Clinical trial • Not applicable • Musculoskeletal|Dermatology|Rare Disease|Other

Romosozumab for Osteogenesis imperfecta

Not applicable trial of Romosozumab for Osteogenesis imperfecta. open-label. 39 participants.

Overview

Trial Therapeutic Area
Musculoskeletal|Dermatology|Rare Disease|Other
Trial Disease
Osteogenesis imperfecta
Trial Stage
Not applicable
Drug Modality
Monoclonal antibody
Paediatric Trial
Yes

Key dates

Initial CTIS Submission Date
15-10-2025
First CTIS Authorization Date
23-02-2026

Trial design

open-label Not applicable trial across 14 sites in Austria, Belgium, Germany and others.

Open Label
Yes
Target Sample Size
39
Trial Duration For Participant
749

Eligibility

Recruits 39 paediatric patients.

Vulnerable Population
Includes children and adolescents. If participant is legally too young to provide informed consent, the participant’s legally authorized representative must provide informed consent; the participant must provide written assent based on local regulations and/or guidelines prior to initiation of study-specific activities/procedures. Age-specific assent/ICF documents are provided (child age groups 5-7 and 8-11, adolescent, parental, adult) and country-specific informed consent/assent materials are included in the documentation.

Inclusion criteria

  • {"criterion_text":"-101 Participant has provided informed consent/assent prior to initiation of any study specific activities/procedures."}
  • {"criterion_text":"-101 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."}
  • {"criterion_text":"-102 Participant was randomized to the romosozumab arm and completed Study 20200105 through the Month 15 Visit, regardless of whether they received investigational product (romosozumab) until the last protocol-specified dose or ended investigational product early."}

Exclusion criteria

  • {"criterion_text":"-201 Currently receiving treatment in another investigational device or drug study, or less than 2 years since ending treatment on another investigational device or drug study(ies) with the exception of Study 20200105. Other investigational procedures while participating in this study are excluded."}
  • {"criterion_text":"-202 Participant likely to not be available to complete all protocol-required study visits or procedures, and/or to comply with all required study procedures to the best of the participant and investigator’s knowledge."}

Endpoints

Primary endpoints

  • {"endpoint_text":"-Adverse events","definition_or_measurement_approach":""}

Recruitment

Planned Sample Size
39
Recruitment Window Months
24
Consent Approach
Informed consent must be provided prior to any study-specific activities. If the participant is legally too young to provide consent, a legally authorized representative provides informed consent and the participant provides written assent per local regulations/guidelines. Age-specific ICF/assent documents are provided (documents for adults, parental ICF, adolescent ICF/assent, child assent for ages 5-7 and 8-11, and other child/adolescent materials). Consent/assent materials are available in multiple languages as provided in the documentation (including English, French, Dutch, German, Italian, Spanish, Hungarian, Slovak).

Geography

Total Number Of Sites
14
Total Number Of Participants
32

Austria

Earliest CTIS Part Ii Submission Date
14-01-2026
Latest Decision Or Authorization Date
23-02-2026
Processing Time Days
40
Number Of Sites
1
Number Of Participants
3

Sites

Site Name
Kepler Universitaetsklinikum GmbH
Department Name
Pediatrics and Adolescent Medicine
Principal Investigator Name
Wolfgang Högler
Principal Investigator Email
Wolfgang.Hoegler@kepleruniklinikum.at
Contact Person Name
Wolfgang Högler
Number Of Participants
3

Belgium

Earliest CTIS Part Ii Submission Date
26-01-2026
Latest Decision Or Authorization Date
27-02-2026
Processing Time Days
32
Number Of Sites
1
Number Of Participants
2

Sites

Site Name
UZ Leuven
Department Name
Pediatrics
Principal Investigator Name
Peter Witters
Principal Investigator Email
peter.witters@uzleuven.be
Contact Person Name
Peter Witters
Contact Person Email
peter.witters@uzleuven.be
Number Of Participants
2

Germany

Earliest CTIS Part Ii Submission Date
16-02-2026
Latest Decision Or Authorization Date
25-02-2026
Processing Time Days
9
Number Of Sites
2
Number Of Participants
12

Sites

Site Name
Universitaetsklinikum Wuerzburg AöR
Department Name
Muskuloskelettales Zentrum
Principal Investigator Name
Lothar Seefried
Principal Investigator Email
lothar.seefried@klh.de
Contact Person Name
Lothar Seefried
Contact Person Email
lothar.seefried@klh.de
Site Name
Universitaetsklinikum Koeln AöR
Department Name
Klinik und Poliklinik fuer Kinder- und Jugendmedizin
Principal Investigator Name
Heike-Katharina Hoyer-Kuhn
Principal Investigator Email
heike-katharina.hoyer-kuhn@uk-koeln.de
Contact Person Name
Heike-Katharina Hoyer-Kuhn

Hungary

Earliest CTIS Part Ii Submission Date
10-12-2025
Latest Decision Or Authorization Date
26-02-2026
Processing Time Days
78
Number Of Sites
1
Number Of Participants
5

Sites

Site Name
Semmelweis University
Department Name
Gyermekgyogyaszati Klinika Tuzolto utcai reszleg
Principal Investigator Name
Eva Hosszu
Principal Investigator Email
hosszu.eva@semmelweis.hu
Contact Person Name
Eva Hosszu
Contact Person Email
hosszu.eva@semmelweis.hu
Number Of Participants
5

Italy

Earliest CTIS Part Ii Submission Date
20-01-2026
Latest Decision Or Authorization Date
02-03-2026
Processing Time Days
41
Number Of Sites
3
Number Of Participants
3

Sites

Site Name
Ospedale Pediatrico Bambino Gesu
Department Name
University Hospital Pediatric Department
Principal Investigator Name
Michaela Veronika Gonfiantini
Principal Investigator Email
mveronik.gonfiantini@opbg.net
Contact Person Name
Michaela Veronika Gonfiantini
Contact Person Email
mveronik.gonfiantini@opbg.net
Site Name
Centro Ricerche Cliniche Di Verona S.r.l.
Department Name
Clinical Pharmacology Unit
Principal Investigator Name
Franco Antoniazzi
Principal Investigator Email
franco.antoniazzi@univr.it
Contact Person Name
Franco Antoniazzi
Contact Person Email
franco.antoniazzi@univr.it
Site Name
IRCCS Istituto Giannina Gaslini
Department Name
Complex Operating Unit for Pediatrics and Endocrinology
Principal Investigator Name
Natascia Di Iorgi
Principal Investigator Email
natasciadiiorgi@gaslini.org
Contact Person Name
Natascia Di Iorgi
Contact Person Email
natasciadiiorgi@gaslini.org

Spain

Earliest CTIS Part Ii Submission Date
04-02-2026
Latest Decision Or Authorization Date
02-03-2026
Processing Time Days
26
Number Of Sites
5
Number Of Participants
5

Sites

Site Name
Hospital Universitario De Getafe
Department Name
Pediatrics
Principal Investigator Name
Nuria Lopez Barrena
Principal Investigator Email
nlbarrena@hotmail.com
Contact Person Name
Nuria Lopez Barrena
Contact Person Email
nlbarrena@hotmail.com
Site Name
Hospital Universitario De Cruces
Department Name
Pediatric Endocrinology
Principal Investigator Name
Gema Grau Bolado
Principal Investigator Email
MARIAGEMA.GRAUBOLADO@osakidetza.eus
Contact Person Name
Gema Grau Bolado
Site Name
Hospital Sant Joan De Deu Barcelona
Department Name
Pediatric Rheumatology
Principal Investigator Name
Rosa Bou Torrent
Principal Investigator Email
rosa.bou@sjd.es
Contact Person Name
Rosa Bou Torrent
Contact Person Email
rosa.bou@sjd.es
Site Name
Hospital Universitari Vall D Hebron
Department Name
Pediatric Rheumatology
Principal Investigator Name
Ariadna Campos Martorell
Principal Investigator Email
ariadna.campos@vallhebron.cat
Contact Person Name
Ariadna Campos Martorell
Contact Person Email
ariadna.campos@vallhebron.cat
Site Name
Hospital Universitario Y Politecnico La Fe
Department Name
Pediatric Rheumatology
Principal Investigator Name
Lucia Lacruz Perez
Principal Investigator Email
lacruz_lucper@gva.es
Contact Person Name
Lucia Lacruz Perez
Contact Person Email
lacruz_lucper@gva.es

Slovakia

Earliest CTIS Part Ii Submission Date
19-02-2026
Latest Decision Or Authorization Date
24-02-2026
Processing Time Days
5
Number Of Sites
1
Number Of Participants
2

Sites

Site Name
Narodny Ustav Detskych Chorob
Department Name
Detska klinika LF UK a NUDCH
Principal Investigator Name
Lubica Ticha
Principal Investigator Email
lubica.ticha@nudch.eu
Contact Person Name
Lubica Ticha
Contact Person Email
lubica.ticha@nudch.eu
Number Of Participants
2

Sponsor

Primary sponsor

Full Name
Amgen Inc.
Organisation Type
Pharmaceutical company
Country Of Registered Address
United States

Contract research organisations

Name
Bioclinica Inc.
Responsibilities
Imaging
Name
Q Squared Solutions Limited

Third parties

  • {"country":"United States","full_name":"Bioclinica Inc.","duties_or_roles":"Imaging","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United Kingdom","full_name":"Q Squared Solutions Limited","duties_or_roles":"","organisation_type":"Non-Pharmaceutical company"}

Investigational products

Investigational Product Name
EVENITY 105 mg solution for injection in pre-filled syringe
Active Substance
Romosozumab
Modality
Monoclonal antibody
Routes Of Administration
SUBCUTANEOUS INJECTION
Route
SUBCUTANEOUS INJECTION
Authorisation Status
Authorised
Starting Dose
105 mg
Dose Levels
105 mg
Investigational Product Name
EVENITY 105 mg solution for injection in pre-filled syringe
Active Substance
Romosozumab
Modality
Monoclonal antibody
Routes Of Administration
SUBCUTANEOUS INJECTION
Route
SUBCUTANEOUS INJECTION
Authorisation Status
Authorised
Starting Dose
105 mg
Dose Levels
105 mg
Investigational Product Name
EVENITY 105 mg solution for injection in pre-filled pen
Active Substance
Romosozumab
Modality
Monoclonal antibody
Routes Of Administration
SUBCUTANEOUS INJECTION
Route
SUBCUTANEOUS INJECTION
Authorisation Status
Authorised
Starting Dose
105 mg
Dose Levels
105 mg
Investigational Product Name
EVENITY 105 mg solution for injection in pre-filled pen
Active Substance
Romosozumab
Modality
Monoclonal antibody
Routes Of Administration
SUBCUTANEOUS INJECTION
Route
SUBCUTANEOUS INJECTION
Authorisation Status
Authorised
Starting Dose
105 mg
Dose Levels
105 mg

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