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BGJ398 for the Treatment of Tumor-Induced Osteomalacia

NCT03510455 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 4

Last updated 2021-04-08

Study results available
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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....

Conditions

  • Tumor-Induced Osteomalacia
  • Oncogenic Osteomalacia

Interventions

DRUG

BGJ398

BGJ398, a pan-fibroblast growth factor receptor (FGFR) kinase inhibitor will be orally administered over six 4-week cycles (4 weeks on drug continuously). After the initial dose, escalation/de- escalation of BGJ398 will be based on FGF- 23 blood levels and adjusted according to protocol procedures. The six cycles of BGJ398 will be followed by 3 months off the drug and an optional extension phase.

Sponsors & Collaborators

  • National Institute of Dental and Craniofacial Research (NIDCR)

    lead NIH

Principal Investigators

  • Rachel I Gafni, M.D. · National Institute of Dental and Craniofacial Research (NIDCR)

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
85 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-02-27
Primary Completion
2020-02-28
Completion
2020-05-04
FDA Drug
Yes

Countries

  • United States

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03510455 on ClinicalTrials.gov