Overview
Sponsor-declared trial summary
Phase
Therapeutic confirmatory (Phase III)
Status
Authorised, recruiting
Participants planned
71
Countries
7
Sites
14
Osteogenesis Imperfecta
To evaluate the safety of romosozumab in participants with osteogenesis imperfecta (OI) that have completed Study 20200105, regardless of whether they received investigational product (romosozumab) until the last protocolspecified dose or ended investigational product early
Key facts
- Sponsor
- Amgen Inc.
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16], Diseases [C] - Skin and Connective Tissue Diseases [C17], Diseases [C] - Musculoskeletal Diseases [C05]
- Trial duration
- 25 Mar 2026 → ongoing
- Decision date (initial)
- 2026-02-23
- Transition trial
- No
- Low-intervention
- Yes
- Rare-disease indication
- Yes
- Vulnerable population
- Yes
- Funding sources
- Amgen Inc.
External identifiers
- EU CT number
- 2023-503293-21-00
- WHO UTN
- U1111-1327-7190
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Safety
To evaluate the safety of romosozumab in participants with osteogenesis imperfecta (OI) that have completed Study 20200105, regardless of whether they received investigational product (romosozumab) until the last protocolspecified dose or ended investigational product early
Conditions and MedDRA coding
Osteogenesis Imperfecta
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.0 | PT | 10031243 | Osteogenesis imperfecta | 100000004850 |
Study design 1 period
| # | Title | Allocation | Blinding | Roles blinded | Arms |
|---|---|---|---|---|---|
| 1 | Safety follow-up period N/A
|
Not Applicable | None |
Regulatory references
- Scientific advice from competent authorities
- European Medicines Agency
- EMA paediatric investigation plan (PIP)
- EMEA-001075-PIP04-15
- Plan to share IPD
- No
| EU CT number | Title | Sponsor |
|---|---|---|
| 2023-503294-37-00 | A Phase 3, Open-Label, Multicenter, Randomized Study to evaluate the Efficacy and Safety of Romosozumab Compared with Bisphophonates in Children and Adolecents with Osteogenesis Imperfecta | Amgen Inc. |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 3
- 101 Participant has provided informed consent/assent prior to initiation of any study specific activities/procedures.
- 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.
- 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 2
- 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.
- 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 and secondary outcome measures (English text)
Primary endpoints 1
- Adverse events
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 4
EVENITY 105 mg solution for injection in pre-filled pen
PRD7764042 · Product
- Active substance
- Romosozumab
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS INJECTION
- Max daily dose
- 00 mg milligram(s)
- Max total dose
- 00 mg milligram(s)
- Max treatment duration
- 9999 Day(s)
- Authorisation status
- Authorised
- ATC code
- M05BX06 — -
- Marketing authorisation
- EU/1/19/1411/001
- MA holder
- UCB PHARMA S.A.
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
EVENITY 105 mg solution for injection in pre-filled pen
PRD7764048 · Product
- Active substance
- Romosozumab
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS INJECTION
- Max daily dose
- 00 mg milligram(s)
- Max total dose
- 00 mg milligram(s)
- Max treatment duration
- 9999 Week(s)
- Authorisation status
- Authorised
- ATC code
- M05BX06 — -
- Marketing authorisation
- EU/1/19/1411/002
- MA holder
- UCB PHARMA S.A.
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
EVENITY 105 mg solution for injection in pre-filled syringe
PRD7764013 · Product
- Active substance
- Romosozumab
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS INJECTION
- Max daily dose
- 00 mg milligram(s)
- Max total dose
- 00 mg milligram(s)
- Max treatment duration
- 9999 Week(s)
- Authorisation status
- Authorised
- ATC code
- M05BX06 — -
- Marketing authorisation
- EU/1/19/1411/004
- MA holder
- UCB PHARMA S.A.
- MA country
- EU
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
EVENITY 105 mg solution for injection in pre-filled syringe
PRD7763545 · Product
- Active substance
- Romosozumab
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- SUBCUTANEOUS INJECTION
- Max daily dose
- 00 mg milligram(s)
- Max total dose
- 00 mg milligram(s)
- Max treatment duration
- 9999 Day(s)
- Authorisation status
- Authorised
- ATC code
- M05BX06 — -
- Marketing authorisation
- EU/1/19/1411/003
- MA holder
- UCB PHARMA S.A.
- MA country
- EU
- Paediatric formulation
- Yes
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Amgen Inc.
- Sponsor organisation
- Amgen Inc.
- Address
- 1 Amgen Center Drive
- City
- Thousand Oaks
- Postcode
- 91320-1799
- Country
- United States
Scientific contact point
- Organisation
- Amgen Inc.
- Contact name
- Medical Information
Public contact point
- Organisation
- Amgen Inc.
- Contact name
- Medical Information
Third parties 2
| Organisation | City, country | Duties |
|---|---|---|
| Bioclinica Inc. ORG-100033079
|
Philadelphia, United States | Other |
| Q Squared Solutions Limited ORG-100042527
|
Reading, United Kingdom | Laboratory analysis |
Locations
7 EU/EEA countries · 14 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Austria | Authorised, recruitment pending | 3 | 1 |
| Belgium | Authorised, recruitment pending | 2 | 1 |
| Germany | Ongoing, recruiting | 12 | 2 |
| Hungary | Authorised, recruitment pending | 5 | 1 |
| Italy | Authorised, recruitment pending | 3 | 3 |
| Slovakia | Authorised, recruitment pending | 2 | 1 |
| Spain | Authorised, recruitment pending | 5 | 5 |
| Rest of world
United Kingdom, Canada, China, Australia, Switzerland, Turkey, Japan
|
— | 39 | — |
Investigational sites
Kepler Universitaetsklinikum GmbH
Pediatrics and Adolescent Medicine, Krankenhausstrasse 26-30, 4020, Linz
Universitaetsklinikum Wuerzburg AöR
Muskuloskelettales Zentrum, Brettreichstrasse 11, Frauenland, Wuerzburg
Universitaetsklinikum Koeln AöR
Klinik und Poliklinik fuer Kinder- und Jugendmedizin, Kerpener Strasse 62, Lindenthal, Cologne
Semmelweis University
Gyermekgyogyaszati Klinika Tuzolto utcai reszleg, Tuzolto Utca 7-9, 1094, Budapest
Centro Ricerche Cliniche Di Verona S.r.l.
Clinical Pharmacology Unit, Piazzale Ludovico Antonio Scuro 10, 37134, Verona
IRCCS Istituto Giannina Gaslini
Complex Operating Unit for Pediatrics and Endocrinology, Via Gerolamo Gaslini 5, 16147, Genoa
Ospedale Pediatrico Bambino Gesu
University Hospital Pediatric Department, Piazza Di Sant'onofrio 4, 00165, Rome
Narodny Ustav Detskych Chorob
Detska klinika LF UK a NUDCH, Limbova 1, 833 40, Bratislava
Hospital Universitario De Cruces
Pediatric Endocrinology, Cruces Plaza S/n, 48903, Barakaldo
Hospital Universitari Vall D Hebron
Pediatric Rheumatology, Passeig De La Vall D'Hebron 119-129, 08035, Barcelona
Hospital Universitario Y Politecnico La Fe
Pediatric Rheumatology, Avenida Fernando Abril Martorell 106, 46026, Valencia
Hospital Universitario De Getafe
Pediatrics, Carretera De Madrid Toledo Km 12,500, 28905, Getafe
Hospital Sant Joan De Deu Barcelona
Pediatric Rheumatology, Passeig De Sant Joan De Deu 2, 08950, Esplugues De Llobregat
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Germany | 2026-03-25 | 2026-05-06 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 93 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol_ENG_2023-503293-21_20220043_For Publication | 2 |
| Recruitment arrangements (for publication) | K1_recruitment arrangement_For publication | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_FP | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_FP | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements_Germany_2022043_ fP | 1.3 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_Not For Publication | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment arrangements_Recruitment and Informed consent procedure_FP | 1.0 |
| Recruitment arrangements (for publication) | K1_Recruitment procedure_EN_FP | 1 |
| Subject information and informed consent form (for publication) | L1_ SIS and ICF Future Research Adult_Spain_For Publication | 1.0 |
| Subject information and informed consent form (for publication) | L1_ SIS and ICF Future Research Parental_Spain_For Publication | 1.0 |
| Subject information and informed consent form (for publication) | L1_ SIS and ICF Pregnancy Follow up father_ Spain_For Publication | 1.0 |
| Subject information and informed consent form (for publication) | L1_ SIS and ICF Pregnancy Follow up mother_ Spain_For Publication | 1.0 |
| Subject information and informed consent form (for publication) | L1_ SIS and ICF Withdrawal Adult_ Spain_For Publication | 1.1 |
| Subject information and informed consent form (for publication) | L1_ SIS and ICF Withdrawal Parental_ Spain_For Publication | 1.1 |
| Subject information and informed consent form (for publication) | L1_Adolescent ICF_EN_Clean_NFP | 1.1 |
| Subject information and informed consent form (for publication) | L1_ICF Adolescent Assent_EN_Clean_FP | 1.1 |
| Subject information and informed consent form (for publication) | L1_ICF Adolescent Assent_EN_Clean_NFP | 1.0 |
| Subject information and informed consent form (for publication) | L1_ICF Adolescent Assent_FR_Clean_FP | 1.1 |
| Subject information and informed consent form (for publication) | L1_ICF Adolescent Assent_NL_Clean_FP | 1.1 |
| Subject information and informed consent form (for publication) | L1_ICF Adult_EN_Clean_FP | 1.1 |
| Subject information and informed consent form (for publication) | L1_ICF Adult_FR_Clean_FP | 1.1 |
| Subject information and informed consent form (for publication) | L1_ICF Adult_NL_Clean_FP | 1.1 |
| Subject information and informed consent form (for publication) | L1_ICF Child Assent_FR_Clean_FP | 1.1 |
| Subject information and informed consent form (for publication) | L1_ICF Child Assent_NL_Clean_FP | 1.1 |
| Subject information and informed consent form (for publication) | L1_ICF Child_EN_Clean_FP | 1.1 |
| Subject information and informed consent form (for publication) | L1_ICF Parental__EN_Clean_FP | 1.1 |
| Subject information and informed consent form (for publication) | L1_ICF Parental_FR_Clean_FP | 1.1 |
| Subject information and informed consent form (for publication) | L1_ICF Parental_NL_Clean_FP | 1.1 |
| Subject information and informed consent form (for publication) | L1_Informed consent procedure_Germany_20220043_fP | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent Assent 12-17 yr_Spain_For Publication | 1.2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent_For publication | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Adolescent_FP | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Child Age 5-7_FP | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Child Age 8-11_FP | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Child_For publication | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Future Research Adult_FP | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Future Research Parental_FP | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Main Adult_For publication | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Main Adult_FP | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Main Adult_Spain_For Publication | 1.2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Main Parent_For publication | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Main Parental_FP | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Main Parental_Spain_For Publication | 1.2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Pregnancy Adult_FP | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF Pregnancy Parental_FP | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_12 17years_FP | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_18 and above_FP | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_6 11 years_FP | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Adolescent_12-17 years old_FP | 1.2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Child Assent_FP | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Future research_FP | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_GDPR Information Notice Form Adults_FP | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main Adults_FP | 1.2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Main Parent_FP | 1.2 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Parental Future Research_FP | 1.0 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_Parental GDPR Information Notice Form_FP | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_parental_FP | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_pregnant breastfeeding_FP | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_pregnant participant_FP | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF_pregnant partner_FP | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS_ICF_FR Adult_20220043_Germany_fP | 1.2 |
| Subject information and informed consent form (for publication) | L1_SIS_ICF_FR Parent_20220043_Germany_fP | 1.2 |
| Subject information and informed consent form (for publication) | L1_SIS-ICF_Main 5 till 7 years_20220043_Germany_fP | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS-ICF_Main 8 till 12 years_20220043_Germany_fP | 1.1 |
| Subject information and informed consent form (for publication) | L1_SIS-ICF_Main Adolescent_20220043_Germany_fP | 1.2 |
| Subject information and informed consent form (for publication) | L1_SIS-ICF_Main Adult_20220043_Germany_fP | 1.2 |
| Subject information and informed consent form (for publication) | L1_SIS-ICF_Main Parent_20220043_Germany_fP | 1.2 |
| Subject information and informed consent form (for publication) | L2_ Other subject info material_Costs reimburse_fp | 1.0 |
| Subject information and informed consent form (for publication) | L2_ Other subject info material_General terms_fp | 1.0 |
| Subject information and informed consent form (for publication) | L2_Informed consent procedure_FP | 1 |
| Subject information and informed consent form (for publication) | L2_Informed Consent Procedure_Spain_Not For Publication | 1.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material GP letter_For publication | 1.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material ICF procedure_For publication | 1.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Clincard Information form_Adult_FP | 1.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Clincard Information form_Adult_Third party_FP | 1.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Clincard Information form_Parent_FP | 1.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_Clincard Information form_Parent_Third party_FP | 1.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_ICF procedure_FP | 1.0 |
| Subject information and informed consent form (for publication) | L2_Other subject information material_patientmat_FP | 1.1 |
| Subject information and informed consent form (for publication) | L2_Other subject information_Sponsor Statement on use of ICF model_EN_NFP | 1 |
| Subject information and informed consent form (for publication) | L3_Other subject informatIon_Patient Card_FP | 1 |
| Synopsis of the protocol (for publication) | D2_Protocol Synopsis_AT DE_Full_2023-503293-21_20220043_For Publication | 1 |
| Synopsis of the protocol (for publication) | D2_Protocol Synopsis_AT DE_PLPS_2023-503293-21_20220043_For Publication | 1 |
| Synopsis of the protocol (for publication) | D2_Protocol Synopsis_BE DE_PLPS_2023-503293-21_20220043_For Publication | 1 |
| Synopsis of the protocol (for publication) | D2_Protocol Synopsis_BE FR_PLPS_2023-503293-21_20220043_For Publication | 1 |
| Synopsis of the protocol (for publication) | D2_Protocol Synopsis_BE NL_PLPS_2023-503293-21_20220043_For Publication | 1 |
| Synopsis of the protocol (for publication) | D2_Protocol Synopsis_ENG_PLPS_2023-503293-21_20220043_For Publication | 1 |
| Synopsis of the protocol (for publication) | D2_Protocol Synopsis_ES_PLPS_2023-503293-21_20220043_For Publication | 1 |
| Synopsis of the protocol (for publication) | D2_Protocol Synopsis_HU_Full_2023-503293-21_20220043_For Publication | 1 |
| Synopsis of the protocol (for publication) | D2_Protocol Synopsis_HU_PLPS_2023-503293-21_20220043_For Publication | 1 |
| Synopsis of the protocol (for publication) | D2_Protocol Synopsis_IT_Full_2023-503293-21_20220043_For Publication | 1 |
| Synopsis of the protocol (for publication) | D2_Protocol Synopsis_IT_PLPS_2023-503293-21_20220043_For Publication | 1 |
| Synopsis of the protocol (for publication) | D2_Protocol Synopsis_SK_PLPS_2023-503293-21_20220043_For Publication | 1 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2025-10-15 | Slovakia | Acceptable 2026-02-23
|
2026-02-23 |