A Phase 1/2, Study to measure the safety, tolerability and the action of drugs in the body (method and rate of excretion; duration of effect on the body) and effectivity of Burosumab in children from birth to less than 1 year of age with inheritable form of rickets
- Conditions
- XLH is a rare, genetic disorder that is serious, chronically debilitating and represents an unmet medical need. This genetic deficiency is estimated to occur in about 1:20,000 live births (Burnett et al. 1964), (Imel et al. 2005). XLH is the most common inherited form of rickets and the most common inherited defect in renal tubular phosphate transport. XLH is transmitted as an X-linked dominant disorder (Dixon et al. 1998).MedDRA version: 20.0Level: LLTClassification code 10016206Term: Familial hypophosphataemic ricketsSystem Organ Class: 100000004850Therapeutic area: Body processes [G] - Bones and nerves physological processes [G11]
- Registration Number
- EUCTR2019-000469-19-SE
- Lead Sponsor
- Kyowa Kirin Pharmaceutical Development Ltd.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 20
Main Criteria for Inclusion:
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
Main Criteria for Exclusion:
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. Nutritional rickets and/or osteopenia to include exclusion of patients with metabolic
bone disease of other origin than XLH at Screening and/or Baseline.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method