Endocrinology, Diabetes and Metabolism Research

Endocrinology Research at Children's Hospital shows the important of exercising as children and adults.The Division of Pediatric Endocrinology, Diabetes and Metabolism at UPMC Children’s Hospital of Pittsburgh is world renowned for its 4 decades of Pediatric Endocrine research, particularly in the area of diabetes and carbohydrate-related metabolic disorders.

Research into the etiology and epidemiology of type 1 diabetes evaluates the immunologic, genetic and environmental determinants in a search for triggers that precipitate clinical diabetes. Prevention of clinical diabetes as well as the physical and psychosocial complications of diabetes and preservation of insulin secretory capacity are the ultimate goals of the research of both local populations and national and international collaborations. Our researchers were amongst the first to recognize the coexistence of features of type 1 and type 2 diabetes and coined the term “double diabetes” Both epidemiologic and intervention strategies are ongoing. In one of the major international intervention study, Children’s serves as the coordinating center for the United States. In that study, researchers are comparing use of hydrolyzed formulas in genetically susceptible infants. If the intervention is effective in delaying autoimmunity or its progression to diabetes, this first-ever primary prevention study of type 1 diabetes will have a far-reaching impact for individuals and the global society.

Type 2 diabetes, a disease once considered almost exclusive to adults, is increasing at an alarming rate among children. Research into the areas of hormones related to childhood obesity, insulin sensitivity/resistance, prediabetes and cardiovascular disease in youth has led to an increased awareness of this epidemic and racial differences in its pathogenesis. This resulted in validated patient care approaches leading to new trials aimed at type 2 diabetes intervention by  our nationally recognized faculty.

Beyond the scope of clinical diabetes, research is progressing in the study of growth hormone and its receptors in rodent models using a conditional knockout genetic approach. A number of clinical growth hormone studies supported by pharmaceutical research, include the GeNeSIS study with a primary goal of evaluating the long-term safety and efficacy of growth hormone use for children with endocrine or growth disorders with 5 substudies: DNA Analysis, Growth Prediction, SHOX Deficiency, Neoplasia and Idiopathic Short Stature. The idiopathic short stature study assesses the effect of different growth hormone doses in short children without growth hormone deficiency. The need to identify and characterize the clinical, endocrine and other features within this area take into consideration not only the physical patient care, but the psychosocial factors, stressing the important of this research, as endocrine disorders affect quality of life throughout the transition from childhood into adolescence.


Clinical Studies

Congenital Adrenal Hyperplasia Treatment with Crinecerfont – Phase III

Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Adult Subjects with Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment

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Diabetes Vaccine Study

Autologous Dendritic Cell Therapy for Type 1 Diabetes Suppression: A Safety Study

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Eli Lilly Growth Study Program

The Genetics and NeuroEndocrinology of Short Stature International Study (GeNeSIS) Consent For Data Collection: Core Program

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Genentech Growth Study Program

Genentech National Cooperative Growth Study Surveillance Program for Nutropin, Nutropin AQ and Protropin

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Pfizer Growth Study Program

KIGS: Pfizer International Growth Study for Genotropin®

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Search for a Cause of Diabetes

Etiology and Epidemiology of Type 1 Diabetes

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TEDDY Study - The Environmental Determinants of Diabetes in the Young

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TrialNet - Abatacept Drug Study

Effects of CTLA-4 Ig on The Progression of Type 1 Diabetes in New Onset Subjects

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TrialNet - Family Screening Study for Type 1 Diabetes

Screening and the Natural History Study of the Development of Type 1 Diabetes

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TrialNet - GAD - Alum Drug Study

Effects of GAD-Alum on the Progression of Type 1 Diabetes in New Onset Subjects

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TrialNet - Prevention of Type 1 Diabetes

Oral Insulin for Prevention of Diabetes in Relatives at Risk for Type 1 Diabetes Mellitus

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TrialNet - Type 1 Diabetes and Rituximab

The Effect of Rituximab on the Progression of Type 1 Diabetes

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