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Found 268 clinical trials
Setmelanotide Phase 2 Treatment Trial in Patients With Rare Genetic Disorders of Obesity

The purpose of the study is to determine the effect of setmelanotide (RM-493) on weight, hunger assessments and other factors in patients with rare genetic disorders of obesity.

body mass index
other disease
genetic disorders
  • 24 Feb, 2021
  • 54 locations
EDS in Ataxia Telangiectasia Patients

This is an international, multi-center, one-year, randomized, prospective, double-blind, placebo-controlled, phase III study, designed to assess the effect of two non-overlapping dose ranges of EDS EP, administered by IV infusion once per month, on neurological symptoms of patients with Ataxia Telangiectasia.

iv infusion
  • 24 Jan, 2021
  • 24 locations
A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent -Thalassemia

This is a single-arm, open-label, multi-site, single-dose Phase 1/2 study in subjects with transfusion-dependent -thalassemia (TDT). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.

blood transfusion
beta thalassemia
stem cell transplantation
hemoglobin e
  • 22 Feb, 2021
  • 12 locations
Program to Establish the Genetic and Immunologic Profile of Patient's Tumor for All Types of Advanced Cancer

potential predictive value and to determine if some genetic disorders are linked to immunity status alterations.

advanced cancer
primary cancer
  • 23 Jan, 2021
  • 8 locations
Evaluating Innovations in Transition From Pediatric to Adult Care - The Transition Navigator Trial

The Transition Navigator Trial (TNT) is a pragmatic randomized controlled trial evaluating the effectiveness of usual care plus a patient navigator service versus usual care plus newsletters and other educational materials, to improve transition outcomes among adolescents aged 16-21 who have chronic health conditions requiring transfer to adult specialty care. …

  • 22 Jan, 2021
  • 1 location
Pre-Test Genetic Education and Remote Genetic Counseling in Communicating Tumor Profiling Results to Patients With Advanced Cancer

This research trial studies how well pre-test genetic education and remote genetic counseling works in communicating tumor profiling results to patients with advanced cancer. Web-based genetic education before receiving tumor profiling results and remote genetic counseling for patients with potential germline mutations may increase genetic knowledge and reduce distress for …

advanced cancer
  • 26 Jan, 2021
  • 1 location
DCM Precision Medicine Study

The aims of the DCM Precision Medicine Study are to test the hypothesis that DCM has substantial genetic basis and to evaluate the effectiveness of a family communication intervention in improving the uptake and impact of family member clinical screening.

  • 26 Jan, 2021
  • 1 location
Genetic Screening and Assisted Oocyte Activation in Couples With Diminished/Aberrant Embryonic Development.

This is an interventional comparative study at the Department of Reproductive Medicine at Ghent University Hospital. Patients with previous embryo developmental problems are eligible for the study. Patients will undergo an ICSI-AOA treatment and will also be screened for genes important in the oocyte activation and embryonic development process. Also, …

  • 23 Jan, 2021
  • 1 location
Screening of Pulmonary Veino Occlusive Disease in Heterozygous EIF2AK4 Mutation Carriers

Pulmonary Veino Occlusive Disease (PVOD) is a rare form of pulmonary arterial hypertension, characterised by a poor prognosis. Recent studies demonstrated that heritable form of pulmonary veino occlusive diseaseis due to bi-allelic mutations in EIF2AK4 gene. heritable pulmonary veino occlusive disease is an autosomal recessive disease. In the french referal …

genetic counseling
  • 23 Jan, 2021
  • 1 location
Effects of Sitagliptin in Individuals With Genetically Decreased DPP4

This is a pilot clinical trial to test the hypothesis that during sitagliptin (DPP4 inhibitor), individuals heterozygous for DPP4 loss of function variants will have a reduction in DPP4 activity and antigen, lower glucose after a mixed meal, and higher levels of intact DPP4 substrates compared to during placebo and …

renal function
sitagliptin 100 mg
  • 03 Feb, 2021
  • 1 location