Study of KRN23 (Burosumab), a Recombinant Fully Human Monoclonal Antibody Against Fibroblast Grow… (NCT02163577) | Clinical Trial Compass
CompletedPhase 2
Study of KRN23 (Burosumab), a Recombinant Fully Human Monoclonal Antibody Against Fibroblast Growth Factor 23 (FGF23), in Pediatric Subjects With X-linked Hypophosphatemia (XLH)
United States, France, Netherlands52 participantsStarted 2014-07-02
Plain-language summary
The objectives of the study are to:
* Identify a dose and dosing regimen of burosumab, based on safety and pharmacodynamic (PD) effect, in pediatric XLH participants
* Establish the safety profile of burosumab for the treatment of children with XLH including ectopic mineralization risk, cardiovascular effects, and immunogenicity profile
* Characterize the pharmacokinetic (PK)/PD profile of the KRN23 doses tested in the monthly (Q4) and biweekly (Q2) dose regimens in pediatric XLH patients
* Determine the PD effects of burosumab treatment on markers of bone health in pediatric XLH patients
* Obtain a preliminary assessment of the clinical effects of burosumab on bone health and deformity, muscle strength, and motor function
* Obtain a preliminary assessment of the effects of burosumab on participant-reported outcomes, including pain, disability, and quality of life in pediatric XLH patients
* Evaluate the long-term safety and efficacy of burosumab
Who can participate
Age range
5 Years – 12 Years
Sex
ALL
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 5 - 12 years, inclusive, with open growth plates
. Tanner stage of 2 or less based on breast and testicular development
. Diagnosis of XLH supported by ONE of the following:
. Biochemical findings associated with XLH including:
. Standing height \< 50th percentile for age and gender using local normative data.
. Radiographic evidence of active bone disease including rickets in the wrists and/or knees, AND/OR femoral/tibial bowing, OR, for expansion subjects, a Rickets Severity Score (RSS) score in the knee of at least 1.5 as determined by central read.
. Willing to provide access to prior medical records for the collection of historical growth, biochemical and radiographic data, and disease history.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Change From Baseline in RSS Total Score Over Time
Timeframe: Baseline, Week 40, 64, 160
2
Change From Baseline in Serum Phosphorus Over Time
Timeframe: Baseline, Week 40, 64, 160
3
Change From Baseline in Serum 1,25(OH)2D Over Time
. Provide written or verbal assent (if possible) 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
. Use of a pharmacologic vitamin D metabolite or analog (e.g. calcitriol, doxercalciferol, alfacalcidiol, and paricalcitol) within 14 days prior to Screening Visit 2; washout will take place during the Screening Period
. Use of oral phosphate within 7 days prior to Screening Visit 2; washout will take place during the Screening Period
. Use of calcimimetics, aluminum hydroxide antacids (e.g. Maalox® and Mylanta®), systemic corticosteroids, and thiazides within 7 days prior to Screening Visit 1
. Use of growth hormone therapy within 3 months before Screening Visit 1
. Use of bisphosphonates for 6 months or more in the 2 years prior to Screening Visit 1
. Presence of nephrocalcinosis on renal ultrasound graded ≥ 3 based on the following scale: 0 = Normal 1 = Faint hyperechogenic rim around the medullary pyramids 2 = More intense echogenic rim with echoes faintly filling the entire pyramid 3 = Uniformly intense echoes throughout the pyramid 4 = Stone formation: solitary focus of echoes at the tip of the pyramid
. Planned or recommended orthopedic surgery, including staples, 8-plates or osteotomy, within the clinical trial period
. Hypocalcemia or hypercalcemia, defined as serum calcium levels outside the age-adjusted normal limits \*