NCT04040348CompletedPhase 1
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
Bernard (Barry) Baumel2 sites in 1 country6 target enrollmentStarted: October 8, 2019Last updated:
Conditions
Interventions
Drugs
Trial Snapshot
- Phase
- Phase 1
- Status
- Completed
- Sponsor
- Enrollment
- 6
- Locations
- 2
- Primary Endpoint
- Number of Incidence of any Treatment-Emergent Serious Adverse Events (TE-SAEs)
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 50 Years to 85 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •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
- •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
- •Exclusion Criteria:
- •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
- •Uncontrolled medical conditions
- •Hypertension
- •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.
Arms & Interventions
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.
Intervention: Approximately 100 million cells allogeneic hMSC (Biological)
Outcomes
Primary Outcomes
Number of Incidence of any Treatment-Emergent Serious Adverse Events (TE-SAEs)
Time Frame: One month post-infusion
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
Secondary Outcomes
- 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)
- 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)
- 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)
- CSF Tau protein level over time(Up to Week 52)
- 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)
- Neuropsychiatric Inventory-Q (NPI-Q) Scores over time(Up to Week 52)
- CSF PRA level over time(Up to Week 52)
- Change in hippocampal volume(Baseline to Week 6, Baseline to Week 52)
- Cognitive function over time as assessed by the Alzheimer's Disease Assessment Scale - Cognitive (ADAS-Cog 11)(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)
- Caregiver's Quality of life over time as assessed by the Caregiver Self-Assessment Questionnaire scores(Up to Week 52)
Investigators
Bernard (Barry) Baumel
Assistant Professor
University of Miami
Study Sites (2)
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