221 Clinical Trials for Various Conditions
Background: - Some growth disorders are caused by a change in genes. Genes are the instructions the body uses to function. Changes in genes often cause them not to work correctly. Researchers want to use a new technology called exome sequencing, to look at many genes at once. This is done by looking at DNA from blood or saliva in a lab. This method may help find the cause of disorders that researchers haven t been able to find using past methods. Objectives: - To better understand genetic causes of growth disorders. Eligibility: - Children and adults with growth disorders and their family members. Design: * Participants will give a small sample of blood and/or saliva. * Researchers will purify DNA from the sample. They will perform exome sequencing and other tests to look for changes in genes. Some participants may receive limited or no genetic tests. Researchers will let them know if exome sequencing is performed. * Participants may have a medical history, physical exam, and lab tests. They may have x-rays or ultrasound tests to study the disorder in their family. * Some participants may be recommended for a specific genetic test from a commercial lab. They may have to pay for that test. * Participants will be told about test results that relate to the growth disorder. This may happen up to years after the testing. They may have to give another blood and/or saliva sample. * Some participants may get results about other health conditions. This will only happen if the information would help the person or their family protect their health. They may have to give another blood and/or saliva sample.
This single-center, randomized controlled pilot study aims to evaluate the efficacy of TruHeight Growth Protein Shake on improving growth patterns in children aged 4-17 years. Over six months, participants will consume the protein shake daily, five times a week, with outcomes measured in terms of type X collagen concentration in urine, height via a stadiometer, and body composition using an INBODY device.
A prospective, randomized, open-label single-blinded study of 50 subjects with growth hormone deficiency, ages 5 to 15 years in which 25 subjects will initiate rhGH therapy at 0.3mg/kg/week and the remaining 25 subjects will initiate their rhGH treatment at 0.2 mg/kg/week for the first 12 months of treatment. Safety parameters, height velocity, and adult height prediction by bone age determination will be assessed at 4-month intervals for 1 year following the initiation of rhGH therapy.
This is a prospective interventional study designed for a single patient with a dominant-negative mutation in the growth hormone receptor gene (GHR) which results in increased levels of growth hormone binding protein (GHBP). The patient will receive escalating doses of growth hormone titrated to achieve an insulin like growth factor-1 level above the mean and then growth response to therapy will be monitored.
Postnatal growth failure occurs in up to 50% of very low birth weight (VLBW, \<1500 grams at birth) infants as assessed by discharge weight. This study will evaluate if a sodium supplementation algorithm guided by spot urine sodium measurements can improve postnatal growth.
This study evaluates the relationship between growth and stool microbiota in premature infants.
This study is conducted in Europe and the United States of America (USA). The aim of the study is to validate two measures for growth hormone deficiency in children, the Treatment Related Impact Measure of Childhood Growth Hormone Deficiency (TRIM-CGHD) and the Treatment Burden Measure of Childhood Growth Hormone Deficiency (TB-CGHD).
This trial is conducted in the United States of America (USA). The aim of the trial is to investigate the bioequivalence (the expected biological equivalence of two pharmaceutical drug products with identical active ingredient) of Norditropin® (somatropin) versus Genotropin® (somatropin) in healthy adult subjects.
After approval of amendment (g), patients who were still receiving study drug at the time were scheduled for a study visit. In addition, patients who had discontinued early from the core, blinded phase of the study were contacted. All of these patients were offered the opportunity to enter the unblinded extension phase (if they met eligibility criteria) and continue somatropin treatment (regardless of initial treatment randomization) until they reached final height.
This study is intended to determine whether twice daily weight based dosing with recombinant human insulin-like growth factor (rhIGF-1) will safely and effectively increase the growth of prepubertal children with short stature associated with low IGF-1 levels but who produce sufficient growth hormone (GH). Subjects will be randomized to either an observation arm or to active treatment.
Background: Endocrine glands give off hormones. Researchers want to learn more about the disorders that affect these glands in children. These disorders might be caused by changes in genes. Genes contain DNA, which is the blueprint of how a cell works. Researchers want to identify the genes involved in endocrine and metabolic disorders. This might help develop new ways to diagnose and treat the disorders. Objective: To study the inheritance of endocrine or metabolism disorders. Eligibility: Children ages 3month-18 with known or suspected endocrine or metabolism disorders. Family members ages 3months-100. They may participate in the DNA part of the study. Design: Participants will be screened with a review of their medical records. Their parents or guardians will allow the records to be released. Participants will have a clinic visit. This may include a physical exam and medical history. Parents or guardians will give their consent for the study. Participants may have tests, surgery, or other procedures to help diagnose or treat their condition. These could include: Blood, urine, and saliva tests Growth hormone test Pituitary and adrenal function tests Picture of chromosomes Imaging tests. These may include X-ray, ultrasound, scans, or a skeletal survey. Genetic tests Sleep study Medical photographs If surgery is done, a tissue sample will be taken. Participants may have follow-up visits for diagnosis and treatment. Participating relatives will have one visit. This will include medical history and blood and saliva tests. The blood and saliva will be used for DNA testing.
Background: - PIK3CA-related overgrowth spectrum (PROS) is caused by changes in the PIK3CA gene. This gene makes a protein that communicates with other proteins in the body to cause cells to grow. Alterations in PIK3CA change the chemical signals in the body and cause overgrowth in fatty, vascular and other tissues. Sirolimus is a drug that reduces the signals sent by one of the proteins in this chemical signaling pathway. Researchers want to learn whether the drug sirolimus can reduce or stabilize some of the overgrowth that patients with PROS experience. Objectives: - To measure how the overgrowth of patients with PROS changes over time and whether taking a drug called sirolimus can reduce or stabilize a person s overgrowth. Eligibility: - People ages 3 to 65 years old with a confirmed mutation or alteration of the PIK3CA gene in the person s affected tissues (a somatic mutation). Design: * Participants will be screened with medical history and genetic counseling. * First 6 months: Participants will have their overgrowth monitored. * Next 6 months: Participants will take sirolimus once or twice a day. * Participants will have to visit the clinic several times, and stay in the area for 4 to 5 days each time. * Participants will have a one month-long visit to the clinic. * During clinic visits, participants will have: * Blood and urine tests. * Photographs of their physical features. * Scans, including an MRI and DEXA, and possibly x-rays and CT scans. * For the MRI and CT scans, participants will lie in a machine that takes pictures of their body. * The DEXA involves a small amount of radiation. * They may have: * Non-invasive heart function tests. * Lung function tests. * Participants will have several blood and urine tests between visits. * Participants will complete surveys and keep a diary of their treatment and side effects. * Participants may visit other health specialists or undergo other tests based on side effects. * One month after stopping the study drug, participants will have 1 clinic visit.
This trial is conducted in United States of America (USA). The aim of this trial is to examine the bioequivalence of Norditropin® versus Genotropin® in healthy adult volunteers.
This trial is conducted in the United States of America (USA). The purpose of the trial is to compare the effect of Norditropin® using different dosing regimens in children suspected of growth hormone deficiency.
The purpose of the study is to find out if patients with constitutional delay of growth have a mismatch between energy intake and utilization (as measured by doubly labeled water) and to compare the improvement in growth and energy balance between a group of boys treated with growth hormone (GH) alone versus those given GH plus added nutritional supplements for one year.
This study is intended to assess the effects of once daily dosing of recombinant human insulin-like growth factor (rhIGF-1) in increasing height velocity.
Children with extreme short stature (height) and their families often experience significant psychological stress related to concerns about adult height. In addition, short stature often results in life-long emotional, social, and physical obstacles to the affected person. Normal growth occurs in two phases. The first phase, known as childhood growth, occurs below the age of 10. The second phase of growth, teen-age or adolescent growth, begins between the ages of 10 and 15. In addition, puberty marks the time when the bone's growth plates (epiphysis) begin to close, initiating the completion of linear growth (height). Some children suffer from a condition called precocious puberty, meaning that puberty begins at a younger age than normal. The development of medications known as synthetic LHRH analogs have provided a method to delay puberty and treat these patients. LHRHa (deslorelin) is a hormone created to act like naturally occurring LHRH. It been used in patient's diagnosed with precocious (early onset) puberty. The drugs were able to regress patient's clinical signs of puberty, decrease the levels of adult sex hormones produced, and slow the rate of bone aging.
This study will examine rare congenital disorders that involve malformations and abnormal growth. It will focus on patients with Proteus syndrome, whose physical features are characterized by overgrowth, benign tumors of fatty tissue or blood vessels, asymmetric arms or legs, and large feet with very thick soles. The study will explore the genetic and biochemical cause and course of the disease, the changes in symptoms over time, and the effects of the disease on patients. Patients with Proteus syndrome may be eligible for this study. Study candidates will have a medical history and physical examination, including X-rays and possibly other imaging tests, such as computerized tomography (CT), magnetic resonance imaging (MRI) and ultrasound. Other tests and examinations may be done if needed. Those enrolled in the study will have will be interviewed or complete questionnaires, or both, about how their disease affects them. Patients will provide a small blood sample for research and may be asked to undergo biopsies from a normal area of skin and from a tumor. ...
This trial is conducted globally. The aim of the trial is to investigate efficacy and safety of once-weekly NNC0195-0092 (somapacitan) treatment compared to daily growth hormone treatment (Norditropin® FlexPro®) in growth hormone treatment naïve pre-pubertal children with growth hormone deficiency. The trial consists of a 26 week main trial period, followed by a 26 week extension trial period, a 104 week safety extension period, a 208 week longterm safety extension trial period and a 30 day follow up period. Participants receive NNC0195-0092 (somapacitan) (0.04 mg/kg/week) during the main trial and the extension period and thereafter NNC0195-0092 (somapacitan) (0.16 mg/kg/week) during the safety extension and the long-term safety extension periods. Two additional age groups, cohort II (age below 2 years and 26 weeks at screening) and cohort III (above 9 years (girls)/ above 10 years (boys) and equal to or below 17 years at screening) are included in the 208 week long-term safety extension trial period only.
This study is conducted globally. The purpose is to demonstrate the efficacy of once weekly dosing of NNC0195-0092 (somapacitan) compared to placebo and once-daily dosing of somatropin (human growth hormone, hGH) after 35 weeks of treatment in adults with growth hormone deficiency.
This study is conducted in the United States of America (USA). The aim of the study is to assess the minimal important difference (MID) of the TRIM-AGHD.
This trial is conducted in the United States of America (USA). The aim of this trial is to evaluate the safety profiles of Norditropin® (lyophilized somatropin) and Norditropin® cartridges (liquid somatropin) in children with growth hormone deficiency.
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.
This trial was conducted in the United States of America (USA). The aim of this clinical trial was to investigate the pharmacokinetics of somatropin in healthy Japanese and Caucasian subjects, and to identify somatostatin-related adverse events.
This trial is conducted in the United States of America (USA). This is a 12 month study to determine if Norditropin is safe and effective in children with IGF deficiency.
This project is designed to answer the question: Is there an acute IGFBP-3 response in normal children? Our specific hypothesis states that under the influence of growth hormone secretagogues, intact IGFBP-3 molecule will undergo proteolysis and liberate IGFBP-3 fragments, along with other components of the ternary complex. This proteolysis will result in measurable rise in IGFBP-3, which will indicate the subject's growth hormone status. Short children with growth hormone deficiency will not show an IGFBP-3 response.
This is a prospective, open label, interventional trial beginning with a phase 1b safety run-in followed by an expansion cohort.
Alterations in the intrauterine environment can have profound effects on fetal development. Diabetes during gestation results in multiple deleterious short-term outcome differences, and is correlated with long-term developmental deficits. Multiple studies, in neonates through school-aged children, have demonstrated differences in language, attention and psychomotor development in offspring of diabetic pregnancies. Neonatal EEG is a promising and non-invasive tool for assessment of abnormal brain development or "dysmaturity" in this population. Multiple conventional EEG (cEEG) and amplitude-integrated EEG (aEEG) parameters change predictably with advancing gestational development and have been used to differentiate between at risk groups in neonatal studies. The investigators hypothesize that neonatal EEG can identify brain dysmaturity in infants of diabetic mothers (IDMs) compared to gestational-age matched controls. The primary aim is documentation of brain dysmaturity in IDMs using cEEG. The secondary aim is establishment of aEEG as a more accessible tool to quantify the effects of maternal diabetes on neonatal brain development. The investigators will conduct a pilot study comparing cEEG and aEEG parameters of cases to gestational-age matched controls. Cases will be IDM neonates of at least 35 weeks' gestation whose mothers were recommended treatment with either insulin or an oral glycemic agent. Video EEG recording will be planned for approximately 60 minutes and obtained between 24 hours and 5 days of life during birth hospitalization. Additional data will be extracted from maternal and neonatal medical records and a maternal questionnaire. In addition to evaluating the measures of cEEG and aEEG, this project will establish a research cohort. A subsequent study involving developmental evaluations will allow for correlation of EEG results with long-term outcomes. The ability to identify those at risk at birth would provide the opportunity to intervene in order to mitigate outcome differences, particularly in language development. More significantly, we hope to establish neonatal CNS outcome measures for future diabetic pregnancy intervention studies. .
This research study will find out if a new growth hormone stimulation test is safe and works as well as other tests to diagnose growth hormone deficiency (GHD) in children. The stimulation test will use a new growth hormone stimulating substance called macimorelin. By now, only adults in the USA can get this new stimulation test. The results of this study are expected to help children and teenagers with suspected GHD to get the macimorelin stimulation test. The macimorelin test will be compared to a clonidine and an arginine test. Both are known standard stimulation tests. Altogether two macimorelin tests are planned to be performed in the study, to show how repeatable macimorelin tests results are (under a set of similar conditions).
This study will determine whether the negative effects of combined oral contraceptive (COC) therapy on the growth hormone/insulin-like growth factor-1 (GH/IGF-1) axis and bone turnover are dependent on the route of administration such that an attenuation of these effects is observed when a comparable dose of non-oral transdermal contraceptive (TDC) and contraceptive vaginal ring therapy (CVR) are also tested.