Safety and Efficacy of Pegvisomant in Children With Growth Hormone Excess
Pituitary DiseaseBackground: 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. ...
A Study to Assess the Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Debio 4126...
AcromegalyGEP-NETThis is an open-label, single treatment arm, multicenter study to assess the pharmacokinetics (PK), pharmacodynamics (PD), safety, and tolerability of Debio 4126 in the treatment of participants with Acromegaly or Functioning Gastroenteropancreatic Neuroendocrine tumors (GEP-NETs).
Diet in the Management of Acromegaly
AcromegalyImpact of low carbohydrate and low gluten diet on acromegaly progression, symptoms, complications, and treatment outcomes.
A Study to Evaluate the Long-Term Safety and Efficacy of Paltusotine for the Treatment of Acromegaly...
AcromegalyA phase 2, open label, long-term extension study designed to evaluate the safety and efficacy of paltusotine (formerly CRN00808; an oral selective nonpeptide somatostatin receptor type 2 biased agonist) in subjects with acromegaly.
Study to Allow Access to Pasireotide for Patients Benefiting From Pasireotide Treatment in Novartis-sponsored...
Cushing's DiseaseAcromegaly7 moreThe purpose of this study is to allow continued use of pasireotide in patients who are on pasireotide treatment in a Novartis-sponsored study and are benefiting from the treatment as judged by the investigator.
A Study to Evaluate the Safety and Efficacy of Paltusotine for the Treatment of Acromegaly
AcromegalyA randomized, placebo-controlled study designed to evaluate the safety and efficacy of paltusotine (also known as CRN00808; an orally administered nonpeptide somatostatin agonist) in subjects with acromegaly previously treated with somatostatin receptor ligand (SRL) based treatment regimens.
A Trial to Assess the Long-term Safety of Octreotide Subcutaneous Depot in Patients With Acromegaly...
AcromegalyThe purpose of this trial is to assess the long-term safety and efficacy of CAM2029 in patients with acromegaly. Patients will be administered CAM2029 subcutaneously once monthly during 12 months. Patients fulfilling trial NCT04076462 will be offered to continue with open-label treatment week 24-52 in this trial. Patients completing the main part of the trial will be offered 52 weeks continued open-label treatment in an extension part.
A Study to Evaluate the Safety and Efficacy of Paltusotine for the Treatment of Acromegaly (PATHFNDR-2)...
AcromegalyA randomized, placebo-controlled study designed to evaluate the safety and efficacy of paltusotine (formerly CRN00808; an oral selective nonpeptide somatostatin receptor type 2 biased agonist) in subjects with non-pharmacologically treated acromegaly.
Effects of Therapies in the Acromegaly Disease: Acral Morpho-functional Study
AcromegalyAcromegaly is a rare chronic disease due to excessive secretion of growth hormone (GH) and insulin-like growth factor-I (IGF-I), caused in over 98% of cases by GH-secreting pituitary adenoma. Prolonged exposure to GH/IGF-I excess is the cause of increased mortality and morbidity in these patients. Arthropathy occurs in about 75% of acromegalic patients. Any joint may be affected, with the development of osteoarthritis, arthralgia, and an increase in fracture risk. The aims of the present project are to evaluate the dimensions of hands and feet with the 3D scanner method and to perform a quantitative analysis of movement through Gait Analysis technique in de novo patients with acromegaly (group # 1) and in patients with different disease status (group #2).
Developing a Simple Recognition System of Acromegaly
AcromegalyWe will compare the features of 3D stereophotography of acromegaly patients with that of healthy people. We hope to develop a computerized model to help screening acromegaly patients for early detection and treatment.