Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Burosumab in Patients … (NCT04188964) | Clinical Trial Compass
CompletedPhase 1/2
Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Burosumab in Patients Less Than 1 Year of Age
Austria, France, Italy16 participantsStarted 2020-02-26
Plain-language summary
A Phase 1/2, Open-label, Multicenter, Non-randomized Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Burosumab in Paediatric Patients from Birth to Less than 1 Year of Age with X-linked Hypophosphatemia (XLH)
Who can participate
Age range1 Year
SexALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
✓. Male or female pediatric subjects, aged \<12 months at burosumab treatment initiation.
✓. 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.
✓. Presenting serum phosphate levels below the age-specific LLN at Screening.
✓. A legally authorized representative has provided written informed consent prior to any research-related procedures.
✓. 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.
Exclusion criteria
✕. 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.
✕. 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.
✕. 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.
What they're measuring
1
To assess the safety and tolerability of Burosumab in paediatric subjects with X-linked Hypophosphatemia (XLH) starting treatment below 12 months of age
Timeframe: From Baseline to scheduled time points (measured throughout the study up to Week 48).