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

NCT ID: NCT05972551

Last Updated: 2025-11-13

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

106 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-04-22

Study Completion Date

2027-08-25

Brief Summary

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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.

Detailed Description

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Conditions

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Osteogenesis Imperfecta

Keywords

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Osteogenesis Imperfecta Romosozumab EVENITY® Bisphosphonates

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Romosozumab

Participants will receive romosozumab once a month (QM) for 12 months.

Group Type EXPERIMENTAL

Romosozumab

Intervention Type DRUG

Subcutaneous (SC) injection

Standard of Care Bisphosphonate

Participants will receive bisphosphonates per local standard of care treatment regimens, as determined by the investigator for 12 months.

Group Type ACTIVE_COMPARATOR

Bisphosphonate

Intervention Type DRUG

Administration determined by investigator according to the local standard of care

Interventions

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Romosozumab

Subcutaneous (SC) injection

Intervention Type DRUG

Bisphosphonate

Administration determined by investigator according to the local standard of care

Intervention Type DRUG

Other Intervention Names

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EVENITY®

Eligibility Criteria

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Inclusion Criteria

* 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:

* 3 or more fractures within the previous 2 years, or
* 1 or more nonvertebral fracture(s) within the previous 2 years and at least 1 prevalent vertebral fracture, or
* 2 or more prevalent vertebral fractures.

Exclusion Criteria

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.
Minimum Eligible Age

5 Years

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Amgen

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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MD

Role: STUDY_DIRECTOR

Amgen

Locations

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Childrens Hospital of Alabama

Birmingham, Alabama, United States

Site Status RECRUITING

Phoenix Childrens Hospital

Phoenix, Arizona, United States

Site Status RECRUITING

Nemours Hospital for Children

Wilmington, Delaware, United States

Site Status RECRUITING

Nemours Childrens Hospital

Orlando, Florida, United States

Site Status RECRUITING

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

Tampa, Florida, United States

Site Status RECRUITING

Emory University

Atlanta, Georgia, United States

Site Status RECRUITING

Indiana University

Indianapolis, Indiana, United States

Site Status RECRUITING

Kennedy Krieger Institute

Baltimore, Maryland, United States

Site Status RECRUITING

Boston Childrens Hospital

Boston, Massachusetts, United States

Site Status RECRUITING

Mayo Clinic Childrens Center

Rochester, Minnesota, United States

Site Status TERMINATED

Gillette Childrens Hospital and Clinic Saint Paul

Saint Paul, Minnesota, United States

Site Status RECRUITING

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status RECRUITING

Monash Childrens Hospital

Clayton, Victoria, Australia

Site Status RECRUITING

Perth Childrens Hospital

Nedlands, Western Australia, Australia

Site Status RECRUITING

Kepler Universitaetsklinikum GmbH

Linz, , Austria

Site Status RECRUITING

Universitair Ziekenhuis Leuven - Campus Gasthuisberg

Leuven, , Belgium

Site Status RECRUITING

Childrens Hospital of Eastern Ontario

Ottawa, Ontario, Canada

Site Status RECRUITING

The Hospital for Sick Children

Toronto, Ontario, Canada

Site Status RECRUITING

Centre Hospitalier Universitaire Sainte Justine

Montreal, Quebec, Canada

Site Status RECRUITING

Peking Union Medical College Hospital

Beijing, Beijing Municipality, China

Site Status RECRUITING

The University of Hong Kong-Shenzhen Hospital

Shenzhen, Guangdong, China

Site Status RECRUITING

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

Wuhan, Hubei, China

Site Status RECRUITING

The First Bethune Hospital of Jilin University

Changchun, Jilin, China

Site Status RECRUITING

West China Hospital of Sichuan University

Chengdu, Sichuan, China

Site Status RECRUITING

Shanghai Sixth Peoples Hospital

Shanghai, , China

Site Status RECRUITING

Centre Hospitalier Universitaire de Bordeaux - Hopital Pellegrin

Bordeaux, , France

Site Status RECRUITING

Hopital Necker Enfants Malades

Paris, , France

Site Status RECRUITING

Universitaetsklinikum Koeln

Cologne, , Germany

Site Status RECRUITING

Universitaetsklinikum Wuerzburg

Würzburg, , Germany

Site Status RECRUITING

Semmelweis Egyetem

Budapest, , Hungary

Site Status RECRUITING

Okayama Saiseikai Outpatient Center Hospital

Okayama, Okayama-ken, Japan

Site Status RECRUITING

Okayama University Hospital

Okayama, Okayama-ken, Japan

Site Status RECRUITING

Osaka Womens and Childrens Hospital

Izumi-shi, Osaka, Japan

Site Status RECRUITING

National Center for Child Health and Development

Setagaya-ku, Tokyo, Japan

Site Status RECRUITING

Centermed Krakow Sp zoo

Krakow, , Poland

Site Status RECRUITING

Samodzielny Publiczny Zaklad Opieki Zdrowotnej Centralny Szpital Kliniczny UMeds

Lodz, , Poland

Site Status RECRUITING

Instytut Centrum Zdrowia Matki Polki

Lodz, , Poland

Site Status RECRUITING

Szpital Miejski w Tychach Spolka z ograniczona odpowiedzialnoscia

Tychy, , Poland

Site Status RECRUITING

Narodny ustav detskych chorob

Bratislava, , Slovakia

Site Status RECRUITING

Hospital de Cruces

Barakaldo, Basque Country, Spain

Site Status RECRUITING

Hospital Universitari Vall d Hebron

Barcelona, Catalonia, Spain

Site Status RECRUITING

Hospital Sant Joan de Deu

Esplugues de Llorbregat, Catalonia, Spain

Site Status RECRUITING

Hospital Universitario de Getafe

Getafe, Madrid, Spain

Site Status RECRUITING

Hospital Universitari i Politecnic La Fe

Valencia, Valencia, Spain

Site Status RECRUITING

Hospital Universitario Infantil Niño Jesus

Madrid, , Spain

Site Status RECRUITING

Universitaets-Kinderspital beider Basel

Basel, , Switzerland

Site Status RECRUITING

Centre Hospitalier Universitaire Vaudois

Lausanne, , Switzerland

Site Status RECRUITING

Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi Gazi Hastanesi

Ankara, , Turkey (Türkiye)

Site Status RECRUITING

Marmara Universitesi Tip Fakultesi Hastanesi

Istanbul, , Turkey (Türkiye)

Site Status RECRUITING

Ege Universitesi Tip Fakultesi Hastanesi

Izmir, , Turkey (Türkiye)

Site Status RECRUITING

Karadeniz Teknik Universitesi Tip Fakultesi

Trabzon, , Turkey (Türkiye)

Site Status RECRUITING

Queen Elizabeth University Hospital

Glasgow, , United Kingdom

Site Status RECRUITING

Royal Manchester Childrens Hospital

Manchester, , United Kingdom

Site Status RECRUITING

Countries

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United States Australia Austria Belgium Canada China France Germany Hungary Japan Poland Slovakia Spain Switzerland Turkey (Türkiye) United Kingdom

Central Contacts

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Amgen Call Center

Role: CONTACT

Phone: 866-572-6436

Email: [email protected]

Related Links

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http://www.amgentrials.com

AmgenTrials clinical trials website

Other Identifiers

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2023-503294-37

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

20200105

Identifier Type: -

Identifier Source: org_study_id