Status and phase
Conditions
Treatments
About
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:
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:
Participants will take the study drug for six 1-month cycles. In each cycle, participants will:
Participants will have 1 visit at the end of the last cycle and another 3 months later....
Full description
Background:
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:
Design:
Enrollment
Sex
Ages
Volunteers
Inclusion and exclusion criteria
Patients eligible for inclusion in this study have to meet all of the following criteria:
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 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:
Insufficient hepatic and renal function defined as one of the following:
Clinically significant cardiac disease including any of the following:
Recent (less than or equal to 3 months) transient ischemic attack or stroke
Patients under age 21 will have a bone age assessed as part of their clinically indicated skeletal survey under 01-D-0184 and will not be offered enrollment if their growth plates are open.
Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test
Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing and for 3 months following the discontinuation of study treatment. Highly effective contraception methods include:
Total abstinence (when this is in line with the preferred and usual lifestyle of the subject. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception
Female sterilization (have had surgical bilateral oophorectomy with or without hysterectomy) or tubal ligation at least six weeks before taking study treatment. In case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment
Male sterilization (at least 6 months prior to screening). For female subjects on the study the vasectomized male partner should be the sole partner for that subject.
Combination of the following (a+b or a+c, or b+c):
Use of oral, injected or implanted hormonal methods of contraception or other forms of hormonal contraception that have comparable efficacy (failure rate <1%), for example hormone vaginal ring or transdermal hormone contraception
Placement of an intrauterine device (IUD) or intrauterine system (IUS)
Barrier methods of contraception: Condom or Occlusive cap (diaphragm or cervical/vault caps) with spermicidal foam/gel/film/cream/vaginal suppository
Primary purpose
Allocation
Interventional model
Masking
4 participants in 1 patient group
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Data sourced from clinicaltrials.gov
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