Safety and Exploratory Efficacy Study of NEUROSTEM® Versus Placebo in Patients With Alzheimer's Disease
A Double-blind, Single-center, Phase 1/2a Clinical Trial to Evaluate the Safety and Exploratory Efficacy of Intraventricular Administrations of NEUROSTEM® Versus Placebo Via an Ommaya Reservoir in Patients With Alzheimer's Disease
1 other identifier
interventional
46
1 country
1
Brief Summary
This combined phase 1/2a clinical trial is to investigate the safety, dose limiting toxicity (DLT), and exploratory efficacy of three repeated intraventricular administrations of NEUROSTEM® (human umbilical cord blood-derived mesenchymal stem cells) versus placebo via an Ommaya reservoir at 4 week intervals in patients with Alzheimer's disease.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_1
Started Mar 2014
Longer than P75 for phase_1
1 active site
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
First Submitted
Initial submission to the registry
January 29, 2014
CompletedFirst Posted
Study publicly available on registry
February 4, 2014
CompletedStudy Start
First participant enrolled
March 1, 2014
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2019
CompletedStudy Completion
Last participant's last visit for all outcomes
December 1, 2019
CompletedAugust 28, 2020
August 1, 2018
5.8 years
January 29, 2014
August 26, 2020
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Number of subjects with adverse events
Number of subjects with adverse event, number of subjects with normal range of vital signs, mixed lymphocyte reaction result, and laboratory examination result
24 weeks after the first dose
Secondary Outcomes (11)
Change from the baseline in ADAS-Cog
24 weeks after the first dose
Change from the baseline in S-IADL
24 weeks after the first dose
Change from the baseline in K-MMSE
24 weeks after the first dose
Change from the baseline in CGA-NPI
24 weeks from the first dose
ADAS-Cog Response Rate
24 weeks after the first dose
- +6 more secondary outcomes
Study Arms (3)
NEUROSTEM (hUCB-MSCs)- low dose
EXPERIMENTALhuman umbilical cord blood derived mesenchymal stem cells Low dose: 1 x 10\^7cells/2mL 3 repeated intraventricular administrations via an Ommaya Reservoir at 4 week intervals
NEUROSTEM (hUCB-MSCs) - high dose
EXPERIMENTALhuman umbilical cord blood derived mesenchymal stem cells High dose: 3 x 10\^7 cells/2mL 3 repeated intraventricular administrations via an Ommaya Reservoir at 4 week intervals
Placebo
PLACEBO COMPARATORnormal saline 2mL, doses separated by 4 weeks for a total of 3 doses
Interventions
Low dose: 1 x 10\^7cells/2mL 3 repeated intraventricular administrations via an Ommaya Reservoir at 4 week intervals High dose: 3 x 10\^7cells/2mL 3 repeated intraventricular administrations via an Ommaya Reservoir at 4 week intervals
Intraventricular administrations of 2mL Normal Saline at 4 week intervals via an Ommaya Reservoir, for a total of 3 administrations
Eligibility Criteria
You may qualify if:
- Korean male or female at 50 -85 years of age
- Diagnosis of Probable Alzheimer type according to NINCDS-ADRDA criteria at Visit 1 (Screening)
- Korea Mini-Mental State Examination (KMMSE) score of 18 - 26 at Visit 1 (Screening)
- Positive for Amyloid on PIB-PET or Florbetaben PET
- A subject who is informed of the clinical trial and signs a consent form (if unable to sign, a consent from a legally acceptable representative is required)
- Korean male or female at 50 -85 years of age
- Diagnosis of Probable Alzheimer type or mild cognitive impairment due to Alzheimer's disease (stage A) according to NIA-AA criteria at Visit 1(Screening)
- Korea Mini-Mental State Examination (KMMSE) score of over 18 at Visit 1 (Screening)
- Positive for Amyloid on Florbetaben PET
- A subject with neurodegeneration (mild atrophy of the brain) as confirmed by MRI
- A subject who is informed of the clinical trial and signs a consent form (if unable to sign, a consent from a legally acceptable representative is required)
You may not qualify if:
- Concurrent mental disorder (such as schizophrenia, depression, bi-polar diseases or others) aside from dementia
- Concurrent dementia as a result of other neurodegenerative disorders (due to infectious disease of the central nervous system such as HIV, syphilis), head injury, Creutzfeld-Jacob disease, Pick's disease, Huntington's disease, or Parkinson's disease
- Diagnosis of severe white matter hyperintensitivity (WMH) according to CREDOS (Clinical REsearch Center for Dementia of South Korea), which is defined as ≥ 25mm of the deep white matter and ≥ 10mm of the periventricular capping/banding in lengths
- History of stroke within 3 months prior to study enrollment
- Severe liver disorder (equivalent to double the normal values of ALT and AST) at Visit 1
- Severe kidney disorder (serum creatinine ≥1.5mg/dL) at Visit 1
- Pregnant or lactating females
- Abnormal Laboratory findings at Visit 1
- Hemoglobin \< 9.5 g/dL for male and \<9.0 g/dL for female
- Total WBC Count \< 3000/mm3
- Total Bilirubin \>= 3 mg/dL
- Suspected active lung disease based on chest X-ray at Visit 1
- Woman of childbearing age who refuses to practice medically acceptable contraceptive method (post menopausal patient with no menstruation for at least 12 months is considered as infertile)
- History of screening failure for the clinical trial of NEUROSTEM® in the past 6 months
- Participation in another clinical trial in the past 3 months prior to the beginning (Week 0) of this clinical trial
- +6 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Medipost Co Ltd.lead
Study Sites (1)
Samsung Medical Center
Seoul, South Korea
Related Publications (3)
Lee NK, Jang H, Choi Y, Hwangbo S, Lee S, Lee JI, Kim YJ, Chin J, Chang JW, Seo SW, Son HJ, Choi SJ, Na DL, Kim HJ. Mesenchymal Stem Cells With Adjuvant Dexamethasone in Patients With Alzheimer's Disease: A Phase IIa Trial. Dement Neurocogn Disord. 2025 Oct;24(4):272-285. doi: 10.12779/dnd.2025.24.4.272. Epub 2025 Oct 24.
PMID: 41220869DERIVEDChoi Y, Shin S, Son HJ, Lee NH, Myeong SH, Lee C, Jang H, Choi SJ, Kim HJ, Na DL. Identification of potential biomarkers related to mesenchymal stem cell response in patients with Alzheimer's disease. Stem Cell Res Ther. 2023 Jul 19;14(1):178. doi: 10.1186/s13287-023-03410-8.
PMID: 37468918DERIVEDKim HJ, Cho KR, Jang H, Lee NK, Jung YH, Kim JP, Lee JI, Chang JW, Park S, Kim ST, Moon SW, Seo SW, Choi SJ, Na DL. Intracerebroventricular injection of human umbilical cord blood mesenchymal stem cells in patients with Alzheimer's disease dementia: a phase I clinical trial. Alzheimers Res Ther. 2021 Sep 14;13(1):154. doi: 10.1186/s13195-021-00897-2.
PMID: 34521461DERIVED
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Wonil Oh, MD, PhD
Medipost Co Ltd.
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
January 29, 2014
First Posted
February 4, 2014
Study Start
March 1, 2014
Primary Completion
December 1, 2019
Study Completion
December 1, 2019
Last Updated
August 28, 2020
Record last verified: 2018-08