Autologous Activated Adipose-derived Stem Cells (RB-ADSC) Injected Directly Into the Brain for Mild to Moderate Alzheimer's Disease

February 8, 2024 updated by: Regeneration Biomedical, Inc.

Ph 1, Open-Label Safety Study of Escalating Doses of Intracerebroventricular Injections of Ex Vivo Expanded, Autologous ADSCs in Participants With Mild-Moderate AD Whose Treatment is Not Addressed Adequately by Available Therapy

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Estimated)

18

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

45 years to 80 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: RB-ADSC low dose
Participants will receive one dose of 2x10^6 RB-ADSC infused in the previously implanted Ommaya reservoir
Biological: RB-ADSC
Ex Vivo Expanded, Autologous Adipose-Derived Stem Cells (ADSCs)
Experimental: RB-ADSC medium dose
Participants will receive one dose of 5x10^6 RB-ADSC infused in the previously implanted Ommaya reservoir
Biological: RB-ADSC
Ex Vivo Expanded, Autologous Adipose-Derived Stem Cells (ADSCs)
Experimental: RB-ADSC high dose
Participants will receive one dose of 10x10^6 RB-ADSC infused in the previously implanted Ommaya reservoir
Biological: RB-ADSC
Ex Vivo Expanded, Autologous Adipose-Derived Stem Cells (ADSCs)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
The safety of RB-ADSC treatment in study participants with AD
Time Frame: 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

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from Baseline in Mini Mental State Examination (MMSE)
Time Frame: 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
Change from Baseline in Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-cog)
Time Frame: 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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Regeneration Biomedical, Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 14, 2023

Primary Completion (Estimated)

February 1, 2025

Study Completion (Estimated)

March 1, 2025

Study Registration Dates

First Submitted

December 1, 2022

First Submitted That Met QC Criteria

December 19, 2022

First Posted (Actual)

December 28, 2022

Study Record Updates

Last Update Posted (Actual)

February 12, 2024

Last Update Submitted That Met QC Criteria

February 8, 2024

Last Verified

February 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • RB-ADSC-02

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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