Cabozantinib for Plexiform Neurofibromas (PN) in Subjects With NF1 in Children and Adults (NCT02101736) | Clinical Trial Compass
CompletedPhase 2
Cabozantinib for Plexiform Neurofibromas (PN) in Subjects With NF1 in Children and Adults
United States45 participantsStarted 2014-06
Plain-language summary
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.
Who can participate
Age range3 Years
SexALL
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Inclusion criteria
✓. Clinical or molecular diagnosis of Neurofibromatosis Type 1
✓. Plexiform neurofibroma that is progressive OR causing significant morbidity.
✓. Measurable disease amenable to volumetric MRI imaging defined as lesion seen on at least 3 consecutive MRI slices and at least 3 mL in volume. Select tumors \<3 cm may be eligible on review.
✓. Central review or MRI required prior to enrollment.
✓. Age ≥ 3 years of age at the time of study entry. Subjects ≥ 16 years will be enrolled in Cohort A. Subjects 3 - 15 years will be enrolled in Cohort B.
✓. Performance Level Karnofsky ≥ 50%. Subjects unable to walk because of paralysis, but up in a wheel chair will be considered ambulatory for purpose of assessing performance score.
✓. Complete resection of plexiform neurofibroma is not feasible or if subject refuses surgery.
✓. Fully recovered from acute toxic effects of all prior chemotherapy or radiotherapy.
Exclusion criteria
What they're measuring
1
The Change in Tumor Size Based on Radiographic Assessment
. Active optic glioma or other low-grade glioma requiring treatment with chemotherapy or radiation therapy.
✕. Malignant glioma, malignant peripheral nerve sheath tumor, or other malignancy requiring treatment in the last 12 months.
✕. Dental braces or prosthesis that interferes with volumetric analysis of the neurofibroma(s).
✕. Unable to swallow tablets.
✕. Women who are pregnant or breast-feeding.
✕. Subjects of reproductive potential who have not agreed to use effective contraception.
✕. Subject has not recovered to baseline or CTCAE ≤ Grade 1 from toxicity due to all prior therapies except alopecia and other non-clinically significant adverse events (AEs).
✕. Subject requires anticoagulants. Low dose aspirin, low-dose warfarin, and prophylactic low molecular weight heparin are permitted.