NCT04308096已完成3 期
A Phase 3 Long-term Extension Study of KRN23 in Patients With X-linked Hypophosphatemic Rickets/Osteomalacia and a Post-marketing Study of KRN23 Switched From the Phase 3 Long-term Extension Study
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 27
- 试验地点
- 10
- 主要终点
- Effect to Pulse rate
研究概览
简要总结
Before switching to the post-marketing study:
Assess the efficacy and safety of KRN23 administered subcutaneously once every 4 or 2 weeks in adult or children with XLH
After switching to the post-marketing study:
To evaluate the safety and efficacy of KRN23, which was switched from the investigational product to the post-marketing investigational product, at the approved dose and dosing regimen in subjects who continued treatment
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Personally submitted voluntary written informed consent to participate in the study; For pediatric patients, 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.
- •Patients meeting any of the followings;
- •For adult XLH patients, completion the final observation at Week 96 in UX023-CL303 or UX023-CL304
- •For pediatric patients, completion the final observation at Week 64 in UX023-CL301
- •For female patients; women of childbearing potential (except for females who have not reached menarche, permanently sterilized, postmenopausal [12 months with no menses without an alternative medical cause] or anatomically not of childbearing potential) with negative pregnancy test at pre-treatment assessment of Week 0
- •For female patient with childbearing potential, or male patients with reproductive capacity; willingness to use acceptable methods of contraception while participating in the study
- •Willingness and ability to cooperatively complete all study procedures, adhere to the visit schedule and follow the investigator's instructions, as considered by investigator or subinvestigator
排除标准
- •Use of oral phosphate for treating XLH, pharmacologic vitamin D metabolites or analogs, aluminum hydroxide antacids, systemic corticosteroids, acetazolamide, and thiazides within 7 days prior to scheduled initial administration of investigational drug
- •Planned or recommended orthopedic surgery (implantation or removal), including staples, 8 plates or osteotomy, during the study period
- •Blood or blood product transfusion within 60 days prior to scheduled initial administration of investigational drug
- •Use of growth hormone therapy within 12 months prior to scheduled initial administration of investigational drug
- •Use of medication to suppress the secretion of parathyroid hormone (e.g., cinacalcet) within 60 days prior to scheduled initial administration of investigational drug
- •Use of any investigational product (except for investigational product of the preceding study) or investigational medical device within 4 months prior to scheduled initial administration of investigational drug, or requirement for any investigational agent prior to completion of all scheduled study assessments
- •Use of a therapeutic monoclonal antibody other than KRN23 within 90 days prior to scheduled initial administration of investigational drug
- •History of being positive for HIV antibody, HBs antigen and/or HCV antibody
- •Anyone otherwise considered unsuitable for the study by the investigator or subinvestigator
- •At the time of switching to the post-marketing clinical study:
- •Subjects eligible for enrollment in the post-marketing clinical study must have met both of the following criteria:
- •Personally submitted voluntary written informed consent to participate in the postmarketing clinical study. For pediatric patients, personally submitted voluntary written informed consent by a legally authorized representative. If appropriate, written or verbal assent to participate in the post-marketing clinical study was to be obtained from subjects.
- •Switching to the post-marketing clinical study was necessary and appropriate for the subject from the viewpoint of efficacy and safety, as judged by the investigator or subinvestigator
研究组 & 干预措施
KRN23
Experimental
Subjects will receive subcutaneous injections of KRN23 every 4 weeks (adult) or 2 weeks (pediatric) from Week 0 through Week 140.
干预措施: KRN23 (Drug)
结局指标
主要结局
Effect to Pulse rate
时间窗: up to week 140
Effect to Respiratory rate
时间窗: up to week 140
Effect to Systolic blood pressure in sitting position
时间窗: up to week 140
Effect to Diastolic blood pressure in sitting position
时间窗: up to week 140
Effect to 12-lead electrocardiogram (ECG)
时间窗: up to week 140
The presence of abnormality in the electrocardiogram
Effect to renal ultrasound
时间窗: up to week 140
The evaluation to nephrocalcinosis in five grades by renal ultrasound
Effect to Echocardiogram
时间窗: up to week 140
The presence of ectopic calcification in the heart by Echocardiogram
Number of subjects for each adverse events
时间窗: up to week 140
Effect to Body temperature
时间窗: up to week 140
次要结局
- Concentration of serum phosphorus(up to week 140)
- Concentration of serum 1,25(OH)2D(up to week 140)
- Concentration of urinary phosphorus(up to week 140)
- Concentration of tubular resorption of phosphorus（TRP）(up to week 140)
- Concentration of maximum tubular reabsorption of phosphate/glomerular filtration rate (TmP/GFR)(up to week 140)
- concentration of Carboxy terminal cross-linked telopeptide of type 1 collagen (CTx) (Adult patients with XLH)(up to week 140)
- concentration of Procollagen type 1 N-propeptide (P1NP) (Adult patients with XLH)(up to week 140)
- concentration of Bone-specific alkaline phosphatase (BALP)(Adult patients with XLH)(up to week 140)
- Concentration of serum alkaline phosphatase (ALP) (Pediatric patients with XLH)(up to week 140)
- Motor functions (6 minutes walk test (6MWT))(up to week 140)
- Radiographic findings of fracture and enthesopathy (Adult patients with XLH)(up to week 140)
- Rickets Severity Score (RSS) (Pediatric patients with XLH)(up to week 140)
- Radiographic Global Impression of Change (RGI-C)(Pediatric patients with XLH)(up to week 140)
- Z score of height (LMS method) (Pediatric patients with XLH)(up to week 140)
研究者
研究点 (10)
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