Trial · NCT02304367
Study of Burosumab (KRN23) in Adults With Tumor-Induced Osteomalacia (TIO) or Epidermal Nevus Syndrome (ENS)
NCT02304367, Phase 2, Completed, Kyowa Kirin, Inc.; results first posted 2020-07-30
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of Burosumab (KRN23) in Adults With Tumor-Induced Osteomalacia (TIO) or Epidermal Nevus Syndrome (ENS). Trials, record NCT02304367. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02304367
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Burosumab (KRN23) in Adults With Tumor-Induced Osteomalacia (TIO) or Epidermal Nevus Syndrome (ENS)
- NCT number
- NCT02304367
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 17 participants, actual
- Lead sponsor
- Kyowa Kirin, Inc.
- Conditions
- Tumor Induced Osteomalacia (TIO), Epidermal Nevus Syndrome (ENS)
- Interventions
- Burosumab
- First posted
- 2014-12-01
- Last updated
- 2024-05-06
- Results first posted
- 2020-07-30
Source ClinicalTrials.gov
Registered outcomes51
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 2.5 mg/dL at the Mid-Point of the Dose Intervals Between Baseline and Week 24 | Mid-point of each dose interval from Baseline to Week 24 (Weeks 2, 6, 10, 14 and 22 [there was no study visit at Week 18]) | The percentage of participants achieving mean serum phosphorus levels above the lower limit of normal (LLN; 2.5 mg/dL \[0.81 mmol/L\]) at the mid-point of the dose interval (2 weeks after dosing), as averaged across dose cycles between Baseline and Week 24 (i.e. the average of serum phosphorus levels at Weeks 2, 6, 10, 14 and 22). |
| Primary | Change From Baseline to Week 48 in Osteoid Thickness | Baseline, Week 48 | Histomorphometry of trans-iliac crest bone biopsies was assessed by a blinded, central reader. Osteoid thickness is the mean thickness of osteoid seams. |
| Primary | Change From Baseline to Week 48 in Osteoid Surface/Bone Surface (OS/BS) | Baseline, Week 48 | Histomorphometry of trans-iliac crest bone biopsies was assessed by a blinded, central reader. Osteoid surface/bone surface is expressed as the percentage of bone surface covered in osteoid. |
| Primary | Change From Baseline to Week 48 in Osteoid Volume/Bone Volume (OV/BV) | Baseline, Week 48 | Histomorphometry of trans-iliac crest bone biopsies was assessed by a blinded, central reader. Osteoid volume/bone volume is expressed as the percentage of a given volume of bone tissue that consists of unmineralized bone (osteoid). |
| Primary | Change From Baseline to Week 48 in Mineralization Lag Time (MLt) | Baseline, Week 48 | Mineralization lag time is a dynamic modeling parameter representing the mean time interval between the formation of osteoid and its subsequent mineralization which can be measured using histomorphometry with double tetracycline labeling. Mlt was calculated by dividing the osteoid width by the mineralizing apposition rate (MAR; the average rate at which new bone mineral is being added on any actively forming surface). If Mlt could not be calculated directly due to low tetracycline uptake, Mlt was imputed according to the following: Mlt = O.Th/(MAR\*MS/OS), where O.Th = osteoid thickness, MAR is imputed as 0.3 μm/day, MS/OS=mineralizing surface/osteoid surface, each measured at the same visit. |
| Secondary | Percentage of Participants Achieving Mean Serum Phosphorus Levels Above 2.5 mg/dL at the End of the Dose Intervals Between Baseline and Week 24 | End of each dose interval from Baseline to Week 24 (Weeks 4, 8, 12, 16, 20, and 24) | The percentage of participants achieving mean serum phosphorus levels above the lower limit of normal (2.5 mg/dL \[0.81 mmol/L\]) at the end of the dose interval (4 weeks post-dose, prior to the next dose), as averaged across dose cycles between Baseline and Week 24 (i.e. the average of serum phosphorus levels at Weeks 4, 8, 12, 16, 20, and 24). |
| Secondary | Mean Change From Baseline in Serum Phosphorus Levels at the Mid-Point of the Dose Interval, as Averaged Across Dose Cycles Between Baseline and Week 24 | Baseline and the mid-point of each dose interval from Baseline to Week 24 (Weeks 2, 6, 10, 14 and 22) | Mean change from Baseline to the mid-point of the dose interval (2 weeks after dosing) averaged across dose cycles between Baseline and Week 24 (i.e. the average of serum phosphorus levels at Weeks 2, 6, 10, 14 and 22). |
| Secondary | Percent Change From Baseline in Serum Phosphorus Levels at the Mid-Point of the Dose Interval, as Averaged Across Dose Cycles Between Baseline and Week 24 | Baseline and the mid-point of each dose interval from Baseline to Week 24 (Weeks 2, 6, 10, 14 and 22) | Mean percent change from Baseline to the mid-point of the dose interval (2 weeks after dosing) averaged across dose cycles between Baseline and Week 24 (i.e. the average of serum phosphorus levels at Weeks 2, 6, 10, 14 and 22). |
| Secondary | Mean Change From Baseline in Serum Phosphorus Levels at the End of the Dosing Cycle, as Averaged Across Dose Cycles Between Baseline and Week 24 | Baseline and Weeks 4, 8, 12, 16, 20, and 24 | Mean change from Baseline at the end of the dose interval (4 weeks post-dose, prior to the next dose) averaged across dose cycles between Baseline and Week 24 (i.e. the average of serum phosphorus levels at Weeks 4, 8, 12, 16, 20, and 24). |
| Secondary | Percent Mean Change From Baseline in Serum Phosphorus Levels at the End of the Dosing Cycle, as Averaged Across Dose Cycles Between Baseline and Week 24 | Baseline and Weeks 4, 8, 12, 16, 20, and 24 | Mean percent change from Baseline at the end of the dose interval (4 weeks post-dose, prior to the next dose) averaged across dose cycles between Baseline and Week 24 (i.e. the average of serum phosphorus levels at Weeks 4, 8, 12, 16, 20, and 24). |
1 to 10 of 51Source ClinicalTrials.gov
See all 51 registered outcomesOutcomes51
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 2.5 mg/dL at the Mid-Point of the Dose Intervals Between Baseline and Week 24 | Mid-point of each dose interval from Baseline to Week 24 (Weeks 2, 6, 10, 14 and 22 [there was no study visit at Week 18]) | TIO analysis set: includes participants with TIO who received at least 1 dose of burosumab during the study. | percentage of participants | NUMBER |
| PRIMARY | Change From Baseline to Week 48 in Osteoid Thickness | Baseline, Week 48 | TIO bone biopsy analysis set | µm | MEAN |
| PRIMARY | Change From Baseline to Week 48 in Osteoid Surface/Bone Surface (OS/BS) | Baseline, Week 48 | TIO biopsy analysis set | percentage of bone surface | MEAN |
| PRIMARY | Change From Baseline to Week 48 in Osteoid Volume/Bone Volume (OV/BV) | Baseline, Week 48 | TIO bone biopsy analysis set | percentage of bone volume | MEAN |
| PRIMARY | Change From Baseline to Week 48 in Mineralization Lag Time (MLt) | Baseline, Week 48 | TIO biopsy analysis set for whom Mlt could be calculated or imputed | days | MEAN |
| SECONDARY | Percentage of Participants Achieving Mean Serum Phosphorus Levels Above 2.5 mg/dL at the End of the Dose Intervals Between Baseline and Week 24 | End of each dose interval from Baseline to Week 24 (Weeks 4, 8, 12, 16, 20, and 24) | TIO analysis set | percentage of participants | NUMBER |
| SECONDARY | Mean Change From Baseline in Serum Phosphorus Levels at the Mid-Point of the Dose Interval, as Averaged Across Dose Cycles Between Baseline and Week 24 | Baseline and the mid-point of each dose interval from Baseline to Week 24 (Weeks 2, 6, 10, 14 and 22) | TIO analysis set | mg/dL | MEAN |
| SECONDARY | Percent Change From Baseline in Serum Phosphorus Levels at the Mid-Point of the Dose Interval, as Averaged Across Dose Cycles Between Baseline and Week 24 | Baseline and the mid-point of each dose interval from Baseline to Week 24 (Weeks 2, 6, 10, 14 and 22) | TIO analysis set | percent change | MEAN |
| SECONDARY | Mean Change From Baseline in Serum Phosphorus Levels at the End of the Dosing Cycle, as Averaged Across Dose Cycles Between Baseline and Week 24 | Baseline and Weeks 4, 8, 12, 16, 20, and 24 | TIO analysis set | mg/dL | MEAN |
| SECONDARY | Percent Mean Change From Baseline in Serum Phosphorus Levels at the End of the Dosing Cycle, as Averaged Across Dose Cycles Between Baseline and Week 24 | Baseline and Weeks 4, 8, 12, 16, 20, and 24 | TIO analysis set | percent change | MEAN |
1 to 10 of 51Source ClinicalTrials.gov
See all 51 outcomesResults by group301
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 2.5 mg/dL at the Mid-Point of the Dose Intervals Between Baseline and Week 24 | Burosumab | 50.0 | 95% Confidence Interval 26.80 to 73.20 | percentage of participants | |
| Change From Baseline to Week 48 in Osteoid Thickness | Burosumab | -5.12 | 95% Confidence Interval -10.04 to -0.20 | µm | |
| Change From Baseline to Week 48 in Osteoid Surface/Bone Surface (OS/BS) | Burosumab | -0.18 | 95% Confidence Interval -13.86 to 13.50 | percentage of bone surface | |
| Change From Baseline to Week 48 in Osteoid Volume/Bone Volume (OV/BV) | Burosumab | -5.47 | 95% Confidence Interval -11.87 to 0.93 | percentage of bone volume | |
| Change From Baseline to Week 48 in Mineralization Lag Time (MLt) | Burosumab | -565.20 | 95% Confidence Interval -2037.42 to 907.02 | days | |
| Percentage of Participants Achieving Mean Serum Phosphorus Levels Above 2.5 mg/dL at the End of the Dose Intervals Between Baseline and Week 24 | Burosumab | 21.4 | 95% Confidence Interval 7.57 to 47.59 | percentage of participants | |
| Mean Change From Baseline in Serum Phosphorus Levels at the Mid-Point of the Dose Interval, as Averaged Across Dose Cycles Between Baseline and Week 24 | Burosumab | 1.039 | Standard Deviation 0.5622 | mg/dL | |
| Percent Change From Baseline in Serum Phosphorus Levels at the Mid-Point of the Dose Interval, as Averaged Across Dose Cycles Between Baseline and Week 24 | Burosumab | 69.77 | Standard Deviation 43.743 | percent change | |
| Mean Change From Baseline in Serum Phosphorus Levels at the End of the Dosing Cycle, as Averaged Across Dose Cycles Between Baseline and Week 24 | Burosumab | 0.550 | Standard Deviation 0.4260 | mg/dL | |
| Percent Mean Change From Baseline in Serum Phosphorus Levels at the End of the Dosing Cycle, as Averaged Across Dose Cycles Between Baseline and Week 24 | Burosumab | 38.56 | Standard Deviation 33.027 | percent change |
1 to 10 of 301Source ClinicalTrials.gov
See all 301 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 | 17 | |
| Overall Study | Completed 48 Weeks of Study Drug | Burosumab | 16 | |
| Overall Study | Completed 144 Weeks of Study Drug | Burosumab | 12 | |
| Overall Study | COMPLETED | Burosumab | 9 | |
| Overall Study | NOT COMPLETED | Burosumab | 8 |
Source ClinicalTrials.gov
Adverse events528
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 | Anaemia | Blood and lymphatic system disorders | KRN23 TIO | 2 | 14 | |
| other | Anaemia | Blood and lymphatic system disorders | KRN23 XLH and ENS | 1 | 3 | |
| other | Coagulopathy | Blood and lymphatic system disorders | KRN23 TIO | 1 | 14 | |
| other | Coagulopathy | Blood and lymphatic system disorders | KRN23 XLH and ENS | 0 | 3 | |
| other | Leukocytosis | Blood and lymphatic system disorders | KRN23 TIO | 1 | 14 | |
| other | Leukocytosis | Blood and lymphatic system disorders | KRN23 XLH and ENS | 0 | 3 | |
| other | Bradycardia | Cardiac disorders | KRN23 TIO | 1 | 14 | |
| other | Bradycardia | Cardiac disorders | KRN23 XLH and ENS | 0 | 3 | |
| other | Tachycardia | Cardiac disorders | KRN23 TIO | 1 | 14 | |
| other | Tachycardia | Cardiac disorders | KRN23 XLH and ENS | 0 | 3 |
1 to 10 of 528Source ClinicalTrials.gov
See all 528 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | KRN23 TIO | 2 | 14 |
| Total, all-cause mortality | KRN23 XLH and ENS | 0 | 3 |
| Total, other adverse events | KRN23 TIO | 14 | 14 |
| Total, other adverse events | KRN23 XLH and ENS | 3 | 3 |
| Total, serious adverse events | KRN23 TIO | 8 | 14 |
| Total, serious adverse events | KRN23 XLH and ENS | 0 | 3 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 34660853 | DERIVED |
Source 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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| KRN23 TIO vs KRN23 XLH and ENS: deaths from any cause | Trial comparisons |
| KRN23 TIO vs KRN23 XLH and ENS: 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
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The record and each of its lists are JSON, twenty rows a page. No key.
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