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Found 81 clinical trials
Featured trial
Eltrombopag for People With Fanconi Anemia

Fanconi anemia is a genetic disease. Some people with it have reduced blood cell counts. This means their bone marrow no longer works properly. These people may need blood transfusions for anemia

anemia studies
fanconi's anemia
fanconi's syndrome
  • 22 Dec, 2020
  • 1 location
The HOME Study (HPN With OMEGA-3) (HOME)

The aim of the trial is to investigate safety and tolerability of an Omega-3-FA-enriched lipid emulsion in adult patients with chronic intestinal failure in need of long-term HPN. It is aimed to

  • 03 Mar, 2022
  • 9 locations
A Monoclonal Antibody Conditioning Regimen for Allogeneic Stem Cell Transplant in Patients With Fanconi Anemia.

Patients eligible for this study have an aggressive blood disease, Fanconi Anemia (FA) that requires an allogeneic (meaning the cells come from a donor) stem cell transplant using a

graft versus host disease
  • 07 Nov, 2020
  • 1 location
NAD+ and Exercise in FA (ExRx in FA)

Randomized, placebo-controlled trial with a 2x2 factorial design testing the effects of an NAD+ precursor (NR) and exercise on VO2max and Si in Friedreich's Ataxia (FA). The primary

  • 02 Mar, 2022
  • 1 location
NAD+ Precursor Supplementation in Friedreich's Ataxia

The primary objective is to test the safety and tolerability of short-term therapy with a nicotinamide adenine dinucleotide (NAD+) precursor (MIB-626) in adults with Friedreich's Ataxia (FA

ejection fraction
cardiac failure
molecular diagnosis
  • 09 May, 2022
  • 1 location
Mechanisms of Ozone-Induced Alterations in Efferocytosis and Phagocytosis

blood draw and breathing testing, return for a two-day series of testing to include blood draw, and brief breathing test before and after an inhaled challenge with either filtered air (FA) or ozone (O3

Accepts healthy volunteers
  • 10 Aug, 2022
  • 1 location
Salt Supplementation in Gitelman Syndrome

The purpose of this study is to determine the effect of salt (NaCl) supplementation on serum potassium and clinical signs and symptoms in patients with Gitelman syndrome.

  • 17 Aug, 2021
  • 2 locations
An Experimental Protocol for the Study of Brain Functional Magnetic Resonance Imaging in Female With Urgent Urinary Incontinence

Background: Patients' life quality and physical and mental health are seriously affected by Urgent Urinary Incontinence(UUI). The cause is not clear at present. It has been found that the injury of the prefrontal lobe and damage to the surrounding cerebral cortex leads to significant lower urinary tract dysfunction. Therefore, some …

Accepts healthy volunteers
  • 27 Feb, 2022
  • 1 location
Long-Term Efficacy and Safety of Intravitreal Aflibercept Injections for the Treatment of Diabetic Retinopathy for Subjects Who Completed the 2-Year PANORAMA Trial (VOYAGE)

The VOYAGE trial will assess diabetic retinopathy severity scale (DRSS) levels, through 112 weeks, while being managed with aflibercept as needed, among subjects who completed the 2-year PANORAMA trial (VGFTe-OD-1411) and were treated in a clinical setting prior to joining the VOYAGE study.

aflibercept injection
  • 26 Mar, 2022
  • 9 locations
Niraparib in Combination With Cabozantinib (XL184) in Patients With Advanced Urothelial Cancer (NICARAGUA) (NICARAGUA)

Cabozantinib is an oral, small-molecule tyrosine kinase inhibitor. Its primary targets are Hepatocyte growth factor receptor protein (MET), vascular endothelial growth factor receptor 1-3 (VEGFR1-3), RET, AXL, FLT3 and KIT. Cabozantinib has been approved by the FDA for clinical treatment of progressive, metastatic medullary thyroid cancer. Recently published trials have …

measurable disease
metastatic cancer
kinase inhibitor
  • 26 Mar, 2022
  • 9 locations