EUCTR2019-000469-19-DE进行中（未招募）1 期
A Phase 1/2, Open-label, Multicenter, Non-randomized Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Burosumab in Pediatric Patients from Birth to Less than 1 Year of Age with X-linked Hypophosphatemia (XLH)
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 20
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
- 性别
- All
入选标准
- •1. Male or female pediatric subjects, aged <12 months at burosumab treatment initiation.
- •2. Pediatric subjects with PHEX mutation or variant of uncertain significance in either the subject or a directly
- •related family member with appropriate X-linked inheritance.
- •3. Presenting serum phosphate levels below the age-specific LLN at Screening.
- •4. A legally authorized representative has provided written informed consent prior to any research-related procedures.
- •5. A legally authorized representative 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 required by the
- •study protocol, including providing access to prior medical records for the collection of historical growth,
- •biochemical, and radiographic data and disease history
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 20
- •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
- •1. The pediatric subject’s legally authorized representative is unwilling or unable to stop the subject’s treatment
- •with oral phosphate and/or pharmacologic vitamin D metabolite or analogue (e.g. calcitriol, alfacalcidol) for
- •at least 1 week before planned treatment start and for the duration of the study.
- •2. Preterm pediatric patients (defined as born before 37 weeks of pregnancy) with a chronological age of
- •<6 months. Enrolment of preterm pediatric patients with a chronological age =6 months must be confirmed
- •by the Study Medical Monitor before study entry.
- •3. Impairment of renal function measured as serum creatinine above the age-adjusted normal range and
- •estimated GFR (calculated using the Bedside Schwartz equation) below the age-adjusted normal range.
- •4. Presence of nephrocalcinosis on renal ultrasound
- •5. Hypocalcemia or hypercalcemia, defined as serum calcium levels outside the age-adjusted normal limits.
- •6. Presence of a concurrent disease or condition that would interfere with study participation or affect subject safety.
- •7. Predisposition to infection or known immunodeficiency.
- •8. Severe dermatological conditions over the available injection sites.
- •9. 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.
- •10. Metabolic bone disease, nutritional rickets and/or osteopenia of other origin than XLH at screening and/or baseline
- •11. Serum Levels of 25-hydroxyvitamin D (25(OH)D) below the LLN that are clinically significant in the opinion of the investigator
- •12 Evidence of hyperthyroidism not associated with XLH as determined by the investigator.
研究者
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