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Open Label Trial Assessing Safety and Efficacy of Burosumab (KRN23), in a Patient With ENS and Hypophosphatemic Rickets

CompletedPhase 3NCT03581591
View the official record on ClinicalTrials.gov

Recruiting status changes often. Contact the study team listed on ClinicalTrials.gov, and talk to your doctor before joining a study.

This study has no listed site in Canada in this snapshot. It is included as part of the worldwide chronic pain corpus.

Brief summary (from the registry)

A 52 week, open label trial to assess the safety and efficacy of KRN23, an investigational antibody to FGF23, in a single pediatric patient with Epidermal Nevus Syndrome(ENS) and associated hypophosphatemic rickets A 26 weeks extension to original study to monitor patient lab results for her safety.

Study details

Official title
An Open Label Trial to Assess the Safety and Efficacy of Burosumab (KRN23), an Investigational Antibody to FGF23, in a Single Pediatric Patient With Epidermal Nevus Syndrome(ENS) and Associated Hypophosphatemic Rickets
Conditions
Interventions
  • Biological: Burosumab
Sponsor
Redwood Dermatology Sciences
Collaborators
Ultragenyx Pharmaceutical Inc
Start date
2018-01-31
Primary completion
2019-12-06
Sex
Female
Age range
to 18 Years
Results posted
No results posted in this snapshot
Countries
United States
Total sites listed
1

Eligibility criteria

Show eligibility criteria (verbatim from the registry)
Inclusion Criteria: 1. Patient has confirmed ENS by physician diagnosis 2. Patient has confirmed FGF23 elevations in the context of low serum phosphorous \< 4.1 mg/dL 3. Patient able to tolerate KRN23 treatment 4. Have a corrected serum calcium level \< 10.8mg/dL 5. Have an eGFR \>60 ml/min 6. Must be willing in the opinion of the investigator, to comply with study procedures and schedule 7. Provide written informed consent by a parent after the study has been explained and prior to any research related procedures begin Exclusion Criteria: 1. Concomitant use of active vitamin D (i.e calcitriol) and/or exogenous phosphate supplementation. Patient will be allowed OTC Vitamin D should levels drop below \<20 ng/ml 2. The use or enrollment in studies using other investigational therapies including other monoclonal antibodies 3. Subject and their parent not willing or not able to give written informed consent 4. In the Investigators opinion, the patient may not be able to meet all the requirements for study participation 5. Patient has a history of hypersensitivity to KRN23 excipients that in the opinion of the investigator, places the patient at an increased risk of adverse effects 6. Patient has a condition that in the opinion of the investigator could present a concern for subject safety or data interpretation.

Source: ClinicalTrials.gov (US National Library of Medicine), snapshot October 2026. Check the registry for current recruiting status. Record: https://clinicaltrials.gov/study/NCT03581591