NCT03213691

Selumetinib Sulfate in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With Activating MAPK Pathway Mutations (A Pediatric MATCH Treatment Trial)

NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice) - Phase 2 Subprotocol of Selumetinib (AZD6244 Hydrogen Sulfate) in Patients With Tumors Harboring Activating MAPK Pathway Mutations

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 21 (actual)
• Sponsor: National Cancer Institute (NCI) · NIH
• Sex: All
• Age: 12 Months – 21 Years
• Healthy volunteers: Not accepted

Summary

This phase II Pediatric MATCH trial studies how well selumetinib sulfate works in treating patients with solid tumors, non-Hodgkin lymphoma, or histiocytic disorders with MAPK pathway activation mutations that have spread to other places in the body and have come back or do not respond to treatment. Selumetinib sulfate may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.

Detailed description

PRIMARY OBJECTIVE: I. To determine the objective response rate (ORR; complete response + partial response) in pediatric patients treated with selumetinib (AZD6244 hydrogen sulfate) with advanced solid tumors (including central nervous system \[CNS\] tumors), non-Hodgkin lymphomas or histiocytic disorders that harbor activating genetic alterations in the MAPK pathway. SECONDARY OBJECTIVES: I. To estimate the progression free survival in pediatric patients treated with selumetinib (AZD6244 hydrogen sulfate) with advanced solid tumors (including CNS tumors), non-Hodgkin lymphomas or histiocytic disorders that harbor MAPK activation mutations. II. To obtain additional information about the tolerability of selumetinib (AZD6244 hydrogen sulfate) in children with relapsed or refractory cancer. EXPLORATORY OBJECTIVES: I. To evaluate other biomarkers as predictors of response to selumetinib (AZD6244 hydrogen sulfate) and specifically, whether tumors that harbor different mutations or fusions will demonstrate differential response to selumetinib (AZD6244 hydrogen sulfate) treatment. II. To explore approaches to profiling changes in tumor genomics over time through evaluation of circulating tumor deoxyribonucleic acid (DNA). OUTLINE: Patients receive selumetinib sulfate 25 mg/m2/dose orally (PO) twice daily (BID) on days 1-28. Cycles repeat every 28 days for up to 2 years in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up periodically.

Conditions

• Advanced Malignant Solid Neoplasm
• Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma
• Ann Arbor Stage IV Childhood Non-Hodgkin Lymphoma
• Recurrent Childhood Central Nervous System Neoplasm
• Recurrent Childhood Non-Hodgkin Lymphoma
• Recurrent Malignant Solid Neoplasm
• Recurrent Neuroblastoma
• Refractory Malignant Solid Neoplasm
• Refractory Neuroblastoma
• Refractory Non-Hodgkin Lymphoma
• Refractory Primary Central Nervous System Neoplasm

Interventions

Type	Name	Description
OTHER	Laboratory Biomarker Analysis	Correlative studies
DRUG	Selumetinib	Given PO
DRUG	Selumetinib Sulfate	Given PO

Timeline

• Start date: 2017-10-30
• Primary completion: 2021-06-30
• Completion: 2024-12-31
• First posted: 2017-07-11
• Last updated: 2025-02-05
• Results posted: 2022-12-13

Locations

110 sites across 2 countries: United States, Puerto Rico

Regulatory

• FDA-regulated drug study