JPRN-jRCT2080223558已完成3 期
A Phase 3 Open-Label Trial to Assess the Efficacy and Safety of KRN23 in Pediatric Patients with X-linked Hypophosphatemic Rickets/Osteomalacia and a Postmarketing Study of KRN23 Switched from the Phase 3 Trial
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 10
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- >= 1age old 至 <= 12age old（—）
- 性别
- All
入选标准
- •1) Personally submitted voluntary written informed consent by a legally authorized representative. If appropriate, written or verbal assent to participate in the study should be obtained from patients.
- •2) Aged >=1 and =<12 years patients at the time of consent
- •3) Patients who have open growth plate at screening
- •4) Willing to perform a self-administration of KRN23 by the patients (if appropriate) and legally authorized representative, and available to perform a self-administration by the investigator or subinvestigator, during the study period at the time of consent
- •5) Diagnosis of XLH, and meeting any of the followings;
- •a) PHEX mutation in either the patient or in a directly related family member with appropriate X-linked inheritance
- •b) Serum intact FGF23 level at screening >= 30 pg/mL
- •6) Finding evidence of rickets (e.g. a cup-shaped depression of the metaphyseal end, enlargement of the epiphyseal line, fuzzing) or clinical symptoms such as X-legs and O-legs, by the judgment of investigator, in the simple X-ray evaluation at screening
- •7) Meeting all of following criteria for laboratory test related to XLH;
- •a) Serum P: < 3.0 mg/dL
- •b) Serum Cr: Within the age-adjusted normal limits
- •c) Serum 25(OH)D: >= 16 ng/mL
- •8) For female patients who have reached menarche with child bearing potential; a negative urine pregnancy test at screening
- •9) For female patients with child baring potential, or male patients with reproductive capacity; willingness to use an acceptable method of contraception while participating in the study
- •10) Willingness to provide access to prior medical records to determine eligibility including data on imaging tests, blood chemistry, diagnosis, medication, and surgical history
- •11) Willingness and ability to cooperatively complete all study procedures, adhere to the visit schedule and follow the investigator instructions, as considered by the investigator or subinvestigator
排除标准
- •1) Height percentile > 50% based on age-adjusted Japanese norms at screening
- •2) Use of aluminum hydroxide antacids, systemic corticosteroids, acetazolamide, and thiazides within 7 days prior to screening
- •3) Current or prior use of leuprorelin, triptorelin, goserelin, or other drugs known to delay puberty
- •4) Use of growth hormone therapy within 12 months before screening
- •5) Use of medication to suppress parathyroid hormone (e.g., cinacalcet) within 60 days prior to screening
- •6) Serum calcium levels outside the age-adjusted normal limits
- •7) Evidence of hyperparathyroidism (iPTH levels >= 163 pg/mL)
- •8) Presence of nephrocalcinosis on renal ultrasound grade 4 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
- •9) Planned or recommended orthopedic surgery (implantation or removal), including staples, 8 plates or osteotomy, during the study period
- •10) Blood or blood product transfusion within 60 days prior to screening
- •11) History of malignancy within 5 years prior to registration
- •12) History of being positive for HIV antibody, HBs antigen and/or HCV antibody
- •13) Predisposition to infection, or history of recurrent infection or known immunodeficiency
- •14) Use of any investigational product or investigational medical device within 4 months prior to screening, or requirement for any investigational agent prior to completion of all scheduled study assessments
- •15) Recieving investigational agent in the UX023-CL301 study
- •16) Use of a therapeutic monoclonal antibody other than KRN23 within 90 days prior to screening
- •17) History of allergic or anaphylactic reactions to KRN23, any of the KRN23 ingredients, or any other monoclonal antibodies
- •18) Anyone otherwise considered unsuitable participation in the study by the investigator or subinvestigator
研究者
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