National Institute of Dental and Craniofacial Research (NIDCR)
Who can join
Adults 18 to 85, any sex, with Tumor-Induced Osteomalacia or Oncogenic Osteomalacia. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Number of Participants With Complete Metabolic Remission After Stopping BGJ398Primary· Up to 12 weeks after stopping BGJ398
Participants were monitored for up to 12 weeks after stopping BGJ398. A participant was considered to have a complete metabolic remission by achieving both normal blood FGF23 and phosphorus levels in the blood for 12 weeks after stopping BGJ398.
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
0
TIO Subjects Who Received BGJ398 (Single Arm)
4
Number of Participants With Complete and Partial Metabolic Response RateSecondary· Every 2 weeks up to 24 weeks
Participants were monitored for metabolic response to BGJ398 every other week. A participant was considered to have a complete metabolic response by achieving both normal c-terminal FGF23 and phosphorus levels at each time point. A participant was considered to have a partial metabolic response by achieving both a decrease of at least 50% in c-terminal FGF23 levels and an increase of at least 50% in phosphorous at each time point
Baseline
Group
Value
95% CI
Single Arm (TIO Subjects)
0
Single Arm (TIO Subjects)
0
Single Arm (TIO Subjects)
4
Week 2
Group
Value
95% CI
Single Arm (TIO Subjects)
2
Single Arm (TIO Subjects)
1
Single Arm (TIO Subjects)
1
Week 4
Group
Value
95% CI
Single Arm (TIO Subjects)
0
Single Arm (TIO Subjects)
1
Single Arm (TIO Subjects)
3
Week 6
Group
Value
95% CI
Single Arm (TIO Subjects)
2
Single Arm (TIO Subjects)
1
Single Arm (TIO Subjects)
1
Week 8
Group
Value
95% CI
Single Arm (TIO Subjects)
1
Single Arm (TIO Subjects)
1
Single Arm (TIO Subjects)
2
Week 10
Group
Value
95% CI
Single Arm (TIO Subjects)
0
Single Arm (TIO Subjects)
0
Single Arm (TIO Subjects)
4
Week 12
Group
Value
95% CI
Single Arm (TIO Subjects)
1
Single Arm (TIO Subjects)
1
Single Arm (TIO Subjects)
2
Week 14
Group
Value
95% CI
Single Arm (TIO Subjects)
1
Single Arm (TIO Subjects)
1
Single Arm (TIO Subjects)
2
Number of Participants With Grade 3 or 4 Adverse Events or Serious Adverse EventsSecondary· 24 week treatment phase followed by 3 month follow up or extension phase
Percentage of patients who incurred grade 3 or 4 adverse events (AEs) or serious adverse events (SAE) or AEs causing dose interruption/reduction
SAE
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
0
Grade 4 AE
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
0
Grade 3 AE
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
1
AE leading to dose interruption/reduction
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
4
Six-Minute Walk TestSecondary· Assessed at baseline and 24 weeks after starting BGJ398
The Six-Minute Walk Test (6MWT), is a self-paced practical test that measures the distance the patient can quickly cover on a flat, hard surface. Each subject was instructed to complete the maximum distance possible in six minutes. Feedback was given in two minute intervals and during the last 30 seconds. Patients were instructed to notify test administrator of leg cramping, pain, nausea, dizziness and shortness of breath. Test administrators counted each lap and upon test completion, the partial distance is measured and added to where the subject stopped.
Baseline
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
320.4
± 221.8
24 Weeks
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
496
± 173.4
Hand Grip Strength TestSecondary· Assessed at baseline and every 4 weeks up to 24 weeks during the treatment phase, and every 4 weeks in the follow up phase, up to 37 weeks
The person is seated, lower extremities flexed at 90 degrees. The individual uses the dominant hand for this activity. The individual sit with shoulder adducted and neutrally rotated elbow flexed at 90 degrees, forearm in neutral position, resting on the arm of a chair. The JAMAR Hand Dynamometer set to the third level position from the inside is used. The examiner lightly supports the readout dial to prevent it from dropping. The patient is asked to "Squeeze as hard as you can….squeeze…..squeeze….relax." Three successive trials is recorded in kilograms. A 10-15 second break occurs to prevent
Baseline
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
14.7
± 11.8
Week 4
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
16.4
± 11.2
Week 8
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
18.5
± 9.4
Week 12
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
17.0
± 7.9
Week 16
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
22.6
± 18.5
Week 20
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
17.3
± 6.6
Week 24
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
15.5
± 1.4
Week 28
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
30.1
± 6.7
Pinch TestSecondary· Assessed at baseline and every 4 weeks for the 24 treatment period, and every 4 weeks in the follow up phase, up to 37 weeks
A lateral pinch is required of the patient, using the dominant hand. The individual is seated, lower extremities flexed at 90 degrees. The examiner stabilizes the patient's wrist as the patient holds the pinch gauge to perform the test. The examiner requests the person to pinch with maximum strength. The peak-hold needle will automatically record the highest force exerted. After the patient uses the Pinch gauge, the examiner records the reading and resets the peak-hold needle to zero before testing again. Three lateral pinch trials are recorded and averaged for the final result.
Baseline
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
6.8
± 4.0
Week 4
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
6.7
± 1.2
Week 8
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
6.8
± 1.3
Week 12
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
7.5
± 2.0
Week 16
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
7.5
± 1.0
Week 20
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
7.7
± 1.5
Week 24
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
7.0
± 1.3
Week 28
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
8.1
± 1.9
Five Times Sit-to-Stand TestSecondary· Assessed at baseline and every 4 weeks during the 24 week treatment period
The Five Times Sit to Stand Test measures an aspect of transfer skill. The test provides a method to quantify functional lower extremity strength and/or identify movement strategies a patient uses to complete transitional movements.
The chair is free standing. Patient sits with arms folded across chest and with their back against the chair. A standard chair height 44 cm was used for each patient. Patient stands fully between repetitions of the test, careful to not touch the back of the chair during each repetition. Patient instructions: "I want you to stand up and sit down 5 times as quickly
Baseline
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
14.7
± 6.5
Week 4
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
13.2
± 4.0
Week 8
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
13.5
± 1.1
Week 12
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
11.2
± 0.7
Week 16
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
10.5
± 1.5
Week 20
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
10.8
± 0.8
Week 24
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
11.1
± 0.8
Week 28
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
11.1
± 1.0
The Disabilities of Arm Shoulder and Hand Outcome Measurement (DASH)Secondary· Assessed at baseline and every 4 weeks up to 24 weeks during the treatment phase, and every 4 weeks in the follow up phase, up to 37 weeks
The Disabilities of Arm Shoulder and Hand Outcome Measurement (DASH) The minimum score value is 0, maximum is 100, where a higher school represents a worse outcome (significant symptoms/disability)
Baseline
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
16.5
± 4.8
Week 4
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
13.3
± 6.8
Week 8
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
14.2
± 4.6
Week 12
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
13.3
± 7.3
Week 16
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
12.7
± 6.7
Week 20
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
21.0
± 23.1
Week 24
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
12.1
± 8.1
Week 28
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
10.0
± 2.3
RAND SF-36 Survey ResultsSecondary· Assessed at baseline and every 4 weeks up to 24 weeks during the treatment phase, and every 4 weeks in the follow up phase, up to 37 weeks
Evaluation using the RAND 36-Item Short Form Survey (SF-36). Results were separated into 8 domains - Physical Functioning, Role limitations due to physical health problems, Role limitations due to emotional problems, Emotional well-being, Energy/fatigue, Social functioning, Bodily Pain, and General health perceptions.
The minimum value is 0, the maximum value is 100, which the higher the score, the better the outcome.
Physical Functioning - Baseline
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
46.3
± 18.9
Physical Functioning - 4 weeks
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
47.5
± 21.0
Physical Functioning - 8 weeks
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
51.3
± 16.5
Physical Functioning - 12 weeks
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
47.5
± 12.6
Physical Functioning - 16 weeks
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
56.0
± 16.2
Physical Functioning - 20 weeks
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
52.5
± 26.0
Physical Functioning - 24 weeks
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
41.0
± 6.4
Physical Functioning - 28 weeks
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
65.0
± 7.1
PROMIS FatigueSecondary· Assessed at baseline and every 4 weeks up to 24 weeks during the treatment phase, and every 4 weeks in the follow up phase, up to 37 weeks
Assessed using the PROMIS (Patient-Reported Outcomes Measurement Information System) Fatigue 8A short form.
Scores are given as T values when compared to a population where the mean is 50 and 1SD is 10.
A higher score represents increased feelings of fatigue.
Baseline
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
55.1
± 8.1
Week 4
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
47.4
± 1.6
Week 8
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
52.6
± 7.9
Week 12
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
52.3
± 9.1
Week 16
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
52.3
± 9.1
Week 20
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
46.6
± 9.1
Week 24
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
52
± 4.9
Week 28
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
46.7
± 8.1
PROMIS Pain Interference ScoreSecondary· Assessed at baseline and every 4 weeks up to 24 weeks during the treatment phase, and every 4 weeks in the follow up phase, up to 37 weeks
Assessed using the PROMIS (Patient-Reported Outcomes Measurement Information System) Pain Interference 8A short form.
Scores are given as T values when compared to a population where the mean is 50 and 1SD is 10.
A higher score represents increased pain interference
Baseline
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
56.4
± 4.5
Week 4
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
52.6
± 9.4
Week 8
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
52.5
± 6.0
Week 12
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
55.7
± 4.2
Week 16
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
49.0
± 10.6
Week 20
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
48.2
± 15
Week 24
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
52.6
± 9.1
Week 28
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
40.7
± 0
PROMIS Mobility ScoreSecondary· Assessed at baseline and every 4 weeks up to 24 weeks during the treatment phase, and every 4 weeks in the follow up phase, up to 37 weeks
Assessed using the PROMIS (Patient-Reported Outcomes Measurement Information System) Mobility bank.
Scores are given as T values when compared to a population where the mean is 50 and 1SD is 10.
A higher score represents a better outcome (unencumbered mobility)
Baseline
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
38.2
± 6.7
Week 4
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
40.0
± 6.4
Week 8
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
39.3
± 5.3
Week 12
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
38.8
± 3.2
Week 16
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
40.2
± 4.5
Week 20
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
40.7
± 7.3
Week 24
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
39.5
± 2.6
Week 28
Group
Value
95% CI
TIO Subjects Who Received BGJ398 (Single Arm)
43.6
± 1.6
Adverse events — posted to ClinicalTrials.gov
Time frame: Patients were monitored for adverse events during 24 weeks of treatment followed by 3 month follow-up period, with optional extension phase up to 37 weeks. If a patient was withdrawn from the study, adverse event data was no longer collected. Subjects who required permanent discontinuation of BGJ398 therapy due to an adverse event continued to be followed in the study until the toxicity had resolved or up to 30 days after study drug discontinuation..
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
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....
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT03773302 — Phase 3 Study of BGJ398 (Oral Infigratinib) in First Line Cholangiocarcinoma With FGFR2 Gene Fusions/Translocations
· Phase 3
· terminated
NCT02706691 — BGJ398 in Treating Patients With FGFR Positive Recurrent Head and Neck Cancer
· Phase 2
· terminated
NCT02657486 — BGJ398 in Non-Muscle-Invasive Urothelial Carcinoma of the Bladder
· NA
· completed
NCT02257541 — BGJ398 in Combination With Imatinib Mesylate in Patients With Untreated Advanced Gastrointestinal Stromal Tumor (GIST)
· Phase 1, PHASE2
· completed
NCT02160041 — BGJ398 for Patients With Tumors With FGFR Genetic Alterations
· Phase 2
· terminated
Other recruiting trials for Tumor-Induced Osteomalacia
Currently open trials in the same condition.
NCT07366099 — Al18F-NOTA-LM3 PET/CT in Patients With TIO
· NA
· recruiting
Other National Institute of Dental and Craniofacial Research (NIDCR) trials
Trials by the same sponsor.
NCT07281456 — Safety of Tofacitinib, an Oral Janus Kinase Inhibitor, in Primary Sjogren Disease
· Phase 2
· recruiting
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by National Institute of Dental and Craniofacial Research (NIDCR)
Last refreshed: 8 April 2021
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03510455.