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

Not Applicable

• Conditions: Alzheimer's Disease
• Interventions: Biological: human umbilical cord blood derived mesenchymal stem cells

• Registration Number: NCT04954534
• Lead Sponsor: Samsung Medical Center

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-06-01
• Status Verified: 2021-07
• Study First Submitted QC: 2021-07-07
• Study First Posted: 2021-07-08
• Last Update Submitted: 2021-07-08
• Study Start: 2021-07-12
• Last Update Posted: 2021-07-14
• Primary Completion: 2022-01-31
• Study Completion: 2022-06-30

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 9

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
NEUROSTEM (hUCB-MSCs) - high dose	human umbilical cord blood derived mesenchymal stem cells	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

Primary Outcome Measures

Name	Time	Method
Change from the baseline in ADAS-Cog	4weeks,8weeks,12weeks,24 weeks after the first dose	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.

Secondary Outcome Measures

Name	Time	Method
Change in CDR-SOB	24 weeks after the first dose	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.
Change from the baseline in CGA-NPI	24 weeks after the first dose	Caregiver-administered Neuropsychiatric Inventory, Measure abnormal behavior. The score range is 0-144. A higher score means severe abnormal behavior.
Change from the baseline in SIB	24 weeks after the first dose	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)
Change in CIBIC-plus	24 weeks after the first dose	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).
Change from the baseline in K-MMSE(korean version)	24 weeks after the first dose	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
ADAS-Cog Response Rate	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	Alzheimer's Disease assessment Scale-Cognitive Subscale
Change from the baseline in CSF biomarkers	24 weeks after the first dose	biomakrers analysis (Amyloid beta 42, Phosphorylated tau, Total tau, RBC, WBC, Protein, Glucose)

Trial Locations

Locations (1)

Samsung Medical Center; 🇰🇷 Seoul, Korea, Republic of