Efficacy and Safety of Burosumab Versus Oral Phosphate and Active Vitamin D Treatment in Pediatri… (NCT02915705) | Clinical Trial Compass
CompletedPhase 3
Efficacy and Safety of Burosumab Versus Oral Phosphate and Active Vitamin D Treatment in Pediatric Patients With XLH
United States61 participantsStarted 2016-09-08
Plain-language summary
The primary objective of this study is to evaluate the effect of KRN23 (burosumab) therapy in improving rickets in children with XLH compared with active control (oral phosphate/active vitamin D).
Who can participate
Age range1 Year – 12 Years
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, aged 1 to ≤12 years with radiographic evidence of rickets as determined by central readers
✓. Phosphate-regulating endopeptidase homolog, X-linked (PHEX) mutation or variant of uncertain significance in either the patient or in a directly related family member with appropriate X-linked inheritance
✓. Serum creatinine below the age-adjusted upper limit of normal
✓. Serum 25(OH)D above the lower limit of normal (≥16 ng/mL) at the Screening Visit
✓. Have received both oral phosphate and active vitamin D therapy for ≥ 12 consecutive months (for children ≥3 years of age) or ≥ 6 consecutive months (for children \<3 years of age) 7 days prior to the Randomization Visit
✓. Willing to provide access to prior medical records for the collection of historical growth and radiographic data and disease history
✓. Provide written or verbal assent (as appropriate for the subject and region) and written informed consent by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures.
Exclusion criteria
✕. Tanner stage 4 or higher in any of the following: genitals, breast, or pubic hair, based on physical examination
✕. Height percentile \> 50th based on country-specific norms
What they're measuring
1
Radiographic Global Impression of Change (RGI-C) Global Score at Week 40
. Use of aluminum hydroxide antacids (eg, Maalox® and Mylanta®), systemic corticosteroids, acetazolamide, and thiazides within 7 days prior to the Screening Visit
✕. Current or prior use of leuprorelin (eg, Lupron®, Viadur®, Eligard®), triptorelin (TRELSTAR®), goserelin (Zoladex®), or other drugs known to delay puberty
✕. Use of growth hormone therapy within 12 months before the Screening Visit
✕. Presence of nephrocalcinosis on renal ultrasound grade 4
✕. Planned orthopedic surgery, including osteotomy or implantation or removal of staples, 8 plates, or any other hardware, within the first 40 weeks of the study
✕. Hypocalcemia or hypercalcemia, defined as serum calcium levels outside the age-adjusted normal limits