LymphocytesPhase 1NCT06047977
Tumor Infiltrating Lymphocyte Therapy for Pediatric High Risk Solid Tumors
A Phase 1 study testing Tumor Infiltrating Lymphocytes, Fludarabine, Cyclophosphamide, Interleukin-2 in people with Lymphocytes, run by H. Lee Moffitt Cancer Center and Research Institute at 1 site in United States.
Trial summary
Part One of this study will determine the feasibility of creating Tumor-Infiltrating Lymphocyte (TIL) product prospectively from high-risk pediatric solid tumors. Part Two of this study will determine the safety of TIL therapy with lymphodepleting chemotherapy and post-TIL Interleukin-2 in high-risk pediatric solid… Read more about this study
- Who is it for?
- Ages 1 year to 21 years
- Conditions the study is for
- Lymphocytes
- What is being tested?
- Tumor Infiltrating Lymphocytes, Fludarabine, Cyclophosphamide, Interleukin-2
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteUnited States
- Study dates
- 2 Jun 2025to 1 Sep 2029
- 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 1 year to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Part One (Prospective biobanking study):
- Patients must be ≥1 year to ≤ 21 years of age at time of enrollment to Part One.
- Initial therapy patients: Patients with a histologic diagnosis of high-risk pediatric malignant solid tumors (pMST), defined as a pediatric malignant solid tumor outside of the central nervous system, newly-diagnosed or being treated with initial therapy, with expected 5-year Event-Free Survival (EFS) <60%. Examples include:
- High-risk neuroblastoma
- Metastatic Ewing sarcoma
- Metastatic osteosarcoma
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- Hepatoblastoma metastatic or not amenable to total resection
- Desmoplastic small round cell tumor
- Metastatic rhabdomyosarcoma
- Metastatic non-rhabdomyosarcoma soft tissue sarcomas (NRSTS)
- Metastatic diffuse anaplastic Wilms tumor
- Malignant rhabdoid tumor of kidney or liver
- Mediastinal mixed germ cell tumors
- Other tumors may be deemed eligible after assessment and documentation of prognosis from an enrolling institution's multidisciplinary tumor board.
- Note: There will be occasions prior to establishment of a histologic diagnosis where an investigator will highly suspect one of the diagnoses above in a patient due to undergo a surgical procedure (initial biopsy, up-front resection, etc.). If this is the case, the investigator may choose to enroll the patient prior to a tissue diagnosis. Their suspicion of a qualifying diagnosis should be documented. If the diagnosis is confirmed, the patient may continue on study. If the diagnosis is not confirmed, the patient will be considered a screening failure and removed from study.
- Relapsed/refractory/recurrent patients: patients must have a histologic diagnosis of pMST (may include diagnoses outside those identified in Part 1) that has relapsed after achieving remission or progressed after completion of all planned initial therapy.
- Patients must have an open surgical biopsy or resection planned (core needle/final needle biopsies are not allowed) for standard of care purposes, at some point in their initial or relapsed therapy for pMST.
- Note: The surgery for tissue procurement may occur at any time during initial therapy including up-front surgeries or after neoadjuvant therapies, including chemotherapy, immunotherapy, and radiation.
- All patients and/or their parents or legal guardians must have the ability to understand and the willingness to sign a written informed consent or assent document.
- Part Two: Phase I Clinical Trial
- ≥1-year-old at enrollment. There is no upper bound age limit, as long as the patient met age criteria at the time of enrollment on Part One.
- Weight ≥ 5 kg at time of enrollment for Part Two.
- Written informed consent from patient/family (separate from Part One).
- Confirmation of satisfactory TIL cellular product availability.
- Karnofsky/Lansky (as age appropriate) score ≥60%.
- Must fit one of the following disease status criteria:
- Relapsed/refractory disease: Patients with a histologic diagnosis of high-risk pMST as defined in Part 1 inclusion who have recurrent/refractory local or metastatic disease that is either measurable or evaluable by RECIST 1.1 51
- OR:
- Adjuvant therapy for poor prognosis tumors: patients qualifying for TIL collection as per Part 1, deemed by the treating investigator to have <30% chance at long-term cure who has reached the end of their primary therapy with a disease status of Stable Disease or better and who have achieved appropriate washout period. Measurable or evaluable disease per RECIST 1.1 is NOT required for these patients.
- Organ function requirements:
- Adequate hematologic function, defined as: Absolute neutrophil count greater than or equal to 1000/mm3, greater than 7 days from short-acting myeloid growth factors and at least 14 days from long-acting myeloid growth factors, Platelet count greater than or equal to 100,000/mm3 without transfusion within 7 days and without platelet growth factors for at least 14 days, and Hemoglobin greater than or equal to 8.0 g/dL without transfusion within 14 days.
- Liver function, defined as serum alanine aminotransferase and aspartate aminotransferase less than 5 times the institutional upper limit of normal and total bilirubin <2.0 mg/dL.
- Adequate renal function, defined as creatinine clearance or radioisotope glomerular filtration rate≥ 70 mL/min/1.73 m2 OR appropriate serum creatinine based on age/sex (see table below).
- Age Maximum Serum Creatinine (mg/dL) Male Female 1 to < 2 years 0.6 0.6 2 to < 6 years 0.8 0.8 6 to < 10 years 1.0 1.0 10 to < 13 years 1.2 1.2 13 to < 16 years 1.5 1.4
- ≥ 16 years 1.7 1.4
- Adequate cardiac function defined as Adequate cardiac function defined as a shortening fraction of ≥ 27% by echocardiogram, or ejection fraction of > 50% by echocardiogram or radionuclide angiogram.
- Adequate pulmonary function defined as forced expiratory volume (FEV1), forced vital capacity (FVC), diffusing capacity of the lungs for carbon monoxide (DLCO) > 50% predicted (corrected for hemoglobin); if unable to perform pulmonary function tests, then O2 saturation > 92% on room air.
- Appropriate time frame from prior therapy:
- Subjects must have fully recovered from the acute toxic effects of all prior anti-cancer chemotherapy.
- Myelosuppressive chemotherapy: At least 21 days after the last dose of myelosuppressive chemotherapy (42 days if prior nitrosourea).
- Hematopoietic growth factors: At least 14 days after the last dose of a long-acting growth factor (e.g. Pegfilgrastim) or 7 days for short acting growth factor. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur. The duration of this interval must be discussed with the study chair.
- Biologic (anti-neoplastic agent): At least 7 days after the last dose of a biologic agent. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur. The duration of this interval must be discussed with the study chair.
- Cellular Therapy: ≥ 42 days must have elapsed from last dose of any type of cellular therapy (e.g. modified T cells, natural killer cells, dendritic cells, etc.)
- Interleukins, Interferons, and Cytokines (other than Hematopoietic Growth Factors): ≥ 21 days must have elapsed from the last dose of interleukins, interferon or cytokines (other than Hematopoietic Growth Factors).
- Antibodies: ≥ 21 days must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to Grade ≤ 1.
- Radiation therapy: At least 28 days after local palliative radiotherapy (XRT) (small port); 6 weeks must have elapsed since treatment with therapeutic doses of 131 I-MIBG; At least 42 days must have elapsed if other substantial bone marrow radiation.
- Stem Cell Infusion without Total Body Irradiation: No evidence of active graft vs. host disease and at least 84 days must have elapsed after transplant and 42 days for autologous stem cell infusion after 131 Iodine meta-iodobenzylguanidine (MIBG) therapy.
- Investigational Agents Not Otherwise Specified: ≥30 days must have elapsed since the last dose of any agents not specified above. For agents with an uncertain washout period or for any questions or uncertainty the study PI should be notified.
Who cannot join
- Part One: There are no exclusion criteria
- Part Two:
- Patients with active systemic infections requiring intravenous antibiotics
- Patients testing positive for HIV titer, hepatitis B surface antigen, human T-cell leukemia-lymphoma virus (HTLV) I or II antibody, or both rapid plasma regain (RPR) and fluorescent treponemal antibody (FTA) are excluded. Patients with hepatitis C antibody must have a negative (undetectable) viral load by polymerase chain reaction (PCR).
- Patients who are pregnant or nursing.
- Sexually active patients of reproductive potential are eligible if they have agreed to use an effective contraceptive method from the time of informed consent through the duration and for 1 month following completion of protocol treatment. The definition of an effective contraceptive method will be at the discretion of the institutional investigator.
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- Patients needing chronic immunosuppressive systemic steroids are excluded. Any supraphysiologic doses of steroids should be discontinued by the time of enrollment.
- Patients with autoimmune diseases that require immunosuppressive medications.
- Patients with central nervous system metastases, currently active or in the past.
- Patients with history of prior solid organ transplant.
- Inability to comprehend and give informed consent.
- Patients receiving concomitant anti-cancer therapies or investigational therapies.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Johns Hopkins All Children's Hospital
St. Petersburg, Florida, 33701
Jade Hanson, 727-767-6468, Jade.Hanson@jhmi.edu
Muaz Alrazzak, MD
About this study
Official title: Tumor Infiltrating Lymphocytes in Pediatric Malignant Solid Tumors: A Prospective Biobanking Study and Phase I Clinical Trial
Part One of this study will determine the feasibility of creating Tumor-Infiltrating Lymphocyte (TIL) product prospectively from high-risk pediatric solid tumors.
Part Two of this study will determine the safety of TIL therapy with lymphodepleting chemotherapy and post-TIL Interleukin-2 in high-risk pediatric solid tumors
- Conditions
- Lymphocytes
- Being tested
- Tumor Infiltrating Lymphocytes, Fludarabine, Cyclophosphamide, Interleukin-2
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Tumor Infiltrating Lymphocytes, Fludarabine, Cyclophosphamide, Interleukin-2Biological
Lymphodepleting Chemotherapy with Fludarabine/Cyclophosphamide followed by TIL and Post-TIL Interleukin-2 (IL-2)
| Study group | Role | What they receive |
|---|---|---|
| TIL Therapy | Experimental | TIL therapy with lymphodepleting chemotherapy and Interleukin 2 Receives: Biological: Tumor Infiltrating Lymphocytes, Fludarabine, Cyclophosphamide, Interleukin-2 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety of TIL therapy in Pediatric Solid Tumors
The regimen will be declared safe if <33% of patients (0 or 1 out of maximum 6 patients on the safety phase) experience a dose-limiting toxicity (DLT).
Measured: 3 years
Secondary outcomes (3)
Feasibility of Generating TIL Product from Pediatric Solid Tumors
The TIL acquisition/production strategies will be considered feasible if 50% or more of all tumors collected on the prospective biobanking study generate a primary TIL culture that is satisfactory for rapid expansion for use on the Phase I trial.
Measured: 3 years
Toxicity of TIL Therapy in Pediatric Solid Tumors
Toxicities of all grades related to therapy and all Grade 3 and higher toxicities will be collected.
Measured: 3 years
Disease Response to TIL Therapy
For recurrent patients with measurable disease, Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 will be used. For all patients, event-free survival (EFS) will be reported.
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Sep 2023
- Study start2 Jun 2025
- Primary completion1 Sep 2029 (estimated)
- Study completion1 Sep 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for LymphocytesStudies 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- H. Lee Moffitt Cancer Center and Research Institute
- Sponsor type
- University, hospital or other
- Collaborators
- Swim Across America, Benjamin Gilkey Fund, Cannonball Kids' Cancer Foundation
- Responsible party
- Sponsor
- Investigators
- Jonathan Metts, MD, Principal investigator, Moffitt Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06047977
- Sponsor's protocol ID
- MCC-23265
- Other IDs
- IRB00486298
- Status last verified
- Sep 2026
- First submitted
- 15 Sep 2023
- Last updated
- 17 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Tumor Infiltrating Lymphocytes in Pediatric Malignant Solid Tumors: A Prospective Biobanking Study and Phase I Clinical Trial
- ClinicalTrials.gov ID
- NCT06047977
- Acronym / sponsor ID
- MCC-23265
- Conditions
- Lymphocytes
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- H. Lee Moffitt Cancer Center and Research InstituteUniversity, hospital or other
- Collaborators
- Swim Across America, Benjamin Gilkey Fund, Cannonball Kids' Cancer Foundation
- Study start
- 2 Jun 2025 (actual)
- Primary completion
- 1 Sep 2029 (estimated)
- Study completion
- 1 Sep 2029 (estimated)
- First posted
- 21 Sep 2023 (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.
Part One of this study will determine the feasibility of creating Tumor-Infiltrating Lymphocyte (TIL) product prospectively from high-risk pediatric solid tumors.
Part Two of this study will determine the safety of TIL therapy with lymphodepleting chemotherapy and post-TIL Interleukin-2 in high-risk pediatric solid tumors
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| TIL Therapy | Experimental | Biological: Tumor Infiltrating Lymphocytes, Fludarabine, Cyclophosphamide, Interleukin-2TIL therapy with lymphodepleting chemotherapy and Interleukin 2 |
Who may qualify
- Ages
- 1 year to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (48 listed)
- Patients must be ≥1 year to ≤ 21 years of age at time of enrollment to Part One.
- Initial therapy patients: Patients with a histologic diagnosis of high-risk pediatric malignant solid tumors (pMST), defined as a pediatric malignant solid tumor outside of the central nervous system, newly-diagnosed or being treated with initial therapy, with expected 5-year Event-Free Survival (EFS) <60%. Examples include:
- Relapsed/refractory/recurrent patients: patients must have a histologic diagnosis of pMST (may include diagnoses outside those identified in Part 1) that has relapsed after achieving remission or progressed after completion of all planned initial therapy.
- Patients must have an open surgical biopsy or resection planned (core needle/final needle biopsies are not allowed) for standard of care purposes, at some point in their initial or relapsed therapy for pMST.
- Note: The surgery for tissue procurement may occur at any time during initial therapy including up-front surgeries or after neoadjuvant therapies, including chemotherapy, immunotherapy, and radiation.
Key exclusion criteria (12 listed)
- Part One: There are no exclusion criteria
- Patients with active systemic infections requiring intravenous antibiotics
- Patients testing positive for HIV titer, hepatitis B surface antigen, human T-cell leukemia-lymphoma virus (HTLV) I or II antibody, or both rapid plasma regain (RPR) and fluorescent treponemal antibody (FTA) are excluded. Patients with hepatitis C antibody must have a negative (undetectable) viral load by polymerase chain reaction (PCR).
- Patients who are pregnant or nursing.
- Sexually active patients of reproductive potential are eligible if they have agreed to use an effective contraceptive method from the time of informed consent through the duration and for 1 month following completion of protocol treatment. The definition of an effective contraceptive method will be at the discretion of the institutional investigator.
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 Infiltrating Lymphocytes, Fludarabine, Cyclophosphamide, Interleukin-2Lymphodepleting Chemotherapy with Fludarabine/Cyclophosphamide followed by TIL and Post-TIL Interleukin-2 (IL-2) | Biological | TIL Therapy |
Primary outcome
Safety of TIL therapy in Pediatric Solid Tumors
Time frame: 3 years
The regimen will be declared safe if <33% of patients (0 or 1 out of maximum 6 patients on the safety phase) experience a dose-limiting toxicity (DLT).
3 secondary outcomes
Feasibility of Generating TIL Product from Pediatric Solid Tumors
Time frame: 3 years
Toxicity of TIL Therapy in Pediatric Solid Tumors
Time frame: 3 years
Disease Response to TIL Therapy
Time frame: 3 years
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 17 Sep 2026.
- 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.
Jonathan Metts, MD principal investigator
Moffitt Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Tumor Infiltrating Lymphocytes, Fludarabine, Cyclophosphamide, Interleukin-2 in people with Lymphocytes, run by H. Lee Moffitt Cancer Center and 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 48 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: looking for participants now, as of the sponsor's last update on 17 Sep 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?
Contact Jade Hanson (727-767-6468, Jade.Hanson@jhmi.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety of TIL therapy in Pediatric Solid Tumors (time frame: 3 years). 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 17 Sep 2026.