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A Phase 3, Double Blind, Randomized, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Setmelanotide in Patients With Acquired Hypothalamic Obesity
This is a sub-study of Study RM-493-040 (NCT05774756). The goal of this sub-study is to learn how well Setmelanotide works to improve weight reduction, hunger…
Pancreatic Cancer Early Detection Consortium
The purpose of the Pancreatic Cancer Early Detection (PRECEDE) Consortium is to conduct research on multiple aspects of early detection and prevention of pancr…
Genotype-guided Comprehensive Medication Management to Improve Depression Outcomes in Pennsylvania
The goal of this prospective, randomized clinical trial is to learn whether pharmacogenomic (PGx)-guided comprehensive medication management delivered by pharm…
Video Education With Result Dependent dIsclosure
The overall study objective of this trial study is to identify and evaluate strategies to improve the accessibility of the video education with result dependen…
ACT-GEN (Adherence And Care Tracking In GENetic Cancer Syndromes)
To help people with high-risk cancer variants to follow cancer surveillance guidelines and lower their risk of developing the disease.
WiTNNess: An International Natural History Study of Autosomal Recessive TNNT1 Myopathy
WiTNNess is designed to accurately document the natural course and variation of muscle disease caused by pathogenic changes of the TNNT1 gene. The primary aim …
Genetic Investigation of Cancer Predisposition
Clinical information and samples (blood, saliva, and tumor) will be collected from patients with multiple cancers and/or a family history of cancer as well as …
Genetic Disorders of Obesity Program Database
This study collects data on children with severe, early-onset obesity.
A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Transfusion-Dependent β-Thalassemia or Severe Sickle Cell Disease
This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will eval…
An Adaptive Clinical Trial of BeginNGS Newborn Screening for Hundreds of Genetic Diseases by Genome Sequencing
The goal of this clinical trial is to test a new method for newborn screening using whole genome sequencing, called BeginNGS. Parents will be approached to pro…
A Randomized, Double-Blind, Placebo-Controlled, Parallel Group, Multi-Dose Study to Evaluate the Efficacy and Safety of VDPHL01 in Female Subjects With Androgenetic Alopecia
This study will evaluate the safety and efficacy of VDPHL01 in female subjects with Androgenetic Alopecia (AGA). AGA is a genetic disorder caused by an excess…
Identification of Genomic Predictors of Adverse Events After Cardiac Surgery
This study aims to identify genetic causes of adverse events after cardiac surgery, such as atrial fibrillation, myocardial infarction, renal dysfunction and h…