Open Label Trial Assessing Safety and Efficacy of Burosumab (KRN23), in a Patient With ENS and Hy… (NCT03581591) | Clinical Trial Compass
CompletedPhase 3
Open Label Trial Assessing Safety and Efficacy of Burosumab (KRN23), in a Patient With ENS and Hypophosphatemic Rickets
United States1 participantsStarted 2018-01-31
Plain-language summary
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.
Who can participate
Age range18 Years
SexFEMALE
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
✓. 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 the study has been explained and prior to any research related procedures begin
Exclusion criteria
✕. 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
✕. 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 patient may not be able to meet all the requirements for study participation
✕. 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
What they're measuring
1
The effect of KRN23 treatment on normalizing age-adjusted serum phosphorous levels in a single pediatric patient with Epidermal Nevus Syndrome associated hypophosphatemic rickets