National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
N/A
2011-03-01
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.
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.
Some people with hypothyroidism have persistent symptoms despite adequate treatment with
thyroid hormones. We are testing whether giving calcitonin to such people will improve their
quality of life.
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