Filter By
Clear all Advanced
I am/have/had
added new label for I am/have/had
more
I am looking for
added new label for I am looking for
more
Advanced Filters
Found 278 clinical trials
featured
Retrospective study examining the differences in vocalizations of babies and toddlers with Rett compared to same age peers

Retrospective study examining the differences in vocalizations of babies and toddlers with Rett compared to same age peers: We are looking to recruit children and their parent(s) to participate in a study examining the early speech development of infants and toddlers later diagnosed with Rett syndrome, compared to girls their …

  • 0 views
  • 02 Oct, 2020
  • 1 location
featured
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 syndrome
fanconi's anemia
anemia
  • 91 views
  • 22 Dec, 2020
  • 1 location
featured
Cardiovascular Disease Discovery Protocol

Background: Researchers are interested in studying individuals who have known or suspected metabolic or genetic diseases that put them at a high risk for heart

angiography
  • 327 views
  • 08 Apr, 2019
  • 1 location
featured
HAE - Patients diagnosed with Acute Hereditary Angioedema

HAE - Patients diagnosed with Acute Hereditary Angioedema

  • 598 views
  • 08 Nov, 2020
  • 1 location
featured
CASCADE FH Registry: Cascade Screening for Awareness and Detection of Familial Hypercholesterolemia

CASCADE FH Registry: Cascade Screening for Awareness and Detection of Familial Hypercholesterolemia

  • 434 views
  • 23 Nov, 2020
  • 1 location
South-seq: Deoxyribonucleic Acid (DNA) Sequencing for Newborn Nurseries in the South

2,000 infants with signs suggestive of a genetic disorder being treated at a neonatal intensive care unit (NICU) in which African-American and rural populations are highly represented will be

  • 0 views
  • 02 Feb, 2021
  • 3 locations
Study of the Anti-FGF23 Antibody Burosumab in Adults With XLH

This is phase 3b open-label, international, multicenter study to continue to monitor the long-term safety and efficacy of burosumab in adult patients with XLH that participated in previous clinical trials with burosumab (UX023-CL303 / UX023-CL304).

  • 32 views
  • 23 Jan, 2021
  • 11 locations
Treatment of Hemochromatosis

This study will evaluate the effectiveness of a test called MCV in guiding phlebotomy (blood drawing) therapy in patients with hemochromatosis an inherited disorder that causes too much iron to be absorbed by the intestine. The excess damages body tissues, most severely in the liver, heart, pancreas and joints. Because …

  • 4 views
  • 14 Feb, 2021
  • 1 location
Propranolol for Epistaxis in Hereditary Hemorrhagic Telangiectasia Patients

Hereditary Hemorrhagic Telangiectasia (HHT) is a genetic disorder of angiogenesis associated with disabling epistaxis. Management of these nose bleedings requires more effective treatment

recurrent nosebleeds
arteriovenous malformation
epistaxis
angiogenesis
genetic disorder
  • 0 views
  • 24 Jan, 2021
  • 1 location
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

  • 0 views
  • 25 Apr, 2021
  • 1 location