Frontotemporal DementiaPhase 1FTD_ET-STEMNCT05315661
The Safety and The Efficacy Evaluation of ET-STEM in Patients With Frontotemporal Dementia
A Phase 1 study testing ET-STEM in people with Frontotemporal Dementia, run by Samsung Medical Center at 1 site in South Korea.
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. Read more about this study
- Who is it for?
- Ages 40 to 85 years
- Conditions the study is for
- Frontotemporal Dementia
- What is being tested?
- ET-STEM
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteSouth Korea
- Study dates
- 6 Jul 2022to 31 Dec 2026
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 40 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- 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)
Who cannot join
- 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)
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- 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.
Trial locations and contacts
1 site in 1 country.
About this study
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)
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.
Read the 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
- Being tested
- ET-STEM
- Medical subject headings
- Frontotemporal Dementia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ET-STEMDrug
mesenchymal stem cells preconditioned with ethionamide
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Drug: ET-STEM |
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine DLT (Dose limiting toxicity)
incidence rate of DLT (Dose limiting toxicity)
Measured: First 3-week cycle of treatment
adverse events as assessed by CTCAE v5.0
all potentially treated subjects to assess the safety
Measured: up to 5years
Secondary outcomes (6)
ADAS-Cog 13 response rate
response rate, no change or improvement on ADAS cog 13 score
Measured: Screening, after the first administration12weeks, 48weeks, 96weeks, 144weeks, 192weeks, 240weeks
The Clinical Dementia Rating Sum of Boxes
Change from the baseline in CDR-SB, min 0, max 24, higher scores mean a worse outcome
Measured: Screening, after the first administration12weeks, 48weeks, 96weeks, 144weeks, 192weeks, 240weeks
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
Measured: the first administration12weeks, 48weeks, 96weeks, 144weeks, 192weeks, 240weeks
Caregiver-administered Neuropsychiatric Inventory
Change from the baseline in CGA-NPI, min 0, max 144, higher scores mean a worse outcome
Measured: the first administration12weeks, 48weeks, 96weeks, 144weeks, 192weeks, 240weeks
preliminary efficacy
Change from the baseline in CSF biomarkers
Measured: up to 12weeks
K-MMSE
Korean Mini-Mental State Examination(MMSE), min 0, max 30, higher scores mean a better outcome
Measured: the first administration12weeks, 48weeks, 96weeks, 144weeks, 192weeks, 240weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Apr 2022
- Study start6 Jul 2022
- Primary completion31 Dec 2026 (estimated)
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Frontotemporal DementiaStudies of the same condition, open and under way.
- More trials run by Samsung Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Single Group
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Samsung Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Hee Jin Kim, Samsung Medical Center
- Investigators
- HeeJin Kim, Principal investigator, Samsung Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05315661
- Sponsor's protocol ID
- 2022-02-089
- Acronym
- FTD_ET-STEM
- Status last verified
- Feb 2024
- First submitted
- 1 Mar 2022
- Last updated
- 21 Feb 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT05315661
- Acronym / sponsor ID
- FTD_ET-STEM / 2022-02-089
- Conditions
- Frontotemporal Dementia
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Samsung Medical CenterUniversity, hospital or other
- Study start
- 6 Jul 2022 (actual)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 7 Apr 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Single Group
| Arm | Type | Receives |
|---|---|---|
| Treatment Arm | Experimental | Drug: ET-STEMinjected with 3x10^7 cells/2mL of ET-STEM to intraventricular space via an Ommaya reservoir. repeated 3 times at 4 week intervals |
Who may qualify
- Ages
- 40 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- 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)
Key exclusion criteria (16 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ET-STEMmesenchymal stem cells preconditioned with ethionamide | Drug | Treatment Arm |
Primary outcome
To determine DLT (Dose limiting toxicity)
Time frame: First 3-week cycle of treatment
incidence rate of DLT (Dose limiting toxicity)
adverse events as assessed by CTCAE v5.0
Time frame: up to 5years
all potentially treated subjects to assess the safety
6 secondary outcomes
ADAS-Cog 13 response rate
Time frame: Screening, after the first administration12weeks, 48weeks, 96weeks, 144weeks, 192weeks, 240weeks
The Clinical Dementia Rating Sum of Boxes
Time frame: Screening, after the first administration12weeks, 48weeks, 96weeks, 144weeks, 192weeks, 240weeks
Alzheimer's Disease Cooperative Study- instrumental items of the Activities of Daily Living Inventory
Time frame: the first administration12weeks, 48weeks, 96weeks, 144weeks, 192weeks, 240weeks
Caregiver-administered Neuropsychiatric Inventory
Time frame: the first administration12weeks, 48weeks, 96weeks, 144weeks, 192weeks, 240weeks
preliminary efficacy
Time frame: up to 12weeks
K-MMSE
Time frame: the first administration12weeks, 48weeks, 96weeks, 144weeks, 192weeks, 240weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Feb 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
HeeJin Kim principal investigator
Samsung Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ET-STEM in people with Frontotemporal Dementia, run by Samsung Medical Center at 1 site in South Korea.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 21 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To determine DLT (Dose limiting toxicity) (time frame: First 3-week cycle of treatment). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Feb 2024.