drugset / Trial / NCT03993821
Burosumab for CSHS
Phase 0
Unknown
1 enrolled
Laura Tosi
Children's National Research Institute · collabUltragenyx Pharmaceutical Inc · collab
NaSingle-groupOpen-labelTreatment
Summary
Burosumab (also known as the drug, Crysvita®) is a fully human immunoglobulin G1 (IgG1) monoclonal antibody (mAb) that binds to and inhibits the activity of fibroblast growth factor 23 (FGF23), leading to an increase in serum phosphorus levels. This drug is already approved for use in patients with X-linked hypophosphatemia (XLH), but not for Cutaneous Skeletal Hypophosphatemia Syndrome (CSHS). It is hypothesized that burosumab may provide clinical benefit to a patient with CSHS due to the common underlying feature in this patient and in patients with XLH - abnormally elevated FGF23 in the context of low age -adjusted serum phosphorous levels.
Timeline
- Start
- 2019-07-01
- Primary completion
- 2022-10-30
- Completion
- 2023-03
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Burosumab | Monoclonal antibody | 0.3 mg/kg | Subcutaneous |