Tumor-Induced Osteomalacia Clinical Trial
Official title:
BGJ398 for the Treatment of Tumor-Induced Osteomalacia
Background: People with tumor-induced osteomalacia (TIO) have small tumors that may cause low blood phosphorus, weak muscles, bone pain, and broken bones. The tumors may be so small they are hard to find or impossible to remove. Researchers want to test a drug that may help treat TIO. Objective: To see how the drug BGJ398 affects people with tumor-induced osteomalacia. Eligibility: People ages 18-85 who are in NIH protocol 01-D-0184 and have TIO that cannot be found or easily removed Design: At every study visit, participants will have: - Medical history - Physical exam - Blood and urine tests - Questions about their health and fatigue At the screening visit, participants will also have a heart and eye tests. They may have other tests to find their tumor. The baseline visit will be a 1-week stay in the clinic. Participants will have the regular study tests, plus: - Their first dose of the study drug capsules - Blood and urine collected every 2-4 hours for 24 hours. A thin plastic tube will be inserted in a vein to collect blood. - Heart and kidney ultrasounds - Activities that test strength - 6-minute walk test Participants will take the study drug for six 1-month cycles. In each cycle, participants will: - Take the study drug every day for 4 weeks. - Have 1 visit. Participants will collect their urine for 24 hours and have their blood drawn. Participants will have the regular study tests and repeat some baseline tests. - Have blood and urine tests at their local lab. Participants will have 1 visit at the end of the last cycle and another 3 months later....
Background: - Tumor-induced osteomalacia (TIO) is a rare disorder in which fibroblast growth factor (FGF23)-producing neoplasms cause renal phosphate wasting and skeletal disease. - Recent studies have shown that chromosomal translocations causing a fibronectin-FGFR1 (FN1/FGFR1) fusion gene have been identified in 40-60% of these tumors. - BGJ398 is an orally bio-available, selective and ATP competitive pan-fibroblast growth factor receptor (FGFR) kinase inhibitor which has demonstrated anti-tumor activity in preclinical, in vitro and in-vivo tumor models harboring FGFR genetic alterations. Objectives: To induce complete metabolic response in subjects with tumor-induced osteomalacia (TIO) with BGJ-398 as demonstrated by normalization of FGF23 and phosphate homeostasis. Eligibility: Patients may be eligible if they: - Are adults 18-85 years with documented evidence of TIO due to a non-localized or unresectable tumor, or metastatic disease, or resectable tumor that cannot be easily removed. - Are not taking any exclusionary medications or foods that may interfere with BGJ398. - Are not pregnant or nursing and are willing to use contraception (at least two forms of contraception), if able to become pregnant. - Have no significant ophthalmologic, gastrointestinal, renal, or hematologic disease. Design: - Phase 2, open-label, non-randomized, single-arm, drug treatment trial. - 10 subjects to be studied. - Treatment duration 6 months with 3 months off drug follow-up and optional extension phase. - Monthly NIH visits with additional labs obtained in between visits. - Imaging performed in those with identifiable tumors. - Analyses to include repeated measures ANOVA assessing changes in biochemical indices over time in response to BGJ398. ;
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