Burosumab for CSHS (NCT03993821) | Clinical Trial Compass
UnknownEarly Phase 1
Burosumab for CSHS
United States1 participantsStarted 2019-07-01
Plain-language 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.
Who can participate
SexALL
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Inclusion criteria
✓. Patient has confirmed CSHS by physician diagnosis
✓. Patient has confirmed FGF23 elevations in the context of a low fasting serum phosphorous \< 2.5 mg/dL
✓. Patient able to tolerate burosumab treatment
✓. Have a corrected serum calcium level \< 10.8 mg/dL
✓. Have an eGFR \>25 mL/min/1.73m2 (using CKD-EPI equation)
✓. Must be willing in the opinion of the investigators, to comply with study procedures and schedule
✓. Provide written informed consent by the subject or a Legal Authorized Representative (LAR) after the study has been explained and prior to any research related procedures begin
✓. Must have a negative pregnancy test at Screening and be willing to have additional pregnancy tests during the study.
Exclusion criteria
✕. Concomitant use of active vitamin D (i.e. calcitriol) and/or exogenous phosphate supplementation during burosumab therapy. Subjects will be allowed over the counter Vitamin D should levels drop below \<20 ng/ml
. Blood phosphorus level within or above the normal range while not taking phosphate or active Vitamin D.
✕. Severe renal impairment or end-stage renal disease, defined as an eGFR of less than 25 ml/min/1.73m2
✕. The use or enrollment in studies using other investigational therapies including other monoclonal antibodies
✕. Subject or Legally Authorized Representative not willing or not able to give written informed consent
✕. In the investigator's opinion, the subject may not be able to meet all the requirements for study participation
✕. History of hypersensitivity to burosumab excipients that in the opinion of the investigator, places the subject at an increased risk of adverse effects
✕. Subject has a condition that in the opinion of the investigator could present a concern for subject safety or data interpretation.