Clinical trials: Hereditary Anemias
Sickle Cell Anemia
Study of Beet Juice for Patients With Sickle Cell Anemia
The investigators hypothesize that increasing plasma nitrite using dietary nitrate will improve platelet function and red cell deformability and decrease MCHC in patients with sickle cell disease.
- Trial name
- NCT02162225
- Testing
- beet juice (Unbeetable)
- Where
- United States
- Sponsor
- Wake Forest University Health Sciences
Chronic Myelomonocytic Leukemia, Chronic Myelomonocytic Leukemia-1
Azacitidine With or Without Lenalidomide or Vorinostat in Treating Patients With Higher-Risk Myelodysplastic Syndromes or Chronic Myelomonocytic Leukemia
This randomized phase II/III trial studies how well azacitidine works with or without lenalidomide or vorinostat in treating patients with higher-risk myelodysplastic syndromes or chronic myelomonocytic leukemia.
- Conditions
- Chronic Myelomonocytic Leukemia, Chronic Myelomonocytic Leukemia-1, Chronic Myelomonocytic Leukemia-2, Myelodysplastic Syndrome
- Trial name
- NCT01522976
- Testing
- Azacitidine, Laboratory Biomarker Analysis, Lenalidomide
- Where
- 294 sites in United States, Canada
- Volunteers
- 282 people
- Sponsor
- National Cancer Institute (NCI)
Sickle Cell Disease
Sickle Cell Pro-Inflammatory Response to Interval Training Study
Recommendations for exercise prescription currently do not exist for individuals with sickle cell anemia (SCA) despite the known impact that SCA-related complications has on physical functioning and fitness.
- Trial name
- SPRINTS (NCT03653676)
- Testing
- Exercise Test (CIIT)
- Where
- 3 sites in United States
- Volunteers
- 110 people, healthy volunteers welcome
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
Anemia
Iron Prophylaxis for Anemia in Infants With Cyanotic Congenital Heart Disease
Anemia is a common disorder in infants with one working chamber of the heart that pumps blood. Anemia is when the level of healthy blood cells becomes too low.
- Trial name
- NCT00459225
- Testing
- Iron (ferrous sulfate)
- Where
- United States
- Sponsor
- Emory University
Thrombocytopenia, Metachromatic Leukodystrophy
Stem Cell Transplantation (SCT) for Genetic Diseases
OBJECTIVES: I. Ascertain whether stem cell transplantation (SCT) is an effective method by which missing or dysfunctional enzymes can be replaced in patients with various inborn errors of metabolism. II.
- Conditions
- Thrombocytopenia, Metachromatic Leukodystrophy, Fanconi's Anemia, Thalassemia Major
- Trial name
- NCT00004378
- Testing
- Stem Cell Transplantation
- Where
- United States
- Sponsor
- National Center for Research Resources (NCRR)
Sickle Cell Anemia in Children, Sickle Cell Disease
Promoting Utilization and Safety of Hydroxyurea Using Precision in Africa
Sickle cell anemia (SCA) is among the world's most common and devastating blood disorders, affecting more than 300,000 newborns per year.
- Trial name
- PUSHUP (NCT05285917)
- Testing
- Hydroxyurea
- Where
- Angola
- Volunteers
- 400 people
- Sponsor
- Brown University
Attention Deficit Disorder With Hyperactivity, Asthma
Study of an Intervention to Improve Problem List Accuracy and Use
The aim of this study is to identify patients with problem list gaps and intervene to correct these gaps by creating clinical decision support interventions that alert providers to likely problem list gaps and offer clinicians…
- Conditions
- Attention Deficit Disorder With Hyperactivity, Asthma, COPD, Breast Cancer
- Trial name
- MAPLE (NCT01105923)
- Testing
- MAPLE
- Where
- United States
- Volunteers
- 140 people
- Sponsor
- Brigham and Women's Hospital
Sickle Cell Disease
The Implementation of the Automated Erythrocytapheresis in Egyptian Sickle Cell Disease Center
Improvements of health infrastructure, preventive care and clinical treatment have reduced the morbidity and mortality of sickle cell disease (SCD).
- Trial name
- NCT03903289
- Testing
- Automated red cell exchange, Manual red cell exchange, Simple red cell transfusion
- Where
- Egypt
- Volunteers
- 20 people
- Sponsor
- Ain Shams University
Sickle Cell Disease
A Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Crovalimab as Adjunct Treatment in Prevention of Vaso-Occlusive Episodes (VOE) in Sickle Cell Disease (SCD)
This study is designed to evaluate the efficacy, safety and pharmacokinetics of crovalimab compared with placebo as adjunct therapy in the prevention of VOEs in participants with SCD.
- Trial name
- CROSSWALK-c (NCT05075824)
- Testing
- Crovalimab
- Where
- 27 sites in United States, Brazil and 9 more
- Volunteers
- 95 people
- Sponsor
- Hoffmann-La Roche
Sickle Cell Anemia, Malaria
Sulfadoxine- Pyrimethamine Versus Weekly Chloroquine for Malaria Prevention in Children With Sickle Cell Anemia
Malaria is fatal and increases the risk of death among children with sickle cell anemia. Chemoprophylaxis significantly improves quality of life in these children.
- Trial name
- NCT00399074
- Testing
- sulfadoxine pyrimethamine
- Where
- Uganda
- Volunteers
- 220 people
- Sponsor
- Makerere University
Alpha and Beta Thalassemia, Sickle Cell Disease
Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants
Background: Blood disorders like sickle cell disease and malaria affect many people around the world. Researchers want to learn more about blood disorders.
- Conditions
- Alpha and Beta Thalassemia, Sickle Cell Disease, Malaria, Human Physiology
- Trial name
- NCT03937817
- Where
- United States
- Volunteers
- 300 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Anemia, Sickle Cell
Effectiveness of Arginine as a Treatment for Sickle Cell Anemia
Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited genetic disease that can cause intense pain episodes.
- Trial name
- Arginine (NCT00513617)
- Testing
- Arginine
- Where
- 17 sites in United States
- Volunteers
- 128 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Gaucher Disease
Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase (prGCD) in Patients With Gaucher Disease
This is an open-label expanded access trial of prGCD in patients with Gaucher disease who require enzyme replacement therapy (ERT) and who have been treated with imiglucerase but for whom the dose has been reduced or discontinued…
- Trial name
- NCT00962260
- Testing
- Plant cell expressed recombinant glucocerebrosidase (prGCD)
- Where
- 15 sites in United States, Israel
- Sponsor
- Pfizer
Beta-Thalassemia
Safety & Efficacy of ICL670 vs. Deferoxamine in Beta-thalassemia Patients With Iron Overload Due to Blood Transfusions
The purpose of this study is to deterimine if the new orally active iron chelator, ICL670, is as effective and as safe as deferoxamine in preventing accumulation of iron in the body while a patient is undergoing repeated blood…
- Trial name
- NCT00061750
- Testing
- ICL670, deferoxamine
- Where
- 7 sites in United States
- Volunteers
- 595 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease
Prevalence of Problematic Use of Equimolar Mixture of Oxygen and Nitrous Oxide and Analgesics in the Sickle-cell Disease
The use of analgesics can lead to cases of drug abuse and dependence. It can also cause pseudo-addiction in patients suffering from pain.
- Trial name
- PHEDRE (NCT02580565)
- Testing
- telephone interview
- Where
- France
- Volunteers
- 1,004 people
- Sponsor
- Nantes University Hospital
Sickle Cell Anemia
Longitudinal Changes in Exercise Capacity in Children and Young Adults With Sickle Cell Anemia
The purpose of this study is to use comprehensive exercise testing to examine longitudinal changes in exercise capacity over a 2 year period in children and young adults with sickle cell anemia.
- Trial name
- NCT01558076
- Where
- United States
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
Sickle Cell Disease
Study of Blood Platelets in Sickle Cell Disease
We are studying if sickle cell disease blood platelets are larger than normal and how they may cause obstruction of blood vessels.
- Trial name
- NCT00226083
- Where
- United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- Rockefeller University
Sickle Cell Disease
Spectralis HRA+OCT Imaging of the Retina With Autofluorescence in Sickle Cell Disease
To determine the retinal and choroidal thickness in patients with sickle cell disease compared to age, race matched population without sickle cell disease to allow a better understanding of the clinical manifestations of sickle…
- Trial name
- NCT01123369
- Where
- 2 sites in United States
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- Medical University of South Carolina
Thalassemia Major
Long Term Outcomes in β Thalassemia Major
Beta thalassemia (β-thalassemia) is the most common genetic disease worldwide. Individuals with thalassemia are born with a defect in hemoglobin.
- Trial name
- NCT02307786
- Where
- United States
- Volunteers
- 176 people
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
Cyanotic Congenital Heart Disease, Anemia
Erythropoietin to Prevent Unnecessary Transfusions In Patients With Cyanotic CHD - A Prospective Control Trial
Cyanotic congenital cardiac patients require higher hemoglobin concentrations (red blood cell levels) for optimal oxygen delivery to the body.
- Conditions
- Cyanotic Congenital Heart Disease, Anemia, Cyanosis, Congenital Heart Disease
- Trial name
- NCT02564796
- Testing
- Epoetin Alfa and Iron, Iron
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- University of California, San Diego