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)

This Phase 1 clinical trial is exploring a next-generation CAR T cell therapy for children and young adults with GD2-positive brain tumors. By adding a constitutively active interleukin-7 receptor gene (C7R) to the CAR T cells, researchers aim to enhance the cells’ persistence in the body and improve their ability to fight tumors over time.  

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

Study aims to determine the optimal safe dosage of GD2-CAR NKT cells (GINAKIT cells), assess their impact on neuroblastoma tumors, duration of detection in patient blood, and their influence on neuroblastoma outcomes in children.

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

Study for patients newly diagnosed with Langerhan's cell histiocytosis to compare vinblastine/prednisone  versus cytarabine (LCH-REASON).

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

Phase 1 immunotherapy trial using next generation CD30.CAR T cells for refractory lymphoma (Hodgkin or non-Hodgkin, including ALCL) (RELY 30).

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

Phase 1 trial using next generation CD5.CAR T cells for recurrent T-cell leukemia/lymphoma (MAGENTA).

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

Phase 1 study of a novel trivalent CAR-T cell therapy for patients with refractory or recurrent B cell acute lymphoblastic leukemia (+CD19, +CD20 and/or +CD22)

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

Phase 1 immunotherapy trial for children with relapsed or refractory sarcoma using a combination of HER2-CAR T cells with checkpoint blockade (HEROS 3.0).

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

Frontline study of cobimetinib in patients with recurrent or refractory histiocytic diseases (NACHO COBI).

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

Phase 1 cell therapy for children with high-risk T-cell malignancies using CD7-specific CAR expressed on autologous T cells (CRIMSON).

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

A Phase I/II study testing a new drug combination for children and young adults with rare, aggressive tumors lacking SMARCB1 (INI1) or SMARCA4 proteins.

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

A pilot feasibility study testing new chemotherapy combinations for children and young adults with intermediate- or high-risk acute myeloid leukemia (AML)

A Phase 2 Study of Mutant-selective PI3Kα Inhibitor, RLY-2608, in Adults and Children with PIK3CA Related Overgrowth Spectrum and Malformations Driven by PIK3CA Mutation

This Phase 2 study evaluates the safety and effectiveness of the RLY-2608 medication in children and adults with PIK3CA-related overgrowth conditions.

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

This Phase 3 study evaluates the QTORIN 3.9% rapamycin gel topical treatment in individuals aged 3 years and older with microcystic lymphatic malformations.

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

This Phase 2/3 study evaluates the safety and effectiveness of the targeted medicine Alpelisib in children and adults with lymphatic malformations caused by PIK3CA mutations.

A Study of the Safety and Tolerability in Participants with PIK3CA-related Overgrowth Spectrum or Proteus Syndrome Who Are Being Treated with Miransertib (MK-7075) in Other Studies (MK-7075-006)

This Phase 2 study evaluates the targeted medicine miransertib in children and adults with PROS or Proteus Syndrome.

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

This Phase 2 study evaluates the targeted medicine Alpelisib in children and adults with symptomatic or progressive PIK3CA-Related Overgrowth Spectrum (PROS).

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

A Phase 1 Study of GRN163L (Imetelstat) in Combination with Fludarabine and Cytarabine for Patients with Acute Myeloid Leukemia that is in Second or Greater Relapse or that is Refractory to Relapse Therapy; Myelodysplastic Syndrome or Juvenile Myelomonocytic Leukemia in First or Greater Relapse or is Refractory to Relapse Therapy.

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

This clinical study will test the effect of 21.15.GPC3-CAR T cells in patients with solid tumors including Hepatoblastoma, Hepatocellular Carcinoma, Wilms Tumor, Malignant Rhabdoid Tumor, Yolk Sac Tumor, Rhabdomyosarcoma, Liposarcoma and Embryonal Sarcoma of Liver that express a molecule called GPC3 on their surface.

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

Phase 1 study of irinotecan liposomal with talazoparib or temozolomide in children with recurrent solid tumors or Ewing sarcoma (ONITT).

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

A Phase 2 study for alpelisib (BYL719) in pediatric and adult patients with PIK3CA-related overgrowth spectrum (EPIK-P2).

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

Evaluation of ELA026 monoclonal antibody for safety and efficacy as a treatment for patients with secondary hemophagocytic lymphohistiocytosis.

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)

Phase 2 trial for MAS825 in patients with monogenic IL-18 driven autoinflammatory diseases, including NLRC4-GOF, XIAP deficiency or CDC42 mutations (MASter-1).

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

A multi-center HER2-CAR T-cell immunotherapy trial for children with relapsed or refractory ependymoma (PBTC-059).