPD Sharing Statement
Citations:
PubMed Identifier
29854930
Citation
Kim HJ, Seo SW, Chang JW, Lee JI, Kim CH, Chin J, Choi SJ, Kwon H, Yun HJ, Lee JM, Kim ST, Choe YS, Lee KH, Na DL. Stereotactic brain injection of human umbilical cord blood mesenchymal stem cells in patients with Alzheimer's disease dementia: A phase 1 clinical trial. Alzheimers Dement (N Y). 2015 Jul 26;1(2):95-102. doi: 10.1016/j.trci.2015.06.007. eCollection 2015 Sep.
Results Reference
derived
Learn more about this trial
The Safety and The Efficacy Evaluation of NEUROSTEM®-AD in Patients With Alzheimer's Disease
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