Albright Hereditary Osteodystrophy: Growth Hormone Trial and Cognitive/Behavioral Assessments
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Phase 2/Phase 3
We, the researchers, have found that growth hormone deficiency is very common in patients
with pseudohypoparathyroidism type 1a, which falls under the broader condition termed
Albright hereditary osteodystrophy. Patients with pseudohypoparathyroidism type 1a typically
are short and obese. Some of these patients are not short during childhood, but due to a
combination of factors, they end up short as adults. We are evaluating the effect of growth
hormone treatment in those patients with pseudohypoparathyroidism type 1a who are found to
be growth hormone deficient. We hypothesize that growth hormone deficiency may contribute to
the short stature and obesity found in this condition. We are also evaluating the effect of
growth hormone on patients with pseudohypoparathyroidism type 1a who are not growth hormone
deficient (i.e., growth hormone sufficient) in those who had been on study drug through R01
FD003409 or who meet the criteria of idiopathic short stature or SGA.
We are also evaluating neurocognitive and psychosocial functioning in participants with AHO
in order to determine the specific impairments that are most common in the condition and to
determine the best approach toward management.
Funding source -- Growth hormone study: FDA OOPD [R01 FD003409 (which has ended) and R01
FD002568 (which has ended)] Cognitive/behavior: NICHD R21 HD078864
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