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

NCT ID: NCT03164928

Last Updated: 2024-07-19

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 24 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-05-07
• Study Completion Date: 2023-12-20

Brief 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).

Conditions

Evaluate the Safety and Efficacy of Denosumab in Pediatric Subjects With; Glucocorticoid-induced Osteoporosis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Placebo

SC Q6M placebo

Group Type: OTHER

Placebo

Intervention Type: OTHER

SC Q6M placebo

Denosumab

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

Group Type: EXPERIMENTAL

Denosumab

Intervention Type: DRUG

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

Interventions

Denosumab

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

Intervention Type: DRUG

Placebo

SC Q6M placebo

Intervention Type: OTHER

Eligibility Criteria

Inclusion Criteria

• 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
• 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

Exclusion Criteria

• 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
• 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

• Minimum Eligible Age: 5 Years
• Maximum Eligible Age: 17 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Amgen

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

MD

Role: STUDY_DIRECTOR

Amgen

Locations

Childrens Hospital of Los Angeles

Los Angeles, California, United States

AI Dupont Hospital for Children

Wilmington, Delaware, United States

Indiana University Hospital

Indianapolis, Indiana, United States

University of Minnesota Masonic Childrens Hospital Discovery Clinic

Minneapolis, Minnesota, United States

Metrohealth Medical Center

Cleveland, Ohio, United States

Nationwide Childrens Hospital

Columbus, Ohio, United States

Perth Childrens Hospital

Nedlands, Western Australia, Australia

Cliniques Universtaire Saint Luc Universite Catholique de Louvain

Brussels, , Belgium

Medical Centre Synexus Sofia EOOD

Sofia, , Bulgaria

Childrens Hospital of Eastern Ontario

Ottawa, Ontario, Canada

Centre Hospitalier Universitaire Sainte Justine

Montreal, Quebec, Canada

Center for Clinical and Basic Research Colombia

Medellín, Antioquia, Colombia

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

Bogota, Cundinamarca, Colombia

Foscal Internacional-Fundacion Oftalmologica de Santander

Floridablanca, Santander Department, Colombia

Gandhi Medical College

Hyderabad, Andhra Pradesh, India

KLES Dr Prabhakar Kore Hospital and Medical Research Centre

Belagavi, Karnataka, India

Sir Ganga Ram Hospital

New Delhi, National Capital Territory of Delhi, India

Christian Medical College

Vellore, Tamil Nadu, India

Azienda Ospedaliera Universitaria Meyer

Florence, , Italy

Istituto Auxologico Italiano Istituto di Ricovero e Cura a Carattere Scientifico

Milan, , Italy

Azienda Ospedaliera Policlinico Umberto I

Roma, , Italy

RM Pharma Specialists SA de CV

Mexico City, , Mexico

Instituto Nacional de Salud del Nino

Breña, Lima region, Peru

Centro Especializado de Enfermedades Neoplasicas

Arequipa, , Peru

Hospital Nacional Alberto Sabogal Sologuren

Callao, , Peru

Clinica Angloamericana

Lima, , Peru

Centro de Investigacion Ricardo Palma

Lima, , Peru

FSAI Scientific Center of Childrens Health of MoH of the RF

Moscow, , Russia

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

Moscow, , Russia

SBHI of Novosibirsk region City Pediatric Clinical Hospital of Emergency Care

Novosibirsk, , Russia

LLC Medical Technologies

Saint Petersburg, , Russia

Ankara Universitesi Tip Fakultesi

Ankara, , Turkey (Türkiye)

Ataturk Universitesi Tip Fakultesi

Erzurum, , Turkey (Türkiye)

Marmara Universitesi Pendik Egitim Arastirma Hastanesi

Istanbul, , Turkey (Türkiye)

Ege Universitesi Tip Fakultesi

Izmir, , Turkey (Türkiye)

CI Dnipropetrovsk Regional Children Clinical Hospital of Dnipropetrovsk Regional Council

Dnipro, , Ukraine

Communal Institution of Healthcare Kharkiv City Clinical Children Hospital 16

Kharkiv, , Ukraine

National Childrens Specialized Hospital OHMATDYT

Kyiv, , Ukraine

Countries

United States; Australia; Belgium; Bulgaria; Canada; Colombia; India; Italy; Mexico; Peru; Russia; Turkey (Türkiye); Ukraine

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

http://www.amgentrials.com

AmgenTrials clinical trials website

Other Identifiers

2016-003083-39

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

20140444

Identifier Type: -

Identifier Source: org_study_id