Study of the Safety, Pharmacodynamics and Efficacy of KRN23 in Children From 1 to 4 Years Old With X-linked Hypophosphatemia (XLH)
This trial is active, not recruiting.
|Sponsor||Ultragenyx Pharmaceutical Inc|
|Start date||May 2016|
|End date||October 2017|
|Trial size||13 participants|
|Trial identifier||NCT02750618, UX023-CL205|
UX023-CL205 is a multicenter, open-label, Phase 2 study in children from 1 to 4 years old with XLH to assess the safety, PD, and efficacy of KRN23 administered via subcutaneous (SC) injections every 2 weeks (Q2W) for a total of 64 weeks.
|United States||No locations recruiting|
|Other Countries||No locations recruiting|
|Endpoint classification||safety/efficacy study|
|Intervention model||single group assignment|
Evaluate safety by the incidence, frequency and severity of AEs and SAEs, including clinically significant changes from baseline to scheduled time points
time frame: 64 weeks
Determine the pharmacodynamics (PD) effects of KRN23 treatment by observing the change from Baseline over time in serum phosphorus
time frame: 64 weeks
Male or female participants from 1 year up to 4 years old.
Inclusion Criteria: 1. Male or female, aged ≥1 year and <5 years 2. Diagnosis of XLH supported by ONE or more of the following - Confirmed PHEX mutation in the patient or a directly related family member with appropriate X-linked inheritance - Serum FGF23 level > 30 pg/mL by Kainos assay 3. Biochemical findings associated with XLH including: - Serum phosphorus < 3.0 mg/dL (0.97 mmol/L) - Serum creatinine within age-adjusted normal range 4. Radiographic evidence of rickets 5. Willing to provide access to prior medical records for the collection of historical growth, biochemical, and radiographic data and disease history 6. Provide written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures 7. Must, in the opinion of the investigator, be willing and able to complete all aspects of the study, adhere to the study visit schedule, and comply with the assessments Exclusion Criteria: 1. Unwilling to stop treatment with oral phosphate and/or pharmacologic vitamin D metabolite or analog (e.g. calcitriol, alfacalcidol) during the screening period and for the duration of the study 2. Planned or recommended orthopedic surgery including staples, 8-plates or osteotomy, within the clinical trial period 3. Hypocalcemia or hypercalcemia, defined as serum calcium levels outside the age-adjusted normal limits 4. Presence or history of any condition that, in the view of the investigator, places the subject at high risk of poor treatment compliance or of not completing the study 5. Presence of a concurrent disease or condition that would interfere with study participation or affect safety 6. History of recurrent infection or predisposition to infection, or of known immunodeficiency 7. Use of any investigational product or investigational medical device within 30 days prior to screening, or requirement for any investigational agent prior to completion of all scheduled study assessments
|Official title||An Open-Label, Phase 2 Study to Assess the Safety, Pharmacodynamics, and Efficacy of KRN23 in Children From 1 to 4 Years Old With X-linked Hypophosphatemia (XLH)|
|Principal investigator||Erik Imel, MD|
|Description||The study will enroll approximately 10 pediatric subjects between 1 and 4 years old, inclusive, with a confirmed diagnosis of XLH and radiographic evidence of rickets. Subjects will discontinue oral phosphate and active vitamin D therapy after initial Screening and for the duration of the study. All subjects will receive treatment with KRN23. Safety, PD, pre-dose KRN23 concentrations, and efficacy measures (rickets, growth, functional outcomes) will be evaluated throughout the study. Radiographs will be taken to assess rickets periodically throughout the study duration.|
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