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Alzheimer DiseasePhase 1NCT04040348

Alzheimer's Disease Stem Cells Multiple Infusions

A Phase 1 study testing Approximately 100 million cells allogeneic hMSC in people with Alzheimer Disease, run by Bernard (Barry) Baumel at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 50 to 85 years
Conditions the study is for
Alzheimer Disease
What is being tested?
Approximately 100 million cells allogeneic hMSC
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
6 participants
Where
1 siteUnited States
Study dates
8 Oct 2019to 25 Apr 2023
Phase
Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 50 to 85 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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)
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  • 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
  • Subjects already taking memantine will not have an effect in the inclusion/exclusion criteria.
  • 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

Who cannot join

  • 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
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  • 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
  • Uncontrolled medical conditions
  • Hypertension
  • Diabetes
  • Unstable angina or history of Myocardial Infarction (MI) within 1 year prior to screening
  • History of alcohol or drug use disorder (except tobacco use disorder) within 2 years before the screening visit
  • Brain MRI at screening that shows evidence of findings incompatible with a diagnosis of Alzheimer's disease. Volumetric MRI scans done within 6 months prior to ICF signature will be accepted if completed locally.
  • History of bleeding disorder
  • History of or positive results for Human Immunodeficiency Virus (HIV)
  • History of or positive results for Hepatitis C Virus (HCV) or Hepatitis B Virus (HBV)
  • Hypersensitivity to dimethyl sulfoxide (DMSO)
  • Inability to perform any of the assessments required for endpoint analysis
  • Currently receiving (or received within four weeks of screening) experimental agents for the treatment of AD or enrolled in clinical trials in the prior 3 months
  • Be a transplant recipient, or on active listing (or expected future listing) for transplant of any organ.
  • Any other condition that, in the opinion of the investigator, may compromise the safety or compliance of the patient or preclude successful completion of the study.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • University of Miami

    Miami, Florida, 33136

Other trials in United States

About this study

Official title: A Phase I, Prospective, Open-label Trial to Evaluate the Safety, Tolerability and Exploratory Outcomes of Multiple Allogeneic Human Mesenchymal Stem Cells (HMSC) Infusions in Patients With Mild to Moderate Alzheimer's Disease

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.

Being tested
Approximately 100 million cells allogeneic hMSC
Keywords
Dementia, Stem cells, Alzheimer's Disease
Medical subject headings
Alzheimer Disease, Dementia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Approximately 100 million cells allogeneic hMSCBiological

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

Study groups in NCT04040348
Study groupRoleWhat they receive
hMSC Treatment groupExperimentalParticipants 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.
Receives: Biological: Approximately 100 million cells allogeneic hMSC

What is being measured

The primary outcome is the main question the study is designed to answer.

  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

    Measured: One month post-infusion

Secondary outcomes (16)

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

    The ADAS-Cog 11 is a 13-item version of ADAS-Cog comprising of the original 11-item ADAS-Cog as well as Delayed Recall and Digit Cancellation items. The total score ranges from 0-85 points, with a lower score indicating better performance.

    Measured: Up to Week 65

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

    The MMSE assesses orientation to time and place, immediate and delayed recall of words, attention and calculation, language (naming, comprehension and repetition), and spatial ability (copying a figure). The total score ranges from 0-30, with a higher score indicating better cognitive performance.

    Measured: Up to Week 65

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

    The GDS is a 15-item questionnaire with each item counting as one point. The total score ranges from 0 to 15 with a score greater than 5 indicating possible depression.

    Measured: Up to Week 65

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

    ADRQL-40 is a questionnaire completed by the caregiver assessing the quality of life of the participant with AD. The total score for the ADRQL is computed by summing the values assigned to the responses, dividing the sum by the maximum value for the scale and multiplying the results by 100 to obtain a percentage score of 0 to 100. A higher score reflects a higher quality of life.

    Measured: Up to Week 65

  5. 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

    The ADCS-ADL is a 23 item questionnaire completed by the caregiver assessing the basic and instrumental activities of daily living by the AD participant. Total score range from 0-78 with the higher score indicating increased independence.

    Measured: Up to Week 65

  6. Neuropsychiatric Inventory-Q (NPI-Q) Scores over time

    The NPI-Q is a questionnaire used to assess behavioral changes common in dementia patients. This questionnaire is completed by the caregiver. The questionnaire consists of 12 items with each item having a scoring range between 0-3. The higher score indicates a more severe neuropsychiatric symptomatology.

    Measured: Up to Week 52

  7. Caregiver's Quality of life over time as assessed by the Caregiver Self-Assessment Questionnaire scores

    The Caregiver Self-Assessment questionnaire is completed by the caregiver. It is an 18-item questionnaire answered with a "yes" or "no". Evidence of distress is indicated for having over 10 "yes" answers.

    Measured: Up to Week 52

  8. Biomarker levels over time

    Serum blood inflammatory and biomarker levels will be evaluated including Interleukin-6 (IL-6), Neurofilament light (NfL), Amyloid Beta 40 (Aβ40) and Amyloid Beta 42 (Aβ42) in pg/mL.

    Measured: Up to Week 65

  9. Serum ApoE level over time

    Serum blood Apolipoprotein E (ApoE) will be evaluated in mg/dL.

    Measured: Up to Week 65

  10. Serum PRA level over time

    Serum blood Plasma Renin Activity (PRA) will be evaluated in ng/mL per hour.

    Measured: Up to Week 65

  11. Serum Tau protein level over time

    Serum blood Tau protein level will be evaluated in ng/L.

    Measured: Up to Week 65

  12. Cerebrospinal Fluid (CSF) Biomarker levels over time

    CSF inflammatory and biomarker levels will be evaluated including Interleukin-6 (IL-6), Neurofilament light (NfL), Amyloid Beta 40 (Aβ40) and Amyloid Beta 42 (Aβ42) in pg/mL.

    Measured: Up to Week 52

  13. CSF ApoE level over time

    CSF Apolipoprotein E (ApoE) levels will be evaluated in mg/dL.

    Measured: Up to Week 52

  14. CSF PRA level over time

    CSF Plasma Renin Activity (PRA) levels will be evaluated in ng/mL per hour.

    Measured: Up to Week 52

  15. CSF Tau protein level over time

    CSF Tau protein levels will be evaluated in ng/L.

    Measured: Up to Week 52

  16. Change in hippocampal volume

    Change in hippocampal volume will be assessed via MRI Brain volumetric studies

    Measured: Baseline to Week 6, Baseline to Week 52

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted31 Jul 2019
  2. Study start8 Oct 2019
  3. Primary completion25 Apr 2023
  4. Study completion25 Apr 2023

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
6 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04040348
Sponsor's protocol ID
20190438
Status last verified
May 2026
First submitted
30 Jul 2019
Last updated
28 May 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Approximately 100 million cells allogeneic hMSC in people with Alzheimer Disease, run by Bernard (Barry) Baumel at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 11 inclusion and 26 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 28 May 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Incidence of any Treatment-Emergent Serious Adverse Events (TE-SAEs) (time frame: One month post-infusion). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04040348.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 May 2026.

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Alzheimer's Disease Stem Cells Multiple Infusions | AzTrials