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Sponsors and Collaborators: |
FDA OPD FDA Office of Orphan Products Development Johns Hopkins University |
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Information provided by: | FDA Office of Orphan Products Development |
ClinicalTrials.gov Identifier: | NCT00209235 |
We, the researchers, have found that growth hormone deficiency is very common in patients with pseudohypoparathyroidism type 1a. These patients 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, the researchers, are evaluating the effect of growth hormone treatment in those pseudohypoparathyroidism type 1a patients 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 patients with pseudohypoparathyroidism type 1a who are not growth hormone deficient and patients with pseudopseudohypoparathyroidism. Both pseudohypoparathyroidism type 1a and pseudopseudohypoparathyroidism are the 2 sub-types that make up a broader condition termed Albright hereditary osteodystrophy.
Condition | Intervention |
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Pseudohypoparathyroidism Pseudopseudohypoparathyroidism Albright Hereditary Osteodystrophy |
Drug: Growth hormone treatment |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Historical Control, Factorial Assignment, Safety/Efficacy Study |
Official Title: | Growth Hormone Use in Pseudohypoparathyroidism Type 1a |
Estimated Enrollment: | 100 |
Study Start Date: | January 2003 |
Estimated Primary Completion Date: | October 2011 (Final data collection date for primary outcome measure) |
Pseudohypoparathyroidism type 1a (PHP type 1a) 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 and obesity, which for many are a source of considerable morbidity and personal distress. These patients require frequent medical care, blood tests, and medication adjustments. PHP type 1a is an inherited condition with an estimated prevalence in the United States of 1:10,000- 50,000, 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 suggests that GH testing should be part of their routine standard of care. We are investigating whether GH treatment can increase height (in children), reduce weight, and improve a variety of metabolic disturbances and overall health.
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 PHP type 1a 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. We are currently not treating patients with PHP type 1a who are not GH deficient unless they qualify for GH treatment based on another FDA approved indication. Pseudopseudohypoparathyroidism, a condition also characterized by obesity and short final adult height, is not associated with hormone abnormalities including GH deficiency. However, it is possible that a subset of these patients may qualify for GH treatment based on another FDA approved indication, and certain patients may qualify for treatment on a research basis in the future.
Ages Eligible for Study: | 2 Months to 89 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | Yes |
Inclusion Criteria:
Exclusion Criteria:
Contact: Emily L Germain-Lee, MD | 410-955-6463 | egermain@jhmi.edu |
United States, Maryland | |
Johns Hopkins Hospital | Recruiting |
Baltimore, Maryland, United States, 21287 | |
Contact: Emily L Germain-Lee, MD 410-955-6463 egermain@jhmi.edu | |
Principal Investigator: Emily L Germain-Lee, MD |
Principal Investigator: | Emily L Germain-Lee, MD | Johns Hopkins University |
Responsible Party: | Johns Hopkins University School of Medicine ( Emily L. Germain-Lee, M.D.; Associate Professor ) |
Study ID Numbers: | FDA OPD R01 FD-R-002568, RPN: 82-02-24-01 (IRB), GCRC:NIH/NCRR(M01 RR00052), (CTSA for GCRC |
Study First Received: | September 13, 2005 |
Last Updated: | January 15, 2009 |
ClinicalTrials.gov Identifier: | NCT00209235 |
Health Authority: | United States: Food and Drug Administration |
Pseudohypoparathyroidism type 1a Pseudopseudohypoparathyroidism Albright Hereditary Osteodystrophy Growth Hormone Deficiency |
Hypopituitary dwarfism Metabolic Diseases Bone Diseases, Metabolic Vitamin D resistant rickets Dwarfism, Pituitary Albright's hereditary osteodystrophy Bone Diseases |
Growth hormone deficiency Metabolism, Inborn Errors Pseudohypoparathyroidism Musculoskeletal Diseases Genetic Diseases, Inborn Pseudopseudohypoparathyroidism Metabolic disorder |
Calcium Metabolism Disorders Metal Metabolism, Inborn Errors |