Open Label Trial Assessing Safety and Efficacy of Burosumab (KRN23), in a Patient With ENS and Hypophosphatemic Rickets
Completed · Phase 3
Conditions studied: Hypophosphatemia, Hypophosphatemic Rickets, Pain, Chronic
In brief
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.
Key facts
- Study ID
- NCT03581591
- Run by
- Redwood Dermatology Sciences
- People needed
- 1
- Starts
- 2018-01-31
- Expected to finish
- 2019-12-06
- Last updated by the study team
- 2020-01-18
Who can join
Age: any, up to 18. Sex: female. Healthy volunteers: not accepted.
You may qualify if…
- 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
You may not qualify if…
- 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
- Patient has a condition that in the opinion of the investigator could present a concern for subject safety or data interpretation.
Where it is running
- The Focus Center, PC — Clinton, Utah, United States
Full record on ClinicalTrials.gov
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