EUCTR2014-000406-35-FR进行中（未招募）1 期
A Randomized, open Label, Dose Finding, Phase 2 Study to Assess the Pharmacodynamics and Safety of the anti-FGF23 antibody, KRN23, in Pediatric Patients with X-linked Hypophosphatemia (XLH)
ltragenyx Pharmaceuticals Inc.0 个研究点目标入组 52 人开始时间: 2015年6月22日最近更新:
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 52
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
入选标准
- •Individuals eligible to participate in this study must meet all of the following criteria:
- •1)Male or female, aged 5 – 12 years, inclusive, with open growth plates
- •2)Tanner stage of 2 or less based on breast and testicular development (assessed only in children = 8 years of age)
- •3)Diagnosis of XLH supported by ONE of the following:
- •-Confirmed PHEX mutation in the patient or a directly related family member with appropriate X-linked inheritance
- •-Serum iFGF23 level > 30 pg/mL by Kainos assay
- •4)Biochemical findings associated with XLH including:
- •-Serum phosphorus = 2.8 mg/dL (0.904 mmol/L)*
- •-Serum creatinine within age-adjusted normal range*
- •5)Short stature as defined by standing height < 25% percentile for age and gender (per CDC 2000)
- •6)Radiographic evidence of active bone disease including rickets in the wrists and/or knees, AND/OR femoral/tibial bowing
- •7)Willing to provide access to prior medical records for the collection of historical growth, biochemical and radiographic data, and disease history.
- •8)Provide written or verbal assent (if possible) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures
- •9)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.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 30
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Individuals who meet any of the following exclusion criteria will not be eligible to participate in the study:
- •1)Use of a pharmacologic vitamin D metabolite or analog (e.g. calcitriol, doxercalciferol, and paricalcitol) within 14 days prior to Screening Visit 2; washout will take place during the Screening Period
- •2)Use of oral phosphate within 7 days prior to Screening Visit 2; washout will take place during the Screening Period
- •3)Use of aluminum hydroxide antacids (e.g. Maalox® and Mylanta®), systemic corticosteroids, and thiazides within 7 days prior to Screening Visit 1
- •4)Use of growth hormone within 1 year prior to Screening Visit 1
- •5)Use of bisphosphonates for 6 months or more in the 2 years prior to Screening Visit 1
- •6)Presence of nephrocalcinosis on renal ultrasound graded = 3 based on the following scale:
- •0 = Normal
- •1 = Faint hyperechogenic rim around the medullary pyramids
- •2 = More intense echogenic rim with echoes faintly filling the entire pyramid
- •3 = Uniformly intense echoes throughout the pyramid
- •4 = Stone formation: solitary focus of echoes at the tip of the pyramid
- •7)Planned or recommended orthopedic surgery, including staples, 8-plates or osteotomy, within the clinical trial period
- •8)Hypocalcemia or hypercalcemia, defined as serum calcium levels outside the age-adjusted normal limits*
- •9)Evidence of tertiary hyperparathyroidism as determined by the Investigator
- •10)Use of medication to suppress PTH (e.g. Sensipar®, cinacalcet) within 2 months prior to Screening Visit 1
- •11)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.
- •12)Presence of a concurrent disease or condition that would interfere with study participation or affect safety
- •13)Positive for human immunodeficiency virus antibody, hepatitis B surface antigen, and/or hepatitis C antibody
- •14)History of recurrent infection or predisposition to infection, or of known immunodeficiency
- •15)Use of a therapeutic monoclonal antibody within 90 days prior to Screening Visit 1 or history of allergic or anaphylactic reactions to any monoclonal antibody
- •16)Presence or history of any hypersensitivity to KRN23 excipients that, in the judgment of the investigator, places the subject at increased risk for adverse effects.
- •17)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.
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