Trial · NCT02526160

Study of KRN23 in Adults With X-linked Hypophosphatemia (XLH)

NCT02526160, Phase 3, Completed, Kyowa Kirin, Inc.; results first posted 2021-02-05

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Study of KRN23 in Adults With X-linked Hypophosphatemia (XLH). Trials, record NCT02526160. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02526160

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Study of KRN23 in Adults With X-linked Hypophosphatemia (XLH)
NCT number: NCT02526160
Status: Completed
Phases: Phase 3
Enrollment: 134 participants, actual
Lead sponsor: Kyowa Kirin, Inc.
Conditions: X-linked Hypophosphatemia
Interventions: burosumab, Placebo
First posted: 2015-08-18
Last updated: 2024-06-18
Other registries' IDs: eudract:2014-005529-11
Results first posted: 2021-02-05

Source ClinicalTrials.gov

Registered outcomes33

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Percentage of Participants Achieving Mean Serum Phosphorus Levels Above the LLN (2.5 mg/dL [0.81 mmol/L]) at the Mid-Point of the Dose Interval, as Averaged Across Dose Cycles Between Baseline and Week 24	Baseline through Week 24
Secondary	Change From Baseline to Week 24 in Brief Pain Inventory (BPI) Question 3 (Q3; Worst Pain in Past 24 Hours) Score	Baseline, 24 weeks	The BPI evaluates the condition of all pain over the previous 24 hours. Two dimensions are measured: pain severity (worst, least, average, and now) and the impact of pain on functioning (pain interference with general activity, walking, work, mood, enjoyment of life, relations with others, and sleep). Question 3 of the short-form BPI (BPI-Q3) asks subjects to rate their pain at its worst in the last 24 hours on a scale of 0 (no pain) to 10 (pain as bad as you can imagine). From the generalized estimating equation (GEE) model, which includes the change from Baseline for the endpoint of interest as the dependent variable; region, visit, treatment, actual randomization stratification (not included for analysis of BPI Worst Pain), and visit by treatment as fixed factors; and Baseline value for the endpoint of interest as a covariate, with compound symmetry covariance structure.
Secondary	Change From Baseline to Week 24 in the Western Ontario and McMaster University Osteoarthritis Index (WOMAC) Stiffness Score	Baseline, 24 weeks	The WOMAC is a 24-item participant-reported questionnaire with two domains, Stiffness (2 questions) and Physical Function (17 questions) over the previous 48 hours. The WOMAC is administered in a 5-point Likert-scale format using descriptors of none, mild, moderate, severe, and extreme corresponding to an ordinal scale of 0-4. Higher scores on the WOMAC indicate worse stiffness and functional limitations. Scores are normalized to a 0-100 metric where 0 was the best health state and 100 the worst. The GEE estimates are from the GEE model which includes the change from baseline for WOMAC Stiffness as the dependent variable, region, visit, treatment, actual randomization stratification and visit by treatment as fixed factors, and baseline of WOMAC Stiffness as a covariate, with compound symmetry covariance structure.
Secondary	Change From Baseline to Week 24 in the WOMAC Physical Function Score	Baseline, 24 weeks	The WOMAC is a 24-item participant-reported questionnaire with two domains, Stiffness (2 questions) and Physical Function (17 questions) over the previous 48 hours. The WOMAC is administered in a 5-point Likert-scale format using descriptors of none, mild, moderate, severe, and extreme corresponding to an ordinal scale of 0-4. Higher scores on the WOMAC indicate worse stiffness and functional limitations. Scores are normalized to a 0-100 metric where 0 was the best health state and 100 the worst. The GEE Estimates are from the GEE model which includes the change from baseline for WOMAC Physical Function as the dependent variable, region, visit, treatment, actual randomization stratification and visit by treatment as fixed factors, and baseline of WOMAC Physical Function as a covariate, with compound symmetry covariance structure.
Secondary	Change From Baseline Over Time in BPI Worst Pain Score	Baseline, Weeks 12, 24, 36, 48, 72, 96	Change from baseline to post-baseline visits in BPI-Q3 (Worst Pain) score as averaged from daily diary scores recorded over 1 week and the study visit score. The BPI evaluates the condition of all pain over the previous 24 hours. Two dimensions are measured: pain severity (worst, least, average, and now) and the impact of pain on functioning (pain interference with general activity, walking, work, mood, enjoyment of life, relations with others, and sleep). Question 3 of the short-form BPI (BPI-Q3) asks subjects to rate their pain at its worst in the last 24 hours on a scale of 0 (no pain) to 10 (pain as bad as you can imagine). The GEE Estimates are from the GEE model which includes the change from baseline for BPI worst pain as the dependent variable, region, visit, treatment and visit by treatment as fixed factors, and baseline of BPI Worst Pain as a covariate, with compound symmetry covariance structure.
Secondary	Change From Baseline Over Time in BPI Pain Severity Score	Baseline, Weeks 12, 24, 36, 48, 72, 96	Change from baseline to post-baseline visits in BPI pain severity score as averaged from daily diary scores recorded over 1 week and the study visit score. The BPI evaluates the condition of all pain over the previous 24 hours. Two dimensions are measured: pain severity (worst, least, average, and now) and the impact of pain on functioning (pain interference with general activity, walking, work, mood, enjoyment of life, relations with others, and sleep). The severity of pain in the last 24 hours is rated on a scale of 0 (no pain) to 10 (pain as bad as you can imagine). The GEE Estimates are from the GEE model which includes the change from baseline for each BPI endpoint as the dependent variable, region, visit, treatment, and visit by treatment as fixed factors, and baseline of each BPI endpoint as a covariate, with compound symmetry covariance structure.
Secondary	Change From Baseline Over Time in BPI Pain Interference Score	Baseline, Weeks 12, 24, 36, 48, 72, 96	Change from baseline to post-baseline visits in BPI pain interference score as recorded on the day of the study visit. The BPI evaluates the condition of all pain over the previous 24 hours. Two dimensions are measured: pain severity (worst, least, average, and now) and the impact of pain on functioning (pain interference with general activity, walking, work, mood, enjoyment of life, relations with others, and sleep). Pain interference in the last 24 hours is rated on a scale of 0 (does not interfere) to 10 (completely interferes). The GEE Estimates are from the GEE model which includes the change from baseline for each BPI endpoint as the dependent variable, region, visit, treatment, and visit by treatment as fixed factors, and baseline of each BPI endpoint as a covariate, with compound symmetry covariance structure.
Secondary	Change From Baseline Over Time in WOMAC Stiffness Score	Baseline, Weeks 12, 24, 36, 48, 72, 96, 120, 144	The WOMAC is a 24-item participant-reported questionnaire with two domains, Stiffness (2 questions) and Physical Function (17 questions) over the previous 48 hours. The WOMAC is administered in a 5-point Likert-scale format using descriptors of none, mild, moderate, severe, and extreme corresponding to an ordinal scale of 0-4. Higher scores on the WOMAC indicate worse stiffness and functional limitations. Scores are normalized to a 0-100 metric where 0 was the best health state and 100 the worst. The GEE Estimates are from the GEE model which includes the change from baseline for WOMAC Stiffness as the dependent variable, region, visit, treatment, actual randomization stratification and visit by treatment as fixed factors, and baseline of WOMAC Stiffness as a covariate, with compound symmetry covariance structure.
Secondary	Change From Baseline Over Time in WOMAC Physical Function Score	Baseline, Weeks 12, 24, 36, 48, 72, 96, 120, 144	The WOMAC is a 24-item participant-reported questionnaire with two domains, Stiffness (2 questions) and Physical Function (17 questions) over the previous 48 hours. The WOMAC is administered in a 5-point Likert-scale format using descriptors of none, mild, moderate, severe, and extreme corresponding to an ordinal scale of 0-4. Higher scores on the WOMAC indicate worse stiffness and functional limitations. Scores are normalized to a 0-100 metric where 0 was the best health state and 100 the worst. The GEE Estimates are from the GEE model which includes the change from baseline for WOMAC Stiffness as the dependent variable, region, visit, treatment, actual randomization stratification and visit by treatment as fixed factors, and baseline of WOMAC Stiffness as a covariate, with compound symmetry covariance structure.
Secondary	Change From Baseline Over Time in BFI Worst Fatigue Score	Baseline, Weeks 12, 24, 36, 48, 72, 96	Change from baseline to post-baseline visits in Brief Fatigue Inventory Question 3 (Worst Fatigue in Past 24 Hours; BFI-Q3) as averaged from daily diary scores recorded over 1 week and the study visit score. The BFI is a self-reported questionnaire consisting of 9 items related to fatigue rated on a 0 to 10 numerical scale with a recall period of 24 hours. Two dimensions are measured: fatigue severity and the interference of fatigue on daily life (activity, mood, walking ability, work, relations with others, and enjoyment of life). Participants are asked to rate their worst fatigue over the past 24 hours from 0 (no fatigue) to 10 (as bad a you can imagine). The GEE Estimates are from the GEE model which includes the change from baseline for each BFI endpoint as the dependent variable, region, visit, treatment, actual randomization stratification and visit by treatment as fixed factors, and baseline of each BFI endpoint as a covariate, with compound symmetry covariance structure.

1 to 10 of 33Source ClinicalTrials.gov

See all 33 registered outcomes

Outcomes33

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Percentage of Participants Achieving Mean Serum Phosphorus Levels Above the LLN (2.5 mg/dL [0.81 mmol/L]) at the Mid-Point of the Dose Interval, as Averaged Across Dose Cycles Between Baseline and Week 24	Baseline through Week 24	Primary Analysis Set: all randomized participants who received at least 1 dose of study drug during the Placebo-Controlled Treatment Period.	percentage of participants	MEAN
SECONDARY	Change From Baseline to Week 24 in Brief Pain Inventory (BPI) Question 3 (Q3; Worst Pain in Past 24 Hours) Score	Baseline, 24 weeks	Primary Analysis Set: all randomized participants who received at least 1 dose of study drug during the Placebo-Controlled Treatment Period.	score on a scale	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline to Week 24 in the Western Ontario and McMaster University Osteoarthritis Index (WOMAC) Stiffness Score	Baseline, 24 weeks	Primary Analysis Set: all randomized participants who received at least 1 dose of study drug during the Placebo-Controlled Treatment Period.	score on a scale	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline to Week 24 in the WOMAC Physical Function Score	Baseline, 24 weeks	Primary Analysis Set: all randomized participants who received at least 1 dose of study drug during the Placebo-Controlled Treatment Period.	score on a scale	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline Over Time in BPI Worst Pain Score	Baseline, Weeks 12, 24, 36, 48, 72, 96	Primary Analysis Set: all randomized participants who received at least 1 dose of study drug during the Placebo-Controlled Treatment Period. Participants with an assessment at given time point.	score on a scale	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline Over Time in BPI Pain Severity Score	Baseline, Weeks 12, 24, 36, 48, 72, 96	Primary Analysis Set: all randomized participants who received at least 1 dose of study drug during the Placebo-Controlled Treatment Period. Participants with an assessment at given time point.	score on a scale	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline Over Time in BPI Pain Interference Score	Baseline, Weeks 12, 24, 36, 48, 72, 96	Primary Analysis Set: all randomized participants who received at least 1 dose of study drug during the Placebo-Controlled Treatment Period. Participants with an assessment at given time point.	score on a scale	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline Over Time in WOMAC Stiffness Score	Baseline, Weeks 12, 24, 36, 48, 72, 96, 120, 144	Primary Analysis Set: all randomized participants who received at least 1 dose of study drug during the Placebo-Controlled Treatment Period. Participants with an assessment at given time point.	score on a scale	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline Over Time in WOMAC Physical Function Score	Baseline, Weeks 12, 24, 36, 48, 72, 96, 120, 144	Primary Analysis Set: all randomized participants who received at least 1 dose of study drug during the Placebo-Controlled Treatment Period. Participants with an assessment at given time point.	score on a scale	LEAST_SQUARES_MEAN
SECONDARY	Change From Baseline Over Time in BFI Worst Fatigue Score	Baseline, Weeks 12, 24, 36, 48, 72, 96	Primary Analysis Set: all randomized participants who received at least 1 dose of study drug during the Placebo-Controlled Treatment Period. Participants with an assessment at given time point.	score on a scale	LEAST_SQUARES_MEAN

1 to 10 of 33Source ClinicalTrials.gov

See all 33 outcomes

Results by group468

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Percentage of Participants Achieving Mean Serum Phosphorus Levels Above the LLN (2.5 mg/dL [0.81 mmol/L]) at the Mid-Point of the Dose Interval, as Averaged Across Dose Cycles Between Baseline and Week 24	Placebo		7.6	95% Confidence Interval 3.3 to 16.5	percentage of participants
Percentage of Participants Achieving Mean Serum Phosphorus Levels Above the LLN (2.5 mg/dL [0.81 mmol/L]) at the Mid-Point of the Dose Interval, as Averaged Across Dose Cycles Between Baseline and Week 24	Burosumab		92.6	95% Confidence Interval 83.9 to 96.8	percentage of participants
Change From Baseline to Week 24 in Brief Pain Inventory (BPI) Question 3 (Q3; Worst Pain in Past 24 Hours) Score	Placebo		-0.32	Standard Error 0.222	score on a scale
Change From Baseline to Week 24 in Brief Pain Inventory (BPI) Question 3 (Q3; Worst Pain in Past 24 Hours) Score	Burosumab		-0.79	Standard Error 0.211	score on a scale
Change From Baseline to Week 24 in the Western Ontario and McMaster University Osteoarthritis Index (WOMAC) Stiffness Score	Placebo		0.46	Standard Error 3.139	score on a scale
Change From Baseline to Week 24 in the Western Ontario and McMaster University Osteoarthritis Index (WOMAC) Stiffness Score	Burosumab		-7.85	Standard Error 3.034	score on a scale
Change From Baseline to Week 24 in the WOMAC Physical Function Score	Placebo		1.79	Standard Error 2.722	score on a scale
Change From Baseline to Week 24 in the WOMAC Physical Function Score	Burosumab		-3.11	Standard Error 2.553	score on a scale
Change From Baseline Over Time in BPI Worst Pain Score	Placebo	Change at Week 12	-0.37	Standard Error 0.216	score on a scale
Change From Baseline Over Time in BPI Worst Pain Score	Burosumab 1 mg/kg	Change at Week 12	-0.62	Standard Error 0.208	score on a scale

1 to 10 of 468Source ClinicalTrials.gov

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Participant flow24

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Double-Blind (Placebo-Controlled) Period	STARTED	Placebo	66
Double-Blind (Placebo-Controlled) Period	STARTED	Burosumab 1 mg/kg	68
Double-Blind (Placebo-Controlled) Period	COMPLETED	Placebo	66
Double-Blind (Placebo-Controlled) Period	COMPLETED	Burosumab 1 mg/kg	67
Double-Blind (Placebo-Controlled) Period	NOT COMPLETED	Placebo	0
Double-Blind (Placebo-Controlled) Period	NOT COMPLETED	Burosumab 1 mg/kg	1
Open-label Treatment Continuation Period	STARTED	Placebo	66
Open-label Treatment Continuation Period	STARTED	Burosumab 1 mg/kg	67
Open-label Treatment Continuation Period	COMPLETED	Placebo	63
Open-label Treatment Continuation Period	COMPLETED	Burosumab 1 mg/kg	63

1 to 10 of 24Source ClinicalTrials.gov

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Adverse events428

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
other	Vertigo	Ear and labyrinth disorders	Placebo (DB Period)	1	66
other	Vertigo	Ear and labyrinth disorders	Placebo -> Burosumab (OL Period)	4	66
other	Vertigo	Ear and labyrinth disorders	Burosumab -> Burosumab (Combined DB and OL Period)	3	68
other	Vertigo	Ear and labyrinth disorders	Total Burosumab (Combined DB and OL Period)	7	134
other	Hyperparathyroidism Secondary	Endocrine disorders	Placebo (DB Period)	0	66
other	Hyperparathyroidism Secondary	Endocrine disorders	Placebo -> Burosumab (OL Period)	3	66
other	Hyperparathyroidism Secondary	Endocrine disorders	Burosumab -> Burosumab (Combined DB and OL Period)	4	68
other	Hyperparathyroidism Secondary	Endocrine disorders	Total Burosumab (Combined DB and OL Period)	7	134
other	Abdominal Discomfort	Gastrointestinal disorders	Placebo (DB Period)	0	66
other	Abdominal Discomfort	Gastrointestinal disorders	Placebo -> Burosumab (OL Period)	3	66

1 to 10 of 428Source ClinicalTrials.gov

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Adverse event totals12

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Placebo (DB Period)	0	66
Total, all-cause mortality	Placebo -> Burosumab (OL Period)	0	66
Total, all-cause mortality	Burosumab -> Burosumab (Combined DB and OL Period)	1	68
Total, all-cause mortality	Total Burosumab (Combined DB and OL Period)	1	134
Total, other adverse events	Placebo (DB Period)	57	66
Total, other adverse events	Placebo -> Burosumab (OL Period)	63	66
Total, other adverse events	Burosumab -> Burosumab (Combined DB and OL Period)	68	68
Total, other adverse events	Total Burosumab (Combined DB and OL Period)	131	134
Total, serious adverse events	Placebo (DB Period)	1	66
Total, serious adverse events	Placebo -> Burosumab (OL Period)	10	66

1 to 10 of 12Source ClinicalTrials.gov

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Papers6

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
29947083	RESULT	PubMed
31165191	RESULT	PubMed
34548383	DERIVED	PubMed
35927518	DERIVED	PubMed
36854566	DERIVED	PubMed
39151033	DERIVED	PubMed

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.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

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.

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