Exploratory Efficacy Study of NEUROSTEM® in Subjects Who Control Group of NEUROSTEM®

Exploratory Efficacy Study of NEUROSTEM® in Subjects Who Control Group of NEUROSTEM® Phase-I/IIa Clinical Trial

This study is to evaluate of Exploratory Efficacy of NEUROSTEM® in Subjects who control group of NEUROSTEM® Phase-I/IIa Clinical Trial

Study Overview

• Status: Not yet recruiting
• Conditions: Alzheimer's Disease
• Intervention / Treatment: Biological: human umbilical cord blood derived mesenchymal stem cells

• Study Type: Interventional
• Enrollment (Anticipated): 9
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Heejin kim, MD; Phone Number: +82-2-3410-1947; Email: evekhj@gmail.com

Study Locations

• Korea, Republic of
  • Seoul, Korea, Republic of, 135-710
    • Samsung Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 50 years to 85 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Subjects who have been treated with placebo in phase 1/2a clinical trial of NEUROSTEM®
2. Subjects who voluntarily decided to participate and signed the consent form after receiving explanations on the clinical trial (in case it is difficult for the participant to sign, the consent of the legal representative)

Exclusion Criteria:

1. History of stroke within 3 months prior to study enrollment
2. Severe liver disorder (equivalent to double the normal values of ALT and AST) at Visit 1
3. Severe kidney disorder (serum creatinine ≥1.5mg/dL) at Visit 1

4. 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
5. Suspected active lung disease based on chest X-ray at Visit 1
6. Bleeding disorder (abnormal blood coagulation test result (i.e. platelet count of < 137,000/mm3, PT ≥ 1.5 INR, or aPTT ≥ 1.5 x control anti-coagulant or anti-platelet, without anticoagulant or anti-platelet therapy)
7. Diagnosis of cancer (of any body system, including brain tumor)
8. Contraindicated for any of the tests performed during the clinical trial period (for example, MRI, CT, PET)
9. Whom the principal investigator considers inappropriate for participation in the study due to any reasons other than those listed above

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
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; High dose: 3 x 10^7cells/2mL 3 repeated intraventricular administrations via an Ommaya Reservoir at 4-week intervals; Other Names: NEUROSTEM

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from the baseline in ADAS-Cog	The ADAS-Cog is a psychometric instrument that evaluates memory, attention, reasoning, language, orientation, and praxis using an 11-point AD Assessment Scale. It has a minimum score of 0 and a maximum severity score of 70, and a higher score indicates more impairment.	4weeks,8weeks,12weeks,24 weeks after the first dose

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in CDR-SOB	The CDR-SOB(Clinical Dementia Rating, Sum of Boxes) is a global rating of dementia severity based on the clinician's interpretation of the history and examination. The range of this instrument is 0 to 18 with higher numbers indicating greater impairment.	24 weeks after the first dose
Change from the baseline in K-MMSE(korean version)	The MMSE(Mini-Mental State Examination) is a brief, practical test for cognitive dysfunction. The test consists of five sections (orientation, registration, attention-calculation, recall, and language) and results in a total possible score from 0 to 30, with higher scores indicating better function	24 weeks after the first dose
Change from the baseline in CGA-NPI	Caregiver-administered Neuropsychiatric Inventory, Measure abnormal behavior. The score range is 0-144. A higher score means severe abnormal behavior.	24 weeks after the first dose
Change from the baseline in SIB	The Severe Impairment Battery (SIB) is an assessment of cognitive dysfunction across nine domains such as memory, language, and orientation. The score ranges from 0 (worst) to 100 (best)	24 weeks after the first dose
ADAS-Cog Response Rate	Alzheimer's Disease assessment Scale-Cognitive Subscale	The 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
Change in CIBIC-plus	The Clinician's Interview-Based Impression of Change-plus(CIBIC-plus) is a rating scale derived from an interview with the patient and caregiver with an independent rater designed to measure several domains of patient function, such as mental/cognitive state, behavior, and activities of daily living. The scores range from 1 (marked improvement) to 7 (marked worsening).	24 weeks after the first dose
Change from the baseline in CSF biomarkers	biomakrers analysis (Amyloid beta 42, Phosphorylated tau, Total tau, RBC, WBC, Protein, Glucose)	24 weeks after the first dose

Collaborators and Investigators

• Sponsor: Samsung Medical Center
• Collaborators: Medipost Co Ltd.
• Investigators: Principal Investigator: Duk L. Na, MD, PhD, Samsung Medical Center

Study record dates

Study Major Dates

• Study Start (Anticipated): July 12, 2021
• Primary Completion (Anticipated): January 31, 2022
• Study Completion (Anticipated): June 30, 2022

Study Registration Dates

• First Submitted: June 1, 2021
• First Submitted That Met QC Criteria: July 7, 2021
• First Posted (Actual): July 8, 2021

Study Record Updates

• Last Update Posted (Actual): July 14, 2021
• Last Update Submitted That Met QC Criteria: July 8, 2021
• Last Verified: July 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Mental Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurocognitive Disorders; Neurodegenerative Diseases; Dementia; Tauopathies; Alzheimer Disease
• Other Study ID Numbers: SARC-CR-01

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No