Open-label Extension Denosumab Study in Children and Young Adults With Osteogenesis Imperfecta

• Register: ClinicalTrials.gov
• Last refreshed on: 25 April 2022
• Main ID: NCT03638128
• Date of registration: 21/06/2018
• Prospective Registration: Yes
• Primary sponsor: Amgen
• Public title: Open-label Extension Denosumab Study 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
• Date of first enrolment: July 26, 2018
• Target sample size: 75
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03638128
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Australia, Belgium, Canada, Czechia, France, Germany, Hungary, Italy, Poland, Spain, United Kingdom, United States

Contacts

• Name: MD
• Affiliation: Amgen

Key inclusion & exclusion criteria

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 subjects who do not reconsent/rassent 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.

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.



Age minimum: 5 Years
Age maximum: 20 Years
Gender: All

Health Condition(s) or Problem(s) studied

Osteogenesis Imperfecta (OI)

Intervention(s)

Drug: Alternative osteoporosis medications

Other: No treatment

Drug: Denosumab

Primary Outcome(s)

Number of subjects with clinically significant changes from baseline in laboratory values. [Time Frame: Baseline to 30 Months]

Subject incidence of abnormal molar eruption. [Time Frame: 30 Months]

Subject incidence of treatment-emergent adverse events. [Time Frame: 30 Months]

Subject incidence of antidenosumab antibodies. [Time Frame: 30 Months]

Subject incidence of serious treatment-emergent adverse events. [Time Frame: 30 Months]

Subject incidence of mandibular shaping. [Time Frame: 30 Months]

Number of subjects with clinically significant changes from baseline in vital signs. [Time Frame: Baseline to 30 Months]

Subject incidence of metaphyseal index Z-score above age-appropriate normal range. [Time Frame: 30 Months]

Subject incidence of treatment-emergent adverse events of special interest. [Time Frame: 30 Months]

Secondary Outcome(s)

Denosumab 3-Month regimen in 20170534: Actual values of bone mineral density (BMD) of lumbar spine and proximal femur [Time Frame: Baseline and at 6, 12, and 24 months]

Denosumab 3-Month regimen in 20170534: Change from baseline in bone mineral density (BMD) of lumbar spine and proximal femur [Time Frame: Baseline to 6, 12, and 24 months]

Secondary ID(s)

20170534

2018-000550-21

Source(s) of Monetary Support

Please refer to primary and secondary sponsors