Calcitonin for Treating X-linked Hypophosphatemia

• Status: Completed
• Trial end date: 2015-09-01
• Target enrollment: 0
• Participant gender: All

Summary

X-linked hypophosphatemia (XLH) is the most common form of inherited rickets in the United States. It also causes bone disease in adults. XLH is caused by overproduction of a hormone call FGF23, which makes the body waste phosphate. This study is designed to determine if nasal calcitonin, an already approved drug in the US, can lower blood levels of FGF23 and reduce phosphate wasting in patients with XLH. In this study the investigators will:

1. Determine whether nasal calcitonin significantly lowers integrated 24-hour blood levels of FGF23 in patients with XLH.

2. Evaluate whether nasal calcitonin improves serum phosphate levels in XLH.

3. Assess whether nasal calcitonin improves blood levels of the active form of vitamin D and calcium absorption from the intestine.

4. Make sure that nasal calcitonin is safe and well tolerated.

• Phase: N/A
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Yale University
• Collaborator: National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
• Treatments: Calcitonin; Calcitonin Gene-Related Peptide; Katacalcin; Salmon calcitonin

Criteria

Inclusion Criteria:

• age ≥18 or greater

• an established diagnosis of XLH

• fasting serum calcium ≤10.5 mg/dl

• fasting PTH at time of screen Exclusion Criteria:

• estimated creatinine clearance < 60 cc/min and/or serum creatinine > 1.5 mg/dl;

• serum 25(OH)vitamin D < 30 ng/ml. Potential study subjects who have a serum 25(OH)vitamin D < 30 ng/ml will be supplemented with 25(OH)vitamin D to achieve a serum value > 30 ng/ml and then re- screened

• inability to comply with instructions and appropriate follow up visits

• treatment with agents that may skeletal metabolism such as glucocorticoids, bisphosphonates, denosumab, teriparatide, estrogen and anticonvulsants.