A Phase 1/2 interventional study of Burosumab in X-linked Hypophosphatemia (XLH), sponsored by Kyowa Kirin Pharmaceutical Development Ltd. Completed at 9 sites in 6 countries. Open to participants aged Up to 1 Year. Per ClinicalTrials.gov, last updated 2024-11-07.
Sponsored by Kyowa Kirin Pharmaceutical Development Ltd (part of Kyowa Kirin Co., Ltd.) · Phase 1/2, Interventional, and Treatment
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)
BUR-CL207 is a multicenter, open-label, non-randomized Phase 1/2 study in pediatric patients with XLH initiating treatment with burosumab at \<12 months of age. The study includes a total treatment period of up to 48 weeks across 3 cohorts. Subjects will be enrolled in 2 age subgroups: (1) ≥6 months to \<12 months of age, and (2) \<6 months of age at initiation of burosumab treatment. Cohorts will aim to include 3 subjects per cohort depending on the starting dose of burosumab and relative response of patients to treatment as assessed by serum phosphate levels and by the treating physician (Investigator). Cohorts will commence in a staggered manner starting with Cohort 1, followed by Cohorts 2 and 3, which may start in parallel after an adequate observation period (4 weeks) in Cohort 1 and with approval from the Sponsor's Medical Monitor and the DSMB. The cohorts are defined as follows:
49 studies on the registry are indexed under Familial Hypophosphatemic Rickets; 8 are open to participants now.
This study's enrollment of 16 is below the median of 28 across 29 interventional studies indexed under Familial Hypophosphatemic Rickets.
Browse Familial Hypophosphatemic Rickets studies →Kyowa Kirin Pharmaceutical Development Ltd is the lead sponsor of 13 studies on the registry; 1 is open to participants now.
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Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Pediatric subjects \> = 6 months to \< 12 months will receive a starting dose of 0.4mg/kg administered subcutaneously (SC) every 2 weeks (Q2W) for 48 weeks with the option of the dose to be increased to 0.8mg/kg upon recommendation of the Data Safety Management Board (DSMB). The dose can be either increased up to a maximum of 2 mg/kg or decreased to 0.2 mg/kg depending on serum phosphate response. Upon recommendation of the DSMB subjects \< 6 months can then start at 0.4mg/kg starting dose administered subcutaneously (SC) every 2 weeks (Q2W) for 48 weeks with the option to be increased to 0.8mg/kg upon recommendation of the DSMB and can be either increased up to a maximum of 2 mg/kg or decreased to 0.2 mg/kg depending on serum phosphate response.
Drug: Burosumab
Burosumab is a sterile clear colourless to slightly yellow and preservative free solution supplied in single use 5ml vials containing 1 mL of Burosumab at a concentration of 10mg/mL,20 mg/mL or 30mg/mL, administered by SC injections every 2 weeks.
Also known as: KRN23, Crysvita
To assess the safety and tolerability of Burosumab in paediatric subjects with X-linked Hypophosphatemia (XLH) starting treatment below 12 months of age
Incidence, frequency, and severity of adverse events (AEs) and serious AEs (SAEs), including clinically significant changes in laboratory, physical examinations, vital signs, ECGs and imaging assessments
Time frame: From Baseline to scheduled time points (measured throughout the study up to Week 48).
To characterize the pharmacokinetics (PK) of Burosumab following subcutaneous (SC) injection in paediatric subjects with XLH below 12 months of age.
Burosumab serum concentrations and PK parameters, including apparent clearance (CL/F), apparent volume of distribution (V/F), area under the serum concentration-time curve (AUC), maximum serum drug concentration (Cmax) and other parameters, as appropriate.
Time frame: Measured throughout the study up to Week 48
To characterize the effect of Burosumab on serum phosphate and 1,25-dihydroxyvitamin D (1,25[OH]2D) in paediatric subjects with XLH starting treatment below 12 months of age
Changes in serum phosphate and 1,25-dihydroxyvitamin D (1,25\[OH\]2D)
Time frame: Change from Baseline at Week 20, 26, 32, 40 and 48
To assess the clinical effects of Burosumab on growth and prevention and/or healing of rickets and skeletal deformities
Change in serum alkaline phosphatase (ALP).
Time frame: Baseline and Week 48
To assess the clinical effects of Burosumab on growth and prevention and/or healing of rickets and skeletal deformities
Appearance in radiographic appearance of rickets severity as assessed by the Radiograph Global Impression of Change (RGI-C) scoring system.
Time frame: At week 48
To assess the clinical effects of burosumab on growth and prevention and/or healing of rickets and skeletal deformities
The appearance in rickets severity assessed by total Rickets Severity Score (RSS).
Time frame: At week 48
To assess the clinical effects of burosumab on growth and prevention and/or healing of rickets and skeletal deformities
Change in lower extremity skeletal abnormalities, including genu varum and genu valgus, as determined by the RGI-C long leg score.
Time frame: At week 48
To assess the clinical effects of burosumab on growth and prevention and/or healing of rickets and skeletal deformities
Change in recumbent length in cm, height-for-age z-scores, and percentiles.
Time frame: At week 48
Plan to share: No
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Ran from Feb 2020 to primary completion in Oct 2023, 43 months.
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This study is completed, as verified in Nov 2024. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Burosumab
Familial Hypophosphatemic Rickets →
Kyowa Kirin Pharmaceutical Development Ltd →