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The primary objectives of the study are to:
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Burosumab Q2W | Experimental | Burosumab subcutaneous (SC) injections every 2 weeks (Q2W) for a total of 160 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Burosumab | Biological | solution for subcutaneous injection |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline at Week 40 in Serum Phosphorus | The Generalized Estimation Equation (GEE) model includes the change from baseline as the dependent variable, time as the categorical variable and adjusted for baseline measurement, with exchangeable covariance structure. | Baseline, Week 40 |
| Number of Participants With Adverse Events (AEs), Treatment Emergent AEs (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | An AE was defined as any untoward medical occurrence associated with the use of a drug, whether or not considered drug related. A serious AE was defined as an AE that at any dose, in the view of either the Investigator or Sponsor, results in any of the following outcomes: death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant incapacity or disability, a congenital anomaly/birth defect, or other important medical events (according to the investigator). An AE was considered a TEAE if it occurred on or after the first dose and was not present prior to the first dose, or it was present prior to the first dose but increased in severity during the study. Events were graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 4.0: grade 1 (mild), grade 2 (moderate), grade 3 (severe), grade 4 (life-threatening), grade 5 (death). | From first dose of study drug through the end of the study (Week 160). Maximum duration of exposure to study drug was 160 weeks. |
| Measure | Description | Time Frame |
|---|---|---|
| Radiographic Global Impression of Change (RGI-C) Score at Week 40 | Changes in the severity of rickets and bowing were assessed centrally by three independent pediatric radiologists contracted by a central imaging facility using a disease specific qualitative RGI-C scoring system. The RGI-C is a seven point ordinal scale with possible values: +3 = very much better (complete or near complete healing of rickets), +2 = much better (substantial healing of rickets), +1 = minimally better (i.e., minimal healing of rickets), 0 = unchanged, -1 = minimally worse (minimal worsening of rickets), -2 = much worse (moderate worsening of rickets), -3 = very much worse (severe worsening of rickets). The Analysis of Covariance (ANCOVA) model includes the RGI-C score as the dependent variable, age and RSS at baseline as covariates. |
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Inclusion Criteria:
Male or female, aged ≥1 year and <5 years
Diagnosis of XLH supported by ONE or more of the following
Biochemical findings associated with XLH including:
Radiographic evidence of rickets
Willing to provide access to prior medical records for the collection of historical growth, biochemical, and radiographic data and disease history
Provide written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures
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
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director | Ultragenyx Pharmaceutical Inc | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Yale University School of Medicine | New Haven | Connecticut | 06510 | United States | ||
| Indiana University School of Medicine |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 39151033 | Derived | Portale AA, Ward L, Dahir K, Florenzano P, Ing SW, Jan de Beur SM, Martin RM, Meza-Martinez AI, Paloian N, Ashraf A, Dixon BP, Khan A, Langman C, Chen A, Wang C, Roberts MS, Tandon PK, Bedrosian C, Imel EA. Nephrocalcinosis and kidney function in children and adults with X-linked hypophosphatemia: baseline results from a large longitudinal study. J Bone Miner Res. 2024 Sep 26;39(10):1493-1502. doi: 10.1093/jbmr/zjae127. | |
| 32721016 |
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The study enrolled pediatric participants between 1 and 4 years old, inclusive, with clinical findings consistent with XLH including hypophosphatemia and radiographic evidence of rickets, and a confirmed PHEX mutation or variant of uncertain significance.
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| ID | Title | Description |
|---|---|---|
| FG000 | Burosumab Q2W | Burosumab subcutaneous (SC) injections every 2 weeks (Q2W) for a total of 160 weeks. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jun 12, 2017 | Feb 19, 2020 |
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| Week 40 |
| RGI-C Score at Week 64 | Changes in the severity of rickets and bowing were assessed centrally by three independent pediatric radiologists contracted by a central imaging facility using a disease specific qualitative RGI-C scoring system. The RGI-C is a seven point ordinal scale with possible values: +3 = very much better (complete or near complete healing of rickets), +2 = much better (substantial healing of rickets), +1 = minimally better (i.e., minimal healing of rickets), 0 = unchanged, -1 = minimally worse (minimal worsening of rickets), -2 = much worse (moderate worsening of rickets), -3 = very much worse (severe worsening of rickets). The GEE model includes the RGI-C score as the dependent variable, visit as a factor, age and RSS at baseline as covariates, with exchangeable covariance structure. | Week 64 |
| Change From Baseline in Rickets at Week 40 as Assessed by the RSS Total Score | The RSS system is a 10-point radiographic scoring method that was developed to assess the severity of nutritional rickets in the wrists and knees based on the degree of metaphyseal fraying, cupping, and the proportion of the growth plate affected. Scores are assigned for the unilateral wrist and knee X-rays deemed by the rater to be the more severe of the bilateral images. The maximum total score on the RSS is 10 points and the minimum score is 0, with a total possible score of 4 points for the wrists and 6 points for the knees. Higher scores indicate greater rickets severity.The ANCOVA model includes the RGI-C score as the dependent variable, age and RSS at baseline as covariates. | Baseline, Week 40 |
| Change From Baseline in Rickets at Week 64 as Assessed by the RSS Total Score | The RSS system is a 10-point radiographic scoring method that was developed to assess the severity of nutritional rickets in the wrists and knees based on the degree of metaphyseal fraying, cupping, and the proportion of the growth plate affected. Scores are assigned for the unilateral wrist and knee X-rays deemed by the rater to be the more severe of the bilateral images. The maximum total score on the RSS is 10 points, with a total possible score of 4 points for the wrists and 6 points for the knees. Higher scores indicate greater rickets severity. The GEE model includes the change from baseline in RSS as the dependent variable, visit as a factor, age and RSS at baseline as covariates, with exchangeable covariance structure. | Baseline, Week 64 |
| RGI-C Lower Limb Deformity Score at Week 40 | Changes in the severity of lower extremity skeletal abnormalities were assessed centrally by three independent pediatric radiologists contracted by a central imaging facility using a disease specific qualitative RGI-C scoring system. The RGI-C is a seven point ordinal scale with possible values: +3 = very much better (complete or near complete healing of rickets), +2 = much better (substantial healing of rickets), +1 = minimally better (i.e., minimal healing of rickets), 0 = unchanged, -1 = minimally worse (minimal worsening of rickets), -2 = much worse (moderate worsening of rickets), -3 = very much worse (severe worsening of rickets). The ANCOVA model includes the RGI-C score as the dependent variable, age and RSS at baseline as covariates. | Week 40 |
| RGI-C Lower Limb Deformity Score at Week 64 | Changes in the severity of lower extremity skeletal abnormalities were assessed centrally by three independent pediatric radiologists contracted by a central imaging facility using a disease specific qualitative RGI-C scoring system. The RGI-C is a seven point ordinal scale with possible values: +3 = very much better (complete or near complete healing of rickets), +2 = much better (substantial healing of rickets), +1 = minimally better (i.e., minimal healing of rickets), 0 = unchanged, -1 = minimally worse (minimal worsening of rickets), -2 = much worse (moderate worsening of rickets), -3 = very much worse (severe worsening of rickets). The GEE model includes the RGI-C score as the dependent variable, visit as a factor, age and RSS at baseline as covariates, with exchangeable covariance structure. | Week 64 |
| Change From Baseline Over Time in Recumbent Length/Standing Height | Baseline, Weeks 12, 24, 40, 64, 88, 112, 136, 160 |
| Change From Baseline Over Time in Recumbent Length/Standing Height as Assessed by Height-for-Age Z-Scores | 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. | Baseline, Weeks 12, 24, 40, 64, 88, 112, 136, 160 |
| Change From Baseline Over Time in Recumbent Length/Standing Height as Assessed by Percentiles | Baseline, Weeks 12, 24, 40, 64, 88, 112, 136, 160 |
| Change From Baseline Over Time in Serum Alkaline Phosphatase (ALP) | The GEE model includes the change from baseline as the dependent variable, time as the categorical variable and adjusted for baseline measurement, with exchangeable covariance structure. | Baseline, Weeks 4, 12, 20, 40, 48, 56, 64, 76, 88, 100, 112, 124, 136, 148, 160 |
| Percent Change From Baseline Over Time in Serum ALP | Baseline, Weeks 4, 12, 20, 40, 48, 56, 64, 76, 88, 100, 112, 124, 136, 148, 160 |
| Indianapolis |
| Indiana |
| 46202 |
| United States |
| Shriners Hospital for Children | St Louis | Missouri | 63110 | United States |
| Derived |
| 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. |
| 30638856 | Derived | Whyte MP, Carpenter TO, Gottesman GS, Mao M, Skrinar A, San Martin J, Imel EA. Efficacy and safety of burosumab in children aged 1-4 years with X-linked hypophosphataemia: a multicentre, open-label, phase 2 trial. Lancet Diabetes Endocrinol. 2019 Mar;7(3):189-199. doi: 10.1016/S2213-8587(18)30338-3. Epub 2019 Jan 9. |
| Completed Week 40 |
|
| Completed Week 64 |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Burosumab Q2W | Burosumab SC injections Q2W for a total of 160 weeks. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| ||||||||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| |||||||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| |||||||||||||||||||||||
| Race/Ethnicity, Customized | Count of Participants | Participants |
| |||||||||||||||||||||||
| Serum Phosphorus | Mean | Standard Deviation | mg/dL |
| ||||||||||||||||||||||
| Rickets Severity Score (RSS) Total Score | 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. | Mean | Standard Deviation | units on a scale |
| |||||||||||||||||||||
| Recumbent length/Standing height | Growth is measured by recumbent length for subjects < 2 years of age or those unable or unwilling to stand for the measurement. | Mean | Standard Deviation | cm |
| |||||||||||||||||||||
| Recumbent length/Standing height (Z score) | 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. | Mean | Standard Deviation | Z score |
| |||||||||||||||||||||
| Recumbent length/Standing height (percentile) | Mean | Standard Deviation | percentile |
| ||||||||||||||||||||||
| Serum Alkaline Phosphatase (ALP) | Mean | Standard Deviation | U/L |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change From Baseline at Week 40 in Serum Phosphorus | The Generalized Estimation Equation (GEE) model includes the change from baseline as the dependent variable, time as the categorical variable and adjusted for baseline measurement, with exchangeable covariance structure. | Pharmacokinetic/Pharmacodynamic (PK/PD) Analysis Set: all participants who received at least one dose of study drug and had evaluable blood samples. | Posted | Least Squares Mean | Standard Error | mg/dL | Baseline, Week 40 |
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| Primary | Number of Participants With Adverse Events (AEs), Treatment Emergent AEs (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation | An AE was defined as any untoward medical occurrence associated with the use of a drug, whether or not considered drug related. A serious AE was defined as an AE that at any dose, in the view of either the Investigator or Sponsor, results in any of the following outcomes: death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant incapacity or disability, a congenital anomaly/birth defect, or other important medical events (according to the investigator). An AE was considered a TEAE if it occurred on or after the first dose and was not present prior to the first dose, or it was present prior to the first dose but increased in severity during the study. Events were graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 4.0: grade 1 (mild), grade 2 (moderate), grade 3 (severe), grade 4 (life-threatening), grade 5 (death). | Safety Analysis Set: all participants who received at least one dose of study drug. | Posted | Count of Participants | Participants | From first dose of study drug through the end of the study (Week 160). Maximum duration of exposure to study drug was 160 weeks. |
|
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| Secondary | Radiographic Global Impression of Change (RGI-C) Score at Week 40 | Changes in the severity of rickets and bowing were assessed centrally by three independent pediatric radiologists contracted by a central imaging facility using a disease specific qualitative RGI-C scoring system. The RGI-C is a seven point ordinal scale with possible values: +3 = very much better (complete or near complete healing of rickets), +2 = much better (substantial healing of rickets), +1 = minimally better (i.e., minimal healing of rickets), 0 = unchanged, -1 = minimally worse (minimal worsening of rickets), -2 = much worse (moderate worsening of rickets), -3 = very much worse (severe worsening of rickets). The Analysis of Covariance (ANCOVA) model includes the RGI-C score as the dependent variable, age and RSS at baseline as covariates. | Efficacy Analysis Set: all participants who received at least one dose of study drug and had at least one post-study drug measurement. | Posted | Least Squares Mean | Standard Error | units on a scale | Week 40 |
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| Secondary | RGI-C Score at Week 64 | Changes in the severity of rickets and bowing were assessed centrally by three independent pediatric radiologists contracted by a central imaging facility using a disease specific qualitative RGI-C scoring system. The RGI-C is a seven point ordinal scale with possible values: +3 = very much better (complete or near complete healing of rickets), +2 = much better (substantial healing of rickets), +1 = minimally better (i.e., minimal healing of rickets), 0 = unchanged, -1 = minimally worse (minimal worsening of rickets), -2 = much worse (moderate worsening of rickets), -3 = very much worse (severe worsening of rickets). The GEE model includes the RGI-C score as the dependent variable, visit as a factor, age and RSS at baseline as covariates, with exchangeable covariance structure. | Efficacy Analysis Set: all participants who received at least one dose of study drug and had at least one post-study drug measurement. | Posted | Least Squares Mean | Standard Error | units on a scale | Week 64 |
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| Secondary | Change From Baseline in Rickets at Week 40 as Assessed by the RSS Total Score | The RSS system is a 10-point radiographic scoring method that was developed to assess the severity of nutritional rickets in the wrists and knees based on the degree of metaphyseal fraying, cupping, and the proportion of the growth plate affected. Scores are assigned for the unilateral wrist and knee X-rays deemed by the rater to be the more severe of the bilateral images. The maximum total score on the RSS is 10 points and the minimum score is 0, with a total possible score of 4 points for the wrists and 6 points for the knees. Higher scores indicate greater rickets severity.The ANCOVA model includes the RGI-C score as the dependent variable, age and RSS at baseline as covariates. | Efficacy Analysis Set: all participants who received at least one dose of study drug and had at least one post-study drug measurement. | Posted | Least Squares Mean | Standard Error | units on a scale | Baseline, Week 40 |
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| Secondary | Change From Baseline in Rickets at Week 64 as Assessed by the RSS Total Score | The RSS system is a 10-point radiographic scoring method that was developed to assess the severity of nutritional rickets in the wrists and knees based on the degree of metaphyseal fraying, cupping, and the proportion of the growth plate affected. Scores are assigned for the unilateral wrist and knee X-rays deemed by the rater to be the more severe of the bilateral images. The maximum total score on the RSS is 10 points, with a total possible score of 4 points for the wrists and 6 points for the knees. Higher scores indicate greater rickets severity. The GEE model includes the change from baseline in RSS as the dependent variable, visit as a factor, age and RSS at baseline as covariates, with exchangeable covariance structure. | Efficacy Analysis Set: all participants who received at least one dose of study drug and had at least one post-study drug measurement. | Posted | Least Squares Mean | Standard Error | units on a scale | Baseline, Week 64 |
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| Secondary | RGI-C Lower Limb Deformity Score at Week 40 | Changes in the severity of lower extremity skeletal abnormalities were assessed centrally by three independent pediatric radiologists contracted by a central imaging facility using a disease specific qualitative RGI-C scoring system. The RGI-C is a seven point ordinal scale with possible values: +3 = very much better (complete or near complete healing of rickets), +2 = much better (substantial healing of rickets), +1 = minimally better (i.e., minimal healing of rickets), 0 = unchanged, -1 = minimally worse (minimal worsening of rickets), -2 = much worse (moderate worsening of rickets), -3 = very much worse (severe worsening of rickets). The ANCOVA model includes the RGI-C score as the dependent variable, age and RSS at baseline as covariates. | Efficacy Analysis Set: all participants who received at least one dose of study drug and had at least one post-study drug measurement. | Posted | Least Squares Mean | Standard Error | units on a scale | Week 40 |
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| Secondary | RGI-C Lower Limb Deformity Score at Week 64 | Changes in the severity of lower extremity skeletal abnormalities were assessed centrally by three independent pediatric radiologists contracted by a central imaging facility using a disease specific qualitative RGI-C scoring system. The RGI-C is a seven point ordinal scale with possible values: +3 = very much better (complete or near complete healing of rickets), +2 = much better (substantial healing of rickets), +1 = minimally better (i.e., minimal healing of rickets), 0 = unchanged, -1 = minimally worse (minimal worsening of rickets), -2 = much worse (moderate worsening of rickets), -3 = very much worse (severe worsening of rickets). The GEE model includes the RGI-C score as the dependent variable, visit as a factor, age and RSS at baseline as covariates, with exchangeable covariance structure. | Efficacy Analysis Set: all participants who received at least one dose of study drug and had at least one post-study drug measurement. | Posted | Least Squares Mean | Standard Error | units on a scale | Week 64 |
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| Secondary | Change From Baseline Over Time in Recumbent Length/Standing Height | Efficacy Analysis Set: all participants who received at least one dose of study drug and had at least one post-study drug measurement at given time point. | Posted | Mean | Standard Deviation | cm | Baseline, Weeks 12, 24, 40, 64, 88, 112, 136, 160 |
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| Secondary | Change From Baseline Over Time in Recumbent Length/Standing Height as Assessed by Height-for-Age Z-Scores | 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. | Efficacy Analysis Set: all participants who received at least one dose of study drug and had at least one post-study drug measurement at given time point. | Posted | Mean | Standard Deviation | Z score | Baseline, Weeks 12, 24, 40, 64, 88, 112, 136, 160 |
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| Secondary | Change From Baseline Over Time in Recumbent Length/Standing Height as Assessed by Percentiles | Efficacy Analysis Set: all participants who received at least one dose of study drug and had at least one post-study drug measurement at given time point. | Posted | Mean | Standard Deviation | percentiles | Baseline, Weeks 12, 24, 40, 64, 88, 112, 136, 160 |
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| Secondary | Change From Baseline Over Time in Serum Alkaline Phosphatase (ALP) | The GEE model includes the change from baseline as the dependent variable, time as the categorical variable and adjusted for baseline measurement, with exchangeable covariance structure. | PK/PD Analysis Set: all participants who received at least one dose of study drug and had evaluable blood samples at given time point. | Posted | Least Squares Mean | Standard Error | U/L | Baseline, Weeks 4, 12, 20, 40, 48, 56, 64, 76, 88, 100, 112, 124, 136, 148, 160 |
|
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| Secondary | Percent Change From Baseline Over Time in Serum ALP | PK/PD Analysis Set: all participants who received at least one dose of study drug and had evaluable blood samples at given time point. | Posted | Mean | Standard Deviation | percent change | Baseline, Weeks 4, 12, 20, 40, 48, 56, 64, 76, 88, 100, 112, 124, 136, 148, 160 |
|
|
From first dose of study drug through the end of the study (at Week 160). Maximum duration of exposure to study drug was 160 weeks.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Burosumab Q2W | Burosumab SC injections Q2W for a total of 160 weeks. | 0 | 13 | 1 | 13 | 13 | 13 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Tooth Abscess | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Skull Malformation | Congenital, familial and genetic disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Syringomyelia | Congenital, familial and genetic disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Deafness Unilateral | Ear and labyrinth disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Ear Haemorrhage | Ear and labyrinth disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Ear Pain | Ear and labyrinth disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Excessive Cerumen Production | Ear and labyrinth disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Hypoacusis | Ear and labyrinth disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Motion Sickness | Ear and labyrinth disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Otorrhoea | Ear and labyrinth disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Tinnitus | Ear and labyrinth disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Ocular Hyperaemia | Eye disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Abdominal Discomfort | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Abdominal Pain Upper | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Aphthous Ulcer | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Dental Caries | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Epigastric Discomfort | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Gingival Blister | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Gingival Erythema | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Gingival Pain | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Haematochezia | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Loose Tooth | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Oral Pain | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Post-Tussive Vomiting | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Stomatitis | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Teething | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Toothache | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Vomiting Projectile | Gastrointestinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Injection Site Bruising | General disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Injection Site Erythema | General disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Injection Site Pain | General disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Injection Site Pruritus | General disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Injection Site Reaction | General disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Non-Cardiac Chest Pain | General disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Pain | General disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Vaccination Site Reaction | General disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Food Allergy | Immune system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Hypersensitivity | Immune system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Atypical Pneumonia | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Croup Infectious | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Ear Infection | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Enterobiasis | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Eye Infection | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Gastrointestinal Viral Infection | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Genital Candidiasis | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Gingival Abscess | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Hand-Foot-And-Mouth Disease | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Hordeolum | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Impetigo | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Molluscum Contagiosum | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Otitis Media | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Pharyngitis Streptococcal | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Tooth Abscess | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Upper Respiratory Tract Infection | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Urinary Tract Infection | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Viral Infection | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Viral Upper Respiratory Tract Infection | Infections and infestations | MedDRA 18.1 | Systematic Assessment |
| |
| Arthropod Bite | Injury, poisoning and procedural complications | MedDRA 18.1 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 18.1 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 18.1 | Systematic Assessment |
| |
| Laceration | Injury, poisoning and procedural complications | MedDRA 18.1 | Systematic Assessment |
| |
| Lip Injury | Injury, poisoning and procedural complications | MedDRA 18.1 | Systematic Assessment |
| |
| Skin Abrasion | Injury, poisoning and procedural complications | MedDRA 18.1 | Systematic Assessment |
| |
| Tooth Fracture | Injury, poisoning and procedural complications | MedDRA 18.1 | Systematic Assessment |
| |
| Tooth Injury | Injury, poisoning and procedural complications | MedDRA 18.1 | Systematic Assessment |
| |
| Traumatic Tooth Displacement | Injury, poisoning and procedural complications | MedDRA 18.1 | Systematic Assessment |
| |
| Amylase Increased | Investigations | MedDRA 18.1 | Systematic Assessment |
| |
| Blood 25-Hydroxycholecalciferol Decreased | Investigations | MedDRA 18.1 | Systematic Assessment |
| |
| Blood Parathyroid Hormone Increased | Investigations | MedDRA 18.1 | Systematic Assessment |
| |
| Blood Phosphorus Decreased | Investigations | MedDRA 18.1 | Systematic Assessment |
| |
| Heart Rate Abnormal | Investigations | MedDRA 18.1 | Systematic Assessment |
| |
| Lipase Increased | Investigations | MedDRA 18.1 | Systematic Assessment |
| |
| Vitamin D Decreased | Investigations | MedDRA 18.1 | Systematic Assessment |
| |
| White Blood Cell Count Increased | Investigations | MedDRA 18.1 | Systematic Assessment |
| |
| Decreased Appetite | Metabolism and nutrition disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Increased Appetite | Metabolism and nutrition disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Polydipsia | Metabolism and nutrition disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Back Pain | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Bone Pain | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Foot Deformity | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Knee Deformity | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Pain In Extremity | Musculoskeletal and connective tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Dysarthria | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Hypersomnia | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Lethargy | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Periodic Limb Movement Disorder | Nervous system disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Dysuria | Renal and urinary disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Polyuria | Renal and urinary disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Urinary Incontinence | Renal and urinary disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Penile Erythema | Reproductive system and breast disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Penile Pain | Reproductive system and breast disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Adenoidal Disorder | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Chronic Throat Clearing | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Nasal Congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Nasal Discharge Discolouration | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Oropharyngeal Pain | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Productive Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Respiratory Tract Congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Sinus Congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Sneezing | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Throat Irritation | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Wheezing | Respiratory, thoracic and mediastinal disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Dermatitis Contact | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Rash Papular | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Skin Disorder | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Swelling Face | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Urticaria | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Urticaria Papular | Skin and subcutaneous tissue disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Flushing | Vascular disorders | MedDRA 18.1 | Systematic Assessment |
| |
| Haematoma | Vascular disorders | MedDRA 18.1 | Systematic Assessment |
|
The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot extend the embargo.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Information | Ultragenyx Pharmaceutical Inc | 1-888-756-8657 | medinfo@ultragenyx.com |
| Prot_002.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Nov 29, 2016 | Apr 19, 2018 | SAP_001.pdf |
| ID | Term |
|---|---|
| D053098 | Familial Hypophosphatemic Rickets |
| 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 |
Not provided
Not provided
| ID | Term |
|---|---|
| C000601956 | burosumab |
Not provided
Not provided
Not provided
| Unknown or Not Reported |
|
| Counts |
|---|
| Participants |
|
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| Title | Denominators | Categories |
|---|
| Week 12 |
|
| ||||
| Week 24 |
|
| ||||
| Week 40 |
|
| ||||
| Week 64 |
|
| ||||
| Week 88 |
|
| ||||
| Week 112 |
|
| ||||
| Week 136 |
|
| ||||
| Week 160 |
|
|
|
| Title | Denominators | Categories |
|---|
| Week 12 |
|
| ||||
| Week 24 |
|
| ||||
| Week 40 |
|
| ||||
| Week 64 |
|
| ||||
| Week 88 |
|
| ||||
| Week 112 |
|
| ||||
| Week 136 |
|
| ||||
| Week 160 |
|
|
|
|
| Title | Denominators | Categories |
|---|
| Week 4 |
|
| ||||
| Week 12 |
|
| ||||
| Week 20 |
|
| ||||
| Week 40 |
|
| ||||
| Week 48 |
|
| ||||
| Week 56 |
|
| ||||
| Week 64 |
|
| ||||
| Week 76 |
|
| ||||
| Week 88 |
|
| ||||
| Week 100 |
|
| ||||
| Week 112 |
|
| ||||
| Week 124 |
|
| ||||
| Week 136 |
|
| ||||
| Week 148 |
|
| ||||
| Week 160 |
|
|