Treatment Trials

47 Clinical Trials for Various Conditions

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Novel Mediators of the Lipodystrophy and Metabolic Consequences of Cushing's Disease
Description

This proposal will evaluate the glucocorticoid mediated changes in body fat distribution and metabolism that occur in patients with Cushing's disease. The objective is to identify the mechanisms that influence both the accumulation of lipodystrophic fat and also the changes in energy expenditure and metabolism that accompany them. The study is designed to determine if the high cortisol and AgRP levels in the blood of people living with Cushing's syndrome, either from taking steroid medications or from tumors, impact body fat and metabolism by turning off brown fat, which is a type of fat that increases one's metabolism.

Conditions

Cushing's Syndrome

Efficacy and Safety Evaluation of Osilodrostat in Cushing's Disease
Description

The purpose of this study was to confirm efficacy and safety of osilodrostat for the treatment of patients with Cushing's disease who are candidates for medical therapy.

Conditions

Cushing's Disease

Long Term Safety and Efficacy of Pasireotide s.c. in Patients With Cushing's Disease
Description

This is a non-interventional, multinational, multi-center post-marketing study, to further document the safety and efficacy of pasireotide s.c. administered in routine clinical practice in patients with Cushing's disease. Patients with Cushing's disease and treated with pasireotide s.c. alone and in combination with other therapies will be monitored. For this study, each enrolled patient will be followed up for 3 years after enrollment. Patients who permanently discontinue pasireotide s.c. prior to completing the 3-year observation period will be followed up for 3 months after the last dose of pasireotide s.c.

Conditions

Cushings Disease

Safety and Efficacy of LCI699 for the Treatment of Patients With Cushing's Disease
Description

The study aimed to confirm long-term efficacy and safety of LCI699 for the treatment of patients with Cushing's disease. It was a pivotal trial which supported the registration of LCI699 for the treatment of patients with Cushing's disease in the US and the EU. This is a phase lll, multi-center, double-blind, randomized withdrawal study of LCI699 following a 24 week, single-arm, open-label dose titration and treatment period which evaluated the safety and efficacy of LCI699 for the treatment of patients with Cushing's disease.

Conditions

Cushings Disease

Treatment of Cushing's Disease With R-roscovitine
Description

The investigators hypothesize that R-roscovitine will suppress pituitary corticotroph tumor ACTH production and normalize urinary free cortisol levels in patients with Cushing disease. To date, R-roscovitine has been evaluated in several Phase I and II studies and has shown early signs of anti-cancer activity in approximately 240 patients.

Conditions

Cushings Disease

Study of Management of Pasireotide-induced Hyperglycemia in Adult Patients With Cushing's Disease or Acromegaly
Description

The study was designed to investigate the optimal management of hyperglycemia developed during pasireotide treatment in participants with Cushing's disease or Acromegaly, which was not manageable with metformin. This was a Phase IV, multi-center, randomized, open-label study. Eligible patients started pasireotide subcutaneously (s.c.) for Cushing's disease and pasireotide LAR (long-acting release) for Acromegaly. Participants being treated with pasireotide s.c or LAR at screening were eligible as long as they met protocol criteria during the screening period. If previously normo-glycemic participants experienced an increase in their fasting blood glucose and met the criteria for diabetes while on pasireotide, they started anti-diabetic treatment using metformin. If they continued to have elevated blood glucose above target on metformin within the first 16 weeks, they were randomized in a 1:1 ratio to receive treatment with incretin based therapy or insulin for approximately 16 weeks. Participants who continued to receive clinical benefit after completing the Core Phase could enter an optional Extension Phase if pasireotide was not commercially available in their country or a local access program was not available to provide drug. Patients continued in the Extension Phase until the last participant randomized in the Core Phase completed 16 weeks of treatment post-randomization.

Conditions

Cushing's Disease, Acromegaly

Mifepristone in Children With Refractory Cushing's Disease
Description

Study objectives are to obtain safety, pharmacokinetic, and pharmacodynamic data on the effect of mifepristone on glucose metabolism, body weight and the growth-hormone-IGF in children with refractory Cushing's disease.

Conditions

Cushing's Disease

Study of the Efficacy and Safety of Pasireotide s.c. +/- Cabergoline in Patients With Cushing's Disease
Description

The main purpose of this prospective, multicenter, open-label phase II study, was to evaluate the efficacy and safety of pasireotide alone or in combination with cabergoline in patients with Cushing's disease.

Conditions

Cushings Disease

An Open-label, Multi-center, Expanded Access Study of Pasireotide s.c. in Patients With Cushing's Disease.
Description

This study provided access to pasireotide sc in patients with Cushing's disease.and provided additional information for safety and efficacy of pasireotide s.c.

Conditions

Cushing's Disease

Efficacy and Safety of Pasireotide Administered Monthly in Patients With Cushing's Disease
Description

This is a randomized, double-blind, multicenter, phase III study to evaluate the safety and efficacy of 2 dosing regiments of Pasireotide long acting release (LAR) in patients with Cushing's disease.

Conditions

Cushing's Disease

Safety and Efficacy of LCI699 in Cushing's Disease Patients
Description

This exploratory study is a proof of concept study to determine whether LCI699 can safely reduce the level of urinary free cortisol in patients with Cushing's disease. In addition, this study evaluated the long term efficacy and safety of LCI699 including an additional 12 week of treatment followed by a 12 month long term optional extension. A second extension provided patients who were clinically benefitting from LCI699 an opportunity to continue to have access to the drug until LCI699 was commercially available and reimbursed or through the availability of a local access program.

Conditions

Cushings Disease, Cushing Disease

Preoperative Bexarotene Treatment for Cushing's Disease
Description

The objective of this pilot study is to establish the safety and tolerability of short-term therapy with bexarotene in patient's with Cushing's disease, and study the clinical, biochemical, and cellular effects of a preoperative five-day course of bexarotene in these patients before undergoing transsphenoidal surgery.

Conditions

Cushing's Disease

Rosiglitazone in Treating Patients With Newly Diagnosed ACTH-Secreting Pituitary Tumor (Cushing Disease)
Description

RATIONALE: Rosiglitazone may help pituitary tumor cells become more like normal cells, and to grow and spread more slowly. PURPOSE: This phase II trial is studying how well rosiglitazone works in treating patients with newly diagnosed ACTH-secreting pituitary tumor (Cushing disease).

Conditions

Cushing's Disease

Safety and Efficacy of Different Dose Levels of Pasireotide in Patients With de Novo, Persistent or Recurrent Cushing's Disease
Description

This study will evaluate the safety and efficacy of two different doses of Pasireotide in patients with de novo or recurrent/persistent Cushing's Disease.

Conditions

Cushing's Disease

Extension Study to Assess the Safety and Efficacy of Pasireotide in Participants With Cushing's Disease
Description

Cushing's disease is a rare serious condition that is caused by an adrenocorticotropic hormone (ACTH) secreting pituitary adenoma. This study assessed the long-term safety and efficacy of pasireotide in participants with Cushing's disease.

Conditions

Cushing Disease

A Study to Assess SOM230 in Patients With Pituitary Cushing's Disease
Description

The study treatment period is 15 days in length and includes patients with pituitary Cushing's disease who are candidates for surgical intervention as well as and patients who have recurrent Cushing's post operatively.

Conditions

Cushing's Syndrome

Cognition, Steroids, and Imaging in Cushings Disease
Description

This study investigates the effects of the glucocorticoid hormone cortisol on brain structure and function. Patients with Cushing's disease are studied before and after treatment. Brain imaging and neuropsychologic tests are used to study changes in the hippocampus and thinking and learning functions as well as mood during the period of elevated cortisol. At several intervals after treatment, these are reexamined to study the degree of reversibility of the abnormalities. The contribution of cortisol as well as testosterone and estrogen to dysfunction and recovery is studied. Since elevated cortisol and dysregulation of its secretory system occurs in a significant proportion of the aged and in Alzheimers Disease and Major Depressive Disorder, these studies will help advance knowledge of the role of cortisol in these conditions.

Conditions

Cushing Syndrome

Fimepinostat, Combination HDAC and Pi3-kinase Inhibitor Tumor-Directed Therapy for Cushing Disease
Description

Supported by the pre-clinical data (summarized in Research Strategy), the investigators propose that Fimepinostat is an ideal candidate drug in the treatment and intervention of patients with Cushing Disease. The investigators propose a pilot, short-term (4 weeks) phase II single-center study to demonstrate the safety and efficacy of Fimepinostat in the treatment of patients with de novo, persistent, and/or recurrent CD recruited at the University of California, Los Angeles. The trial will have a 2-arm design and will simultaneously examine two different doses of Fimepinostat. The study will allow the investigators to determine the efficacy and safety of these doses in the treatment of CD and guide dose selection for subsequent, larger studies.

Conditions

Cushing Disease

Long-Term Follow-Up of Survivors of Pediatric Cushing Disease
Description

Background: The pituitary gland produces hormones. A tumor in this gland can cause it to produce too much of the hormone cortisol. Too much cortisol in the body causes Cushing disease. This disease causes many problems. Some of these problems might persist after the disease is cured. Objective: To find out the long-term effects of exposure to high levels of cortisol during childhood and adolescence. Eligibility: People ages 10-42years who were diagnosed with Cushing disease before age 21 and are now cured and have normal or low cortisol levels People related to someone with Cushing disease Design: Participants will be screened with a medical history. Participants will complete an online survey. This will include questions about their or their child s physical and mental health. All participants will be seen at 5 -year intervals after cure of Cushing disease (5yr, 10yr, 15yr, 20yr (last visit)) Participants who have a relative with Cushing disease will have a medical history and blood tests or cheek swabs. Participants who have the disease will have: Physical exam Blood tests Cheek swab DXA scan: A machine will x-ray the participant s body to measure bone mineral content. For participants who are still growing, a hand x-ray Participants with the disease may also have: Hormone stimulation test: Participants will get a hormone or another substance that will be measured. Serial hormone sampling: Participants blood will be measured several times through a thin plastic tube in an arm vein. Urine tests: Participants urine may be collected over 24 hours. MRI: Participants may have a dye injected into a vein. They will lie on a table that slides into a machine. The machine will take pictures of the body. ...

Conditions

Cushing Disease

Multicenter Study of Seliciclib (R-roscovitine) for Cushing Disease
Description

This phase 2 multicenter, open-label clinical trial will evaluate safety and efficacy of 4 weeks of oral seliciclib in patients with newly diagnosed, persistent, or recurrent Cushing disease. Funding Source - FDA Office of Orphan Products Development (OOPD)

Conditions

Cushing Disease

Corticotrophin-releasing Hormone (CRH) Stimulation for 18F-FDG-PET Detection of Pituitary Adenoma in Cushing s Disease
Description

Background: Cushing s disease is caused by a pituitary gland tumor. Patients with Cushing s disease suffer obesity, diabetes, osteoporosis, weakness, and hypertension. The cure is surgery to remove the pituitary tumor. Currently, MRI is the best way to find these tumors. But not all tumors can be seen with an MRI. Researchers hope giving the hormone CRH before a PET scan can help make these tumors more visible. Objective: To test whether giving CRH before a PET scan will help find pituitary gland tumors that might be causing Cushing s disease. Eligibility: People ages 8 and older with Cushing s disease that is caused by a pituitary gland tumor that cannot be reliably seen on MRI Design: Participants will be screened with their medical history, a physical exam, an MRI, and blood tests. Participants will have at least one hospital visit. During their time in the hospital, they will have a physical exam and a neurological exam. They will have a PET scan of the brain. A thin plastic tube will be inserted into an arm vein. A small amount of radioactive sugar and CRH will be injected through the tube. Participants will lie in a darkened room for about an hour and be asked to urinate. Then they will lie inside the scanner for about 40 minutes. After the scan, they will be asked to urinate every 2-3 hours for the rest of the day. Blood will be drawn through a needle in the arm. Participants will have surgery to remove their tumor within 3 months after the scan. Participants will then continue regular follow-up in the clinic.

Conditions

Cushing's Disease, Pituitary Adenoma

Effect of Vorinostat on ACTH Producing Pituitary Adenomas in Cushing s Disease
Description

Background: Cushing s disease is caused by excess ACTH hormone release by a benign tumor of the pituitary gland. It can lead to decreased quality of life and early death. The current best treatment for Cushing s disease is surgery. If surgery does not work or if the tumor returns, there are no more good treatment options. Vorinostat, which is approved to treat a type of lymphoma, might be a treatment option. Objective: To test vorinostat to see if it can kill tumor cells and change the number of hormones released in people with Cushing s disease. Eligibility: People ages 18 and older who have Cushing s disease and are scheduled for surgery under protocol 03-N-0164 to remove a tumor in their pituitary gland Design: Participants will be screened under protocol 03-N-0164. Participants will stay in the hospital for 8 days before their surgery. On the first day, participants will have a physical exam and blood tests. They will have their urine collected for testing all day. They will have an ECG: For this, small metal disks or sticky electrode pads will be placed on their chest to record heart activity. For the next 7 days, participants will have blood tests and all-day urine collection. They will drink at least 2 liters of fluid per day. They will take the study drug by mouth each morning. On the eighth day, participants will have their surgery. Leftover tissue will be collected for research. On the day they are discharged from the hospital, participants will have a physical exam and blood tests.

Conditions

Cushing's Disease

Collecting Information About Treatment Results for Patients With Cushing's Syndrome
Description

The purpose of this study is to follow participants with Cushing's syndrome during the course of their routine care and to form a data registry to study long term participant outcomes.

Conditions

Cushing's Disease, Cushing Syndrome, Cushing Disease

Study to Allow Access to Pasireotide for Patients Benefiting From Pasireotide Treatment in Novartis-sponsored Studies
Description

The 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.

Conditions

Cushing's Disease, Acromegaly, Neuroendocrine Tumors, Pituitary Tumors, Ectopic ACTH Secreting (EAS) Tumors, Dumping Syndrome, Prostate Cancer, Melanoma Negative for bRAF, Melanoma Negative for nRAS

Compassionate Use of CORLUX® (Mifepristone) in the Treatment of Signs and Symptoms of Endogenous Cushing's Syndrome
Description

This is a compassionate use study. In addition to providing compassionate use access to mifepristone, objectives of the study will be to evaluate the safety and utility of mifepristone in the treatment of the signs and symptoms of endogenous Cushing's syndrome when given on a compassionate use basis. The study will only enroll subjects whose physicians have determined that medical treatment is needed to control the symptoms or signs of hypercortisolemia.

Conditions

Cushing's Disease, Cushing's Syndrome

Long-term Cardiovascular Risk in Cured Cushing's Patients
Description

The purpose of this study is to determine whether subjects who have been affected by Cushing's disease in the past maintain some different characteristics compared with subjects who have never been diagnosed with the disease.

Conditions

Cushing's Disease

A Study to Evaluate the Safety and PK of CRN04894 for the Treatment of Cushing's Syndrome
Description

A Phase 1b/2a, first-in-disease, open-label, multiple-ascending dose exploratory study to evaluate safety, tolerability, pharmacokinetics (PK), and pharmacodynamic biomarker responses associated with CRN04894 (an adrenocorticotropic hormone \[ACTH\] receptor antagonist) in participants with ACTH-dependent Cushing's syndrome (Cushing's disease or Ectopic ACTH Syndrome \[EAS\])

Conditions

Cushing Syndrome, Cushing Disease, Ectopic ACTH Syndrome

SPI-62 as a Treatment for Adrenocorticotropic Hormone-dependent Cushing's Syndrome
Description

This is a randomized, placebo-controlled, study of SPI-62 in subjects with ACTH-dependent Cushing's syndrome caused by a non-adrenal tumor. Subjects will receive each of the following 2 treatments for 24 weeks: SPI-62 and matching placebo with the option of long-term extension.

Conditions

Cushing's Syndrome I, Cushing Disease Due to Increased ACTH Secretion, Cortisol Excess, Cortisol; Hypersecretion, Cortisol Overproduction, Ectopic ACTH Secretion

Open-label Treatment in Cushing's Syndrome
Description

This is a long-term, open-label extension study of levoketoconazole in subjects with endogenous Cushing's Syndrome.

Conditions

Cushing Syndrome, Cushing Disease

Rhinological Outcomes in Endonasal Pituitary Surgery
Description

This is a prospective, multi-center observational study designed to address patient-reported nasal outcomes in adults undergoing endoscopic and microscopic surgical removal of pituitary tumors. The primary objective of this study is to determine the difference in nasal outcomes by using the Anterior Skull Base (ASK) Nasal survey between patients treated with endoscopic surgical technique and those treated with microscopic surgical technique. Patients will be given the ASK Nasal survey to assess their nasal functioning and overall health before their surgery, and at post-operative visits 1-2 weeks, 3 months, and 6 months.

Conditions

Pituitary Neoplasm, Pituitary Adenoma, Prolactinoma, Cushings Disease, Acromegaly