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

Frontotemporal Dementia Clinical Trial

— FTD_ET-STEM  

Official title:

Clinical Assessment on the Safety and Potential Efficacy of Mesenchymal Stem Cells Preconditioned With Ethionamide (ET-STEM) in Patients With Frontotemporal Dementia (FTD)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05315661
• Other study ID #: 2022-02-089
• Status: Active, not recruiting
• Phase: Phase 1
• Start date: July 6, 2022
• Est. completion date: December 31, 2026

Study information

• Verified date: February 2024
• Source: Samsung Medical Center
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 12
• Est. completion date: December 31, 2026
• Est. primary completion date: December 31, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 40 Years to 85 Years

Eligibility

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

? Probable bvFTD (behavior variant FTD)

? svPPA (semantic variant primary progressive aphasia)

? 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.

Related Conditions & MeSH terms

• Aphasia, Primary Progressive
• Dementia
• Frontotemporal Dementia
• Pick Disease of the Brain

Intervention

Drug:
• ET-STEM
mesenchymal stem cells preconditioned with ethionamide

Locations

Country	Name	City	State
Korea, Republic of	Samsung Medical Center	Seoul	Gangnam-gu

Sponsors (1)

Lead Sponsor	Collaborator
Samsung Medical Center

Country where clinical trial is conducted

Korea, Republic of, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To determine DLT (Dose limiting toxicity)	incidence rate of DLT (Dose limiting toxicity)	First 3-week cycle of treatment
Primary	adverse events as assessed by CTCAE v5.0	all potentially treated subjects to assess the safety	up to 5years
Secondary	ADAS-Cog 13 response rate	response rate, no change or improvement on ADAS cog 13 score	Screening, after the first administration12weeks, 48weeks, 96weeks, 144weeks, 192weeks, 240weeks
Secondary	The Clinical Dementia Rating Sum of Boxes	Change from the baseline in CDR-SB, min 0, max 24, higher scores mean a worse outcome	Screening, after the first administration12weeks, 48weeks, 96weeks, 144weeks, 192weeks, 240weeks
Secondary	Alzheimer's Disease Cooperative Study- instrumental items of the Activities of Daily Living Inventory	Change from the baseline in ADCS-iADL, min 0, max78, higher scores mean a better outcome	the first administration12weeks, 48weeks, 96weeks, 144weeks, 192weeks, 240weeks
Secondary	Caregiver-administered Neuropsychiatric Inventory	Change from the baseline in CGA-NPI, min 0, max 144, higher scores mean a worse outcome	the first administration12weeks, 48weeks, 96weeks, 144weeks, 192weeks, 240weeks
Secondary	preliminary efficacy	Change from the baseline in CSF biomarkers	up to 12weeks
Secondary	K-MMSE	Korean Mini-Mental State Examination(MMSE), min 0, max 30, higher scores mean a better outcome	the first administration12weeks, 48weeks, 96weeks, 144weeks, 192weeks, 240weeks