RecruitingPhase 3NCT05972551

Study to Evaluate Efficacy and Safety of Romosozumab Compared With Bisphosphonates in Children and Adolescents With Osteogenesis Imperfecta

A Phase 3, Open-Label, Multicenter, Randomized Study to Evaluate Efficacy and Safety of Romosozumab Compared With Bisphosphonates in Children and Adolescents With Osteogenesis Imperfecta


Sponsor

Amgen

Enrollment

106 participants

Start Date

Apr 22, 2024

Study Type

INTERVENTIONAL

Conditions

Summary

The primary objective of this study is to evaluate the effect of romosozumab treatment for 12-months compared with bisphosphonate(s) on the number of clinical fractures at 12-months; the number of any fractures at 12-months and change in lumbar spine bone mineral density (BMD) Z-score at 6-months.


Eligibility

Min Age: 5 YearsMax Age: 17 Years

Inclusion Criteria10

  • Participant has provided informed consent/assent prior to initiation of any study specific activities/procedures.
  • OR
  • Participant's legally authorized representative has provided informed consent when the participant is legally too young to provide informed consent and the participant has provided written assent based on local regulations and/or guidelines prior to any study-specific activities/procedures being initiated.
  • Ambulatory male and female children and adolescents, age 5 to <18 years, including ambulatory with assistance as defined in the pediatric osteogenesis imperfecta (OI) population.
  • Clinical diagnosis of OI, defined as clinical history consistent with type I, III, or IV OI as determined by presence of expected phenotype (examples include: facial shape, voice, blue sclera, dentinogenesis imperfecta, typical radiographic features, fracture pattern) and lack of additional features unrelated to type I, III, or IV OI (eg, blindness, mental retardation, neuropathy, and craniosynostosis).
  • o If familial, also must be autosomal dominant.
  • Meets at least one of the following:
  • or more fractures within the previous 2 years, or
  • or more nonvertebral fracture(s) within the previous 2 years and at least 1 prevalent vertebral fracture, or
  • or more prevalent vertebral fractures.

Exclusion Criteria4

  • Disease Related
  • History of an electrophoresis pattern inconsistent with type I, III or IV OI.
  • History of known mutation in a gene other than collagen type I alpha 1/collagen type I alpha 2 (COL1A1/COL1A2) causing OI or other metabolic bone disease.
  • History of congenital dislocation of the radial head, interosseous membrane calcification, or exuberant callus formation.

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Interventions

DRUGRomosozumab

Subcutaneous (SC) injection

DRUGBisphosphonate

Administration determined by investigator according to the local standard of care


Locations(65)

Childrens Hospital of Alabama

Birmingham, Alabama, United States

Phoenix Childrens Hospital

Phoenix, Arizona, United States

University of California, Los Angeles Interventional Clinical Trials

Los Angeles, California, United States

Nemours Hospital for Children

Wilmington, Delaware, United States

Nemours Childrens Hospital

Orlando, Florida, United States

University of South Florida - Carol and Frank Morsani Center for Advanced Health Care

Tampa, Florida, United States

Emory University

Atlanta, Georgia, United States

Indiana University

Indianapolis, Indiana, United States

Kennedy Krieger Institute

Baltimore, Maryland, United States

Boston Childrens Hospital

Boston, Massachusetts, United States

Mayo Clinic Childrens Center

Rochester, Minnesota, United States

Gillette Childrens Hospital and Clinic Saint Paul

Saint Paul, Minnesota, United States

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Marshfield Clinic

Marshfield, Wisconsin, United States

Monash Childrens Hospital

Clayton, Victoria, Australia

Perth Childrens Hospital

Nedlands, Western Australia, Australia

Kepler Universitaetsklinikum GmbH

Linz, Austria

Universitair Ziekenhuis Leuven - Campus Gasthuisberg

Leuven, Belgium

Childrens Hospital of Eastern Ontario

Ottawa, Ontario, Canada

The Hospital for Sick Children

Toronto, Ontario, Canada

Centre Hospitalier Universitaire Sainte Justine

Montreal, Quebec, Canada

Peking Union Medical College Hospital

Beijing, Beijing Municipality, China

The University of Hong Kong-Shenzhen Hospital

Shenzhen, Guangdong, China

Tongji Hospital , Tongji Medical College, Huazhong University of Science and Technology

Wuhan, Hubei, China

Childrens Hospital of Soochow University

Suzhou, Jiangsu, China

The First Bethune Hospital of Jilin University

Changchun, Jilin, China

Shandong Provincial Hospital

Jinan, Shandong, China

West China Hospital Sichuan University

Chengdu, Sichuan, China

Shanghai Sixth Peoples Hospital

Shanghai, China

Centre Hospitalier Universitaire de Bordeaux - Hopital Pellegrin

Bordeaux, France

Centre Hospitalier Regional Universitaire de Lille

Lille, France

Hopital Necker Enfants Malades

Paris, France

Universitaetsklinikum Koeln

Cologne, Germany

Universitaetsklinikum Wuerzburg

Würzburg, Germany

Semmelweis Egyetem

Budapest, Hungary

IRCCS Istituto Giannina Gaslini

Genova, Italy

Centro Ricerche Cliniche Di Verona Societa responsabilita limitata

Verona, Italy

Okayama Saiseikai Outpatient Center Hospital

Okayama, Okayama-ken, Japan

Okayama University Hospital

Okayama, Okayama-ken, Japan

Osaka Womens and Childrens Hospital

Izumi-shi, Osaka, Japan

Tokyo Metropolitan Children's Medical Center

Fuchu-shi, Tokyo, Japan

National Center for Child Health and Development

Setagaya-ku, Tokyo, Japan

Tottori University Hospital

Yonago-shi, Tottori, Japan

Centermed Krakow Sp zoo

Krakow, Poland

Samodzielny Publiczny Zaklad Opieki Zdrowotnej Centralny Szpital Kliniczny UMeds

Lodz, Poland

Instytut Centrum Zdrowia Matki Polki

Lodz, Poland

Centrum Medyczne Medyk Spolka z Ograniczona Odpowiedzialnoscia Spolka Komandytowa

Rzeszów, Poland

Szpital Miejski w Tychach Spolka z ograniczona odpowiedzialnoscia

Tychy, Poland

King Faisal Specialist Hospital and Research Centre

Riyadh, Saudi Arabia

Narodny ustav detskych chorob

Bratislava, Slovakia

Hospital de Cruces

Barakaldo, Basque Country, Spain

Hospital Universitari Vall d Hebron

Barcelona, Catalonia, Spain

Hospital Sant Joan de Deu

Esplugues de Llobregat, Catalonia, Spain

Hospital Universitario de Getafe

Getafe, Madrid, Spain

Hospital Universitari i Politecnic La Fe

Valencia, Valencia, Spain

Hospital Universitario Infantil Niño Jesus

Madrid, Spain

Universitaets-Kinderspital beider Basel

Basel, Switzerland

Centre Hospitalier Universitaire Vaudois

Lausanne, Switzerland

Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi Gazi Hastanesi

Ankara, Turkey (Türkiye)

Marmara Universitesi Tip Fakultesi Hastanesi

Istanbul, Turkey (Türkiye)

Ege Universitesi Tip Fakultesi Hastanesi

Izmir, Turkey (Türkiye)

Karadeniz Teknik Universitesi Tip Fakultesi

Trabzon, Turkey (Türkiye)

Queen Elizabeth University Hospital

Glasgow, United Kingdom

Royal Manchester Childrens Hospital

Manchester, United Kingdom

Sheffield Childrens Hospital

Sheffield, United Kingdom

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