Study to Assess Long term Safety and Efficacy of Current or Prior Treatment with Denosumab in Children/Young Adults with Osteogenesis Imperfecta
- Conditions
- Osteogenesis Imperfecta (OI)MedDRA version: 20.0Level: PTClassification code 10031243Term: Osteogenesis imperfectaSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
- Registration Number
- EUCTR2018-000550-21-DE
- Lead Sponsor
- Amgen Inc
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 150
Subjects are eligible to be included in the study only if all of the following criteria apply:
-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 was enrolled in Study 20130173 and:
? - completed the 20130173 EOS visit (regardless of completing or ending investigational product early).
OR
?- Subjects who do not reconsent to transition to 3-Month Dosing Regimen on Study 20130173 are also eligible for enrollment
OR
? Early terminated from Study 20130173 as a result of meeting BMD Z-score investigational product stopping criteria and was required to early terminate from the study.
Are the trial subjects under 18? yes
Number of subjects for this age range: 114
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 36
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
Subjects are excluded from the study if any of the following criteria
apply:
Prior/Concomitant Therapy
-Treatment with any prohibited proscribed medications during Study
20130173 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.
Prior/Concurrent Clinical Study Experience
-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.
Other Exclusions
-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 = 2) should only be included in the study after a negative
highly sensitive urine 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 (IP)
(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 IP (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
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: -To evaluate long-term safety of denosumab in subjects with pediatric osteogenesis imperfecta (OI) completing Study 20130173;Secondary Objective: - To describe changes in bone mineral density (BMD) of lumbar spine<br>and proximal femur (total hip and femoral neck) from baseline to 12 and<br>24 months<br>;Primary end point(s): -The primary endpoints are subject incidence<br>of adverse events, serious adverse events and<br>adverse events of special interest, subject prevalence of<br>immunogenicity, changes from baseline in laboratory values and vital<br>signs, and subject incidence of metaphyseal index Z score above age-appropriate<br>normal range, abnormal molar eruption, and mandibular shaping;Timepoint(s) of evaluation of this end point: -at 3, 12 and 24 months
- Secondary Outcome Measures
Name Time Method Secondary end point(s): - Actual values and changes in BMD Z score of lumbar spine and proximal<br>femur (total hip and femoral neck) from Study 20170534 baseline, as assessed by dual X-ray<br>absorptiometry (DXA), at 6, 12 and 24 months<br>;Timepoint(s) of evaluation of this end point: -at 6, 12 and 24 months