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

NCT04228666 | Withdrawn Phase 1 DMC FDA Drug

• 1 other identifier: HBALZ01
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

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.

Conditions

Alzheimer Disease

Keywords

Alzheimer's disease; stem cells; adipose derived mesenchymal stem cells

Outcome Measures

Primary Outcomes (37)

• Glucose

  clinical lab evaluation of level of glucose in the blood (mg/dL)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• Calcium

  clinical lab evaluation of level of calcium in the blood (mg/dL)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• Albumin

  clinical lab evaluation of level of albumin in the blood (g/dL)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• Total Protein

  clinical lab evaluation of level of protein in the blood (g/dL)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• Sodium

  clinical lab evaluation of level of sodium in the blood (mol/L)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• Total carbon dioxide

  clinical lab evaluation of level of carbon dioxide in the blood (mmol/L)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• Potassium

  clinical lab evaluation of level of potassium in the blood (mmol/L)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• Chloride

  clinical lab evaluation of level of chloride in the blood (mmol/L)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• BUN

  clinical lab evaluation of level of BUN in the blood (mg/dL)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• Creatinine

  clinical lab evaluation of level of creatinine in the blood (mg/dL)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• Alkaline phosphatase

  clinical lab evaluation of level of alkaline phosphatase in the blood (IU/L)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• Alanine aminotransferase

  clinical lab evaluation of level of alanine aminotransferase in the blood (IU/L)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• Aspartate aminotransferase

  clinical lab evaluation of level of aspartate aminotransferase in the blood (IU/L)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• Total Bilirubin

  clinical lab evaluation of level of bilirubin in the blood (mg/dL)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• White blood cell

  clinical lab evaluation of level of white blood cells in the blood (x 10\^3/uL)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• Red blood cell

  clinical lab evaluation of level of red blood cells in the blood (x 10\^6/uL)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• Hemoglobin

  clinical lab evaluation of level of hemoglobin in the blood (g/dL)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• Hematocrit

  clinical lab evaluation of level of hematocrit in the blood (%)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• Mean corpuscular volume

  clinical lab evaluation of mean corpuscular volume in the blood (fL)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• Mean corpuscular hemoglobin

  clinical lab evaluation of mean corpuscular hemoglobin in the blood (pg)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• Mean corpuscular hemoglobin concentration

  clinical lab evaluation of level of hemoglobin concentration in the blood (g/dL)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• Red cell distribution width

  clinical lab evaluation of distribution width in the blood (%)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• Neutrophils

  clinical lab evaluation of neutrophils in the blood (%)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• Lymphs

  clinical lab evaluation of lymphocytes in the blood (%)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• Monocytes

  clinical lab evaluation of monocytes in the blood (%)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• Eos

  clinical lab evaluation of eosinophils in the blood (%)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• Basophils

  clinical lab evaluation of basophils in the blood (%)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• Absolute neutrophils

  clinical lab evaluation of absolute neutrophils in the blood (x 10\^3/uL)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• Absolute lymphs

  clinical lab evaluation of absolute lymphocytes in the blood (x 10\^3/uL)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• Absolute monocytes

  clinical lab evaluation of absolute monocytes in the blood (x 10\^3/uL)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• Absolute Eos

  clinical lab evaluation of absolute eosinophils in the blood (x 10\^3/uL)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• Absolute Basos

  clinical lab evaluation of absolute basophils in the blood (x 10\^3/uL)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• Immature granulocytes

  clinical lab evaluation of granulocytes in the blood (%)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• Absolute Immature granulocytes

  clinical lab evaluation of absolute immature granulocytes in the blood (x 10\^3/uL)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• Platelets

  clinical lab evaluation of platelets in the blood (x 10\^3/uL)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• Prothrombin time

  clinical lab evaluation of time for blood to coagulate (seconds)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

• INR

  clinical lab evaluation of international normalized ratio of blood coagulation (no unit)

  Screening, change from screening at week 0, week 4, week 8, week 13, week 26, week 52.

Secondary Outcomes (12)

• Tumor necrosis factor-alpha

  week 0, change from baseline at week 13, change from baseline at week 52

• Interleukin-1

  week 0, change from baseline at week 13, change from baseline at week 52

• Interleukin-6

  week 0, change from baseline at week 13, change from baseline at week 52

• C-reactive protein

  week 0, change from baseline at week 13, change from baseline at week 52

• Amyloid beta 40

  week 0, change from baseline at week 13, change from baseline at week 52

Study Arms (1)

HB-adMSCs

EXPERIMENTAL

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.

Biological: HB-adMSCs

Interventions

HB-adMSCs 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;

HB-adMSCs

Eligibility Criteria

• Age: 50 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• Informed consent signed by the subject
• Documented Amyloid PET Scan (images and report) positive to amyloid plaques deposits on the brain.
• 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).

You may not qualify if:

• Hospitalization or change of chronic concomitant medication within one month prior to screening.
• 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.
• Records of PET Scan negative to Amyloid plaques deposition in the brain.
• 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.
• Acute intercurrent infections such as Hepatitis C Virus (HCV), Hepatitis B Virus (HBV), Human Immunodeficiency Virus (HIV) or Syphilis.
• 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.
• Is unable or unwilling to comply with protocol follow-up requirements.
• Enrollment in another investigational study or intake of investigational drug within the previous 30 days.

Sponsors & Collaborators

• Hope Biosciences LLC: lead
• Hope Biosciences Research Foundation: collaborator

Study Sites (1)

Clinical Trial Network

Houston, Texas, 77074, United States

Location

MeSH Terms

Conditions

Alzheimer Disease

Condition Hierarchy (Ancestors)

Dementia; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Tauopathies; Neurodegenerative Diseases; Neurocognitive Disorders; Mental Disorders

Study Officials

• Djamchid Lotfi, MD

  Clinical Trial Network

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 3, 2020
• First Posted: January 14, 2020
• Study Start: March 1, 2020
• Primary Completion: February 1, 2022
• Study Completion: February 1, 2022
• Last Updated: September 29, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)