A Clinical Trial to Determine the Safety and Efficacy of Hope Biosciences Autologous Mesenchymal Stem Cell Therapy (HB-adMSCs) for the Treatment of Alzheimer's Disease

NCT ID: NCT04228666

Last Updated: 2025-09-29

Basic Information

• Recruitment Status: WITHDRAWN
• Clinical Phase: PHASE1/PHASE2
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-03-01
• Study Completion Date: 2022-02-01

Brief Summary

Hope Biosciences is conducting a research study of an investigational product called autologous adipose-derived mesenchymal stem cells (abbreviated as HB-adMSCs) as a possible treatment for Alzheimer's disease (AD). The study purpose is to evaluate the safety profile of four IV infusions of HB-adMSCs in subjects with clinical diagnosis of AD.

Detailed Description

This is a Phase 1/2a, open-label, non-randomized study in subjects with Alzheimer's disease. 24 patients will be enrolled for the study. The overall objective of this study is to evaluate the safety profile of four IV infusions of autologous adipose-derived mesenchymal stem cells (HB-adMSCs) in subjects with clinical diagnosis of AD. The primary endpoint of this study is to measure the number and frequency of adverse event(s) and/or severe adverse event(s) throughout the study duration. The second endpoint of this study is to evaluate the ability of HB-adMSCs to alter AD-related inflammation via measuring levels of Tumor Necrosis Factor alpha (TNF-a), Interleukin-1 (IL-1), Interleukin-6 (IL-6), C-Reactive Protein (CRP), and markers associated with amyloid deposition, Amyloid beta 40 and Amyloid beta 42. Subjects will also be assessed for cognitive deficits measured by changes from baseline values using Mini Mental Status Examination (MMSE), Alzheimer's disease Cooperative Study Activities of Daily Living (ADCS-ADL), Alzheimer's disease Related Quality of Life (ADRQL), Altoida Neuro Motor Index (NMI) for Digital Biomarkers, and Clinical Dementia Rating Questionnaire (CDR).

Conditions

Alzheimer Disease

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

HB-adMSCs

HB-adMSCs are autologous, adipose-derived mesenchymal stem cells. Four intravenous infusions will be administered on weeks 0, 2, 6, and 8 at a dose of 2 x 10\^8 total HB-adMSC cells.

Group Type: EXPERIMENTAL

HB-adMSCs

Intervention Type: BIOLOGICAL

Four IV infusions of autologous adipose-derived mesenchymal stem cells. Baseline laboratory data will be collected prior to first infusion; follow-up data will be compared against baseline according to the following schedule: safety laboratory tests follow-up on weeks 4, 8, 13, 26, and 52; inflammation and amyloid markers follow-up on weeks 13 and 52; MMSE and ADCS-ADL follow-up on weeks 13, 19, 26, 33, 40, 46 and 52; Altoida NMI follow-up will occur weekly from week 0 to week 52; CDR follow-up will occur weeks 4, 10, 13, 19, 26, 33, 40, 46 and 52; C-SSRS follow-up will occur on weeks 4, 10, 26, and 52; Amyloid PET imaging follow-up occurs week 26 and 52;

Interventions

HB-adMSCs

Four IV infusions of autologous adipose-derived mesenchymal stem cells. Baseline laboratory data will be collected prior to first infusion; follow-up data will be compared against baseline according to the following schedule: safety laboratory tests follow-up on weeks 4, 8, 13, 26, and 52; inflammation and amyloid markers follow-up on weeks 13 and 52; MMSE and ADCS-ADL follow-up on weeks 13, 19, 26, 33, 40, 46 and 52; Altoida NMI follow-up will occur weekly from week 0 to week 52; CDR follow-up will occur weeks 4, 10, 13, 19, 26, 33, 40, 46 and 52; C-SSRS follow-up will occur on weeks 4, 10, 26, and 52; Amyloid PET imaging follow-up occurs week 26 and 52;

Intervention Type: BIOLOGICAL

Eligibility Criteria

Inclusion Criteria

1. Men, and women of non-childbearing potential, 50-85 years of age inclusively, with a diagnosis of early stage's (preclinical/mild cognitive impairment) Probable Alzheimer's Disease according to the 2011 NIA-AA criteria.

• Non-childbearing potential for women is defined as postmenopausal [last natural menses greater than 24 months; in women under age 55, menopausal status will be documented with serum follicle stimulating hormone (FSH) test] or undergone a documented bilateral tubal ligation or hysterectomy.
• Male participants who are sexually active with a woman of childbearing potential must agree to use condoms during the trial unless the woman is using an acceptable means of birth control. Acceptable forms of birth control include abstinence, birth control pills, or any double combination of intrauterine device (IUD), male or female condom and diaphragm.

2. Informed consent signed by the subject

3. Documented Amyloid PET Scan (images and report) positive to amyloid plaques deposits on the brain.

4. If the patient is under any treatment, should have been on a stable dose for at least 30 days prior to signing the informed consent form and there is no intention to modify the dose over the course of the study. (NOTE: Cholinesterase inhibitors (AChEI) (donepezil, galantamine, or rivastigmine) may not be initiated, discontinued or modified after study initiation for the 12-months control period).

Exclusion Criteria

1. Hospitalization or change of chronic concomitant medication within one month prior to screening.

2. Clinically significant or unstable disease that may interfere with outcome evaluations, including but not limited to:

• Respiratory Insufficiency
• Poorly managed hypertension (systolic >160 mm Hg and/or diastolic >95 mm Hg) or hypotension (systolic <90 mm Hg and/or diastolic <60 mm Hg); or
• Bradycardia (<50 beats/min.) or tachycardia (>100 beats/min.). Otherwise healthy subjects with borderline bradycardia may be discussed with the medical monitor to determine eligibility.
• Renal insufficiency, defined as eGFR <40 mL/min based on the CKD-EPI (Chronic Kidney Disease Epidemiology Collaboration) formula, https://www.mdcalc.com/ckd-epi-equations-glomerular-filtration-rate-gfr
• Heart disease (myocardial infarction, unstable angina, heart failure, cardiomyopathy within 3 months before screening). If a subject has a history of heart disease of questionable clinical significance, the medical monitor may be contacted to discuss eligibility.

3. Records of PET Scan negative to Amyloid plaques deposition in the brain.

4. Suspected or known drug or alcohol abuse, i.e. more than approximately 60 g alcohol (approximately 1 liter of beer or 0.5 liter of wine) per day.

5. Acute intercurrent infections such as Hepatitis C Virus (HCV), Hepatitis B Virus (HBV), Human Immunodeficiency Virus (HIV) or Syphilis.

6. Contraindications for PET scanning, including implanted metallic devices (e.g. non-MRI-safe cardiac pacemaker or neurostimulator; some artificial joints metal pins; surgical clips; or other implanted metal parts), or claustrophobia or discomfort in confined spaces.

7. Is unable or unwilling to comply with protocol follow-up requirements.

8. Enrollment in another investigational study or intake of investigational drug within the previous 30 days.

9. Any condition, which in the opinion of the investigator or the sponsor makes the patient unsuitable for inclusion.

• Minimum Eligible Age: 50 Years
• Maximum Eligible Age: 85 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Hope Biosciences Research Foundation

INDUSTRY

Sponsor Role: collaborator

Hope Biosciences LLC

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Djamchid Lotfi, MD

Role: PRINCIPAL_INVESTIGATOR

Clinical Trial Network

Locations

Clinical Trial Network

Houston, Texas, United States

Countries

United States

Other Identifiers

HBALZ01

Identifier Type: -

Identifier Source: org_study_id