Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Trial Review
The ANZCTR website will be unavailable from 1pm until 3pm (AEDT) on Wednesday the 30th of October for website maintenance. Please be sure to log out of the system in order to avoid any loss of data.
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT02915705
Registration number
NCT02915705
Ethics application status
Date submitted
23/05/2016
Date registered
27/09/2016
Date last updated
29/08/2024
Titles & IDs
Public title
Efficacy and Safety of Burosumab Versus Oral Phosphate and Active Vitamin D Treatment in Pediatric Patients With XLH
Query!
Scientific title
A Randomized, Open-Label, Phase 3 Study to Assess the Efficacy and Safety of KRN23 Versus Oral Phosphate and Active Vitamin D Treatment in Pediatric Patients With X Linked Hypophosphatemia (XLH)
Query!
Secondary ID [1]
0
0
UX023-CL301
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
X-Linked Hypophosphatemia
0
0
Query!
Condition category
Condition code
Musculoskeletal
0
0
0
0
Query!
Other muscular and skeletal disorders
Query!
Metabolic and Endocrine
0
0
0
0
Query!
Metabolic disorders
Query!
Human Genetics and Inherited Disorders
0
0
0
0
Query!
Other human genetics and inherited disorders
Query!
Metabolic and Endocrine
0
0
0
0
Query!
Other metabolic disorders
Query!
Intervention/exposure
Study type
Interventional(has expanded access)
Query!
Description of intervention(s) / exposure
Treatment: Other - burosumab
Treatment: Drugs - Oral Phosphate Supplement
Treatment: Drugs - active vitamin D
Experimental: Burosumab - Burosumab 0.8 mg/kg starting dose, administered Q2W by SC injection during the Treatment Period (up to Week 64). During the Treatment Extension Period (Week 64 to Week 140), participants continued to receive a starting dose of SC burosumab 0.8 mg/kg Q2W. Participants in Japan and Korea did not enter the Treatment Extension Period.
Active comparator: Active Control - Multiple daily doses of oral phosphate and one or more daily doses of active vitamin D therapy, titrated and individualized by the investigator based on published recommendations during the Treatment Period (up to Week 64). During the Treatment Extension Period (Week 64 to Week 140), participants crossed over to receive a starting dose of SC burosumab 0.8 mg/kg Q2W. Participants in Japan and Korea did not enter the Treatment Extension Period.
Treatment: Other: burosumab
solution for subcutaneous (SC) injection
Treatment: Drugs: Oral Phosphate Supplement
oral tablet; oral solution; oral powder
Treatment: Drugs: active vitamin D
tablet, oral solution
Query!
Intervention code [1]
0
0
Treatment: Other
Query!
Intervention code [2]
0
0
Treatment: Drugs
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Radiographic Global Impression of Change (RGI-C) Global Score at Week 40
Query!
Assessment method [1]
0
0
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).
Query!
Timepoint [1]
0
0
Week 40
Query!
Secondary outcome [1]
0
0
Percentage of Participants With a Mean RGI-C Global Score = +2.0 (Responders) at Week 40
Query!
Assessment method [1]
0
0
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).
Query!
Timepoint [1]
0
0
Week 40
Query!
Secondary outcome [2]
0
0
Percentage of Participants With a Mean RGI-C Global Score = +2.0 (Responders) at Week 64
Query!
Assessment method [2]
0
0
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).
Query!
Timepoint [2]
0
0
Week 64
Query!
Secondary outcome [3]
0
0
RGI-C Global Score at Week 64
Query!
Assessment method [3]
0
0
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).
Query!
Timepoint [3]
0
0
Week 64
Query!
Secondary outcome [4]
0
0
Change From Baseline in RSS Total Score at Week 40
Query!
Assessment method [4]
0
0
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.
Query!
Timepoint [4]
0
0
Baseline, Week 40
Query!
Secondary outcome [5]
0
0
Change From Baseline in RSS Total Score at Week 64
Query!
Assessment method [5]
0
0
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.
Query!
Timepoint [5]
0
0
Baseline, Week 64
Query!
Secondary outcome [6]
0
0
RGI-C Long Leg Score at Week 40
Query!
Assessment method [6]
0
0
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).
Query!
Timepoint [6]
0
0
Week 40
Query!
Secondary outcome [7]
0
0
RGI-C Long Leg Score at Week 64
Query!
Assessment method [7]
0
0
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).
Query!
Timepoint [7]
0
0
Week 64
Query!
Secondary outcome [8]
0
0
Change From Baseline in Height-For-Age Z-Scores to Week 40
Query!
Assessment method [8]
0
0
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.
Query!
Timepoint [8]
0
0
Baseline, Week 40
Query!
Secondary outcome [9]
0
0
Change From Baseline in Height-For-Age Z-Scores to Week 64
Query!
Assessment method [9]
0
0
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.
Query!
Timepoint [9]
0
0
Baseline, Week 64
Query!
Secondary outcome [10]
0
0
Change in Growth Velocity Z Score From Baseline to Week 40
Query!
Assessment method [10]
0
0
A growth velocity Z score was calculated based on Tanner's standard. The Z score indicates the number of standard deviations away from a reference population (from Tanner's standard) in the same age range and with the same sex. The baseline growth velocity was calculated for participants who had data available from within 1.5 years prior to baseline. The Week 64 growth velocity was calculated using data between baseline and Week 64. The mid-point of the age interval was used to locate the closest reference age provided by Tanner's Standard. Children with a mid-point age under 2.25 years were excluded, because younger ages are not available in Tanner's standard. To smoothly transition from recumbent length to standing height, 0·8 cm was subtracted from recumbent length before pooling with standing height. 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.
Query!
Timepoint [10]
0
0
Baseline, Week 40
Query!
Secondary outcome [11]
0
0
Change in Growth Velocity Z Score From Baseline to Week 64
Query!
Assessment method [11]
0
0
A growth velocity Z score was calculated based on Tanner's standard. The Z score indicates the number of standard deviations away from a reference population (from Tanner's standard) in the same age range and with the same sex. The baseline growth velocity was calculated for participants who had data available from within 1.5 years prior to baseline. The Week 64 growth velocity was calculated using data between baseline and Week 64. The mid-point of the age interval was used to locate the closest reference age provided by Tanner's Standard. Children with a mid-point age under 2.25 years were excluded, because younger ages are not available in Tanner's standard. To smoothly transition from recumbent length to standing height, 0·8 cm was subtracted from recumbent length before pooling with standing height. 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.
Query!
Timepoint [11]
0
0
Baseline, Week 64
Query!
Secondary outcome [12]
0
0
Change From Baseline Over Time in Serum Phosphorus Concentration, up to Week 64
Query!
Assessment method [12]
0
0
The GEE model includes change from baseline for serum phosphorous measurement as the dependent variable, treatment group, visit, interaction between treatment group by visit, baseline age and baseline RSS stratification as factors, baseline phosphorous measure as a covariate, with exchangeable covariance structure. The GEE model included data up to Week 64.
Query!
Timepoint [12]
0
0
Baseline, Weeks 1, 2, 4, 8, 12, 16, 24, 32, 33, 40, 52, 64
Query!
Secondary outcome [13]
0
0
Change From Baseline Over Time in Serum Phosphorus Concentration, Weeks 66-112
Query!
Assessment method [13]
0
0
Query!
Timepoint [13]
0
0
Baseline, Weeks 66, 68, 76, 88, 100, 112
Query!
Secondary outcome [14]
0
0
Change From Baseline in Mean Post-Baseline Serum Phosphorus Level to Week 64
Query!
Assessment method [14]
0
0
The ANCOVA model includes change in serum phosphorus from baseline to mean post-baseline as the dependent variable, treatment group, baseline age and baseline RSS stratification as factors, baseline phosphorous measure as a covariate.
Query!
Timepoint [14]
0
0
Baseline, Weeks 1, 4, 8, 16, 24, 32, 40, 52, 64
Query!
Secondary outcome [15]
0
0
Change From Baseline in Mean Post-Baseline Serum Phosphorus Level to Week 140 (During Treatment With Burosumab)
Query!
Assessment method [15]
0
0
Query!
Timepoint [15]
0
0
Burosumab arm: Baseline, Week 1, 4, 8, 16, 24, 32, 40, 52, 64, 66, 68, 76, 88, 100, 112, 124, 140; Active Control arm: Baseline, Week 68, 76, 88, 100, 112, 124, 140
Query!
Secondary outcome [16]
0
0
Percentage of Participants Reaching the Normal Range of Serum Phosphorus Concentration (3.2 - 6.1 mg/dL)
Query!
Assessment method [16]
0
0
Query!
Timepoint [16]
0
0
Burosumab arm: Baseline, up to Week 140; Active Control arm: Baseline, Week 68 up to Week 140
Query!
Secondary outcome [17]
0
0
Change From Baseline Over Time in 1,25-Dihydroxyvitamin D, up to Week 64
Query!
Assessment method [17]
0
0
The GEE model includes change from baseline for 1, 25-Dihydroxyvitamin D measurement as the dependent variable, treatment group, visit, interaction between treatment group by visit, baseline age and baseline RSS stratification as factors, baseline 1, 25-Dihydroxyvitamin D measure as a covariate, with exchangeable covariance structure. The GEE model included data up to Week 64.
Query!
Timepoint [17]
0
0
Baseline, Weeks 1, 2, 4, 8, 12, 16, 24, 32, 33, 40, 52, 64
Query!
Secondary outcome [18]
0
0
Change From Baseline Over Time in 1,25-Dihydroxyvitamin D, Weeks 68 to 112
Query!
Assessment method [18]
0
0
Query!
Timepoint [18]
0
0
Baseline, Weeks 68, 76, 88, 100, 112
Query!
Secondary outcome [19]
0
0
Change From Baseline Over Time in TmP/GFR, up to Week 64
Query!
Assessment method [19]
0
0
Serum phosphorus and TRP measurements were used in the calculation of TmP/GFR.
The GEE model includes change from baseline for TmP/GFR measurement as the dependent variable, treatment group, visit, interaction between treatment group by visit, baseline age and baseline RSS stratification as factors, baseline TmP/GFR measure as a covariate, with exchangeable covariance structure. The GEE model included data up to Week 64.
Query!
Timepoint [19]
0
0
Baseline, Weeks 4, 8, 16, 24, 32, 40, 52, 64
Query!
Secondary outcome [20]
0
0
Change From Baseline Over Time in TmP/GFR, Week 68 to 112
Query!
Assessment method [20]
0
0
Serum phosphorus and TRP measurements were used in the calculation of TmP/GFR.
Query!
Timepoint [20]
0
0
Baseline, Weeks 68, 76, 88, 112
Query!
Secondary outcome [21]
0
0
Change From Baseline Over Time in Serum ALP, up to Week 64
Query!
Assessment method [21]
0
0
The GEE model includes change from baseline for ALP measurement as the dependent variable, treatment group, visit, interaction between treatment group by visit, baseline age and baseline RSS stratification as factors, baseline ALP measure as a covariate, with exchangeable covariance structure. The GEE model included data up to Week 64.
Query!
Timepoint [21]
0
0
Baseline, Weeks 16, 24, 40, 52, 64
Query!
Secondary outcome [22]
0
0
Change From Baseline Over Time in Serum ALP, Week 68 to 112
Query!
Assessment method [22]
0
0
Query!
Timepoint [22]
0
0
Baseline, Weeks 68, 76, 88, 100, 112
Query!
Secondary outcome [23]
0
0
Percent Change From Baseline Over Time in Serum ALP, up to Week 112
Query!
Assessment method [23]
0
0
Decreases indicate improvement.
Query!
Timepoint [23]
0
0
Baseline, Weeks 16, 24, 40, 52, 64, 68, 76, 88, 100, 112
Query!
Secondary outcome [24]
0
0
Change From Baseline in the PROMIS Pediatric Pain Interference, Physical Function Mobility and Fatigue Domain Scores (For Participants = 5 Years of Age at the Screening Visit) at Week 40
Query!
Assessment method [24]
0
0
The PROMIS was developed by the National Institutes of Health and uses domain-specific measures to assess patient well-being (Broderick et al. 2013), (NIH 2015). It uses a T-score metric in which 50 is the mean of a relevant reference population and 10 is the standard deviation (SD) of that population. For the Pain Interference Domain, decreases indicate less pain, for the Physical Function Mobility Domain, increases indicate greater mobility and for the Fatigue Domain, decreases indicate less fatigue.
Query!
Timepoint [24]
0
0
Baseline, Week 40
Query!
Secondary outcome [25]
0
0
Change From Baseline in the PROMIS Pediatric Pain Interference, Physical Function Mobility and Fatigue Domain Scores (For Participants = 5 Years of Age at the Screening Visit) at Week 64
Query!
Assessment method [25]
0
0
The PROMIS was developed by the National Institutes of Health and uses domain-specific measures to assess patient well-being (Broderick et al. 2013), (NIH 2015). It uses a T-score metric in which 50 is the mean of a relevant reference population and 10 is the standard deviation (SD) of that population. For the Pain Interference Domain, decreases indicate less pain, for the Physical Function Mobility Domain, increases indicate greater mobility and for the Fatigue Domain, decreases indicate less fatigue.
Query!
Timepoint [25]
0
0
Baseline, Week 64
Query!
Secondary outcome [26]
0
0
Change From Baseline in the FPS-R (For Participants = 5 Years of Age at the Screening Visit) at Week 40
Query!
Assessment method [26]
0
0
The FPS-R is a dimensionless 10 point Likert scale used to assess self-reported pain intensity on a scale from 0 (no pain) to 10 (most pain you can imagine). Greater pain scores are indicative of more severe pain.
Query!
Timepoint [26]
0
0
Baseline, Week 40
Query!
Secondary outcome [27]
0
0
Change From Baseline in the FPS-R (For Participants = 5 Years of Age at the Screening Visit) at Week 64
Query!
Assessment method [27]
0
0
The FPS-R is a dimensionless 10 point Likert scale used to assess self-reported pain intensity on a scale from 0 (no pain) to 10 (most pain you can imagine). Greater pain scores are indicative of more severe pain.
Query!
Timepoint [27]
0
0
Baseline, Week 64
Query!
Secondary outcome [28]
0
0
Change From Baseline in the 6MWT Total Distance at Week 40
Query!
Assessment method [28]
0
0
The total distance walked (meters) in a 6-minute period was measured in participants = 5 years of age at the Screening Visit who were able to complete the test.
Query!
Timepoint [28]
0
0
Baseline, Week 40
Query!
Secondary outcome [29]
0
0
Change From Baseline in the 6MWT Total Distance at Week 64
Query!
Assessment method [29]
0
0
The total distance walked (meters) in a 6-minute period was measured in participants = 5 years of age at the Screening Visit who were able to complete the test.
Query!
Timepoint [29]
0
0
Baseline, Week 64
Query!
Secondary outcome [30]
0
0
Percent of Predicted Normal in the 6MWT Total Distance at Week 40
Query!
Assessment method [30]
0
0
The total distance walked (meters) in a 6-minute period was measured in participants = 5 years of age at the Screening Visit who were able to complete the test, and the percent predicted distance based on normative data for age and gender was estimated.
Query!
Timepoint [30]
0
0
Baseline, Week 40
Query!
Secondary outcome [31]
0
0
Percent of Predicted Normal in the 6MWT Total Distance at Week 64
Query!
Assessment method [31]
0
0
The total distance walked (meters) in a 6-minute period was measured in participants = 5 years of age at the Screening Visit who were able to complete the test, and the percent predicted distance based on normative data for age and gender was estimated.
Query!
Timepoint [31]
0
0
Baseline, Week 64
Query!
Eligibility
Key inclusion criteria
1. Male or female, aged 1 to =12 years with radiographic evidence of rickets as determined by central readers
2. Phosphate-regulating endopeptidase homolog, X-linked (PHEX) mutation or variant of uncertain significance in either the patient or in a directly related family member with appropriate X-linked inheritance
3. Biochemical findings associated with XLH: serum phosphorus <3.0 mg/dL (<0.97 mmol/L)
4. Serum creatinine below the age-adjusted upper limit of normal
5. Serum 25(OH)D above the lower limit of normal (=16 ng/mL) at the Screening Visit
6. Have received both oral phosphate and active vitamin D therapy for = 12 consecutive months (for children =3 years of age) or = 6 consecutive months (for children <3 years of age) 7 days prior to the Randomization Visit
7. Willing to provide access to prior medical records for the collection of historical growth and radiographic data and disease history
8. Provide written or verbal assent (as appropriate for the subject and region) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures.
9. Must, in the opinion of the investigator, be willing and able to complete all aspects of the study, adhere to the study visit schedule and comply with the assessments
10. Females who have reached menarche must have a negative pregnancy test at Screening and undergo additional pregnancy testing during the study. Female subjects of childbearing potential must be willing to use a highly effective method of contraception for the duration of the study plus 12 weeks after stopping the study drug. Sexually active male subjects with female partners of childbearing potential must consent to use a condom with spermicide or a highly effective method of contraception for the duration of the study plus 12 weeks after stopping the study drug
Query!
Minimum age
1
Year
Query!
Query!
Maximum age
12
Years
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
Key exclusion criteria
1. Tanner stage 4 or higher in any of the following: genitals, breast, or pubic hair, based on physical examination
2. Height percentile > 50th based on country-specific norms
3. Use of aluminum hydroxide antacids (eg, Maalox® and Mylanta®), systemic corticosteroids, acetazolamide, and thiazides within 7 days prior to the Screening Visit
4. Current or prior use of leuprorelin (eg, Lupron®, Viadur®, Eligard®), triptorelin (TRELSTAR®), goserelin (Zoladex®), or other drugs known to delay puberty
5. Use of growth hormone therapy within 12 months before the Screening Visit
6. Presence of nephrocalcinosis on renal ultrasound grade 4
7. Planned orthopedic surgery, including osteotomy or implantation or removal of staples, 8 plates, or any other hardware, within the first 40 weeks of the study
8. Hypocalcemia or hypercalcemia, defined as serum calcium levels outside the age-adjusted normal limits
9. Evidence of hyperparathyroidism (parathyroid hormone [PTH] levels 2.5X upper limit of normal [ULN])
10. Use of medication to suppress PTH (eg, cinacalcet, calcimimetics) within 2 months prior to the Screening Visit
11. Presence or history of any condition that, in the view of the investigator, places the subject at high risk of poor treatment compliance or of not completing the study.
12. Presence of a concurrent disease or condition that would interfere with study participation or affect safety
13. History of recurrent infection or predisposition to infection, or of known immunodeficiency
14. Use of a therapeutic monoclonal antibody within 90 days prior to the Screening Visit or history of allergic or anaphylactic reactions to any monoclonal antibody
15. Presence or history of any hypersensitivity to KRN23 excipients that, in the judgment of the investigator, places the subject at increased risk for adverse effects
16. Use of any investigational product or investigational medical device within 30 days prior to screening, or requirement for any investigational agent prior to completion of all scheduled study assessments. OR, in Japan, use of any investigational product or investigational medical device within 4 months prior to screening, or requirement for any investigational agent prior to completion of all scheduled study assessments
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
Randomised controlled trial
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Open (masking not used)
Query!
Who is / are masked / blinded?
Query!
Query!
Query!
Query!
Intervention assignment
Parallel
Query!
Other design features
Query!
Phase
Phase 3
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Completed
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
8/09/2016
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
Query!
Actual
15/07/2019
Query!
Sample size
Target
Query!
Accrual to date
Query!
Final
61
Query!
Recruitment in Australia
Recruitment state(s)
NSW
Query!
Recruitment hospital [1]
0
0
The Children's Hospital at Westmead - Westmead
Query!
Recruitment postcode(s) [1]
0
0
2145 - Westmead
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
California
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
Indiana
Query!
Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
Missouri
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
Tennessee
Query!
Country [5]
0
0
Canada
Query!
State/province [5]
0
0
Ontario
Query!
Country [6]
0
0
Canada
Query!
State/province [6]
0
0
Quebec
Query!
Country [7]
0
0
Japan
Query!
State/province [7]
0
0
Kanagawa
Query!
Country [8]
0
0
Japan
Query!
State/province [8]
0
0
Okayama
Query!
Country [9]
0
0
Japan
Query!
State/province [9]
0
0
Osaka
Query!
Country [10]
0
0
Korea, Republic of
Query!
State/province [10]
0
0
Seoul
Query!
Country [11]
0
0
Sweden
Query!
State/province [11]
0
0
Stockholm
Query!
Country [12]
0
0
United Kingdom
Query!
State/province [12]
0
0
Birmingham
Query!
Country [13]
0
0
United Kingdom
Query!
State/province [13]
0
0
Manchester
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Query!
Name
Kyowa Kirin, Inc.
Query!
Address
Query!
Country
Query!
Other collaborator category [1]
0
0
Commercial sector/industry
Query!
Name [1]
0
0
Kyowa Kirin Co., Ltd.
Query!
Address [1]
0
0
Query!
Country [1]
0
0
Query!
Ethics approval
Ethics application status
Query!
Summary
Brief summary
The primary objective of this study is to evaluate the effect of KRN23 (burosumab) therapy in improving rickets in children with XLH compared with active control (oral phosphate/active vitamin D).
Query!
Trial website
https://clinicaltrials.gov/study/NCT02915705
Query!
Trial related presentations / publications
Ward LM, Glorieux FH, Whyte MP, Munns CF, Portale AA, Hogler W, Simmons JH, Gottesman GS, Padidela R, Namba N, Cheong HI, Nilsson O, Mao M, Chen A, Skrinar A, Roberts MS, Imel EA. Effect of Burosumab Compared With Conventional Therapy on Younger vs Older Children With X-linked Hypophosphatemia. J Clin Endocrinol Metab. 2022 Jul 14;107(8):e3241-e3253. doi: 10.1210/clinem/dgac296. Padidela R, Whyte MP, Glorieux FH, Munns CF, Ward LM, Nilsson O, Portale AA, Simmons JH, Namba N, Cheong HI, Pitukcheewanont P, Sochett E, Hogler W, Muroya K, Tanaka H, Gottesman GS, Biggin A, Perwad F, Williams A, Nixon A, Sun W, Chen A, Skrinar A, Imel EA. Patient-Reported Outcomes from a Randomized, Active-Controlled, Open-Label, Phase 3 Trial of Burosumab Versus Conventional Therapy in Children with X-Linked Hypophosphatemia. Calcif Tissue Int. 2021 May;108(5):622-633. doi: 10.1007/s00223-020-00797-x. Epub 2021 Jan 23. Mao M, Carpenter TO, Whyte MP, Skrinar A, Chen CY, San Martin J, Rogol AD. Growth Curves for Children with X-linked Hypophosphatemia. J Clin Endocrinol Metab. 2020 Oct 1;105(10):3243-9. doi: 10.1210/clinem/dgaa495. Imel EA, Glorieux FH, Whyte MP, Munns CF, Ward LM, Nilsson O, Simmons JH, Padidela R, Namba N, Cheong HI, Pitukcheewanont P, Sochett E, Hogler W, Muroya K, Tanaka H, Gottesman GS, Biggin A, Perwad F, Mao M, Chen CY, Skrinar A, San Martin J, Portale AA. Burosumab versus conventional therapy in children with X-linked hypophosphataemia: a randomised, active-controlled, open-label, phase 3 trial. Lancet. 2019 Jun 15;393(10189):2416-2427. doi: 10.1016/S0140-6736(19)30654-3. Epub 2019 May 16. Erratum In: Lancet. 2019 Jul 13;394(10193):120. doi: 10.1016/S0140-6736(19)31426-6.
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Medical Director
Query!
Address
0
0
Ultragenyx Pharmaceutical Inc
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Type
Other Details
Attachment
Study protocol
https://cdn.clinicaltrials.gov/large-docs/05/NCT02915705/Prot_000.pdf
Statistical analysis plan
https://cdn.clinicaltrials.gov/large-docs/05/NCT02915705/SAP_001.pdf
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT02915705
Download to PDF