TerminatedPhase 2

BGJ398 for the Treatment of Tumor-Induced Osteomalacia

Stopped: Study was stopped early due to a greater than expected incidence of ocular AEs and analysis of the data from the first 4 subjects indicating that the likelihood of permanent remission with this therapy was low.

United States4 participantsStarted 2019-02-27

Plain-language summary

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....

Who can participate

Age range18 Years – 85 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

* INCLUSION CRITERIA: Patients eligible for inclusion in this study have to meet all of the following criteria: * Aged 18-85 years * Diagnosis of TIO due to a non-localized or unresectable tumor, or metastatic disease or resectable tumor that cannot be removed by minor surgical procedure. This diagnosis will be confirmed prior to enrollment on protocol 01-D-0184. Where clinically indicated, genetic testing to rule-out heritable causes of FGF23 excess will also be performed on 01-D-0184. * Willing and able to comply with scheduled visits, treatment plan and laboratory tests. * Able to swallow and retain oral medication. * Able to provide informed consent EXCLUSION CRITERIA: Patients eligible for this study must not meet any of the following criteria: * Have another genetic or secondary cause of hypophosphatemia. * History of any other malignancy that has not been cured/in remission for 5 years. * Patients who previously received treatment with an FGFR inhibitor other than BGJ398. * Current evidence of corneal or retinal disorder/keratopathy including, but not limited to: bullous/band keratopathy, corneal abrasion, inflammation/ulceration, keratoconjuctivitis, confirmed by ophthalmologic examination * Impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of oral BGJ398 (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, small bowel resection) * Patients who are currently receiving tre…

What they're measuring

Number of Participants With Complete Metabolic Remission After Stopping BGJ398

Timeframe: Up to 12 weeks after stopping BGJ398

Trial details

NCT IDNCT03510455

SponsorNational Institute of Dental and Craniofacial Research (NIDCR)

Sponsor typeNIH

Study typeINTERVENTIONAL

Primary completion2020-02-28

Results submitted2021-02-16