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

NCT ID: NCT05315661

Last Updated: 2024-02-21

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE1
• Total Enrollment: 12 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-07-06
• Study Completion Date: 2026-12-31

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.

Detailed Description

Subjects with FTD, who signed the informed consent form and meet the eligibility criteria will undergo Ommaya reservoir insertion. 2 weeks after Ommaya reservoir insertion, the subjects will be injected with 3x10^7 cells/2mL of ET-STEM to intraventricular space via an Ommaya reservoir. The injection will be repeated 3 times at 4 week intervals. The subjects will be hospitalized for 24 hours and observed for acute adverse events. 4 weeks after the 3rd injection, safety and potential efficacy will be assessed.

Conditions

Frontotemporal Dementia

Study Design

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

Study Groups

Treatment Arm

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

Group Type: EXPERIMENTAL

ET-STEM

Intervention Type: DRUG

mesenchymal stem cells preconditioned with ethionamide

Interventions

ET-STEM

mesenchymal stem cells preconditioned with ethionamide

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Korean male or female at 40-85 years of age

2. 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)

3. K-MMSE ≥ 10

4. Subjects with trusted caregivers who regularly contact the subjects and can accompany the subjects when visiting the hospital.

5. Negative result of amyloid PET imaging

6. 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)

Exclusion Criteria

1. 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)

2. 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)

3. Subjects with uncontrolled hypotension, hypertension, diabetes and thyroid disease.

4. Subjects with a cancer (including brain tumor)

5. Subjects with bleeding disorder

6. 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)

7. Pregnant or lactating females

8. History of stroke within 3 months prior to study enrollment

9. Substance/alcohol abuse 1

10. Contraindicated for any of the tests performed during the clinical trial period(for example, MRI, CT,PET)

11. A subject in whom Ommaya reservoir insertion and general anesthesia are considered difficult

12. Abnormal Laboratory findings at Screening

13. Suspected active lung disease based on chest X-ray at Screening

14. Positive hepatitis B nuclear antibody and hpatitis C antibody

15. 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

16. Subjects who the principal investigator considers impossible to comply with clinical research procedures.

• Minimum Eligible Age: 40 Years
• Maximum Eligible Age: 85 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Samsung Medical Center

OTHER

Sponsor Role: lead

Responsible Party

Hee Jin Kim

assistant professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

HeeJin Kim

Role: PRINCIPAL_INVESTIGATOR

Samsung Medical Center

Locations

Samsung Medical Center

Seoul, Gangnam-gu, South Korea

Countries

South Korea

Other Identifiers

2022-02-089

Identifier Type: -

Identifier Source: org_study_id