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Study to Evaluate Romosozumab in Children and Adolescents With Osteogenesis Imperfecta

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04545554
Recruitment Status : Active, not recruiting
First Posted : September 11, 2020
Last Update Posted : December 8, 2022
Sponsor:
Information provided by (Responsible Party):
Amgen

Study Description
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Brief Summary:
The primary objective of this study is to evaluate the pharmacokinetics (PK) profile following multiple subcutaneous (SC) doses of romosozumab in children and adolescents with Osteogenesis Imperfecta (OI).

Condition or disease Intervention/treatment Phase
Osteogenesis Imperfecta Drug: Romosozumab Dietary Supplement: Calcium Dietary Supplement: Vitamin D Phase 1

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 25 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label, Ascending Multiple-dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Romosozumab in Children and Adolescents With Osteogenesis Imperfecta
Actual Study Start Date : January 21, 2021
Estimated Primary Completion Date : March 30, 2023
Estimated Study Completion Date : March 30, 2023

Resource links provided by the National Library of Medicine

Drug Information available for: Romosozumab

Arms and Interventions
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Arm Intervention/treatment
Experimental: Romosozumab Drug: Romosozumab
Participants will receive 3 doses of romosozumab via a subcutaneous (SC) injection.

Dietary Supplement: Calcium
All participants will receive daily supplements of elemental calcium.

Dietary Supplement: Vitamin D
All participants will receive daily supplementation with vitamin D.


Outcome Measures
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Primary Outcome Measures :
  1. Maximum-observed Concentration (Cmax) of Romosozumab in Serum [ Time Frame: Up to Day 169 ]
  2. Time to Maximum-observed Concentration (tmax) of Romosozumab in Serum [ Time Frame: Up to Day 169 ]
  3. Area Under the Curve (AUC) of Romosozumab in Serum [ Time Frame: Up to Day 169 ]
  4. Terminal Half-life (t1/2) of Romosozumab in Serum [ Time Frame: Up to Day 169 ]

Secondary Outcome Measures :
  1. Number of Participants who Experience Treatment-emergent Adverse Events [ Time Frame: Day 0 (post study drug administration) to Day 169 ]
  2. Number of Participants with a Clinically Significant Change from Baseline in Vital Sign Measurements [ Time Frame: Baseline to Day 169 ]
  3. Number of Participants with Clinically Significant Changes from Baseline in Electrocardiogram (ECG) Results [ Time Frame: Baseline to Day 169 ]
  4. Number of Participants with Clinically Significant Changes from Baseline in Physical Examination Results [ Time Frame: Baseline to Day 169 ]
  5. Number of Participants with Clinically Significant Changes from Baseline in Safety Laboratory Tests [ Time Frame: Baseline to Day 169 ]
  6. Number of Participants with Antiromosuzomab Antibodies [ Time Frame: Up to Day 169 ]
  7. Percent Change from Baseline in Bone Turnover Marker: Procollagen Type 1 N-terminal Propeptide (P1NP) [ Time Frame: Baseline to Day 169 ]
  8. Percent Change from Baseline in Bone Turnover Marker: Serum C-telopeptide (CTX) [ Time Frame: Baseline to Day 169 ]
  9. Percent Change from Baseline in Bone Mineral Density (BMD) at the Anteroposterior Lumbar Spine [ Time Frame: Baseline to Day 169 ]
  10. Percent Change from Baseline in Bone Mineral Content (BMC) [ Time Frame: Baseline to Day 169 ]
  11. Percent Change from Baseline in Bone Area [ Time Frame: Baseline to Day 169 ]
  12. Percent Change from Baseline in Bone Mineral Density (BMD) Z-Score at Anteroposterior Lumbar Spine [ Time Frame: Baseline to Day 169 ]

Eligibility Criteria
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Ages Eligible for Study:   5 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Ambulatory male or female children 5 to 18 years of age upon entry into screening
  • Clinical diagnosis of OI defined as a clinical history consistent with type I-IV OI as determined by presence of expected phenotype and lack of additional features unrelated to type I-IV OI

Exclusion Criteria

  • History of an electrophoresis pattern inconsistent with type I to type IV OI
  • History of known mutation in a gene other than collagen type I alpha/collagen type I alpha 2 (COL1AI/COL1A2) causing OI or other metabolic bone disease
  • History of other bone diseases that affect bone metabolism (eg, osteoporosis pseudoglioma syndrome, idiopathic juvenile osteoporosis, osteopetrosis, hypophosphatasia)
  • History of Kawasaki disease, rheumatic myocarditis, ischemic cardiomyopathy, inherited cardiomyopathies, nephrotic syndrome, familial hypercholesterolemia, stroke, or any thromboembolic disorder
  • Unhealed fracture as defined by orthopedic opinion
  • Symptoms associated with skull abnormalities such as basilar invagination, basilar impression or Chiari malformation
  • Prior treatment with anti-sclerostin antibody, fluoride or strontium, parathyroid hormone (PTH) within 12 months prior to screening, denosumab within 12 months or zoledronic acide with in 6 months prior to first dose
  • Less than 2 evaluable vertebrae by DXA evaluation in the region of interest, L1 L4, as confirmed by the central imaging laboratory.
  • Clinically significant valvular heart disease based on local echocardiogram (ECHO) results.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04545554


Locations
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United States, Indiana
Riley Hospital for Children
Indianapolis, Indiana, United States, 46202
United States, Tennessee
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37212-3157
United States, Wisconsin
The Medical College of Wisconsin
Milwaukee, Wisconsin, United States, 53226
Austria
Kepler Universitaetsklinikum GmbH
Linz, Austria, 4020
Germany
Uniklinik Köln
Koeln, Germany, 50937
Greece
General Children Hospital Panagioti and Aglaias Kyriakou
Athens, Greece, 11527
Hungary
Semmelweis Egyetem
Budapest, Hungary, 1094
Italy
IRCCS Ospedale Pediatrico Bambino Gesu
Roma, Italy, 00165
Spain
Hospital Sant Joan de Deu
Esplugues de Llobregat, Cataluña, Spain, 08950
Hospital Universitari i Politecnic La Fe
Valencia, Comunidad Valenciana, Spain, 46026
Hospital Universitario de Getafe
Getafe, Madrid, Spain, 28905
Hospital de Cruces
Baracaldo, País Vasco, Spain, 48903
Turkey
Gazi Universitesi Tip Fakultesi
Ankara, Turkey, 06500
Koc Universitesi Hastanesi
Istanbul, Turkey, 34010
Ege Universitesi Ilac Gelistirme ve Farmakokinetik Arastirma Uygulama Merkezi (ARGEFAR)
Izmir, Turkey, 35100
Sponsors and Collaborators
Amgen
Investigators
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Study Director: MD Amgen
More Information
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Additional Information:

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Responsible Party: Amgen
ClinicalTrials.gov Identifier: NCT04545554    
Other Study ID Numbers: 20160227
2017-004972-74 ( EudraCT Number )
First Posted: September 11, 2020    Key Record Dates
Last Update Posted: December 8, 2022
Last Verified: December 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Clinical Study Report (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 (or other new use) 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, and if not approved, may be further arbitrated by a Data Sharing Independent Review Panel. 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.
URL: http://www.amgen.com/datasharing

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Additional relevant MeSH terms:
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Osteogenesis Imperfecta
Osteochondrodysplasias
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Genetic Diseases, Inborn
Collagen Diseases
Connective Tissue Diseases
 Vitamin D
Calcium
Vitamins
Micronutrients
Physiological Effects of Drugs
Calcium-Regulating Hormones and Agents
Bone Density Conservation Agents