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Found 297 clinical trials
Senicapoc and Dehydrated Stomatocytosis

Dehydrated stomatocytosis is a genetic disorder characterized by chronic hemolysis, variable anemia and erythrocyte dehydration. Causative mutations have been identified in either the Gardos

  • 13 Jun, 2021
  • 1 location
Cascade Genetic Testing of Familial Hypercholesterolemia (CATCH)

Familial hypercholesterolemia (FH) is a frequent genetic disorder (1/200) associated with an increased risk of early-onset myocardial infarction. To improve detection and treatment of patient

genetic analysis
myocardial infarction
genetic disorder
cardiovascular disease
  • 03 Jun, 2022
Effectiveness of the Hippotherapy Simulator in Children and Adolescents With Cystic Fibrosis

Cystic fibrosis (CF) is a genetic disease that affects many organs and systems, especially respiratory system problems due to lung damage. Patients often have difficulty in removing the sticky

  • 19 Jun, 2022
  • 1 location
Coronary Artery Disease in Patients With Friedreich's Ataxia

Friedreich's ataxia is a debilitating, inherited disease cause by mutations in a protein called frataxin (FXN). FXN is one of several proteins that controls the production of iron-sulfur

  • 23 Mar, 2022
  • 1 location
Pilot Study of Reduced-Intensity Hematopoietic Stem Cell Transplant of DOCK8 Deficiency

Background -DOCK8 deficiency is a genetic disorder that affects the immune system and can lead to severe recurrent infections and possible death from infections or

Accepts healthy volunteers
  • 28 Jun, 2022
  • 1 location
Multi-center, Open-label, Phase 1b Clinical Trial to Evaluate the Safety, Tolerability, and Exploratory Efficacy of TEW-7197 in Combination With FOLFOX in Patients With Metastatic Pancreatic Ductal Adenocarcinoma Who Have Failed First-Line Gemcitabine and Nab-Paclitaxel (MP-PDAC-01)

reference limit) at Screening (based on the normal range of relevant study center) Subjects who have risk factors for ascending aortic aneurysm such as genetic disorder and trauma and risk

neutrophil count
pancreatic ductal adenocarcinoma
biliary stents
  • 29 Jun, 2022
  • 1 location
RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)

RGX-121 is a gene therapy which is designed to deliver a functional copy of the iduronate-2-sulfatase (IDS) gene to the central nervous system. This study is a phase I/II study to determine whether RGX-121 is safe, well tolerated, and potentially effective in children five years of age and over who …

  • 12 May, 2022
  • 2 locations
CHED - Congenital Hereditary Endothelial Dystrophy: New Paradigm Shift in Therapy Using Topical Eye Drops

CHED- Congenital Endothelial Endothelial Dystrophy is a condition that causes corneal cloudiness. Since, currently only surgery is being done to cure this condition, we are taking up the research of using topical eye drops for this condition which is a very simple and easy method. Also, there are no significant …

  • 27 Apr, 2021
  • 1 location
A Trial for Prevention of Recurrent Ischemic Priapism in Men With Sickle Cell Disease: A Pilot Study (PIN)

To conduct a randomized controlled internal pilot feasibility trial for the prevention of recurrent ischemic priapism referred to as the Priapism in Nigeria (PIN) trial. The study team will enroll a minimum of 30 participants and a maximum of 200 participants. Study investigators hypothesize that hydroxyurea therapy combined with tadalafil …

  • 29 Apr, 2022
  • 1 location
Dupilumab for Prevention of Recurrence of CRSwNP After ESS

The investigators believe that administering Dupilumab during the pre- and peri-operative period of surgery for chronic rhinosinusitis with nasal polyps (CRSwNP) will safely downregulate Type 2 inflammation of the healing sinus environment and will allow for better coordinated and more effective mucosal healing. Specifically, the investigators believe that endoscopic signs …

  • 13 Jun, 2021
  • 1 location