Solid TumorsPhase 1RESTNCT02789228
Research Study Utilizing Expanded Multi-antigen Specific Lymphocytes for the Treatment of Solid Tumors
A Phase 1 study testing Tumor associated antigen lymphocytes (TAA-CTL) in people with Solid Tumors, run by Children's National Research Institute at 1 site in United States.
Trial summary
Patients with high-risk solid tumors, those that are refractory to standard up front therapy or relapse after completion of therapy, have a very poor prognosis despite attempts to induce remission with salvage regimen. Novel therapies are critical for this patient population with high-risk cancer. Read more about this study
- Who is it for?
- Ages 6 months to 60 years
- Conditions the study is for
- Solid Tumors
- What is being tested?
- Tumor associated antigen lymphocytes (TAA-CTL)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 1 siteUnited States
- Study dates
- 12 Oct 2016to Dec 2027
- 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 6 months to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Recipient procurement inclusion criteria
- Diagnosis of high-risk solid tumors: Ewing sarcoma, Wilms tumor, neuroblastoma, rhabdomyosarcoma, soft tissue sarcomas, osteosarcoma, adenocarcinoma, and esophageal carcinoma and renal cell carcinoma.
- Refractory disease, residual detectable disease following conventional therapy or relapsed disease.
- 6 months to 60 years of age at enrollment.*
- Karnofsky/Lansky score of ≥ 50%.*
- Absolute neutrophil count (ANC) greater than 500/µL. *
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- Absolute lymphocyte count (ALC) greater than 1000/µL.*
- Bilirubin ≤ 2.5 mg/dL. *
- Aspartate aminotransferase (AST)/ Alanine transaminase (ALT) ≤ 5x the upper limit of normal for age. *
- Serum creatinine < 1.0 mg/dL or 2 x the upper limit of normal for age (whichever is higher).*
- Pulse oximetry of > 90% on room air.*
- Agree to use contraceptive measures during study protocol participation (when age appropriate).*
- LVEF > 50% or LVSF > 27 % if history of total body irradiation (TBI).
- Patient or parent/guardian capable of providing informed consent.
Who cannot join
- Recipient Procurement exclusion criteria
- Patients with uncontrolled infections
- Patients with active HIV
- Current evidence of GVHD > grade 2 or chronic GVHD manifestations: bronchiolitis obliterans syndrome, sclerotic GVHD, or serositis.
- Pregnant or lactating females
- Prior immunotherapy with an investigational agent within the last 28 days prior to procurement
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- Recipient Inclusion to administer cells:
- Steroids less than 0.5 mg/kg/day prednisone (or equivalent).
- Karnofsky/Lansky score of ≥ 50% %.
- Bilirubin ≤ 2.5 mg/dL.
- AST/ALT ≤ 5x the upper limit of normal for age.
- Serum creatinine < 1.0 mg/dL or 2x the upper limit of normal for age (whichever is higher).
- Pulse oximetry of > 90% on room air.
- Patients receiving lymphodepleting chemotherapy must have:
- ANC >750 /uL Platelet count >75,000 /uL
- Recipient Exclusion to administer cells:
- Patients with uncontrolled infections
- Patients who received ATG, Campath, or other T cell immunosuppressive monoclonal antibodies within 28 days prior to TAA-T cell infusion
- Acute GVHD > grade 2 or chronic GVHD manifestations: bronchiolitis obliterans syndrome, sclerotic GVHD, or serositis
- Pregnant or lactating females
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's National Medical Center
Washington D.C., District of Columbia, 20010
About this study
Patients with high-risk solid tumors, those that are refractory to standard up front therapy or relapse after completion of therapy, have a very poor prognosis despite attempts to induce remission with salvage regimen. Novel therapies are critical for this patient population with high-risk cancer.
The ability of tumors to be recognized and lysed by the immune system offers a unique opportunity to aid in tumor eradication by expanding and activating these anti-tumor cells. Through this ability to harness sophisticated and specific immunotherapy, residual or relapsed disease that is resistant to chemotherapy and/or radiotherapy could be eradicated. Prior studies have suggested both safety of expanded specific T cells and efficacy in the setting of melanoma, lymphoma or viral eradication. While this therapy has previously been limited by the versatility of the tumor to down-regulate antigens and evade a single immune-target, the use of multi-antigen specific T cells may permit better and more durable anti-tumor immunity. Thus, the investigators propose to infuse these specific multi-antigen anti-tumor T lymphocytes into patients with high risk solid tumors. This trial will be conducted to demonstrate safety of these cells and generate efficacy and biology data that may be important for future studies that may enhance tumor immunotherapy.
Read the detailed description
This protocol is designed as a phase I dose-escalation study. In each treatment group (A and B), patients will be enrolled to one of the following TAA-CTL dose levels:
Dose Level One: 1 x 107 cells/m2 Dose Level Two: 2 x 107 cells/m2 Dose Level Three: 4 x 107 cells/m2
Patients will receive cells due to the presence of refractory disease and/or high risk for disease relapse and/or residual detectable disease following HSCT or conventional therapy at the time of the infusion. Group A and Group B patients will use the dose escalation strategy described above. Ideally, patients should not receive other systemic antineoplastic agents for at least 45 days after the infusion of TAA- CTL (for purposes of evaluation), although such treatment may be added if deemed critical for patient care by the attending physician.
Two to four patients will be enrolled at each dose level until the maximum tolerated dose (MTD) is determined at which point to ensure safety a total 8 patients will be treated at the MTD. Each patient will receive at least one TAA-CTL infusion and may receive a maximum of 8 doses total. Dose escalation will occur once at least 2 patients have completed the 45 day follow up period following their first TAA-CTL infusion. The first and second doses will be administered 45 days apart then additional doses will be spaced every 28 days. The expected volume of each infusion is 1 to 10 cc.
Expansion cohorts of Group B patients with Wilms tumor, neuroblastoma, rhabdomyosarcoma, adenocarcinoma and esophageal cancer will be permitted to enroll up to 6 additional patients in each disease group, to be treated at the MTD.
Within group B, a cohort of patients with relapsed or refractory Wilms tumor will be enrolled and receive a lymphodepleting chemotherapy regimen followed by TAA-T.
If patients with measurable or evaluable disease have a response of stable disease or better by RECIST criteria at the day 28 evaluation after dose 2 or subsequent evaluations they are eligible to receive up to 6 additional doses of CTLs at 28 day intervals. Each subsequent doseis expected to be at the enrollment dose level (i.e. no subsequent dose escalation). Following dose 1, if a patient's T cell supply is insufficient for subsequent doses at the enrollment dose level, further treatments may be administered at a lower dose level at the treating physician's discretion.
- Conditions
- Solid Tumors
- Being tested
- Tumor associated antigen lymphocytes (TAA-CTL)
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Tumor associated antigen lymphocytes (TAA-CTL)Biological
This protocol is designed as a phase I dose-escalation study. In each treatment group (A and B), patients will be enrolled to one of the following TAA-T dose levels:
Dose Level One: 1 x 107 cells/m2 Dose Level Two: 2 x 107 cells/m2 Dose Level Three: 4 x 107 cells/m2
| Study group | Role | What they receive |
|---|---|---|
| Group A | Experimental | Group A includes patients who have undergone an allogeneic hematopoietic stem cell transplant (HSCT) as part of their prior therapy.
Group A patients (post allogeneic HSCT): TAA-T will be infused any time after neutrophil engraftment post-HSCT or day 30, whichever comes first. Receives: Biological: Tumor associated antigen lymphocytes (TAA-CTL) |
| Group B | Experimental | Group B includes patients who have undergone conventional (standard) therapy which does not include an allogeneic HSCT. Within group B, a cohort of patients with relapsed or refractory Wilms tumor will be enrolled and receive a lymphodepleting chemotherapy regimen followed by TAA-T.
Group B patients (no prior allogeneic HSCT): TAA-T will be infused any time >1 week after completing most recent course of conventional (non-investigational) therapy for their disease. Patients receiving lymphodepletion will be >2 weeks from most recent course of conventional therapy and have nadired and recovered before beginning protocol therapy. Receives: Biological: Tumor associated antigen lymphocytes (TAA-CTL) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Product-Emergent Adverse Events
Primary endpoint of the study is defined grade ≥3 infusion-related adverse event occurring within 45 days of the last TAA-CTL dose, grade ≥4 non-hematologic adverse event occurring within 45 days of the last TAA-CTL dose and that are not due to the patient's underlying malignancy or pre-existing co-morbidities or grade ≥3 acute GVHD occurring within 45 days of the last TAA-CTL dose, or any unexpected toxicity of any grade attributed to the infusion of TAA-CTL occurring within 45 days of the last TAA-CTL dose. Toxicities will be defined by the NCI Common Terminology Criteria for Adverse Events (CTCAE), Version 4.03
Measured: Within 45 days of the last dose of TAA-CT of first infusion and 28 days after the final TAA CTL dose
Secondary outcomes (1)
Tumor associated antigen lymphocytes (TAA-CTL) responses
- determine the number of patients who respond to tumor associated antigen lymphocytes (TAA-CTL)
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Jun 2016 (estimated)
- Study start12 Oct 2016
- Primary completion18 Apr 2022
- Study completionDec 2027 (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 Solid TumorsStudies 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
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's National Research Institute
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, HMeany, Children's National Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02789228
- Sponsor's protocol ID
- 7497
- Acronym
- REST
- Status last verified
- Oct 2026
- First submitted
- 24 May 2016
- Last updated
- 29 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
Hont AB, Cruz CR, Ulrey R, O'Brien B, Stanojevic M, Datar A, Albihani S, Saunders D, Hanajiri R, Panchapakesan K, Darko S, Banerjee P, Fortiz MF, Hoq F, Lang H, Wang Y, Hanley PJ, Dome JS, Bollard CM, Meany HJ. Immunotherapy of Relapsed and Refractory Solid Tumors With Ex Vivo Expanded Multi-Tumor Associated Antigen Specific Cytotoxic T Lymphocytes: A Phase I Study. J Clin Oncol. 2019 Sep 10;37(26):2349-2359. doi: 10.1200/JCO.19.00177. Epub 2019 Jul 29.
PubMed 31356143
Study at a glance
From the registry record, last updated by the sponsor on 29 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Research Study Utilizing Expanded Multi-antigen Specific Lymphocytes for the Treatment of Solid Tumors
- ClinicalTrials.gov ID
- NCT02789228
- Acronym / sponsor ID
- REST / 7497
- Conditions
- Solid Tumors
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 36 participants (actual)
- Lead sponsor
- Children's National Research InstituteUniversity, hospital or other
- Study start
- 12 Oct 2016 (actual)
- Primary completion
- 18 Apr 2022 (actual)
- Study completion
- Dec 2027 (estimated)
- First posted
- 2 Jun 2016 (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.
Patients with high-risk solid tumors, those that are refractory to standard up front therapy or relapse after completion of therapy, have a very poor prognosis despite attempts to induce remission with salvage regimen. Novel therapies are critical for this patient population with high-risk cancer.
The ability of tumors to be recognized and lysed by the immune system offers a unique opportunity to aid in tumor eradication by expanding and activating these anti-tumor cells. Through this ability to harness sophisticated and specific immunotherapy, residual or relapsed disease that is resistant to chemotherapy and/or radiotherapy could be eradicated. Prior studies have suggested both safety of expanded specific T cells and efficacy in the setting of melanoma, lymphoma or viral eradication. While this therapy has previously been limited by the versatility of the tumor to down-regulate antigens and evade a single immune-target, the use of multi-antigen specific T cells may permit better and more durable anti-tumor immunity. Thus, the investigators propose to infuse these specific multi-antigen anti-tumor T lymphocytes into patients with high risk solid tumors. This trial will be conducted to demonstrate safety of these cells and generate efficacy and biology data that may be important for future studies that may enhance tumor immunotherapy.
Detailed description
This protocol is designed as a phase I dose-escalation study. In each treatment group (A and B), patients will be enrolled to one of the following TAA-CTL dose levels:
Dose Level One: 1 x 107 cells/m2 Dose Level Two: 2 x 107 cells/m2 Dose Level Three: 4 x 107 cells/m2
Patients will receive cells due to the presence of refractory disease and/or high risk for disease relapse and/or residual detectable disease following HSCT or conventional therapy at the time of the infusion. Group A and Group B patients will use the dose escalation strategy described above. Ideally, patients should not receive other systemic antineoplastic agents for at least 45 days after the infusion of TAA- CTL (for purposes of evaluation), although such treatment may be added if deemed critical for patient care by the attending physician.
Two to four patients will be enrolled at each dose level until the maximum tolerated dose (MTD) is determined at which point to ensure safety a total 8 patients will be treated at the MTD. Each patient will receive at least one TAA-CTL infusion and may receive a maximum of 8 doses total. Dose escalation will occur once at least 2 patients have completed the 45 day follow up period following their first TAA-CTL infusion. The first and second doses will be administered 45 days apart then additional doses will be spaced every 28 days. The expected volume of each infusion is 1 to 10 cc.
Expansion cohorts of Group B patients with Wilms tumor, neuroblastoma, rhabdomyosarcoma, adenocarcinoma and esophageal cancer will be permitted to enroll up to 6 additional patients in each disease group, to be treated at the MTD.
Within group B, a cohort of patients with relapsed or refractory Wilms tumor will be enrolled and receive a lymphodepleting chemotherapy regimen followed by TAA-T.
If patients with measurable or evaluable disease have a response of stable disease or better by RECIST criteria at the day 28 evaluation after dose 2 or subsequent evaluations they are eligible to receive up to 6 additional doses of CTLs at 28 day intervals. Each subsequent doseis expected to be at the enrollment dose level (i.e. no subsequent dose escalation). Following dose 1, if a patient's T cell supply is insufficient for subsequent doses at the enrollment dose level, further treatments may be administered at a lower dose level at the treating physician's discretion.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group A | Experimental | Biological: Tumor associated antigen lymphocytes (TAA-CTL)Group A includes patients who have undergone an allogeneic hematopoietic stem cell transplant (HSCT) as part of their prior therapy. Group A patients (post allogeneic HSCT): TAA-T will be infused any time after neutrophil engraftment post-HSCT or day 30, whichever comes first. |
| Group B | Experimental | Biological: Tumor associated antigen lymphocytes (TAA-CTL)Group B includes patients who have undergone conventional (standard) therapy which does not include an allogeneic HSCT. Within group B, a cohort of patients with relapsed or refractory Wilms tumor will be enrolled and receive a lymphodepleting chemotherapy regimen followed by TAA-T. Group B patients (no prior allogeneic HSCT): TAA-T will be infused any time >1 week after completing most recent course of conventional (non-investigational) therapy for their disease. Patients receiving lymphodepletion will be >2 weeks from most recent course of conventional therapy and have nadired and recovered before beginning protocol therapy. |
Who may qualify
- Ages
- 6 months to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (14 listed)
- Recipient procurement inclusion criteria
- Diagnosis of high-risk solid tumors: Ewing sarcoma, Wilms tumor, neuroblastoma, rhabdomyosarcoma, soft tissue sarcomas, osteosarcoma, adenocarcinoma, and esophageal carcinoma and renal cell carcinoma.
- Refractory disease, residual detectable disease following conventional therapy or relapsed disease.
- 6 months to 60 years of age at enrollment.*
- Karnofsky/Lansky score of ≥ 50%.*
Key exclusion criteria (20 listed)
- Recipient Procurement exclusion criteria
- Patients with uncontrolled infections
- Patients with active HIV
- Current evidence of GVHD > grade 2 or chronic GVHD manifestations: bronchiolitis obliterans syndrome, sclerotic GVHD, or serositis.
- Pregnant or lactating females
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 |
|---|---|---|
| Tumor associated antigen lymphocytes (TAA-CTL)This protocol is designed as a phase I dose-escalation study. In each treatment group (A and B), patients will be enrolled to one of the following TAA-T dose levels: Dose Level One: 1 x 107 cells/m2 Dose Level Two: 2 x 107 cells/m2 Dose Level Three: 4 x 107 cells/m2 | Biological | Group A, Group B |
Primary outcome
Incidence of Product-Emergent Adverse Events
Time frame: Within 45 days of the last dose of TAA-CT of first infusion and 28 days after the final TAA CTL dose
Primary endpoint of the study is defined grade ≥3 infusion-related adverse event occurring within 45 days of the last TAA-CTL dose, grade ≥4 non-hematologic adverse event occurring within 45 days of the last TAA-CTL dose and that are not due to the patient's underlying malignancy or pre-existing co-morbidities or grade ≥3 acute GVHD occurring within 45 days of the last TAA-CTL dose, or any unexpected toxicity of any grade attributed to the infusion of TAA-CTL occurring within 45 days of the last TAA-CTL dose. Toxicities will be defined by the NCI Common Terminology Criteria for Adverse Events (CTCAE), Version 4.03
1 secondary outcome
Tumor associated antigen lymphocytes (TAA-CTL) responses
Time frame: 1 year
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 29 Oct 2026.
- Derived from this study: PubMed 31356143Hont AB, Cruz CR, Ulrey R, O'Brien B, Stanojevic M, Datar A, Albihani S, Saunders D, Hanajiri R, Panchapakesan K, Darko S, Banerjee P, Fortiz MF, Hoq F, Lang H, Wang Y, Hanley PJ, Dome JS, Bollard CM, Meany HJ. Immunotherapy of Relapsed and Refractory Solid Tumors With Ex Vivo Expanded Multi-Tumor Associated Antigen Specific Cytotoxic T Lymphocytes: A Phase I Study. J Clin Oncol. 2019 Sep 10;37(26):2349-2359. doi: 10.1200/JCO.19.00177. Epub 2019 Jul 29.
- 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.
The record lists no contact. Use the official record to reach the sponsor.
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 Tumor associated antigen lymphocytes (TAA-CTL) in people with Solid Tumors, run by Children's National Research Institute at 1 site 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?
The sponsor lists 14 inclusion and 20 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 29 Oct 2026. 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 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?
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?
Incidence of Product-Emergent Adverse Events (time frame: Within 45 days of the last dose of TAA-CT of first infusion and 28 days after the final TAA CTL dose). 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 NCT02789228, 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 29 Oct 2026.