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Table 2 Ongoing stem cell trials in humans with Alzheimer’s disease

From: Alzheimer’s disease, dementia, and stem cell therapy

Trial ID NCT01547689 NCT02054208 NCT02600130 NCT02912169 NCT02833792 NCT02672306 NCT02899091
Date 03/2012 to 12/2016 02/2014 to 02/2018 11/2015 to 10/2019 11/2015 to 12/2017 06/2016 to 06/2018 05/2016 to 10/2019 09/2016 to 06/2018
Study design Phase I/II
Safety and efficacy
Intervention
Single group open-label
Phase I/II
Safety and efficacy
Intervention
Randomized
Double-blind
Placebo-controlled
Phase I
Safety and efficacy
Intervention
Randomized
Double-blind
Placebo-controlled
Phase I/II
Safety and efficacy
Intervention
Non-randomized
Single group
Open-label
Multi-centre
Phase II
Safety and efficacy
Intervention
Randomized
Single-blind
Placebo-controlled
Multi-centre
Phase I/II
Safety and efficacy
Intervention
Randomized
Double-blind
Placebo-controlled
Phase I/II
Safety and efficacy
Intervention
Randomized
Double-blind
Placebo-controlled
Stage Active Recruiting Recruiting Recruiting Recruiting Not yet recruiting Not yet recruiting
Cell type hUCB-MSCs hUCB-MSCs hBM-MSCs hAD-SVF hBM-MSCs hUCB-MSCs hPD-MSCs
Inclusion criteria Age 50–85
Probable AD
K-MMSE 3–20
Age 50–85
Probable AD
K-MMSE 18–26
Amyloid+ PIB/florbetaben-PET
Age 50–80
Diagnosed AD
K-MMSE 18–24
Amyloid+ PET
Age ≥55
Probable AD (NINCDS-ADRDA and DSM IV)
Age 55–80
Mild-moderate AD
K-MMSE 12–24
Amyloid+
florbetapir-PET
Age 50–85
Probable AD
K-MMSE 3–20
Age ≥50
Probable AD
K-MMSE 10–26
Amyloid+ PET
Delivery route Intravenous infusion Ommaya Reservoir intraventricular injection Intravenous infusion Intravenous and intranasal infusion Intravenous infusion Intravenous infusion Intravenous infusion
Arms n = 30
Eight infusions once every 2 weeks in the first month of each quarter
2 × 107 cells per infusion
n = 42
Three injections at 4-week intervals
Low-dose group: 1 × 107 cells per injection
High-dose group: 3 × 107 cells per injection
Placebo group: saline
n = 30
Single infusion
Low-dose group: 2 × 107 cells
High-dose group: 1 × 108 cells
Placebo group: Plasmalyte A and 1% human serum albumin
n = 100
Single intravenous infusion or intravenous and intranasal infusion
n = 40
Single infusion
Crossover at 6 months post-infusion
Group 1: 1.5 × 106 cells/kg bodyweight
Group 2: lactated Ringer’s Solution
n = 40
Eight infusions at 2-week intervals
Treatment group: 2 × 107 cells per infusion
Placebo group: saline
n = 24
Single or repeat (day 0 and week 4) infusions
Arm 1:
K-MMSE 20–26
Arm 2:
K-MMSE 10–19
Group1: 2 × 108 cells
Group 2: two infusions of 2 × 108 cells
Placebo group:
placebo infusion
Outcome
measures
10 weeks FU
No. of adverse events
Change from baseline:
ADAS-cog, MMSE, CIBIC, ADCS-ADL and CGA-NPI
CSF transthyretin, Aβ and tau
Blood Thl/Th2 cytokines
24 weeks FU
No. of adverse events
Change from baseline:
ADAS-cog, S-IADL, K-MMSE, CIBIC, CGA-NPI and CDR
CSF biomarkers
MRI DTI mapping, PIB-PET and FDG-PET
30 days FU
No. of adverse events
2, 4, 13, 39, and 52 weeks FU
Change from baseline:
ADAS-cog, MMSE, CGA-NPI and GDS
CSF inflammatory markers, Aβ and tau
Blood inflammatory and AD biomarkers
MRI brain volumetry
12 months FU
No. of adverse events
3 and 6 months FU
Change from baseline:
FAQ, GDS, MMSE and ADCS-ADL
18 months FU
No. of adverse events
Change from baseline:
Neurological examinations
10 weeks FU
No. of adverse events
Change from baseline:
ADAS-cog, MMSE, ADCS-CCGIC, ADCS-ADL and CGA-NPI
CSF Aβ and tau
Blood Aβ
48 weeks FU
No. of adverse events
Change from baseline:
ADAS-cog, K-MMSE, GDS, CDR, K-IADL, CGA-NPI, CIBIC and SF-36
CSF Aβ and tau
Brain MRI, amyloid-PET, FDG-PET, CMRglc
Quantitative ECG
  1. amyloid beta, AD Alzheimer’s disease, ADAS-cog Alzheimer’s Disease Assessment Scale-Cognitive Subscale, ADCS-ADL Alzheimer’s Disease Cooperative Study Activities of Daily Living, ADCS-CCGIC Alzheimer’s Disease Cooperative Study Clinician’s Global Impression of Change, CDR Clinical Dementia Rating, CGA-NPI Caregiver-administered Neuropsychiatric Inventory, CIBIC Clinician’s Interview-Based Impression of Change, CMRglc cerebral metabolic rate for glucose, CSF cerebrospinal fluid, DSM Diagnostic and Statistical Manual of Mental Disorders, DTI diffusion tensor imaging, ECG electrocardiogram, FAQ Functional Activities Questionnaire, FDG fluorodeoxyglucose, FU follow-up, GDS Geriatric Depression Scale, hAD-SVF human adipose-derived stromal vascular fraction, hBM-MSC human bone marrow-derived mesenchymal stem cell, hPD-MSC human placenta-derived mesenchymal stem cell, hUCB-MSC human umbilical cord blood-derived mesenchymal stem cell, K-IADL Korean Instrumental Activities of Daily Living, K-MMSE Korean version of Mini-Mental State Evaluation, MMSE Mini-Mental State Evaluation, MRI magnetic resonance imaging, NINCDS-ADRDA National Institute of Neurological and Communicative Disorders and Stroke and the Alzheimer's Disease and Related Disorders Association, PET positron emission tomography, PIB Pittsburgh compound B, SF-36 36-item Short Form Health Survey, S-IADL Seoul-Instrumental Activities of Daily Living, Th T helper