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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03164928




Registration number
NCT03164928
Ethics application status
Date submitted
8/05/2017
Date registered
24/05/2017

Titles & IDs
Public title
Safety and Efficiency of Denosumab in Pediatric Subjects With Glucocorticoid-induced Osteoporosis
Scientific title
A Phase 3 Randomized, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Safety and Efficacy of Denosumab in Pediatric Subjects With Glucocorticoid-induced Osteoporosis
Secondary ID [1] 0 0
2016-003083-39
Secondary ID [2] 0 0
20140444
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Evaluate the Safety and Efficacy of Denosumab in Pediatric Subjects With 0 0
Glucocorticoid-induced Osteoporosis 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Osteoporosis

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Denosumab
Other interventions - Placebo

Other: Placebo - SC Q6M placebo

Experimental: Denosumab - 1 mg/kg BW (up to a maximum of 60 mg) SC Q6M


Treatment: Drugs: Denosumab
1mg/kg BW (up to a maximum of 60 mg) SC Q6M

Other interventions: Placebo
SC Q6M placebo
Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change From Baseline in Lumbar Spine BMD Z-score as Assessed by Dual-energy X-ray Absorptiometry (DXA) at 12 Months
Timepoint [1] 0 0
Baseline and 12 Months
Secondary outcome [1] 0 0
Change From Baseline in Lumbar Spine BMD Z-score as Assessed by DXA at 6, 18, 24, and 36 Months
Timepoint [1] 0 0
Baseline and 6, 18, 24, and 36 Months
Secondary outcome [2] 0 0
Change From Baseline in Proximal Femur BMD Z-score as Assessed by DXA at 6, 12, 18, 24, and 36 Months
Timepoint [2] 0 0
Baseline and 6, 12, 18, 24, and 36 Months
Secondary outcome [3] 0 0
Number of Participants With X-ray Confirmed Long-bone Fractures and/or Vertebral Fractures at 12, 24, and 36 Months
Timepoint [3] 0 0
Month 12, 24, and 36
Secondary outcome [4] 0 0
Number of Participants With Improving Vertebral Fractures at 12, 24, and 36 Months
Timepoint [4] 0 0
Month 12, 24, and 36
Secondary outcome [5] 0 0
Number of Participants With New and Worsening Vertebral and Non-vertebral Fractures at 12, 24, and 36 Months
Timepoint [5] 0 0
Month 12, 24, and 36
Secondary outcome [6] 0 0
Change From Baseline in Child Health Questionnaire-Parent Form-50 (CHQ-PF-50) Physical Summary Score at 12, 24, and 36 Months
Timepoint [6] 0 0
Baseline and month 12, 24, and 36
Secondary outcome [7] 0 0
Change From Baseline in CHQ-PF-50 Psychological Summary Score at 12, 24, and 36 Months
Timepoint [7] 0 0
Baseline and Month 12, 24, and 36
Secondary outcome [8] 0 0
Change From Baseline in Childhood Health Assessment Questionnaire (CHAQ) Disability Index Score at 12, 24, and 36 Months
Timepoint [8] 0 0
Baseline and Month 12, 24, and 36
Secondary outcome [9] 0 0
Change From Baseline in Wong-Baker FACES Pain Rating Scale (WBFPRS) at 12, 24, and 36 Months
Timepoint [9] 0 0
Baseline and Month 12, 24, and 36
Secondary outcome [10] 0 0
Change From Baseline in Growth Velocity Z-score (Height) at 12, 24, and 36 Months
Timepoint [10] 0 0
Baseline and Month 12, 24, and 36
Secondary outcome [11] 0 0
Change From Baseline in Growth Velocity Z-score (Weight) at 12, 24, and 36 Months
Timepoint [11] 0 0
Baseline and Month 12, 24, and 36
Secondary outcome [12] 0 0
Change From Baseline in Growth Velocity Z-score (BMI) at 12, 24, and 36 Months
Timepoint [12] 0 0
Baseline and Month 12, 24, and 36
Secondary outcome [13] 0 0
Mean Serum Concentration of Denosumab
Timepoint [13] 0 0
Day 1, Day 10, Day 30, Month 3, Month 6, Month 12, and Month 18

Eligibility
Key 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
Minimum age
5 Years
Maximum age
17 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
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 causes of primary or secondary osteoporosis (other than GC use), or previous exposure to non-GC medications, which the investigator considers to have been a major factor contributing to the patient's fracture(s)
* Current adrenal insufficiency as the sole indication for GC therapy
* Duchenne muscular dystrophy with symptomatic cardiac abnormality
* Current malabsorption (in children with serum albumin -lower limit of normal [LLN], malabsorption should be clinically ruled out by the investigator to confirm eligibility)
* Known intolerance to calcium or vitamin D supplements
* Active infection or history of infections, defined as follows:
* 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
7/05/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
20/12/2023
Sample size
Target
Accrual to date
Final
24
Recruitment in Australia
Recruitment state(s)
WA
Recruitment hospital [1] 0 0
Perth Childrens Hospital - Nedlands
Recruitment postcode(s) [1] 0 0
6909 - Nedlands
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Delaware
Country [3] 0 0
United States of America
State/province [3] 0 0
Indiana
Country [4] 0 0
United States of America
State/province [4] 0 0
Minnesota
Country [5] 0 0
United States of America
State/province [5] 0 0
Ohio
Country [6] 0 0
Belgium
State/province [6] 0 0
Bruxelles
Country [7] 0 0
Bulgaria
State/province [7] 0 0
Sofia
Country [8] 0 0
Canada
State/province [8] 0 0
Ontario
Country [9] 0 0
Canada
State/province [9] 0 0
Quebec
Country [10] 0 0
Colombia
State/province [10] 0 0
Antioquia
Country [11] 0 0
Colombia
State/province [11] 0 0
Cundinamarca
Country [12] 0 0
Colombia
State/province [12] 0 0
Santander
Country [13] 0 0
India
State/province [13] 0 0
Andhra Pradesh
Country [14] 0 0
India
State/province [14] 0 0
Delhi
Country [15] 0 0
India
State/province [15] 0 0
Karnataka
Country [16] 0 0
India
State/province [16] 0 0
Tamil Nadu
Country [17] 0 0
Italy
State/province [17] 0 0
Firenze
Country [18] 0 0
Italy
State/province [18] 0 0
Milan
Country [19] 0 0
Italy
State/province [19] 0 0
Roma
Country [20] 0 0
Mexico
State/province [20] 0 0
Ciudad de Mexico
Country [21] 0 0
Peru
State/province [21] 0 0
Lima
Country [22] 0 0
Peru
State/province [22] 0 0
Arequipa
Country [23] 0 0
Peru
State/province [23] 0 0
Callao
Country [24] 0 0
Russian Federation
State/province [24] 0 0
Moscow
Country [25] 0 0
Russian Federation
State/province [25] 0 0
Novosibirsk
Country [26] 0 0
Russian Federation
State/province [26] 0 0
Saint Petersburg
Country [27] 0 0
Turkey
State/province [27] 0 0
Ankara
Country [28] 0 0
Turkey
State/province [28] 0 0
Erzurum
Country [29] 0 0
Turkey
State/province [29] 0 0
Istanbul
Country [30] 0 0
Turkey
State/province [30] 0 0
Izmir
Country [31] 0 0
Ukraine
State/province [31] 0 0
Dnipro
Country [32] 0 0
Ukraine
State/province [32] 0 0
Kharkiv
Country [33] 0 0
Ukraine
State/province [33] 0 0
Kyiv

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Amgen
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
MD
Address 0 0
Amgen
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request

Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Informed consent form (ICF), Clinical study report (CSR)
When will data be available (start and end dates)?
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.
Available to whom?
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.
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://www.amgen.com/datasharing


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT03164928