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Alzheimer's DiseasePhase 1/2NCT02054208

Safety and Exploratory Efficacy Study of NEUROSTEM® Versus Placebo in Patients With Alzheimer's Disease

A Phase 1/2 study testing human umbilical cord blood derived mesenchymal stem cells and Normal saline 2mL in people with Alzheimer's Disease, run by Medipost Co Ltd. at 1 site in South Korea.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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… Read more about this study

Who is it for?
Ages 50 to 85 years
Conditions the study is for
Alzheimer's Disease
What is being tested?
human umbilical cord blood derived mesenchymal stem cells and Normal saline 2mL
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
46 participants
Where
1 siteSouth Korea
Study dates
Mar 2014to Dec 2019
Phase
Phase 1/2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 50 to 85 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

None listed.

Who cannot join

  • 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
Show all 21Show fewer
  • 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
  • Bleeding disorder (abnormal blood coagulation test result (i.e. platelet count of < 150,000/mm3, PT ≥ 1.5 INR, or aPTT ≥ 1.5 x control anti-coagulant or anti-platelet, without anticoagulant or anti-platelet therapy)
  • Diagnosis of cancer (of any body system, including brain tumor)
  • Substance/alcohol abuse
  • Contraindicated for any of the tests performed during the clinical trial period (for example, MRI, CT, PET)
  • A subject in whom Ommaya reservoir insertion is considered difficult
  • Whom the principal investigator considers inappropriate for participation in the study due to any reasons other than those listed above

Trial locations and contacts

1 site in 1 country.

South Korea1 site

  • Samsung Medical Center

    Seoul

Other trials in South Korea

About this study

Official title: 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

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.

Read the detailed description

The study is divided into the 2 stages: dose-escalation in stage 1 and randomized and multiple-dose cohort parallel design in stage 2.The target population for enrollment in this study is patients with mild to moderate Alzheimer's disease.

Being tested
human umbilical cord blood derived mesenchymal stem cells, Normal saline 2mL
Keywords
human umbilical cord blood derived mesenchymal stem cells, stem cells, alzheimer's disease, cognitive ability, mesenchymal stem cells, cord blood
Medical subject headings
Alzheimer Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • human umbilical cord blood derived mesenchymal stem cellsBiological

    Also called NEUROSTEM

    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

  • Normal saline 2mLOther

    Intraventricular administrations of 2mL Normal Saline at 4 week intervals via an Ommaya Reservoir, for a total of 3 administrations

Study groups in NCT02054208
Study groupRoleWhat they receive
NEUROSTEM (hUCB-MSCs)- low doseExperimentalhuman 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
Receives: Biological: human umbilical cord blood derived mesenchymal stem cells
NEUROSTEM (hUCB-MSCs) - high doseExperimentalhuman 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
Receives: Biological: human umbilical cord blood derived mesenchymal stem cells
PlaceboPlacebo comparatornormal saline 2mL, doses separated by 4 weeks for a total of 3 doses
Receives: Other: Normal saline 2mL

What is being measured

The primary outcome is the main question the study is designed to answer.

  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

    Measured: 24 weeks after the first dose

Secondary outcomes (11)

  1. Change from the baseline in ADAS-Cog

    Alzheimer's Disease assessment Scale-Cognitive Subscale

    Measured: 24 weeks after the first dose

  2. Change from the baseline in S-IADL

    Seoul Instrumental Activities of Daily Living

    Measured: 24 weeks after the first dose

  3. Change from the baseline in K-MMSE

    Mini Mental State Exmination Korean version

    Measured: 24 weeks after the first dose

  4. Change from the baseline in CGA-NPI

    Caregiver-administered Neuropsychiatric Inventory

    Measured: 24 weeks from the first dose

  5. ADAS-Cog Response Rate

    ADAS-cog response is defined as no worsening (no change or improvement on ADAS-cog score) of the ADAS-cog score at 24 weeks after the first administration compared to the baseline

    Measured: 24 weeks after the first dose

  6. Change in CDR-SOB

    Clinical Dementia Rating-Sum of Box

    Measured: 24 weeks after the first dose

  7. Change in Florbetaben-PET

    Florbetaben - Pittsburgh Compound B-positron emission tomography

    Measured: 24 weeks after the first dose

  8. Change in FDG-PET (CMRglc: regional cerebral metabolic rate for glucose)

    fluorodeoxyglucose positron emission tomography

    Measured: 24 weeks after the first dose

  9. Change in CIBIC-plus

    The Clinician's Interview Based Impression of Change-plus

    Measured: 24 weeks after the first dose

  10. Change from baseline in MRI (DTI mapping)

    MRI Analysis

    Measured: 24 weeks after the first dose

  11. Change from the baseline in CSF biomarkers

    biomakrer analysis

    Measured: 24 weeks after the first dose

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted4 Feb 2014 (estimated)
  2. Study startMar 2014
  3. Primary completionDec 2019
  4. Study completionDec 2019

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Model description
Stage 1: 9 subjects (3 subjects for low dose and 6 subjects for high dose) Stage 2: 36 subjects ( 24 subjects for high dose and 12 subjects for placebo) A total of 45 subjects to be enrolled
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
46 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02054208
Sponsor's protocol ID
MP-CR-010
Status last verified
Aug 2018
First submitted
29 Jan 2014
Last updated
28 Aug 2020
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Undecided

Publications

  • Myeong SH, Lee NK, Lee NH, Choi SJ, Son HJ, Chang JW, Kim HJ, Na DL. Intracerebroventricular human mesenchymal stem cells induce MMP9-driven transient inflammation in Alzheimer's disease. Stem Cell Res Ther. 2026 Mar 11;17(1):145. doi: 10.1186/s13287-026-04958-x.

    PubMed 41814444

  • 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.

    PubMed 41220869

  • Choi 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.

    PubMed 37468918

  • Kim 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.

    PubMed 34521461

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing human umbilical cord blood derived mesenchymal stem cells and Normal saline 2mL in people with Alzheimer's Disease, run by Medipost Co Ltd. at 1 site in South Korea.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 0 inclusion and 21 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 28 Aug 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of subjects with adverse events (time frame: 24 weeks after the first dose). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02054208, which also lists 4 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Aug 2020.

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