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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03638128




Registration number
NCT03638128
Ethics application status
Date submitted
21/06/2018
Date registered
20/08/2018

Titles & IDs
Public title
Open-label Extension of Study 20130173 of Denosumab in Children and Young Adults With Osteogenesis Imperfecta
Scientific title
Multicenter, Single-arm Open-label Extension Study to Assess Long-term Safety and Efficacy of Current or Prior Treatment With Denosumab in Children/Young Adults With Osteogenesis Imperfecta
Secondary ID [1] 0 0
2018-000550-21
Secondary ID [2] 0 0
20170534
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Osteogenesis Imperfecta (OI) 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 - Denosumab
Treatment: Drugs - Alternative osteoporosis medications

Other: Alternative Medications / Observational - Participants who received non-denosumab alternative therapy during the study or who were not receiving any medication at baseline.

Alternative osteoporosis medication(s) were determined at the investigator's discretion and per standard of care and local guidelines.

Experimental: Denosumab 1 mg/kg Q6M - Participants who received at least 1 dose of 1 mg/kg denosumab administered once every 6 months (Q6M) by subcutaneous injection, but no Q3M denosumab during this study.

Experimental: Denosumab 1 mg/kg Q3M - Participants who received at least one dose of 1 mg/kg denosumab administered once every 3 months (Q3M) by subcutaneous injection during this study.


Treatment: Drugs: Denosumab
Solution for injection

Treatment: Drugs: Alternative osteoporosis medications
Alternative osteoporosis medication/s at the discretion of the investigator.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With Adverse Events, Serious Adverse Events, and Adverse Events of Special Interest
Timepoint [1] 0 0
From enrollment to end of study, including 24 weeks after last dose of denosumab for participants who received denosumab; the maximum time on study was 24 months.
Primary outcome [2] 0 0
Number of Participants With Anti-denosumab Antibodies
Timepoint [2] 0 0
From enrollment to end of study, including 24 weeks after last dose of denosumab for participants who received denosumab; the maximum time on study was 24 months
Primary outcome [3] 0 0
Number of Participants With Clinical Laboratory Toxicities Grade = 3
Timepoint [3] 0 0
From enrollment to end of study, including 24 weeks after last dose of denosumab for participants who received denosumab; the maximum time on study was 24 months
Primary outcome [4] 0 0
Number of Participants With Clinically Significant Vital Sign Findings
Timepoint [4] 0 0
From enrollment to end of study, including 24 weeks after last dose of denosumab for participants who received denosumab; the maximum time on study was 24 months
Primary outcome [5] 0 0
Number of Participants With Metaphyseal Index Z-score Above Age-appropriate Normal Range
Timepoint [5] 0 0
Baseline, month 12 and month 24
Primary outcome [6] 0 0
Number of Participants With Abnormal Molar Eruption of the First or Second Molar Based on Radiological Findings
Timepoint [6] 0 0
Baseline, month 12, and month 24
Primary outcome [7] 0 0
Percent Change From Baseline in Mandibular Shaping Parameters
Timepoint [7] 0 0
Baseline and month 12 and month 24
Secondary outcome [1] 0 0
Change From Baseline in Lumbar Spine Bone Mineral Density (BMD) Z-score
Timepoint [1] 0 0
Baseline and months 6, 12, and 24
Secondary outcome [2] 0 0
Change From Baseline in Total Hip BMD Z-score
Timepoint [2] 0 0
Baseline, months 6, 12, and 24
Secondary outcome [3] 0 0
Change From Baseline in Femoral Neck BMD Z-score
Timepoint [3] 0 0
Baseline and months 6, 12, and 24

Eligibility
Key inclusion criteria
* Subject has provided informed consent/assent prior to initiation of any Study 20170534 specific activities/procedures. 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 written assent based on local regulations and/or guidelines prior to any study-specific activities/procedures being initiated.
* Subject is currently/was enrolled in Study 20130173 and

* completed the 20130173 End of Study (EOS) visit (regardless of completing or ending investigational product early) OR
* did not reconsent/reassent to transition to 3-month dosing regimen on Study 20130173 OR
* early terminated from Study 20130173 as a result of meeting bone mineral density (BMD) Z-score investigational product stopping criteria.
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Treatment with any prohibited proscribed medications while receiving denosumab. Eligibility into study treatment with alternative osteoporosis medication/s of investigator's choice, follow guidelines per the specific alternative osteoporosis medication/s selected. For subjects off-treatment (observation only), no prohibited medications apply.
* Subjects currently receiving treatment in another investigational device or drug study other than Study 20130173. Other investigational procedures while participating in this study are excluded.
* For subjects expected to receive investigational product (denosumab) at study day 1: Female subject is pregnant or breastfeeding or planning to become pregnant or breastfeed during treatment and for an additional 5 months after the last dose of denosumab. Females of childbearing potential (Tanner Stage greater than or equal to 2) should only be included in the study after a negative highly sensitive urine or serum pregnancy test. For study treatment with alternative osteoporosis medication/s of investigator's choice, follow guidelines per the specific alternative osteoporosis medication/s selected. For Subjects off-treatment (observation only), no exclusion applies.
* For subjects expected to receive investigational product (denosumab) at study day 1: Female subjects of childbearing potential unwilling to practice true sexual abstinence (refrain from heterosexual intercourse) or use 1 highly effective method of contraception during treatment and for an additional 5 months after the last dose of investigational product (denosumab). For study treatment with alternative osteoporosis medication/s of investigator's choice, follow contraception guidelines per the specific alternative osteoporosis medication/s selected. For subjects not receiving any investigational product (observation only), no contraception required.
* History or evidence of any other clinically significant disorder, condition or disease (with the exception of those outlined above) that, in the opinion of the investigator or Amgen physician, if consulted, would pose a risk to subject safety or interfere with the study evaluation, procedures or completion.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised 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
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
26/07/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
28/03/2022
Sample size
Target
Accrual to date
Final
75
Recruitment in Australia
Recruitment state(s)
NSW,WA
Recruitment hospital [1] 0 0
The Childrens Hospital at Westmead - Westmead
Recruitment hospital [2] 0 0
Perth Childrens Hospital - Nedlands
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment postcode(s) [2] 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
Indiana
Country [3] 0 0
Belgium
State/province [3] 0 0
Bruxelles
Country [4] 0 0
Canada
State/province [4] 0 0
Ontario
Country [5] 0 0
Canada
State/province [5] 0 0
Quebec
Country [6] 0 0
Czechia
State/province [6] 0 0
Plzen
Country [7] 0 0
Czechia
State/province [7] 0 0
Praha 4
Country [8] 0 0
France
State/province [8] 0 0
Bordeaux Cedex
Country [9] 0 0
France
State/province [9] 0 0
Paris Cedex 15
Country [10] 0 0
Germany
State/province [10] 0 0
Köln
Country [11] 0 0
Hungary
State/province [11] 0 0
Budapest
Country [12] 0 0
Italy
State/province [12] 0 0
Roma
Country [13] 0 0
Poland
State/province [13] 0 0
Lodz
Country [14] 0 0
Spain
State/province [14] 0 0
Cataluña
Country [15] 0 0
Spain
State/province [15] 0 0
Comunidad Valenciana
Country [16] 0 0
United Kingdom
State/province [16] 0 0
Birmingham
Country [17] 0 0
United Kingdom
State/province [17] 0 0
Bristol
Country [18] 0 0
United Kingdom
State/province [18] 0 0
Glasgow
Country [19] 0 0
United Kingdom
State/province [19] 0 0
Sheffield

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 URL 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/NCT03638128