Study of KRN23 in Adults With X-linked Hypophosphatemia (... | NCT02526160

NCT02526160

ID	Type	Description	Link
2014-005529-11	EudraCT Number

Status Module

Sponsor/Collaborators Module

Name	Class
Kyowa Kirin Co., Ltd.	INDUSTRY

Oversight Module

Description Module

Conditions Module

Design Module

Interventional Study Design

Biospecimen

No data available

No data is available for this block.

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Burosumab 1 mg/kg	Experimental	Burosumab 1 mg/kg administered subcutaneously (SC) every 4 weeks, for the duration of the study.	Biological: burosumab
Placebo	Placebo Comparator	Placebo administered SC every 4 weeks through Week 24, followed by burosumab 1 mg/kg, for the duration of the study.	Biological: burosumab Other: Placebo

Name	Type	Description	Arm Group Labels	Other Names
burosumab	Biological	solution for SC injection	Burosumab 1 mg/kg Placebo

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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 Outcomes

Measure	Description	Time Frame
Change From Baseline to Week 24 in Brief Pain Inventory (BPI) Question 3 (Q3; Worst Pain in Past 24 Hours) 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). 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.

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Male or female, aged 18 - 65 years, inclusive
Diagnosis of XLH supported by classic clinical features of adult XLH (such as short stature or bowed legs) and at least ONE of the following at Screening:
- Documented phosphate-regulating gene with homologies to endopeptidases on the X chromosome (PHEX) mutation in the patient or a directly related family member with appropriate X-linked inheritance
- Serum intact FGF23 (iFGF23) level > 30 pg/mL by Kainos assay
Biochemical findings consistent with XLH at Screening Visit 2 following overnight fasting (min. 8 hours):
- Serum phosphorus < 2.5 mg/dL
- TmP/GFR of < 2.5 mg/dL
Presence of skeletal pain attributed to XLH/osteomalacia, as defined by a score of ≥ 4 on Brief Pain Inventory (BPI) Questions 3 (Worst Pain) at Screening Visit 1. (Skeletal pain that, in the opinion of the investigator, is attributed solely to causes other than XLH/osteomalacia-for example, back or joint pain in the presence of severe osteoarthritis by radiograph in that anatomical location-in the absence of any skeletal pain likely attributed to XLH/osteomalacia should not be considered for eligibility)
Estimated glomerular filtration rate (eGFR) ≥ 60 mL/min (using the Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI] equation) or eGFR of 45 60 mL/min at Screening Visit 2 with confirmation that the renal insufficiency is not due to nephrocalcinosis
If taking chronic pain medications (including narcotic pain medications/opioids), must be on a stable regimen for at least 21 days prior to Screening Visit 1, and be willing to maintain medications at the same stable dose(s) and schedule throughout the Placebo-controlled Treatment Period of the study. The dose must not exceed 60 mg oral morphine equivalents/day
Provide written informed consent or if a minor, provide written consent and have a legally authorized representative willing and able to provide written informed consent, after the nature of the study has been explained, and prior to any research-related procedures
Willing to provide access to prior medical records for the collection of biochemical and radiographic data and disease history
Females of child-bearing potential must have a negative urine pregnancy test at Screening and be willing to have additional pregnancy tests during the study. Females considered not to be of childbearing potential include those who have been in menopause for at least two years prior to Screening, or have had tubal ligation at least one year prior to Screening, or have had a total hysterectomy or bilateral salpingo-oophorectomy
Participants of child-bearing potential or with partners of child-bearing potential who have not undergone a bilateral salpingo-oophorectomy and are sexually active must consent to use an effective method of contraception as determined by the site investigator (i.e. oral hormonal contraceptives, patch hormonal contraceptives, vaginal ring, intrauterine device, physical double-barrier methods, surgical hysterectomy, vasectomy, tubal ligation, or true abstinence) from the period following the signing of the informed consent through 12 weeks after last dose of study drug
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
Must have completed at least 4 of 7 days of the patient diaries before the Baseline visit.

Exclusion Criteria:

Use of a pharmacologic vitamin D metabolite or analog (calcitriol, doxercalciferol, and paricalcitol) within 14 days prior to Screening Visit 2
Use of oral phosphate within 14 days prior to Screening Visit 2
Use of aluminum hydroxide antacids, acetazolamides, and thiazides within 7 days prior to Screening Visit 2
Chronic use of systemic corticosteroids defined as more than 10 days in the previous 2 months
Corrected serum calcium level ≥ 10.8 mg/dL (2.7 mmol/L) at Screening Visit 2
Serum intact parathyroid hormone (iPTH) ≥ 2.5 upper limit of normal (ULN) at Screening Visit 1
Use of medication to suppress parathyroid hormone (PTH) (cinacalcet for example) within 60 days prior to Screening Visit 1
Use of oral bisphosphonates for 6 months or more in the 2 years prior to Screening Visit 1
Use of denosumab in the 6 months prior to Screening Visit 1
Use of teriparatide in the 2 months prior to Screening Visit 1
Planned or recommended orthopedic surgery within the first 24 weeks of the clinical trial period
History of traumatic fracture or orthopedic surgery within 6 months prior to Screening Visit 1
Use of KRN23, or any other therapeutic monoclonal antibody within 90 days prior to Screening Visit 1
Use of any investigational product or investigational medical device within 30 days prior to Screening Visit 1, 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.
Pregnant or breastfeeding at Screening /Baseline or planning to become pregnant (self or partner) at any time during the study
Unable or unwilling to withhold prohibited medications throughout the study
Presence or history of any hypersensitivity, allergic or anaphylactic reactions to any monoclonal antibody or KRN23 excipients that, in the judgment of the investigator, places the subject at increased risk for adverse effects.
Prior history of positive test for human immunodeficiency virus antibody, hepatitis B surface antigen, and/or hepatitis C antibody
History of recurrent infection or predisposition to infection, or of known immunodeficiency
Presence of malignant neoplasm (except basal cell carcinoma)
Presence of a concurrent disease or condition that would interfere with study participation or affect safety
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

Contacts/Locations Module

Name	Affiliation	Role
Medical Director	Ultragenyx Pharmaceutical Inc	Study Director

Facility	Status	City	State	ZIP	Country	Contacts
Children's Hospital of Los Angeles		Los Angeles	California	90027	United States
University of California-San Francisco Medical Center

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
29947083	Result	Insogna KL, Briot K, Imel EA, Kamenicky P, Ruppe MD, Portale AA, Weber T, Pitukcheewanont P, Cheong HI, Jan de Beur S, Imanishi Y, Ito N, Lachmann RH, Tanaka H, Perwad F, Zhang L, Chen CY, Theodore-Oklota C, Mealiffe M, San Martin J, Carpenter TO; AXLES 1 Investigators. A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial Evaluating the Efficacy of Burosumab, an Anti-FGF23 Antibody, in Adults With X-Linked Hypophosphatemia: Week 24 Primary Analysis. J Bone Miner Res. 2018 Aug;33(8):1383-1393. doi: 10.1002/jbmr.3475. Epub 2018 Jun 26.
31165191

IPD Sharing Statement Module

No data available

No data is available for this block.

Results Section

Participant Flow Module

Arm/Group Information

ID	Title	Description
FG000	Placebo	Subcutaneous (SC) injection of placebo every 4 weeks (Q4W) for 24 weeks (Double-blind Placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (Open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (Open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (Open-label Treatment Extension Period II).

Baseline Characteristics Module

Outcome Measures Module

Adverse Events Module

More Info Module

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Submission Tracking

No data available

No data is available for this block.

Condition Browse Module

Intervention Browse Module

Masking Info

FG00066 subjects
FG00168 subjects

Type of Units Analyzed

Baseline Measures

Title

Description

Population Description

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Denominator Units Selected

Denominators

Classes

Age, Continuous

Mean

Standard Deviation

years

Title

Denominators

Categories

Title	Measurements
	OG0007.6(3.3 to 16.5) OG00192.6(83.9 to 96.8)

Group IDs	Group Description	Statistical Method	Statistical Comment	P-Value	P-Value Comment	Parameter Type	Parameter Value	Dispersion Type	Dispersion Value	Confidence Interval Sides	Confidence Interval %	CI Lower Limit	CI Upper Limit	CI Lower Limit Comment	CI Upper Limit Comment	Estimate Comment	Tested Non-Inferiority	Non-Inferiority Type	Non-Inferiority Comment	Other Analysis Description
OG000 OG001		Cochran-Mantel-Haenszel		< 0.0001	From Cochran-Mantel-Haenszel (CMH) testing for association between achieving mean serum phosphorus levels above lower limit of normal (LLN) and treatment group, adjusting for stratification of Brief Pain Inventory (BPI) Average Pain and region.													Superiority

Secondary

Change From Baseline to Week 24 in Brief Pain Inventory (BPI) Question 3 (Q3; Worst Pain in Past 24 Hours) 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).

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.

Primary Analysis Set: all randomized participants who received at least 1 dose of study drug during the Placebo-Controlled Treatment Period.

Posted

Least Squares Mean

Standard Error

score on a scale

Baseline, 24 weeks

ID	Title	Description
OG000	Placebo	Placebo SC Q4W through Week 24
OG001	Burosumab	Burosumab 1 mg/kg SC Q4W

Secondary

Change From Baseline to Week 24 in the Western Ontario and McMaster University Osteoarthritis Index (WOMAC) Stiffness Score

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.

Primary Analysis Set: all randomized participants who received at least 1 dose of study drug during the Placebo-Controlled Treatment Period.

Posted

Least Squares Mean

Standard Error

score on a scale

Baseline, 24 weeks

ID	Title	Description
OG000	Placebo	Placebo SC Q4W through Week 24
OG001	Burosumab	Burosumab 1 mg/kg SC Q4W

Secondary

Change From Baseline to Week 24 in the WOMAC Physical Function Score

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.

Primary Analysis Set: all randomized participants who received at least 1 dose of study drug during the Placebo-Controlled Treatment Period.

Posted

Least Squares Mean

Standard Error

score on a scale

Baseline, 24 weeks

ID	Title	Description
OG000	Placebo	Placebo SC Q4W through Week 24
OG001	Burosumab	Burosumab 1 mg/kg SC Q4W

Secondary

Change From Baseline Over Time in BPI Worst Pain Score

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.

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.

Posted

Least Squares Mean

Standard Error

score on a scale

Baseline, Weeks 12, 24, 36, 48, 72, 96

ID	Title	Description
OG000	Placebo	Subcutaneous (SC) injection of placebo every 4 weeks (Q4W) for 24 weeks (Double-blind Placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (Open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (Open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (Open-label Treatment Extension Period II).

Secondary

Change From Baseline Over Time in BPI Pain Severity Score

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.

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.

Posted

Least Squares Mean

Standard Error

score on a scale

Baseline, Weeks 12, 24, 36, 48, 72, 96

ID	Title	Description
OG000	Placebo	Subcutaneous (SC) injection of placebo every 4 weeks (Q4W) for 24 weeks (Double-blind Placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (Open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (Open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (Open-label Treatment Extension Period II).

Secondary

Change From Baseline Over Time in BPI Pain Interference Score

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).

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.

Posted

Least Squares Mean

Standard Error

score on a scale

Baseline, Weeks 12, 24, 36, 48, 72, 96

ID	Title	Description
OG000	Placebo	Subcutaneous (SC) injection of placebo every 4 weeks (Q4W) for 24 weeks (Double-blind Placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (Open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (Open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (Open-label Treatment Extension Period II).

Secondary

Change From Baseline Over Time in WOMAC Stiffness Score

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.

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.

Posted

Least Squares Mean

Standard Error

score on a scale

Baseline, Weeks 12, 24, 36, 48, 72, 96, 120, 144

ID	Title	Description
OG000	Placebo	Subcutaneous (SC) injection of placebo every 4 weeks (Q4W) for 24 weeks (Double-blind Placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (Open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (Open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (Open-label Treatment Extension Period II).

Secondary

Change From Baseline Over Time in WOMAC Physical Function Score

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.

Posted

Least Squares Mean

Standard Error

score on a scale

Baseline, Weeks 12, 24, 36, 48, 72, 96, 120, 144

ID	Title	Description
OG000	Placebo	Subcutaneous (SC) injection of placebo every 4 weeks (Q4W) for 24 weeks (Double-blind Placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (Open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (Open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (Open-label Treatment Extension Period II).

Secondary

Change From Baseline Over Time in BFI Worst Fatigue Score

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.

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.

Posted

Least Squares Mean

Standard Error

score on a scale

Baseline, Weeks 12, 24, 36, 48, 72, 96

ID	Title	Description
OG000	Placebo	Subcutaneous (SC) injection of placebo every 4 weeks (Q4W) for 24 weeks (Double-blind Placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (Open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (Open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (Open-label Treatment Extension Period II).

Secondary

Change From Baseline Over Time in BFI Global Fatigue Score

Change from baseline to post-baseline visits in BFI global fatigue score, calculated by averaging all 9 BFI items as recorded on the day of the study visit. The BFI is a self-reported questionnaire consisting of 9 items related to fatigue that are rated on a 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). BFI Global Fatigue score was calculated by averaging all 9 items on the BFI. Global scores range from 0 to 10, with higher score indicating worse fatigue severity and interference.

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.

Posted

Least Squares Mean

Standard Error

score on a scale

Baseline, Weeks 12, 24, 36, 48, 72, 96

ID	Title	Description
OG000	Placebo	SC injection of placebo Q4W for 24 weeks (Double-blind Placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (Open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (Open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (Open-label Treatment Extension Period II).

Secondary

Change From Baseline Over Time in Biochemical Marker of Bone Remodeling Procollagen Type 1 N-Propeptide (P1NP)

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.

Posted

Least Squares Mean

Standard Error

ng/mL

Baseline, Weeks 12, 24, 36, 48, 72, 96

ID	Title	Description
OG000	Placebo	SC injection of placebo Q4W for 24 weeks (Double-blind Placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (Open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (Open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (Open-label Treatment Extension Period II).
OG001	Burosumab 1 mg/kg	SC injection of 1.0 mg/kg burosumab Q4W for 24 weeks (double-blind placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (open-label Treatment Extension Period II).

Units	Counts

Secondary

Percent Change From Baseline Over Time in Biochemical Marker of Bone Remodeling P1NP

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.

Posted

Least Squares Mean

Standard Error

percentage change

Baseline, Weeks 12, 24, 36, 48, 72, 96

ID	Title	Description
OG000	Placebo	SC injection of placebo Q4W for 24 weeks (Double-blind Placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (Open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (Open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (Open-label Treatment Extension Period II).
OG001	Burosumab 1 mg/kg	SC injection of 1.0 mg/kg burosumab Q4W for 24 weeks (double-blind placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (open-label Treatment Extension Period II).

Units	Counts

Secondary

Change From Baseline Over Time in Biochemical Marker of Bone Remodeling Carboxy-Terminal Cross-Linked Telopeptide of Type I Collagen (CTx)

The GEE Estimates are from the GEE model which includes the change from baseline for CTx as the dependent variable, region, visit, treatment, actual randomization stratification and visit by treatment as fixed factors, and baseline of CTx as a covariate, with compound symmetry covariance structure.

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.

Posted

Least Squares Mean

Standard Error

pg/mL

Baseline, Weeks 12, 24, 36, 48, 72, 96

ID	Title	Description
OG000	Placebo	SC injection of placebo Q4W for 24 weeks (Double-blind Placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (Open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (Open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (Open-label Treatment Extension Period II).
OG001	Burosumab 1 mg/kg	SC injection of 1.0 mg/kg burosumab Q4W for 24 weeks (double-blind placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (open-label Treatment Extension Period II).

Secondary

Percent Change From Baseline Over Time in Biochemical Marker of Bone Remodeling CTx

The GEE Estimates are from the GEE model which includes the percent change from baseline for CTx as the dependent variable, region, visit, treatment, actual randomization stratification and visit by treatment as fixed factors, and baseline of CTx as a covariate, with compound symmetry covariance structure.

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.

Posted

Least Squares Mean

Standard Error

percentage change

Baseline, Weeks 12, 24, 36, 48, 72, 96

ID	Title	Description
OG000	Placebo	SC injection of placebo Q4W for 24 weeks (Double-blind Placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (Open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (Open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (Open-label Treatment Extension Period II).
OG001	Burosumab 1 mg/kg	SC injection of 1.0 mg/kg burosumab Q4W for 24 weeks (double-blind placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (open-label Treatment Extension Period II).

Secondary

Change From Baseline Over Time in Biochemical Marker of Bone Remodeling Bone-Specific Alkaline Phosphatase (BALP)

The GEE Estimates are from the GEE model which includes the change from baseline for BALP as the dependent variable, region, visit, treatment, actual randomization stratification and visit by treatment as fixed factors, and baseline of BALP as a covariate, with compound symmetry covariance structure.

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.

Posted

Least Squares Mean

Standard Error

μg/L

Baseline, Weeks 12, 24, 36, 48, 72, 96

ID	Title	Description
OG000	Placebo	SC injection of placebo Q4W for 24 weeks (Double-blind Placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (Open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (Open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (Open-label Treatment Extension Period II).
OG001	Burosumab 1 mg/kg	SC injection of 1.0 mg/kg burosumab Q4W for 24 weeks (double-blind placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (open-label Treatment Extension Period II).

Secondary

Percent Change From Baseline Over Time in Biochemical Marker of Bone Remodeling BALP

The GEE Estimates are from the GEE model which includes the percent change from baseline for BALP as the dependent variable, region, visit, treatment, actual randomization stratification and visit by treatment as fixed factors, and baseline of Bone ALP as a covariate, with compound symmetry covariance structure.

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.

Posted

Least Squares Mean

Standard Error

percentage change

Baseline, Weeks 12, 24, 36, 48, 72, 96

ID	Title	Description
OG000	Placebo	SC injection of placebo Q4W for 24 weeks (Double-blind Placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (Open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (Open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (Open-label Treatment Extension Period II).
OG001	Burosumab 1 mg/kg	SC injection of 1.0 mg/kg burosumab Q4W for 24 weeks (double-blind placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (open-label Treatment Extension Period II).

Secondary

Change From Baseline Over Time in Serum Phosphorus

The GEE Estimates are from the GEE model which includes the change from baseline for serum phosphorus as the dependent variable, region, visit, treatment, actual randomization stratification and visit by treatment as fixed factors, and baseline of serum phosphorus as a covariate, with compound symmetry covariance structure.

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.

Posted

Least Squares Mean

Standard Error

mmol/L

Baseline, Weeks 1, 2, 4, 6, 10, 12, 14, 18, 20, 21, 22, 24, 26, 28, 34, 36, 46, 48, 60, 70, 72, 84, 94, 96, 108, 120, 132, 144

ID	Title	Description
OG000	Placebo	SC injection of placebo Q4W for 24 weeks (Double-blind Placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (Open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (Open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (Open-label Treatment Extension Period II).
OG001	Burosumab 1 mg/kg	SC injection of 1.0 mg/kg burosumab Q4W for 24 weeks (double-blind placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (open-label Treatment Extension Period II).

Secondary

Percent Change From Baseline Over Time in Serum Phosphorus

The GEE Estimates are from the GEE model which includes the percent change from baseline for serum phosphorus as the dependent variable, region, visit, treatment, actual randomization stratification and visit by treatment as fixed factors, and baseline of serum phosphorus as a covariate, with compound symmetry covariance structure.

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.

Posted

Least Squares Mean

Standard Error

percentage change

Baseline, Weeks 1, 2, 4, 6, 10, 12, 14, 18, 20, 21, 22, 24, 26, 28, 34, 36, 46, 48, 60, 70, 72, 84, 94, 96, 108, 120, 132, 144

ID	Title	Description
OG000	Placebo	SC injection of placebo Q4W for 24 weeks (Double-blind Placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (Open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (Open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (Open-label Treatment Extension Period II).
OG001	Burosumab 1 mg/kg	SC injection of 1.0 mg/kg burosumab Q4W for 24 weeks (double-blind placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (open-label Treatment Extension Period II).

Secondary

Change From Baseline Over Time in Serum 1,25(OH)2D

The GEE Estimates are from the GEE model which includes the change from baseline for 1,25(OH)2D as the dependent variable, region, visit, treatment, actual randomization stratification and visit by treatment as fixed factors, and baseline of 1, 25 (OH)2 D as a covariate, with compound symmetry covariance structure.

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.

Posted

Least Squares Mean

Standard Error

pg/mL

Baseline, Weeks 1, 2, 4, 20, 21, 22, 46, 48, 60, 70, 72, 84, 94, 96, 108, 120, 132, 144

ID	Title	Description
OG000	Placebo	SC injection of placebo Q4W for 24 weeks (Double-blind Placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (Open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (Open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (Open-label Treatment Extension Period II).
OG001	Burosumab 1 mg/kg	SC injection of 1.0 mg/kg burosumab Q4W for 24 weeks (double-blind placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (open-label Treatment Extension Period II).

Secondary

Percent Change From Baseline Over Time in Serum 1,25(OH)2D

The GEE Estimates are from the GEE model which includes the percent change from baseline for 1,25(OH)2D as the dependent variable, region, visit, treatment, actual randomization stratification and visit by treatment as fixed factors, and baseline of 1, 25 (OH)2 D as a covariate, with compound symmetry covariance structure.

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.

Posted

Least Squares Mean

Standard Error

percentage change

Baseline, Weeks 1, 2, 4, 20, 21, 22, 46, 48, 60, 70, 72, 84, 94, 96, 108, 120, 132, 144

ID	Title	Description
OG000	Placebo	SC injection of placebo Q4W for 24 weeks (Double-blind Placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (Open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (Open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (Open-label Treatment Extension Period II).
OG001	Burosumab 1 mg/kg	SC injection of 1.0 mg/kg burosumab Q4W for 24 weeks (double-blind placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (open-label Treatment Extension Period II).

Secondary

Change From Baseline Over Time in 24-Hour Urinary Phosphorus

The GEE Estimates are from the GEE model which includes the change from baseline for 24-Hour urinary phosphorus as the dependent variable, region, visit, treatment, actual randomization stratification and visit by treatment as fixed factors, and baseline of urinary phosphorus as a covariate, with compound symmetry covariance structure.

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.

Posted

Least Squares Mean

Standard Error

g/24 hour

Baseline, Week 12, 24, 36, 48, 72, 96

ID	Title	Description
OG000	Placebo	SC injection of placebo Q4W for 24 weeks (Double-blind Placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (Open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (Open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (Open-label Treatment Extension Period II).
OG001	Burosumab 1 mg/kg	SC injection of 1.0 mg/kg burosumab Q4W for 24 weeks (double-blind placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (open-label Treatment Extension Period II).

Secondary

Percent Change From Baseline Over Time in 24-Hour Urinary Phosphorus

The GEE Estimates are from the GEE model which includes the percent change from baseline for 24-hour urinary phosphorus as the dependent variable, region, visit, treatment, actual randomization stratification and visit by treatment as fixed factors, and baseline of urinary phosphorus as a covariate, with compound symmetry covariance structure.

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.

Posted

Least Squares Mean

Standard Error

percentage change

Baseline, Week 12, 24, 36, 48, 72, 96

ID	Title	Description
OG000	Placebo	SC injection of placebo Q4W for 24 weeks (Double-blind Placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (Open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (Open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (Open-label Treatment Extension Period II).
OG001	Burosumab 1 mg/kg	SC injection of 1.0 mg/kg burosumab Q4W for 24 weeks (double-blind placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (open-label Treatment Extension Period II).

Secondary

Change From Baseline Over Time in Ratio of Renal Tubular Maximum Reabsorption Rate of Phosphate to Glomerular Filtration Rate (TmP/GFR)

The GEE Estimates are from the GEE model which includes the change from baseline for TmP/GFR as the dependent variable, region, visit, treatment, actual randomization stratification and visit by treatment as fixed factors, and baseline of TmP/GFR as a covariate, with compound symmetry covariance structure.

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.

Posted

Least Squares Mean

Standard Error

mg/dL

Baseline, Weeks 2, 4, 12, 22, 24, 48, 60, 72, 84, 96

ID	Title	Description
OG000	Placebo	SC injection of placebo Q4W for 24 weeks (Double-blind Placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (Open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (Open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (Open-label Treatment Extension Period II).
OG001	Burosumab 1 mg/kg	SC injection of 1.0 mg/kg burosumab Q4W for 24 weeks (double-blind placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (open-label Treatment Extension Period II).

Secondary

Percent Change From Baseline Over Time in TmP/GFR

The GEE Estimates are from the GEE model which includes the percent change from baseline for TmP/GFR as the dependent variable, region, visit, treatment, actual randomization stratification and visit by treatment as fixed factors, and baseline of TmP/GFR as a covariate, with compound symmetry covariance structure.

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.

Posted

Least Squares Mean

Standard Error

percentage change

Baseline, Weeks 2, 4, 12, 22, 24, 48, 60, 72, 84, 96

ID	Title	Description
OG000	Placebo	SC injection of placebo Q4W for 24 weeks (Double-blind Placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (Open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (Open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (Open-label Treatment Extension Period II).
OG001	Burosumab 1 mg/kg	SC injection of 1.0 mg/kg burosumab Q4W for 24 weeks (double-blind placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (open-label Treatment Extension Period II).

Secondary

Change From Baseline Over Time in Tubular Reabsorption of Phosphate (TRP)

The GEE Estimates are from the GEE model which includes the change from baseline for TRP as the dependent variable, region, visit, treatment, actual randomization stratification and visit by treatment as fixed factors, and baseline of TRP as a covariate, with compound symmetry covariance structure.

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.

Posted

Least Squares Mean

Standard Error

fraction of phosphate reabsorbed

Baseline, Weeks 2, 4, 12, 22, 24, 48, 60, 72, 84, 96

ID	Title	Description
OG000	Placebo	SC injection of placebo Q4W for 24 weeks (Double-blind Placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (Open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (Open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (Open-label Treatment Extension Period II).
OG001	Burosumab 1 mg/kg	SC injection of 1.0 mg/kg burosumab Q4W for 24 weeks (double-blind placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (open-label Treatment Extension Period II).

Secondary

Percent Change From Baseline Over Time in TRP

The GEE Estimates are from the GEE model which includes the percent change from baseline for TRP as the dependent variable, region, visit, treatment, actual randomization stratification and visit by treatment as fixed factors, and baseline of TRP as a covariate, with compound symmetry covariance structure.

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.

Posted

Least Squares Mean

Standard Error

percentage change

Baseline, Weeks 2, 4, 12, 22, 24, 48, 60, 72, 84, 96

ID	Title	Description
OG000	Placebo	SC injection of placebo Q4W for 24 weeks (Double-blind Placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (Open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (Open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (Open-label Treatment Extension Period II).
OG001	Burosumab 1 mg/kg	SC injection of 1.0 mg/kg burosumab Q4W for 24 weeks (double-blind placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (open-label Treatment Extension Period II).

Secondary

Percentage of Participants Achieving Mean Serum Phosphorus Levels Above the LLN (2.5 mg/dL [0.81 mmol/L]) at the End of the Dosing Cycle, as Averaged Across Dose Cycles Between Baseline and Week 24

Primary Analysis Set: all randomized participants who received at least 1 dose of study drug during the Placebo-Controlled Treatment Period.

Posted

Number

95% Confidence Interval

percentage of participants

Baseline through Week 24

ID	Title	Description
OG000	Placebo	Placebo SC Q4W through Week 24
OG001	Burosumab	Burosumab 1 mg/kg SC Q4W

Units	Counts
Participants	OG000

Secondary

Change From Baseline in Serum Phosphorus at Each Mid-Point of Dosing Cycle, as Averaged Across Dose Cycles Between Baseline and Week 24

Primary Analysis Set: all randomized participants who received at least 1 dose of study drug during the Placebo-Controlled Treatment Period.

Posted

Mean

Standard Deviation

mg/dL

Baseline through Week 24

ID	Title	Description
OG000	Placebo	Placebo SC Q4W through Week 24
OG001	Burosumab	Burosumab 1 mg/kg SC Q4W

Units	Counts
Participants	OG000

Secondary

Percent Change From Baseline in Serum Phosphorus at Each Mid-Point of Dosing Cycle, as Averaged Across Dose Cycles Between Baseline and Week 24

Primary Analysis Set: all randomized participants who received at least 1 dose of study drug during the Placebo-Controlled Treatment Period.

Posted

Mean

Standard Deviation

percentage change

Baseline through Week 24

ID	Title	Description
OG000	Placebo	Placebo SC Q4W through Week 24
OG001	Burosumab	Burosumab 1 mg/kg SC Q4W

Units	Counts
Participants	OG000

Secondary

Change From Baseline in Serum Phosphorus at Each End of Dosing Cycle, as Averaged Across Dose Cycles Between Baseline and Week 24

Primary Analysis Set: all randomized participants who received at least 1 dose of study drug during the Placebo-Controlled Treatment Period.

Posted

Mean

Standard Deviation

mg/dL

Baseline through Week 24

ID	Title	Description
OG000	Placebo	Placebo SC Q4W through Week 24
OG001	Burosumab	Burosumab 1 mg/kg SC Q4W

Units	Counts
Participants	OG000

Secondary

Percent Change From Baseline in Serum Phosphorus at Each End of Dosing Cycle, as Averaged Across Dose Cycles Between Baseline and Week 24

Primary Analysis Set: all randomized participants who received at least 1 dose of study drug during the Placebo-Controlled Treatment Period.

Posted

Mean

Standard Deviation

percentage change

Baseline through Week 24

ID	Title	Description
OG000	Placebo	Placebo SC Q4W through Week 24
OG001	Burosumab	Burosumab 1 mg/kg SC Q4W

Units	Counts
Participants	OG000

Secondary

Time-Adjusted Area Under the Curve (AUC) of Serum Phosphorus Between Baseline and Week 24

Primary Analysis Set: all randomized participants who received at least 1 dose of study drug during the Placebo-Controlled Treatment Period.

Posted

Mean

Standard Deviation

mg/dL

Baseline through Week 24

ID	Title	Description
OG000	Placebo	Placebo SC Q4W through Week 24
OG001	Burosumab	Burosumab 1 mg/kg SC Q4W

Units	Counts
Participants	OG000

Arm/Groups

ID	Title	Description	Deaths (Affected)	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	Placebo (DB Period)	SC injection of placebo Q4W for 24 weeks (double-blind placebo-controlled Treatment Period)	0	66	1	66	57	66
EG001	Placebo -> Burosumab (OL Period)	SC injection of placebo Q4W for 24 weeks (double-blind placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (open label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (open-label Treatment Extension Period II [US only]).	0	66	10	66	63	66
EG002	Burosumab -> Burosumab (Combined DB and OL Period)	SC injection of 1.0 mg/kg burosumab Q4W for 24 weeks (double-blind placebo-controlled Treatment Period), SC injection of burosumab 1 mg/kg Q4W for 24 weeks (open-label Treatment Continuation Period), SC injection of burosumab 1 mg/kg Q4W for 48 weeks (open-label Treatment Extension Period I), and SC injection of burosumab 1 mg/kg Q4W up to 53 weeks (open-label Treatment Extension Period II [US only]).	1	68	12	68	68	68
EG003	Total Burosumab (Combined DB and OL Period)	SC injection of 1.0 mg/kg burosumab at any time during the study.	1	134	22	134	131	134

Serious Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Palpitations	Cardiac disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0011 affected66 at risk EG0020 affected68 at risk EG0031 affected134 at risk
Colitis	Gastrointestinal disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0010 affected66 at risk EG0021 affected68 at risk EG003
Duodenal Ulcer	Gastrointestinal disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0010 affected66 at risk EG0021 affected68 at risk EG003
Ileus	Gastrointestinal disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0010 affected66 at risk EG0021 affected68 at risk EG003
Irritable Bowel Syndrome	Gastrointestinal disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0010 affected66 at risk EG0021 affected68 at risk EG003
Periodontal Disease	Gastrointestinal disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0011 affected66 at risk EG0020 affected68 at risk EG003
Peritoneal Adhesions	Gastrointestinal disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0011 affected66 at risk EG0020 affected68 at risk EG003
Cholelithiasis	Hepatobiliary disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0010 affected66 at risk EG0021 affected68 at risk EG003
Gastroenteritis	Infections and infestations	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0010 affected66 at risk EG0021 affected68 at risk EG003
Medical Device Site Infection	Infections and infestations	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0010 affected66 at risk EG0021 affected68 at risk EG003
Overdose	Injury, poisoning and procedural complications	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0010 affected66 at risk EG0021 affected68 at risk EG003
Procedural Nausea	Injury, poisoning and procedural complications	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0010 affected66 at risk EG0021 affected68 at risk EG003
Procedural Vomiting	Injury, poisoning and procedural complications	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0010 affected66 at risk EG0021 affected68 at risk EG003
Road Traffic Accident	Injury, poisoning and procedural complications	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0010 affected66 at risk EG0021 affected68 at risk EG003
Subdural Haematoma	Injury, poisoning and procedural complications	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0011 affected66 at risk EG0020 affected68 at risk EG003
Arthralgia	Musculoskeletal and connective tissue disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0011 affected66 at risk EG0021 affected68 at risk EG003
Back Pain	Musculoskeletal and connective tissue disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0010 affected66 at risk EG0021 affected68 at risk EG003
Cervical Spinal Stenosis	Musculoskeletal and connective tissue disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0011 affected66 at risk EG0020 affected68 at risk EG003
Joint Range Of Motion Decreased	Musculoskeletal and connective tissue disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0011 affected66 at risk EG0020 affected68 at risk EG003
Knee Deformity	Musculoskeletal and connective tissue disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0010 affected66 at risk EG0021 affected68 at risk EG003
Musculoskeletal Pain	Musculoskeletal and connective tissue disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0010 affected66 at risk EG0021 affected68 at risk EG003
Osteoarthritis	Musculoskeletal and connective tissue disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0010 affected66 at risk EG0021 affected68 at risk EG003
Pseudarthrosis	Musculoskeletal and connective tissue disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0011 affected66 at risk EG0020 affected68 at risk EG003
Spinal Column Stenosis	Musculoskeletal and connective tissue disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0010 affected66 at risk EG0021 affected68 at risk EG003
Spondylolisthesis	Musculoskeletal and connective tissue disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0010 affected66 at risk EG0021 affected68 at risk EG003
Invasive Ductal Breast Carcinoma	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	MedDRA 18.1	Systematic Assessment	EG0001 affected66 at risk EG0010 affected66 at risk EG0020 affected68 at risk EG003
Parathyroid Tumour Benign	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0011 affected66 at risk EG0020 affected68 at risk EG003
Cerebrospinal Fluid Leakage	Nervous system disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0010 affected66 at risk EG0021 affected68 at risk EG003
Cervical Radiculopathy	Nervous system disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0010 affected66 at risk EG0021 affected68 at risk EG003
Dizziness	Nervous system disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0011 affected66 at risk EG0020 affected68 at risk EG003
Myelopathy	Nervous system disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0010 affected66 at risk EG0022 affected68 at risk EG003
Neuralgia	Nervous system disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0011 affected66 at risk EG0020 affected68 at risk EG003
Paraesthesia	Nervous system disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0011 affected66 at risk EG0020 affected68 at risk EG003
Partial Seizures	Nervous system disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0011 affected66 at risk EG0020 affected68 at risk EG003
Presyncope	Nervous system disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0011 affected66 at risk EG0020 affected68 at risk EG003

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Vertigo	Ear and labyrinth disorders	MedDRA 18.1	Systematic Assessment	EG0001 affected66 at risk EG0014 affected66 at risk EG0023 affected68 at risk EG0037 affected134 at risk
Hyperparathyroidism Secondary	Endocrine disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0013 affected66 at risk EG0024 affected68 at risk EG003
Abdominal Discomfort	Gastrointestinal disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0013 affected66 at risk EG0024 affected68 at risk EG003
Abdominal Pain	Gastrointestinal disorders	MedDRA 18.1	Systematic Assessment	EG0002 affected66 at risk EG0012 affected66 at risk EG0026 affected68 at risk EG003
Abdominal Pain Upper	Gastrointestinal disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0016 affected66 at risk EG0023 affected68 at risk EG003
Constipation	Gastrointestinal disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0014 affected66 at risk EG00210 affected68 at risk EG003
Diarrhoea	Gastrointestinal disorders	MedDRA 18.1	Systematic Assessment	EG0005 affected66 at risk EG0015 affected66 at risk EG00213 affected68 at risk EG003
Nausea	Gastrointestinal disorders	MedDRA 18.1	Systematic Assessment	EG0006 affected66 at risk EG0015 affected66 at risk EG00211 affected68 at risk EG003
Toothache	Gastrointestinal disorders	MedDRA 18.1	Systematic Assessment	EG0001 affected66 at risk EG00110 affected66 at risk EG00212 affected68 at risk EG003
Vomiting	Gastrointestinal disorders	MedDRA 18.1	Systematic Assessment	EG0002 affected66 at risk EG0014 affected66 at risk EG0026 affected68 at risk EG003
Asthenia	General disorders	MedDRA 18.1	Systematic Assessment	EG0002 affected66 at risk EG0011 affected66 at risk EG0025 affected68 at risk EG003
Fatigue	General disorders	MedDRA 18.1	Systematic Assessment	EG0007 affected66 at risk EG00114 affected66 at risk EG00216 affected68 at risk EG003
Injection Site Erythema	General disorders	MedDRA 18.1	Systematic Assessment	EG0002 affected66 at risk EG0016 affected66 at risk EG0026 affected68 at risk EG003
Injection Site Pruritus	General disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0018 affected66 at risk EG0023 affected68 at risk EG003
Injection Site Reaction	General disorders	MedDRA 18.1	Systematic Assessment	EG0002 affected66 at risk EG0018 affected66 at risk EG0027 affected68 at risk EG003
Pain	General disorders	MedDRA 18.1	Systematic Assessment	EG0006 affected66 at risk EG0019 affected66 at risk EG00212 affected68 at risk EG003
Pyrexia	General disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0014 affected66 at risk EG0025 affected68 at risk EG003
Seasonal Allergy	Immune system disorders	MedDRA 18.1	Systematic Assessment	EG0001 affected66 at risk EG0016 affected66 at risk EG0025 affected68 at risk EG003
Bronchitis	Infections and infestations	MedDRA 18.1	Systematic Assessment	EG0001 affected66 at risk EG0016 affected66 at risk EG0024 affected68 at risk EG003
Gastroenteritis	Infections and infestations	MedDRA 18.1	Systematic Assessment	EG0002 affected66 at risk EG0014 affected66 at risk EG0022 affected68 at risk EG003
Influenza	Infections and infestations	MedDRA 18.1	Systematic Assessment	EG0003 affected66 at risk EG0016 affected66 at risk EG0029 affected68 at risk EG003
Nasopharyngitis	Infections and infestations	MedDRA 18.1	Systematic Assessment	EG0006 affected66 at risk EG00124 affected66 at risk EG00228 affected68 at risk EG003
Oral Herpes	Infections and infestations	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0014 affected66 at risk EG0024 affected68 at risk EG003
Pharyngitis	Infections and infestations	MedDRA 18.1	Systematic Assessment	EG0001 affected66 at risk EG0010 affected66 at risk EG0024 affected68 at risk EG003
Rhinitis	Infections and infestations	MedDRA 18.1	Systematic Assessment	EG0001 affected66 at risk EG0014 affected66 at risk EG0021 affected68 at risk EG003
Sinusitis	Infections and infestations	MedDRA 18.1	Systematic Assessment	EG0002 affected66 at risk EG0012 affected66 at risk EG0026 affected68 at risk EG003
Tooth Abscess	Infections and infestations	MedDRA 18.1	Systematic Assessment	EG0006 affected66 at risk EG0015 affected66 at risk EG00219 affected68 at risk EG003
Upper Respiratory Tract Infection	Infections and infestations	MedDRA 18.1	Systematic Assessment	EG0006 affected66 at risk EG0012 affected66 at risk EG00212 affected68 at risk EG003
Urinary Tract Infection	Infections and infestations	MedDRA 18.1	Systematic Assessment	EG0004 affected66 at risk EG0016 affected66 at risk EG0025 affected68 at risk EG003
Contusion	Injury, poisoning and procedural complications	MedDRA 18.1	Systematic Assessment	EG0001 affected66 at risk EG0015 affected66 at risk EG0024 affected68 at risk EG003
Fall	Injury, poisoning and procedural complications	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0017 affected66 at risk EG0026 affected68 at risk EG003
Ligament Sprain	Injury, poisoning and procedural complications	MedDRA 18.1	Systematic Assessment	EG0006 affected66 at risk EG0012 affected66 at risk EG0024 affected68 at risk EG003
Procedural Pain	Injury, poisoning and procedural complications	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0016 affected66 at risk EG0029 affected68 at risk EG003
Blood 25-Hydroxycholecalciferol Decreased	Investigations	MedDRA 18.1	Systematic Assessment	EG0001 affected66 at risk EG0013 affected66 at risk EG0026 affected68 at risk EG003
Blood Glucose Increased	Investigations	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0011 affected66 at risk EG0025 affected68 at risk EG003
Blood Parathyroid Hormone Increased	Investigations	MedDRA 18.1	Systematic Assessment	EG0001 affected66 at risk EG0011 affected66 at risk EG0025 affected68 at risk EG003
Blood Pressure Increased	Investigations	MedDRA 18.1	Systematic Assessment	EG0001 affected66 at risk EG0011 affected66 at risk EG0026 affected68 at risk EG003
Lipase Increased	Investigations	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0010 affected66 at risk EG0024 affected68 at risk EG003
Vitamin D Decreased	Investigations	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0019 affected66 at risk EG0028 affected68 at risk EG003
Decreased Appetite	Metabolism and nutrition disorders	MedDRA 18.1	Systematic Assessment	EG0001 affected66 at risk EG0010 affected66 at risk EG0024 affected68 at risk EG003
Hyperphosphataemia	Metabolism and nutrition disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0014 affected66 at risk EG0021 affected68 at risk EG003
Vitamin D Deficiency	Metabolism and nutrition disorders	MedDRA 18.1	Systematic Assessment	EG0003 affected66 at risk EG0017 affected66 at risk EG00215 affected68 at risk EG003
Arthralgia	Musculoskeletal and connective tissue disorders	MedDRA 18.1	Systematic Assessment	EG00016 affected66 at risk EG00123 affected66 at risk EG00227 affected68 at risk EG003
Back Pain	Musculoskeletal and connective tissue disorders	MedDRA 18.1	Systematic Assessment	EG0006 affected66 at risk EG00117 affected66 at risk EG00218 affected68 at risk EG003
Bone Pain	Musculoskeletal and connective tissue disorders	MedDRA 18.1	Systematic Assessment	EG0004 affected66 at risk EG0016 affected66 at risk EG0028 affected68 at risk EG003
Joint Stiffness	Musculoskeletal and connective tissue disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0014 affected66 at risk EG0024 affected68 at risk EG003
Joint Swelling	Musculoskeletal and connective tissue disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0017 affected66 at risk EG0025 affected68 at risk EG003
Muscle Spasms	Musculoskeletal and connective tissue disorders	MedDRA 18.1	Systematic Assessment	EG0002 affected66 at risk EG0019 affected66 at risk EG00211 affected68 at risk EG003
Muscular Weakness	Musculoskeletal and connective tissue disorders	MedDRA 18.1	Systematic Assessment	EG0001 affected66 at risk EG0012 affected66 at risk EG0026 affected68 at risk EG003
Musculoskeletal Chest Pain	Musculoskeletal and connective tissue disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0014 affected66 at risk EG0023 affected68 at risk EG003
Musculoskeletal Pain	Musculoskeletal and connective tissue disorders	MedDRA 18.1	Systematic Assessment	EG0004 affected66 at risk EG00110 affected66 at risk EG00212 affected68 at risk EG003
Musculoskeletal Stiffness	Musculoskeletal and connective tissue disorders	MedDRA 18.1	Systematic Assessment	EG0003 affected66 at risk EG0014 affected66 at risk EG0024 affected68 at risk EG003
Myalgia	Musculoskeletal and connective tissue disorders	MedDRA 18.1	Systematic Assessment	EG0001 affected66 at risk EG0015 affected66 at risk EG0028 affected68 at risk EG003
Neck Pain	Musculoskeletal and connective tissue disorders	MedDRA 18.1	Systematic Assessment	EG0001 affected66 at risk EG0015 affected66 at risk EG0023 affected68 at risk EG003
Pain In Extremity	Musculoskeletal and connective tissue disorders	MedDRA 18.1	Systematic Assessment	EG00010 affected66 at risk EG00110 affected66 at risk EG00218 affected68 at risk EG003
Dizziness	Nervous system disorders	MedDRA 18.1	Systematic Assessment	EG0004 affected66 at risk EG0015 affected66 at risk EG00211 affected68 at risk EG003
Headache	Nervous system disorders	MedDRA 18.1	Systematic Assessment	EG0005 affected66 at risk EG00118 affected66 at risk EG00222 affected68 at risk EG003
Hypoaesthesia	Nervous system disorders	MedDRA 18.1	Systematic Assessment	EG0001 affected66 at risk EG0013 affected66 at risk EG0027 affected68 at risk EG003
Migraine	Nervous system disorders	MedDRA 18.1	Systematic Assessment	EG0001 affected66 at risk EG0012 affected66 at risk EG0027 affected68 at risk EG003
Restless Legs Syndrome	Nervous system disorders	MedDRA 18.1	Systematic Assessment	EG0005 affected66 at risk EG00110 affected66 at risk EG00211 affected68 at risk EG003
Depression	Psychiatric disorders	MedDRA 18.1	Systematic Assessment	EG0001 affected66 at risk EG0013 affected66 at risk EG0029 affected68 at risk EG003
Insomnia	Psychiatric disorders	MedDRA 18.1	Systematic Assessment	EG0001 affected66 at risk EG0016 affected66 at risk EG0029 affected68 at risk EG003
Nephrocalcinosis	Renal and urinary disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0014 affected66 at risk EG0022 affected68 at risk EG003
Asthma	Respiratory, thoracic and mediastinal disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0015 affected66 at risk EG0022 affected68 at risk EG003
Cough	Respiratory, thoracic and mediastinal disorders	MedDRA 18.1	Systematic Assessment	EG0003 affected66 at risk EG00111 affected66 at risk EG00210 affected68 at risk EG003
Nasal Congestion	Respiratory, thoracic and mediastinal disorders	MedDRA 18.1	Systematic Assessment	EG0002 affected66 at risk EG0015 affected66 at risk EG0025 affected68 at risk EG003
Oropharyngeal Pain	Respiratory, thoracic and mediastinal disorders	MedDRA 18.1	Systematic Assessment	EG0007 affected66 at risk EG0018 affected66 at risk EG0024 affected68 at risk EG003
Rhinorrhoea	Respiratory, thoracic and mediastinal disorders	MedDRA 18.1	Systematic Assessment	EG0003 affected66 at risk EG0012 affected66 at risk EG0024 affected68 at risk EG003
Pruritus	Skin and subcutaneous tissue disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0011 affected66 at risk EG0025 affected68 at risk EG003
Rash	Skin and subcutaneous tissue disorders	MedDRA 18.1	Systematic Assessment	EG0003 affected66 at risk EG0011 affected66 at risk EG0024 affected68 at risk EG003
Hot Flush	Vascular disorders	MedDRA 18.1	Systematic Assessment	EG0000 affected66 at risk EG0012 affected66 at risk EG0026 affected68 at risk EG003
Hypertension	Vascular disorders	MedDRA 18.1	Systematic Assessment	EG0002 affected66 at risk EG0015 affected66 at risk EG0028 affected68 at risk EG003

ID	Term
D053098	Familial Hypophosphatemic Rickets

Ancestor Terms

ID	Term
D063730	Rickets, Hypophosphatemic
D012279	Rickets
D001851	Bone Diseases, Metabolic
D001847	Bone Diseases
D009140	Musculoskeletal Diseases
D007015	Hypophosphatemia, Familial
D015499	Renal Tubular Transport, Inborn Errors
D007674	Kidney Diseases
D014570	Urologic Diseases
D052776	Female Urogenital Diseases
D005261	Female Urogenital Diseases and Pregnancy Complications
D000091642	Urogenital Diseases
D052801	Male Urogenital Diseases
D008664	Metal Metabolism, Inborn Errors
D008661	Metabolism, Inborn Errors
D030342	Genetic Diseases, Inborn
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities
D008659	Metabolic Diseases
D009750	Nutritional and Metabolic Diseases
D002128	Calcium Metabolism Disorders
D017674	Hypophosphatemia
D010760	Phosphorus Metabolism Disorders
D014808	Vitamin D Deficiency
D001361	Avitaminosis
D003677	Deficiency Diseases
D044342	Malnutrition
D009748	Nutrition Disorders

ID	Term
C000601956	burosumab

ParticipantsBG002134

Units	Counts
Participants	OG00066 OG00168

Units	Counts
Participants	OG00066 OG00168

ID	Title	Description
BG000	Placebo	Placebo SC Q4W through Week 24
BG001	Burosumab	Burosumab 1 mg/kg SC Q4W
BG002	Total	Total of all reporting groups