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

NCT05972551 | Recruiting Phase 3 DMC FDA Drug

• 2 other identifiers: 202001052023-503294-37
• Study Type: interventional
• Target: 106
• Locations: 17 countries
• Sites: 63

Brief 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.

Conditions

Osteogenesis Imperfecta

Keywords

Osteogenesis Imperfecta; Romosozumab; EVENITY®; Bisphosphonates

Outcome Measures

Primary Outcomes (3)

• Number of Clinical Fractures

  Clinical fractures include clinical vertebral fractures and nonvertebral fractures.

  12 months

• Number of Any Fractures

  Fractures include new and worsening vertebral compression fractures, whether clinically silent or manifest, and nonvertebral fractures.

  12 months

• Change from Baseline in Lumbar Spine BMD Z-score at 12 Months, as assessed by DXA

  Baseline and 12 months

Secondary Outcomes (21)

• Change from Baseline in lumbar spine BMD Z-score at 6 months and 12 months, as assessed by DXA

  Baseline, 6 months, and 12 months

• Change from Baseline in Total Hip BMD Z-score at 6 Months and at 12 Months, as assessed by DXA

  Baseline, 6 months, and 12 months

• Change from Baseline in Femoral Neck BMD Z-score at 6 Months and at 12 Months, as assessed by DXA

  Baseline, 6 months, and 12 months

• Number of Participants with Any Fractures

  12 months

• Number of Participants with Clinical Fractures

  12 months

Study Arms (2)

Romosozumab

EXPERIMENTAL

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

Drug: Romosozumab

Standard of Care Bisphosphonate

ACTIVE COMPARATOR

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

Drug: Bisphosphonate

Interventions

Romosozumab DRUG

Subcutaneous (SC) injection

Also known as: EVENITY®

Romosozumab

Bisphosphonate DRUG

Administration determined by investigator according to the local standard of care

Standard of Care Bisphosphonate

Eligibility Criteria

• Age: 5 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Participant has provided informed consent/assent prior to initiation of any study specific activities/procedures.
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Amgen: lead

Study Sites (65)

Childrens Hospital of Alabama

Birmingham, Alabama, 35233, United States

TERMINATED

Phoenix Childrens Hospital

Phoenix, Arizona, 85016, United States

RECRUITING

University of California, Los Angeles Interventional Clinical Trials

Los Angeles, California, 90095, United States

RECRUITING

Nemours Hospital for Children

Wilmington, Delaware, 19803, United States

RECRUITING

Nemours Childrens Hospital

Orlando, Florida, 32827, United States

RECRUITING

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

Tampa, Florida, 33606, United States

RECRUITING

Emory University

Atlanta, Georgia, 30322, United States

TERMINATED

Indiana University

Indianapolis, Indiana, 46202, United States

RECRUITING

Kennedy Krieger Institute

Baltimore, Maryland, 21287, United States

RECRUITING

Boston Childrens Hospital

Boston, Massachusetts, 02115, United States

RECRUITING

Mayo Clinic Childrens Center

Rochester, Minnesota, 55905, United States

TERMINATED

Gillette Childrens Hospital and Clinic Saint Paul

Saint Paul, Minnesota, 55101, United States

RECRUITING

Vanderbilt University Medical Center

Nashville, Tennessee, 37212-3157, United States

RECRUITING

Marshfield Clinic

Marshfield, Wisconsin, 54449, United States

RECRUITING

Monash Childrens Hospital

Clayton, Victoria, 3168, Australia

RECRUITING

Perth Childrens Hospital

Nedlands, Western Australia, 6909, Australia

RECRUITING

Kepler Universitaetsklinikum GmbH

Linz, 4020, Austria

RECRUITING

Universitair Ziekenhuis Leuven - Campus Gasthuisberg

Leuven, 3000, Belgium

RECRUITING

Childrens Hospital of Eastern Ontario

Ottawa, Ontario, K1H 8L1, Canada

RECRUITING

The Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

RECRUITING

Centre Hospitalier Universitaire Sainte Justine

Montreal, Quebec, H3T 1C5, Canada

RECRUITING

Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100730, China

RECRUITING

The University of Hong Kong-Shenzhen Hospital

Shenzhen, Guangdong, 518053, China

RECRUITING

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

Wuhan, Hubei, 430030, China

RECRUITING

Childrens Hospital of Soochow University

Suzhou, Jiangsu, 215000, China

RECRUITING

The First Bethune Hospital of Jilin University

Changchun, Jilin, 130021, China

RECRUITING

Shandong Provincial Hospital

Jinan, Shandong, 250021, China

RECRUITING

West China Hospital Sichuan University

Chengdu, Sichuan, 610044, China

RECRUITING

Shanghai Sixth Peoples Hospital

Shanghai, 200233, China

RECRUITING

Centre Hospitalier Universitaire de Bordeaux - Hopital Pellegrin

Bordeaux, 33076, France

RECRUITING

Centre Hospitalier Regional Universitaire de Lille

Lille, 59037, France

RECRUITING

Hopital Necker Enfants Malades

Paris, 75743, France

RECRUITING

Universitaetsklinikum Koeln

Cologne, 50937, Germany

RECRUITING

Universitaetsklinikum Wuerzburg

Würzburg, 97074, Germany

RECRUITING

Semmelweis Egyetem

Budapest, 1094, Hungary

RECRUITING

IRCCS Istituto Giannina Gaslini

Genova, 16147, Italy

RECRUITING

Centro Ricerche Cliniche Di Verona Societa responsabilita limitata

Verona, 37134, Italy

RECRUITING

Okayama Saiseikai Outpatient Center Hospital

Okayama, Okayama-ken, 700-0013, Japan

RECRUITING

Okayama University Hospital

Okayama, Okayama-ken, 700-8558, Japan

RECRUITING

Osaka Womens and Childrens Hospital

Izumi-shi, Osaka, 594-1101, Japan

RECRUITING

Tokyo Metropolitan Children's Medical Center

Fuchu-shi, Tokyo, 183-8561, Japan

RECRUITING

National Center for Child Health and Development

Setagaya-ku, Tokyo, 157-8535, Japan

RECRUITING

Tottori University Hospital

Yonago-shi, Tottori, 683-8504, Japan

RECRUITING

Centermed Krakow Sp zoo

Krakow, 31-530, Poland

RECRUITING

Samodzielny Publiczny Zaklad Opieki Zdrowotnej Centralny Szpital Kliniczny UMeds

Lodz, 91-738, Poland

RECRUITING

Instytut Centrum Zdrowia Matki Polki

Lodz, 93-338, Poland

RECRUITING

Centrum Medyczne Medyk Spolka z Ograniczona Odpowiedzialnoscia Spolka Komandytowa

Rzeszów, 35-326, Poland

RECRUITING

Szpital Miejski w Tychach Spolka z ograniczona odpowiedzialnoscia

Tychy, 43-100, Poland

RECRUITING

King Faisal Specialist Hospital and Research Centre

Riyadh, 11564, Saudi Arabia

RECRUITING

Narodny ustav detskych chorob

Bratislava, 833 40, Slovakia

RECRUITING

Hospital de Cruces

Barakaldo, Basque Country, 48903, Spain

RECRUITING

Hospital Universitari Vall d Hebron

Barcelona, Catalonia, 08035, Spain

RECRUITING

Hospital Sant Joan de Deu

Esplugues de Llobregat, Catalonia, 08950, Spain

RECRUITING

Hospital Universitario de Getafe

Getafe, Madrid, 28905, Spain

RECRUITING

Hospital Universitari i Politecnic La Fe

Valencia, Valencia, 46026, Spain

RECRUITING

Hospital Universitario Infantil Niño Jesus

Madrid, 28009, Spain

RECRUITING

Universitaets-Kinderspital beider Basel

Basel, 4031, Switzerland

RECRUITING

Centre Hospitalier Universitaire Vaudois

Lausanne, 1011, Switzerland

RECRUITING

Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi Gazi Hastanesi

Ankara, 06500, Turkey (Türkiye)

RECRUITING

Marmara Universitesi Tip Fakultesi Hastanesi

Istanbul, 34890, Turkey (Türkiye)

RECRUITING

Ege Universitesi Tip Fakultesi Hastanesi

Izmir, 35100, Turkey (Türkiye)

RECRUITING

Karadeniz Teknik Universitesi Tip Fakultesi

Trabzon, 61080, Turkey (Türkiye)

RECRUITING

Queen Elizabeth University Hospital

Glasgow, G51 4TF, United Kingdom

RECRUITING

Royal Manchester Childrens Hospital

Manchester, M13 9WL, United Kingdom

RECRUITING

Sheffield Childrens Hospital

Sheffield, S10 2TH, United Kingdom

RECRUITING

Related Links

• AmgenTrials clinical trials website

MeSH Terms

Conditions

Osteogenesis Imperfecta

Interventions

romosozumab; Diphosphonates

Condition Hierarchy (Ancestors)

Osteochondrodysplasias; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Collagen Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases

Intervention Hierarchy (Ancestors)

Organophosphonates; Organophosphorus Compounds; Organic Chemicals

Study Officials

• MD

  Amgen

  STUDY DIRECTOR

Central Study Contacts

Amgen Call Center

CONTACT

866-572-6436; medinfo@amgen.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 24, 2023
• First Posted: August 2, 2023
• Study Start: April 22, 2024
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): August 25, 2027
• Last Updated: May 1, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Time Frame: Data sharing requests relating to this study will be considered beginning 18 months after the study has ended and either 1) the product and indication have been granted marketing authorization in both the US and Europe or 2) clinical development for the product and/or indication discontinues and the data will not be submitted to regulatory authorities. There is no end date for eligibility to submit a data sharing request for this study.
• Access Criteria: Qualified researchers may submit a request containing the research objectives, the Amgen product(s) and Amgen study/studies in scope, endpoints/outcomes of interest, statistical analysis plan, data requirements, publication plan, and qualifications of the researcher(s). In general, Amgen does not grant external requests for individual patient data for the purpose of re-evaluating safety and efficacy issues already addressed in the product labelling. Requests are reviewed by a committee of internal advisors. If not approved, a Data Sharing Independent Review Panel will arbitrate and make the final decision. Upon approval, information necessary to address the research question will be provided under the terms of a data sharing agreement. This may include anonymized individual patient data and/or available supporting documents, containing fragments of analysis code where provided in analysis specifications. Further details are available at the URL below.

Locations

SL (1); ES (6); JP (6); DE (2); IT (2); TU (4); CA (3); PL (5); CH (2); BE (1); CN (8); HU (1); FR (3); US (14); GB (3); SA (1); AU (1)