Principal Investigators
Bryan A. Dickson, M.D.
Michele R. Hutchison, M.D., Ph.D.
Ellen C. Kaizer, M.D.
Jon D. Oden, M.D.
Grace M. Tannin, M.D.
Perrin C. White, M.D. & James F. Marks, M.D., M.P.H.
Studies
A randomized, multi-center, double-blind, trial of Sandostatin LAR© Depot (40 mg) versus saline-control in the treatment of pediatric hypothalamic obesity with open label extension phase
Sponsor: Novartis
Status: Completed September 7, 2005
Principal Investigator: Bryan A. Dickson, M.D.
Study Coordinator: Martha Danton, R.N., B.S.N.
The purpose of this study was to see how this medicine lowers insulin levels in the body and potentially inducing weight loss in children who are at least 6 years of age but less than 18 years of age.
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KABI International Growth Study (KIGS)
Sponsor: Pfizer
Status: Active
Principal Investigator: Michele R. Hutchison, M.D., Ph.D.
Study Coordinator: Jennifer Klawinsky, R.N., B.S.N.
The primary objectives of the study are to assess the long-term safety of Genotropin and the growth responses of children treated with Genotropin to, thereby, establish a database suitable for the scientific investigation of questions that remain unanswered in this therapeutic area. Secondary objectives include comparative aspects of growth response variables among the treated US population with the International database and the tracking of the Genotropin convenience system.
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National Cooperative Growth Study (NCGS)
Sponsor: Genentech
Status: Active
Principal Investigator: Michele R. Hutchison, M.D., Ph.D.
Study Coordinator: Jennifer Klawinsky, R.N., B.S.N.
The purpose of this study is to collect long-term safety and efficacy information on Nutropin Depot, Nutropin AQ, Nutropin, and Protropin Growth Hormone (GH) regarding treatment of children who have growth failure due to lack of adequate endogenous GH secretion.
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Norditropin National Registry Program
Sponsor: Novo Nordisk
Status: Active
Principal Investigator: Michele R. Hutchison, M.D., Ph.D.
Study Coordinator: Jennifer Klawinsky, R.N., B.S.N.
The Norditropin© National Registry Program is a post-marketing registry of pediatric patients using Norditropin© therapy. Participating physicians will make all treatment decisions. The data collected in the Norditropin© National Registry Program will thus be observational as no treatment specifics will be mandated. A large body of data will be generated to meet the following objectives:
- To develop a pharmacodynamic model defining the relationship of Norditropin©, accounting for effects of known or suspected independent variables (e.g., age, gender, puberty).
- To develop a model defining the relationship of growth hormone (GH) dose and IGF exposure to treatment (height) outcomes, accounting for effects of known or suspected independent variables (e.g., age, gender, puberty).To develop a safety model that relates GH doses to adverse event occurrence, again accounting for the effects of known or suspected independent variables (e.g., GH dose, IGF-1 and IGFBP-3 responses, age, gender).
- To determine the relative predictive values of pre-treatment GH stimulation tests and centralized assays of pre-treatment IGF-1 and IGFBP-3 levels.
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Blood Gene Signatures in Pediatric Patients with Newly Diagnosed Insulin Dependent Diabetes Mellitus
Status: Active
Principal Investigator: Ellen C. Kaizer, M.D.
The purpose of this study is to analyze the blood gene signatures of newly diagnosed Type 1 diabetes patients.
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Blood Gene Signatures in Pediatric Patients with Newly Diagnosed Type 2 Diabetes
Status: Active
Principal Investigator: Ellen C. Kaizer, M.D.
The purpose of this study is to analyze the blood gene signatures of newly diagnosed Type II diabetes patients.
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Characterization of Autoreactive T cells in Type I Diabetes Patient
Status: Active
Principal Investigator: Ellen C. Kaizer, M.D.
The objective of this research study is to investigate whether there is an imbalance of islet-specific T cell subsets in type 1 diabetic patients as compared to healthy individuals.
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Role of Growth Hormone in the Development of Abnormal Glucose Tolerance in Obese Youth
Status: Pending
Principal Investigator: Jon D. Oden, M.D.
A rapidly growing number of U.S. children are overweight, which places them at increased risk of developing hypertension, dyslipidemia, coronary artery disease, and type 2 diabetes (T2DM). Initially, however, obese children destined to develop T2DM suffer from insulin resistance, a state of inefficient insulin action. However, not all insulin-resistant individuals develop T2DM. In fact, it has been estimated that only 6% of significantly obese individuals will contract the disease. However, as obesity and insulin resistance are common factors in those who do progress to T2DM, insulin resistance has become the target of much clinical investigation. Unfortunately, ways to reliably identify those subjects at highest risk for developing T2DM have yet to be identified.
Clinical observations suggest that growth hormone (GH) may adversely affect insulin sensitivity. The peak incidence of Type 1 diabetes occurs during puberty when endogenous growth hormone (GH) levels are highest. Conditions of GH excess such as acromegaly are associated with insulin resistance, which improves when the GH excess resolves. Interestingly, several sources have cited reduced GH concentrations in obese individuals. However, these studies did not document their subjects’ insulin sensitivity leaving room for question concerning GH role in obese children with insulin resistance.
Therefore, we propose a study comparing stimulated growth hormone values in obese children who are mildly or severely insulin resistant. We expect that severely insulin resistant children will have significantly higher GH values. If successful, our future investigation would be to follow our patient population to determine if these children will go on to develop T2DM. T2DM is a complex and poorly understood disease demanding further research into potential risk factors. A relative excess in endogenous GH could be one of those risk factors.
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Glycomark as a Screening Tool for Impaired Glucose Tolerance in Pediatric Obese Patients
Status: Pending
Principal Investigator: Jon D. Oden, M.D.
The purpose of this study is to determine whether Glycomark can discriminate between patients with and without impaired glucose tolerance in the obese, insulin resistant pediatric population.
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The Genetics of Neuroendocrinology of Short Stature International Study
Sponsor: Eli Lilly
Status: Active
Principal Investigator: Grace M. Tannin, M.D.
Study Coordinator: Shanda Grant
This is a multinational, muticenter, observational, post-marketing research program. The patient can not have been on any other form of growth hormone. Our facility is participating in 3 parts of the study outside of the mandatory Core Study.
The Core Study is evaluating the long-term effectiveness and efficacity of Humantrope. The child can be either naive to treatment and just starting or already on treatment.
The 1st arm of participation is the DNA Analysis. This Sub-Study is collecting blood samples from the participant and possibly the family members. This is to predict the impending hormone deficiencies, determine the disease status in siblings born after the patient, and genetic counseling.
The 2nd arm is Shox deficiency characterized by clinical , endocrine, and radiological features. These patients are identified as having Turner syndrome, Leri-Weil syndrome, Langer syndrome, or other condition with proven SHOX deletion or mutation.
The 3rd arm is the Idiopathic Short Stature (ISS) Sub Study. This is to characterized and understand patients with variations on genes, proteins, and other biomarkers that may be related to the growth in children with ISS.
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Duration of the Honeymoon Phase of Type 1 Diabetes: A Comparison of Insulins Detemir, Glargine and NPH
Sponsor: Novo Nordisk
Status: Active
Principal Investigator: James F. Marks, M.P.H.
Type 1 diabetes is a very common disorder affecting 1/400 persons by the age of 18 years in the United States alone. It is responsible for a disproportionate percentage of the morbidity and mortality associated with diabetes because of its typically young age of onset and difficulty achieving good glycemic control. Although exogenous administration of insulin allows for normal growth and development in children with this condition, it is not a cure. Studies which have attempted to modify the autoimmune process which underlies the development of hyperglycemia in patients with type 1 diabetes have focused on the preservation of the innate insulin secretory capacity of the pancreas as the primary outcome variable. Interventions that might preserve residual b-cell function and thereby improve glycemic control have the potential to have significant effects on long-term morbidity and mortality.
In recent years, a number of insulin analogs have been developed which have varying time-action profiles due to varying mechanisms of absorption. In comparison to children on the moderate-acting analog NPH, we have retrospective data to suggest that children placed on the long-acting analog glargine achieve significantly better average glycemic control for at least the first nine months after diagnosis of diabetes. This contrasts to children with long-standing diabetes who are switched from NPH to glargine, who show no significant change in Hemoglobin A1c values. Therefore, we propose a randomized trial in patients aged 6-18 years newly diagnosed with type 1 diabetes to address whether the choice of the longer-acting insulin affects the preservation of the innate insulin secretory capacity of the pancreas. In order to evaluate whether there is a difference in b-cell function between patients treated with basal bolus regimens (utilizing glargine or detemir) in comparison to multiple daily injections (utilizing NPH), we will perform mixed meal tolerance tests at study entry and again at 6 and 12 months, with C-peptide secretion as the primary outcome variable. If a difference is found, this could have significant implications for future intervention studies in patients with type 1 diabetes, as failing to control for insulin regimen could be a potential confounding variable in such studies.
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