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Found 338 clinical trials
A Non-Interventional National Study in Pediatric Patients With Unexplained Enlarged Spleen (OPPUS)

Primary Objective: To assess prevalence of Gaucher disease (GD) diagnosed in pediatric patients presenting with unexplained splenomegaly (SMG) after exclusion of first intention-diagnoses (e.g. portal hypertension, haematological malignancy, hemolytic anemia, infection) based on clinical examination and routine biological tests (full blood count, reticulocytes, liver tests, abdominal ultrasound, Coombs test and …

  • 0 views
  • 04 Oct, 2022
  • 40 locations
A Non-Interventional National Study in Pediatric Patients With Unexplained Enlarged Spleen (OPPUS)

Primary Objective: To assess prevalence of Gaucher disease (GD) diagnosed in pediatric patients presenting with unexplained splenomegaly (SMG) after exclusion of first intention-diagnoses (e.g. portal hypertension, haematological malignancy, hemolytic anemia, infection) based on clinical examination and routine biological tests (full blood count, reticulocytes, liver tests, abdominal ultrasound, Coombs test and …

  • 0 views
  • 04 Oct, 2022
  • 40 locations
Pompe Pregnancy Sub-Registry

This Sub-registry is a multicenter, international, longitudinal, observational, and voluntary program designed to track pregnancy outcomes for any pregnant woman enrolled in the Pompe Registry, regardless of whether she is receiving disease-specific therapy (such as ERT with alglucosidase alfa) and irrespective of the commercial product with which she may be …

alglucosidase alfa
  • 0 views
  • 23 Jan, 2022
  • 14 locations
Pompe Pregnancy Sub-Registry

This Sub-registry is a multicenter, international, longitudinal, observational, and voluntary program designed to track pregnancy outcomes for any pregnant woman enrolled in the Pompe Registry, regardless of whether she is receiving disease-specific therapy (such as ERT with alglucosidase alfa) and irrespective of the commercial product with which she may be …

alglucosidase alfa
  • 84 views
  • 23 Jan, 2022
  • 14 locations
A Phase IV Study to Investigate the Safety and Effectiveness of Rivaroxaban(Xarelto) 2.5mg [BID]+Acetylsalicylic Acid(ASA) 75mg [OD] in Indian Patients With Coronary and/or Symptomatic Peripheral Artery Disease

This is an observational study in which data from Indian people with coronary artery disease and / or symptomatic peripheral artery disease who will be receiving the drug rivaroxaban (Xarelto) are studied. Coronary artery disease (CAD) is a condition where the arteries that bring blood and oxygen to the heart …

clot
aspirin
stroke
  • 0 views
  • 13 Oct, 2022
  • 1 location
Longitudinal Study of Mitochondrial Hepatopathies (MITOHEP)

The specific aims of this study are (1) to determine the clinical phenotypes and natural history of hepatic RC and FAO disorders, (2) to determine the correlation between genotype and phenotype, (3) to determine if circulating biomarkers reflect diagnosis and predict liver disease progression and survival with the native liver, …

acyl carnitine
liver transplant
chronic liver failure
fibrosis
cirrhosis
  • 34 views
  • 28 Apr, 2022
  • 12 locations
Pediatric Hypertension and the Renin-Angiotensin SystEm (PHRASE) (PHRASE)

Studying the causal roles of components of the renin-angiotensin-aldosterone system (including angiotensin-(1-7) (Ang-(1-7)), angiotensin-converting enzyme 2 (ACE2), Ang II, and ACE), uric acid, and klotho in pediatric hypertension and related target organ injury, including in the heart, kidneys, vasculature, and brain. Recruiting children with a new hypertension diagnosis over a …

angiotensin
Uric Acid Test
Accepts healthy volunteers
renin
aldosterone
  • 0 views
  • 21 Mar, 2022
  • 1 location
The NIH MINI Study: Metabolism, Infection, and Immunity in Inborn Errors of Metabolism

The Metabolism, Infection and Immunity (MINI) Study is a longitudinal natural history study at the National Institutes of Health (NIH) that aims to define the relationship between infection, immunity and clinical decline in individuals with mitochondrial disease. Mitochondrial diseases are a group of disorders caused by problems with the cell …

Accepts healthy volunteers
influenza
vaccination
lab testing
  • 39 views
  • 07 Oct, 2022
  • 1 location