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Evaluate the Safety and Efficacy of Denosumab in Pediatric Subjects WithPhase 3NCT03164928

Safety and Efficiency of Denosumab in Pediatric Subjects With Glucocorticoid-induced Osteoporosis

A Phase 3 study testing Denosumab in people with Evaluate the Safety and Efficacy of Denosumab in Pediatric Subjects With and Glucocorticoid-induced Osteoporosis, run by Amgen at 38 sites in 13 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

To evaluate the effect of denosumab on lumbar spine bone mineral density (BMD) Z-score as assessed by dual-energy X-ray absorptiometry (DXA) at 12 months in children 5 to 17 year of age with Glucocorticoid (GC)-induced osteoporosis (GiOP). Read more about this study

Who is it for?
Ages 5 to 17 years
What is being tested?
Denosumab
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
24 participants
Where
38 sitesin 13 countries
Study dates
7 May 2018to 20 Dec 2023
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 5 to 17 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • Male or female subjects, age 5 to 17 years, inclusive, at the time of informed consent.
  • Clinical diagnosis of GiOP as defined by the following (and consistent with the International Society for Clinical Densitometry definition of osteoporosis in children and adolescents [Bishop et al, 2014])
  • A confirmed diagnosis of non-malignant condition(s) requiring treatment with systemic GC (including, but not limited to, chronic rheumatologic, gastrointestinal, neurologic, respiratory, and/or nephrological conditions)
  • Subjects who are on systemic GC only as replacement therapy for adrenal insufficiency are not eligible for the study - Treatment with systemic GC (intravenous or oral) of any duration for the underlying non-malignant condition(s) within the 12 months prior to screening
  • Evidence of at least 1 vertebral compression fracture of Genant grade 1 or higher, as assessed by the central imaging vendor on lateral spine X-rays performed at screening or within 2 months prior to screening; OR, in the absence of vertebral compression fractures, presence of both clinically significant fracture history (ie, ≥ 2 long-bone fractures by age 10 years or ≥ 3 long-bone fractures at any age up to 17 years) and lumbar spine BMD Z-score ≤ -2.0, as assessed by the central imaging vendor.
  • 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 assent based on local regulations and/or guidelines prior to any study-specific activities/procedures being initiated
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  • A confirmed diagnosis of non-malignant condition(s) requiring treatment with systemic GC (including, but not limited to, chronic rheumatologic, gastrointestinal, neurologic, respiratory, and/or nephrological conditions)
  • Subjects who are on systemic GC only as replacement therapy for adrenal insufficiency are not eligible for the study
  • Treatment with systemic GC (intravenous or oral) of any duration for the underlying non malignant condition(s) within the 12 months prior to screening
  • Prepubertal children should be expected to require significant GC use during the study, per investigator opinion

Who cannot join

  • Current hyperthyroidism (unless well controlled on stable antithyroid therapy)
  • Current clinical hypothyroidism (unless well controlled on stable thyroid replacement therapy)
  • History of hyperparathyroidism
  • Current hypoparathyroidism
  • Duchenne muscular dystrophy with symptomatic cardiac abnormality
  • Current malabsorption
Show all 17Show fewer
  • Active infection or history of infections
  • History of malignancy
  • Any causes of primary or secondary osteoporosis (other than GC use), or previous exposure to non-GC medications, which the investigator considers to have been a major factor contributing to the patient's fracture(s)
  • Current adrenal insufficiency as the sole indication for GC therapy
  • Duchenne muscular dystrophy with symptomatic cardiac abnormality
  • Current malabsorption (in children with serum albumin -lower limit of normal [LLN], malabsorption should be clinically ruled out by the investigator to confirm eligibility)
  • Known intolerance to calcium or vitamin D supplements
  • Active infection or history of infections, defined as follows:
  • Any active infection for which systemic anti-infectives were used within 4 weeks prior to screening
  • Serious infection, defined as requiring hospitalization or intravenous anti infectives within 8 weeks prior to screening
  • Recurrent or chronic infection or other active infection that, in the opinion of the investigator, might compromise the safety of the subject

Trial locations and contacts

38 sites in 13 countries.

38 sites in 13 countries

United States6 sites
  • Metrohealth Medical Center

    Cleveland, Ohio, 44109

  • Nationwide Childrens Hospital

    Columbus, Ohio, 43205

  • Indiana University Hospital

    Indianapolis, Indiana, 46202

  • Childrens Hospital of Los Angeles

    Los Angeles, California, 90027

  • University of Minnesota Masonic Childrens Hospital Discovery Clinic

    Minneapolis, Minnesota, 55454

  • AI Dupont Hospital for Children

    Wilmington, Delaware, 19803

Other trials in United States

Peru5 sites
  • Centro Especializado de Enfermedades Neoplasicas

    Arequipa, 040001

  • Instituto Nacional de Salud del Nino

    Breña, Lima region, Lima 5

  • Hospital Nacional Alberto Sabogal Sologuren

    Callao, Callao 2

  • Clinica Angloamericana

    Lima, Lima 27

  • Centro de Investigacion Ricardo Palma

    Lima, Lima27

Other trials in Peru

India4 sites
  • KLES Dr Prabhakar Kore Hospital and Medical Research Centre

    Belagavi, Karnataka, 590010

  • Gandhi Medical College

    Hyderabad, Andhra Pradesh, 500 025

  • Sir Ganga Ram Hospital

    New Delhi, National Capital Territory of Delhi, 110 022

  • Christian Medical College

    Vellore, Tamil Nadu, 632 004

Other trials in India

Russia4 sites
  • FSAI Scientific Center of Childrens Health of MoH of the RF

    Moscow, 119991

  • SBEI of HPE First Moscow state medical university na I M Sechenov of MoH of Russian Federation

    Moscow, 119991

  • SBHI of Novosibirsk region City Pediatric Clinical Hospital of Emergency Care

    Novosibirsk, 630007

  • LLC Medical Technologies

    Saint Petersburg, 191025

Other trials in Russia

Turkey (Türkiye)4 sites
  • Ankara Universitesi Tip Fakultesi

    Ankara, 06590

  • Ataturk Universitesi Tip Fakultesi

    Erzurum, 25240

  • Marmara Universitesi Pendik Egitim Arastirma Hastanesi

    Istanbul, 34890

  • Ege Universitesi Tip Fakultesi

    Izmir, 35100

Other trials in Turkey (Türkiye)

Colombia3 sites
  • Solano y Terront Servicios Medicos Ltda - Unidad Integral de Endocrinologia Uniendo

    Bogota, Cundinamarca, 110221

  • Foscal Internacional-Fundacion Oftalmologica de Santander

    Floridablanca, Santander Department, 681004

  • Center for Clinical and Basic Research Colombia

    Medellín, Antioquia, 050021

Other trials in Colombia

Italy3 sites
  • Azienda Ospedaliera Universitaria Meyer

    Florence, 50139

  • Istituto Auxologico Italiano Istituto di Ricovero e Cura a Carattere Scientifico

    Milan, 20145

  • Azienda Ospedaliera Policlinico Umberto I

    Roma, 00161

Other trials in Italy

Ukraine3 sites
  • CI Dnipropetrovsk Regional Children Clinical Hospital of Dnipropetrovsk Regional Council

    Dnipro, 49100

  • Communal Institution of Healthcare Kharkiv City Clinical Children Hospital 16

    Kharkiv, 61075

  • National Childrens Specialized Hospital OHMATDYT

    Kyiv, 01025

Other trials in Ukraine

Canada2 sites
  • Centre Hospitalier Universitaire Sainte Justine

    Montreal, Quebec, H3T 1C5

  • Childrens Hospital of Eastern Ontario

    Ottawa, Ontario, K1H 8L1

Other trials in Canada

Australia1 site
  • Perth Childrens Hospital

    Nedlands, Western Australia, 6909

Other trials in Australia

Belgium1 site
  • Cliniques Universtaire Saint Luc Universite Catholique de Louvain

    Brussels, 1200

Other trials in Belgium

Bulgaria1 site
  • Medical Centre Synexus Sofia EOOD

    Sofia, 1784

Other trials in Bulgaria

Mexico1 site
  • RM Pharma Specialists SA de CV

    Mexico City, 03100

Other trials in Mexico

About this study

Official title: A Phase 3 Randomized, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Safety and Efficacy of Denosumab in Pediatric Subjects With Glucocorticoid-induced Osteoporosis

To evaluate the effect of denosumab on lumbar spine bone mineral density (BMD) Z-score as assessed by dual-energy X-ray absorptiometry (DXA) at 12 months in children 5 to 17 year of age with Glucocorticoid (GC)-induced osteoporosis (GiOP).

Being tested
Denosumab, Placebo
Keywords
Denosumab, Pediatric GIOP, Glucocorticoid-induced, Osteoporosis
Medical subject headings
Osteoporosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • DenosumabDrug

    1mg/kg BW (up to a maximum of 60 mg) SC Q6M

  • PlaceboOther

    SC Q6M placebo

Study groups in NCT03164928
Study groupRoleWhat they receive
PlaceboOtherSC Q6M placebo
Receives: Other: Placebo
DenosumabExperimental1 mg/kg BW (up to a maximum of 60 mg) SC Q6M
Receives: Drug: Denosumab

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change From Baseline in Lumbar Spine BMD Z-score as Assessed by Dual-energy X-ray Absorptiometry (DXA) at 12 Months

    Lumbar spine BMD was assessed by DXA and analyzed by analysis of covariance (ANCOVA) including treatment (denosumab vs placebo), baseline age, and baseline BMD z-score. DXA results were converted to z-scores, indicating number of standard deviations from the reference population's mean, with 0 denoting the mean. Positive changes from baseline signify lumbar spine BMD improvement.

    Measured: Baseline and 12 Months

Secondary outcomes (13)
  1. Change From Baseline in Lumbar Spine BMD Z-score as Assessed by DXA at 6, 18, 24, and 36 Months

    Lumbar spine BMD was assessed by DXA and analyzed by repeated measures analysis with randomization group, visit, baseline age, and baseline BMD z-score as fixed effects. Treatment-by-visit was included as an interaction term. DXA results were converted to z-scores, indicating number of standard deviations from the reference population's mean, with 0 denoting the mean. Positive changes from baseline signify lumbar spine BMD improvement.

    Measured: Baseline and 6, 18, 24, and 36 Months

  2. Change From Baseline in Proximal Femur BMD Z-score as Assessed by DXA at 6, 12, 18, 24, and 36 Months

    Proximal femur (total hip and femoral neck) BMD was assessed by DXA and analyzed by repeated measures analysis with randomization group, visit, baseline age, and baseline BMD z-score as fixed effects. Treatment-by-visit was included as an interaction term. DXA results were converted to z-scores, indicating number of standard deviations from the reference population's mean, with 0 denoting the mean. Positive changes from baseline signify proximal femur BMD improvement.

    Measured: Baseline and 6, 12, 18, 24, and 36 Months

  3. Number of Participants With X-ray Confirmed Long-bone Fractures and/or Vertebral Fractures at 12, 24, and 36 Months

    Number of participants who have at least one long bone fracture or vertebral fracture, and number of participants who have more than one long bone fracture or vertebral fracture.

    Measured: Month 12, 24, and 36

  4. Number of Participants With Improving Vertebral Fractures at 12, 24, and 36 Months

    Number of participants with improving vertebral fractures. An improving fracture is defined as one showing signs of healing/repair from baseline as assessed by X-ray.

    Measured: Month 12, 24, and 36

  5. Number of Participants With New and Worsening Vertebral and Non-vertebral Fractures at 12, 24, and 36 Months

    Number of participants who have at least one vertebral fracture or non-vertebral fracture, and number of participants who have more than one vertebral fracture or non-vertebral fracture.

    Measured: Month 12, 24, and 36

  6. Change From Baseline in Child Health Questionnaire-Parent Form-50 (CHQ-PF-50) Physical Summary Score at 12, 24, and 36 Months

    The CHQ-PF-50 is a 50-item questionnaire to be completed by the parents or guardians of children between 5 and 18 years of age. The physical summary score ranges from 0-100 with higher scores indicating better physical health.

    Measured: Baseline and month 12, 24, and 36

  7. Change From Baseline in CHQ-PF-50 Psychological Summary Score at 12, 24, and 36 Months

    The CHQ-PF-50 is a 50-item questionnaire to be completed by the parents or guardians of children between 5 and 18 years of age. The psychological summary score ranges from 0-100 with higher scores indicating better psychological health.

    Measured: Baseline and Month 12, 24, and 36

  8. Change From Baseline in Childhood Health Assessment Questionnaire (CHAQ) Disability Index Score at 12, 24, and 36 Months

    The CHAQ was developed to measure the physical functioning in children 6 months to 18 years of age. It consists of 54 questions related to the child's ability to perform various activities of daily living. Depending on the question asked, each question is scored either 0 to 3 based on the level of difficulty experienced by the child or 0-1 based on whether the child required assistance from another person or used an aid or other device. All CHAQ questions were scored and converted to a total index score ranging from 0-3, where higher scores indicate greater disability.

    Measured: Baseline and Month 12, 24, and 36

  9. Change From Baseline in Wong-Baker FACES Pain Rating Scale (WBFPRS) at 12, 24, and 36 Months

    The WBFPRS is a horizontal pain scale for children 3-18 years which consists of 6 faces that range from a smiling "no hurt" face with a score of 0 to a crying "hurts worst" face with a score of 10.

    Measured: Baseline and Month 12, 24, and 36

  10. Change From Baseline in Growth Velocity Z-score (Height) at 12, 24, and 36 Months

    Growth velocity was determined by calculating age-adjusted z-scores for height, weight, and body mass index (BMI). Z-scores represent the number of standard deviations from the reference population's mean, with 0 denoting the mean. Positive changes from baseline signify increased growth velocity.

    Measured: Baseline and Month 12, 24, and 36

  11. Change From Baseline in Growth Velocity Z-score (Weight) at 12, 24, and 36 Months

    Growth velocity was determined by calculating age-adjusted z-scores for height, weight, and BMI. Z-scores represent the number of standard deviations from the reference population's mean, with 0 denoting the mean. Positive changes from baseline signify increased growth velocity.

    Measured: Baseline and Month 12, 24, and 36

  12. Change From Baseline in Growth Velocity Z-score (BMI) at 12, 24, and 36 Months

    Growth velocity was determined by calculating age-adjusted z-scores for height, weight, and BMI. Z-scores represent the number of standard deviations from the reference population's mean, with 0 denoting the mean. Positive changes from baseline signify increased growth velocity.

    Measured: Baseline and Month 12, 24, and 36

  13. Mean Serum Concentration of Denosumab

    Measured: Day 1, Day 10, Day 30, Month 3, Month 6, Month 12, and Month 18

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted24 May 2017
  2. Study start7 May 2018
  3. Primary completion13 Dec 2021
  4. Study completion20 Dec 2023

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
24 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03164928
Sponsor's protocol ID
20140444
Other IDs
2016-003083-39, 2016
Status last verified
Jul 2024
First submitted
8 May 2017
Last updated
19 Jul 2024
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request

Publications

  • Storm NE, Kubik C, Priem G, Kim M, Lin TC, Lange J, O'Connell C, Glennane A, Bradbury BD, Taisey MJ. Case Study on the Use of Real-World Evidence in a Feasibility Assessment of a Randomized Controlled Trial Design to Support the Fulfillment of Pediatric Requirements with the FDA and EMA. Ther Innov Regul Sci. 2026 Jun 19. doi: 10.1007/s43441-026-00977-1. Online ahead of print.

    PubMed 42319705

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Denosumab in people with Evaluate the Safety and Efficacy of Denosumab in Pediatric Subjects With and Glucocorticoid-induced Osteoporosis, run by Amgen at 38 sites in 13 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 10 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 19 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 38 sites in United States, Peru, India, Russia, Turkey (Türkiye) and 8 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 19 Jul 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change From Baseline in Lumbar Spine BMD Z-score as Assessed by Dual-energy X-ray Absorptiometry (DXA) at 12 Months (time frame: Baseline and 12 Months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03164928, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Jul 2024.

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