Clinical trials: Sickle Cell Thalassemia
Sickle Cell Disease
Aspirin Prophylaxis in Sickle Cell Disease
Neurologic complications secondary to cerebrovascular damage are prevalent in children with sickle cell disease. These patients experience both clinically overt cerebrovascular accidents and "silent infarctions" demonstrated by…
- Trial name
- START (NCT00178464)
- Testing
- aspirin
- Volunteers
- 11 people
- Sponsor
- University of Rochester
Transfusion-dependent Iron Overload
A Study Assessing the Efficacy and Safety of Deferasirox in Patients With Transfusion-dependent Iron Overload
This study uses a single arm, multi-center, open-label trial design. The study will assess the efficacy and safety of 52 weeks of treatment with deferasirox (ICL670) in patients with evidence of transfusion induced iron overload.
- Trial name
- NCT00171821
- Testing
- Deferasirox
- Where
- 97 sites in Australia, Austria and 21 more
- Volunteers
- 1,784 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Anemia, Thalassemia
Bone Marrow for Hemoglobinopathy Research
Human participants affected with sickle cell disease or thalassemia will donate bone marrow for use in experimental laboratory models to study potential new treatments.
- Trial name
- NCT00669305
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- St. Jude Children's Research Hospital
Sickle Cell Disease, Sickle Cell Hemoglobin C
Home-based Assessment of PRO Measures in SCD Using A Smartphone App Platform: A Feasibility Study
The overarching goal of this proposal is to identify modifiable behavioral strategies based on patient-reported outcomes (PROs) and health-related quality of life (HRQOL) that will improve hydroxyurea (HU) adherence among…
- Conditions
- Sickle Cell Disease, Sickle Cell Hemoglobin C, Sickle Beta Zero Thalassemia, Sickle B+ Thalassemia
- Trial name
- NCT04678037
- Testing
- PROs assessment using sickle cell disease mobile app (SCD-app)
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
Chronic Kidney Disease, Acute Myeloid Leukemia (AML)
Bone Marrow and Kidney Transplant for Patients With Chronic Kidney Disease and Blood Disorders
The main purpose of this study is to examine the outcome of a combined bone marrow and kidney transplant from a partially matched related (haploidentical or "haplo") donor.
- Conditions
- Chronic Kidney Disease, Acute Myeloid Leukemia (AML), Acute Lymphoblastic Leukemia (ALL), Chronic Myelogenous Leukemia (CML)
- Trial name
- BMT (NCT01758042)
- Testing
- Haploidentical Bone Marrow/Kidney
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Massachusetts General Hospital
Anemia, Sickle Cell, Sickle Cell Disease
Epidemiology of Silent and Overt Strokes in Sickle Cell Disease
Sickle Cell Disease (SCD) is a rare disease occurring in an estimated 100,000 individuals, often poor and underserved, in the US.
- Conditions
- Anemia, Sickle Cell, Sickle Cell Disease, Stroke, Sickle Cell Thalassemia
- Trial name
- ESCD (NCT03376893)
- Where
- 4 sites in United States
- Volunteers
- 102 people
- Sponsor
- Vanderbilt University Medical Center
Alpha-Thalassemia, Beta-Thalassemia
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases.
- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis
- Trial name
- NCT06539169
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- xCures
Sickle Cell Anemia, Sickle - Beta Thalassemia
Hepcidin Levels in Sickle Cell Disease (SCD)
The investigators propose that patients with HbSβ-thalassemia have lower levels of hepcidin and higher levels of GDF-15 than HbSS patients during the non-crisis, "steady states." In addition, the investigators propose that when…
- Trial name
- NCT02258997
- Volunteers
- 42 people
- Sponsor
- Kenneth Ataga, MD
Thalassemia, Sickle Cell Anemia
Sickle Cell Anemia Screening and Prevention in Northern Israel
Since 1987, a screening for β Thalassemia in pregnant women is carried on in northern Israel, and from 1999 all the samples were tested also for Hgb S, Hgb C, Hgb D, Hgb O Arab and others.
- Trial name
- NCT00735488
- Testing
- Observational
- Where
- Israel
- Volunteers
- 69 people
- Sponsor
- HaEmek Medical Center, Israel
Sickle Cell Disease, Thalassaemia
Integrative Diagnosis for SCD and Other RADs
INTEGRA aims at enabling personalized medicine for RHADs patients by the establishment of an integrative diagnostic approach based on deep phenotypic and genetic characterization through combining new generation methodologies.
- Conditions
- Sickle Cell Disease, Thalassaemia, Congenital Dyserythropoietic Anemia (CDA), Enzyme Disorder; Anemia
- Trial name
- INTEGRA (NCT07206095)
- Testing
- Analysis of genetic modifiers, Disease phenotyping
- Where
- 9 sites in Spain
- Volunteers
- 200 people
- Sponsor
- Hospital Universitari Vall d'Hebron Research Institute
Sickle Cell Disease
Sickle Cell Anemia in an Arab Bedouin Village in the Northern Israel
Sickle cell anemia and sickle cell thalassemia are frequent diseases among the israeli arab population. The purpose of this study is to assess the clinical characteristics of the patients in one arab village and the laboratory…
- Trial name
- NCT00481039
- Testing
- Medical history and basic laboratory analysis
- Where
- Israel
- Volunteers
- 300 people
- Sponsor
- HaEmek Medical Center, Israel
Anemia, Sickle Cell
Study to Determine the Maximum Tolerated Dose, Safety and Effectiveness of Pomalidomide for Patients With Sickle Cell Disease
The purpose of the study is to determine the maximum tolerated dose, safety and effect on induction of fetal hemoglobin of pomalidomide in patients with Sickle Cell Disease.
- Trial name
- SCD-001 (NCT01522547)
- Testing
- pomalidomide
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Celgene
Sickle Cell Disease, Thalassemia
Determination of Red Cell Survival in Sickle Cell Disease and Other Hemoglobinopathies Using Biotin Labeling
Background: Sickle cell disease (SCD) is an inherited disorder of the blood. SCD causes red blood cells (RBCs) to die early. This can lead to a shortage of healthy cells.
- Conditions
- Sickle Cell Disease, Thalassemia, Hemoglobinopathy
- Trial name
- NCT06313398
- Testing
- Biotin-labeled red blood cells
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Iron, Abnormal Blood Level, Other Abnormal Blood Chemistry
Fresh Versus Old Red Blood Cells for Transfusion
Packed red blood cell units destined for transfusion can be stored for up to 42 days prior to transfusion based on Food and Drug Administration (FDA) guidelines.
- Trial name
- NCT01319552
- Testing
- Fresh transfusion, Old transfusion
- Where
- United States
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Columbia University
Sickle Cell Disease
Expanded Access Protocol for Gene Therapy Utilizing shmiR Lentivirus Vector to Induce Fetal Hemoglobin in Sickle Cell Disease
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine in which genetic material (mostly DNA) in the patient is changed to treat his or her own…
- Trial name
- GRASP-EA (NCT07640815)
- Testing
- Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a
- Where
- United States
- Sponsor
- David Williams
Sickle Cell Disease & Thalassemia
Allogeneic SCT of NiCord®, UCB-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients With Hemoglobinopathies
Allogeneic Stem Cell Transplantation of NiCord®, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients with Hemoglobinopathies
- Trial name
- NCT01590628
- Testing
- NiCord
- Where
- 3 sites in United States
- Volunteers
- 16 people
- Sponsor
- Gamida Cell ltd
Hurler Syndrome, Fanconi Anemia
Bone Marrow Transplant With Abatacept for Non-Malignant Diseases
This is a single arm, phase I study to assess the tolerability of abatacept when combined with cyclosporine and mycophenolate mofetil as graft versus host disease prophylaxis in children undergoing unrelated hematopoietic stem…
- Conditions
- Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome
- Trial name
- NCT01917708
- Testing
- Abatacept
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Emory University
Sickle Red Blood Cell, Fetal Hemoglobin
Effect of Broccoli Sprouts Homogenate on SS RBC
The overall purpose of this study is to obtain a better understanding of the biological response of red blood cells to sulforaphane contained in fresh broccoli sprouts that have been put through a blending process.
- Conditions
- Sickle Red Blood Cell, Fetal Hemoglobin, Oxidative Stress
- Trial name
- BSH (NCT01715480)
- Testing
- Broccosprouts® (Brassica Protection Products LLC) homogenate
- Where
- United States
- Volunteers
- 21 people, healthy volunteers welcome
- Sponsor
- Duke University
Sickle Cell Disease, Thalassemia, Beta
Study of the Role of Genetic Modifiers in Hemoglobinopathies
This study will investigate the role of genetic modifiers in hemoglobinopathies through a large-scale, multi-ethnic genome-wide association study (GWAS).
- Conditions
- Sickle Cell Disease, Thalassemia, Beta, Thalassemia Alpha, Hemoglobinopathies
- Trial name
- INHERENT (NCT05799118)
- Testing
- GWAS
- Where
- 26 sites in United States, Angola and 14 more
- Volunteers
- 30,000 people
- Sponsor
- Cyprus Institute of Neurology and Genetics