Trial Outcomes & Findings for BGJ398 for the Treatment of Tumor-Induced Osteomalacia (NCT NCT03510455)
NCT ID: NCT03510455
Last Updated: 2021-04-08
Results Overview
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.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
4 participants
Primary outcome timeframe
Up to 12 weeks after stopping BGJ398
Results posted on
2021-04-08
Participant Flow
Participant milestones
| Measure |
TIO Subjects Who Received BGJ398 (Single Arm)
TIO subjects were treated with BGJ398 for 24 weeks with optional extension phase
|
|---|---|
|
Overall Study
STARTED
|
4
|
|
Overall Study
Treated
|
4
|
|
Overall Study
Extension Phase
|
1
|
|
Overall Study
Screening Failures
|
0
|
|
Overall Study
COMPLETED
|
3
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
TIO Subjects Who Received BGJ398 (Single Arm)
TIO subjects were treated with BGJ398 for 24 weeks with optional extension phase
|
|---|---|
|
Overall Study
Physician Decision
|
1
|
Baseline Characteristics
BGJ398 for the Treatment of Tumor-Induced Osteomalacia
Baseline characteristics by cohort
| Measure |
Single Arm (TIO Subjects)
n=4 Participants
Phase 2, open-label, non-randomized, single-arm, drug treatment trial. 10 subjects will be studied. Treatment duration of 6 months with 3 months off drug follow-up and optional extension phase.
BGJ398: BGJ398, a pan-fibroblast growth factor receptor (FGFR) kinase inhibitor will be orally administered over six 4-week cycles (3 weeks on drug, 1 week off drug). 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.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
4 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
33.75 years
STANDARD_DEVIATION 7.8 • n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
4 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Type of TIO Disease
Localized
|
2 Participants
n=5 Participants
|
|
Type of TIO Disease
Non-localized
|
2 Participants
n=5 Participants
|
|
Type of TIO Disease
Metastatic
|
0 Participants
n=5 Participants
|
|
Blood Intact FGF23 on Initiation of Sutdy
|
1384 pg/mL
STANDARD_DEVIATION 1467 • n=5 Participants
|
|
Blood C-Terminal FGF23 on Initiation of Study
|
814 RU/mL
STANDARD_DEVIATION 911 • n=5 Participants
|
|
Blood Phosphate on Initiation of Study
|
1.6 mg/dL
STANDARD_DEVIATION 0.3 • n=5 Participants
|
|
Blood Alkaline Phosphatase on Initiation of Study
|
134 U/L
STANDARD_DEVIATION 54 • n=5 Participants
|
PRIMARY outcome
Timeframe: Up to 12 weeks after stopping BGJ398Population: The population consisted of all participants enrolled in the study
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.
Outcome measures
| Measure |
TIO Subjects Who Received BGJ398 (Single Arm)
n=4 Participants
TIO subjects were treated with BGJ398 for 24 weeks with optional extension phase
|
|---|---|
|
Number of Participants With Complete Metabolic Remission After Stopping BGJ398
Complete Metabolic Remission
|
0 Participants
|
|
Number of Participants With Complete Metabolic Remission After Stopping BGJ398
No Complete Metabolic Remission
|
4 Participants
|
SECONDARY outcome
Timeframe: Every 2 weeks up to 24 weeksParticipants 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
Outcome measures
| Measure |
TIO Subjects Who Received BGJ398 (Single Arm)
n=4 Participants
TIO subjects were treated with BGJ398 for 24 weeks with optional extension phase
|
|---|---|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Baseline · Complete Metabolic Response
|
0 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Baseline · Partial Metabolic Response
|
0 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Baseline · Neither Complete or Partial Metabolic Response
|
4 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 2 · Complete Metabolic Response
|
2 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 2 · Partial Metabolic Response
|
1 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 2 · Neither Complete or Partial Metabolic Response
|
1 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 4 · Complete Metabolic Response
|
0 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 4 · Partial Metabolic Response
|
1 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 4 · Neither Complete or Partial Metabolic Response
|
3 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 6 · Complete Metabolic Response
|
2 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 6 · Partial Metabolic Response
|
1 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 6 · Neither Complete or Partial Metabolic Response
|
1 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 8 · Complete Metabolic Response
|
1 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 8 · Partial Metabolic Response
|
1 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 8 · Neither Complete or Partial Metabolic Response
|
2 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 10 · Complete Metabolic Response
|
0 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 10 · Partial Metabolic Response
|
0 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 10 · Neither Complete or Partial Metabolic Response
|
4 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 12 · Complete Metabolic Response
|
1 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 12 · Partial Metabolic Response
|
1 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 12 · Neither Complete or Partial Metabolic Response
|
2 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 14 · Complete Metabolic Response
|
1 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 14 · Partial Metabolic Response
|
1 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 14 · Neither Complete or Partial Metabolic Response
|
2 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 16 · Complete Metabolic Response
|
0 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 16 · Partial Metabolic Response
|
1 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 16 · Neither Complete or Partial Metabolic Response
|
3 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 18 · Complete Metabolic Response
|
1 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 18 · Partial Metabolic Response
|
2 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 18 · Neither Complete or Partial Metabolic Response
|
1 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 20 · Complete Metabolic Response
|
1 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 20 · Partial Metabolic Response
|
1 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 20 · Neither Complete or Partial Metabolic Response
|
2 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 22 · Complete Metabolic Response
|
1 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 22 · Partial Metabolic Response
|
1 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 22 · Neither Complete or Partial Metabolic Response
|
2 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 24 - prior to drug discontinuation · Complete Metabolic Response
|
0 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 24 - prior to drug discontinuation · Partial Metabolic Response
|
0 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 24 - prior to drug discontinuation · Neither Complete or Partial Metabolic Response
|
4 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 24 - after drug discontinuation · Complete Metabolic Response
|
0 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 24 - after drug discontinuation · Partial Metabolic Response
|
0 Participants
|
|
Number of Participants With Complete and Partial Metabolic Response Rate
Week 24 - after drug discontinuation · Neither Complete or Partial Metabolic Response
|
4 Participants
|
SECONDARY outcome
Timeframe: 24 week treatment phase followed by 3 month follow up or extension phasePercentage of patients who incurred grade 3 or 4 adverse events (AEs) or serious adverse events (SAE) or AEs causing dose interruption/reduction
Outcome measures
| Measure |
TIO Subjects Who Received BGJ398 (Single Arm)
n=4 Participants
TIO subjects were treated with BGJ398 for 24 weeks with optional extension phase
|
|---|---|
|
Number of Participants With Grade 3 or 4 Adverse Events or Serious Adverse Events
SAE
|
0 Participants
|
|
Number of Participants With Grade 3 or 4 Adverse Events or Serious Adverse Events
Grade 4 AE
|
0 Participants
|
|
Number of Participants With Grade 3 or 4 Adverse Events or Serious Adverse Events
Grade 3 AE
|
1 Participants
|
|
Number of Participants With Grade 3 or 4 Adverse Events or Serious Adverse Events
AE leading to dose interruption/reduction
|
4 Participants
|
SECONDARY outcome
Timeframe: Assessed at baseline and 24 weeks after starting BGJ398The 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.
Outcome measures
| Measure |
TIO Subjects Who Received BGJ398 (Single Arm)
n=4 Participants
TIO subjects were treated with BGJ398 for 24 weeks with optional extension phase
|
|---|---|
|
Six-Minute Walk Test
Baseline
|
320.4 Meters
Standard Deviation 221.8
|
|
Six-Minute Walk Test
24 Weeks
|
496 Meters
Standard Deviation 173.4
|
SECONDARY outcome
Timeframe: 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 weeksPopulation: Only 2 patients performed strength testing at 28 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.
Outcome measures
| Measure |
TIO Subjects Who Received BGJ398 (Single Arm)
n=4 Participants
TIO subjects were treated with BGJ398 for 24 weeks with optional extension phase
|
|---|---|
|
Hand Grip Strength Test
Baseline
|
14.7 kg
Standard Deviation 11.8
|
|
Hand Grip Strength Test
Week 4
|
16.4 kg
Standard Deviation 11.2
|
|
Hand Grip Strength Test
Week 8
|
18.5 kg
Standard Deviation 9.4
|
|
Hand Grip Strength Test
Week 12
|
17.0 kg
Standard Deviation 7.9
|
|
Hand Grip Strength Test
Week 16
|
22.6 kg
Standard Deviation 18.5
|
|
Hand Grip Strength Test
Week 20
|
17.3 kg
Standard Deviation 6.6
|
|
Hand Grip Strength Test
Week 24
|
15.5 kg
Standard Deviation 1.4
|
|
Hand Grip Strength Test
Week 28
|
30.1 kg
Standard Deviation 6.7
|
SECONDARY outcome
Timeframe: Assessed at baseline and every 4 weeks for the 24 treatment period, and every 4 weeks in the follow up phase, up to 37 weeksPopulation: Only 2 patients performed strength testing at 28 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.
Outcome measures
| Measure |
TIO Subjects Who Received BGJ398 (Single Arm)
n=4 Participants
TIO subjects were treated with BGJ398 for 24 weeks with optional extension phase
|
|---|---|
|
Pinch Test
Baseline
|
6.8 kg
Standard Deviation 4.0
|
|
Pinch Test
Week 4
|
6.7 kg
Standard Deviation 1.2
|
|
Pinch Test
Week 8
|
6.8 kg
Standard Deviation 1.3
|
|
Pinch Test
Week 12
|
7.5 kg
Standard Deviation 2.0
|
|
Pinch Test
Week 16
|
7.5 kg
Standard Deviation 1.0
|
|
Pinch Test
Week 20
|
7.7 kg
Standard Deviation 1.5
|
|
Pinch Test
Week 24
|
7.0 kg
Standard Deviation 1.3
|
|
Pinch Test
Week 28
|
8.1 kg
Standard Deviation 1.9
|
SECONDARY outcome
Timeframe: Assessed at baseline and every 4 weeks during the 24 week treatment periodPopulation: Only 2 patients performed strength testing at 28 weeks.
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.
Outcome measures
| Measure |
TIO Subjects Who Received BGJ398 (Single Arm)
n=4 Participants
TIO subjects were treated with BGJ398 for 24 weeks with optional extension phase
|
|---|---|
|
Five Times Sit-to-Stand Test
Baseline
|
14.7 seconds
Standard Deviation 6.5
|
|
Five Times Sit-to-Stand Test
Week 4
|
13.2 seconds
Standard Deviation 4.0
|
|
Five Times Sit-to-Stand Test
Week 8
|
13.5 seconds
Standard Deviation 1.1
|
|
Five Times Sit-to-Stand Test
Week 12
|
11.2 seconds
Standard Deviation 0.7
|
|
Five Times Sit-to-Stand Test
Week 16
|
10.5 seconds
Standard Deviation 1.5
|
|
Five Times Sit-to-Stand Test
Week 20
|
10.8 seconds
Standard Deviation 0.8
|
|
Five Times Sit-to-Stand Test
Week 24
|
11.1 seconds
Standard Deviation 0.8
|
|
Five Times Sit-to-Stand Test
Week 28
|
11.1 seconds
Standard Deviation 1.0
|
SECONDARY outcome
Timeframe: 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 weeksPopulation: 2 patients did not fill out their DASH at week 28
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)
Outcome measures
| Measure |
TIO Subjects Who Received BGJ398 (Single Arm)
n=4 Participants
TIO subjects were treated with BGJ398 for 24 weeks with optional extension phase
|
|---|---|
|
The Disabilities of Arm Shoulder and Hand Outcome Measurement (DASH)
Baseline
|
16.5 DASH Disability/Symptom Score
Standard Deviation 4.8
|
|
The Disabilities of Arm Shoulder and Hand Outcome Measurement (DASH)
Week 4
|
13.3 DASH Disability/Symptom Score
Standard Deviation 6.8
|
|
The Disabilities of Arm Shoulder and Hand Outcome Measurement (DASH)
Week 8
|
14.2 DASH Disability/Symptom Score
Standard Deviation 4.6
|
|
The Disabilities of Arm Shoulder and Hand Outcome Measurement (DASH)
Week 12
|
13.3 DASH Disability/Symptom Score
Standard Deviation 7.3
|
|
The Disabilities of Arm Shoulder and Hand Outcome Measurement (DASH)
Week 16
|
12.7 DASH Disability/Symptom Score
Standard Deviation 6.7
|
|
The Disabilities of Arm Shoulder and Hand Outcome Measurement (DASH)
Week 20
|
21.0 DASH Disability/Symptom Score
Standard Deviation 23.1
|
|
The Disabilities of Arm Shoulder and Hand Outcome Measurement (DASH)
Week 24
|
12.1 DASH Disability/Symptom Score
Standard Deviation 8.1
|
|
The Disabilities of Arm Shoulder and Hand Outcome Measurement (DASH)
Week 28
|
10.0 DASH Disability/Symptom Score
Standard Deviation 2.3
|
SECONDARY outcome
Timeframe: 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 weeksPopulation: Only 2 patients completed week 28 surveys
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.
Outcome measures
| Measure |
TIO Subjects Who Received BGJ398 (Single Arm)
n=4 Participants
TIO subjects were treated with BGJ398 for 24 weeks with optional extension phase
|
|---|---|
|
RAND SF-36 Survey Results
Physical Functioning - Baseline
|
46.3 score on a scale
Standard Deviation 18.9
|
|
RAND SF-36 Survey Results
Physical Functioning - 4 weeks
|
47.5 score on a scale
Standard Deviation 21.0
|
|
RAND SF-36 Survey Results
Physical Functioning - 8 weeks
|
51.3 score on a scale
Standard Deviation 16.5
|
|
RAND SF-36 Survey Results
Physical Functioning - 12 weeks
|
47.5 score on a scale
Standard Deviation 12.6
|
|
RAND SF-36 Survey Results
Physical Functioning - 16 weeks
|
56.0 score on a scale
Standard Deviation 16.2
|
|
RAND SF-36 Survey Results
Physical Functioning - 20 weeks
|
52.5 score on a scale
Standard Deviation 26.0
|
|
RAND SF-36 Survey Results
Physical Functioning - 24 weeks
|
41.0 score on a scale
Standard Deviation 6.4
|
|
RAND SF-36 Survey Results
Physical Functioning - 28 weeks
|
65.0 score on a scale
Standard Deviation 7.1
|
|
RAND SF-36 Survey Results
Role limitations due to physical health problems - Baseline
|
43.8 score on a scale
Standard Deviation 12.5
|
|
RAND SF-36 Survey Results
Role limitations due to physical health problems - 4 weeks
|
93.8 score on a scale
Standard Deviation 12.5
|
|
RAND SF-36 Survey Results
Role limitations due to physical health problems - 8 weeks
|
68.8 score on a scale
Standard Deviation 31.5
|
|
RAND SF-36 Survey Results
Role limitations due to physical health problems - 12 weeks
|
50 score on a scale
Standard Deviation 35.4
|
|
RAND SF-36 Survey Results
Role limitations due to physical health problems - 16 weeks
|
50 score on a scale
Standard Deviation 57.7
|
|
RAND SF-36 Survey Results
Role limitations due to physical health problems - 20 weeks
|
75 score on a scale
Standard Deviation 50
|
|
RAND SF-36 Survey Results
Role limitations due to physical health problems - 24 weeks
|
25 score on a scale
Standard Deviation 50
|
|
RAND SF-36 Survey Results
Role limitations due to physical health problems - 28 weeks
|
12.5 score on a scale
Standard Deviation 17.7
|
|
RAND SF-36 Survey Results
Role limitations due to emotional problems - Baseline
|
66.7 score on a scale
Standard Deviation 27.2
|
|
RAND SF-36 Survey Results
Role limitations due to emotional problems - 4 weeks
|
83.3 score on a scale
Standard Deviation 33.3
|
|
RAND SF-36 Survey Results
Role limitations due to emotional problems - 8 weeks
|
75 score on a scale
Standard Deviation 31.9
|
|
RAND SF-36 Survey Results
Role limitations due to emotional problems - 12 weeks
|
66.7 score on a scale
Standard Deviation 47.1
|
|
RAND SF-36 Survey Results
Role limitations due to emotional problems - 16 weeks
|
50 score on a scale
Standard Deviation 43
|
|
RAND SF-36 Survey Results
Role limitations due to emotional problems - 20 weeks
|
91.7 score on a scale
Standard Deviation 16.7
|
|
RAND SF-36 Survey Results
Role limitations due to emotional problems - 24 weeks
|
41.7 score on a scale
Standard Deviation 50
|
|
RAND SF-36 Survey Results
Role limitations due to emotional problems - 28 weeks
|
33.3 score on a scale
Standard Deviation 47.1
|
|
RAND SF-36 Survey Results
Emotional well-being - Baseline
|
67 score on a scale
Standard Deviation 15.1
|
|
RAND SF-36 Survey Results
Emotional well-being - 4 weeks
|
72 score on a scale
Standard Deviation 15.7
|
|
RAND SF-36 Survey Results
Emotional well-being - 8 weeks
|
72 score on a scale
Standard Deviation 12.6
|
|
RAND SF-36 Survey Results
Emotional well-being - 12 weeks
|
72 score on a scale
Standard Deviation 17.6
|
|
RAND SF-36 Survey Results
Emotional well-being - 16 weeks
|
69.8 score on a scale
Standard Deviation 21.5
|
|
RAND SF-36 Survey Results
Emotional well-being - 20 weeks
|
73 score on a scale
Standard Deviation 8.2
|
|
RAND SF-36 Survey Results
Emotional well-being - 24 weeks
|
62 score on a scale
Standard Deviation 10.1
|
|
RAND SF-36 Survey Results
Emotional well-being - 28 weeks
|
72 score on a scale
Standard Deviation 5.7
|
|
RAND SF-36 Survey Results
Energy/fatigue - Baseline
|
50 score on a scale
Standard Deviation 29.4
|
|
RAND SF-36 Survey Results
Energy/fatigue - Week 4
|
58.8 score on a scale
Standard Deviation 8.5
|
|
RAND SF-36 Survey Results
Energy/fatigue - week 8
|
52.5 score on a scale
Standard Deviation 22.2
|
|
RAND SF-36 Survey Results
Energy/fatigue - week 12
|
46.3 score on a scale
Standard Deviation 28.4
|
|
RAND SF-36 Survey Results
Energy/fatigue - week 16
|
61.3 score on a scale
Standard Deviation 31.5
|
|
RAND SF-36 Survey Results
Energy/fatigue - week 20
|
57.5 score on a scale
Standard Deviation 18.5
|
|
RAND SF-36 Survey Results
Energy/fatigue - week 24
|
48.8 score on a scale
Standard Deviation 33.3
|
|
RAND SF-36 Survey Results
Energy/fatigue - week 28
|
47.5 score on a scale
Standard Deviation 53.0
|
|
RAND SF-36 Survey Results
Social functioning - Baseline
|
59.4 score on a scale
Standard Deviation 37.3
|
|
RAND SF-36 Survey Results
Social functioning - 4 weeks
|
87.5 score on a scale
Standard Deviation 14.4
|
|
RAND SF-36 Survey Results
Social functioning - 8 weeks
|
78.1 score on a scale
Standard Deviation 21.3
|
|
RAND SF-36 Survey Results
Social functioning - 12 weeks
|
81.3 score on a scale
Standard Deviation 16.1
|
|
RAND SF-36 Survey Results
Social functioning - 16 weeks
|
75 score on a scale
Standard Deviation 27
|
|
RAND SF-36 Survey Results
Social functioning - 20 weeks
|
78.1 score on a scale
Standard Deviation 25.8
|
|
RAND SF-36 Survey Results
Social functioning - 24 weeks
|
71.9 score on a scale
Standard Deviation 32.9
|
|
RAND SF-36 Survey Results
Social functioning - 28 weeks
|
75 score on a scale
Standard Deviation 17.7
|
|
RAND SF-36 Survey Results
Bodily Pain - Baseline
|
51.9 score on a scale
Standard Deviation 29.2
|
|
RAND SF-36 Survey Results
Bodily Pain - week 4
|
66.3 score on a scale
Standard Deviation 28
|
|
RAND SF-36 Survey Results
Bodily Pain - week 8
|
63.1 score on a scale
Standard Deviation 37.4
|
|
RAND SF-36 Survey Results
Bodily Pain - week 12
|
43.1 score on a scale
Standard Deviation 26.8
|
|
RAND SF-36 Survey Results
Bodily Pain - week 16
|
72.5 score on a scale
Standard Deviation 34.6
|
|
RAND SF-36 Survey Results
Bodily Pain - week 20
|
75 score on a scale
Standard Deviation 20.7
|
|
RAND SF-36 Survey Results
Bodily Pain - week 24
|
36.2 score on a scale
Standard Deviation 2.5
|
|
RAND SF-36 Survey Results
Bodily Pain - week 28
|
90 score on a scale
Standard Deviation 0
|
|
RAND SF-36 Survey Results
General health perceptions - Baseline
|
60 score on a scale
Standard Deviation 10.8
|
|
RAND SF-36 Survey Results
General health perceptions - week 4
|
68.8 score on a scale
Standard Deviation 13.1
|
|
RAND SF-36 Survey Results
General health perceptions - week 8
|
72.5 score on a scale
Standard Deviation 16.6
|
|
RAND SF-36 Survey Results
General health perceptions - week 12
|
73.8 score on a scale
Standard Deviation 25.9
|
|
RAND SF-36 Survey Results
General health perceptions - week 16
|
71.3 score on a scale
Standard Deviation 14.9
|
|
RAND SF-36 Survey Results
General health perceptions - week 20
|
67.5 score on a scale
Standard Deviation 23.3
|
|
RAND SF-36 Survey Results
General health perceptions - week 24
|
71.3 score on a scale
Standard Deviation 18.0
|
|
RAND SF-36 Survey Results
General health perceptions - week 28
|
67.5 score on a scale
Standard Deviation 31.8
|
SECONDARY outcome
Timeframe: 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 weeksAssessed 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.
Outcome measures
| Measure |
TIO Subjects Who Received BGJ398 (Single Arm)
n=4 Participants
TIO subjects were treated with BGJ398 for 24 weeks with optional extension phase
|
|---|---|
|
PROMIS Fatigue
Baseline
|
55.1 T score
Standard Deviation 8.1
|
|
PROMIS Fatigue
Week 4
|
47.4 T score
Standard Deviation 1.6
|
|
PROMIS Fatigue
Week 8
|
52.6 T score
Standard Deviation 7.9
|
|
PROMIS Fatigue
Week 12
|
52.3 T score
Standard Deviation 9.1
|
|
PROMIS Fatigue
Week 16
|
52.3 T score
Standard Deviation 9.1
|
|
PROMIS Fatigue
Week 20
|
46.6 T score
Standard Deviation 9.1
|
|
PROMIS Fatigue
Week 24
|
52 T score
Standard Deviation 4.9
|
|
PROMIS Fatigue
Week 28
|
46.7 T score
Standard Deviation 8.1
|
SECONDARY outcome
Timeframe: 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 weeksPopulation: Only 2 patients completed the week 28 surveys
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
Outcome measures
| Measure |
TIO Subjects Who Received BGJ398 (Single Arm)
n=4 Participants
TIO subjects were treated with BGJ398 for 24 weeks with optional extension phase
|
|---|---|
|
PROMIS Pain Interference Score
Baseline
|
56.4 T score
Standard Deviation 4.5
|
|
PROMIS Pain Interference Score
Week 4
|
52.6 T score
Standard Deviation 9.4
|
|
PROMIS Pain Interference Score
Week 8
|
52.5 T score
Standard Deviation 6.0
|
|
PROMIS Pain Interference Score
Week 12
|
55.7 T score
Standard Deviation 4.2
|
|
PROMIS Pain Interference Score
Week 16
|
49.0 T score
Standard Deviation 10.6
|
|
PROMIS Pain Interference Score
Week 20
|
48.2 T score
Standard Deviation 15
|
|
PROMIS Pain Interference Score
Week 24
|
52.6 T score
Standard Deviation 9.1
|
|
PROMIS Pain Interference Score
Week 28
|
40.7 T score
Standard Deviation 0
|
SECONDARY outcome
Timeframe: 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 weeksPopulation: Only 2 patients completed the week 28 surveys
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)
Outcome measures
| Measure |
TIO Subjects Who Received BGJ398 (Single Arm)
n=4 Participants
TIO subjects were treated with BGJ398 for 24 weeks with optional extension phase
|
|---|---|
|
PROMIS Mobility Score
Baseline
|
38.2 T score
Standard Deviation 6.7
|
|
PROMIS Mobility Score
Week 4
|
40.0 T score
Standard Deviation 6.4
|
|
PROMIS Mobility Score
Week 8
|
39.3 T score
Standard Deviation 5.3
|
|
PROMIS Mobility Score
Week 12
|
38.8 T score
Standard Deviation 3.2
|
|
PROMIS Mobility Score
Week 16
|
40.2 T score
Standard Deviation 4.5
|
|
PROMIS Mobility Score
Week 20
|
40.7 T score
Standard Deviation 7.3
|
|
PROMIS Mobility Score
Week 24
|
39.5 T score
Standard Deviation 2.6
|
|
PROMIS Mobility Score
Week 28
|
43.6 T score
Standard Deviation 1.6
|
SECONDARY outcome
Timeframe: Assessed at baseline and every 2 weeks up to 24 weeks during the treatment phase, and every 4 weeks in the follow up phase, up to 37 weeksPopulation: There were dose interruptions throughout the 24 week treatment phase for 3 of the 4 subjects. Only 3 patients provided follow up samples.
Measured Blood Intact FGF23 Levels.
Outcome measures
| Measure |
TIO Subjects Who Received BGJ398 (Single Arm)
n=4 Participants
TIO subjects were treated with BGJ398 for 24 weeks with optional extension phase
|
|---|---|
|
Blood Intact FGF23 Levels
Baseline
|
1384 pg/mL
Standard Deviation 1467
|
|
Blood Intact FGF23 Levels
4 Weeks
|
282 pg/mL
Standard Deviation 254
|
|
Blood Intact FGF23 Levels
8 Weeks
|
286 pg/mL
Standard Deviation 236
|
|
Blood Intact FGF23 Levels
12 Weeks
|
596 pg/mL
Standard Deviation 924
|
|
Blood Intact FGF23 Levels
16 Weeks
|
310 pg/mL
Standard Deviation 202
|
|
Blood Intact FGF23 Levels
20 Weeks
|
135 pg/mL
Standard Deviation 67
|
|
Blood Intact FGF23 Levels
24 Weeks
|
439 pg/mL
Standard Deviation 230
|
|
Blood Intact FGF23 Levels
Follow up
|
2534.8 pg/mL
Standard Deviation 2824.6
|
SECONDARY outcome
Timeframe: Assessed at baseline and every 2 weeks up to 24 weeks during the treatment phase, and every 4 weeks in the follow up phase, up to 37 weeksPopulation: There were dose interruptions throughout the 24 week treatment phase for 3 of the 4 subjects. Only 3 patients provided follow up samples. There is missing data for 1 patient at week 10 and 22.
Outcome measures
| Measure |
TIO Subjects Who Received BGJ398 (Single Arm)
n=4 Participants
TIO subjects were treated with BGJ398 for 24 weeks with optional extension phase
|
|---|---|
|
Blood C-terminal FGF23 Level
Baseline
|
814 RU/mL
Standard Deviation 911
|
|
Blood C-terminal FGF23 Level
2 Weeks
|
213 RU/mL
Standard Deviation 158
|
|
Blood C-terminal FGF23 Level
4 Weeks
|
330 RU/mL
Standard Deviation 87
|
|
Blood C-terminal FGF23 Level
6 Weeks
|
229 RU/mL
Standard Deviation 123
|
|
Blood C-terminal FGF23 Level
8 Weeks
|
304 RU/mL
Standard Deviation 154
|
|
Blood C-terminal FGF23 Level
10 Weeks
|
2225 RU/mL
Standard Deviation 2015
|
|
Blood C-terminal FGF23 Level
12 Weeks
|
430 RU/mL
Standard Deviation 409
|
|
Blood C-terminal FGF23 Level
14 Weeks
|
270 RU/mL
Standard Deviation 65
|
|
Blood C-terminal FGF23 Level
16 Weeks
|
360 RU/mL
Standard Deviation 75
|
|
Blood C-terminal FGF23 Level
18 Weeks
|
185 RU/mL
Standard Deviation 151
|
|
Blood C-terminal FGF23 Level
20 Weeks
|
274 RU/mL
Standard Deviation 118
|
|
Blood C-terminal FGF23 Level
22 Weeks
|
368 RU/mL
Standard Deviation 272
|
|
Blood C-terminal FGF23 Level
24 Weeks
|
463 RU/mL
Standard Deviation 102
|
|
Blood C-terminal FGF23 Level
Follow up
|
1568 RU/mL
Standard Deviation 1863
|
SECONDARY outcome
Timeframe: Assessed at baseline and every 2 weeks up to 24 weeks during the treatment phase, and every 4 weeks in the follow up phase, up to 37 weeksPopulation: There were dose interruptions throughout the 24 week treatment phase for 3 of the 4 subjects. Only 3 patients provided follow up samples. There is missing data for 1 patient at week 10 and 22.
Outcome measures
| Measure |
TIO Subjects Who Received BGJ398 (Single Arm)
n=4 Participants
TIO subjects were treated with BGJ398 for 24 weeks with optional extension phase
|
|---|---|
|
Blood Phosphate Levels
Baseline
|
1.6 mg/dL
Standard Deviation 0.3
|
|
Blood Phosphate Levels
Week 2
|
2.7 mg/dL
Standard Deviation 0.9
|
|
Blood Phosphate Levels
Week 4
|
2.9 mg/dL
Standard Deviation 1
|
|
Blood Phosphate Levels
Week 6
|
3.2 mg/dL
Standard Deviation 1.3
|
|
Blood Phosphate Levels
Week 8
|
3.6 mg/dL
Standard Deviation 1.5
|
|
Blood Phosphate Levels
Week 10
|
3.1 mg/dL
Standard Deviation 0.7
|
|
Blood Phosphate Levels
Week 12
|
2.9 mg/dL
Standard Deviation 1.2
|
|
Blood Phosphate Levels
Week 14
|
2.8 mg/dL
Standard Deviation 1.2
|
|
Blood Phosphate Levels
Week 16
|
3.4 mg/dL
Standard Deviation 1.6
|
|
Blood Phosphate Levels
Week 18
|
2.5 mg/dL
Standard Deviation 0.2
|
|
Blood Phosphate Levels
Week 20
|
3.0 mg/dL
Standard Deviation 1.3
|
|
Blood Phosphate Levels
Week 22
|
3.3 mg/dL
Standard Deviation 2.8
|
|
Blood Phosphate Levels
Week 24
|
3.0 mg/dL
Standard Deviation 1.2
|
|
Blood Phosphate Levels
Follow up
|
2.3 mg/dL
Standard Deviation 0.4
|
SECONDARY outcome
Timeframe: 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 weeksPopulation: There were dose interruptions throughout the 24 week treatment phase for 3 of the 4 subjects. Only 3 patients provided follow up samples. There is missing data for 1 patient at week 10 and 22.
Outcome measures
| Measure |
TIO Subjects Who Received BGJ398 (Single Arm)
n=4 Participants
TIO subjects were treated with BGJ398 for 24 weeks with optional extension phase
|
|---|---|
|
Blood 1,25-(OH)2-Vitamin D Level
Baseline
|
22.5 pg/mL
Standard Deviation 16.1
|
|
Blood 1,25-(OH)2-Vitamin D Level
Week 2
|
112.3 pg/mL
Standard Deviation 94.5
|
|
Blood 1,25-(OH)2-Vitamin D Level
Week 4
|
79 pg/mL
Standard Deviation 68.4
|
|
Blood 1,25-(OH)2-Vitamin D Level
Week 6
|
202.8 pg/mL
Standard Deviation 246.5
|
|
Blood 1,25-(OH)2-Vitamin D Level
Week 8
|
64.7 pg/mL
Standard Deviation 30.7
|
|
Blood 1,25-(OH)2-Vitamin D Level
Week 10
|
85.7 pg/mL
Standard Deviation 63.8
|
|
Blood 1,25-(OH)2-Vitamin D Level
Week 12
|
96.5 pg/mL
Standard Deviation 61.4
|
|
Blood 1,25-(OH)2-Vitamin D Level
Week 14
|
59.8 pg/mL
Standard Deviation 28.9
|
|
Blood 1,25-(OH)2-Vitamin D Level
Week 16
|
36 pg/mL
Standard Deviation 6.1
|
|
Blood 1,25-(OH)2-Vitamin D Level
Week 18
|
86.5 pg/mL
Standard Deviation 56.3
|
|
Blood 1,25-(OH)2-Vitamin D Level
Week 20
|
33 pg/mL
Standard Deviation 12.1
|
|
Blood 1,25-(OH)2-Vitamin D Level
Week 22
|
22.5 pg/mL
Standard Deviation 19.1
|
|
Blood 1,25-(OH)2-Vitamin D Level
Week 24
|
80.3 pg/mL
Standard Deviation 35.7
|
|
Blood 1,25-(OH)2-Vitamin D Level
Follow up
|
51 pg/mL
Standard Deviation 32.2
|
SECONDARY outcome
Timeframe: Assessed at baseline and every 2 weeks up to 24 weeks during the treatment phase, and every 4 weeks in the follow up phase, up to 37 weeksPopulation: There were dose interruptions throughout the 24 week treatment phase for 3 of the 4 subjects. Only 3 patients provided follow up samples. There is missing data for 1 patient at week 10 and 22.
Outcome measures
| Measure |
TIO Subjects Who Received BGJ398 (Single Arm)
n=4 Participants
TIO subjects were treated with BGJ398 for 24 weeks with optional extension phase
|
|---|---|
|
Blood Alkaline Phosphatase
Baseline
|
132 IU/L
Standard Deviation 52.3
|
|
Blood Alkaline Phosphatase
Week 2
|
178.8 IU/L
Standard Deviation 50.7
|
|
Blood Alkaline Phosphatase
Week 4
|
186.5 IU/L
Standard Deviation 42.1
|
|
Blood Alkaline Phosphatase
Week 6
|
198 IU/L
Standard Deviation 36.6
|
|
Blood Alkaline Phosphatase
Week 8
|
171.8 IU/L
Standard Deviation 26.6
|
|
Blood Alkaline Phosphatase
Week 10
|
181.3 IU/L
Standard Deviation 22
|
|
Blood Alkaline Phosphatase
Week 12
|
170 IU/L
Standard Deviation 18.4
|
|
Blood Alkaline Phosphatase
Week 14
|
188.3 IU/L
Standard Deviation 29.4
|
|
Blood Alkaline Phosphatase
Week 16
|
175.5 IU/L
Standard Deviation 14.3
|
|
Blood Alkaline Phosphatase
Week 18
|
183 IU/L
Standard Deviation 40.4
|
|
Blood Alkaline Phosphatase
Week 20
|
190.3 IU/L
Standard Deviation 33.6
|
|
Blood Alkaline Phosphatase
Week 22
|
200.7 IU/L
Standard Deviation 48
|
|
Blood Alkaline Phosphatase
Week 24
|
192.3 IU/L
Standard Deviation 67.4
|
|
Blood Alkaline Phosphatase
Follow up
|
185.7 IU/L
Standard Deviation 17
|
SECONDARY outcome
Timeframe: Assessed at baseline and every 2 weeks up to 24 weeks during the treatment phase, and every 4 weeks in the follow up phase, up to 37 weeksPopulation: There were dose interruptions throughout the 24 week treatment phase for 3 of the 4 subjects. Missing urine samples resulted in several time points having fewer than 4 subjects.
Tubular Reabsorption of Phosphate was calculated using blood phosphate and creatinine, as well as either 24 hour urine or spot urine samples.
Outcome measures
| Measure |
TIO Subjects Who Received BGJ398 (Single Arm)
n=4 Participants
TIO subjects were treated with BGJ398 for 24 weeks with optional extension phase
|
|---|---|
|
Tubular Reabsorption of Phosphate
Baseline
|
67 % Filtered Phosphate
Standard Deviation 20
|
|
Tubular Reabsorption of Phosphate
Week 2
|
82 % Filtered Phosphate
Standard Deviation 13
|
|
Tubular Reabsorption of Phosphate
Week 4
|
79 % Filtered Phosphate
Standard Deviation 14
|
|
Tubular Reabsorption of Phosphate
Week 6
|
84 % Filtered Phosphate
Standard Deviation 14
|
|
Tubular Reabsorption of Phosphate
Week 8
|
78 % Filtered Phosphate
Standard Deviation 19
|
|
Tubular Reabsorption of Phosphate
Week 10
|
50 % Filtered Phosphate
Standard Deviation 36
|
|
Tubular Reabsorption of Phosphate
Week 12
|
45 % Filtered Phosphate
Standard Deviation 36
|
|
Tubular Reabsorption of Phosphate
Week 14
|
82 % Filtered Phosphate
Standard Deviation 15
|
|
Tubular Reabsorption of Phosphate
Week 16
|
81 % Filtered Phosphate
Standard Deviation 15
|
|
Tubular Reabsorption of Phosphate
Week 18
|
77 % Filtered Phosphate
Standard Deviation 13
|
|
Tubular Reabsorption of Phosphate
Week 20
|
75 % Filtered Phosphate
Standard Deviation 12
|
|
Tubular Reabsorption of Phosphate
Week 22
|
81 % Filtered Phosphate
Standard Deviation 11
|
|
Tubular Reabsorption of Phosphate
Week 24
|
74 % Filtered Phosphate
Standard Deviation 19
|
SECONDARY outcome
Timeframe: Assessed at baseline and every 2 weeks up to 24 weeks during the treatment phase, and every 4 weeks in the follow up phase, up to 37 weeksPopulation: There were dose interruptions throughout the 24 week treatment phase for 3 of the 4 subjects. Missing urine samples resulted in several time points having fewer than 4 subjects.
Tubular maximum reabsorption rate of phosphate to glomerular filtration rate (TmP/GFR) was calculated using blood phosphate and creatinine, as well as either 24 hour urine or spot urine samples.
Outcome measures
| Measure |
TIO Subjects Who Received BGJ398 (Single Arm)
n=4 Participants
TIO subjects were treated with BGJ398 for 24 weeks with optional extension phase
|
|---|---|
|
Tubular Maximum Reabsorption Rate of Phosphate to Glomerular Filtration Rate (TmP/GFR)
Baseline
|
1.1 mg/dL
Standard Deviation 0.5
|
|
Tubular Maximum Reabsorption Rate of Phosphate to Glomerular Filtration Rate (TmP/GFR)
Week 2
|
2.5 mg/dL
Standard Deviation 1.2
|
|
Tubular Maximum Reabsorption Rate of Phosphate to Glomerular Filtration Rate (TmP/GFR)
Week 4
|
2.3 mg/dL
Standard Deviation 1.3
|
|
Tubular Maximum Reabsorption Rate of Phosphate to Glomerular Filtration Rate (TmP/GFR)
Week 6
|
3.5 mg/dL
Standard Deviation 2.2
|
|
Tubular Maximum Reabsorption Rate of Phosphate to Glomerular Filtration Rate (TmP/GFR)
Week 8
|
3.4 mg/dL
Standard Deviation 2.3
|
|
Tubular Maximum Reabsorption Rate of Phosphate to Glomerular Filtration Rate (TmP/GFR)
Week 10
|
1.32 mg/dL
Standard Deviation 0.9
|
|
Tubular Maximum Reabsorption Rate of Phosphate to Glomerular Filtration Rate (TmP/GFR)
Week 12
|
1.52 mg/dL
Standard Deviation 1.86
|
|
Tubular Maximum Reabsorption Rate of Phosphate to Glomerular Filtration Rate (TmP/GFR)
Week 14
|
2.76 mg/dL
Standard Deviation 1.61
|
|
Tubular Maximum Reabsorption Rate of Phosphate to Glomerular Filtration Rate (TmP/GFR)
Week 16
|
3.44 mg/dL
Standard Deviation 2.47
|
|
Tubular Maximum Reabsorption Rate of Phosphate to Glomerular Filtration Rate (TmP/GFR)
Week 18
|
1.98 mg/dL
Standard Deviation 0.34
|
|
Tubular Maximum Reabsorption Rate of Phosphate to Glomerular Filtration Rate (TmP/GFR)
Week 20
|
2.31 mg/dL
Standard Deviation 1.25
|
|
Tubular Maximum Reabsorption Rate of Phosphate to Glomerular Filtration Rate (TmP/GFR)
Week 22
|
3.86 mg/dL
Standard Deviation 2.99
|
|
Tubular Maximum Reabsorption Rate of Phosphate to Glomerular Filtration Rate (TmP/GFR)
Week 24
|
2.61 mg/dL
Standard Deviation 1.87
|
SECONDARY outcome
Timeframe: Baseline and at 24 weeksIn patients with radiographic evidence of TIO, 18FDG PET scans were performed
Outcome measures
| Measure |
TIO Subjects Who Received BGJ398 (Single Arm)
n=2 Participants
TIO subjects were treated with BGJ398 for 24 weeks with optional extension phase
|
|---|---|
|
Radiographic Evidence of Tumor-Induced Osteomalacia
Baseline SUVmax Tumor
|
7.9 Units
Standard Deviation 1.3
|
|
Radiographic Evidence of Tumor-Induced Osteomalacia
24 Week SUVmax tumor
|
3.9 Units
Standard Deviation 1.3
|
Adverse Events
TIO Subjects Who Received BGJ398 (Single Arm)
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
TIO Subjects Who Received BGJ398 (Single Arm)
n=4 participants at risk
TIO subjects were treated with BGJ398 for 24 weeks with optional extension phase
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
25.0%
1/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Ear and labyrinth disorders
Ear Pain
|
25.0%
1/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Ear and labyrinth disorders
Hearing impairment
|
25.0%
1/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Eye disorders
Blurred vision
|
100.0%
4/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Eye disorders
Dry eye
|
75.0%
3/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Eye disorders
Corneal inflamation/keratitis
|
100.0%
4/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Eye disorders
Nuclear Sclerosis
|
25.0%
1/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Eye disorders
Glaucoma
|
25.0%
1/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Ear and labyrinth disorders
Hypertrichosis of eyelashes
|
50.0%
2/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Infections and infestations
Rash, pustular
|
50.0%
2/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Infections and infestations
C. difficile colitis
|
25.0%
1/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Infections and infestations
Pelvic Infection
|
25.0%
1/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Investigations
Alanine aminotransferase increased
|
50.0%
2/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Investigations
Aspartate aminotransferase increased
|
25.0%
1/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Investigations
Platelet count decreased
|
50.0%
2/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Investigations
Lymphocyte count decreased
|
75.0%
3/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Investigations
Neutrophil count decreased
|
25.0%
1/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Investigations
Parathyroid hormone increased
|
75.0%
3/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Investigations
Creatinine increased
|
25.0%
1/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Investigations
Hypercalcemia
|
25.0%
1/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Investigations
Hypercalciuria
|
25.0%
1/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Investigations
Hyperphosphatemia
|
75.0%
3/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Investigations
Weight loss
|
25.0%
1/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Gastrointestinal disorders
Vomiting
|
50.0%
2/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Gastrointestinal disorders
Nausea
|
25.0%
1/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Gastrointestinal disorders
Mucositis
|
50.0%
2/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Gastrointestinal disorders
Dysgeusia
|
50.0%
2/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Gastrointestinal disorders
Dry mouth
|
75.0%
3/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Gastrointestinal disorders
Diarrhea
|
50.0%
2/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Gastrointestinal disorders
Gastroesophageal reflux
|
25.0%
1/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Musculoskeletal and connective tissue disorders
Pain, musculoskeletal
|
75.0%
3/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Nervous system disorders
Headache
|
25.0%
1/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Nervous system disorders
Paresthesia
|
25.0%
1/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Psychiatric disorders
Insomnia
|
25.0%
1/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
50.0%
2/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
25.0%
1/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Skin and subcutaneous tissue disorders
Dry lips
|
25.0%
1/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Skin and subcutaneous tissue disorders
Pain, nails
|
100.0%
4/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Skin and subcutaneous tissue disorders
Nail change/separation
|
100.0%
4/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
25.0%
1/4 • 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.
Adverse Events information was collected through investigator assessment every 4 weeks and labs every 2 weeks while on treatment. During the extension phase, adverse event information was collected through investigator and lab assessment every 4 weeks.
|
Additional Information
Rachel I. Gafni
National Institute of Dental and Craniofacial Research
Phone: 301-594-9924
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place