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

Primary Purpose

Frontotemporal Dementia

Status

Recruiting

Phase

Phase 1

Locations

Korea, Republic of

Study Type

Interventional

Intervention

ET-STEM

About this trial

This is an interventional treatment trial for Frontotemporal Dementia

Eligibility Criteria

40 Years - 85 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

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)

Exclusion Criteria:

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

Sites / Locations

Samsung Medical CenterRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Treatment Arm

Arm Description

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

Outcomes

Primary Outcome Measures

To determine DLT (Dose limiting toxicity)

incidence rate of DLT (Dose limiting toxicity)

adverse events as assessed by CTCAE v5.0

all potentially treated subjects to assess the safety

Secondary Outcome Measures

ADAS-Cog 13 response rate

response rate, no change or improvement on ADAS cog 13 score

The Clinical Dementia Rating Sum of Boxes

Change from the baseline in CDR-SB, min 0, max 24, higher scores mean a worse outcome

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

Caregiver-administered Neuropsychiatric Inventory

Change from the baseline in CGA-NPI, min 0, max 144, higher scores mean a worse outcome

preliminary efficacy

Change from the baseline in CSF biomarkers

K-MMSE

Korean Mini-Mental State Examination(MMSE), min 0, max 30, higher scores mean a better outcome

Full Information

NCT ID

NCT05315661

First Posted

March 1, 2022

Last Updated

October 24, 2022

Sponsor

Samsung Medical Center

1. Study Identification

Unique Protocol Identification Number

NCT05315661

Brief Title

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

Acronym

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)

Study Type

Interventional

2. Study Status

Record Verification Date

October 2022

Overall Recruitment Status

Recruiting

Study Start Date

July 6, 2022 (Actual)

Primary Completion Date

December 31, 2026 (Anticipated)

Study Completion Date

December 31, 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Samsung Medical Center

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Frontotemporal Dementia

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Model Description

Single Group

Masking

None (Open Label)

Allocation

N/A

Enrollment

12 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Treatment Arm

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

ET-STEM

Intervention Description

mesenchymal stem cells preconditioned with ethionamide

Primary Outcome Measure Information:

Title

To determine DLT (Dose limiting toxicity)

Description

incidence rate of DLT (Dose limiting toxicity)

Time Frame

First 3-week cycle of treatment

Title

adverse events as assessed by CTCAE v5.0

Description

all potentially treated subjects to assess the safety

Time Frame

up to 5years

Secondary Outcome Measure Information:

Title

ADAS-Cog 13 response rate

Description

response rate, no change or improvement on ADAS cog 13 score

Time Frame

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

Title

The Clinical Dementia Rating Sum of Boxes

Description

Change from the baseline in CDR-SB, min 0, max 24, higher scores mean a worse outcome

Time Frame

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

Title

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

Description

Change from the baseline in ADCS-iADL, min 0, max78, higher scores mean a better outcome

Time Frame

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

Title

Caregiver-administered Neuropsychiatric Inventory

Description

Change from the baseline in CGA-NPI, min 0, max 144, higher scores mean a worse outcome

Time Frame

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

Title

preliminary efficacy

Description

Change from the baseline in CSF biomarkers

Time Frame

up to 12weeks

Title

K-MMSE

Description

Korean Mini-Mental State Examination(MMSE), min 0, max 30, higher scores mean a better outcome

Time Frame

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

10. Eligibility

Sex

All

Minimum Age & Unit of Time

40 Years

Maximum Age & Unit of Time

85 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 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) Exclusion Criteria: 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 Subjects who the principal investigator considers impossible to comply with clinical research procedures.

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

HeeJin Kim

Phone

82-10-8654-7347

evekhj@gmail.com

First Name & Middle Initial & Last Name or Official Title & Degree

HeeJin Kim

Phone

82-10-8654-7347

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

HeeJin Kim

Organizational Affiliation

Samsung Medical Center

Official's Role

Principal Investigator

Facility Information:

Facility Name

Samsung Medical Center

City

Seoul

State/Province

Gangnam-gu

ZIP/Postal Code

06351

Country

Korea, Republic of

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Heejin Kim

Phone

82-10-8654-7347

evekhj@gmail.com

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

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

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