ECE2020 Audio ePoster Presentations Bone and Calcium (121 abstracts)
Phase 3b open-label study of burosumab in adults with X-linked hypophosphatemia (XLH): Baseline and Week 12 results
Peter Kamenický 1 , Kassim Javaid 2 , Richard Keen 3 , Robin Lachmann 4 , Stuart Ralston 5 , Martine Cohen-Solal 6 , Maria Brandi 7 , Karine Briot 8 , Rachel Crowley 9 , Jennifer Walsh 10 , Sami Kolta 8 , Angela Rylands 11 , Wei Sun 12 & Annabel Nixon 13
1CHU de Bicêtre, France; 2University of Oxford; 3Royal National Orthopaedic Hospital, Stanmore, United Kingdom; 4University College of London Hospital, London, United Kingdom; 5University of Edinburgh, Edinburgh, United Kingdom; 6Hôpital Lariboisière, Paris, France; 7Azienda ospedaliera universitaria Careggi, Florence, Italy; 8Assistance Publique – Hôpitaux de Paris, Paris, France; 9St. Vincent’s University Hospital, Dublin, Ireland; 10Northern General Hospital, Sheffield, United Kingdom; 11Kyowa Kirin International, Marlow, United Kingdom; 12Kyowa Kirin Pharmaceutical Development, Princeton NJ, United States; 13Chilli Consultancy, Salisbury, United Kingdom
Introduction: Burosumab, a fully human monoclonal antibody to fibroblast growth factor 23 (FGF23), is the only approved treatment for XLH, a rare genetic disorder characterized by renal phosphate wasting and substantial cumulative musculoskeletal morbidity. BUR02 (NCT03920072) is a European phase 3b open-label study monitoring the long-term safety and efficacy of burosumab in adults with XLH from sites who participated in the CL303/CL304 studies (NCT02526160/02537431).
Objective: To describe the patients in the open label follow-up and the first 12 weeks data from BUR02.
Methods: Forty-eight adults with XLH who participated in CL303/CL304 studies from EU sites were invited to take part in BUR02 and continue to receive burosumab every 4 weeks. Thirty-seven patients are expected to be recruited in total. Patients attended the clinic at baseline and week 12 for measurement of serum phosphate (primary endpoint) and patient-relevant secondary endpoints, and collection of safety data.
Results: Baseline and Week 12 data from BUR02 are available for 27 subjects (median age 46 years [range 22–63 years]; 66.7% female).At BUR02 baseline their median height was 150 cm, height z-score -2.2, BMI 25.6 kg/m2 and 96.3% had a pathogenic PHEX gene variant, 52% were taking pain medication, 81% had joint pain and 67% had received prior orthopaedic surgery. Median serum phosphate was 0.7 mmol/l (2.3 mg/dl) at baseline and 0.8 mmol/l (2.4 mg/dl) at Week 12. Median Brief Pain Inventory (BPI) scores at baseline and Week 12 were 5.8 and 6.0 for worst pain, and 3.0 and 3.6 for pain interference. Median Brief Fatigue Inventory (BFI) scores were 5.8 and 6.0 for worst fatigue, and 2.7 at both timepoints for fatigue interference. Median Western Ontario and McMaster Universities Osteoarthritis Index (WOMAC) scores were 45.6 and 36.8 for physical functioning, 50.0 at both timepoints for stiffness, 45.0 at both timepoints for pain, and 46.9 and 41.7 for total score respectively. Median Short-form-36 v2 scores at Week 12 were 39.0 for the physical component score (PCS) and 49.3 for the mental component score (MCS).Median 6-minute walk test (6MWT) distance was 412 m at baseline (58.8% predicted) and 423 m (62.9%) at Week 12. There were no serious adverse events in the first 12 weeks of BUR02.
Conclusion: Early analysis of the first 12 weeks of BUR02 data demonstrates that continuation of burosumab from the CL303/304 studies into BUR02 sustains the correction of serum phosphate levels and maintains clinical benefits in patients.
Volume 70
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Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
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