Texas Children's Cancer and Hematology Center

Clinical Trials and Novel Therapies

C7R-GD2.CAR T Cells for Patients with GD2-expressing Brain Tumors (GAIL-B)

In this study, a new gene will be added to the GD2 T cells that can cause the cells to live longer. T cells need substances called cytokines to survive. The gene C7R has been added that gives the cells a constant supply of cytokine and helps them to survive for a longer period of time.

GD2 Specific CAR and Interleukin-15 Expressing Autologous NKT Cells to Treat Children With Neuroblastoma (GINAKIT2)

This research study combines two different ways of fighting cancer: antibodies and Natural Killer T cells (NKT).

Vinblastine/Prednisone versus Single Therapy with Cytarabine for Langerhans Cell Histiocytosis (LCH)

The purpose of this research study is to compare previously used vinblastine/prednisone to single therapy with cytarabine for LCH.

CD30 CAR T Cells, Relapsed CD30 Expressing Lymphoma (RELY-30)

Investigators have found from previous research that they can put a new gene into T cells that will make them recognize cancer cells and kill them. They now want to test whether these genetically modified T cells given after chemotherapy will be more effective at killing cancer cells.

Autologous T-Cells Expressing a Second Generation CAR for Treatment of T-Cell Malignancies Expressing CD5 Antigen (MAGENTA)

In this study investigators will attach the CD5 chimeric receptor with CD28 added to it to the patient's T cells or the previous bone marrow transplant donor's T cells. The investigators will then test how long the cells last.

Trivalent CAR-T Cell in Acute B-Lineage Leukemia (TRICAR-ALL)

The antibody used in this study targets CD19, CD20 and CD22. This antibody sticks to ALL cells because of a substance on the outside of these cells called CD19, CD20 and/or CD22. For this study, the antibody to CD19, CD20 and CD22 has been changed so that instead of floating free in the blood, it is now joined to the T cells.

HER2 Chimeric Antigen Receptor (CAR) T Cells in Combination with Checkpoint Blockade in Patients With Advanced Sarcoma (HEROS 3.0)

The purpose of this study is to learn whether it is safe to give HER2-CAR T cells in combination with an immune checkpoint inhibitor drug (pembrolizumab or nivolumab), to learn what the side effects are, and to see whether this therapy might help patients with sarcoma.

Cobimetinib in Refractory Langerhans Cell Histiocytosis (LCH), and Other Histiocytic Disorders (NACHO COBI)

The purpose of this research study is to learn whether cobimetinib is safe and effective in subjects diagnosed with LCH, LCH-ND, RDD, JXG and ECD which may have a specific mutation called BRAF-V600E.

Cell Therapy for High Risk T-Cell Malignancies Using CD7-Specific CAR Expressed On Autologous T Cells (CRIMSON)

In this study, investigators attach the CD7 chimeric receptor with CD28 added to it to T cells. Investigators will then test how long the cells last. 

TCH Histiocytosis Biology Study (H-25667)

This study seeks to understand the biology of histiocytic disorders by analyzing tissue and clinical data from patients with histiocytic disorders. 

NACHO-BIO: Biology Study for Histiocytic Disorders

This study collects biological samples from patients with histiocytic disorders to understand the disease’s biology and identify biomarkers that could improve future diagnosis and treatment. 

Tovorafenib for Relapsed or Refractory Langerhans Cell Histiocytosis

This phase II trial tests the safety, side effects, best dose and activity of tovorafenib (DAY101) in treating patients with Langerhans cell histiocytosis that is growing, spreading, or getting worse (progressive), has come back (relapsed) after previous treatment, or does not respond to therapy (refractory).

U1 Mutation Registry for SHH Medulloblastoma (Children and Adults)

The purpose of this study is to create a biobank for patients diagnosed with Sonic Hedgehog Medulloblastoma at Baylor College of Medicine/Texas Children's Cancer Center.

TETRAVI Multivirus CTL for Treatment of EBV, CMV, Adenovirus, and BK Infections Post Allogeneic SCT

The purpose of this study is to use VSTs (virus-specific T cells) from a donor that is a partial HLA (human leukocyte antigen) match with the patient to treat viral infections after an allogeneic hematopoietic stem cell transplant (HSCT). These cells may also have value in CAR-T recipients who have received a product that depletes virus specific T cells.

EBV Specific T Lymphocytes for Treatment of EBV-Positive Lymphoma (CILESTE)

The purpose of this study is to find the largest safe dose of C7R-EBV T cells, and additionally to evaluate how long they can be detected in the blood and what affect they have on cancer.

Constitutive IL7R (C7R) Modified Banked Allogeneic CD30.CAR EBVSTS for CD30-Positive Lymphomas (CABAL2)

The aim is to study the safety and effectiveness of allogeneic banked CD30.CAR-EBVST cells that also carry the C7R molecule, to learn the side effects of C7R modified CD30.CAR-EBVST cells in lymphoma patients, and to see whether this therapy may help them.

Allogeneic CD30.CAR-EBVSTs in Patients with Relapsed or Refractory CD30-Positive Lymphomas

The purpose of this study is to find out the highest safe dose of allogeneic CD30.CAR-EBVST cells given following chemotherapy and used to treat lymphoma. The investigators will learn the side effects of CD30.CAR-EBVST cells in patients and see whether this therapy may help lymphoma patients.

Tazemetostat + Nivolumab/Ipilimumab for SMARCB1-Negative or SMARCA4-Deficient Tumors (TAZNI)

This research study involves a combination of three drugs given together as a possible treatment for malignant rhabdoid tumor, atypical teratoid rhabdoid tumor, epithelioid sarcoma, chordoma or other tumors that are deficient in one of two possible proteins, either INI-1 (SMARCB1) or SMARCA4.

UPDATE AML: A New Chemotherapy Approach for Pediatric Acute Myeloid Leukemia

This research study investigates the tolerability of substituting two cycles of chemotherapy into the standard pediatric acute myeloid leukemia (AML) chemotherapy treatment regimen for patients with newly diagnosed AML at intermediate-risk (IR) and high-risk (HR) of relapse.

A Phase 2 Study of Mutant-selective PI3Kα Inhibitor, RLY-2608, to treat PIK3CA Related Overgrowth Spectrum (PROS) and Malformations Driven by PIK3CA Mutation

This is a 3-part Phase 2 randomized study evaluating the safety and efficacy of the mutant-selective PI3Kα inhibitor, RLY-2608, in adults and children with PIK3CA Related Overgrowth Spectrum (PROS) and malformations driven by PIK3CA mutation.

A Phase 3 Study Evaluating QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Microcystic Lymphatic Malformations (SELVA)

The main purpose of this study is to assess the change in microcystic lymphatic malformations IGA after 24 weeks of treatment with QTORIN 3.9% Rapamycin Anhydrous Gel in approximately 40 participants with microcystic lymphatic malformations.

Alpelisib in Pediatric and Adult Patients with Lymphatic Malformations Associated with a PIK3CA Mutation (EPIK-L1)

The main purpose of this study in participants with PIK3CA-mutated LyM is to assess the change in radiological response and symptom severity upon treatment with alpelisib film-coated tablets (FCT) as compared to placebo.

Safety and Tolerability of Miransertib (MK-7075) in Participants with PIK3CA-related Overgrowth Spectrum or Proteus Syndrome Enrolled in Other Studies (MK-7075-006)

This is a study of the safety and tolerability of oral miransertib (MK-7075) administered to participants at least 2 years of age with phosphatidylinositol-4,5-bisphosphate 3-kinase catalytic subunit alpha (PIK3CA)-related overgrowth spectrum (PROS) or Proteus Syndrome (PS).

Study Assessing the Efficacy, Safety and PK of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum (EPIK-P2)

This is a prospective Phase II multi-center study with an initial 16-week, randomized, double-blind, placebo-controlled period, followed by two extension periods to assess the efficacy, safety and pharmacokinetics (PK) of alpelisib in pediatric and adult patients with PIK3CA-related overgrowth spectrum (PROS).

PEPN2312: Imetelstat with Fludarabine and Cytarabine for AML, MDS, or JMML

This phase I trial tests the safety, side effects, and best dose of imetelstat in combination with fludarabine and cytarabine in treating patients with acute myeloid leukemia (AML), myelodysplastic syndrome (MDS) or juvenile myelomonocytic leukemia (JMML) that is refractory or recurrent.

New Immunotherapy For Children With GPC3-Positive Solid Tumors Using IL-15 and IL-21 Armored GPC3-CAR T Cells (CARE)

This study will test the effect of 21.15.GPC3-CAR T cells in patients with solid tumors that express GPC3 on their surface.

Study of Onivyde With Talazoparib or Temozolomide in Children With Recurrent Solid Tumors and Ewing Sarcoma (ONITT)

The trial will test 2 combinations of therapy and participants will be randomly assigned to either Arm A or Arm B. The purpose of the phase I study is to determine the highest tolerable doses of the combinations of treatment given in each Arm.

Study Assessing the Efficacy, Safety and PK of Alpelisib (BYL719) in Pediatric and Adult Patients With PIK3CA-related Overgrowth Spectrum (EPIK-P2)

This is a prospective Phase II multi-center study with an initial 16-week, randomized, double-blind, placebo-controlled period, followed by two extension periods to assess the efficacy, safety and pharmacokinetics (PK) of alpelisib in pediatric and adult patients with PIK3CA-related overgrowth spectrum (PROS).

Evaluation of the Safety and Efficacy of ELA026 in Participants With Secondary Hemophagocytic Lymphohistiocytosis

The purpose of this study is to assess the safety, efficacy pharmacokinetics and pharmacodynamics of ELA026 in participants with sHLH.

Study to Evaluate the Efficacy, Safety and Tolerability of MAS825 in Patients With Monogenic IL-18 Driven Autoinflammatory Diseases, Including NLRC4-GOF, XIAP Deficiency, or CDC42 Mutations (MASter-1)

This study is a Phase 2 trial designed to evaluate the clinical efficacy, safety, and tolerability of MAS825 in patients with NLRC4-GOF, XIAP deficiency, or CDC42 mutations.

HER2-specific Chimeric Antigen Receptor (CAR) T Cells for Children with Ependymoma (PBTC-059)

This is a Phase I study to evaluate the safety profile of a type of immune therapy called HER2 CAR T cells. In addition to looking for side effects, we will study how well this treatment works against a brain tumor called ependymoma that has come back after treatment (recurrent) or has not responded well to treatment (progressive) in children.