Examining the Effect of Burosumab on Muscle Function

• Status: Recruiting
• Trial end date: 2021-11-01
• Target enrollment: 0
• Participant gender: All

Summary

Patients with X-linked hypophosphatemia (XLH) often report symptoms of fatigue and weakness particularly after exertion, in addition to their skeletal complaints. In previous trials using KRN23 (same drug as burosumab/Crysvita®), patients report these symptoms improve. The investigators wish to test this hypothesis directly by measuring muscle energy when patients begin treatment with Crysvita® for the first time.

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Yale University
• Treatments: Antibodies, Monoclonal

Criteria

Inclusion Criteria:

1. 18-65 years of age

2. Diagnosis of XLH

3. eGFR ≥ 50

4. Normal serum calcium

5. Phosphate ≤ 2.5 mg/dl

6. Deemed clinically appropriate for starting therapy with Burosumab/Crysvita® (based on the treating physician's evaluation)

7. Deemed appropriate for MR Spectroscopy

Exclusion Criteria:

1. Patients with fixed skeletal abnormalities which would prevent them from successfully completing study-related functional assessments

2. Patients unwilling to stop therapy with supplemental phosphate and calcitriol 2 weeks prior to enrollment.

3. Patients who have undergone an orthopaedic procedure within the previous 6 months involving implantation of metal hardware