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Burosumab for CSHS

NCT03993821 · Status: UNKNOWN · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 1

Last updated 2022-03-07

No results posted yet for this study

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.

Conditions

  • Cutaneous Skeletal Hypophosphatemia Syndrome (CSHS)
  • Epidermal Nevus Syndrome

Interventions

DRUG

Burosumab

Burosumab, the investigational product, is a recombinant human IgG1 monoclonal antibody targeting FGF23. It is supplied as a sterile, clear, colorless and preservative-free solution and is administered via subcutaneous injection.

Sponsors & Collaborators

Principal Investigators

  • Laura Tosi, MD · Children's National Research Institute

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-07-01
Primary Completion
2022-10-30
Completion
2023-03-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03993821 on ClinicalTrials.gov