Selpercatinib for the Treatment of Advanced Solid Tumors, Lymphomas, or Histiocytic Disorders With Activating RET Gene Alterations, a Pediatric MATCH Treatment Trial
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|ClinicalTrials.gov Identifier: NCT04320888|
Recruitment Status : Not yet recruiting
First Posted : March 25, 2020
Last Update Posted : May 15, 2020
|Condition or disease||Intervention/treatment||Phase|
|Hematopoietic and Lymphoid Cell Neoplasm Recurrent Ependymoma Recurrent Ewing Sarcoma Recurrent Hepatoblastoma Recurrent Histiocytic and Dendritic Cell Neoplasm Recurrent Langerhans Cell Histiocytosis Recurrent Lymphoma Recurrent Malignant Germ Cell Tumor Recurrent Malignant Glioma Recurrent Malignant Solid Neoplasm Recurrent Medulloblastoma Recurrent Neuroblastoma Recurrent Non-Hodgkin Lymphoma Recurrent Osteosarcoma Recurrent Peripheral Primitive Neuroectodermal Tumor Recurrent Rhabdoid Tumor Recurrent Rhabdomyosarcoma Recurrent Soft Tissue Sarcoma Recurrent WHO Grade II Glioma Refractory Ependymoma Refractory Ewing Sarcoma Refractory Hepatoblastoma Refractory Histiocytic and Dendritic Cell Neoplasm Refractory Langerhans Cell Histiocytosis Refractory Lymphoma Refractory Malignant Germ Cell Tumor Refractory Malignant Glioma Refractory Malignant Solid Neoplasm Refractory Medulloblastoma Refractory Neuroblastoma Refractory Non-Hodgkin Lymphoma Refractory Osteosarcoma Refractory Peripheral Primitive Neuroectodermal Tumor Refractory Rhabdoid Tumor Refractory Rhabdomyosarcoma Refractory Soft Tissue Sarcoma Refractory WHO Grade II Glioma Wilms Tumor||Drug: Selpercatinib||Phase 2|
I. To determine the objective response rate (ORR; complete response + partial response) in pediatric patients treated with selpercatinib (LOXO-292) with advanced solid tumors (including central nervous system [CNS] tumors), lymphomas or histiocytic disorders that harbor activating genetic alterations in the RET pathway.
I. To estimate the progression free survival in pediatric patients treated with LOXO-292 with advanced solid tumors (including CNS tumors), lymphomas or histiocytic disorders that harbor activating genetic alterations in the RET pathway.
II. To obtain information about the tolerability of LOXO-292 in children and adolescents with relapsed or refractory cancer.
I. To explore approaches to profiling changes in tumor genomics over time through evaluation of circulating tumor deoxyribonucleic acid (DNA).
Patients receive selpercatinib orally (PO) twice daily (BID) on days 1-28. Treatment repeats every 28 days for up to 26 cycles (2 years) in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed for 30 days, then periodically thereafter.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||49 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 Subprotocol of LOXO-292 in Patients With Tumors Harboring RET Gene Alterations|
|Estimated Study Start Date :||October 30, 2020|
|Estimated Primary Completion Date :||September 30, 2027|
|Estimated Study Completion Date :||September 30, 2027|
Experimental: Treatment (selpercatinib)
Patients receive selpercatinib PO BID on days 1-28. Treatment repeats every 28 days for up to 26 cycles (2 years) in the absence of disease progression or unacceptable toxicity.
- Objective response rate (complete response + partial response) in pediatric patients treated with selpercatinib (LOXO-292) [ Time Frame: Up to completion of Pediatric MATCH Screening Trial (APEC1621) ]Will be determined by Response Evaluation Criteria in Solid Tumors. Response rates will be calculated as the percent of evaluable patients who are responders, and confidence intervals will be constructed using the Wilson score interval method.
- Progression-free survival (PFS) [ Time Frame: From the initiation of subprotocol (APEC1621N) treatment to the occurrence of any of the following events: disease progression or disease recurrence or death from any cause, assessed up to completion of Pediatric MATCH Screening Trial (APEC1621) ]PFS along with the confidence intervals will be estimated using the Kaplan-Meier method.
- Incidence of adverse events [ Time Frame: Up to completion of Pediatric MATCH Screening Trial (APEC1621) ]Evaluated by Common Terminology Criteria for Adverse Events version 5. Toxicity tables will be constructed to summarize the observed incidence by type of toxicity and grade. A patient will be counted only once for a given toxicity for the worst grade of that toxicity reported for that patient. Toxicity information recorded will include the type, severity, time of onset, time of resolution, and the probable association with the study regimen.
- Profiling changes in tumor genomics [ Time Frame: Up to time of disease progression or end of protocol therapy ]Will explore approaches to the profiling changes in tumor genomics over time through evaluation of circulating tumor deoxyribonucleic acid. A descriptive analysis will be performed and will be summarized with simple summary statistics. All of these analyses will be descriptive in nature.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04320888
|Principal Investigator:||Andrea T Franson||Children's Oncology Group|