Trial · NCT02750618
Study of the Safety, Pharmacodynamics (PD) and Efficacy of KRN23 in Children From 1 to 4 Years Old With X-linked Hypophosphatemia (XLH)
NCT02750618, Phase 2, Completed, Kyowa Kirin, Inc.; results first posted 2018-06-19
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of the Safety, Pharmacodynamics (PD) and Efficacy of KRN23 in Children From 1 to 4 Years Old With X-linked Hypophosphatemia (XLH). Trials, record NCT02750618. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02750618
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of the Safety, Pharmacodynamics (PD) and Efficacy of KRN23 in Children From 1 to 4 Years Old With X-linked Hypophosphatemia (XLH)
- NCT number
- NCT02750618
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 13 participants, actual
- Lead sponsor
- Kyowa Kirin, Inc.
- Conditions
- X-Linked Hypophosphatemia
- Interventions
- Burosumab
- First posted
- 2016-04-25
- Last updated
- 2024-05-06
- Results first posted
- 2018-06-19
Source ClinicalTrials.gov
Registered outcomes13
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change From Baseline at Week 40 in Serum Phosphorus | Baseline, Week 40 | The Generalized Estimation Equation (GEE) model includes the change from baseline as the dependent variable, time as the categorical variable and adjusted for baseline measurement, with exchangeable covariance structure. |
| Primary | Number of Participants With Adverse Events (AEs), Treatment Emergent AEs (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | From first dose of study drug through the end of the study (Week 160). Maximum duration of exposure to study drug was 160 weeks. | An AE was defined as any untoward medical occurrence associated with the use of a drug, whether or not considered drug related. A serious AE was defined as an AE that at any dose, in the view of either the Investigator or Sponsor, results in any of the following outcomes: death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant incapacity or disability, a congenital anomaly/birth defect, or other important medical events (according to the investigator). An AE was considered a TEAE if it occurred on or after the first dose and was not present prior to the first dose, or it was present prior to the first dose but increased in severity during the study. Events were graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 4.0: grade 1 (mild), grade 2 (moderate), grade 3 (severe), grade 4 (life-threatening), grade 5 (death). |
| Secondary | Radiographic Global Impression of Change (RGI-C) Score at Week 40 | Week 40 | 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). The Analysis of Covariance (ANCOVA) model includes the RGI-C score as the dependent variable, age and RSS at baseline as covariates. |
| Secondary | RGI-C Score at Week 64 | Week 64 | 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). The GEE model includes the RGI-C score as the dependent variable, visit as a factor, age and RSS at baseline as covariates, with exchangeable covariance structure. |
| Secondary | Change From Baseline in Rickets at Week 40 as Assessed by the RSS Total Score | 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, 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.The ANCOVA model includes the RGI-C score as the dependent variable, age and RSS at baseline as covariates. |
| Secondary | Change From Baseline in Rickets at Week 64 as Assessed by the RSS Total Score | 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, with a total possible score of 4 points for the wrists and 6 points for the knees. Higher scores indicate greater rickets severity. The GEE model includes the change from baseline in RSS as the dependent variable, visit as a factor, age and RSS at baseline as covariates, with exchangeable covariance structure. |
| Secondary | RGI-C Lower Limb Deformity Score at Week 40 | Week 40 | Changes in the severity of lower extremity skeletal abnormalities 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). The ANCOVA model includes the RGI-C score as the dependent variable, age and RSS at baseline as covariates. |
| Secondary | RGI-C Lower Limb Deformity Score at Week 64 | Week 64 | Changes in the severity of lower extremity skeletal abnormalities 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). The GEE model includes the RGI-C score as the dependent variable, visit as a factor, age and RSS at baseline as covariates, with exchangeable covariance structure. |
| Secondary | Change From Baseline Over Time in Recumbent Length/Standing Height | Baseline, Weeks 12, 24, 40, 64, 88, 112, 136, 160 | |
| Secondary | Change From Baseline Over Time in Recumbent Length/Standing Height as Assessed by Height-for-Age Z-Scores | Baseline, Weeks 12, 24, 40, 64, 88, 112, 136, 160 | 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 13Source ClinicalTrials.gov
See all 13 registered outcomesOutcomes13
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline at Week 40 in Serum Phosphorus | Baseline, Week 40 | Pharmacokinetic/Pharmacodynamic (PK/PD) Analysis Set: all participants who received at least one dose of study drug and had evaluable blood samples. | mg/dL | LEAST_SQUARES_MEAN |
| PRIMARY | Number of Participants With Adverse Events (AEs), Treatment Emergent AEs (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | From first dose of study drug through the end of the study (Week 160). Maximum duration of exposure to study drug was 160 weeks. | Safety Analysis Set: all participants who received at least one dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Radiographic Global Impression of Change (RGI-C) Score at Week 40 | Week 40 | Efficacy Analysis Set: all participants who received at least one dose of study drug and had at least one post-study drug measurement. | units on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | RGI-C Score at Week 64 | Week 64 | Efficacy Analysis Set: all participants who received at least one dose of study drug and had at least one post-study drug measurement. | units on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in Rickets at Week 40 as Assessed by the RSS Total Score | Baseline, Week 40 | Efficacy Analysis Set: all participants who received at least one dose of study drug and had at least one post-study drug measurement. | units on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in Rickets at Week 64 as Assessed by the RSS Total Score | Baseline, Week 64 | Efficacy Analysis Set: all participants who received at least one dose of study drug and had at least one post-study drug measurement. | units on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | RGI-C Lower Limb Deformity Score at Week 40 | Week 40 | Efficacy Analysis Set: all participants who received at least one dose of study drug and had at least one post-study drug measurement. | units on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | RGI-C Lower Limb Deformity Score at Week 64 | Week 64 | Efficacy Analysis Set: all participants who received at least one dose of study drug and had at least one post-study drug measurement. | units on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline Over Time in Recumbent Length/Standing Height | Baseline, Weeks 12, 24, 40, 64, 88, 112, 136, 160 | Efficacy Analysis Set: all participants who received at least one dose of study drug and had at least one post-study drug measurement at given time point. | cm | MEAN |
| SECONDARY | Change From Baseline Over Time in Recumbent Length/Standing Height as Assessed by Height-for-Age Z-Scores | Baseline, Weeks 12, 24, 40, 64, 88, 112, 136, 160 | Efficacy Analysis Set: all participants who received at least one dose of study drug and had at least one post-study drug measurement at given time point. | Z score | MEAN |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 outcomesResults by group70
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline at Week 40 in Serum Phosphorus | Burosumab Q2W | 0.96 | Standard Error 0.117 | mg/dL | |
| Number of Participants With Adverse Events (AEs), Treatment Emergent AEs (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | Burosumab Q2W | Adverse Event Starting during Screening Period | 5 | Participants | |
| Number of Participants With Adverse Events (AEs), Treatment Emergent AEs (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | Burosumab Q2W | TEAEs | 13 | Participants | |
| Number of Participants With Adverse Events (AEs), Treatment Emergent AEs (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | Burosumab Q2W | Related TEAEs | 5 | Participants | |
| Number of Participants With Adverse Events (AEs), Treatment Emergent AEs (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | Burosumab Q2W | Serious TEAEs | 1 | Participants | |
| Number of Participants With Adverse Events (AEs), Treatment Emergent AEs (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | Burosumab Q2W | Serious Related TEAEs | 0 | Participants | |
| Number of Participants With Adverse Events (AEs), Treatment Emergent AEs (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | Burosumab Q2W | Grade 3 or 4 TEAE | 2 | Participants | |
| Number of Participants With Adverse Events (AEs), Treatment Emergent AEs (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | Burosumab Q2W | TEAE Leading to Study Discontinuation | 0 | Participants | |
| Number of Participants With Adverse Events (AEs), Treatment Emergent AEs (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | Burosumab Q2W | TEAE Leading to Treatment Discontinuation | 0 | Participants | |
| Number of Participants With Adverse Events (AEs), Treatment Emergent AEs (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | Burosumab Q2W | TEAE Leading to Death | 0 | Participants |
1 to 10 of 70Source ClinicalTrials.gov
See all 70 results by groupParticipant flow5
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Burosumab Q2W | 13 | |
| Overall Study | Completed Week 40 | Burosumab Q2W | 13 | |
| Overall Study | Completed Week 64 | Burosumab Q2W | 13 | |
| Overall Study | COMPLETED | Burosumab Q2W | 12 | |
| Overall Study | NOT COMPLETED | Burosumab Q2W | 1 |
Source ClinicalTrials.gov
Adverse events132
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 | Skull Malformation | Congenital, familial and genetic disorders | Burosumab Q2W | 1 | 13 | |
| other | Syringomyelia | Congenital, familial and genetic disorders | Burosumab Q2W | 1 | 13 | |
| other | Deafness Unilateral | Ear and labyrinth disorders | Burosumab Q2W | 1 | 13 | |
| other | Ear Haemorrhage | Ear and labyrinth disorders | Burosumab Q2W | 1 | 13 | |
| other | Ear Pain | Ear and labyrinth disorders | Burosumab Q2W | 4 | 13 | |
| other | Excessive Cerumen Production | Ear and labyrinth disorders | Burosumab Q2W | 1 | 13 | |
| other | Hypoacusis | Ear and labyrinth disorders | Burosumab Q2W | 1 | 13 | |
| other | Motion Sickness | Ear and labyrinth disorders | Burosumab Q2W | 1 | 13 | |
| other | Otorrhoea | Ear and labyrinth disorders | Burosumab Q2W | 1 | 13 | |
| other | Tinnitus | Ear and labyrinth disorders | Burosumab Q2W | 1 | 13 |
1 to 10 of 132Source ClinicalTrials.gov
See all 132 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Burosumab Q2W | 0 | 13 |
| Total, other adverse events | Burosumab Q2W | 13 | 13 |
| Total, serious adverse events | Burosumab Q2W | 1 | 13 |
Source ClinicalTrials.gov
Papers3
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.
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/NCT02750618
GET https://conductscience.org/api/trials/v1/NCT02750618/results?page=2