Synovial SarcomaPhase 1/2NCT03250325
Study of TBI-1301 (NY-ESO-1 T Cell Receptor Gene Transduced Autologous T Lymphocytes) in Patients with Synovial Sarcoma
A Phase 1/2 study testing TBI-1301 and Cyclophosphamide in people with Synovial Sarcoma, run by Takara Bio Inc. at 5 sites in Japan.
Trial summary
The purpose of this study is to evaluate the safety and the efficacy of TBI-1301 for NY-ESO-1 expressing synovial sarcoma when administered following cyclophosphamide pre-treatment. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Synovial Sarcoma
- What is being tested?
- TBI-1301 and Cyclophosphamide
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 8 participants
- Where
- 5 sitesJapan
- Study dates
- 20 Sep 2017to 9 Mar 2022
- Phase
- Phase 1/2
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Histologically confirmed synovial sarcoma
- Surgically unresectable tumor
- Progressing or recurrent synovial sarcoma which has been treated with 1-4 regimens of systemic chemotherapies including anthracycline
- HLA-A*02:01 or HLA-A*02:06 positive
- Tumor that express NY-ESO-1 by immunohistochemistry
- ≥ 18 years of age
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- Measurable lesions that are evaluable by the RECIST ver1.1
- ECOG Performance Status of 0, 1 or 2
- No treatment such as chemotherapy and be expected to recover fully from the previous treatment at the time of the lymphocytes collection for manufacturing
- Life expectancy ≥ 16 weeks after consent
- No severe damage on the major organs (bone marrow, heart, lung, liver, kidney, etc) and meet the following lab value criteria; Total bilirubin ≤ 1.5 x upper limit of normal (ULN); AST(GOT), ALT(GPT) < 3.0 x ULN; Creatinine < 1.5 x ULN; 2,500/μL < WBC ≤ULN; Hemoglobin ≥ 8.0g/dL; Platelets ≥ 75,000/μL
- Patients must be able to understand the study contents and to give a written consent at his/her free will. Additionally, if patients are below 20 years of age, proxies must be able to give a written consent.
Who cannot join
- Patients with the following conditions are excluded from the study; Unstable angina, cardiac infarction, or heart failure; Uncontrolled diabetes or hypertension; Active infection; Obvious interstitial pneumonia or lung fibrosis by chest X-ray; Active autoimmune disease requiring steroids or immunosuppressive therapy.
- Active metastatic tumor cell invasion into CNS
- Active multiple cancer
- Positive for HBs antigen or HBV-DNA observed in serum
- Positive for HCV antibody and HCV-RNA observed in serum
- Positive for antibodies against HIV or HTLV-1
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- Left Ventricular Ejection Fraction (LVEF) ≤ 50%
- History of serious hypersensitivity reactions to bovine or murine derived substances.
- History of hypersensitivity reaction to ingredients or excipients of investigational drugs used in this study
- History of hypersensitivity reaction to antibiotics used in manufacturing for the investigational drug used in this study.
- Pregnant females, lactating females (except when they cease and do not resume lactation) or female and male patients who cannot agree to practice the adequate birth control from the consent to 6 months after infusion of the investigational drug.
- Clinically significant systemic illness that in the judgment of the PI or sub-investigator would compromise the patient's ability to tolerate protocol therapy or significantly increase the risk of complications.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
Japan5 sites
National Cancer Center Hospital
Chuo-ku, Tokyo, 104-0045
Kyushu University Hospital
Fukuoka, 812-8582
National Hospital Organization Osaka National Hospital
Osaka, Osaka, 540-0006
Sapporo Medical University Hospital
Sapporo, Hokkaido, 060-8543
Mie University Hospital
Tsu, Mie-ken, 514-8507
About this study
Official title: Multi-center Phase I/II Study of NY-ESO-1 T Cell Receptor Gene Transferred T Lymphocytes in Patients with Synovial Sarcoma
The purpose of this study is to evaluate the safety and the efficacy of TBI-1301 for NY-ESO-1 expressing synovial sarcoma when administered following cyclophosphamide pre-treatment.
Read the detailed description
Following pre-treatment with cyclophosphamide, NY-ESO-1-specific T cell receptor (TCR) gene transduced T lymphocytes are transferred to human leukocyte antigen (HLA)-A*02:01 or HLA-A*02:06 positive patients with synovial sarcoma expressing NY-ESO-1, which are surgically unresectable and refractory to anthracycline therapy. The primary objective is to evaluate the safety in the phase 1 and the efficacy in the phase 2.
- Conditions
- Synovial Sarcoma
- Being tested
- TBI-1301, Cyclophosphamide
- Keywords
- Adoptive cell transfer, Cell therapy, Immunotherapy, NY-ESO-1, T cell receptor gene therapy, Synovial sarcoma
- Medical subject headings
- Sarcoma, Synovial
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TBI-1301Biological
Split dose of TBI-1301 is administered intravenously for 2 days following cyclophosphamide pre-treatment.
CyclophosphamideDrug
Cyclophosphamide (750mg/m2/day x 2 days Intravenous (IV)) is administered as pre-treatment medication of TBI-1301.
| Study group | Role | What they receive |
|---|---|---|
| Split dose of 5x10^9 TBI-1301 | Experimental | Split dose of 5x10^9 TBI-1301 will be administered intravenously for 2 days following cyclophosphamide pre-treatment 750 mg/m2/d for 2 days. Receives: Biological: TBI-1301; Drug: Cyclophosphamide |
What is being measured
The primary outcome is the main question the study is designed to answer.
(Phase I) Adverse event, mortality, severe adverse event, discontinuation due to adverse event, laboratory test values
Confirm the toxicity profile, which is measured by the degree of grade and seriousness, duration, causality, classification, etc. of the adverse events.
Measured: 52 weeks
(Phase I) Appearance of replication competent retrovirus (RCR) by PCR
Confirm that no replication competent retrovirus observed.
Measured: 52 weeks
(Phase I) Appearance of clonality by linear amplification mediated (LAM)-PCR
Confirm that no clonality is observed.
Measured: 52 weeks
(Phase I) Blood kinetics of TBI-1301 by realtime-PCR
Evaluate persistence and expansion of transferred TBI-1301.
Measured: 52 weeks
(Phase II) Overall response rate
Evaluate response rate by measuring response using RECIST v1.1 and irRECIST
Measured: 52 weeks
Secondary outcomes (8)
(Phase I) Objective response rate
Evaluate response rate by measuring response using RECIST v1.1 and irRECIST
Measured: 52 weeks
(Phase I/II) Progression free rate
Evaluate progression free rate by measuring response using RECIST v1.1 and irRECIST
Measured: 12 weeks
(Phase I/II) Progression free survival
Evaluate progression free survival
Measured: 52 weeks
(Phase I/II) Overall survival
Evaluate overall survival
Measured: 52 weeks
(Phase II) Adverse event, mortality, severe adverse event, discontinuation due to adverse event, laboratory test values
Confirm the toxicity profile, which is measured by the degree of grade and seriousness, duration, causality, classification, etc. of the adverse events.
Measured: 52 weeks
(Phase II) Appearance of RCR
Confirm that no replication competent retrovirus observed.
Measured: 52 weeks
(Phase II) Appearance of clonality (LAM-PCR)
Confirm that no clonality is observed.
Measured: 52 weeks
(Phase II) Blood kinetics of TBI-1301 by realtime-PCR
Evaluate persistence and expansion of transferred TBI-1301.
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Aug 2017
- Study start20 Sep 2017
- Primary completion23 Jan 2020
- Study completion9 Mar 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Synovial SarcomaStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Takara Bio Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Masanobu Kimura, Study director, Takara Bio Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03250325
- Sponsor's protocol ID
- 1301-03
- Status last verified
- Nov 2024
- First submitted
- 8 Aug 2017
- Last updated
- 20 Nov 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 20 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multi-center Phase I/II Study of NY-ESO-1 T Cell Receptor Gene Transferred T Lymphocytes in Patients with Synovial Sarcoma
- ClinicalTrials.gov ID
- NCT03250325
- Acronym / sponsor ID
- 1301-03
- Conditions
- Synovial Sarcoma
- Keywords
- Adoptive cell transfer, Cell therapy, Immunotherapy, NY-ESO-1, T cell receptor gene therapy, Synovial sarcoma
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Takara Bio Inc.Industry (pharma, biotech, devices)
- Study start
- 20 Sep 2017 (actual)
- Primary completion
- 23 Jan 2020 (actual)
- Study completion
- 9 Mar 2022 (actual)
- First posted
- 15 Aug 2017 (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 purpose of this study is to evaluate the safety and the efficacy of TBI-1301 for NY-ESO-1 expressing synovial sarcoma when administered following cyclophosphamide pre-treatment.
Detailed description
Following pre-treatment with cyclophosphamide, NY-ESO-1-specific T cell receptor (TCR) gene transduced T lymphocytes are transferred to human leukocyte antigen (HLA)-A*02:01 or HLA-A*02:06 positive patients with synovial sarcoma expressing NY-ESO-1, which are surgically unresectable and refractory to anthracycline therapy. The primary objective is to evaluate the safety in the phase 1 and the efficacy in the phase 2.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Split dose of 5x10^9 TBI-1301 | Experimental | Biological: TBI-1301, Drug: CyclophosphamideSplit dose of 5x10^9 TBI-1301 will be administered intravenously for 2 days following cyclophosphamide pre-treatment 750 mg/m2/d for 2 days. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- Histologically confirmed synovial sarcoma
- Surgically unresectable tumor
- Progressing or recurrent synovial sarcoma which has been treated with 1-4 regimens of systemic chemotherapies including anthracycline
- HLA-A*02:01 or HLA-A*02:06 positive
- Tumor that express NY-ESO-1 by immunohistochemistry
Key exclusion criteria (12 listed)
- Patients with the following conditions are excluded from the study; Unstable angina, cardiac infarction, or heart failure; Uncontrolled diabetes or hypertension; Active infection; Obvious interstitial pneumonia or lung fibrosis by chest X-ray; Active autoimmune disease requiring steroids or immunosuppressive therapy.
- Active metastatic tumor cell invasion into CNS
- Active multiple cancer
- Positive for HBs antigen or HBV-DNA observed in serum
- Positive for HCV antibody and HCV-RNA observed in serum
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 |
|---|---|---|
| TBI-1301Split dose of TBI-1301 is administered intravenously for 2 days following cyclophosphamide pre-treatment. | Biological | Split dose of 5x10^9 TBI-1301 |
| CyclophosphamideCyclophosphamide (750mg/m2/day x 2 days Intravenous (IV)) is administered as pre-treatment medication of TBI-1301. | Drug | Split dose of 5x10^9 TBI-1301 |
Primary outcome
(Phase I) Adverse event, mortality, severe adverse event, discontinuation due to adverse event, laboratory test values
Time frame: 52 weeks
Confirm the toxicity profile, which is measured by the degree of grade and seriousness, duration, causality, classification, etc. of the adverse events.
(Phase I) Appearance of replication competent retrovirus (RCR) by PCR
Time frame: 52 weeks
Confirm that no replication competent retrovirus observed.
(Phase I) Appearance of clonality by linear amplification mediated (LAM)-PCR
Time frame: 52 weeks
Confirm that no clonality is observed.
(Phase I) Blood kinetics of TBI-1301 by realtime-PCR
Time frame: 52 weeks
Evaluate persistence and expansion of transferred TBI-1301.
(Phase II) Overall response rate
Time frame: 52 weeks
Evaluate response rate by measuring response using RECIST v1.1 and irRECIST
8 secondary outcomes
(Phase I) Objective response rate
Time frame: 52 weeks
(Phase I/II) Progression free rate
Time frame: 12 weeks
(Phase I/II) Progression free survival
Time frame: 52 weeks
(Phase I/II) Overall survival
Time frame: 52 weeks
(Phase II) Adverse event, mortality, severe adverse event, discontinuation due to adverse event, laboratory test values
Time frame: 52 weeks
(Phase II) Appearance of RCR
Time frame: 52 weeks
(Phase II) Appearance of clonality (LAM-PCR)
Time frame: 52 weeks
(Phase II) Blood kinetics of TBI-1301 by realtime-PCR
Time frame: 52 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Nov 2024.
- Individual participant dataSharing planned: Undecided
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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.
Masanobu Kimura study director
Takara Bio Inc.
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing TBI-1301 and Cyclophosphamide in people with Synovial Sarcoma, run by Takara Bio Inc. at 5 sites in Japan.
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 12 inclusion and 12 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: finished, as of the sponsor's last update on 20 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in Japan. 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
(Phase I) Adverse event, mortality, severe adverse event, discontinuation due to adverse event, laboratory test values (time frame: 52 weeks). The study lists 5 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 20 Nov 2024.