Clinical trials: Anemia, Neonatal
Beta-Thalassemia Major, Sickle Cell Disease
A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease
This is a Phase 1/2, open label, safety, and efficacy study of the administration of LentiGlobin BB305 Drug Product to participants with either transfusion dependent beta-thalassemia (TDT) or sickle cell disease (SCD).
- Trial name
- NCT02151526
- Testing
- LentiGlobin BB305 Drug Product
- Where
- France
- Volunteers
- 7 people
- Sponsor
- Genetix Biotherapeutics Inc.
Sickle Cell Disease, Iron Overload
Study of Deferasirox Relative to Subcutaneous Deferoxamine in Sickle Cell Disease Patients
This study will examine the long-term safety and efficacy of Deferasirox in patients with sickle cell disease and iron overload from repeated blood transfusions.
- Conditions
- Sickle Cell Disease, Iron Overload, Hemolytic Anemia
- Trial name
- NCT00110617
- Testing
- Deferasirox (ICL670), Deferoxamine (DFO)
- Where
- 59 sites in United States, Canada
- Volunteers
- 212 people
- Sponsor
- Novartis Pharmaceuticals
Priapism, Anemia, Sickle Cell
Priapism in Boys and Men With Sickle Cell Disease - Demographics, Characteristics and Prevalence
Priapism, a prolonged erection of the penis, is a medical issue that often affects men with sickle cell disease. The purpose of this study is to collect demographic and clinical information on priapism by interviewing men with…
- Trial name
- NCT00300235
- Where
- 27 sites in United States
- Volunteers
- 1,464 people
- Sponsor
- University of Texas Southwestern Medical Center
Cardiovascular Diseases, Heart Diseases
Combination Therapy Compared With Single-Drug Therapy in Patients With Cardiac Diseases
The purpose of this study is to determine whether left ventricular function improves more rapidly with deferoxamine (DFO) and deferiprone (L1) combination therapy than with DFO monotherapy in patients with thalassemia and…
- Conditions
- Cardiovascular Diseases, Heart Diseases, Beta-Thalassemia
- Trial name
- NCT00115349
- Testing
- Deferoxamine, Deferiprone (L1)
- Where
- 6 sites in United States
- Volunteers
- 20 people
- Sponsor
- Carelon Research
Thalassemia
Evaluating the Safety and Effectiveness of Decitabine in People With Thalassemia Intermedia
Thalassemia intermedia (TI) is an inherited blood disorder that can cause anemia due to low levels of hemoglobin. Decitabine is a medication that may be effective at increasing hemoglobin levels.
- Trial name
- NCT00661726
- Testing
- Decitabine (USAN, INN)
- Where
- 3 sites in United States, Canada
- Volunteers
- 6 people
- Sponsor
- Carelon Research
Leukemia, Lymphoma
Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer
RATIONALE: Giving chemotherapy and total-body irradiation before a donor peripheral stem cell transplant helps stop the growth of cancer and abnormal cells and helps stop the patient's immune system from rejecting the donor's…
- Conditions
- Leukemia, Lymphoma, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
- Trial name
- NCT00054327
- Testing
- busulfan, cyclophosphamide, cytarabine
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- Case Comprehensive Cancer Center
Sickle Cell Disease (SCD)
Study Exploring the Effect of Crizanlizumab on Kidney Function in Patients With Chronic Kidney Disease Caused by Sickle Cell Disease
The goal of the study was to evaluate descriptively the effect of crizanlizumab + standard of care and standard of care alone on renal function in sickle cell disease patients ≥ 16 years with chronic kidney disease due to sickle…
- Trial name
- STEADFAST (NCT04053764)
- Testing
- Crizanlizuamb, Standard of Care
- Where
- 24 sites in United States, Brazil and 9 more
- Volunteers
- 58 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease, Beta Thalassemia-Major
HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity
This study will evaluate the use of reduced intensity conditioning regimen in patients with high risk hemoglobinopathy Sickle Cell and B-Thalassemia Major in combination with standard immunosuppressive medications, followed by a…
- Trial name
- NCT02435901
- Testing
- alemtuzumab (Campath IH), Fludarabine, Melphalan
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- Northwell Health
Sickle Cell Disease, Sickle Cell Nephropathy
Effect of Atorvastatin on Endothelial Dysfunction and Albuminuria in Sickle Cell Disease
The purpose of this research study is to learn about the effect of the drug, atorvastatin, on blood vessels in patients with sickle cell disease.
- Trial name
- ENDO (NCT01732718)
- Testing
- Atorvastatin
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- University of North Carolina, Chapel Hill
Sickle Cell Disease
Patient-Provider Tools to Improve the Transition to Adult Care in Sickle Cell Disease
The purpose of the study is to develop patient-provider clinical support tools to improve clinical practice, patient self-management, and disease outcomes in sickle cell disease during transition to adult care.
- Trial name
- iTransition (NCT02200510)
- Testing
- Self-management intervention for Adolescents with SCD, Patient Portal Intervention for Adolescents with SCD
- Where
- United States
- Volunteers
- 78 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Fanconi Anemia
TBI Dose De-escalation for Fanconi Anemia
This is a single arm, total body irradiation (TBI) trial. All patients will be prescribed TBI 300 cGy with the goal of evaluating secondary endpoints.
- Trial name
- NCT00352976
- Testing
- Cyclophosphamide, Fludarabine, Total Body Irradiation
- Where
- United States
- Volunteers
- 83 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Sickle Cell Disease
A GBT021601 ADME Microtracer Study in Healthy Volunteers
An Open-label Study of GBT021601 in 8 to 10 healthy male or female participants to evaluate the absorption, distribution, metabolism, and excretion (ADME) of GBT021601.
- Trial name
- NCT05718687
- Testing
- GBT021601
- Where
- Netherlands
- Volunteers
- 9 people, healthy volunteers welcome
- Sponsor
- Pfizer
Acute Respiratory Distress Syndrome
Sigh Ventilation to Increase Ventilator-Free Days in Victims of Trauma at Risk for Acute Respiratory Distress Syndrome
A randomized, concurrent controlled trial to assess if adding sigh breaths to usual invasive mechanical ventilation of victims of trauma who are at risk of developing ARDS will decrease the number of days they require invasive…
- Trial name
- SiVent (NCT02582957)
- Testing
- Sigh breaths
- Where
- 15 sites in United States
- Volunteers
- 524 people
- Sponsor
- University of Minnesota
Sickle Cell Disease
SMYLS: A Self-management Program for Youth Living With Sickle Cell Disease
The purpose of this study is to find out whether a web-based intervention using a mobile device is helpful for teens learning to care for and manage symptoms of sickle cell disease.
- Trial name
- NCT04289311
- Testing
- Voice Crisis Alert V2
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Medical University of South Carolina
Non-Neoplastic Hematologic and Lymphocytic Disorder
Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders
This phase II clinical trial studies how well treosulfan and fludarabine phosphate with or without low dose radiation before donor stem cell transplantation works in treating patients with nonmalignant (noncancerous) diseases.
- Trial name
- NCT00919503
- Testing
- Allogeneic Bone Marrow Transplantation, Anti-Thymocyte Globulin, Cyclosporine
- Where
- 6 sites in United States
- Volunteers
- 98 people
- Sponsor
- Fred Hutchinson Cancer Center
Sickle Cell Disease
Fixed Dose Flavonoid Isoquercetin on Thrombo-Inflammatory Biomarkers in Subjects With Stable Sickle Cell Disease
Background: Sickle cell disease (SCD) is an inherited hemoglobin disorder. People with SCD have an increased chance for getting blood clots.
- Trial name
- NCT04514510
- Testing
- Isoquercetin
- Where
- United States
- Volunteers
- 46 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease
Evaluating the Safety and Effectiveness of Bone Marrow Transplants in Children With Sickle Cell Disease (BMT CTN 0601)
Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited blood disease that can cause organ damage, stroke, and intense pain episodes.
- Trial name
- SCURT (NCT00745420)
- Testing
- Hematopoietic Stem Cell Transplantation
- Where
- 19 sites in United States
- Volunteers
- 30 people
- Sponsor
- Medical College of Wisconsin
Thalassemia Major
Study of Efficacy and Safety of INC424 in Regularly Transfused Patients With Thalassemia.
Patients with severe thalassemia (thalassemia major) present with severe anemia that required life-long transfusion therapy, spleen enlargement that led to increased transfusion requirement, and other serious complications as…
- Trial name
- NCT02049450
- Testing
- ruxolitinib
- Where
- 7 sites in Greece, Italy and 3 more
- Volunteers
- 30 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease, Hemoglobin SC
Bone Marrow Transplantation, Hemoglobinopathies, SCALLOP
Patients are being asked to participate in this study because they have severe sickle cell anemia (SCD) with or without the beta thalassemia trait.
- Trial name
- SCALLOP (NCT00578344)
- Testing
- Busulfan, Campath 1H, Cyclophosphamide and MESNA
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- Tami D. John
Sickle Cell Disease, COVID-19
COVID-19 Vaccine Response in Sickle Cell Disease
The purpose of this study is to assess the antibody response to COVID-19 vaccination in a cohort of patients with sickle cell disease (SCD) and to assess vaccine and SCD related complications around the time of vaccination.
- Trial name
- NCT05139992
- Testing
- COVID-19 Vaccine
- Where
- 8 sites in United States
- Volunteers
- 59 people
- Sponsor
- ASH Research Collaborative