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| ID | Type | Description | Link |
|---|---|---|---|
| 2016-003083-39 | EudraCT Number |
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To evaluate the effect of denosumab on lumbar spine bone mineral density (BMD) Z-score as assessed by dual-energy X-ray absorptiometry (DXA) at 12 months in children 5 to 17 year of age with Glucocorticoid (GC)-induced osteoporosis (GiOP).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo | Other | SC Q6M placebo |
|
| Denosumab | Experimental | 1 mg/kg BW (up to a maximum of 60 mg) SC Q6M |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Denosumab | Drug | 1mg/kg BW (up to a maximum of 60 mg) SC Q6M |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Lumbar Spine BMD Z-score as Assessed by Dual-energy X-ray Absorptiometry (DXA) at 12 Months | Lumbar spine BMD was assessed by DXA and analyzed by analysis of covariance (ANCOVA) including treatment (denosumab vs placebo), baseline age, and baseline BMD z-score. DXA results were converted to z-scores, indicating number of standard deviations from the reference population's mean, with 0 denoting the mean. Positive changes from baseline signify lumbar spine BMD improvement. | Baseline and 12 Months |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Lumbar Spine BMD Z-score as Assessed by DXA at 6, 18, 24, and 36 Months | Lumbar spine BMD was assessed by DXA and analyzed by repeated measures analysis with randomization group, visit, baseline age, and baseline BMD z-score as fixed effects. Treatment-by-visit was included as an interaction term. DXA results were converted to z-scores, indicating number of standard deviations from the reference population's mean, with 0 denoting the mean. Positive changes from baseline signify lumbar spine BMD improvement. |
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Inclusion Criteria:
Male or female subjects, age 5 to 17 years, inclusive, at the time of informed consent.
Clinical diagnosis of GiOP as defined by the following (and consistent with the International Society for Clinical Densitometry definition of osteoporosis in children and adolescents [Bishop et al, 2014])
A confirmed diagnosis of non-malignant condition(s) requiring treatment with systemic GC (including, but not limited to, chronic rheumatologic, gastrointestinal, neurologic, respiratory, and/or nephrological conditions)
Subjects who are on systemic GC only as replacement therapy for adrenal insufficiency are not eligible for the study - Treatment with systemic GC (intravenous or oral) of any duration for the underlying non-malignant condition(s) within the 12 months prior to screening
Evidence of at least 1 vertebral compression fracture of Genant grade 1 or higher, as assessed by the central imaging vendor on lateral spine X-rays performed at screening or within 2 months prior to screening; OR, in the absence of vertebral compression fractures, presence of both clinically significant fracture history (ie, ≥ 2 long-bone fractures by age 10 years or ≥ 3 long-bone fractures at any age up to 17 years) and lumbar spine BMD Z-score ≤ -2.0, as assessed by the central imaging vendor.
• Subject's legally acceptable representative has provided informed consent when the subject is legally too young to provide informed consent and the subject has provided assent based on local regulations and/or guidelines prior to any study-specific activities/procedures being initiated
A confirmed diagnosis of non-malignant condition(s) requiring treatment with systemic GC (including, but not limited to, chronic rheumatologic, gastrointestinal, neurologic, respiratory, and/or nephrological conditions)
Subjects who are on systemic GC only as replacement therapy for adrenal insufficiency are not eligible for the study
Treatment with systemic GC (intravenous or oral) of any duration for the underlying non malignant condition(s) within the 12 months prior to screening
Prepubertal children should be expected to require significant GC use during the study, per investigator opinion
Exclusion criteria will include the following:
Current hyperthyroidism (unless well controlled on stable antithyroid therapy)
Current clinical hypothyroidism (unless well controlled on stable thyroid replacement therapy)
History of hyperparathyroidism
Current hypoparathyroidism
Duchenne muscular dystrophy with symptomatic cardiac abnormality
Current malabsorption
Active infection or history of infections
History of malignancy
Any active infection for which systemic anti-infectives were used within 4 weeks prior to screening
Serious infection, defined as requiring hospitalization or intravenous anti infectives within 8 weeks prior to screening
Recurrent or chronic infection or other active infection that, in the opinion of the investigator, might compromise the safety of the subject
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| Name | Affiliation | Role |
|---|---|---|
| MD | Amgen | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Childrens Hospital of Los Angeles | Los Angeles | California | 90027 | United States | ||
| AI Dupont Hospital for Children |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 42319705 | Derived | Storm NE, Kubik C, Priem G, Kim M, Lin TC, Lange J, O'Connell C, Glennane A, Bradbury BD, Taisey MJ. Case Study on the Use of Real-World Evidence in a Feasibility Assessment of a Randomized Controlled Trial Design to Support the Fulfillment of Pediatric Requirements with the FDA and EMA. Ther Innov Regul Sci. 2026 Jun 19. doi: 10.1007/s43441-026-00977-1. Online ahead of print. |
| Label | URL |
|---|---|
| AmgenTrials clinical trials website | View source |
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De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request
Data sharing requests relating to this study will be considered beginning 18 months after the study has ended and either 1) the product and indication have been granted marketing authorization in both the US and Europe or 2) clinical development for the product and/or indication discontinues and the data will not be submitted to regulatory authorities. There is no end date for eligibility to submit a data sharing request for this study.
Qualified researchers may submit a request containing the research objectives, the Amgen product(s) and Amgen study/studies in scope, endpoints/outcomes of interest, statistical analysis plan, data requirements, publication plan, and qualifications of the researcher(s). In general, Amgen does not grant external requests for individual patient data for the purpose of re-evaluating safety and efficacy issues already addressed in the product labelling. Requests are reviewed by a committee of internal advisors. If not approved, a Data Sharing Independent Review Panel will arbitrate and make the final decision. Upon approval, information necessary to address the research question will be provided under the terms of a data sharing agreement. This may include anonymized individual patient data and/or available supporting documents, containing fragments of analysis code where provided in analysis specifications. Further details are available at the link below.
Participants were randomized in a 2:1 allocation ratio to receive either denosumab or placebo respectively, in a double-blind manner during the 12-month placebo-controlled double-blind Treatment Period. This was followed by a 12-month denosumab Open-label Treatment Period and a 12-month Off-treatment Observation Period.
This study was conducted at 12 center(s) in Australia, Canada, Columbia, India, Peru, Russia, Turkey, Ukraine, and the United States between May 2018 and December 2023.
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| ID | Title | Description |
|---|---|---|
| FG000 | Denosumab/Denosumab | Participants received 1 mg/kg Denosumab by SC injection up to a maximum of 60 mg, Q6M for 24 months during the Treatment Period. Participants were then followed for an additional 12 months during the Off-treatment Observation Period. |
| FG001 | Placebo/Denosumab |
| 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 | Jul 10, 2023 | May 29, 2024 |
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Double-blind
| Placebo |
| Other |
SC Q6M placebo |
|
| Baseline and 6, 18, 24, and 36 Months |
| Change From Baseline in Proximal Femur BMD Z-score as Assessed by DXA at 6, 12, 18, 24, and 36 Months | Proximal femur (total hip and femoral neck) BMD was assessed by DXA and analyzed by repeated measures analysis with randomization group, visit, baseline age, and baseline BMD z-score as fixed effects. Treatment-by-visit was included as an interaction term. DXA results were converted to z-scores, indicating number of standard deviations from the reference population's mean, with 0 denoting the mean. Positive changes from baseline signify proximal femur BMD improvement. | Baseline and 6, 12, 18, 24, and 36 Months |
| Number of Participants With X-ray Confirmed Long-bone Fractures and/or Vertebral Fractures at 12, 24, and 36 Months | Number of participants who have at least one long bone fracture or vertebral fracture, and number of participants who have more than one long bone fracture or vertebral fracture. | Month 12, 24, and 36 |
| Number of Participants With Improving Vertebral Fractures at 12, 24, and 36 Months | Number of participants with improving vertebral fractures. An improving fracture is defined as one showing signs of healing/repair from baseline as assessed by X-ray. | Month 12, 24, and 36 |
| Number of Participants With New and Worsening Vertebral and Non-vertebral Fractures at 12, 24, and 36 Months | Number of participants who have at least one vertebral fracture or non-vertebral fracture, and number of participants who have more than one vertebral fracture or non-vertebral fracture. | Month 12, 24, and 36 |
| Change From Baseline in Child Health Questionnaire-Parent Form-50 (CHQ-PF-50) Physical Summary Score at 12, 24, and 36 Months | The CHQ-PF-50 is a 50-item questionnaire to be completed by the parents or guardians of children between 5 and 18 years of age. The physical summary score ranges from 0-100 with higher scores indicating better physical health. | Baseline and month 12, 24, and 36 |
| Change From Baseline in CHQ-PF-50 Psychological Summary Score at 12, 24, and 36 Months | The CHQ-PF-50 is a 50-item questionnaire to be completed by the parents or guardians of children between 5 and 18 years of age. The psychological summary score ranges from 0-100 with higher scores indicating better psychological health. | Baseline and Month 12, 24, and 36 |
| Change From Baseline in Childhood Health Assessment Questionnaire (CHAQ) Disability Index Score at 12, 24, and 36 Months | The CHAQ was developed to measure the physical functioning in children 6 months to 18 years of age. It consists of 54 questions related to the child's ability to perform various activities of daily living. Depending on the question asked, each question is scored either 0 to 3 based on the level of difficulty experienced by the child or 0-1 based on whether the child required assistance from another person or used an aid or other device. All CHAQ questions were scored and converted to a total index score ranging from 0-3, where higher scores indicate greater disability. | Baseline and Month 12, 24, and 36 |
| Change From Baseline in Wong-Baker FACES Pain Rating Scale (WBFPRS) at 12, 24, and 36 Months | The WBFPRS is a horizontal pain scale for children 3-18 years which consists of 6 faces that range from a smiling "no hurt" face with a score of 0 to a crying "hurts worst" face with a score of 10. | Baseline and Month 12, 24, and 36 |
| Change From Baseline in Growth Velocity Z-score (Height) at 12, 24, and 36 Months | Growth velocity was determined by calculating age-adjusted z-scores for height, weight, and body mass index (BMI). Z-scores represent the number of standard deviations from the reference population's mean, with 0 denoting the mean. Positive changes from baseline signify increased growth velocity. | Baseline and Month 12, 24, and 36 |
| Change From Baseline in Growth Velocity Z-score (Weight) at 12, 24, and 36 Months | Growth velocity was determined by calculating age-adjusted z-scores for height, weight, and BMI. Z-scores represent the number of standard deviations from the reference population's mean, with 0 denoting the mean. Positive changes from baseline signify increased growth velocity. | Baseline and Month 12, 24, and 36 |
| Change From Baseline in Growth Velocity Z-score (BMI) at 12, 24, and 36 Months | Growth velocity was determined by calculating age-adjusted z-scores for height, weight, and BMI. Z-scores represent the number of standard deviations from the reference population's mean, with 0 denoting the mean. Positive changes from baseline signify increased growth velocity. | Baseline and Month 12, 24, and 36 |
| Mean Serum Concentration of Denosumab | Day 1, Day 10, Day 30, Month 3, Month 6, Month 12, and Month 18 |
| Wilmington |
| Delaware |
| 19803 |
| United States |
| Indiana University Hospital | Indianapolis | Indiana | 46202 | United States |
| University of Minnesota Masonic Childrens Hospital Discovery Clinic | Minneapolis | Minnesota | 55454 | United States |
| Metrohealth Medical Center | Cleveland | Ohio | 44109 | United States |
| Nationwide Childrens Hospital | Columbus | Ohio | 43205 | United States |
| Perth Childrens Hospital | Nedlands | Western Australia | 6909 | Australia |
| Cliniques Universtaire Saint Luc Universite Catholique de Louvain | Brussels | 1200 | Belgium |
| Medical Centre Synexus Sofia EOOD | Sofia | 1784 | Bulgaria |
| Childrens Hospital of Eastern Ontario | Ottawa | Ontario | K1H 8L1 | Canada |
| Centre Hospitalier Universitaire Sainte Justine | Montreal | Quebec | H3T 1C5 | Canada |
| Center for Clinical and Basic Research Colombia | Medellín | Antioquia | 050021 | Colombia |
| Solano y Terront Servicios Medicos Ltda - Unidad Integral de Endocrinologia Uniendo | Bogota | Cundinamarca | 110221 | Colombia |
| Foscal Internacional-Fundacion Oftalmologica de Santander | Floridablanca | Santander Department | 681004 | Colombia |
| Gandhi Medical College | Hyderabad | Andhra Pradesh | 500 025 | India |
| KLES Dr Prabhakar Kore Hospital and Medical Research Centre | Belagavi | Karnataka | 590010 | India |
| Sir Ganga Ram Hospital | New Delhi | National Capital Territory of Delhi | 110 022 | India |
| Christian Medical College | Vellore | Tamil Nadu | 632 004 | India |
| Azienda Ospedaliera Universitaria Meyer | Florence | 50139 | Italy |
| Istituto Auxologico Italiano Istituto di Ricovero e Cura a Carattere Scientifico | Milan | 20145 | Italy |
| Azienda Ospedaliera Policlinico Umberto I | Roma | 00161 | Italy |
| RM Pharma Specialists SA de CV | Mexico City | 03100 | Mexico |
| Instituto Nacional de Salud del Nino | Breña | Lima region | Lima 5 | Peru |
| Centro Especializado de Enfermedades Neoplasicas | Arequipa | 040001 | Peru |
| Hospital Nacional Alberto Sabogal Sologuren | Callao | Callao 2 | Peru |
| Clinica Angloamericana | Lima | Lima 27 | Peru |
| Centro de Investigacion Ricardo Palma | Lima | Lima27 | Peru |
| FSAI Scientific Center of Childrens Health of MoH of the RF | Moscow | 119991 | Russia |
| SBEI of HPE First Moscow state medical university na I M Sechenov of MoH of Russian Federation | Moscow | 119991 | Russia |
| SBHI of Novosibirsk region City Pediatric Clinical Hospital of Emergency Care | Novosibirsk | 630007 | Russia |
| LLC Medical Technologies | Saint Petersburg | 191025 | Russia |
| Ankara Universitesi Tip Fakultesi | Ankara | 06590 | Turkey (Türkiye) |
| Ataturk Universitesi Tip Fakultesi | Erzurum | 25240 | Turkey (Türkiye) |
| Marmara Universitesi Pendik Egitim Arastirma Hastanesi | Istanbul | 34890 | Turkey (Türkiye) |
| Ege Universitesi Tip Fakultesi | Izmir | 35100 | Turkey (Türkiye) |
| CI Dnipropetrovsk Regional Children Clinical Hospital of Dnipropetrovsk Regional Council | Dnipro | 49100 | Ukraine |
| Communal Institution of Healthcare Kharkiv City Clinical Children Hospital 16 | Kharkiv | 61075 | Ukraine |
| National Childrens Specialized Hospital OHMATDYT | Kyiv | 01025 | Ukraine |
Participants received matching placebo by SC injection Q6M for the first 12 months of the Treatment Period. This was followed by 1 mg/kg Denosumab administered by SC injection, up to a maximum of 60 mg, Q6M for the second 12 months of the Treatment Period. Participants were then followed for an additional 12 months during the Off-treatment Observation Period. |
| COMPLETED |
|
| NOT COMPLETED |
|
|
Full Analysis Set (FAS): all participants enrolled into the study.
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| ID | Title | Description |
|---|---|---|
| BG000 | Denosumab/Denosumab | Participants received 1 mg/kg Denosumab by SC injection up to a maximum of 60 mg, Q6M for 24 months during the Treatment Period. Participants were then followed for an additional 12 months during the Off-treatment Observation Period. |
| BG001 | Placebo/Denosumab | Participants received matching placebo by SC injection Q6M for the first 12 months of the Treatment Period. This was followed by 1 mg/kg Denosumab administered by SC injection, up to a maximum of 60 mg, Q6M for the second 12 months of the Treatment Period. Participants were then followed for an additional 12 months during the Off-treatment Observation Period. |
| BG002 | Total | Total of all reporting groups |
| 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 (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Bone Mineral Density (BMD) Z-score at Baseline | BMD was assessed by DXA and results were converted to z-scores, indicating number of standard deviations from the reference population's mean, with 0 denoting the mean. | Only participants with available data have been included. | Mean | Standard Deviation | Z-score |
|
| 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 in Lumbar Spine BMD Z-score as Assessed by Dual-energy X-ray Absorptiometry (DXA) at 12 Months | Lumbar spine BMD was assessed by DXA and analyzed by analysis of covariance (ANCOVA) including treatment (denosumab vs placebo), baseline age, and baseline BMD z-score. DXA results were converted to z-scores, indicating number of standard deviations from the reference population's mean, with 0 denoting the mean. Positive changes from baseline signify lumbar spine BMD improvement. | Primary DXA Analysis Set: all participants in the FAS with baseline and ≥ 1 postbaseline valid DXA assessment of lumbar spine provided by the central imaging vendor during the first 12 months. | Posted | Least Squares Mean | 95% Confidence Interval | Z-score | Baseline and 12 Months |
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| Secondary | Change From Baseline in Lumbar Spine BMD Z-score as Assessed by DXA at 6, 18, 24, and 36 Months | Lumbar spine BMD was assessed by DXA and analyzed by repeated measures analysis with randomization group, visit, baseline age, and baseline BMD z-score as fixed effects. Treatment-by-visit was included as an interaction term. DXA results were converted to z-scores, indicating number of standard deviations from the reference population's mean, with 0 denoting the mean. Positive changes from baseline signify lumbar spine BMD improvement. | DXA Analysis Set: all participants in the FAS with baseline and ≥ 1 postbaseline valid DXA assessment for lumbar spine as provided by the central imaging vendor. All participants included in the Overall Number of Participants Analyzed contributed data to this outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | Z-score | Baseline and 6, 18, 24, and 36 Months |
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| Secondary | Change From Baseline in Proximal Femur BMD Z-score as Assessed by DXA at 6, 12, 18, 24, and 36 Months | Proximal femur (total hip and femoral neck) BMD was assessed by DXA and analyzed by repeated measures analysis with randomization group, visit, baseline age, and baseline BMD z-score as fixed effects. Treatment-by-visit was included as an interaction term. DXA results were converted to z-scores, indicating number of standard deviations from the reference population's mean, with 0 denoting the mean. Positive changes from baseline signify proximal femur BMD improvement. | DXA Analysis Set: all participants in the FAS with baseline and ≥ 1 postbaseline valid DXA assessment for total hip and femoral neck as provided by the central imaging vendor. | Posted | Least Squares Mean | 95% Confidence Interval | Z-score | Baseline and 6, 12, 18, 24, and 36 Months |
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| Secondary | Number of Participants With X-ray Confirmed Long-bone Fractures and/or Vertebral Fractures at 12, 24, and 36 Months | Number of participants who have at least one long bone fracture or vertebral fracture, and number of participants who have more than one long bone fracture or vertebral fracture. | FAS: all participants randomized into the study. | Posted | Count of Participants | Participants | Month 12, 24, and 36 |
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| Secondary | Number of Participants With Improving Vertebral Fractures at 12, 24, and 36 Months | Number of participants with improving vertebral fractures. An improving fracture is defined as one showing signs of healing/repair from baseline as assessed by X-ray. | Vertebral Fracture Analysis Set: all participants in the FAS who have a non-missing baseline and ≥ 1 non-missing postbaseline X-ray vertebral evaluation as provided by the central imaging vendor, on or before the time point under consideration. | Posted | Count of Participants | Participants | Month 12, 24, and 36 |
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| Secondary | Number of Participants With New and Worsening Vertebral and Non-vertebral Fractures at 12, 24, and 36 Months | Number of participants who have at least one vertebral fracture or non-vertebral fracture, and number of participants who have more than one vertebral fracture or non-vertebral fracture. | FAS: all participants randomized into the study. | Posted | Count of Participants | Participants | Month 12, 24, and 36 |
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| Secondary | Change From Baseline in Child Health Questionnaire-Parent Form-50 (CHQ-PF-50) Physical Summary Score at 12, 24, and 36 Months | The CHQ-PF-50 is a 50-item questionnaire to be completed by the parents or guardians of children between 5 and 18 years of age. The physical summary score ranges from 0-100 with higher scores indicating better physical health. | Patient Reported Outcomes (PRO) Analysis Set: all participants in the FAS with baseline and at least one valid CHQ-PF-50 response at post-baseline. All participants included in the Overall Number of Participants Analyzed contributed data to this outcome measure. | Posted | Mean | Standard Deviation | Scores on a scale | Baseline and month 12, 24, and 36 |
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| Secondary | Change From Baseline in CHQ-PF-50 Psychological Summary Score at 12, 24, and 36 Months | The CHQ-PF-50 is a 50-item questionnaire to be completed by the parents or guardians of children between 5 and 18 years of age. The psychological summary score ranges from 0-100 with higher scores indicating better psychological health. | PRO Analysis Set: all participants in the FAS with baseline and at least one valid CHQ-PF-50 response at post-baseline. All participants included in the Overall Number of Participants Analyzed contributed data to this outcome measure. | Posted | Mean | Standard Deviation | Scores on a scale | Baseline and Month 12, 24, and 36 |
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| Secondary | Change From Baseline in Childhood Health Assessment Questionnaire (CHAQ) Disability Index Score at 12, 24, and 36 Months | The CHAQ was developed to measure the physical functioning in children 6 months to 18 years of age. It consists of 54 questions related to the child's ability to perform various activities of daily living. Depending on the question asked, each question is scored either 0 to 3 based on the level of difficulty experienced by the child or 0-1 based on whether the child required assistance from another person or used an aid or other device. All CHAQ questions were scored and converted to a total index score ranging from 0-3, where higher scores indicate greater disability. | PRO Analysis Set: all participants in the FAS with baseline and at least one valid CHAQ response at post-baseline. All participants included in the Overall Number of Participants Analyzed contributed data to this outcome measure. | Posted | Mean | Standard Deviation | Scores on a scale | Baseline and Month 12, 24, and 36 |
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| Secondary | Change From Baseline in Wong-Baker FACES Pain Rating Scale (WBFPRS) at 12, 24, and 36 Months | The WBFPRS is a horizontal pain scale for children 3-18 years which consists of 6 faces that range from a smiling "no hurt" face with a score of 0 to a crying "hurts worst" face with a score of 10. | PRO Analysis Set: all participants in the FAS with baseline and at least one valid WBFPRS response at post-baseline. All participants included in the Overall Number of Participants Analyzed contributed data to this outcome measure. | Posted | Mean | Standard Deviation | Scores on a scale | Baseline and Month 12, 24, and 36 |
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| Secondary | Change From Baseline in Growth Velocity Z-score (Height) at 12, 24, and 36 Months | Growth velocity was determined by calculating age-adjusted z-scores for height, weight, and body mass index (BMI). Z-scores represent the number of standard deviations from the reference population's mean, with 0 denoting the mean. Positive changes from baseline signify increased growth velocity. | Growth Velocity Analysis Set: all participants in the FAS who have non-missing height and age in total months at baseline and postbaseline. Only participants with available data for growth velocity (height) are included. All participants included in the Overall Number of Participants Analyzed contributed data to this outcome measure. | Posted | Mean | Standard Deviation | Z-score | Baseline and Month 12, 24, and 36 |
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| Secondary | Change From Baseline in Growth Velocity Z-score (Weight) at 12, 24, and 36 Months | Growth velocity was determined by calculating age-adjusted z-scores for height, weight, and BMI. Z-scores represent the number of standard deviations from the reference population's mean, with 0 denoting the mean. Positive changes from baseline signify increased growth velocity. | Growth Velocity Analysis Set: all participants in the FAS who have non-missing weight and age in total months at baseline and postbaseline. Only participants with available data for growth velocity (weight) are included. | Posted | Mean | Standard Deviation | Z-score | Baseline and Month 12, 24, and 36 |
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| Secondary | Change From Baseline in Growth Velocity Z-score (BMI) at 12, 24, and 36 Months | Growth velocity was determined by calculating age-adjusted z-scores for height, weight, and BMI. Z-scores represent the number of standard deviations from the reference population's mean, with 0 denoting the mean. Positive changes from baseline signify increased growth velocity. | Growth Velocity Analysis Set: all participants in the FAS who have non-missing BMI and age in total months at baseline and postbaseline. Only participants with available data for growth velocity (BMI) are included. All participants included in the Overall Number of Participants Analyzed contributed data to this outcome measure. | Posted | Mean | Standard Deviation | Z-score | Baseline and Month 12, 24, and 36 |
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| Secondary | Mean Serum Concentration of Denosumab | PK Analysis Set: all participants in the FAS who have ≥ 1 denosumab reported result. | Posted | Mean | Standard Deviation | ng/mL | Day 1, Day 10, Day 30, Month 3, Month 6, Month 12, and Month 18 |
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From date of enrollment through last dose, up to 36 months.
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Baseline-Month 12: Placebo | Participants received placebo by SC injection during the first 12 months of the Treatment Period. | 0 | 8 | 1 | 8 | 5 | 8 |
| EG001 | Baseline-Month 12: Denosumab | Participants received 1 mg/kg Denosumab up to a maximum of 60 mg, Q6M by SC injection during the first 12 months of the Treatment Period. | 0 | 16 | 3 | 16 | 11 | 16 |
| EG002 | Baseline-Month 24: Placebo/Denosumab | Participants received matching placebo by SC injection Q6M for the first 12 months of the Treatment Period. This was followed by 1 mg/kg Denosumab administered by SC injection Q6M for the second 12 months of the Treatment Period. | 0 | 8 | 1 | 8 | 5 | 8 |
| EG003 | Baseline-Month 24: Denosumab/Denosumab | Participants received 1 mg/kg Denosumab by SC injection up to a maximum of 60 mg, Q6M for 24 months during the Treatment Period. | 0 | 16 | 3 | 16 | 12 | 16 |
| EG004 | Baseline-Month 36: Placebo/Denosumab | Participants received matching placebo by SC injection Q6M for the first 12 months of the Treatment Period. This was followed by 1 mg/kg Denosumab up to a maximum of 60 mg, Q6M administered by SC injection for the second 12 months of the Treatment Period. Participants were then followed for an additional 12 months during the Off-treatment Observation Period. | 0 | 8 | 2 | 8 | 5 | 8 |
| EG005 | Baseline-Month 36: Denosumab/Denosumab | Participants received 1 mg/kg Denosumab by SC injection up to a maximum of 60 mg, Q6M for 24 months during the Treatment Period. Participants were then followed for an additional 12 months during the Off-treatment Observation Period. | 0 | 16 | 4 | 16 | 13 | 16 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Cardiomyopathy | Cardiac disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Autoimmune hepatitis | Hepatobiliary disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Brain contusion | Injury, poisoning and procedural complications | MedDRA 26.1 | Systematic Assessment |
| |
| Femur fracture | Injury, poisoning and procedural complications | MedDRA 26.1 | Systematic Assessment |
| |
| Tibia fracture | Injury, poisoning and procedural complications | MedDRA 26.1 | Systematic Assessment |
| |
| Ureterolithiasis | Renal and urinary disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Uterine haemorrhage | Reproductive system and breast disorders | MedDRA 26.1 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Iron deficiency anaemia | Blood and lymphatic system disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Cardiac failure | Cardiac disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Cardiomyopathy | Cardiac disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Intracranial lipoma | Congenital, familial and genetic disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Delayed puberty | Endocrine disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Astigmatism | Eye disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Cataract | Eye disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Cataract subcapsular | Eye disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Abdominal discomfort | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Dental caries | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Duodenal bulb deformity | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Duodenogastric reflux | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Gastrointestinal disorder | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Oesophagitis | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Hyperthermia | General disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Injection site pain | General disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Autoimmune hepatitis | Hepatobiliary disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Biliary tract disorder | Hepatobiliary disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Balanitis candida | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| COVID-19 pneumonia | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Chronic tonsillitis | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Coronavirus infection | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Epstein-Barr virus infection | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Fungal skin infection | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Gastrointestinal infection | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Gingivitis | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Hordeolum | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Skin infection | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Varicella | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Viral tonsillitis | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Vulvitis | Infections and infestations | MedDRA 26.1 | Systematic Assessment |
| |
| Femur fracture | Injury, poisoning and procedural complications | MedDRA 26.1 | Systematic Assessment |
| |
| Tooth fracture | Injury, poisoning and procedural complications | MedDRA 26.1 | Systematic Assessment |
| |
| Bone density decreased | Investigations | MedDRA 26.1 | Systematic Assessment |
| |
| Iron deficiency | Metabolism and nutrition disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Vitamin D deficiency | Metabolism and nutrition disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Autoimmune arthritis | Musculoskeletal and connective tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Costochondritis | Musculoskeletal and connective tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Dwarfism | Musculoskeletal and connective tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Osteonecrosis | Musculoskeletal and connective tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Hypoaesthesia | Nervous system disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Myelopathy | Nervous system disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Anxiety | Psychiatric disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Metabolic nephropathy | Renal and urinary disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Dysmenorrhoea | Reproductive system and breast disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Central sleep apnoea syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Nasal septum deviation | Respiratory, thoracic and mediastinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Obstructive sleep apnoea syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Vasomotor rhinitis | Respiratory, thoracic and mediastinal disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Acne | Skin and subcutaneous tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Chronic spontaneous urticaria | Skin and subcutaneous tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Dermal cyst | Skin and subcutaneous tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Eczema | Skin and subcutaneous tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA 26.1 | Systematic Assessment |
| |
| Ingrowing nail | Skin and subcutaneous tissue disorders | MedDRA 26.1 | Systematic Assessment |
|
The Clinical Trial Agreement generally does not restrict an investigator's discussion of trial results after completion. The Agreement permits Amgen a limited period of time to review material discussing trial results (typically up to 45 days and possible extension). Amgen may remove confidential information, but authors have final control and approval of publication content. For multicenter studies, the investigator agrees not to publish any results before the first multi-center publication.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Amgen Inc. | 866-572-6436 | medinfo@amgen.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Nov 12, 2023 | May 29, 2024 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D010024 | Osteoporosis |
| ID | Term |
|---|---|
| D001851 | Bone Diseases, Metabolic |
| D001847 | Bone Diseases |
| D009140 | Musculoskeletal Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| D000069448 | Denosumab |
| ID | Term |
|---|---|
| D061067 | Antibodies, Monoclonal, Humanized |
| D000911 | Antibodies, Monoclonal |
| D000906 | Antibodies |
| D007136 | Immunoglobulins |
| D007162 | Immunoproteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D012712 | Serum Globulins |
| D005916 | Globulins |
Not provided
Not provided
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