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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05972551




Registration number
NCT05972551
Ethics application status
Date submitted
24/07/2023
Date registered
2/08/2023

Titles & IDs
Public title
Study to Evaluate Efficacy and Safety of Romosozumab Compared With Bisphosphonates in Children and Adolescents With Osteogenesis Imperfecta
Scientific title
A Phase 3, Open-Label, Multicenter, Randomized Study to Evaluate Efficacy and Safety of Romosozumab Compared With Bisphosphonates in Children and Adolescents With Osteogenesis Imperfecta
Secondary ID [1] 0 0
2023-503294-37
Secondary ID [2] 0 0
20200105
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Osteogenesis Imperfecta 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Injuries and Accidents 0 0 0 0
Fractures

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Romosozumab
Treatment: Drugs - Bisphosphonate

Experimental: Romosozumab - Participants will receive romosozumab once a month (QM) for 12 months.

Active comparator: Standard of Care Bisphosphonate - Participants will receive bisphosphonates per local standard of care treatment regimens, as determined by the investigator for 12 months.


Treatment: Drugs: Romosozumab
Subcutaneous (SC) injection

Treatment: Drugs: Bisphosphonate
Administration determined by investigator according to the local standard of care
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Clinical Fractures
Timepoint [1] 0 0
12 months
Primary outcome [2] 0 0
Number of Any Fractures
Timepoint [2] 0 0
12 months
Primary outcome [3] 0 0
Change from Baseline to 12 Months in Lumbar Spine BMD Z-score
Timepoint [3] 0 0
Baseline and 12 months
Secondary outcome [1] 0 0
Change from Baseline in Lumbar Spine BMD Z-score at 6 Months and at 12 Months
Timepoint [1] 0 0
Baseline, 6 months, and 12 months
Secondary outcome [2] 0 0
Change from Baseline in Total Hip BMD Z-score at 6 Months and at 12 Months
Timepoint [2] 0 0
Baseline, 6 months, and 12 months
Secondary outcome [3] 0 0
Change from Baseline in Femoral Neck BMD Z-score at 6 Months and at 12 Months
Timepoint [3] 0 0
Baseline, 6 months, and 12 months
Secondary outcome [4] 0 0
Number of Participants with Any Fractures
Timepoint [4] 0 0
12 months
Secondary outcome [5] 0 0
Number of Participants with Clinical Fractures
Timepoint [5] 0 0
12 months
Secondary outcome [6] 0 0
Number of Participants with New or Worsening Vertebral Fractures
Timepoint [6] 0 0
12 months
Secondary outcome [7] 0 0
Number of Participants with Nonvertebral Fractures
Timepoint [7] 0 0
12 months
Secondary outcome [8] 0 0
Number of Participants with Long Bone Fractures
Timepoint [8] 0 0
12 months
Secondary outcome [9] 0 0
Number of New or Worsening Vertebral Fractures
Timepoint [9] 0 0
12 months
Secondary outcome [10] 0 0
Number of Nonvertebral Fractures
Timepoint [10] 0 0
12 months
Secondary outcome [11] 0 0
Number of Long Bone Fractures
Timepoint [11] 0 0
12 months
Secondary outcome [12] 0 0
Change from Baseline in Child Health Questionnaire - Parent Version (CHQ-PF-50) Physical Summary Score
Timepoint [12] 0 0
Baseline and 12 months
Secondary outcome [13] 0 0
Change from Baseline in Childhood Health Assessment Questionnaire (CHAQ) Disability Score
Timepoint [13] 0 0
Baseline and 12 months
Secondary outcome [14] 0 0
Change from Baseline in the Wong-Baker Faces Pain Rating Scale
Timepoint [14] 0 0
Baseline and 12 months
Secondary outcome [15] 0 0
Serum Concentration of Romosozumab
Timepoint [15] 0 0
Day 1 to Month 12
Secondary outcome [16] 0 0
Number of Participants who Experience Treatment-emergent Adverse Events (TEAEs) at 12 Months
Timepoint [16] 0 0
12 months
Secondary outcome [17] 0 0
Number of Participants who Experience TEAEs from Month 12 to Month 15
Timepoint [17] 0 0
Month 12 to Month 15
Secondary outcome [18] 0 0
Number of Participants with Anti-drug Antibodies (ADA) to Romosozumab
Timepoint [18] 0 0
Up to 15 months
Secondary outcome [19] 0 0
Number of Participants who Experience TEAEs at 15 Months
Timepoint [19] 0 0
15 months
Secondary outcome [20] 0 0
Number of Participants with a Narrowing from Baseline to 6 Months in the Intracranial Nerve Tract in the Cranium and Vault of the Skull
Timepoint [20] 0 0
Baseline and 6 months
Secondary outcome [21] 0 0
Number of Participants with a Narrowing from Baseline to 12 Months in the Intracranial Nerve Tract in the Cranium and Vault of the Skull
Timepoint [21] 0 0
Baseline and 12 months

Eligibility
Key inclusion criteria
* Participant has provided informed consent/assent prior to initiation of any study specific activities/procedures.

OR

* Participant's legally authorized representative has provided informed consent when the participant is legally too young to provide informed consent and the participant has provided written assent based on local regulations and/or guidelines prior to any study-specific activities/procedures being initiated.
* Ambulatory male and female children and adolescents, age 5 to <18 years, including ambulatory with assistance as defined in the pediatric osteogenesis imperfecta (OI) population.
* Clinical diagnosis of OI, defined as clinical history consistent with type I, III, or IV OI as determined by presence of expected phenotype (examples include: facial shape, voice, blue sclera, dentinogenesis imperfecta, typical radiographic features, fracture pattern) and lack of additional features unrelated to type I, III, or IV OI (eg, blindness, mental retardation, neuropathy, and craniosynostosis).

o If familial, also must be autosomal dominant.
* Meets at least one of the following:

* 3 or more fractures within the previous 2 years, or
* 1 or more nonvertebral fracture(s) within the previous 2 years and at least 1 prevalent vertebral fracture, or
* 2 or more prevalent vertebral fractures.
Minimum age
5 Years
Maximum age
17 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Disease Related

* History of an electrophoresis pattern inconsistent with type I, III or IV OI.
* History of known mutation in a gene other than collagen type I alpha 1/collagen type I alpha 2 (COL1A1/COL1A2) causing OI or other metabolic bone disease.
* History of congenital dislocation of the radial head, interosseous membrane calcification, or exuberant callus formation.

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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
22/04/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
27/05/2027
Actual
Sample size
Target
106
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC,WA
Recruitment hospital [1] 0 0
Monash Childrens Hospital - Clayton
Recruitment hospital [2] 0 0
Perth Childrens Hospital - Nedlands
Recruitment postcode(s) [1] 0 0
3168 - Clayton
Recruitment postcode(s) [2] 0 0
6909 - Nedlands
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Georgia
Country [2] 0 0
United States of America
State/province [2] 0 0
Indiana
Country [3] 0 0
United States of America
State/province [3] 0 0
Maryland
Country [4] 0 0
United States of America
State/province [4] 0 0
Massachusetts
Country [5] 0 0
United States of America
State/province [5] 0 0
Minnesota
Country [6] 0 0
United States of America
State/province [6] 0 0
Tennessee
Country [7] 0 0
Austria
State/province [7] 0 0
Linz
Country [8] 0 0
Belgium
State/province [8] 0 0
Leuven
Country [9] 0 0
Canada
State/province [9] 0 0
Ontario
Country [10] 0 0
Canada
State/province [10] 0 0
Quebec
Country [11] 0 0
France
State/province [11] 0 0
Paris Cedex 15
Country [12] 0 0
Germany
State/province [12] 0 0
Koeln
Country [13] 0 0
Germany
State/province [13] 0 0
Wuerzburg
Country [14] 0 0
Hungary
State/province [14] 0 0
Budapest
Country [15] 0 0
Japan
State/province [15] 0 0
Okayama
Country [16] 0 0
Japan
State/province [16] 0 0
Osaka
Country [17] 0 0
Japan
State/province [17] 0 0
Tokyo
Country [18] 0 0
Poland
State/province [18] 0 0
Lodz
Country [19] 0 0
Poland
State/province [19] 0 0
Tychy
Country [20] 0 0
Slovakia
State/province [20] 0 0
Bratislava
Country [21] 0 0
Spain
State/province [21] 0 0
Cataluña
Country [22] 0 0
Spain
State/province [22] 0 0
Madrid
Country [23] 0 0
Spain
State/province [23] 0 0
País Vasco
Country [24] 0 0
Switzerland
State/province [24] 0 0
Basel
Country [25] 0 0
Switzerland
State/province [25] 0 0
Lausanne
Country [26] 0 0
Turkey
State/province [26] 0 0
Ankara
Country [27] 0 0
Turkey
State/province [27] 0 0
Istanbul
Country [28] 0 0
Turkey
State/province [28] 0 0
Izmir
Country [29] 0 0
Turkey
State/province [29] 0 0
Trabzon
Country [30] 0 0
United Kingdom
State/province [30] 0 0
Glasgow

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
Amgen Call Center
Address 0 0
Country 0 0
Phone 0 0
866-572-6436
Fax 0 0
Email 0 0
medinfo@amgen.com
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 URL below.
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: http://www.amgen.com/datasharing


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05972551