Trial intelligence / Profile preview

Efficacy and Safety of Burosumab in Pediatric Chinese Patients With XLH

NCT04842032Phase 4

Key facts

Status: Completed
Start: Nov 2021
Primary completion: Dec 2023
Countries: 1
Sponsor: Kyowa Kirin
Modality: Monoclonal Antibodies

Drugs: burosumab
Conditions: X-linked hypophosphatemia (XLH)

Overview

Phase 4, open-label, single-arm trial of burosumab in X-linked hypophosphatemia (XLH), sponsored by Kyowa Kirin. Primary endpoint: Change from Baseline in mean serum phosphorus level. Started Nov 2021; status: completed.

Design

1 arm

Study design: Non-randomizedOpen-labelSingle group

KRN23
Patients receive KRN23 after conventional therapy run-in.

Who it enrols

Pediatric patients (1-12 years) with confirmed XLH (PHEX mutation or high FGF23) and radiographic evidence of rickets. Patients must have hypophosphatemia and be able to receive conventional therapy during run-in. Excludes patients with Tanner stage >=4, height percentile >50%, severe nephrocalcinosis, or hyperparathyroidism.

Endpoints

Primary

Change from Baseline in mean serum phosphorus levelWeeks 2, 4, 8, 12, 16, 24, 32, 40, 52, 64

Secondary

Radiograph Global Impression of Change global scoreWeeks 40, 64
Rickets Severity Score total scoreWeeks 40, 64
RGI-C long leg scoreWeeks 40, 64
Change in serum 1,25-dihydroxyvitamin Dup to Week 74
Change in alkaline phosphataseup to Week 74
Change in urinary phosphorusup to Week 74
Change in ratio of renal tubular maximum reabsorption rate of phosphate to glomerular filtration rateup to Week 74
Change in recumbent length/standing height and Z-scoresWeeks 0, 24, 40, 64

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