Trial · NCT02915705
Efficacy and Safety of Burosumab Versus Oral Phosphate and Active Vitamin D Treatment in Pediatric Patients With XLH
NCT02915705, Phase 3, Completed, Kyowa Kirin, Inc.; results first posted 2019-04-11
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Efficacy and Safety of Burosumab Versus Oral Phosphate and Active Vitamin D Treatment in Pediatric Patients With XLH. Trials, record NCT02915705. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02915705
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Efficacy and Safety of Burosumab Versus Oral Phosphate and Active Vitamin D Treatment in Pediatric Patients With XLH
- NCT number
- NCT02915705
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 61 participants, actual
- Lead sponsor
- Kyowa Kirin, Inc.
- Conditions
- X-Linked Hypophosphatemia
- Interventions
- burosumab, Oral Phosphate Supplement, active vitamin D
- First posted
- 2016-09-27
- Last updated
- 2024-08-29
- Results first posted
- 2019-04-11
Source ClinicalTrials.gov
Registered outcomes32
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Radiographic Global Impression of Change (RGI-C) Global Score at Week 40 | Week 40 | Changes in the severity of rickets and bowing were assessed using a disease specific qualitative RGI-C scoring system. The RGI-C is a 7-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 | Percentage of Participants With a Mean RGI-C Global Score ≥ +2.0 (Responders) at Week 40 | Week 40 | RGI-C responders are defined as participants with a mean RGI-C global score \>= +2.0. The RGI-C is a 7-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 | Percentage of Participants With a Mean RGI-C Global Score ≥ +2.0 (Responders) at Week 64 | Week 64 | RGI-C responders are defined as participants with a mean RGI-C global score \>= +2.0. The RGI-C is a 7-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 Global Score at Week 64 | Week 64 | Changes in the severity of rickets and bowing were assessed using a disease specific qualitative RGI-C scoring system. The RGI-C is a 7-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 RSS Total Score at Week 40 | Baseline, Week 40 | 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, lucency, separation, 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 (the total score is the sum of the wrist and knee score). Higher scores indicate greater rickets severity. |
| Secondary | Change From Baseline in RSS Total Score at Week 64 | Baseline, Week 64 | 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 | RGI-C Long Leg Score at Week 40 | Week 40 | Changes in the severity of lower extremity skeletal abnormalities, including genu varum and genu valgus, were assessed using a disease specific qualitative RGI-C scoring system. The RGI-C is a 7-point ordinal scale with possible values: +3 = very much better (complete or near complete healing), +2 = much better (substantial healing), +1 = minimally better (i.e., minimal healing), 0 = unchanged, -1 = minimally worse (minimal worsening), -2 = much worse (moderate worsening), -3 = very much worse (severe worsening). |
| Secondary | RGI-C Long Leg Score at Week 64 | Week 64 | Changes in the severity of lower extremity skeletal abnormalities, including genu varum and genu valgus, were assessed using a disease specific qualitative RGI-C scoring system. The RGI-C is a 7-point ordinal scale with possible values: +3 = very much better (complete or near complete healing), +2 = much better (substantial healing), +1 = minimally better (i.e., minimal healing), 0 = unchanged, -1 = minimally worse (minimal worsening), -2 = much worse (moderate worsening), -3 = very much worse (severe worsening). |
| Secondary | Change From Baseline in Height-For-Age Z-Scores to Week 40 | Baseline, Week 40 | Recumbent length/Standing height z scores are measures of height adjusted for a child's age and sex. The Z-score indicates the number of standard deviations away from a reference population (from the Centers for Disease Control \[CDC\] growth charts) in the same age range and with the same sex. A Z-score of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher. Higher Z-scores indicate a better outcome. |
| Secondary | Change From Baseline in Height-For-Age Z-Scores to Week 64 | Baseline, Week 64 | Recumbent length/Standing height z scores are measures of height adjusted for a child's age and sex. The Z-score indicates the number of standard deviations away from a reference population (from the CDC growth charts) in the same age range and with the same sex. A Z-score of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher. Higher Z-scores indicate a better outcome. |
1 to 10 of 32Source ClinicalTrials.gov
See all 32 registered outcomesOutcomes32
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Radiographic Global Impression of Change (RGI-C) Global Score at Week 40 | Week 40 | Full Analysis Set: all randomized participants who received at least one dose of assigned medication and had at least one post-baseline assessment. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Percentage of Participants With a Mean RGI-C Global Score ≥ +2.0 (Responders) at Week 40 | Week 40 | Full Analysis Set: all randomized participants who received at least one dose of assigned medication and had at least one post-baseline assessment. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With a Mean RGI-C Global Score ≥ +2.0 (Responders) at Week 64 | Week 64 | Full Analysis Set: all randomized participants who received at least one dose of assigned medication and had at least one post-baseline assessment. | percentage of participants | NUMBER |
| SECONDARY | RGI-C Global Score at Week 64 | Week 64 | Full Analysis Set: all randomized participants who received at least one dose of assigned medication and had at least one post-baseline assessment. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in RSS Total Score at Week 40 | Baseline, Week 40 | Full Analysis Set: all randomized participants who received at least one dose of assigned medication and had at least one post-baseline RSS Total Score assessment. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in RSS Total Score at Week 64 | Baseline, Week 64 | Full Analysis Set: all randomized participants who received at least one dose of assigned medication and had at least one post-baseline RSS Total Score assessment. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | RGI-C Long Leg Score at Week 40 | Week 40 | Full Analysis Set: all randomized participants who received at least one dose of assigned medication and had at least one post-baseline assessment. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | RGI-C Long Leg Score at Week 64 | Week 64 | Full Analysis Set: all randomized participants who received at least one dose of assigned medication and had at least one post-baseline assessment. | score on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in Height-For-Age Z-Scores to Week 40 | Baseline, Week 40 | Full Analysis Set: all randomized participants who received at least one dose of assigned medication and had at least one post-baseline assessment. Participants with an assessment at Week 40. | Z score | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in Height-For-Age Z-Scores to Week 64 | Baseline, Week 64 | Full Analysis Set: all randomized participants who received at least one dose of assigned medication and had at least one post-baseline assessment. Participants with an assessment at Week 64. | Z score | LEAST_SQUARES_MEAN |
1 to 10 of 32Source ClinicalTrials.gov
See all 32 outcomesResults by group181
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Radiographic Global Impression of Change (RGI-C) Global Score at Week 40 | Active Control | 0.77 | Standard Error 0.107 | score on a scale | |
| Radiographic Global Impression of Change (RGI-C) Global Score at Week 40 | Burosumab | 1.92 | Standard Error 0.110 | score on a scale | |
| Percentage of Participants With a Mean RGI-C Global Score ≥ +2.0 (Responders) at Week 40 | Active Control | 6.3 | percentage of participants | ||
| Percentage of Participants With a Mean RGI-C Global Score ≥ +2.0 (Responders) at Week 40 | Burosumab | 72.4 | percentage of participants | ||
| Percentage of Participants With a Mean RGI-C Global Score ≥ +2.0 (Responders) at Week 64 | Active Control | 18.8 | percentage of participants | ||
| Percentage of Participants With a Mean RGI-C Global Score ≥ +2.0 (Responders) at Week 64 | Burosumab | 86.2 | percentage of participants | ||
| RGI-C Global Score at Week 64 | Active Control | 1.03 | Standard Error 0.136 | score on a scale | |
| RGI-C Global Score at Week 64 | Burosumab | 2.06 | Standard Error 0.072 | score on a scale | |
| Change From Baseline in RSS Total Score at Week 40 | Active Control | -0.71 | Standard Error 0.138 | score on a scale | |
| Change From Baseline in RSS Total Score at Week 40 | Burosumab | -2.04 | Standard Error 0.145 | score on a scale |
1 to 10 of 181Source ClinicalTrials.gov
See all 181 results by groupParticipant flow16
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Treatment Period | STARTED | Active Control | 32 | |
| Treatment Period | STARTED | Burosumab | 29 | |
| Treatment Period | Completed Week 40 | Active Control | 32 | |
| Treatment Period | Completed Week 40 | Burosumab | 29 | |
| Treatment Period | Completed Week 64 | Active Control | 32 | |
| Treatment Period | Completed Week 64 | Burosumab | 29 | |
| Treatment Period | COMPLETED | Active Control | 32 | |
| Treatment Period | COMPLETED | Burosumab | 29 | |
| Treatment Period | NOT COMPLETED | Active Control | 0 | |
| Treatment Period | NOT COMPLETED | Burosumab | 0 |
1 to 10 of 16Source ClinicalTrials.gov
See all 16 participant flowAdverse events360
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 | Tooth Hypoplasia | Congenital, familial and genetic disorders | Oral Phosphate/Active Vitamin D (Treatment Period) | 0 | 32 | |
| other | Tooth Hypoplasia | Congenital, familial and genetic disorders | Burosumab (Treatment Period) | 2 | 29 | |
| other | Tooth Hypoplasia | Congenital, familial and genetic disorders | Oral Phosphate/Active Vitamin D->Burosumab (Extension Period) | 0 | 26 | |
| other | Tooth Hypoplasia | Congenital, familial and genetic disorders | Burosumab->Burosumab (Treatment Period and Extension Period) | 2 | 29 | |
| other | Tooth Hypoplasia | Congenital, familial and genetic disorders | Total Burosumab (Treatment Period and Extension Period) | 2 | 55 | |
| other | Ear Discomfort | Ear and labyrinth disorders | Oral Phosphate/Active Vitamin D (Treatment Period) | 0 | 32 | |
| other | Ear Discomfort | Ear and labyrinth disorders | Burosumab (Treatment Period) | 1 | 29 | |
| other | Ear Discomfort | Ear and labyrinth disorders | Oral Phosphate/Active Vitamin D->Burosumab (Extension Period) | 0 | 26 | |
| other | Ear Discomfort | Ear and labyrinth disorders | Burosumab->Burosumab (Treatment Period and Extension Period) | 2 | 29 | |
| other | Ear Discomfort | Ear and labyrinth disorders | Total Burosumab (Treatment Period and Extension Period) | 2 | 55 |
1 to 10 of 360Source ClinicalTrials.gov
See all 360 adverse eventsAdverse event totals15
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Oral Phosphate/Active Vitamin D (Treatment Period) | 0 | 32 |
| Total, all-cause mortality | Burosumab (Treatment Period) | 0 | 29 |
| Total, all-cause mortality | Oral Phosphate/Active Vitamin D->Burosumab (Extension Period) | 0 | 26 |
| Total, all-cause mortality | Burosumab->Burosumab (Treatment Period and Extension Period) | 0 | 29 |
| Total, all-cause mortality | Total Burosumab (Treatment Period and Extension Period) | 0 | 55 |
| Total, other adverse events | Oral Phosphate/Active Vitamin D (Treatment Period) | 23 | 32 |
| Total, other adverse events | Burosumab (Treatment Period) | 21 | 29 |
| Total, other adverse events | Oral Phosphate/Active Vitamin D->Burosumab (Extension Period) | 9 | 26 |
| Total, other adverse events | Burosumab->Burosumab (Treatment Period and Extension Period) | 25 | 29 |
| Total, other adverse events | Total Burosumab (Treatment Period and Extension Period) | 34 | 55 |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 adverse event totalsPapers6
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 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.
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