Clinical Trial: Albright Hereditary Osteodystrophy: Growth Hormone Trial and Cognitive/Behavioral Assessments
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: Study of Growth Hormone Use in Patients With Pseudohypoparathyroidism Type 1a and Assessment of Cognitive/Behavioral Status in Albright Hereditary Osteodystrophy
Brief Summary:
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
Detailed Summary:
Pseudohypoparathyroidism type 1a (PHP1a) is a disorder that causes many endocrine and developmental problems. To date, medical treatment has focused primarily on maintenance of normal serum levels of calcium, phosphorous, and thyroid hormone. However, these therapeutic interventions do not address the problems of short stature, obesity, and subcutaneous ossifications, which for many are a source of considerable morbidity and personal distress. These patients require frequent medical care, blood tests, and medication adjustments. PHP1a is an inherited condition with an estimated prevalence in the United States of 1:15,000- 20,000, and the studies that we propose provide an opportunity to improve the quality of life in affected patients. We have found that growth hormone (GH) deficiency is common in these patients, and our data suggest that GH testing should be part of their routine standard of care. We are investigating whether GH treatment can increase linear growth velocity and final adult height in children. We are also investigating whether GH treatment can reduce weight and improve a variety of metabolic disturbances and overall health in both children and adults.
GH deficiency not only leads to short stature and obesity, but also to osteoporosis, hyperlipidemia, depressed cardiac and renal function, as well as an overall lack of energy. It is quite possible that treatment of GH-deficient patients with PHP1a could improve any or all of the above problems. GH treatment has been FDA approved for use in both children and adults with GH deficiency. Therefore, it may be possible to provide improvement in health and overall quality of life in these patients.
Additionally, we have initiated a research study for which we are treating children with PHP1a who are not GH deficient (i.e., GH sufficient). The rationale is that GH t
Sponsor: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Current Primary Outcome:
- PHP1a: Effect of GH on height, growth velocity, final height in children. Effect on weight, BMI, lipids, self-esteem in all ages. [ Time Frame: until achieve final height (approximately 12-15 years) ]
- Cognitive and behavioral function in Albright hereditary osteodystrophy [ Time Frame: participant will be assessed on day 1; assessment may extend into day 2 ]
Original Primary Outcome: Effect of growth hormone treatment on height, growth velocity, body mass index, lipid profiles every 3 months; effect on percent body fat and bone mineral density annually
Current Secondary Outcome:
Original Secondary Outcome: As above
Information By: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Dates:
Date Received: September 13, 2005
Date Started: January 2003
Date Completion: December 2025
Last Updated: August 23, 2016
Last Verified: August 2016
