Cabozantinib for Plexiform Neurofibromas (PN) in Subjects With NF1 in Children and Adults

PHASE2CompletedINTERVENTIONAL
Enrollment

45

Participants

Timeline

Start Date

June 30, 2014

Primary Completion Date

February 16, 2022

Study Completion Date

February 16, 2022

Conditions
NF1NeurofibromatosisPlexiform Neurofibromas
Interventions
DRUG

Cabozantinib

This is an open label Phase II clinical trial. For both cohorts, subjects will receive cabozantinib orally in continuous cycles. Each cycle is 28 days. In absence of progressive disease or dose limiting toxicity (DLT), subjects may continue therapy for a total of 24 cycles. Subjects with radiographic response (20% or greater reduction in tumor volume) at the end of 12 cycles can continue on therapy for up to an additional year. However, for Cohort A, subjects who do not achieve 15% reduction in tumor volume after 8 cycles will be considered treatment failure and taken off study. For Cohort B, the criteria that subjects who do not achieve 15% reduction by 8 cycles are considered treatment failure and taken off study was eliminated. Subjects will be carefully monitored for toxicities associated with cabozantinib.

Trial Locations (12)

10016

New York University Medical Center, New York

19096

Children's Hospital of Philadelphia, Philadelphia

20010

Children's National Medical Center, Washington D.C.

20892

National Cancer Institute (NCI), Bethesda

45229

Cincinnati Children's Hospital Medical Center, Cincinnati

46202

Indiana Unversity, Indianapolis

60611

Lurie Children's Hospital of Chicago, Chicago

60637

University of Chicago, Chicago

63110

Washington University - St. Louis, St Louis

84132

University of Utah, Salt Lake City

90027

Children's Hospital Los Angeles, Los Angeles

02115

Children' Hospital Boston and Massachusetts General Hospital, Boston

All Listed Sponsors
lead

University of Alabama at Birmingham

OTHER

NCT02101736 - Cabozantinib for Plexiform Neurofibromas (PN) in Subjects With NF1 in Children and Adults | Biotech Hunter | Biotech Hunter