Trial · NCT02163577
Study of KRN23 (Burosumab), a Recombinant Fully Human Monoclonal Antibody Against Fibroblast Growth Factor 23 (FGF23), in Pediatric Subjects With X-linked Hypophosphatemia (XLH)
NCT02163577, Phase 2, Completed, Kyowa Kirin, Inc.; results first posted 2019-05-22
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of KRN23 (Burosumab), a Recombinant Fully Human Monoclonal Antibody Against Fibroblast Growth Factor 23 (FGF23), in Pediatric Subjects With X-linked Hypophosphatemia (XLH). Trials, record NCT02163577. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02163577
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of KRN23 (Burosumab), a Recombinant Fully Human Monoclonal Antibody Against Fibroblast Growth Factor 23 (FGF23), in Pediatric Subjects With X-linked Hypophosphatemia (XLH)
- NCT number
- NCT02163577
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 52 participants, actual
- Lead sponsor
- Kyowa Kirin, Inc.
- Conditions
- X-linked Hypophosphatemia
- Interventions
- burosumab
- First posted
- 2014-06-13
- Last updated
- 2024-05-06
- Results first posted
- 2019-05-22
Source ClinicalTrials.gov
Registered outcomes29
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline in RSS Total Score Over Time | Baseline, Week 40, 64, 160 | The RSS system is a 10-point radiographic scoring method that was developed to assess the severity of nutritional rickets in the wrists and knees based on the degree of metaphyseal fraying, cupping, and the proportion of the growth plate affected. Scores are assigned for the unilateral wrist and knee X-rays deemed by the rater to be the more severe of the bilateral images. The maximum total score on the RSS is 10 points and the minimum score is 0, with a total possible score of 4 points for the wrists and 6 points for the knees. Higher scores indicate greater rickets severity. |
| Primary | Change From Baseline in Serum Phosphorus Over Time | Baseline, Week 40, 64, 160 | |
| Primary | Change From Baseline in Serum 1,25(OH)2D Over Time | Baseline, Week 40, 64, 160 | |
| Primary | Change From Baseline in TmP/GFR Over Time | Baseline, Week 40, 64, 160 | Data for urinary phosphorus and TRP were used in calculation TmP/GFR. |
| Secondary | Change From Baseline in RSS Knee Scores Over Time | Baseline, Week 40, 64, 160 | The RSS system is a 10-point radiographic scoring method that was developed to assess the severity of nutritional rickets in the wrists and knees based on the degree of metaphyseal fraying, cupping, and the proportion of the growth plate affected. Scores are assigned for the unilateral wrist and knee X-rays deemed by the rater to be the more severe of the bilateral images. The maximum total score on the RSS is 10 points and the minimum score is 0, with a total possible score of 4 points for the wrists and 6 points for the knees. Higher scores indicate greater rickets severity. |
| Secondary | Change From Baseline in RSS Wrist Scores Over Time | Baseline, Week 40, 64, 160 | The RSS system is a 10-point radiographic scoring method that was developed to assess the severity of nutritional rickets in the wrists and knees based on the degree of metaphyseal fraying, cupping, and the proportion of the growth plate affected. Scores are assigned for the unilateral wrist and knee X-rays deemed by the rater to be the more severe of the bilateral images. The maximum total score on the RSS is 10 points and the minimum score is 0, with a total possible score of 4 points for the wrists and 6 points for the knees. Higher scores indicate greater rickets severity. |
| Secondary | Radiographic Global Impression of Change (RGI-C) Global Scores Over Time | Baseline, Week 40, 64, 160 | Changes in the severity of rickets and bowing were assessed centrally by three independent pediatric radiologists contracted by a central imaging facility using a disease specific qualitative RGI-C scoring system. The RGI-C is a seven point ordinal scale with possible values: +3 = very much better (complete or near complete healing of rickets), +2 = much better (substantial healing of rickets), +1 = minimally better (i.e., minimal healing of rickets), 0 = unchanged, -1 = minimally worse (minimal worsening of rickets), -2 = much worse (moderate worsening of rickets), -3 = very much worse (severe worsening of rickets). |
| Secondary | RGI-C Knee Scores Over Time | Baseline, Week 40, 64, 160 | Changes in the severity of rickets and bowing were assessed centrally by three independent pediatric radiologists contracted by a central imaging facility using a disease specific qualitative RGI-C scoring system. The RGI-C is a seven point ordinal scale with possible values: +3 = very much better (complete or near complete healing of rickets), +2 = much better (substantial healing of rickets), +1 = minimally better (i.e., minimal healing of rickets), 0 = unchanged, -1 = minimally worse (minimal worsening of rickets), -2 = much worse (moderate worsening of rickets), -3 = very much worse (severe worsening of rickets). |
| Secondary | RGI-C Wrist Scores Over Time | Baseline, Week 40, 64, 160 | Changes in the severity of rickets and bowing were assessed centrally by three independent pediatric radiologists contracted by a central imaging facility using a disease specific qualitative RGI-C scoring system. The RGI-C is a seven point ordinal scale with possible values: +3 = very much better (complete or near complete healing of rickets), +2 = much better (substantial healing of rickets), +1 = minimally better (i.e., minimal healing of rickets), 0 = unchanged, -1 = minimally worse (minimal worsening of rickets), -2 = much worse (moderate worsening of rickets), -3 = very much worse (severe worsening of rickets). |
| Secondary | Change From Baseline in Growth Velocity Over Time | Baseline, Week 40, 64, 160 |
1 to 10 of 29Source ClinicalTrials.gov
See all 29 registered outcomesOutcomes29
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in RSS Total Score Over Time | Baseline, Week 40, 64, 160 | Intent to Treat (ITT) Analysis Set: all participants who received at least 1 dose of study therapy and had at least 1 post-dose measurement at given time point. | score on a scale | LEAST_SQUARES_MEAN |
| PRIMARY | Change From Baseline in Serum Phosphorus Over Time | Baseline, Week 40, 64, 160 | Pharmacokinetic/Pharamcodynamic (PK/PD) Analysis Set: all participants who received at least 1 dose of therapy and had evaluable serum data at given time point. | mg/dL | MEAN |
| PRIMARY | Change From Baseline in Serum 1,25(OH)2D Over Time | Baseline, Week 40, 64, 160 | PK/PD Analysis Set: all participants who received at least 1 dose of therapy and had evaluable serum data at given time point. | pg/mL | MEAN |
| PRIMARY | Change From Baseline in TmP/GFR Over Time | Baseline, Week 40, 64, 160 | PK/PD Analysis Set: all participants who received at least 1 dose of therapy and had evaluable serum data at given time point. | mg/dL | MEAN |
| SECONDARY | Change From Baseline in RSS Knee Scores Over Time | Baseline, Week 40, 64, 160 | ITT Analysis Set: all participants who received at least 1 dose of study therapy and had at least 1 post-dose measurement at given time point. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in RSS Wrist Scores Over Time | Baseline, Week 40, 64, 160 | ITT Analysis Set: all participants who received at least 1 dose of study therapy and had at least 1 post-dose measurement at given time point. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Radiographic Global Impression of Change (RGI-C) Global Scores Over Time | Baseline, Week 40, 64, 160 | ITT Analysis Set: all participants who received at least 1 dose of study therapy and had at least 1 post-dose measurement at given time point. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | RGI-C Knee Scores Over Time | Baseline, Week 40, 64, 160 | ITT Analysis Set: all participants who received at least 1 dose of study therapy and had at least 1 post-dose measurement at given time point. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | RGI-C Wrist Scores Over Time | Baseline, Week 40, 64, 160 | ITT Analysis Set: all participants who received at least 1 dose of study therapy and had at least 1 post-dose measurement at given time point. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in Growth Velocity Over Time | Baseline, Week 40, 64, 160 | ITT Analysis Set: all participants who received at least 1 dose of study therapy and had at least 1 post-dose measurement at given time point. | cm/year | MEAN |
1 to 10 of 29Source ClinicalTrials.gov
See all 29 outcomesResults by group182
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in RSS Total Score Over Time | Burosumab Q2W | Change to Week 40 | -1.06 | Standard Error 0.100 | score on a scale |
| Change From Baseline in RSS Total Score Over Time | Burosumab Q4W Then Q2W | Change to Week 40 | -0.73 | Standard Error 0.100 | score on a scale |
| Change From Baseline in RSS Total Score Over Time | Burosumab Q2W | Change to Week 64 | -1.00 | Standard Error 0.110 | score on a scale |
| Change From Baseline in RSS Total Score Over Time | Burosumab Q4W Then Q2W | Change to Week 64 | -0.84 | Standard Error 0.098 | score on a scale |
| Change From Baseline in RSS Total Score Over Time | Burosumab Q2W | Change to Week 160 | -0.98 | Standard Error 0.129 | score on a scale |
| Change From Baseline in RSS Total Score Over Time | Burosumab Q4W Then Q2W | Change to Week 160 | -0.83 | Standard Error 0.122 | score on a scale |
| Change From Baseline in Serum Phosphorus Over Time | Burosumab Q2W | Change at Week 40 | 0.92 | Standard Deviation 0.480 | mg/dL |
| Change From Baseline in Serum Phosphorus Over Time | Burosumab Q4W Then Q2W | Change at Week 40 | 0.57 | Standard Deviation 0.265 | mg/dL |
| Change From Baseline in Serum Phosphorus Over Time | Burosumab Q2W | Change at Week 64 | 0.99 | Standard Deviation 0.502 | mg/dL |
| Change From Baseline in Serum Phosphorus Over Time | Burosumab Q4W Then Q2W | Change at Week 64 | 0.69 | Standard Deviation 0.370 | mg/dL |
1 to 10 of 182Source ClinicalTrials.gov
See all 182 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Burosumab Q2W | 26 | |
| Overall Study | STARTED | Burosumab Q4W Then Q2W | 26 | |
| Overall Study | COMPLETED | Burosumab Q2W | 26 | |
| Overall Study | COMPLETED | Burosumab Q4W Then Q2W | 26 | |
| Overall Study | NOT COMPLETED | Burosumab Q2W | 0 | |
| Overall Study | NOT COMPLETED | Burosumab Q4W Then Q2W | 0 |
Source ClinicalTrials.gov
Adverse events220
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 | Ear Pain | Ear and labyrinth disorders | Burosumab Q2W | 10 | 26 | |
| other | Ear Pain | Ear and labyrinth disorders | Burosumab Q4W Then Q2W | 8 | 26 | |
| other | Dry Eye | Eye disorders | Burosumab Q2W | 0 | 26 | |
| other | Dry Eye | Eye disorders | Burosumab Q4W Then Q2W | 3 | 26 | |
| other | Eye Pain | Eye disorders | Burosumab Q2W | 2 | 26 | |
| other | Eye Pain | Eye disorders | Burosumab Q4W Then Q2W | 2 | 26 | |
| other | Eye Pruritus | Eye disorders | Burosumab Q2W | 3 | 26 | |
| other | Eye Pruritus | Eye disorders | Burosumab Q4W Then Q2W | 2 | 26 | |
| other | Lacrimation Increased | Eye disorders | Burosumab Q2W | 1 | 26 | |
| other | Lacrimation Increased | Eye disorders | Burosumab Q4W Then Q2W | 3 | 26 |
1 to 10 of 220Source ClinicalTrials.gov
See all 220 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Burosumab Q2W | 0 | 26 |
| Total, all-cause mortality | Burosumab Q4W Then Q2W | 0 | 26 |
| Total, other adverse events | Burosumab Q2W | 26 | 26 |
| Total, other adverse events | Burosumab Q4W Then Q2W | 26 | 26 |
| Total, serious adverse events | Burosumab Q2W | 0 | 26 |
| Total, serious adverse events | Burosumab Q4W Then Q2W | 1 | 26 |
Source ClinicalTrials.gov
Papers4
The papers the trial lists on ClinicalTrials.gov.
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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Burosumab Q2W vs Burosumab Q4W Then Q2W: deaths from any cause | Trial comparisons |
| Burosumab Q2W vs Burosumab Q4W Then Q2W: serious adverse events | Trial comparisons |
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/NCT02163577
GET https://conductscience.org/api/trials/v1/NCT02163577/registered?page=2