RhabdomyosarcomaPhase 1NCT02239861
TAA-Specific CTLS for Solid Tumors (TACTASOM)
A Phase 1 study testing TAA-Specific CTLs in people with Rhabdomyosarcoma, run by Baylor College of Medicine at 2 sites in United States.
Trial summary
This is a clinical trial for patients with a solid tumor which has come back, or may come back, or has not gone away after treatment, including the standard treatment we know for these diseases. Read more about this study
- Who is it for?
- Ages 2 to 80 years
- Conditions the study is for
- Rhabdomyosarcoma
- What is being tested?
- TAA-Specific CTLs
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 2 sitesUnited States
- Study dates
- Apr 2015to 7 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 2 to 80 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
THIS PROTOCOL IS CURRENTLY NOT RECRUITING ADULTS.
Procurement Inclusion Criteria:
- Any patient regardless of sex with a solid tumor expressing any of the following antigens (PRAME, SSX2, MAGEA4, NY-ESO1-1 and/or Survivin) with:
- Active disease after first line therapy;
- Refractory disease;
- As adjuvant therapy for high risk disease (high risk disease is a disease that has a >50% risk of progression within 5 years)
- Patients with life expectancy at least 6 weeks.
- Age greater than or equal to 2 and less than or equal to 80 years old.
- Hgb >8.0
- Informed Consent explained to, understood by and signed by patient/guardian. Patient/guardian given copy of informed consent.
Procurement Exclusion Criteria:
- Diagnosis of primary CNS tumor.
- Patients with severe intercurrent infection.
- Patients with active HIV infection at time of procurement (can be pending at the time of blood draw).
- Patients in remission who are enrolled on another study where time to progression or disease-free survival is a primary endpoint.
Treatment Inclusion Criteria:
- Any patient regardless of sex with a solid tumor expressing any of the following antigens (PRAME, SSX2, MAGEA4, NY-ESO1-1 and/or Survivin) with:
- Active disease after first line therapy;
- Refractory disease;
- As adjuvant therapy for high risk disease (high risk disease is a disease that has a >50% risk of progression within 5 years)
- Patients with life expectancy at least 6 weeks.
- Age greater than or equal to 2 and less than or equal to 80 years old.
- Pulse oximetry of >95% on room air in patients who previously received radiation therapy.
- Patients with a Karnofsky/Lansky score of greater than or equal to 50.
- Patients with bilirubin less than or equal to 2x upper limit of normal, AST less than or equal to 3x upper limit of normal, and Hgb >8.0
- Patients with a creatinine less than or equal to 2x upper limit of normal for age.
- Patients should have been off other investigational therapy for one month prior to entry in this study.
- Patients should have been off conventional therapy for at least 1 week prior to entry in this study. PD1/PDL1 inhibitors will be allowed if medically indicated.
- Informed Consent explained to, understood by and signed by patient/guardian. Patient/guardian given copy of informed consent.
- Due to unknown effects of this therapy on a fetus, pregnant women are excluded from this research. The male partner should use a condom Females of child-bearing potential must be willing to utilize one of the more effective birth control methods during the study unless female has had a hysterectomy or tubal ligation.
Treatment Exclusion Criteria:
- Diagnosis of primary CNS tumor.
- Patients with severe intercurrent infection.
- Patients receiving systemic corticosteroids (patients off steroids for at least 48 hours are eligible).
- Pregnant or breastfeeding
- HIV positive.
- Patients in remission who are enrolled on another study where time to progression or disease-free survival is a primary endpoint.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Houston Methodist Hospital
Houston, Texas, 77030
Texas Children's Hospital
Houston, Texas, 77030
About this study
Official title: Tumor Associated Antigen (TAA)-Specific Cytotoxic T-Lymphocytes Administered to Patients With Solid Tumors
This is a clinical trial for patients with a solid tumor which has come back, or may come back, or has not gone away after treatment, including the standard treatment we know for these diseases. This is a study using special immune system cells called tumor-associated antigen (TAA)-specific cytotoxic T lymphocytes, a new experimental therapy.
The proteins that the investigators are targeting in this study are called tumor-associated antigens (TAAs). These are cell proteins that are specific to the cancer cell, so they either do not show or show up in low quantities on normal human cells. In this study, the investigators target five common TAAs called NY-ESO-1, MAGEA4, PRAME, Survivin and SSX. On a different study, patients have been treated and so far this treatment has shown to be safe.
Read more
The investigators now want to try this treatment in patients with solid tumors.
This protocol is designed as a Phase I dose-escalation study.
Read the detailed description
Blood will be collected from the patient and TAA-specific CTLs will be made.
The cells will be injected by IV into the patient over 1 - 10 minutes. The patient may be pre-treated with acetaminophen (Tylenol) and diphenhydramine (Benadryl). Acetaminophen (Tylenol) and diphenhydramine (Benadryl) are given to prevent a possible allergic reaction to the T cell infusion. Initially, two doses of T cells will be given two weeks apart. The patient's disease will be assessed pre-infusion and then 6 weeks after the second infusion. If after the second infusion, there is a reduction in size of the patient's tumor or if the tumor remains stable on CT or MRI scan as assessed by a radiologist, the patient can receive up to six (6) additional doses of the T cells at monthly intervals. All of the treatments will be given by the Center for Cell and Gene Therapy at Houston Methodist Hospital or Texas Children's Hospital.
In between the first and second T cell infusions, and for 6 weeks after the last infusion, the investigators ask that the patient not receive any other anti-cancer treatments, such as radiation therapy or chemotherapy, except PD1/PDL1 inhibitors as clinically indicated. If the patient does receive any other therapies in-between the first and second infusion of T cells, they will be taken off treatment and will not be able to receive the second infusion of T cells.
This is a dose escalation study. This means that at the beginning, patients will be started on the lowest dose (1 of 4 different levels) of T cells. Once that dose schedule proves safe, the next group of patients will be started at a higher dose. This process will continue until all 4 dose levels are studied. If the side effects are too severe, the dose will be lowered or the T cell injections will be stopped.
MEDICAL TESTS BEFORE TREATMENT:
Before being treated, patients will receive a series of standard medical tests:
- Physical exam.
- Blood tests to measure blood cells, kidney and liver function.
- Measurements of the patient's tumor by routine imaging studies. The investigators will use the imaging study that was previously performed to follow the patient's tumor: Computer Tomogram (CT), Magnetic Resonance Imaging (MRI), or Positron Emission Tomography (PET).
- Pregnancy test if the patient is a female who can have children.
MEDICAL TEST AFTER TREATMENT:
Patients will receive standard medical test after infusion:
- Patients will receive standard medical test after infusion:
- Blood tests to measure blood cells, kidney and liver function.
- Imaging study 6 weeks after the 2nd CTL infusion.
To learn more about the way the T cells are working in the patient's body, an extra 20-40 mL (4-8 teaspoons) of blood will be taken before each infusion, and at Weeks 1, 2, 4 and 6. Afterwards, blood will be collected at 3, 6, 9 and 12 months after the last infusion. The investigators will use this blood to see how long the T cells last, and to look at the immune response to the patient's response to cancer. Patients will then be contacted once a year for up to 4 additional years (total of 5 years follow-up) to evaluate their disease response long-term.
Study Duration: The patient's active participation in this study will last for approximately one (1) year. If the patient receives additional doses of the TAA-CTLs as described above, then the patient's active participation will last until one (1) year after his/her last dose. We will then contact the patient once a year for up to 4 additional years (total of 5 years follow-up) in order to evaluate his/her disease response long-term.
- Conditions
- Rhabdomyosarcoma
- Being tested
- TAA-Specific CTLs
- Keywords
- Rhabdomyosarcoma, cytotoxic T lymphocytes
- Medical subject headings
- Rhabdomyosarcoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TAA-Specific CTLsBiological
Also called Tumor Associated Antigen-Specific Cytotoxic T-Lymphocytes
Patients may be pre-medicated with Benadryl up to 1 mg/kg IV (max 50 mg) and Tylenol 10 mg/kg po (max 650 mg).
Cell Administration: Tumor-specific T cells will be given by intravenous injection over 1-10 minutes through either a peripheral or a central line.
Patients with stable disease or better will receive up to 6 further doses of CTLs, as long as they continue to show disease stabilization or improvement by RECIST criteria at their 8 week or subsequent evaluations. Each dose will consist of the same number as their second injection or less (if there is not enough product available for the subject's original dose).
Patients will not be able to receive additional doses until the initial safety profile is completed at 6 weeks following the second infusion.
| Study group | Role | What they receive |
|---|---|---|
| TAA-Specific CTLs | Experimental | 4 different dosing schedules will be evaluated. 2 to 4 patients will be evaluated on each dosing schedule. The first 2 patients on each dose level will be staggered by 4 weeks (which starts when the first infusion is given, Day 0). No subjects between the ages of 2-18 will be enrolled to a dose level on this protocol, until an adult has been enrolled to and treated on that dose level on one of the protocols being conducted under this same IND. Each patient will receive 2 injections at the same dose,14 days apart: The expected volume of infusion will be 1 to 10 cc.
Dose Level One:
Day 0 and 14: 5 x 10^6 cells/m^2
Dose Level Two:
Day 0 and 14: 1 x 10^7 cells/m^2
Dose Level Three:
Day 0 and 14: 2 x 10^7 cells/m^2
Dose Level Four:
Day 0 and 14: 4 x 10^7 cells/m^2 Receives: Biological: TAA-Specific CTLs |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of patients with dose-limiting toxicity.
The Phase I dose escalation trial is designed for the primary goal of evaluating the safety and feasibility of administering TAA-CTLs to patients with solid tumors.
Measured: 8 weeks
Secondary outcomes (1)
Number of patients with a disease response to the CTLs.
The Phase I dose escalation trial is designed for the primary goal of evaluating the safety and feasibility of administering TAA-CTLs to patients with solid tumors.
Measured: 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Sep 2014 (estimated)
- Study startApr 2015
- Primary completionOct 2018
- Study completion7 Apr 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for RhabdomyosarcomaStudies of the same condition, open and under way.
- More trials run by Baylor College of MedicineThe 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
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baylor College of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- The Methodist Hospital Research Institute, Center for Cell and Gene Therapy, Baylor College of Medicine, Solving Kids' Cancer, Pierce Phillips Charity
- Responsible party
- Principal investigator, Sarah Whittle, Baylor College of Medicine
- Investigators
- Ann Leen, PhD, Principal investigator, Baylor College of MedicineCliona Rooney, PhD, Principal investigator, Baylor College of MedicineHelen Heslop, MD, Principal investigator, Baylor College of MedicineSarah Whittle, MD, Principal investigator, Baylor College of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02239861
- Sponsor's protocol ID
- H-35425, TACTASOM
- Status last verified
- Apr 2022
- First submitted
- 11 Sep 2014
- Last updated
- 12 Apr 2022
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Apr 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Tumor Associated Antigen (TAA)-Specific Cytotoxic T-Lymphocytes Administered to Patients With Solid Tumors
- ClinicalTrials.gov ID
- NCT02239861
- Acronym / sponsor ID
- H-35425, TACTASOM
- Conditions
- Rhabdomyosarcoma
- Keywords
- Rhabdomyosarcoma, cytotoxic T lymphocytes
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- Baylor College of MedicineUniversity, hospital or other
- Collaborators
- The Methodist Hospital Research Institute, Center for Cell and Gene Therapy, Baylor College of Medicine, Solving Kids' Cancer, Pierce Phillips Charity
- Study start
- Apr 2015 (actual)
- Primary completion
- Oct 2018 (actual)
- Study completion
- 7 Apr 2022 (actual)
- First posted
- 15 Sep 2014 (estimated)
- 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.
This is a clinical trial for patients with a solid tumor which has come back, or may come back, or has not gone away after treatment, including the standard treatment we know for these diseases. This is a study using special immune system cells called tumor-associated antigen (TAA)-specific cytotoxic T lymphocytes, a new experimental therapy.
The proteins that the investigators are targeting in this study are called tumor-associated antigens (TAAs). These are cell proteins that are specific to the cancer cell, so they either do not show or show up in low quantities on normal human cells. In this study, the investigators target five common TAAs called NY-ESO-1, MAGEA4, PRAME, Survivin and SSX. On a different study, patients have been treated and so far this treatment has shown to be safe.
The investigators now want to try this treatment in patients with solid tumors.
This protocol is designed as a Phase I dose-escalation study.
Detailed description
Blood will be collected from the patient and TAA-specific CTLs will be made.
The cells will be injected by IV into the patient over 1 - 10 minutes. The patient may be pre-treated with acetaminophen (Tylenol) and diphenhydramine (Benadryl). Acetaminophen (Tylenol) and diphenhydramine (Benadryl) are given to prevent a possible allergic reaction to the T cell infusion. Initially, two doses of T cells will be given two weeks apart. The patient's disease will be assessed pre-infusion and then 6 weeks after the second infusion. If after the second infusion, there is a reduction in size of the patient's tumor or if the tumor remains stable on CT or MRI scan as assessed by a radiologist, the patient can receive up to six (6) additional doses of the T cells at monthly intervals. All of the treatments will be given by the Center for Cell and Gene Therapy at Houston Methodist Hospital or Texas Children's Hospital.
In between the first and second T cell infusions, and for 6 weeks after the last infusion, the investigators ask that the patient not receive any other anti-cancer treatments, such as radiation therapy or chemotherapy, except PD1/PDL1 inhibitors as clinically indicated. If the patient does receive any other therapies in-between the first and second infusion of T cells, they will be taken off treatment and will not be able to receive the second infusion of T cells.
This is a dose escalation study. This means that at the beginning, patients will be started on the lowest dose (1 of 4 different levels) of T cells. Once that dose schedule proves safe, the next group of patients will be started at a higher dose. This process will continue until all 4 dose levels are studied. If the side effects are too severe, the dose will be lowered or the T cell injections will be stopped.
MEDICAL TESTS BEFORE TREATMENT:
Before being treated, patients will receive a series of standard medical tests:
- Physical exam.
- Blood tests to measure blood cells, kidney and liver function.
- Measurements of the patient's tumor by routine imaging studies. The investigators will use the imaging study that was previously performed to follow the patient's tumor: Computer Tomogram (CT), Magnetic Resonance Imaging (MRI), or Positron Emission Tomography (PET).
- Pregnancy test if the patient is a female who can have children.
MEDICAL TEST AFTER TREATMENT:
Patients will receive standard medical test after infusion:
- Patients will receive standard medical test after infusion:
- Blood tests to measure blood cells, kidney and liver function.
- Imaging study 6 weeks after the 2nd CTL infusion.
To learn more about the way the T cells are working in the patient's body, an extra 20-40 mL (4-8 teaspoons) of blood will be taken before each infusion, and at Weeks 1, 2, 4 and 6. Afterwards, blood will be collected at 3, 6, 9 and 12 months after the last infusion. The investigators will use this blood to see how long the T cells last, and to look at the immune response to the patient's response to cancer. Patients will then be contacted once a year for up to 4 additional years (total of 5 years follow-up) to evaluate their disease response long-term.
Study Duration: The patient's active participation in this study will last for approximately one (1) year. If the patient receives additional doses of the TAA-CTLs as described above, then the patient's active participation will last until one (1) year after his/her last dose. We will then contact the patient once a year for up to 4 additional years (total of 5 years follow-up) in order to evaluate his/her disease response long-term.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| TAA-Specific CTLs | Experimental | Biological: TAA-Specific CTLs4 different dosing schedules will be evaluated. 2 to 4 patients will be evaluated on each dosing schedule. The first 2 patients on each dose level will be staggered by 4 weeks (which starts when the first infusion is given, Day 0). No subjects between the ages of 2-18 will be enrolled to a dose level on this protocol, until an adult has been enrolled to and treated on that dose level on one of the protocols being conducted under this same IND. Each patient will receive 2 injections at the same dose,14 days apart: The expected volume of infusion will be 1 to 10 cc. Dose Level One: Day 0 and 14: 5 x 10^6 cells/m^2 Dose Level Two: Day 0 and 14: 1 x 10^7 cells/m^2 Dose Level Three: Day 0 and 14: 2 x 10^7 cells/m^2 Dose Level Four: Day 0 and 14: 4 x 10^7 cells/m^2 |
Who may qualify
- Ages
- 2 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
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 |
|---|---|---|
| TAA-Specific CTLsAlso: Tumor Associated Antigen-Specific Cytotoxic T-LymphocytesPatients may be pre-medicated with Benadryl up to 1 mg/kg IV (max 50 mg) and Tylenol 10 mg/kg po (max 650 mg). Cell Administration: Tumor-specific T cells will be given by intravenous injection over 1-10 minutes through either a peripheral or a central line. Patients with stable disease or better will receive up to 6 further doses of CTLs, as long as they continue to show disease stabilization or improvement by RECIST criteria at their 8 week or subsequent evaluations. Each dose will consist of the same number as their second injection or less (if there is not enough product available for the subject's original dose). Patients will not be able to receive additional doses until the initial safety profile is completed at 6 weeks following the second infusion. | Biological | TAA-Specific CTLs |
Primary outcome
Number of patients with dose-limiting toxicity.
Time frame: 8 weeks
The Phase I dose escalation trial is designed for the primary goal of evaluating the safety and feasibility of administering TAA-CTLs to patients with solid tumors.
1 secondary outcome
Number of patients with a disease response to the CTLs.
Time frame: 8 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 12 Apr 2022.
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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.
Ann Leen, PhD principal investigator
Baylor College of Medicine
Cliona Rooney, PhD principal investigator
Baylor College of Medicine
Helen Heslop, MD principal investigator
Baylor College of Medicine
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing TAA-Specific CTLs in people with Rhabdomyosarcoma, run by Baylor College of Medicine at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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 12 Apr 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. 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?
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?
Number of patients with dose-limiting toxicity. (time frame: 8 weeks). 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 12 Apr 2022.