Alzheimer's Disease Stem Cells Multiple Infusions

NCT04040348 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: 20190438
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this research study is to test the safety, possible side effects, and possible effectiveness of mesenchymal stem cell infusions when given to people with a diagnosis of mild to moderate Alzheimer's disease.

Conditions

Alzheimer Disease

Keywords

Dementia; Stem cells; Alzheimer's Disease

Outcome Measures

Primary Outcomes (1)

• Number of Incidence of any Treatment-Emergent Serious Adverse Events (TE-SAEs)

  All adverse events will be evaluated by the investigator for relationship with the study intervention. Treatment-Emergent Serious Adverse Events is defined as any untoward medical occurrence with a reasonable possibility that it is caused by the study intervention that: * Is life-threatening (e.g.; leads to stroke or non-fatal pulmonary embolism); * Requires inpatient hospitalization or prolongation of existing hospitalization; * Results in persistent or significant disability/incapacity * Results in other clinically significant sign(s) or symptom(s), (e.g.; clinically asymptomatic brain microhemorrhages); or * Results in death

  One month post-infusion

Secondary Outcomes (16)

• Cognitive function over time as assessed by the Alzheimer's Disease Assessment Scale - Cognitive (ADAS-Cog 11)

  Up to Week 65

• Cognitive function over time as assessed by the Mini Mental State Examination (MMSE) of Folstein test

  Up to Week 65

• Depressive symptoms over time as assessed by the Geriatric Depression Scale (GDS) Short Version

  Up to Week 65

• Participant quality of life over time assessed via Alzheimer's Disease Related Quality of Life (ADRQL-40) Questionnaire as completed by the caregiver

  Up to Week 65

• Participant quality of life over time as assessed via the Alzheimer's Disease Cooperative Study Activities of Daily Living (ADCS-ADL) Questionnaire as completed by the caregiver

  Up to Week 65

Study Arms (1)

hMSC Treatment group

EXPERIMENTAL

Participants in the hMSC treatment group will receive a total of 4 doses of the hMSC intervention. Each dose will be administered once about every 13 weeks within a year period.

Biological: Approximately 100 million cells allogeneic hMSC

Interventions

Approximately 100 million cells allogeneic hMSC BIOLOGICAL

Umbilical cord-derived, allogeneic hMSC administered intravenously at a dose of approximately 100 million cells per infusion.

hMSC Treatment group

Eligibility Criteria

• Age: 50 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• All subjects enrolled in this trial must:
• Provide written informed consent
• Male or female subjects aged 50-85 years at time of signing Informed Consent
• Mini-Mental State Examination (MMSE) between 20-26
• Amyloid PET scan or CSF Aß1-42 positive for the presence of amyloid
• Meet criteria for either Alzheimer's Disease or probable Alzheimer's Disease (AD) according to National Institute of Neurological and Communicative Disorders and Stroke/Alzheimer's Disease and Related Disorders Association (NINDCDS/ARDRA)
• Subjects, if taking cholinesterase inhibitor medications (donepezil, rivastigmine (oral or transdermal) or galantamine), are required to have been taking them on a stable dose for at least 3 months prior to Baseline Visit These medicines are not required

You may not qualify if:

• Have a study partner
• No clinically significant abnormal screening laboratory values, as determined by the investigator
• Women must be postmenopausal, surgically sterile, or having infertility. A postmenopausal woman is defined as either having an intact uterus with at least 12 months of spontaneous amenorrhea or a diagnosis of menopause, defined as an Follicular Stimulating Hormone (FSH) level of \> 25 IU/L
• All subjects enrolled must not have:
• Dementia other than AD
• A negative Amyloid PET scan
• Other neurodegenerative disease
• Significant psychiatric illness (e.g., uncontrolled major depression, schizophrenia, bipolar affective disorder)
• History of seizures
• Contraindication for Magnetic Resonance Imaging (MRI)
• History of malignancy, except:
• \> 5 years in remission prior to screening
• Be excised or treated basal cell, squamous carcinoma or melanoma in situ
• Prostate cancer in situ
• Cervical carcinoma in situ

Sponsors & Collaborators

• Bernard (Barry) Baumel: lead

Study Sites (1)

University of Miami

Miami, Florida, 33136, United States

Location

MeSH Terms

Conditions

Alzheimer Disease; Dementia

Condition Hierarchy (Ancestors)

Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Tauopathies; Neurodegenerative Diseases; Neurocognitive Disorders; Mental Disorders

Study Officials

• Bernard (Barry) Baumel, MD

  University of Miami

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Assistant Professor

Study Record Dates

• First Submitted: July 30, 2019
• First Posted: July 31, 2019
• Study Start: October 8, 2019
• Primary Completion: April 25, 2023
• Study Completion: April 25, 2023
• Last Updated: April 27, 2025

Record last verified: 2023-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)