BGJ398 for the Treatment of Tumor-Induced Osteomalacia
Stopped early · Phase 2
Conditions studied: Tumor-Induced Osteomalacia, Oncogenic Osteomalacia
In brief
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....
Key facts
- Study ID
- NCT03510455
- Run by
- National Institute of Dental and Craniofacial Research (NIDCR)
- People needed
- 4
- Starts
- 2019-02-27
- Expected to finish
- 2020-05-04
- Last updated by the study team
- 2021-04-08
Who can join
Age: 18 and older, up to 85. Sex: any. Healthy volunteers: not accepted.
You may not qualify if…
- 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 treatment with agents that are known strong inducers or inhibitors of CYP3A4 are prohibited. This includes treatment with enzyme-inducing antiepileptic drugs including carbamazepine, phenytoin, phenobarbital, and primidone.
- Consumption of grapefruit, grapefruit juice, pomegranates, star fruits, Seville oranges or products within 7 days prior to first dose
- Use of amiodarone within 90 days prior to first dose
- Current use of therapeutic doses of warfarin sodium or any other coumadin-derivative anticoagulants. Heparin and/or low molecular weight heparins are allowed.
- Insufficient bone marrow function defined as all of the following:
- ANC <1,500/mm\^3 [1.0 x 10\^9/L] AND
- Platelets < 75,000/mm\^3 [75 x 10\^9/L] AND
- Hemoglobin < 10.0 g/dL
- Insufficient hepatic and renal function defined as one of the following:
- Total bilirubin > 1.5x ULN OR
- AST/SGOT and ALT/SGPT > 2x ULN OR
- Blood creatinine > 1.5xULN and/or calculated eGFR < 45 ml/min/1.73 m\^2 (calculated by CKD-Epi)
- Clinically significant cardiac disease including any of the following:
- Congestive heart failure requiring treatment (NY Heart Association grade >= 2),
- History or presence of clinically significant ventricular arrhythmias, atrial fibrillation, resting bradycardia, or conduction abnormality
- Unstable angina pectoris or acute myocardial infarction less than or equal to 3 months prior to starting study drug
- QTcF > 450 msec (males); > 470 msec (females)
- History of congenital long QT syndrome
- Recent (less than or equal to 3 months) transient ischemic attack or stroke
Where it is running
- National Institutes of Health Clinical Center — Bethesda, Maryland, United States
Full record on ClinicalTrials.gov
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