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Trial record 2 of 5 for:    setrusumab

Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta (Cosmic)

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ClinicalTrials.gov Identifier: NCT05768854
Recruitment Status : Recruiting
First Posted : March 14, 2023
Last Update Posted : April 9, 2024
Sponsor:
Information provided by (Responsible Party):
Ultragenyx Pharmaceutical Inc

Brief Summary:
The primary objective of the study is to evaluate the effect of setrusumab vs intravenous bisphosphonates (IV-BP) on reduction in fracture rate, including morphometric vertebral fractures in pediatric participants.

Condition or disease Intervention/treatment Phase
Osteogenesis Imperfecta Drug: Bisphosphonate Biological: Setrusumab Phase 3

Detailed Description:
Participants will be randomized 1:1 to receive either setrusumab or IV-BP. Following randomization, participants will receive setrusumab or IV-BP for up to 24 months during the Active-controlled Period. At the end of the Active-controlled Period all participants will enter the Extension Period and participants assigned to IV-BP will transition to setrusumab. During the Extension Period, all participants will receive setrusumab for a minimum of 12 months or until setrusumab becomes commercially available in their respective country or the study is discontinued. The use of any bisphosphonate is prohibited throughout the Extension Period.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 66 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label, Randomized, Active-Controlled, Phase 3 Study of Setrusumab Compared With Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta Types I, III or IV
Actual Study Start Date : June 14, 2023
Estimated Primary Completion Date : December 2025
Estimated Study Completion Date : June 2026


Arm Intervention/treatment
Active Comparator: Intravenous Bisphosphonates (IV-BP) -> Setrusumab

Participants on IV-BP will continue their existing dose/regimen per investigator discretion; for participants not on IV-BP, the dose/regimen will be determined by the investigator.

After the active-controlled period, participants will receive Setrusumab during the extension period

Drug: Bisphosphonate
Administered per investigator discretion via intravenous (IV) infusion

Biological: Setrusumab
A fully human sclerostin neutralizing monoclonal antibody (mAb) administered once a month (QM) via intravenous (IV) infusion
Other Names:
  • BPS804
  • UX143

Experimental: Setrusumab
Participants will receive Setrusumab during the active-controlled and extension period
Biological: Setrusumab
A fully human sclerostin neutralizing monoclonal antibody (mAb) administered once a month (QM) via intravenous (IV) infusion
Other Names:
  • BPS804
  • UX143




Primary Outcome Measures :
  1. Annualized Rate of all Radiographically-confirmed Fractures, Including Morphometric Vertebral Fractures During the Active-controlled Period [ Time Frame: Up to 24 Months ]

Secondary Outcome Measures :
  1. Annualized Rate of all Radiographically-confirmed Fractures, Excluding Morphometric Vertebral Fractures During the Active-controlled Period [ Time Frame: Up to 24 Months ]
  2. Change from Baseline in Pediatric Orthopedic Society of North America Pediatric Outcomes Data Collection Instrument (POSNA-PODCI) Sports/Physical Functioning and Pain/Comfort Subscale Scores at Month 12 of the Active-controlled Period [ Time Frame: Baseline, Up to 12 Months ]
  3. Serum Setrusumab Concentration [ Time Frame: Up to 24 Months ]
  4. Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and Adverse Events of Special Interest (AESIs) [ Time Frame: Up to 24 Months ]
  5. Number of Participants With Anti-setrusumab Binding and Neutralizing Antibodies [ Time Frame: Up to 24 Months ]


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Ages Eligible for Study:   2 Years to 6 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male or female 2 to < 7 years of age at time of informed consent
  • Clinical diagnosis of OI Types I, III, or IV confirmed by identification of genetic mutation in COL1A1 or COL1A2
  • History of ≥ 1 fracture in the past 12 months, ≥ 2 fractures in the past 24 months, or ≥ 1 femur, tibia, or humerus fracture in the past 24 months
  • Any prior exposure to, or currently receiving, IV-bisphosphonate therapy for treatment of OI
  • Serum 25-hydroxyvitamin D level ≥ 20 ng/mL at the Screening visit. If 25-hydroxyvitamin D levels are below 20 ng/mL, the subject may be rescreened after a minimum of 14 days of vitamin D supplementation as directed by the Investigator

Exclusion Criteria:

  • Contraindication for the use of IV bisphosphonates based on clinical judgment of the Investigator
  • History of skeletal malignancies or bone metastases at any time
  • History of neural foraminal stenosis (except if due to scoliosis)
  • Clinical manifestations of Chiari malformation or basilar invagination. Presence of any other neurologic disease that has been clinically unstable within past 2 years requires review by the Medical Monitor.
  • History of or current uncontrolled concomitant diseases that may impact bone metabolism, such as hypo/hyperparathyroidism, abnormal thyroid function, nephrotic syndrome, or Stage IV/V renal disease
  • Any skeletal condition (other than OI) leading to bone deformity and/or increased risk of fractures, such as rickets, osteopetrosis, idiopathic juvenile osteoporosis, or skeletal dysplasia
  • History of known cardiovascular disease such as coronary artery anomaly, Kawasaki disease, myocarditis, cardiomyopathy, myocardial infarction, stroke, or thromboembolic disease. Individuals with other congenital or acquired cardiovascular disease necessitating echocardiogram require Medical Monitor review. Investigators should consider whether the potential benefits of treatment outweigh the potential risks in patients with cardiovascular risk factors such as confirmed arterial hypertension.
  • Hypocalcemia, defined as serum calcium levels below the age-adjusted normal limit reference ranges after a recommended ≥ 4 hour fast, at Screening
  • Estimated glomerular filtration rate <=35 mL/min/1.73 m2 at Screening
  • Prior treatment with growth hormone, denosumab, anti-sclerostin antibody, or other anabolic or anti-resorptive medications impacting the bone (other than bisphosphonates) at any time
  • History of external radiation therapy
  • Known hypersensitivity to setrusumab or its excipients that, in the judgment of the Investigator, places the subject at increased risk for adverse effects
  • Presence or history of any condition that, in the view of the Investigator, would interfere with participation, pose undue risk, or would confound interpretation of results
  • Use of any investigational product or investigational medical device within 4 weeks or 5 half-lives (whichever is longer) of investigational drug prior to Screening, or during the study (per discretion of the Investigator in consultation with the Medical Monitor)
  • Concurrent participation in another clinical study without prior approval from the study Medical Monitor

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): NCT05768854


Contacts
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Contact: Patients Contact: Trial Recruitment 18887568657 trialrecruitment@ultragenyx.com
Contact: HCPs Contact: Medical Information 18887568657 medinfo@ultragenyx.com

Locations
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Sponsors and Collaborators
Ultragenyx Pharmaceutical Inc
Investigators
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Study Director: Medical Director Ultragenyx Pharmaceutical Inc
Additional Information:
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Responsible Party: Ultragenyx Pharmaceutical Inc
ClinicalTrials.gov Identifier: NCT05768854    
Other Study ID Numbers: UX143-CL314
2023-504196-24-00 ( Other Identifier: EU CT Number )
First Posted: March 14, 2023    Key Record Dates
Last Update Posted: April 9, 2024
Last Verified: April 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
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
Diphosphonates
Bone Density Conservation Agents
Physiological Effects of Drugs