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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