The Safety and The Efficacy Evaluation of ET-STEM in Patients With Frontotemporal Dementia

NCT05315661 | Active Not Recruiting Phase 1

• 1 other identifier: 2022-02-089
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

The primary purpose of this study is to evaluate the safety and the tolerability of 3 repeated doses of ET-STEM (Mesenchymal stem cells preconditioned with ethionamide) in patients with FTD.

Conditions

Frontotemporal Dementia

Outcome Measures

Primary Outcomes (2)

• To determine DLT (Dose limiting toxicity)

  incidence rate of DLT (Dose limiting toxicity)

  First 3-week cycle of treatment

• adverse events as assessed by CTCAE v5.0

  all potentially treated subjects to assess the safety

  up to 5years

Secondary Outcomes (6)

• ADAS-Cog 13 response rate

  Screening, after the first administration12weeks, 48weeks, 96weeks, 144weeks, 192weeks, 240weeks

• The Clinical Dementia Rating Sum of Boxes

  Screening, after the first administration12weeks, 48weeks, 96weeks, 144weeks, 192weeks, 240weeks

• Alzheimer's Disease Cooperative Study- instrumental items of the Activities of Daily Living Inventory

  the first administration12weeks, 48weeks, 96weeks, 144weeks, 192weeks, 240weeks

• Caregiver-administered Neuropsychiatric Inventory

  the first administration12weeks, 48weeks, 96weeks, 144weeks, 192weeks, 240weeks

• preliminary efficacy

  up to 12weeks

Study Arms (1)

Treatment Arm

EXPERIMENTAL

injected with 3x10\^7 cells/2mL of ET-STEM to intraventricular space via an Ommaya reservoir. repeated 3 times at 4 week intervals

Drug: ET-STEM

Interventions

ET-STEM DRUG

mesenchymal stem cells preconditioned with ethionamide

Treatment Arm

Eligibility Criteria

• Age: 40 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Korean male or female at 40-85 years of age
• Diagnosis of one of the 3 subtyes of FTD according to the diagnostic criteria for 3 subtypes of FTD
• ① Probable bvFTD (behavior variant FTD)
• ② svPPA (semantic variant primary progressive aphasia)
• ③ nfvPPA (nonfluent/agrammatic variant primary progressive aphasia)
• K-MMSE ≥ 10
• Subjects with trusted caregivers who regularly contact the subjects and can accompany the subjects when visiting the hospital.
• Negative result of amyloid PET imaging
• A subject who is informed of the clinical trial and signs a consent form (If unable to sign, a consent from a legally acceptable representative is required)

You may not qualify if:

• Subjects with dementia cause by other than FTD (i.e. infection of central nervous system, Creutzfeld-Jacob disease, severer head trauma, Huntington's disease, Parkinson's disease, Alzheimer's disease and vascular dementia)
• Subjects with psychological disorder. (i.e. depression, schizophrenia , bipolar disorder, etc) (except for subjects who were misdiagnosed with psychological disease due to the initial neuropsychiatric symptoms of FTD)
• Subjects with uncontrolled hypotension, hypertension, diabetes and thyroid disease.
• Subjects with a cancer (including brain tumor)
• Subjects with bleeding disorder
• Woman of childbearing age who refused to practice medically acceptable contraceptive method (post menopausal patient with no menstruation for at least 12 months is considered as infertile)
• Pregnant or lactating females
• History of stroke within 3 months prior to study enrollment
• Substance/alcohol abuse 1
• Contraindicated for any of the tests performed during the clinical trial period(for example, MRI, CT,PET)
• A subject in whom Ommaya reservoir insertion and general anesthesia are considered difficult
• Abnormal Laboratory findings at Screening
• Suspected active lung disease based on chest X-ray at Screening
• Positive hepatitis B nuclear antibody and hpatitis C antibody
• Subjects who the principal investigator considers inappropriate for participation in the study due to the possible harmful effect on the subjects,difficulty in study completion, or previous or current medical conditions that may disturb evaluation of study results

Sponsors & Collaborators

• Samsung Medical Center: lead

Study Sites (1)

Samsung Medical Center

Seoul, Gangnam-gu, 06351, South Korea

Location

MeSH Terms

Conditions

Frontotemporal Dementia

Condition Hierarchy (Ancestors)

Frontotemporal Lobar Degeneration; Dementia; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; TDP-43 Proteinopathies; Neurodegenerative Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases; Neurocognitive Disorders; Mental Disorders

Study Officials

• HeeJin Kim

  Samsung Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: assistant professor

Model Details: Single Group

Study Record Dates

• First Submitted: March 1, 2022
• First Posted: April 7, 2022
• Study Start: July 6, 2022
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: February 21, 2024

Record last verified: 2024-02

Data Sharing

• IPD Sharing: Will not share

Locations

KR (1)