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CompletedNCT02054208Updated Aug 28, 2020

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

A Phase 1/2 interventional study of human umbilical cord blood derived mesenchymal stem cells and Normal saline 2mL in Alzheimer's Disease, sponsored by Medipost Co Ltd.. Completed at 1 site in Korea, Republic of. Open to participants aged 50 Years to 85 Years. Per ClinicalTrials.gov, last updated 2020-08-28.

Sponsored by Medipost Co Ltd. · Phase 1/2, Interventional, and Treatment

Phase
Phase 1/2
Study type
Interventional
Enrollment
46
Allocation
Randomized
Ages
50 Years to 85 Years
Sex
All
01

Study 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.

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.

02

Conditions studied

  • Alzheimer's Disease

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Keywords

  • human umbilical cord blood derived mesenchymal stem cells
  • stem cells
  • alzheimer's disease
  • cognitive ability
  • mesenchymal stem cells
  • cord blood
03

In context

Alzheimer Disease

3,678 studies on the registry are indexed under Alzheimer Disease; 872 are open to participants now.

This study's enrollment of 46 is below the median of 70 across 2,808 interventional studies indexed under Alzheimer Disease.

Browse Alzheimer Disease studies →

Lead sponsor

Medipost Co Ltd. is the lead sponsor of 16 studies on the registry; 1 is open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
50 Years to 85 Years
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

1 stage Inclusion Criteria:

  1. Korean male or female at 50 -85 years of age
  2. Diagnosis of Probable Alzheimer type according to NINCDS-ADRDA criteria at Visit 1 (Screening)
  3. Korea Mini-Mental State Examination (KMMSE) score of 18 - 26 at Visit 1 (Screening)
  4. Positive for Amyloid on PIB-PET or Florbetaben PET
  5. 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)

2 stage Inclusion Criteria:

  1. Korean male or female at 50 -85 years of age
  2. 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)
  3. Korea Mini-Mental State Examination (KMMSE) score of over 18 at Visit 1 (Screening)
  4. Positive for Amyloid on Florbetaben PET
  5. A subject with neurodegeneration (mild atrophy of the brain) as confirmed by MRI
  6. 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)

Exclusion Criteria:

  1. Concurrent mental disorder (such as schizophrenia, depression, bi-polar diseases or others) aside from dementia
  2. 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
  3. 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
  4. History of stroke within 3 months prior to study enrollment
  5. Severe liver disorder (equivalent to double the normal values of ALT and AST) at Visit 1
  6. Severe kidney disorder (serum creatinine ≥1.5mg/dL) at Visit 1
  7. Pregnant or lactating females

  8. 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
  9. Suspected active lung disease based on chest X-ray at Visit 1
  10. 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)
  11. History of screening failure for the clinical trial of NEUROSTEM® in the past 6 months
  12. Participation in another clinical trial in the past 3 months prior to the beginning (Week 0) of this clinical trial
  13. 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)
  14. Diagnosis of cancer (of any body system, including brain tumor)
  15. Substance/alcohol abuse
  16. Contraindicated for any of the tests performed during the clinical trial period (for example, MRI, CT, PET)
  17. A subject in whom Ommaya reservoir insertion is considered difficult
  18. Whom the principal investigator considers inappropriate for participation in the study due to any reasons other than those listed above
05

Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
46 participants (actual)

Study arms

  • Experimental
    NEUROSTEM (hUCB-MSCs)- low dose

    human 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

    Biological: human umbilical cord blood derived mesenchymal stem cells

  • Experimental
    NEUROSTEM (hUCB-MSCs) - high dose

    human 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

    Biological: human umbilical cord blood derived mesenchymal stem cells

  • Placebo comparator
    Placebo

    normal saline 2mL, doses separated by 4 weeks for a total of 3 doses

    Other: Normal saline 2mL

Interventions

  • Biologicalhuman 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 High dose: 3 x 10\^7cells/2mL 3 repeated intraventricular administrations via an Ommaya Reservoir at 4 week intervals

    Also known as: NEUROSTEM

  • OtherNormal saline 2mL

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

06

What researchers measure

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

    Time frame: 24 weeks after the first dose

Secondary outcomes

  1. Change from the baseline in ADAS-Cog

    Alzheimer's Disease assessment Scale-Cognitive Subscale

    Time frame: 24 weeks after the first dose

  2. Change from the baseline in S-IADL

    Seoul Instrumental Activities of Daily Living

    Time frame: 24 weeks after the first dose

  3. Change from the baseline in K-MMSE

    Mini Mental State Exmination Korean version

    Time frame: 24 weeks after the first dose

  4. Change from the baseline in CGA-NPI

    Caregiver-administered Neuropsychiatric Inventory

    Time frame: 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

    Time frame: 24 weeks after the first dose

  6. Change in CDR-SOB

    Clinical Dementia Rating-Sum of Box

    Time frame: 24 weeks after the first dose

  7. Change in Florbetaben-PET

    Florbetaben - Pittsburgh Compound B-positron emission tomography

    Time frame: 24 weeks after the first dose

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

    fluorodeoxyglucose positron emission tomography

    Time frame: 24 weeks after the first dose

  9. Change in CIBIC-plus

    The Clinician's Interview Based Impression of Change-plus

    Time frame: 24 weeks after the first dose

  10. Change from baseline in MRI (DTI mapping)

    MRI Analysis

    Time frame: 24 weeks after the first dose

  11. Change from the baseline in CSF biomarkers

    biomakrer analysis

    Time frame: 24 weeks after the first dose

07

Study locations

1 site
  • Samsung Medical Center
    Seoul, Korea, Republic of
08

References and documents

Publications

  • 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 ↗

Individual participant data

Plan to share: Undecided

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Aug 28, 2020, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT02054208
Lead sponsor
Medipost Co Ltd.
Responsible party
Sponsor
First posted
Feb 4, 2014
Start date
Mar 2014
Primary completion
Dec 2019
Completion
Dec 2019
Last update
Aug 28, 2020

Study contacts

Wonil Oh, MD, PhD
study director · Medipost Co Ltd.

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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This study is completed, as verified in Aug 2018. You cannot join it, but the record below documents what was studied.

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