Osteogenesis Imperfecta (OI) MedDRA version: 20.0 Level: PT Classification code 10031243 Term: Osteogenesis imperfecta System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Sponsors
Amgen Inc
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 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 IP 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
Exclusion criteria
Exclusion criteria: 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 month 36 visit (regardless of completing or ending investigational product early). 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 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 (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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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 and proximal femur (total hip and femoral neck) from baseline to 12 and 24 months; - To describe the incidence of X-ray confirmed new and worsening vertebral fractures from baseline to 12 and 24 months; - To describe the incidence of X-ray confirmed new vertebral fractures from baseline to 12 and 24 months; - To describe the incidence of X-ray confirmed long bone and new and worsening vertebral fractures from baseline to 12 and 24 months - To describe the incidence of vertebral and nonvertebral fractures from baseline to 12 months and 24 months; - To describe the change in growth velocity (determined by calculating age-adjusted Z-scores for height, weight, and body mass index [BMI]) at 12 and 24 months;;Primary end point(s): -The primary endpoint is safety monitoring, including subject incidence of adverse events, serious adverse events and adverse events of special interest, subject prevalence of immunogenicity, changes from baseline in laboratory values and vital signs, and subject incidence of metaphyseal index Z score above ageappropriate normal range, abnormal molar eruption, and mandibular shaping;Timepoint(s) of evaluation of this end point: -at 3, 12 and 24 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): -Actual values and changes in BMD Z score of lumbar spine and proximal femur (total hip and femoral neck) from Study 20170534 baseline, and from Study 20130173 baseline, as assessed by dual X-ray absorptiometry (DXA), at 12 and 24 months - Subject incidence of X-ray confirmed new and worsening vertebral fractures from Study 20170534 baseline to 12 and 24 months - Subject incidence of X-ray confirmed new vertebral fractures from Study 20170534 baseline to 12 and 24 months - Subject incidence of X-ray confirmed long bone, and new and worsening vertebral fractures from Study 20170534 baseline to 12 and 24 months - Incidence of vertebral and nonvertebral fractures from Study 20170534 baseline to 12 and 24 months - Change from Study 20170534 baseline and from Study 20130173 baseline in growth velocity (determined by calculating age-adjusted Zscores for height, weight, and BMI) at 12 and 24 months;Timepoint(s) of evaluation of this end point: -at 3, 12 and 24 months | — |
Countries
Australia, Belgium, Bulgaria, Canada, Czechia, Czech Republic, France, Germany, Hungary, Italy, Poland, Spain, United Kingdom, United States
Contacts
Public ContactMedical Information
Amgen Biotechnologia Sp. z o.o.
Outcome results
None listed