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

Part of paid clinical trials in Los Angeles, California.

Sponsor
University of Alabama at Birmingham
Study ID
NCT02101736
Phase
PHASE2
Status
Completed

Conditions

  • NF1
  • Neurofibromatosis
  • Plexiform Neurofibromas

Eligibility Criteria

Sex
ALL
Age
3 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Cabozantinib — DRUG
    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.

Study Details

This study, "A Phase II Study of Cabozantinib (XL l84) for Plexiform Neurofibromas in Subjects with Neurofibromatosis Type I in Children and Adults diagnosed with Neurofibromatosis Type 1 (NF1) and have a type of tumor called a plexiform neurofibroma (PN). Neurofibromas are tumors that develop from the cells and tissues that cover the nerves. Plexiform neurofibromas can be disfiguring, painful, and life-threatening. These types of tumors typically do not respond well to most treatment approaches such as chemotherapy, radiation, and surgery because of their slow growth and location near vital structures of the body such as nerves, blood vessels, and the airway. The primary objective is to determine the response rate of NF1 patients with plexiform neurofibromas treated with Cabozantinib therapy using MRI scans. The objective response rate to cabozantinib is defined as ≥ 20% reduction in tumor volume at the end of 12 cycles.

Key Dates

Start date
Jun 30, 2014
Status verified
May 2023
Primary completion
Feb 16, 2022
Completion
Feb 16, 2022

Study Design

Enrollment
45 participants (actual)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Experimental Agent XL184 (Cabozantinib)
    Cohort A (≥ 16 years - closed to accrual): Starting cabozantinib of 40 mg daily by mouth per cycle. Duration of each cycle is 28 days. Subjects will dose escalate after 2 cycles to 60 mg based on dose tolerability. Subjects who do not tolerate 40 mg will dose reduce to 20 mg. Doses will be capped at 60 mg. Cohort B (3 - 15 years). The starting cabozantinib dose is 30 mg/m2/day by mouth per cycle. Duration of each cycle is 28 days. Subjects will dose escalate after 2 cycles to 40 mg/m2/day based on dose tolerability. Subjects who do not tolerate 30 mg/m2/day will dose reduce to 23 mg/m2/day. Doses will be capped at 60 mg/day max daily dose Each cohort will enroll up to 24 evaluable subjects with a target minimum of 17 evaluable subjects per cohort.

Primary Outcome Measure

The Change in Tumor Size Based on Radiographic Assessment [ Time Frame: baseline to 12 Months ]

Locations (12)

FacilityCityStateZIPSite coordinators
Children's Hospital Los AngelesLos AngelesCalifornia90027-
Children's National Medical CenterWashington D.C.District of Columbia20010-
Lurie Children's Hospital of ChicagoChicagoIllinois60611-
University of ChicagoChicagoIllinois60637-
Indiana UnversityIndianapolisIndiana46202-
National Cancer Institute (NCI)BethesdaMaryland20892-
Children' Hospital Boston and Massachusetts General HospitalBostonMassachusetts02115-
Washington University - St. LouisSt LouisMissouri63110-
New York University Medical CenterNew YorkNew York10016-
Cincinnati Children's Hospital Medical CenterCincinnatiOhio45229-
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19096-
University of UtahSalt Lake CityUtah84132-

Find similar trials in Los Angeles, CA

By research site
Children's Hospital Los Angeles· Los Angeles, CAChildren's National Medical Center· Washington D.C., DCLurie Children's Hospital of Chicago· Chicago, ILUniversity of Chicago· Chicago, ILIndiana Unversity· Indianapolis, INNational Cancer Institute (NCI)· Bethesda, MD

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