Alzheimer DiseasePhase 1NCT05016427
A Study of Possibility of Using Regulatory T Cells(VT301) for Treatment of Alzheimer's Disease
A Phase 1 study testing VT301 in people with Alzheimer Disease, run by VTBIO Co. LTD at 1 site in South Korea.
Trial summary
The overall study methods are as follows. [Clinical Trials Schedules] The study consists of a screening period(Visit 1) of up to 30 to 50 days, blood collection visits(Visit 2) for IP generation and administration visits for IP administration(Visit 3), with a follow-up(FU) period of 90 days(Visit 4~7). Read more about this study
- Who is it for?
- Ages 50 to 79 years
- Conditions the study is for
- Alzheimer Disease
- What is being tested?
- VT301
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteSouth Korea
- Study dates
- 1 Nov 2020to 1 Apr 2022
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 50 to 79 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female 79≥ aged ≥50 years at the time of signing Informed Consent Form.
- Patients (or their legally acceptable representative) can understand and provide informed consent to participate in the study.
- Diagnosis of mild-to-moderate AD according to National Institute of Aging and Alzheimer Association (NIA-AA) diagnostic guidelines.
- Mild-to-moderate AD with MMSE ≥ 10 points and CDR Global Score (CDR-GS) of 0.5 to 2.0 points at Screening.
- Have ≥1 identified adult caregiver (study partner) who is able to read, understand, and speak the designated language at the study site; either lives with the subject or sees the subject for ≥8 hours/week; and agrees to accompany the subject to each study visit and participate in clinical assessments.
Who cannot join
- A medical history of any of the following:
- Stroke, transient ischemic attack (TIA), or unexpected loss of consciousness within one year at Screening.
- Clinically significant cerebral hemorrhage, bleeding lesion, or cerebrovascular abnormality.
- Malignant tumor within 5 years at Screening (no time limit for stable non-metastatic prostate cancer or completely resected non-melanoma skin cancer of 6 months or longer).
- Unable to perform MRI tests by enthesis/transplantation of metallic substances (metallic bone fixings, heart devices, etc.) in the body.
- Allergic or hypersensitive to the treatment of regulatory T cell components.
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- Patients who have any of the following accompanying diseases/symptoms:
- Medical conditions or neurological/neural degenerative disease (excluding AD) considered to cause cognitive impairment or to affect the evaluation of clinical trials (discontinuation, nonconformity, interference, etc.) in the judgment of the investigator.
- History of clinically significant gastrointestinal, endocrine, inflammatory or cardiovascular disease that is not controlled by drug/non-drug treatment.
- History of mental illness (e.g., schizophrenia, major depression disorder, bipolar disorder, delirium, etc.) that is considered to affect participation in clinical trials under the judgment of the investigator.
- History of alcohol or drug abuse or dependence (except caffeine or nicotine).
- Vision, hearing, or mobility (behavior) has deteriorated to a degree that interferes with or is unable to perform clinical trial procedures.
- Hypersensitive to bee venom.
- High blood pressure who are not controlled by drug/non-drug treatment (SBP > 165 mmHg or DBP > 96 mmHg).
- Hypersensitive to gentamycin.
- Patients who have any of the following abnormal lab values at Screening:
- Urine Drug Screening Test: positive.
- Hepatitis B virus surface Antigen (HBsAg): positive.
- Anti-Hepatitis C Virus (Anti-HCV): positive.
- Anti- Human Immunodeficiency Virus (Anti-HIV): positive.
- Sensitization test for bee venom: positive.
- Serious renal dysfunction: Serum Creatinine ≥ 1.7 mg/dL.
- Clinically significant hepatic dysfunction (one or more of the following):.
- Serum Alanine Transaminase (ALT) ≥ 2 x upper limit of normal (ULN)
- Serum Aspartate Transaminase (AST) ≥ 2 x ULN
- Serum Bilirubin ≥ organ ULN x 2
- The person with the following drug or need to be administered during the clinical trial period:
- Acetylcholine Esterase(AChE) inhibitor, N-methyl-D-aspartate(NMDA) receptor antagonist, or a co-administration of these two drugs (Except if the dose was started more than 90 days before the date of the acquisition of the consent form and can be maintained reliably during administration and clinical trials without changing the dose for more than 60 days).
- However, the drug corresponding to (a) shall not be duplicated in (b).
- Drugs affecting the central nervous system (However, the dose was administered stably 30 days prior to the date of the acquisition of the consent form, except if it can be maintained during the clinical trial period).
- Drugs that are not properly administered during the clinical trial period as determined by the other investigator's.
- The person with the following one or more applicable:
- A female subject who is pregnant or breast-feeding.
- Fertile female@ subjects who have plans for pregnancy or do not use effective contraception method# during clinical trials period.
- A male subject who does not undergo surgical sterilization procedures or surgery without effective contraception method# (with a Fertile female@ partner).
- A fertile women is a women whose menopause has not occurred since the first menstrual and who has not undergone surgical sterilization procedures or surgery. Non-fertile women are defined as having one or more of the following:
- 12 months of natural menopause.
- For natural menopause for 6 months, the concentration of hCG(human Chorionic Gonadotropin) in the blood is 0 to 5 mIU/mL.
- In case of surgical sterilization procedures or surgery (bilateral ovariectomy, bilateral tubal ligation, etc.)
- Effective contraception methods recognized in this clinical trial are as follows:
- Combining either hormonal contraceptives(subdermal implant agents, injections, oral contraceptive, etc.) or spermicide with physical barrier method(condom, contraceptive vaginal diaphragm, vaginal sponge, cervical cap).
- Transplantation of intrauterine device(IUD) or intrauterine system(copper-loop, hormone-containing intrauterine system).
- Both male(condom) and female(contraceptive vaginal diaphragm, vaginal sponge, or cervical cap) use physical barrier method.
- surgical sterilization procedures or surgery (bilateral ovariectomy, bilateral tubal ligation, etc.)
- A person who has blood donation within 30 days of the date of the acquisition of the consent form.
- A person who has participated in another clinical trial within 60 days from the time of the acquisition of the consent form and has been given a investigational product(IP) or applied a clinical trial medical instrument.
- Other persons who are not qualified to participate in clinical trials under the judgment of the investigator's.
Trial locations and contacts
1 site in 1 country.
South Korea1 site, 1 recruiting
- Recruiting
Vtbio Co., Ltd
Seoul
hansaem Son, +82-2-553-9777, hsson@vtbio.co.kr
dongyoung Lee
About this study
Official title: A Phase 1 Open-Label Dose-Escalating Study to Determine the Safety, and Tolerability of VT301 in Subjects With Mild-to-Moderate Alzheimer's Disease
The overall study methods are as follows.
[Clinical Trials Schedules] The study consists of a screening period(Visit 1) of up to 30 to 50 days, blood collection visits(Visit 2) for IP generation and administration visits for IP administration(Visit 3), with a follow-up(FU) period of 90 days(Visit 4~7). During the Follow-up(FU) Period, subjects will visit 4 times for safety, tolerability and efficacy evaluation, with 90 Day being the End of Study(EOS) Visit.
[Subject screening and blood collection for IP generation] During Screening Period, subjects will be informed about the study and asked if they want to participate. The subjects and representatives and the caregiver/study partner will be asked to sign consent forms before any study-specific procedures are performed. Screening procedures will be performed to assess whether the subject is eligible to participate in the study. A minimum of approximately 200 mL of the subject's blood will be collected ≥30 days before Baseline and shipped to the IP Manufacturing Agency for generation of the IP. Subjects are required to refrain from consuming alcohol ≥3 days before any blood samples for IP generation are collected. If required (e.g. due to contamination), additional blood samples for IP generation may be collected during an unscheduled visit.
- Conditions
- Alzheimer Disease
- Being tested
- VT301
- Medical subject headings
- Alzheimer Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VT301Biological
VT301 is off-white suspension of regulatory T cells (Tregs) (1.7x10^5 cells/kg±15%) for injection diluted with sterile saline solution and supplied in clear, colorless, polypropylene vials.
| Study group | Role | What they receive |
|---|---|---|
| VT301(low dose) | Experimental | VT301 low dose: 8.5x10^4 cells/kg Receives: Biological: VT301 |
| VT301(high dose) | Experimental | VT301 high dose: 1.7x10^5 cells/kg Receives: Biological: VT301 |
What is being measured
The primary outcome is the main question the study is designed to answer.
All Adverse Events(AE) that occurred from the time of acquisition of consent of the subjects to the time of EOS(End of Study) shall be collected.
The collected AE (or ADR) should be monitored until possible recovery (or the investigator is determined to be normalized) or until the EOS can be determined to be meaningless for further monitoring.
Measured: Change from Baseline AE at 3 months
Number of subjects with abnormal clinical Physical examination
The number of subjects with normal and abnormal Physical examination findings will be summarized for each treatment group at each time point. Clinical significance was determined by the investigator.
Measured: Change from Baseline Physical examination at 3 months
Number of subjects With Clinically Significant Abnormalities in 12-lead Electrocardiogram
The number of subjects with normal and abnormal ECG findings will be summarized for each treatment group at each time point. Clinical significance was determined by the investigator.
ECG measures PR interval (ms), QRS interval, QT interval(ms), QTc interval (ms), and heart rate(bpm) for each treatment group at each time point.
Measured: Change from Baseline 12-lead Electrocardiogram at 3 months
Number of subjects with abnormal clinical Laboratory Tests
The number of subjects with normal and abnormal Laboratory Tests findings will be summarized for each treatment group at each time point. Clinical significance was determined by the investigator.
Blood and urine samples will be collected for the assessment of following clinical Laboratory Tests
Measured: Change from Baseline clinical Laboratory Tests at 3 months
Number of subjects with abnormal vital signs
Vital signs, including height (only assessed at Screening), weight, systolic and diastolic blood pressure, heart rate, and body temperature, will be measured after the subject has been in a sitting position for 5 minutes.
Measured: Change from Baseline vital signs at 3 months
Change from Screening "Questionnaire: Columbia Suicide Severity Rating Scale(C-SSRS)" at 90 days
C-SSRS will be assessed for the risk of suicide by an interview with the subject.
Measured: Change from Baseline C-SSRS at 3 months
Secondary outcomes (4)
Change from Baseline "Questionnaire: Alzheimer's Disease Assessment Scale-Cognitive Subscale-13 task(ADAS-Cog-13)" at 90 days
ADAS-Cog-13 will be assessed for cognitive evaluation by interviews with the subject and caregiver/study partner.
Measured: Change from Baseline ADAS-Cog-13 at 3 months
Change from Baseline "Questionnaire: Alzheimer's Disease Cooperative Study-Activities of Daily Living(ADCS-ADL)" at 90 days
ADCS-ADL will be assessed by caregiver/study partner interview regarding activities of daily living of the subject.
Measured: Change from Baseline ADCS-ADL at 3 months
Change from Screening "Questionnaire: Mini-Mental State Examination(MMSE)" at 90 days.
MMSE will be assessed for the evaluation of cognitive function and severity of the disease by an interview with the subject.
Measured: Change from Baseline MMSE at 3 months
Change from Screening "Questionnaire: Clinical Dementia Rating(CDR)" at 90 days.
CDR will be assessed for severity of the disease evaluation by interviews with the subject and caregiver/study partner.
Measured: Change from Baseline CDR at 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Aug 2021
- Study start1 Nov 2020
- Primary completion1 Nov 2021 (estimated)
- Study completion1 Apr 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Alzheimer DiseaseStudies of the same condition, open and under way.
- 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
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- VTBIO Co. LTD
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05016427
- Sponsor's protocol ID
- GB301-A01
- Status last verified
- Aug 2021
- First submitted
- 25 Jul 2021
- Last updated
- 23 Aug 2021
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Yang H, Byun MS, Ha NY, Yang J, Park SY, Park JE, Yi D, Chang YT, Jung WS, Kim JY, Kim J, Lee DY, Bae H. A preclinical and phase I clinical study of ex vivo-expanded amyloid beta-specific human regulatory T cells in Alzheimer's disease. Biomed Pharmacother. 2024 Dec;181:117721. doi: 10.1016/j.biopha.2024.117721. Epub 2024 Dec 2.
PubMed 39626378
Study at a glance
From the registry record, last updated by the sponsor on 23 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Open-Label Dose-Escalating Study to Determine the Safety, and Tolerability of VT301 in Subjects With Mild-to-Moderate Alzheimer's Disease
- ClinicalTrials.gov ID
- NCT05016427
- Acronym / sponsor ID
- GB301-A01
- Conditions
- Alzheimer Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- VTBIO Co. LTDIndustry (pharma, biotech, devices)
- Study start
- 1 Nov 2020 (actual)
- Primary completion
- 1 Nov 2021 (estimated)
- Study completion
- 1 Apr 2022 (estimated)
- First posted
- 23 Aug 2021 (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 overall study methods are as follows.
[Clinical Trials Schedules] The study consists of a screening period(Visit 1) of up to 30 to 50 days, blood collection visits(Visit 2) for IP generation and administration visits for IP administration(Visit 3), with a follow-up(FU) period of 90 days(Visit 4~7). During the Follow-up(FU) Period, subjects will visit 4 times for safety, tolerability and efficacy evaluation, with 90 Day being the End of Study(EOS) Visit.
[Subject screening and blood collection for IP generation] During Screening Period, subjects will be informed about the study and asked if they want to participate. The subjects and representatives and the caregiver/study partner will be asked to sign consent forms before any study-specific procedures are performed. Screening procedures will be performed to assess whether the subject is eligible to participate in the study. A minimum of approximately 200 mL of the subject's blood will be collected ≥30 days before Baseline and shipped to the IP Manufacturing Agency for generation of the IP. Subjects are required to refrain from consuming alcohol ≥3 days before any blood samples for IP generation are collected. If required (e.g. due to contamination), additional blood samples for IP generation may be collected during an unscheduled visit.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| VT301(low dose) | Experimental | Biological: VT301VT301 low dose: 8.5x10^4 cells/kg |
| VT301(high dose) | Experimental | Biological: VT301VT301 high dose: 1.7x10^5 cells/kg |
Who may qualify
- Ages
- 50 to 79 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Male or female 79≥ aged ≥50 years at the time of signing Informed Consent Form.
- Patients (or their legally acceptable representative) can understand and provide informed consent to participate in the study.
- Diagnosis of mild-to-moderate AD according to National Institute of Aging and Alzheimer Association (NIA-AA) diagnostic guidelines.
- Mild-to-moderate AD with MMSE ≥ 10 points and CDR Global Score (CDR-GS) of 0.5 to 2.0 points at Screening.
- Have ≥1 identified adult caregiver (study partner) who is able to read, understand, and speak the designated language at the study site; either lives with the subject or sees the subject for ≥8 hours/week; and agrees to accompany the subject to each study visit and participate in clinical assessments.
Key exclusion criteria (47 listed)
- A medical history of any of the following:
- Patients who have any of the following accompanying diseases/symptoms:
- Patients who have any of the following abnormal lab values at Screening:
- The person with the following drug or need to be administered during the clinical trial period:
- The person with the following one or more applicable:
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 |
|---|---|---|
| VT301VT301 is off-white suspension of regulatory T cells (Tregs) (1.7x10^5 cells/kg±15%) for injection diluted with sterile saline solution and supplied in clear, colorless, polypropylene vials. | Biological | VT301(high dose), VT301(low dose) |
Primary outcome
All Adverse Events(AE) that occurred from the time of acquisition of consent of the subjects to the time of EOS(End of Study) shall be collected.
Time frame: Change from Baseline AE at 3 months
The collected AE (or ADR) should be monitored until possible recovery (or the investigator is determined to be normalized) or until the EOS can be determined to be meaningless for further monitoring.
Number of subjects with abnormal clinical Physical examination
Time frame: Change from Baseline Physical examination at 3 months
The number of subjects with normal and abnormal Physical examination findings will be summarized for each treatment group at each time point. Clinical significance was determined by the investigator.
Number of subjects With Clinically Significant Abnormalities in 12-lead Electrocardiogram
Time frame: Change from Baseline 12-lead Electrocardiogram at 3 months
The number of subjects with normal and abnormal ECG findings will be summarized for each treatment group at each time point. Clinical significance was determined by the investigator. ECG measures PR interval (ms), QRS interval, QT interval(ms), QTc interval (ms), and heart rate(bpm) for each treatment group at each time point.
Number of subjects with abnormal clinical Laboratory Tests
Time frame: Change from Baseline clinical Laboratory Tests at 3 months
The number of subjects with normal and abnormal Laboratory Tests findings will be summarized for each treatment group at each time point. Clinical significance was determined by the investigator. Blood and urine samples will be collected for the assessment of following clinical Laboratory Tests
Number of subjects with abnormal vital signs
Time frame: Change from Baseline vital signs at 3 months
Vital signs, including height (only assessed at Screening), weight, systolic and diastolic blood pressure, heart rate, and body temperature, will be measured after the subject has been in a sitting position for 5 minutes.
Change from Screening "Questionnaire: Columbia Suicide Severity Rating Scale(C-SSRS)" at 90 days
Time frame: Change from Baseline C-SSRS at 3 months
C-SSRS will be assessed for the risk of suicide by an interview with the subject.
4 secondary outcomes
Change from Baseline "Questionnaire: Alzheimer's Disease Assessment Scale-Cognitive Subscale-13 task(ADAS-Cog-13)" at 90 days
Time frame: Change from Baseline ADAS-Cog-13 at 3 months
Change from Baseline "Questionnaire: Alzheimer's Disease Cooperative Study-Activities of Daily Living(ADCS-ADL)" at 90 days
Time frame: Change from Baseline ADCS-ADL at 3 months
Change from Screening "Questionnaire: Mini-Mental State Examination(MMSE)" at 90 days.
Time frame: Change from Baseline MMSE at 3 months
Change from Screening "Questionnaire: Clinical Dementia Rating(CDR)" at 90 days.
Time frame: Change from Baseline CDR at 3 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Aug 2021.
- Derived from this study: PubMed 39626378Yang H, Byun MS, Ha NY, Yang J, Park SY, Park JE, Yi D, Chang YT, Jung WS, Kim JY, Kim J, Lee DY, Bae H. A preclinical and phase I clinical study of ex vivo-expanded amyloid beta-specific human regulatory T cells in Alzheimer's disease. Biomed Pharmacother. 2024 Dec;181:117721. doi: 10.1016/j.biopha.2024.117721. Epub 2024 Dec 2.
- 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.
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 VT301 in people with Alzheimer Disease, run by VTBIO Co. LTD 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 5 inclusion and 47 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: status not confirmed, as of the sponsor's last update on 23 Aug 2021. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
All Adverse Events(AE) that occurred from the time of acquisition of consent of the subjects to the time of EOS(End of Study) shall be collected. (time frame: Change from Baseline AE at 3 months). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05016427, which also lists 1 linked publication.
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 23 Aug 2021.