Ignite Creation Date:
2024-05-05 @ 12:00 PM
Last Modification Date:
2024-10-26 @ 9:18 AM
Study NCT ID:
NCT00209235
Status:
RECRUITING
Last Update Posted:
2024-05-07
First Post:
2005-09-13
Brief Title:
Albright Hereditary Osteodystrophy Natural History Growth and CognitiveBehavioral Assessments
Sponsor:
Connecticut Childrens Medical Center
Organization:
Connecticut Childrens Medical Center
Study Overview
Official Title:
Natural History Study of Albright Hereditary Osteodystrophy Includes Substudies on Effects of Growth Hormone in Patients With Pseudohypoparathyroidism Type 1A and Cognitive Behavioral Studies in Albright Hereditary Osteodystrophy
Status:
RECRUITING
Status Verified Date:
2024-05
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
We the researchers are following the natural history of Albright hereditary osteodystrophy We 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 under R01 FD002568 IND 67148 which ended those who are growth hormone sufficient and were found to have a positive clinical response to growth hormone in a prior clinical trial under R01 FD00FD003409 IND 67148 which ended or those 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 Cognitivebehavior NICHD R21 HD078864 which has ended
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 Cognitivebehavior NICHD R21 HD078864 which has ended
Detailed Description:
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 115000- 20000 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 final adult height 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 completed a study in which we treated children with PHP1A who are not GH deficient ie GH sufficient The rationale is that GH treatment could maximize linear growth velocity prior to the premature bone fusion that occurs in this condition and potentially improve final adult height The supply of growth hormone has ended for this study and we are following those participants who were in this study and received the growth hormone supply Some of these patients remain on growth hormone as per clinical care secondary to their responses
This study also seeks to define the specific neurocognitive and psychosocial disabilities in individuals with AHO in order to develop therapies and improve quality of life AHO includes two subtypes pseudohypoparathyroidism type 1A PHP1A and pseudopseudohypoparathyroidism PPHP
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 completed a study in which we treated children with PHP1A who are not GH deficient ie GH sufficient The rationale is that GH treatment could maximize linear growth velocity prior to the premature bone fusion that occurs in this condition and potentially improve final adult height The supply of growth hormone has ended for this study and we are following those participants who were in this study and received the growth hormone supply Some of these patients remain on growth hormone as per clinical care secondary to their responses
This study also seeks to define the specific neurocognitive and psychosocial disabilities in individuals with AHO in order to develop therapies and improve quality of life AHO includes two subtypes pseudohypoparathyroidism type 1A PHP1A and pseudopseudohypoparathyroidism PPHP
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| R21HD078864 | NIH | None | httpsreporternihgovquickSearchR21HD078864 |