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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

Tracking Information
First Submitted Date  ICMJE September 4, 2020
First Posted Date  ICMJE September 11, 2020
Last Update Posted Date December 8, 2022
Actual Study Start Date  ICMJE January 21, 2021
Estimated Primary Completion Date March 30, 2023   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 4, 2020)
  • Maximum-observed Concentration (Cmax) of Romosozumab in Serum [ Time Frame: Up to Day 169 ]
  • Time to Maximum-observed Concentration (tmax) of Romosozumab in Serum [ Time Frame: Up to Day 169 ]
  • Area Under the Curve (AUC) of Romosozumab in Serum [ Time Frame: Up to Day 169 ]
  • Terminal Half-life (t1/2) of Romosozumab in Serum [ Time Frame: Up to Day 169 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: September 4, 2020)
  • Number of Participants who Experience Treatment-emergent Adverse Events [ Time Frame: Day 0 (post study drug administration) to Day 169 ]
  • Number of Participants with a Clinically Significant Change from Baseline in Vital Sign Measurements [ Time Frame: Baseline to Day 169 ]
  • Number of Participants with Clinically Significant Changes from Baseline in Electrocardiogram (ECG) Results [ Time Frame: Baseline to Day 169 ]
  • Number of Participants with Clinically Significant Changes from Baseline in Physical Examination Results [ Time Frame: Baseline to Day 169 ]
  • Number of Participants with Clinically Significant Changes from Baseline in Safety Laboratory Tests [ Time Frame: Baseline to Day 169 ]
  • Number of Participants with Antiromosuzomab Antibodies [ Time Frame: Up to Day 169 ]
  • Percent Change from Baseline in Bone Turnover Marker: Procollagen Type 1 N-terminal Propeptide (P1NP) [ Time Frame: Baseline to Day 169 ]
  • Percent Change from Baseline in Bone Turnover Marker: Serum C-telopeptide (CTX) [ Time Frame: Baseline to Day 169 ]
  • Percent Change from Baseline in Bone Mineral Density (BMD) at the Anteroposterior Lumbar Spine [ Time Frame: Baseline to Day 169 ]
  • Percent Change from Baseline in Bone Mineral Content (BMC) [ Time Frame: Baseline to Day 169 ]
  • Percent Change from Baseline in Bone Area [ Time Frame: Baseline to Day 169 ]
  • Percent Change from Baseline in Bone Mineral Density (BMD) Z-Score at Anteroposterior Lumbar Spine [ Time Frame: Baseline to Day 169 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study to Evaluate Romosozumab in Children and Adolescents With Osteogenesis Imperfecta
Official Title  ICMJE An Open-label, Ascending Multiple-dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Romosozumab in Children and Adolescents With Osteogenesis Imperfecta
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).
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Osteogenesis Imperfecta
Intervention  ICMJE
  • 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.
Study Arms  ICMJE Experimental: Romosozumab
Interventions:
  • Drug: Romosozumab
  • Dietary Supplement: Calcium
  • Dietary Supplement: Vitamin D
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: April 22, 2022)
25
Original Estimated Enrollment  ICMJE
 (submitted: September 4, 2020)
16
Estimated Study Completion Date  ICMJE March 30, 2023
Estimated Primary Completion Date March 30, 2023   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 5 Years to 17 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Austria,   Germany,   Greece,   Hungary,   Italy,   Spain,   Turkey,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04545554
Other Study ID Numbers  ICMJE 20160227
2017-004972-74 ( EudraCT Number )
Has Data Monitoring Committee No
U.S. FDA-regulated Product
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
IPD Sharing Statement  ICMJE
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
Supporting Materials: Statistical Analysis Plan (SAP)
Supporting Materials: Informed Consent Form (ICF)
Supporting Materials: 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
Current Responsible Party Amgen
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Amgen
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: MD Amgen
PRS Account Amgen
Verification Date December 2022

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP