Alzheimer Disease
Conditions
Keywords
Alzheimer's Disease, AD, Autologous, Stem cells
Brief summary
This is a research study to evaluate the safety of an investigational autologous cell product obtained from participant's own adipose tissue as a possible treatment for Alzheimer disease.
Detailed description
This is a Phase 1, Open-Label Safety Study of Escalating Doses of Intracerebroventricular Injections of Ex Vivo Expanded, Autologous Adipose-Derived Stem Cells (ADSCs) in Participants with Mild to Moderate Alzheimer's Disease (AD) whose Treatment is not Addressed Adequately by Available Therapy (i.e., an unmet medical need). The investigational product, which is referred to as RB-ADSC, consists of stem cells obtained from the participant's own adipose tissue by lipoaspirate. After collection, the stem cells are cultured and expanded outside the body, and then reintroduced into the same patient. A soft plastic reservoir (Ommaya reservoir) is implanted under the scalp, communicating with the brain cavities (ventricles). This study will primarily evaluate the safety of RB-ADSC injected in the Ommaya reservoir in a 3 + 3 dose escalation study. The planned enrollment will be 9 participants, 3 participants per escalation Cohort. The primary objectives will evaluate adverse events, serious adverse events, and dose limiting toxicities to determine a recommended phase 2 clinical trial dose. Secondary objectives will evaluate preliminary efficacy measured by clinical assessments, volumetric MRI (Neuro Quant®), CSF biomarkers (Phospho-Tau, Total Tau, AB-42), and diagnostic imaging comparison (Amyloid PET). Each participant will be followed for 12 months after treatment.
Interventions
BIOLOGICALRB-ADSC
Ex Vivo Expanded, Autologous Adipose-Derived Stem Cells (ADSCs)
Sponsors
Regeneration Biomedical, Inc.
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
45 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
* ≥45 and ≤80 years of age * Mild to moderate AD diagnosis * Adequate cognitive function * Non-remarkable clinical laboratory * Ability to voluntarily provide written informed consent * No tumors or other disease responsible for dementia * Well-controlled comorbidities, on stable medications for 3 months * The participant is otherwise in good general health * The participant must have a relative/caregiver * Participant must be able to donate adequate amount of lipoaspirate to establish the final product * Caregiver separately meets the specified inclusion/
Exclusion criteria
for caregivers
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| The safety of RB-ADSC treatment in study participants with AD | up to 28 weeks | Safety will be determined by incidence, type and severity of adverse events (AE) and serious adverse events (SAE) graded according to CTCAE v5.0 and CRS revised grading system and defined by clinical relevant findings at every visit and week 28 post-treatment in physical examination, vital signs and laboratory data |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from Baseline in Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-cog) | up to 52 weeks | Measured by the Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-cog) is a cognitive performance test. The ADAS-cog score ranges from 0 to 70, with higher scores indicating greater cognitive impairment (higher score is worse outcome). |
| Change from Baseline in Mini Mental State Examination (MMSE) | up to 52 weeks | Measured by the Mini Mental State Examination (MMSE). MMSE is a performance-based test of global cognitive status.. The MMSE score ranges from 0 to 30, with lower scores indicating greater cognitive impairment (lower score is worse outcome). |
Countries
United States
Outcome results
None listed