Stem Cell Therapy for Early Alzheimer's Disease

NCT06775964 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: HSC-MS-24-0516
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

The goal of this clinical trial is to learn if stem cell therapy works to treat brain inflammation in adults. Inflammation in the brain may be involved in adults who have memory or thinking problems. The stem cells will be taken from participant's fat samples, processed and given back to participants, so they are their own donor. The main questions this trial aims to answer are:

• Does stem cell therapy reduce inflammation in the brain?
• Does stem cell therapy improve brain activity?
• Does stem cell therapy slow down progression to Alzheimer's disease? Participants will:
• Have a small fat biopsy taken at a doctor's office to process stem cells
• Receive 4 infusions of stem cells, through a vein in the arm over 12 weeks
• Visit the clinic every 2-4 weeks for the first 4 months and then every 1-2 months for 8 months for checkups and tests

Conditions

Cognitive Dysfunction

Keywords

Mild Cognitive Impairment; Cognitive Decline; Alzheimer's disease

Outcome Measures

Primary Outcomes (2)

• Change in TSPO levels, measured by the PET scan, from baseline to midpoint and baseline to end of study

  Level of TSPO PET tracer will be measured for each PET scan. TSPO positivity, which is a marker for activated microglia, correlates with neuroinflammation

  Baseline, midpoint (169 days from 1st infusion)

• Inflammatory cytokines in CSF following adMSC therapy

  Inflammatory cytokine panel in CSF will be measured using commercially available immunosorbent assays to determine potential treatment effects. Aggregate values and percentages will be reported.

  Baseline, midpoint (169 days from 1st infusion)

Secondary Outcomes (11)

• Number of participants with treatment-related adverse events

  Baseline - End of Study (337 days from 1st infusion)

• Changes in neurofilament light chain (Nf-L) in CSF following adMSC therapy

  Baseline, midpoint (169 days from 1st infusion)

• Changes in Glial fibrillary acidic protein (GFAP) in CSF following adMSC therapy

  Baseline, midpoint (169 days from 1st infusion)

• Changes in Total Tau/phosphor-Tau ratios in CSF following adMSC therapy

  Baseline, midpoint (169 days from 1st infusion)

• Changes in cerebral metabolism activity via FDG PET imaging from image baseline to midpoint

  Baseline, midpoint (169 days from 1st infusion)

Study Arms (1)

adMSC

EXPERIMENTAL

IV-infusions of autologous, adipose-derived, Mesenchymal Stem Cells (adMSCs)

Biological: adMSC

Interventions

adMSC BIOLOGICAL

IV-infusion of autologous, adipose-derived, Mesenchymal Stem Cells (adMSCs), of approximately 2x10(8) adMSCs in 250mL saline.

adMSC

Eligibility Criteria

• Age: 60 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Has signed an informed consent form before any assessment is performed as part of the study.
• Be male or female between 60 and 80 years old.
• Subject has been or is in process of being clinically diagnosed with late pre-symptomatic or mild cognitive impairment (MCI) due to AD (prodromal AD).
• Mini-Mental State Examination (MMSE) score of ≥ 22
• Has an MRI to evaluate AD pathology (may use previous if within 6mo.)
• Has APOE status to evaluate AD pathology (may use previous result)
• Proficiency in English is required because cognitive tests are administered in English only.
• Has evidence of brain amyloidosis via PET Scan or Aβ42/40 ratios in CSF.
• Has evidence of peripheral inflammatory profile based on CRP (≥ 8 mg/L), IL-6 (≥ 3.1 pg/mL), TNF-α (10 pg/mL), or erythrocyte sedimentation rate (ESR) (≥20 mm/h) in blood assays.
• Is in the opinion of the Investigator, in good general medical health based upon medical history, physical examination, laboratory tests, vital signs and EKG.

You may not qualify if:

• Current medical or neurological condition that might impact cognition or performance on cognitive assessments. (e.g., traumatic brain injury (TBI), Parkinson's disease (PD), multiple sclerosis, etc.)
• Inability or unwillingness of patient to undergo neuropsychological testing.
• Advanced, severe, progressive or unstable disease that may interfere with the safety, tolerability and study assessments, or put the subject at special risk. (e.g., significant cardiac disease, severe renal impairment, severe hepatic impairment, autoimmune disease, etc.)
• History of malignancy of any organ system within the past 60 months, that in the opinion of the investigator would impede evaluation or interpretation of subject safety or study results.
• Females of childbearing potential must not be pregnant.
• Inability or unwillingness to undergo PET Scans.
• Inability or unwillingness to undergo MRI Scans.
• Positive blood test for either HIV, Hepatitis B, Hepatitis C or Syphilis
• Positive for TSPO SNP rs6971
• Inability or unwillingness to undergo Lumbar Punctures.
• Inability or unwillingness to undergo infusions.
• Any condition, which in the opinion of the investigator, would put the subject at undue risk or would interfere with evaluation of the investigational product or interpretation of subject safety or study results.

Sponsors & Collaborators

• Paul E Schulz: lead
• Weston Brain Institute: collaborator

Study Sites (1)

The University of Texas Health Science Center at Houston (UTHealth)

Houston, Texas, 77054, United States

RECRUITING

MeSH Terms

Conditions

Cognitive Dysfunction; Alzheimer Disease

Condition Hierarchy (Ancestors)

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

Study Officials

• Paul E Schulz, MD

  The University of Texas Health Science Center, Houston

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Harshali Patel

CONTACT

713-486-0531; Harshali.Patel@uth.tmc.edu

Javier Ortiz, PhD

CONTACT

713-486-0505; Guadalupe.J.Ortiz@uth.tmc.edu

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Principal Investigator, Director, Memory Disorders and Dementia Clinic, Professor of Neurology, Professor of Neurodegenerative Disorders

Model Details: This is a Phase 1b/2a open label study to assess the safety and tolerability, as well as reduction of neuroinflammation after four IV-infusions of autologous, adipose-derived, Mesenchymal Stem Cells (adMSCs) over a 13-week treatment period in 12 subjects who are clinically diagnosed with late pre-symptomatic or prodromal AD, exhibit an Alzheimer's pathology and peripheral inflammatory profile.

Study Record Dates

• First Submitted: January 9, 2025
• First Posted: January 15, 2025
• Study Start: March 11, 2026
• Primary Completion (Estimated): January 1, 2027
• Study Completion (Estimated): January 1, 2028
• Last Updated: April 20, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)