Erdafitinib in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With FGFR Mutations (A Pediatric MATCH Treatment Trial)
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|ClinicalTrials.gov Identifier: NCT03210714|
Recruitment Status : Recruiting
First Posted : July 7, 2017
Last Update Posted : September 12, 2018
|Condition or disease||Intervention/treatment||Phase|
|Advanced Malignant Solid Neoplasm Ann Arbor Stage III Childhood Non-Hodgkin Lymphoma Ann Arbor Stage IV Childhood Non-Hodgkin Lymphoma FGFR1 Gene Mutation FGFR2 Gene Mutation FGFR3 Gene Mutation FGFR4 Gene Mutation Histiocytosis Low Grade Glioma Malignant Glioma Recurrent Central Nervous System Neoplasm Recurrent Childhood Ependymoma Recurrent Childhood Malignant Germ Cell Tumor Recurrent Childhood Medulloblastoma Recurrent Childhood Non-Hodgkin Lymphoma Recurrent Childhood Rhabdomyosarcoma Recurrent Childhood Soft Tissue Sarcoma Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor Recurrent Hepatoblastoma Recurrent Langerhans Cell Histiocytosis Recurrent Malignant Solid Neoplasm Recurrent Neuroblastoma Recurrent Osteosarcoma Refractory Central Nervous System Neoplasm Refractory Langerhans Cell Histiocytosis Refractory Malignant Solid Neoplasm Refractory Neuroblastoma Refractory Non-Hodgkin Lymphoma Rhabdoid Tumor Stage III Soft Tissue Sarcoma AJCC v7 Stage IV Soft Tissue Sarcoma AJCC v7 Wilms Tumor||Drug: Erdafitinib Other: Laboratory Biomarker Analysis Other: Pharmacological Study||Phase 2|
I. To determine the objective response rate (ORR; complete response + partial response) in pediatric patients treated with JNJ-42756493 (erdafitinib) with advanced solid tumors (including central nervous system [CNS] tumors), non-Hodgkin lymphomas or histiocytic disorders that harbor genetic alterations in the FGFR1/2/3/4 pathway.
I. To estimate the progression free survival in pediatric patients treated with JNJ-42756493 (erdafitinib) with advanced solid tumors (including CNS tumors), non-Hodgkin lymphomas or histiocytic disorders that harbor genetic alterations in the FGFR1/2/3/4.
II. To obtain information about the tolerability of JNJ-42756493 (erdafitinib) in children with relapsed or refractory cancer.
III. To provide preliminary estimates of the pharmacokinetics of JNJ-42756493 (erdafitinib) in children 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 erdafitinib orally (PO) once daily (QD) on days 1-28. Courses 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.
|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 JNJ-42756493 (Erdafitinib) in Patients With Tumors Harboring FGFR1/2/3/4 Alterations|
|Actual Study Start Date :||November 6, 2017|
|Estimated Primary Completion Date :||December 31, 2024|
|Estimated Study Completion Date :||December 31, 2024|
Experimental: Treatment (erdafitinib)
Patients receive erdafitinib PO QD on days 1-28. Courses repeat every 28 days for up to 2 years in the absence of disease progression or unacceptable toxicity.
Other Name: JNJ-42756493
Other: Laboratory Biomarker Analysis
Other: Pharmacological Study
- Response rate [ Time Frame: 3 years ]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.
- Incidence of adverse events graded by National Cancer Institute Common Terminology Criteria for Adverse Events version 4.0 [ Time Frame: Up to 3 years ]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.
- Progression free survival (PFS) [ Time Frame: From the initiation of protocol treatment to the occurrence of any of the following events: disease progression or disease recurrence or death from any cause, assessed up to 3 years ]PFS along with the confidence intervals will be estimated using the Kaplan-Meier method.
- Pharmacokinetic (PK) parameters [ Time Frame: Course 2 day 1 ]A descriptive analysis of PK parameters will be performed to define systemic exposure, drug clearance, and other pharmacokinetic parameters. The PK parameters will be summarized with simple summary statistics, including means, medians, ranges, and standard deviations (if numbers and distribution permit).
- Changes in tumor genomic profile [ Time Frame: Up to 3 years ]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): NCT03210714
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|Principal Investigator:||Alice Lee||Children's Oncology Group|