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

Alzheimer's Disease Stem Cells Multiple Infusions

Sponsors

Lead sponsor: Bernard (Barry) Baumel

Source University of Miami
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.

Overall Status Recruiting
Start Date October 8, 2019
Completion Date September 1, 2021
Primary Completion Date September 1, 2021
Phase Phase 1
Study Type Interventional
Primary Outcome
Measure Time Frame
Number of Incidence of any Treatment-Emergent Serious Adverse Events (TE-SAEs) One month post-infusion
Secondary Outcome
Measure Time Frame
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
Olfactory function over time as assessed by the Odor Identification test. 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
Neuropsychiatric Inventory-Q (NPI-Q) Scores over time Up to Week 52
Caregiver's Quality of life over time as assessed by the Caregiver Self-Assessment Questionnaire scores Up to Week 52
Biomarker levels over time Up to Week 65
Serum ApoE level over time Up to Week 65
Serum PRA level over time Up to Week 65
Serum Tau protein level over time Up to Week 65
Cerebrospinal Fluid (CSF) Biomarker levels over time Up to Week 52
CSF ApoE level over time Up to Week 52
CSF PRA level over time Up to Week 52
CSF Tau protein level over time Up to Week 52
Change in hippocampal volume Baseline to Week 6, Baseline to Week 52
Enrollment 6
Condition
Intervention

Intervention type: Biological

Intervention name: 100 million cells allogeneic hMSC

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

Arm group label: hMSC Treatment group

Eligibility

Criteria:

Inclusion Criteria:

All subjects enrolled in this trial must:

1. Provide written informed consent

2. Male or female subjects aged 50-85 years at time of signing Informed Consent

3. Mini-Mental State Examination (MMSE) between 20-26

4. Amyloid PET scan or CSF Aß1-42 positive for the presence of amyloid

5. Meet criteria for 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)

6. Subjects are required to have been taking cholinesterase inhibitor medications (donepezil, rivastigmine (oral or transdermal) or galantamine) on a stable dose for at least 3 months prior to Baseline Visit

7. 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

8. Have a study partner

9. No clinically significant abnormal screening laboratory values, as determined by the investigator

10. 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

Exclusion Criteria:

All subjects enrolled must not have:

1. Dementia other than AD

2. A negative Amyloid PET scan

3. Other neurodegenerative disease

4. Significant psychiatric illness (e.g., uncontrolled major depression, schizophrenia, bipolar affective disorder)

5. History of seizures

6. Contraindication for Magnetic Resonance Imaging (MRI)

7. 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

8. 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

9. 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.

10. History of bleeding disorder

11. History of or positive results for Human Immunodeficiency Virus (HIV)

12. History of or positive results for Hepatitis C Virus (HCV) or Hepatitis B Virus (HBV)

13. Hypersensitivity to dimethyl sulfoxide (DMSO)

14. Inability to perform any of the assessments required for endpoint analysis

15. 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

16. Be a transplant recipient, or on active listing (or expected future listing) for transplant of any organ.

17. 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.

Gender: All

Minimum age: 50 Years

Maximum age: 85 Years

Healthy volunteers: No

Overall Official
Last Name Role Affiliation
Bernard (Barry) Baumel, MD Principal Investigator University of Miami
Overall Contact

Last name: Carmen Perez

Phone: 305-243-0184

Email: [email protected]

Location
facility status contact investigator University of Miami Carmen Perez 305-243-0184 [email protected] Bernard (Barry) Baumel, MD Principal Investigator
Location Countries

United States

Verification Date

May 2020

Responsible Party

Responsible party type: Sponsor-Investigator

Investigator affiliation: University of Miami

Investigator full name: Bernard (Barry) Baumel

Investigator title: Assistant Professor

Keywords
Has Expanded Access No
Condition Browse
Number Of Arms 1
Arm Group

Arm group label: hMSC Treatment group

Arm group type: Experimental

Description: 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.

Patient Data No
Study Design Info

Allocation: N/A

Intervention model: Single Group Assignment

Primary purpose: Treatment

Masking: None (Open Label)

Source: ClinicalTrials.gov