Clinical trials: Sickle Cell Thalassemia
Hemoglobinopathies
A Clinical Exploratory Study of YOLT-204 in the Treatment of Hemoglobinopathies
This is a single-arm, open-label, dose-escalation study designed to enroll approximately 5-30 patients with TDT or SCD. The objectives are to evaluate the safety and tolerability in patients following intravenous administration…
- Trial name
- NCT07774351
- Testing
- YOLT-204
- Volunteers
- 30 people
- Sponsor
- Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
Sickle Cell Anemia, Hemoglobinopathy
Bone Marrow Transplant From Related Donor for Patients With High Risk Hemoglobinopathies
The major goal of this study is to determine the risks and benefits of bone marrow transplants in patients with severe thalassemia or sickle cell disease. Participation in this project will be for two years.
- Conditions
- Sickle Cell Anemia, Hemoglobinopathy, Thalassemia
- Trial name
- NCT00040469
- Testing
- Campath -1H, Dilantin, Busulfan
- Where
- 2 sites in United States
- Volunteers
- 15 people
- Sponsor
- Baylor College of Medicine
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially…
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndrome
- Trial name
- NCT02065869
- Testing
- BPX-501 T cells, Rimiducid
- Where
- 4 sites in Italy, United Kingdom
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Sickle Cell Anemia, Sickle Cell Thalassemia
Iron Overload Assesment in Sickle Cell Anemia and Sickle Cell Thalassemia
Iron overload is well study in Thalassemia patients and it's not only related to blood transfusions, since intestinal iron absorption is also increased in those patients.
- Conditions
- Sickle Cell Anemia, Sickle Cell Thalassemia, Iron Overload, MRI
- Trial name
- NCT00512226
- Testing
- Cardiac and Liver T2* MRI
- Where
- Israel
- Volunteers
- 50 people
- Sponsor
- HaEmek Medical Center, Israel
Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat his or her own disease.
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0), Beta-Thalassemia, Transfusion Dependent Beta-Thalassaemia
- Trial name
- NCT06647979
- Testing
- autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, Sequencing Assay for Variant rs114518452
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Daniel Bauer
Hemoglobinopathies
Screening for Hemoglobinopathies in Pregnant Women
This prospective monocentric study project is to identify hemoglobinopathies in pregnant women in order to optimize antenatal care and to investigate the prevalence of hemoglobinopathies in pregnant women in Switzerland.
- Trial name
- NCT04029142
- Testing
- Review of routine lab data
- Where
- Switzerland
- Volunteers
- 1,785 people, healthy volunteers welcome
- Sponsor
- University Hospital, Basel, Switzerland
All Diagnosed Health Conditions, ADD/ADHD
At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions
We are the missing link in clinical trials, connecting patients and researchers seamlessly and conveniently using a mobile health platform to advance medical research.
- Conditions
- All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis
- Trial name
- NCT01931644
- Where
- United States
- Volunteers
- 17,667 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
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, Thalassemia
Allogeneic SCT of CordIn™, in Patients With Hemoglobinopathies
CordIn™ is a cryopreserved stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells. The overall study objectives are to evaluate the safety and efficacy of CordIn™.
- Trial name
- NCT02504619
- Testing
- CordIn
- Where
- 3 sites in United States, France
- Volunteers
- 1 people
- Sponsor
- Gamida Cell ltd
Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML)
CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant
The purpose of this study is to learn more about the effects of (classification determinant) CD34+ stem cell selection on graft versus host disease (GVHD) in children, adolescents, and young adults.
- Conditions
- Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome (MDS), Juvenile Myelomonocytic Leukemia (JMML)
- Trial name
- NCT02061800
- Testing
- CliniMACS CD34+ Reagent System, Thiotepa, Cyclophosphamide
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Diane George
Sickle Cell Disease, HEMOGLOBIN SS
A Randomized Trial of LOVAZA in Pediatric Sickle Cell Disease (SCD)
The purpose of the study is to determine the effectiveness of LOVAZA (fish oil capsules) to decrease inflammation in children and adolescents with Sickle Cell Disease (SCD).
- Conditions
- Sickle Cell Disease, HEMOGLOBIN SS, Hemoglobin S Beta-0 Thalassemia, Inflammation
- Trial name
- NCT01202812
- Testing
- Omega-3 Fatty Acids: Eicosapentaenoic Acid (EPA) and Docosahexaenoic Acid (DHA)
- Where
- 2 sites in United States
- Volunteers
- 48 people
- Sponsor
- Thomas Jefferson University
Sickle Cell Disease, Thalassemia
A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood disorders that affect haemoglobin.
- Trial name
- FLORAL (NCT06609226)
- Testing
- Etavopivat A, Etavopivat B, Etavopivat C
- Where
- 107 sites in United States, Canada and 15 more
- Volunteers
- 480 people
- Sponsor
- Novo Nordisk A/S
Sickle Cell Disease, Sickle Cell Anemia
Efficacy of Vorinostat to Induce Fetal Hemoglobin in Sickle Cell Disease
Sickle Cell Disease (SCD) is a hereditary anemia that causes the red blood cells to change their shape from a round and doughnut-like shape to a half-moon/crescent, or sickled shape.
- Trial name
- NCT01000155
- Testing
- vorinostat
- Where
- 3 sites in United States
- Volunteers
- 5 people
- Sponsor
- Dana-Farber Cancer Institute
Anemia, Sickle Cell
Far Infrared Radiation for Sickle Cell Pain Management
Most patients with sickle-cell disease have periodic intensely painful episodes. To manage this pain, we are proposing the drinking of at least 500 mL of water followed by far infrared radiation.
- Trial name
- NCT00599482
- Testing
- Far Infrared Radiation (5μm to 20μm wavelength)
- Where
- Canada
- Volunteers
- 10 people
- Sponsor
- GAAD Medical Research Institute Inc.
Transfusional Iron Overload, Beta-Thalassemia
Safety, Efficacy and Pharmacokinetics of an Oral Iron Chelator Given for a Year to Pediatric Patients With Iron Overload
This is an open-label study to assess the pharmacokinetics, safety, efficacy and tolerability of SSP-004184AQ. The study consists of two phases: the pharmacokinetic phase, using a single 16 mg/kg dose of SSP-004184AQ; and the…
- Trial name
- NCT01363908
- Testing
- SPD602
- Where
- 9 sites in United States, Canada and 3 more
- Volunteers
- 30 people
- Sponsor
- Shire
Sickle Cell Disease
Screening Patients With Sickle Cell Disease for Kidney Damage
This study aims to study the temporal course of sickle nephropathy and assess novel biomarkers that can predict patients prone to nephropathy.
- Trial name
- NCT02239016
- Where
- 4 sites in United States
- Volunteers
- 320 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Hemoglobinopathies, Anemia, Sickle Cell
Stem Cell Transplantation After Reduced-Dose Chemotherapy for Patients With Sickle Cell Disease or Thalassemia
The purpose of this study is to find out if using a lower dose of chemotherapy before stem cell transplantation can cure patients of sickle cell anemia or thalassemia while causing fewer severe side effects than conventional high…
- Conditions
- Hemoglobinopathies, Anemia, Sickle Cell, Hemoglobin SC Disease, Thalassemia
- Trial name
- NCT00034528
- Testing
- Busulfan, Fludarabine, FK506
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Transfusional Iron Overload, Beta-thalassemia
Safety and Pharmacokinetic Study of Escalating Multiple Doses of an Iron Chelator in Patients With Iron Overload
The purpose of this research study is to study the safety of increasing doses of FBS0701, and to see how quickly the study medication is absorbed and how quickly it disappears from the bloodstream.
- Trial name
- NCT01039636
- Testing
- FBS0701
- Where
- 5 sites in United States, Australia and 1 more
- Volunteers
- 20 people
- Sponsor
- FerroKin BioSciences, Inc.
Acute Leukemia, Severe Aplastic Anemia
AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion
Children, adolescents, and young adults with malignant and non-malignant conditionsundergoing an allogeneic stem cell transplantation (AlloSCT) will have the stem cells selected utilizing α/β CD3+/CD19+ cell depletion.
- Conditions
- Acute Leukemia, Severe Aplastic Anemia, Non-hodgkin Lymphoma, Hodgkin Lymphoma
- Trial name
- NCT04099966
- Testing
- alpha beta depletion
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Mitchell Cairo
Sickle Cell Disease
Gene Transfer for 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 that uses genetic material (mostly DNA) from the patient to treat his or her own disease.
- Trial name
- NCT03282656
- Testing
- single infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11a
- Where
- 2 sites in United States
- Volunteers
- 10 people
- Sponsor
- David Williams