Setrusumab vs Bisphosphonates for Osteogenesis Imperfecta
(Cosmic Trial)
Trial Summary
Do I need to stop my current medications to join the trial?
The trial does not specify if you need to stop taking your current medications, but it does require that you have not used certain bone-impacting medications other than bisphosphonates before. It's best to discuss your current medications with the study team.
What data supports the effectiveness of the drug Setrusumab vs Bisphosphonates for Osteogenesis Imperfecta?
What is the safety profile of bisphosphonates in humans?
Bisphosphonates are generally safe and well-tolerated, but they can cause side effects like stomach discomfort and flu-like symptoms. Rarely, they may lead to serious issues like jaw bone problems or unusual fractures. The risk of these serious side effects is low compared to the benefits, especially when used correctly.678910
How does the drug Setrusumab differ from bisphosphonates in treating osteogenesis imperfecta?
Setrusumab is a newer treatment option for osteogenesis imperfecta that may offer a different mechanism of action compared to traditional bisphosphonates, which are known to increase bone mineral density. While bisphosphonates like alendronate and zoledronic acid have been extensively studied and used to strengthen bones in OI patients, Setrusumab represents a novel approach that could potentially provide additional benefits.123411
What is the purpose of this trial?
This trial compares setrusumab, a new bone-strengthening drug, with standard treatments in children. It aims to see if setrusumab can better prevent bone fractures. Both treatments work by making bones stronger. Standard treatments have been used for a long time to increase bone mineral density and improve bone resistance, leading to a decrease in fracture rate.
Research Team
Medical Director
Principal Investigator
Ultragenyx Pharmaceutical Inc
Eligibility Criteria
This trial is for children with Osteogenesis Imperfecta (OI) types I, III, or IV who have had fractures in the past year and are currently on or have had bisphosphonate therapy. They must not have severe kidney issues, untreated hypocalcemia, a history of certain bone diseases other than OI, cardiovascular disease without clearance, or recent use of certain bone-impacting drugs.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Active-controlled Period
Participants are randomized to receive either setrusumab or IV-BP for up to 24 months
Extension Period
All participants receive setrusumab for a minimum of 12 months or until it becomes commercially available
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- Bisphosphonate
- Setrusumab
Bisphosphonate is already approved in European Union, United States, Canada, Japan, China, Switzerland for the following indications:
- Osteoporosis
- Paget's disease
- High calcium levels in cancer patients
- Bone metastases
- Osteoporosis
- Paget's disease
- Glucocorticoid-induced osteoporosis
- High calcium levels in cancer patients
- Bone metastases
- Osteoporosis
- Paget's disease
- High calcium levels in cancer patients
- Bone metastases
- Osteoporosis
- Paget's disease
- High calcium levels in cancer patients
- Bone metastases
- Osteoporosis
- Paget's disease
- High calcium levels in cancer patients
- Bone metastases
- Osteoporosis
- Paget's disease
- High calcium levels in cancer patients
- Bone metastases
Find a Clinic Near You
Who Is Running the Clinical Trial?
Ultragenyx Pharmaceutical Inc
Lead Sponsor
Dr. Emil D. Kakkis
Ultragenyx Pharmaceutical Inc
Chief Executive Officer since 2010
MD/PhD in Biological Chemistry from UCLA
Dr. Eric Crombez
Ultragenyx Pharmaceutical Inc
Chief Medical Officer since 2023
MD from Wayne State University School of Medicine