Status:
COMPLETED
A Trial to Assess the Safety and Efficacy of KRN23 in Epidermal Nevus Syndrome (ENS)
Lead Sponsor:
University of Alabama at Birmingham
Collaborating Sponsors:
Ultragenyx Pharmaceutical Inc
Conditions:
Epidermal Nevus Syndrome
Eligibility:
MALE
6+ years
Phase:
PHASE4
Brief Summary
KRN23 is a fully human immunoglobulin monoclonal antibody (mAb) that binds to and inhibits the activity of fibroblast growth factor 23 (FGF23), leading to an increase in serum phosphorus levels. There...
Detailed Description
KRN23 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 lev...
Eligibility Criteria
Inclusion
- Patient has confirmed ENS by physician diagnosis
- Patient has confirmed FGF23 elevations in the context of low serum phosphorous \< 4.1 mg/dL
- Patient able to tolerate KRN23 treatment
- Have a corrected serum calcium level \< 10.8mg/dL
- Have an eGFR \>60 ml/min
- Must be willing in the opinion of the investigator, to comply with study procedures and schedule
- Provide written informed consent by a parent after
Exclusion
- Patient should not use CRYSVITA with Oral phosphate or active Vitamin D analogs.
- Patient and investigator should not initiate CRYSVITA if Phosphorus level is within or above normal.
- CRYSVITA is contraindicated in patients with severe renal impairment or end stage renal disease because these conditions are associated with abnormal mineral metabolism.
- The use or enrollment in studies using other investigational therapies including other monoclonal antibodies
- Subject and their Parent not willing or not able to give written informed consent
- In the Investigators opinion, the subject may not be able to meet all the requirements for study participation
- Subject has a history of hypersensitivity to KRN23 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.
Key Trial Info
Start Date :
July 31 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
August 31 2021
Estimated Enrollment :
1 Patients enrolled
Trial Details
Trial ID
NCT04320316
Start Date
July 31 2020
End Date
August 31 2021
Last Update
October 7 2022
Active Locations (1)
Enter a location and click search to find clinical trials sorted by distance.
1
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233