Clinical trials: Sickle Cell Thalassemia
Non Malignant Disorders, Immunodeficiencies
Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders
The primary objective is to determine the feasibility of attaining acceptable rates of donor cell engraftment (>25% donor chimerism at 180 days) following reduced intensity conditioning (RIC) regimens in pediatric patients < 21…
- Conditions
- Non Malignant Disorders, Immunodeficiencies, Congenital Marrow Failures, Hemoglobinopathies
- Trial name
- NCT00744692
- Testing
- Unrelated Umbilical Cord Blood Transplant, Reduced Intensity Conditioning
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Duke University
Sickle Cell Disease, Beta Thalassemia Intermedia
Pumilio1 (PUM1) Expression, Sickle Cell Anemia, β-thalassemia Intermedia
1. To study the expression pattern of PUM1 gene in patients with sickle cell anemia and β-thalassemia intermedia. 2. To detect PUM1 protein levels in sickle cell anemia and β-thalassemia intermedia patients. 3.
- Trial name
- NCT05883254
- Testing
- RNA Binding Protein Pumilio1 (PUM1) Expression by reverse transcriptase quantatative PCR
- Volunteers
- 80 people
- Sponsor
- Assiut University
Sickle Cell-thalassemia Disease, Thalassemia
Haploidentical Hematopoietic Stem Cell Transplantation
The study is designed as a Pilot/Phase 1 trial of reduced intensity Haploidentical HSCT in patients with sickle cell disease and thalassemia.
- Trial name
- NCT02165007
- Testing
- peripheral blood stem cell graft that are CD34+ selected
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Catherine Bollard
Sickle Cell Anemia, Sickle Cell-hemoglobin C Disease
Treatment of Sickle Cell Anemia With Stem Cell Transplant
This is a clinical research trial in which a novel preparatory regimen was developed for bone marrow transplant (BMT) which eliminates the primary obstacle to transplant, the lack of a matched sibling donor.
- Conditions
- Sickle Cell Anemia, Sickle Cell-hemoglobin C Disease, Sickle Cell-β0-thalassemia
- Trial name
- NCT01350232
- Testing
- Fludarabine, Cytarabine, Cellular Infusions
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
Sickle Cell Anemia, Thalassemia
L-Glutamine Therapy for Sickle Cell Anemia
The primary purpose is to evaluate the effect of L-glutamine therapy on exercise endurance and breath by breath exercise response of sickle cell anemia patients The secondary purpose is to assess the effect of L-glutamine on…
- Trial name
- NCT00586209
- Testing
- L-Glutamine
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Bone Marrow Failure Syndrome, Thalassemia
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum…
- Conditions
- Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia
- Trial name
- NCT03513328
- Testing
- Thiotepa--single daily dose, Thiotepa--escalated dose
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Florida
Sickle Cell Disease
Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease
This is a follow-up trial to NYMC 526 (NCT01461837) to assess the safety, efficacy and toxicity of administering Defibrotide prophylaxis for high-risk sickle cell or beta thalassemia patients undergoing a familial haploidentical…
- Trial name
- NYMC-571 (NCT02675959)
- Testing
- Defibrotide
- Where
- 4 sites in United States
- Volunteers
- 40 people
- Sponsor
- New York Medical College
Transfusion-dependent Hemachromatosis, Thalassemia Major
Pilot Study for Patients With Poor Response to Deferasirox
This purpose of this study is to understand the differences between people who have a good response to deferasirox (exjade) compared to people who have a poor response to this medication when used for transfusion-dependent iron…
- Conditions
- Transfusion-dependent Hemachromatosis, Thalassemia Major, Sickle Cell Disease
- Trial name
- NCT00749515
- Testing
- Deferoxamine, Deferasirox, HIDA
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Boston Children's Hospital
Graft Versus Host Disease
Acute GVHD Suppression Using Costimulation Blockade to Expand Non-malignant Transplant
This trial will see if extended abatacept administration (combined with a standard regimen of tacrolimus and mycophenolate mofetil) will prevent acute and chronic graft-versus-host disease (GVHD) in children and adolescents…
- Trial name
- ASCENT (NCT03924401)
- Testing
- Abatacept
- Where
- 9 sites in United States
- Volunteers
- 30 people
- Sponsor
- Emory University
Sickle Cell Disease, Thalassemia
Genes Influencing Iron Overload State
Iron overload, which can be defined operationally as too much iron in the body, develops as a consequence of too many blood transfusions given, or due to genetic defects hereditary hemochromatosis).
- Conditions
- Sickle Cell Disease, Thalassemia, Marrow Aplasia
- Trial name
- NCT01158794
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- St. Jude Children's Research Hospital
Vitamin D Deficiency, Stem Cell Transplant Complications
A Randomized Phase IV Control Trial of Single High Dose Oral Vitamin D3 in Pediatric Patients Undergoing HSCT
Research has suggested that children with sufficient vitamin D levels undergoing hematopoietic stem cell transplant (HSCT) have improved outcomes, including lower incidences of infection and graft-versus-host disease (GVHD), as…
- Conditions
- Vitamin D Deficiency, Stem Cell Transplant Complications, Pediatric Cancer, Blood Disorder
- Trial name
- NCT03176849
- Testing
- Vitamin D3, Standard Vitamin D3 Supplementation
- Where
- United States
- Volunteers
- 49 people
- Sponsor
- Phoenix Children's Hospital
Sickle Cell Disease
Enhancing Use of Hydroxyurea In Sickle Cell Disease Using Patient Navigators
Multi-phase, patient navigator-based program in the Richmond and Tidewater regions of Virginia to demonstrate: 1. the feasibility of using patient navigators to improve the percentage of children and adult (age 15 and older)…
- Trial name
- SHiP HU (NCT02197845)
- Testing
- Patient Navigator, Recruitment into Specialty Care
- Where
- United States
- Volunteers
- 353 people
- Sponsor
- Virginia Commonwealth University
Sickle Cell Anemia, Sickle Cell-Beta0-Thalassemia
The Effect of Rivaroxaban in Sickle Cell Disease
The primary study hypothesis is that inhibition of factor Xa with rivaroxaban will reduce inflammation, coagulation and endothelial cell activation, and improve microvascular blood flow in patients with sickle cell disease (SCD)…
- Trial name
- NCT02072668
- Testing
- rivaroxaban
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- University of North Carolina, Chapel Hill
Sickle Cell Disease, Thalassemia
Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure
Background: Some sickle cell disease or beta-thalassemia can be cured with transplant. Researchers want to test a variation of transplant that uses low dose radiation and a combination of immunosuppressive drugs.
- Conditions
- Sickle Cell Disease, Thalassemia, Stem Cell Transplantation, Graft vs Host Disease
- Trial name
- NCT02105766
- Testing
- Alemtuzumab, Sirolimus, Cyclophosphamide
- Where
- United States
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Anemia, Sickle Cell Thalassemia
Investigating the Mechanistic Effects of Mitapivat in Subjects With Sickle Cell Disease
Background: Sickle cell disease (SCD) is an inherited blood disorder. The disease affects the ability of red blood cells to carry oxygen; this in turn can injure organs including the heart, lungs, and kidneys.
- Conditions
- Sickle Cell Anemia, Sickle Cell Thalassemia, Sickle Cell Pain, Hbss
- Trial name
- NCT05675436
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia
Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies
This is a study to collect the outcomes of stem cell transplantation for patients with hematologic diseases other than cancer.
- Conditions
- Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia, Diamond Blackfan Anemia, Paroxysmal Nocturnal Hemoglobinuria
- Trial name
- NCT02179359
- Testing
- Reduced Toxicity Ablative Regimen, Reduced Intensity Preparative Regimen, Myeloablative Preparative Regimen
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Sickle Cell Anemia
The Role of Endothelin-1 in Sickle Cell Disease
The primary goal of the study is to determine the safety and tolerability of ambrisentan. It is also expected that ambrisentan will improve blood flow in the lungs, decrease inflammation, and reduce pain in sickle cell patients.
- Trial name
- NCT02712346
- Testing
- Ambrisentan
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- Augusta University
Iron Overload
MRI Evaluation of Iron Overload in the Heart, Liver and Pancreas in Patients Receiving Multiple Blood Transfusions.
Sickle cell anemia and Sickle cell β thalassemia patients require multiple transfusions in order to avoid chronic anemia sequel.
- Trial name
- NCT00629291
- Where
- Israel
- Volunteers
- 60 people
- Sponsor
- Sheba Medical Center
Hematologic Diseases, Anemia, Sickle Cell
Cord Blood Transplantation for Sickle Cell Anemia and Thalassemia
This study will develop a national cord blood bank for siblings of patients with hemoglobinopathies and thalassemia.
- Conditions
- Hematologic Diseases, Anemia, Sickle Cell, Beta-Thalassemia, Hematopoietic Stem Cell Transplantation
- Trial name
- NCT00029380
- Testing
- Sangstat, Cyclophosphamide, Busulfan
- Where
- 15 sites in United States, Canada
- Volunteers
- 30 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease, Thalassemia
Hydroxyurea Therapy: Optimizing Access in Pediatric Populations Everywhere
Primary Objective 1. Define the pharmacokinetics of liquid-formulated HU in infants (9 months to <2 years) 2. Assess the relative bioavailability of HU "sprinkles" compared to capsules in children and adolescents (≥2 to 18…
- Trial name
- NCT03825341
- Testing
- Hydroxyurea, Hydroxyurea Oral Capsule
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- St. Jude Children's Research Hospital