A Trial to Compare the Efficacy and Safety of Once-weekly Lonapegsomatropin With Placebo and a Daily...
Growth Hormone DeficiencyEndocrine System Diseases1 moreA 38 week dosing trial of lonapegsomatropin, a long-acting growth hormone product, administered once-a-week versus placebo-control. A daily somatropin product arm is also included to assist clinical judgement on the trial results. Approximately 240 adults (males and females) with growth hormone deficiency will be included. Randomization will occur in a 1:1:1 ratio (lonapegsomatropin : placebo : daily somatropin product). This is a global trial that will be conducted in, but not limited to, the United States, Europe, and Asia.
Safety and Efficacy of Y-shape Pegylated Somatropin in Growth Hormone Deficiency Children
Growth Hormone DeficiencyThis is a multicenter, randomized, open-labeled, positive controlled phase 2&3 combined study to evaluate the safety and efficacy of weekly Y-shape pegylated somatropin, compared to daily somatropin (Norditropin®), in prepubertal, treatment-naive children with growth hormone deficiency.
Phase 1/2 Study of the Highly-selective RET Inhibitor, Pralsetinib (BLU-667), in Participants With...
RET-altered Non Small Cell Lung CancerMedullary Thyroid Cancer46 moreThis is a Phase 1/2, open-label, first-in-human (FIH) study designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary antineoplastic activity of pralsetinib (BLU-667) administered orally in participants with medullary thyroid cancer (MTC), RET-altered NSCLC and other RET-altered solid tumors.
Diagnosis of Pheochromocytoma
PheochromocytomaEndocrine Disease1 moreThe goal of this study is to develop better methods of diagnosis, localization, and treatment for pheochromocytomas. These tumors, which usually arise from the adrenal glands, are often difficult to detect with current methods. Pheochromocytomas release chemicals called catecholamines, causing high blood pressure. Undetected, the tumors can lead to severe medical consequences, including stroke, heart attack and sudden death, in situations that would normally pose little or no risk, such as surgery, general anesthesia or childbirth. Patients with pheochromocytoma may be eligible for this study. Candidates will be screened with a medical history and physical examination, electrocardiogram, and blood and urine tests. Study participants will undergo blood, urine, and imaging tests, described below, to detect pheochromocytoma. If a tumor is found, the patient will be offered surgery. If surgery is not feasible (for example, if there are multiple tumors that cannot be removed), evaluations will continue in follow-up visits. If the tumor cannot be found, the patient will be offered medical treatment and efforts to detect the tumor will continue. Main diagnostic and research tests may include the following: Blood tests - mainly measurements of plasma or urine catecholamines and metanephrines as well as methoxytyramine. If necessary the clonidine suppression test can be carried out. Standard imaging tests - Non-investigational imaging tests include computed tomography (CT), magnetic resonance imaging (MRI), sonography, and 123I-MIBG scintigraphy and FDG (positron emission tomography) PET/CT. These scans may be done before and/or after surgical removal of pheochromocytoma. Research PET scanning is done using an injection of radioactive compounds. Patients may undergo 18F-FDOPA, 18F-DA, as well as 68Ga-DOTATATE PET/CT . Each scan takes up to about 2 hours. Genetic testing - A small blood sample is collected for DNA analysis and other analyses....
Phenotype and Epidemiology of the Polycystic Ovary Syndrome (PCOS) in Colombia (PEP - Colombia)...
Polycystic Ovary SyndromeHyperandrogenism1 moreThe goal of this observational study is to know the prevalence of PCOS among economically productive and reproductive age women from Medellín and the Valle de Aburrá, Colombia. The main questions it aims to answer are: What is the phenotypic distribution of PCOS detected in women seeking medical attention as a requirement for employment in Medellín and the Valle de Aburrá, Colombia? What is the effect of environmental factors, such as geographical location and diet, and biological factors (such as obesity and ethnicity/race) on the prevalence and phenotype of PCOS in this populatión? Participants will undergo anthropometric measurements and physical examination for hirsutism, acne, alopecia, acanthosis nigricans, and thyroid enlargement. During the initial visit, a transvaginal or transabdominal pelvic ultrasonography will be performed. A sample of venous blood will be collected in plain tubes for serum cryopreservation and for immediate glucose estimation. Some participants will be rescheduled for a second evaluation visit for additional assessment when they have a possible PCOs.
Growth Hormone Replacement Therapy in Veterans With Mild Traumatic Brain Injury (mTBI) and Adult...
Adult Growth Hormone DeficiencyMild Traumatic Brain InjuryThe purpose of this study is to determine whether growth hormone replacement therapy (GHRT) is effective versus placebo in the improvement of Quality of Life in patients with adult growth hormone deficiency (AGHD) and mild traumatic brain injury (mTBI).
Spanish Series of Patients Treated With the Radionuclide Lutetium177
Neuroendocrine TumorsIntestinal Neoplasms18 moreThis study aims to pool the clinical experience of Spanish centers treating patients with 177Lu-DOTATATE to evaluate the efficacy, tolerance, and safety of the drug in routine clinical practice and to learn about the profiles of patients and tumors treated and the results in each type of patient and tumor.
Growth Hormone Deficiency in Mild Traumatic Brain Injury
Growth Hormone TreatmentGrowth Hormone Deficiency3 moreThe goal of this randomized control trial is to test if growth hormone therapy is a safe and effective treatment for patients suffering from growth hormone deficiency and persistent post-concussion symptoms. The main questions it aims to answer are: Is growth hormone therapy effective at mitigating persisting post-concussion symptoms in patients with growth hormone deficiency? Is it feasible to conduct a larger trial to examine efficacy of growth hormone therapy in patients with persistent post-concussion symptoms and growth hormone deficiency? Participants will be asked to complete an initial assessment for study inclusion and to complete clinical outcome questionnaires. If a participant meets study criteria they will be randomized to receive either growth hormone therapy (provided by Pfizer) or a placebo (provided by Pfizer). Participants will be instructed on how to self-administer their assigned drug daily for three months. Monthly follow-up visits will include a blood draw to measure a biomarker and clinical outcome questionnaires. At the final follow-up visit after three months, participants will learn what group they were assigned and given the option to complete the growth hormone therapy if they were originally assigned to the placebo group. Researchers will compare the growth hormone therapy group to the placebo group to identify any potential differences in outcomes.
Brain Imaging of Childhood Onset Psychiatric Disorders, Endocrine Disorders and Healthy Volunteers...
Attention Deficit Hyperactivity DisorderSchizophrenia1 moreMagnetic Resonance Imaging (MRI) unlike X-rays and CT-scans does not use radiation to create a picture. MRI use as the name implies, magnetism to create pictures with excellent anatomical resolution. Functional MRIs are diagnostic tests that allow doctors to not only view anatomy, but physiology and function. It is for these reasons that MRIs are excellent methods for studying the brain. In this study, researchers will use MRI to assess brain anatomy and function in X and Y chromosome variation, healthy volunteers, and patients with a variety of childhood onset psychiatric disorders. The disorders include attention deficit disorder, autism, congenital adrenal hyperplasia, childhood-onset schizophrenia, dyslexia, obsessive compulsive disorder, Sydenham's chorea, and Tourette's syndrome. Results of the MRIs showing the anatomy of the brain and brain function will be compared across age, sex (gender), and diagnostic groups. Correlations between brain and behavioral measures will be examined for normal and clinical populations.
Anlotinib Combined With Carboplatin/Paclitaxel as First-line Treatment in Patients With Advanced...
Ovarian NeoplasmsFallopian Tube Neoplasms17 moreIt has been reported that antiangiogenic drugs combined with chemotherapy as first-line treatment, and subsequent antiangiogenic drugs as maintenance therapy for ovarian cancer can achieve better clinical benefits. Therefore, this study is expected to investigate the efficacy and safety of anlotinib combined with carboplatin/paclitaxel as first-line treatment in patients with advanced ovarian cancer.