View clinical trials related to Dwarfism.
Filter by:GeNeSIS is an open-label, multinational, multicenter, observational study to evaluate the safety and effectiveness of Humatrope treatment. GeNeSIS is a modular program that includes: - Core study: Evaluating the safety and effectiveness of Humatrope in the observational setting - Genetic Analysis Sub-study: Investigating the genetic defects underlying growth hormone (GH) deficiency and non-GH-deficient growth disorders - Growth Prediction Sub-study: Working to validate and refine specific models to accurately predict growth response to GH - Short Stature Homeobox containing gene (SHOX) Deficiency Sub-study: Elucidating the clinical, endocrine and radiological features of participants with SHOX deficiency due to loss of, or mutation in the SHOX gene (including participants with Turner syndrome) - Neoplasia Sub-study: To characterize the natural history of neoplastic disease, especially in relation to recurrence/progression of primary neoplasia or development of secondary neoplasia in children with a history of neoplasia
The investigators hypothesize that low serum ghrelin levels may characterize a group of patients with poor weight gain and/or linear growth who do not have any other identified cause for growth failure. These patients may present with a variety of complaints and are often evaluated by both pediatric endocrinologists and pediatric gastroenterologists. The investigators hypothesize that ghrelin has a physiologically important role in linear growth and that chronic diseases of the gastrointestinal system, such as H. Pylori infection or celiac disease, may alter serum ghrelin levels in children. Low ghrelin levels may be a factor leading to poor growth, potentially by altering growth hormone secretion and/or by decreasing appetite. By measuring ghrelin levels in children with short stature and in children with gastrointestinal disease, the investigators will further elucidate the possible physiologic role of ghrelin in childhood growth and how it may be altered in conditions causing short stature and in certain gastrointestinal diseases.
The objective of this study is to evaluate the efficacy and safety of recombinant human growth hormone (r-hGH) treatment in girls with Turner Syndrome under the age of 4 years. After 4 years of treatment, height in these girls will be compared with an historical control group of untreated girls with Turner Syndrome, matched for age and height at baseline.
This study is conducted in the United States of America (USA). The aim of this observational study is to collect data concerning the treatment outcomes and safety for children and adults who are prescribed Norditropin®. Specific objectives include: 1) developing models defining the relationship of Norditropin dose to changes in insulin-like growth factor (IGF-I) and treatment outcomes, accounting for independent factors such as age, gender and puberty and 2) determining the relative predictive values of peak growth hormone (GH) and IGF-I levels and other factors before treatment to clinical outcomes.
Assessment of Genotropin patient and caregiver (Dyad) perception of convenience and preference of Genotropin injection pen. Patients already on genotropin will be asked to use a genotropin pen for 2 months. Patient and caregiver will be asked to complete a questionnaire at baseline and 2 months.
Estrogens are responsible for the disappearance of growth cartilage in the long bones at the end of the pubertal growth spurt both in boys and in girls. It is therefore hypothesized that stopping pubertal development and hence estrogen production, will prolong and increase the pubertal growth spurt, especially when growth hormone is given concommitantly. Boys in early puberty, with a bone age between 11 and 13 years and a predicted adult height below 163 cm or girls in early puberty with a bone age between 10 and 12 years and a predicted height under 151 cm will be treated with triptorelin 3.75 mg and Zomacton growth hormone for 4 years.
Obesity, now a global epidemic, is a leading cause of illness and mortality in the developed world. To better understand the pathophysiological mechanisms that underlie weight disorders, increasing attention is being paid to central regulatory elements in energy homeostasis, including food intake and energy expenditure. The human hormone ghrelin is secreted as a preprohormone (preproghrelin), from which two hormones with antagonistic effects are derived: ghrelin, which has orexigenic effects and obestatin, which has anorexigenic effects. Ghrelin's actions are mediated by GH secretagogue receptor (GHSR). Ghrelin synthesis occurs predominantly in epithelial cells of the fundus of the stomach. . As the ligand for GHSR, ghrelin stimulates secretion of GH. In both rodents and humans, ghrelin regulates hunger though its action on hypothalamic feeding centers. Other effects of ghrelin include stimulating gastric emptying, positive effects on cardiovascular function, increasing intestinal peristalsis, and positive exocrine and paracrine pancreatic secretion. Despite its important physiological role, its precise regulatory mechanisms remain ambiguous. Thus, it has been suggested that mutations in ghrelin and its receptor will present clinically with obesity, eating disorders or growth disturbances. To date, only four different mutations have been reported in GHSR and no mutations have been found in the ghrelin gene. Working hypothesis and aims: We hypothesize that mutations in ghrelin or in its receptor, GHSR, affect appetite regulation and cause growth and eating disorders.
The purpose of the protocol is to describe the distribution of IGF-1 deficiency in the studied population of Idiopathic Short Children without Growth Hormone Deficiency or any other identified cause of short stature and not treated with recombinant Growth Hormone or IGF-1
IGF-1 (insulin-like growth factor-1) is a hormone that is normally produced in the body in response to another hormone called growth hormone. Growth Hormone is produced by a small gland at the base of the brain (the pituitary). Together IGF-1 and GH are large contributors to growth during infancy, childhood, and adolescence. Children with IGF Deficiency are short and have an imbalance in the levels of growth hormone and IGF-1 that their body produces. Their growth hormone levels are normal or even high, but IGF-1 levels do not increase normally in response to growth hormone. As a result, they have a type of growth hormone insensitivity and an inability to grow normally. This study is a test to see whether daily dosing with a combination of rhIGF-1 and rhGH will help children with IGFD grow taller more quickly than children treated with rhGH alone. The study medications, rhIGF-1 and rhGH, are approved by the US Food and Drug Administration (FDA) for use in some growth disorders in children, but the combination of rhIGF-1 and rhGH in children with IGF-1 deficiency (IGFD) is investigational.
This is a two arm, randomized, prospective, intervention study in order to determine the effects of growth hormone treatment on eating regulation and to compare between the growth responses with or without nutritional intervention in short stature children. The study will include 30 short stature children that are about to initiate growth hormone treatment and will last for one year. After 4 months of treatment, children will be randomized into two groups: 1. Control group that will continue with growth hormone treatment without any other intervention. 2. Study group that will be a given a nutritional intervention in addition to growth hormone treatment. At screening visit and during the study the following parameters will be evaluated: height, weight, growth markers in the blood and urine, child eating behavior questionnaire, blood tests, hormonal tests and resting energy expenditure measurements.