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Found 268 clinical trials
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 …

sitagliptin 100 mg
hypertension
diabetes
renal function
  • 0 views
  • 03 Feb, 2021
  • 1 location
Ensuring Patients' Informed Access to Noninvasive Prenatal Testing

Noninvasive prenatal genetic testing (NIPT) is an important new screening test option provided to pregnant women in the first trimester of pregnancy. The advantage of this screen is that is provides information about the risk of trisomy 13, trisomy 18, and trisomy 21 with greater accuracy than conventional screens. At …

prenatal
trisomy 13
nipt
genetic testing
  • 32 views
  • 04 Feb, 2021
  • 2 locations
Implementing an Individualized Pain Plan (IPP) for ED Treatment of VOE's in Sickle Cell Disease

The overall purpose of this proposed study is to improve management of vaso-occlusive episodes (VOEs) in adult EDs. We aim to implement NHLBI recommendations for VOE treatment by embedding Individualized Pain Plans (IPPs) in the electronic health record (EHR). The EHR-embedded IPP will serve as a record of patients' SCD …

  • 0 views
  • 29 Jan, 2021
  • 8 locations
Genomic Medicine for Ill Neonates and Infants (The GEMINI Study)

a targeted genomic sequencing panel on neonates and infants suspected of having a genetic disorder. This study is funded by the National Institutes of Health. This multicenter, prospective

  • 0 views
  • 23 Jan, 2021
  • 5 locations
Method of Genetic Analysis in Genodermatoses

The goal of the study is to develop a method of genetic diagnosis in two stages, by mendelioma then by genome and transcriptome on fibroblast culture, in genodermatoses and rare diseases with cutaneous expression in the child.

  • 0 views
  • 23 Jan, 2021
  • 1 location
Theophylline Treatment for Pseudohypoparathyroidism

Pseudohypoparathyroidism is a genetic disorder with limited treatment options. Patients have early-onset obesity, short stature and increased risk of type 2 diabetes. This phase 2 clinical trial

diabetes
  • 40 views
  • 26 Jan, 2021
  • 1 location
Crizanlizumab for Treatment of Retinal Vasculopathy With Cerebral Leukoencephalopathy (RVCL)

fatal genetic condition that affects the microvasculature, especially of the brain and eye. There currently is no treatment for RVCL. A maximum of 20 patients will be enrolled.

  • 0 views
  • 17 Feb, 2021
  • 1 location
A Phase 2 Study to Evaluate the Safety Tolerability PK and PD in Cystic Fibrosis Patients With at Least 1 G542X Allele

This is a Phase 2 open label study to evaluate the safety, tolerability, PK, and PD of multiple dose levels of SC administered ELX-02 in patients with CF with at least one G542X allele. Up to 16 patients will be enrolled in the trial; up 4 patients will be homozygotes …

body mass index
cftr modulators
fibrosis
  • 1 views
  • 27 Jan, 2021
  • 9 locations
Theophylline Treatment for Pseudohypoparathyroidism - Children 2-12 Years Old

Pseudohypoparathyroidism is a genetic disorder with limited treatment options, characterized by early-onset obesity, short stature and resistance to multiple hormones. This phase 2 clinical

  • 0 views
  • 26 Jan, 2021
  • 1 location
Cystic Fibrosis - Insulin Deficiency Early Action

Cystic Fibrosis (CF) is the most common life-threatening genetic condition affecting Australian children. As well as repeated lung infections, children with CF develop insulin deficiency and

genetic disorder
antibiotics
malnutrition
oral glucose tolerance test
insulin treatment
  • 50 views
  • 22 Jan, 2021
  • 5 locations