Follow-up Study of Safety and Efficacy in Subjects Who Completed NEUROSTEM® Phase-I/IIa Clinical Trial.

Phase 1

Completed

• Conditions: Alzheimer's Disease
• Interventions: Biological: human umbilical cord blood derived mesenchymal stem cells; Other: Normal saline 2mL

• Registration Number: NCT03172117
• Lead Sponsor: Medipost Co Ltd.

Brief Summary

A long-term follow-up study to obtain safety and efficacy data in subjects who completed phase 1/2a clinical trial of NEUROSTEM® (NCT02054208), comparing NEUROSTEM and placebo groups for up to 36 months after the initial administration in patients suffering from Alzheimer's disease

Detailed Description

The subjects will be followed up at 12-month, 24-month, and 36-month (phone call) after the initial administration of NEUROSTEM®.

Study Timeline

• Study Start: 2017-05-19
• Study First Submitted: 2017-05-29
• Study First Submitted QC: 2017-05-29
• Study First Posted: 2017-06-01
• Primary Completion: 2022-03-18
• Study Completion: 2022-03-18
• Status Verified: 2023-03
• Last Update Submitted: 2023-03-13
• Last Update Posted: 2023-03-15

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 36

Inclusion Criteria

• Assessment of safety and exploratory treatment efficacy in subjects who were enrolled and completed phase 1/2a clinical trial of NEUROSTEM®.
• Subjects who have been treated with either NEUROSTEM® or placebo at least 12 months ago.
• 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

• Subjects who were not enrolled in phase 1/2a clinical trial of Neurostem® for assessing safety and exploratory treatment efficacy
• Other subjects, excluding those listed above, who were deemed unsuitable by the PI

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

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
NEUROSTEM® (hUCB-MSCs)- low dose	human umbilical cord blood derived mesenchymal stem cells	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
Placebo	Normal saline 2mL	normal saline 2mL, doses separated by 4 weeks for a total of 3 doses

Primary Outcome Measures

Name	Time	Method
Change from the baseline in ADAS-Cog	24 month after the first dose	Alzheimer's Disease assessment Scale-Cognitive Subscale

Secondary Outcome Measures

Name	Time	Method
Change from the baseline in CGA-NPI	24 month after the first dose	Caregiver-administered Neuropsychiatric Inventory
Change in Florbetaben-PET	24 month after the first dose	Florbetaben - Pittsburgh Compound B-positron emission tomography
Change in CDR-SOB	24 month after the first dose	Clinical Dementia Rating-Sum of Box
Change from baseline in MRI (DTI mapping)	24 month after the first dose	MRI Analysis
Change from the baseline in K-MMSE	24 month after the first dose	Mini Mental State Exmination Korean version
Change in CIBIC-plus	24 month after the first dose	The Clinician's Interview Based Impression of Change-plus
Change in FDG-PET (CMRglc: regional cerebral metabolic rate for glucose)	24 month after the first dose	fluorodeoxyglucose positron emission tomography
Change from the baseline in S-IADL	24 month after the first dose	Seoul Instrumental Activities of Daily Living
ADAS-Cog Response Rate	24 month after the first dose	the ADAS-cog score at 24 month after the first administration compared to the baseline
Change from the baseline in CSF biomarkers	24 month after the first dose	biomakrer analysis

Trial Locations

Locations (1)

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