4 Clinical Trials for Various Conditions
There is a variety of tumors affecting the pituitary gland in childhood; some of these tumors (eg craniopharyngioma) are included among the most common central nervous system tumors in childhood. The gene(s) involved in the pathogenesis of these tumors are largely not known; their possible association with other developmental defects or inheritance pattern(s) has not been investigated. The present study serves as a (i) screening/training, and, (ii) a research protocol. As a screening and training study, this protocol allows our Institute to admit children with tumors of the hypothalamic-pituitary unit to the pediatric endocrine clinics and wards of the NIH Clinical Center for the purposes of (i)\<TAB\>training our fellows and students in the identification of genetic defects associated with pituitary tumor formation, and (ii)\<TAB\>teaching our fellows and students the recognition, management and complications of pituitary tumors As a research study, this protocol aims at (i)\<TAB\>developing new clinical studies for the recognition and therapy of pituitary tumors; as an example, two new studies have emerged within the context of this protocol: (a) investigation of a new research magnetic resonance imaging (MRI) tool and its usefulness in the identification of pituitary tumors, and (b) investigation of the psychological effects of cortisol secretion in pediatric patients with Cushing disease. Continuation of this protocol will eventually lead to new, separate protocols that will address all aspects of diagnosis of pituitary tumors and their therapy in childhood. (ii)\<TAB\>Identifying the genetic components of pituitary oncogenesis; those will be investigated by (a) studying the inheritance pattern of pituitary tumors in childhood and their possible association with other conditions in the families of the patients, and (ii) collecting tumor tissues and examining their molecular genetics. As with the clinical studies, the present protocol may help generate ideas for future studies on the treatment and clinical follow up of pediatric patients with tumors of the pituitary gland and, thus, lead to the development of better therapeutic regimens for these neoplasms.
The primary purpose of this study is to assess the effect of Debio 4126 in the maintenance of the levels of insulin-like growth factor 1 (IGF-1) ≤1x upper limit of normal (ULN) in the double-blind period (Period 1) in comparison to placebo at week 36.
Background: For children with gigantism, too much growth hormone (GH) in the body causes abnormal growth and many other problems. Current treatments often don t work; no medical treatment is approved by FDA. Researchers want to see if the drug pegvisomant can help. Objective: To test the role of pegvisomant in children and adolescents with gigantism. Eligibility: People ages 2-18 with GH excess for whom usual treatments have not worked or who are not eliginle for them Design: Participants will be screened with a medical history. The study will last 60 weeks and include at least 3 visits: baseline, 6-month, and 12-month visits. For the baseline visit, participants will stay a few nights for testing. They may stay overnight for the other visits. All visits will include: Medical history Physical exam Questionnaires Heart and liver tests Participants may be photographed in their underwear if they agree. Blood tests: Participants will get a catheter: A small plastic tube will be placed in an arm vein. For some tests, the blood may be drawn every 30 minutes over 3 hours. For other tests, blood will be drawn every 20 minutes over 12 hours. Only clinically necessary tests will be done in each patient. At the baseline visit, participants will have the study drug injected under the skin. They will learn to take the injection at home. They will take the injection daily during the study. The baseline and 12-month visits will include: MRI: Participants will have a dye injected into a vein. They will lie in a machine that takes pictures of the body. Hand X-ray Participants must get their height and weight at their local doctor s office monthly. Participants must have blood and urine tests at their local lab monthly for the first 6 months then every 3 months until the study ends. ...
If someone is diagnosed with a pituitary tumor that causes acromegaly (too much growth hormone) the treatment is to have it surgically removed. This study has two phases. The first phase provides medical treatment with a drug that will be provided for 3 months before surgery to see if complications of surgery are reduced and to see whether or not remission improves following surgery if you have this medical treatment. The drug administered is approved by the FDA for long-term treatment of acromegaly. It is not routinely administered before surgery, and is therefore experimental as used in this way. All other procedures performed during this research are standard of care with the exception of the 3 questionnaires to be completed at each visit. The second phase of this study is from 3 months until 12 months after surgery and is only for people who do not go into remission after the operation. This phase assesses the possible remission of acromegaly after resuming the drug treatment for an additional 3 to 9 months. The drug will be prescribed by your physician as part of your regular medical care and will not be included as part of the study. All other procedures performed during this research are standard of care with the exception of the 3 questionnaires to be completed at each visit. The study lasts approximately 16 months - 3 month before surgery and 12 months after surgery.