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Found 298 clinical trials
Featured trial
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 …

  • 9 views
  • 27 May, 2021
  • 1 location
Featured trial
CAHtalyst Study: A Research Study for Adults with Classic Congenital Adrenal Hyperplasia (CAH)

The CAHtalyst Study is a clinical research study evaluating an investigational medication for adults 18 years of age and older who have been diagnosed with classic congenital adrenal hyperplasia (CAH). The investigational medication is an oral capsule that will be taken twice daily with breakfast and evening meals. All participants who …

congenital adrenal hyperplasia
classic congenital adrenal hyperplasia
  • 50 views
  • 23 Nov, 2020
  • 1 location
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

fanconi's syndrome
fanconi's anemia
anemia
anemia studies
  • 143 views
  • 22 Dec, 2020
  • 1 location
Featured trial
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
  • 587 views
  • 08 Apr, 2019
  • 1 location
Featured trial
HAE - Patients diagnosed with Acute Hereditary Angioedema

HAE - Patients diagnosed with Acute Hereditary Angioedema

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

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

  • 529 views
  • 23 Nov, 2020
  • 1 location
Efficacy and Safety Comparison of Niraparib to Placebo in Participants With Human Epidermal Growth Factor 2 Negative (HER2-) Breast Cancer Susceptibility Gene Mutation (BRCAmut) or Triple-Negative Breast Cancer (TNBC) With Molecular Disease (ZEST)

] status, including HR positive [+] and TNBC) or tumor BRCA wild type (tBRCAwt) TNBC with molecular disease based on the presence of circulating tumor Deoxyribonucleic acid (ctDNA) following surgery or

epidermal growth factor
erbb2
growth factor
HER2
adjuvant chemotherapy
  • 3 views
  • 17 Jun, 2022
  • 143 locations
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

Accepts healthy volunteers
  • 0 views
  • 19 Jun, 2022
  • 3 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 …

Accepts healthy volunteers
  • 114 views
  • 17 Jun, 2022
  • 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
  • 13 Jun, 2021
  • 1 location