Trial · NCT03510455
BGJ398 for the Treatment of Tumor-Induced Osteomalacia
NCT03510455, Phase 2, Terminated, National Institute of Dental and Craniofacial Research (NIDCR); results first posted 2021-04-08
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. BGJ398 for the Treatment of Tumor-Induced Osteomalacia. Trials, record NCT03510455. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03510455
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- BGJ398 for the Treatment of Tumor-Induced Osteomalacia
- NCT number
- NCT03510455
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 4 participants, actual
- Lead sponsor
- National Institute of Dental and Craniofacial Research (NIDCR)
- Conditions
- Tumor-Induced Osteomalacia, Oncogenic Osteomalacia
- Interventions
- BGJ398
- First posted
- 2018-04-27
- Last updated
- 2026-11-10
- Results first posted
- 2021-04-08
- Limitations and caveats
This study was terminated early due to a lack of efficacy in the first 4 patients and an unexpectedly high rate of ocular adverse events. As a result, only 4 patients were enrolled in this study. Because of the small sample size, we were unable to perform the planned statistical analysis, and instead the results are primarily descriptive.
Source ClinicalTrials.gov
Registered outcomes20
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Complete Metabolic Remission After Stopping BGJ398 | 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. |
| Secondary | Number of Participants With Complete and Partial Metabolic Response Rate | 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 |
| Secondary | Number of Participants With Grade 3 or 4 Adverse Events or Serious Adverse Events | 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 |
| Secondary | Six-Minute Walk Test | 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. |
| Secondary | Hand Grip Strength Test | 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 muscle fatigue. Scores are recorded and averaged for the final result. Scores within two standard deviations of the mean are considered within normal limits. |
| Secondary | Pinch Test | 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. |
| Secondary | Five Times Sit-to-Stand Test | 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 as you can when I say Go." Timing begins at "Go" and ends when the buttocks touches the chair after the 5th repetition. The results are recorded in seconds. |
| Secondary | The Disabilities of Arm Shoulder and Hand Outcome Measurement (DASH) | 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) |
| Secondary | RAND SF-36 Survey Results | 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. |
| Secondary | PROMIS Fatigue | 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. |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 registered outcomesOutcomes20
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Complete Metabolic Remission After Stopping BGJ398 | Up to 12 weeks after stopping BGJ398 | The population consisted of all participants enrolled in the study | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Complete and Partial Metabolic Response Rate | Every 2 weeks up to 24 weeks | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Number of Participants With Grade 3 or 4 Adverse Events or Serious Adverse Events | 24 week treatment phase followed by 3 month follow up or extension phase | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Six-Minute Walk Test | Assessed at baseline and 24 weeks after starting BGJ398 | Meters | MEAN | |
| SECONDARY | Hand Grip Strength Test | 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 | Only 2 patients performed strength testing at 28 weeks. | kg | MEAN |
| SECONDARY | Pinch Test | 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 | Only 2 patients performed strength testing at 28 weeks. | kg | MEAN |
| SECONDARY | Five Times Sit-to-Stand Test | Assessed at baseline and every 4 weeks during the 24 week treatment period | Only 2 patients performed strength testing at 28 weeks. | seconds | MEAN |
| SECONDARY | The Disabilities of Arm Shoulder and Hand Outcome Measurement (DASH) | 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 | 2 patients did not fill out their DASH at week 28 | DASH Disability/Symptom Score | MEAN |
| SECONDARY | RAND SF-36 Survey Results | 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 | Only 2 patients completed week 28 surveys | score on a scale | MEAN |
| SECONDARY | PROMIS Fatigue | 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 | T score | MEAN |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 outcomesResults by group262
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Complete Metabolic Remission After Stopping BGJ398 | TIO Subjects Who Received BGJ398 (Single Arm) | Complete Metabolic Remission | 0 | Participants | |
| Number of Participants With Complete Metabolic Remission After Stopping BGJ398 | TIO Subjects Who Received BGJ398 (Single Arm) | No Complete Metabolic Remission | 4 | Participants | |
| Number of Participants With Complete and Partial Metabolic Response Rate | Single Arm (TIO Subjects) | Baseline / Complete Metabolic Response | 0 | Participants | |
| Number of Participants With Complete and Partial Metabolic Response Rate | Single Arm (TIO Subjects) | Baseline / Partial Metabolic Response | 0 | Participants | |
| Number of Participants With Complete and Partial Metabolic Response Rate | Single Arm (TIO Subjects) | Baseline / Neither Complete or Partial Metabolic Response | 4 | Participants | |
| Number of Participants With Complete and Partial Metabolic Response Rate | Single Arm (TIO Subjects) | Week 2 / Complete Metabolic Response | 2 | Participants | |
| Number of Participants With Complete and Partial Metabolic Response Rate | Single Arm (TIO Subjects) | Week 2 / Partial Metabolic Response | 1 | Participants | |
| Number of Participants With Complete and Partial Metabolic Response Rate | Single Arm (TIO Subjects) | Week 2 / Neither Complete or Partial Metabolic Response | 1 | Participants | |
| Number of Participants With Complete and Partial Metabolic Response Rate | Single Arm (TIO Subjects) | Week 4 / Complete Metabolic Response | 0 | Participants | |
| Number of Participants With Complete and Partial Metabolic Response Rate | Single Arm (TIO Subjects) | Week 4 / Partial Metabolic Response | 1 | Participants |
1 to 10 of 262Source ClinicalTrials.gov
See all 262 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | TIO Subjects Who Received BGJ398 (Single Arm) | 4 | |
| Overall Study | Treated | TIO Subjects Who Received BGJ398 (Single Arm) | 4 | |
| Overall Study | Extension Phase | TIO Subjects Who Received BGJ398 (Single Arm) | 1 | |
| Overall Study | Screening Failures | TIO Subjects Who Received BGJ398 (Single Arm) | 0 | |
| Overall Study | COMPLETED | TIO Subjects Who Received BGJ398 (Single Arm) | 3 | |
| Overall Study | NOT COMPLETED | TIO Subjects Who Received BGJ398 (Single Arm) | 1 |
Source ClinicalTrials.gov
Adverse events40
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Anemia | Blood and lymphatic system disorders | TIO Subjects Who Received BGJ398 (Single Arm) | 1 | 4 | |
| other | Ear Pain | Ear and labyrinth disorders | TIO Subjects Who Received BGJ398 (Single Arm) | 1 | 4 | |
| other | Hearing impairment | Ear and labyrinth disorders | TIO Subjects Who Received BGJ398 (Single Arm) | 1 | 4 | |
| other | Blurred vision | Eye disorders | TIO Subjects Who Received BGJ398 (Single Arm) | 4 | 4 | |
| other | Dry eye | Eye disorders | TIO Subjects Who Received BGJ398 (Single Arm) | 3 | 4 | |
| other | Corneal inflamation/keratitis | Eye disorders | TIO Subjects Who Received BGJ398 (Single Arm) | 4 | 4 | |
| other | Nuclear Sclerosis | Eye disorders | TIO Subjects Who Received BGJ398 (Single Arm) | 1 | 4 | |
| other | Glaucoma | Eye disorders | TIO Subjects Who Received BGJ398 (Single Arm) | 1 | 4 | |
| other | Hypertrichosis of eyelashes | Ear and labyrinth disorders | TIO Subjects Who Received BGJ398 (Single Arm) | 2 | 4 | |
| other | Rash, pustular | Infections and infestations | TIO Subjects Who Received BGJ398 (Single Arm) | 2 | 4 |
1 to 10 of 40Source ClinicalTrials.gov
See all 40 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | TIO Subjects Who Received BGJ398 (Single Arm) | 0 | 4 |
| Total, other adverse events | TIO Subjects Who Received BGJ398 (Single Arm) | 4 | 4 |
| Total, serious adverse events | TIO Subjects Who Received BGJ398 (Single Arm) | 0 | 4 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03510455
GET https://conductscience.org/api/trials/v1/NCT03510455/results?page=2