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
Overview
- Phase
- Phase 1
- Intervention
- Approximately 100 million cells allogeneic hMSC
- Conditions
- Alzheimer Disease
- Sponsor
- Bernard (Barry) Baumel
- Enrollment
- 6
- Locations
- 1
- Primary Endpoint
- Number of Incidence of any Treatment-Emergent Serious Adverse Events (TE-SAEs)
- Status
- Completed
- Last Updated
- last year
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.
Investigators
Bernard (Barry) Baumel
Assistant Professor
University of Miami
Eligibility Criteria
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
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
Arms & Interventions
hMSC Treatment group
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
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)