Pain, Chronic Clinical Trial
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
Verified date | January 2020 |
Source | Redwood Dermatology Sciences |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 1 |
Est. completion date | December 6, 2019 |
Est. primary completion date | December 6, 2019 |
Accepts healthy volunteers | No |
Gender | Female |
Age group | N/A to 18 Years |
Eligibility |
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. |
Country | Name | City | State |
---|---|---|---|
United States | The Focus Center, PC | Clinton | Utah |
Lead Sponsor | Collaborator |
---|---|
Redwood Dermatology Sciences | Ultragenyx Pharmaceutical Inc |
United States,
Aono Y, Hasegawa H, Yamazaki Y, Shimada T, Fujita T, Yamashita T, Fukumoto S. Anti-FGF-23 neutralizing antibodies ameliorate muscle weakness and decreased spontaneous movement of Hyp mice. J Bone Miner Res. 2011 Apr;26(4):803-10. doi: 10.1002/jbmr.275. — View Citation
Aono Y, Yamazaki Y, Yasutake J, Kawata T, Hasegawa H, Urakawa I, Fujita T, Wada M, Yamashita T, Fukumoto S, Shimada T. Therapeutic effects of anti-FGF23 antibodies in hypophosphatemic rickets/osteomalacia. J Bone Miner Res. 2009 Nov;24(11):1879-88. doi: 10.1359/jbmr.090509. — View Citation
Carpenter TO, Imel EA, Ruppe MD, Weber TJ, Klausner MA, Wooddell MM, Kawakami T, Ito T, Zhang X, Humphrey J, Insogna KL, Peacock M. Randomized trial of the anti-FGF23 antibody KRN23 in X-linked hypophosphatemia. J Clin Invest. 2014 Apr;124(4):1587-97. doi: 10.1172/JCI72829. Epub 2014 Feb 24. — View Citation
Lim YH, Ovejero D, Sugarman JS, Deklotz CM, Maruri A, Eichenfield LF, Kelley PK, Jüppner H, Gottschalk M, Tifft CJ, Gafni RI, Boyce AM, Cowen EW, Bhattacharyya N, Guthrie LC, Gahl WA, Golas G, Loring EC, Overton JD, Mane SM, Lifton RP, Levy ML, Collins MT, Choate KA. Multilineage somatic activating mutations in HRAS and NRAS cause mosaic cutaneous and skeletal lesions, elevated FGF23 and hypophosphatemia. Hum Mol Genet. 2014 Jan 15;23(2):397-407. doi: 10.1093/hmg/ddt429. Epub 2013 Sep 4. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | The effect of KRN23 treatment on normalizing age-adjusted serum phosphorous levels in a single pediatric patient with Epidermal Nevus Syndrome associated hypophosphatemic rickets | 18 months |
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