RB-ADSC för behandling av mild till måttlig Alzheimers sjukdom
Ph 1, öppen säkerhetsstudie av eskalerande doser av intracerebroventrikulära injektioner av ex vivo expanderade, autologa ADSC:er hos deltagare med mild-måttlig AD vars behandling inte behandlas adekvat av tillgänglig terapi
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.
Studieöversikt
Detaljerad beskrivning
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.
Studietyp
Inskrivning (Faktisk)
Fas
- Fas 1
Kontakter och platser
Studieorter
-
-
California
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Newport Beach, California, Förenta staterna, 92663
- Hoag Memorial Hospital Presbyterian
-
-
Deltagandekriterier
Urvalskriterier
Åldrar som är berättigade till studier
Tar emot friska volontärer
Kön som är behöriga för studier
Beskrivning
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
Exclusion Criteria:
- Taking other medications for AD, except that donepezil memantine, AChEIs including patches, Vitamin E, fish oil, and/or gingko biloba are allowed if doses have been stable for at least 3 months prior to the Screening visit
- Stem cell implantation of any type within 3 months
- Existing ventriculoperitoneal shunts
- Neurological disorders except AD
- Psychiatric disorders including schizophrenia, bipolar/unipolar depressive disorder, delirium
- Drug or alcohol abuse or dependence within the past 5 years
- Participants with a history of cancer in the past 5 years
- No caregiver available to meet the inclusion criteria for caregivers
Studieplan
Hur är studien utformad?
Designdetaljer
- Primärt syfte: Behandling
- Tilldelning: Icke-randomiserad
- Interventionsmodell: Sekventiell tilldelning
- Maskning: Ingen (Open Label)
Vapen och interventioner
Deltagargrupp / Arm |
Intervention / Behandling |
|---|---|
|
Experimentell: RB-ADSC low dose
Participants will receive one dose of 2x10^6 RB-ADSC infused in the previously implanted Ommaya reservoir |
Ex Vivo Expanded, Autologous Adipose-Derived Stem Cells (ADSCs) |
|
Experimentell: RB-ADSC medium dose
Participants will receive one dose of 5x10^6 RB-ADSC infused in the previously implanted Ommaya reservoir |
Ex Vivo Expanded, Autologous Adipose-Derived Stem Cells (ADSCs) |
|
Experimentell: RB-ADSC high dose
Participants will receive one dose of 10x10^6 RB-ADSC infused in the previously implanted Ommaya reservoir |
Ex Vivo Expanded, Autologous Adipose-Derived Stem Cells (ADSCs) |
Vad mäter studien?
Primära resultatmått
Resultatmått |
Åtgärdsbeskrivning |
Tidsram |
|---|---|---|
|
The safety of RB-ADSC treatment in study participants with AD
Tidsram: 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 |
up to 28 weeks
|
Sekundära resultatmått
Resultatmått |
Åtgärdsbeskrivning |
Tidsram |
|---|---|---|
|
Change from Baseline in Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-cog)
Tidsram: 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). |
up to 52 weeks
|
|
Change from Baseline in Mini Mental State Examination (MMSE)
Tidsram: 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). |
up to 52 weeks
|
Samarbetspartners och utredare
Sponsor
Publikationer och användbara länkar
Studieavstämningsdatum
Studera stora datum
Studiestart (Faktisk)
Primärt slutförande (Faktisk)
Avslutad studie (Faktisk)
Studieregistreringsdatum
Först inskickad
Först inskickad som uppfyllde QC-kriterierna
Första postat (Faktisk)
Uppdateringar av studier
Senaste uppdatering publicerad (Faktisk)
Senaste inskickade uppdateringen som uppfyllde QC-kriterierna
Senast verifierad
Mer information
Termer relaterade till denna studie
Nyckelord
Ytterligare relevanta MeSH-villkor
Andra studie-ID-nummer
- RB-ADSC-02
Plan för individuella deltagardata (IPD)
Planerar du att dela individuella deltagardata (IPD)?
Läkemedels- och apparatinformation, studiedokument
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