Clinical trials: Thalassemia, Beta
Asplenia, β-thalassemia Major
B Memory Cell Response to Vaccination With the 13-valent Pneumococcal Conjugate Vaccine in Asplenic Individuals
The purpose of this study is to determine whether one dose of the 13-valent pneumococcal conjugate vaccine (PCV13) induces immunological memory in asplenic adults and whether previously administered immunizations with the…
- Trial name
- NCT01846923
- Testing
- PCV13
- Where
- Greece
- Volunteers
- 37 people
- Sponsor
- Aghia Sophia Children's Hospital of Athens
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, Thalassemia
RADeep Multicenter European Epidemiological Platform for Patients Diagnosed With Rare Anemia Disorders (RADs)
Rare Anaemia Disorders (RADs) is a group of rare diseases characterized for presenting anaemia as the main clinical manifestation.
- Conditions
- Sickle Cell Disease, Thalassemia, Hemolytic; Anemia, Hereditary, Due to Enzyme Disorder, Anemia Due to Membrane Defect
- Trial name
- RADeep (NCT06213402)
- Testing
- Data collection from EHR.
- Where
- Spain
- Volunteers
- 32,564 people
- Sponsor
- Hospital Universitari Vall d'Hebron Research Institute
Beta Thalassemia Major
Effect of Spirulina Compared to Amlodipine on Cardiac Iron Overload in Children With Beta Thalassemia
the aim of this study is to evaluate the effect of Spirulina compared to Amlodipine on cardiac iron overload and cardiac functions in multi-transfused children with beta thalassemia major
- Trial name
- NCT02671695
- Testing
- Spirulina, Amlodipine
- Where
- Egypt
- Volunteers
- 40 people
- Sponsor
- Tanta University
Beta-Thalassemia
A Study of EPEG in Beta Thalassemia Patients
An open-label study in which 6 patients will receive once-weekly subcutaneous injections of EPEG for 4 weeks. Final visit will occur 60 days after study entry
- Trial name
- NCT02950857
- Testing
- EPEG
- Where
- Thailand
- Volunteers
- 6 people
- Sponsor
- Prolong Pharmaceuticals
Transfusional Iron Overload, Beta-thalassemia
Safety and Pharmacodynamic Study of an Oral Iron Chelator Given for 6 Months to Patients With Iron Overload
The purpose of this research study is to evaluate the safety of two doses of FBS0701, a new oral iron chelator, and its effectiveness in clearing iron from the liver.
- Trial name
- NCT01186419
- Testing
- SPD602 (FBS0701, SSP-004184)
- Where
- 9 sites in United States, Italy and 3 more
- Volunteers
- 51 people
- Sponsor
- Shire
Sickle Cell Disease, Sickle Beta Zero Thalassemia
An mHealth Strategy to Improve Medication Adherence in Adolescents With Sickle Cell Disease
The primary objective of this study is to evaluate a potential behavioral intervention (MED-Go app). To meet this objective, the researchers will conduct a pilot randomized controlled trial to test the feasibility and…
- Conditions
- Sickle Cell Disease, Sickle Beta Zero Thalassemia, Sickle B+ Thalassemia, Sickle Cell Hemoglobin C
- Trial name
- NCT04688411
- Testing
- MED-Go App
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
Thalassemia, Beta, Thalassemia Major
Safety and Efficacy Evaluation of Autologous CRISPR-Cas12b Edited Hematopoietic Stem Cells
This is a single-arm, open, single-injection exploratory clinical study with two transfusion-dependent β thalassemia (β-TDT) participants planned to enroll.
- Trial name
- NCT06041620
- Testing
- VGB-Ex01
- Where
- China
- Volunteers
- 2 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Thalassemia in Children, Pulmonary Hypertension
Erythrocyte Glutamine Level Relation to Pulmonary Hypertension Risk in Beta Thalassemia Major Children
The study will investigate the relation between erythrocyte glutamine/glutamate ratio and pulmonary hypertension risk in Egyptian thalassemic children in Assiut University Children Hospital
- Conditions
- Thalassemia in Children, Pulmonary Hypertension, Hemolysis
- Trial name
- NCT03133169
- Testing
- blood sample, Tricuspid regurge velocity
- Where
- Egypt
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Assiut University
Beta-Thalassemia
A Study of Bitopertin (RO4917838) in Adults With Non-Transfusion-Dependent (NTD) Beta-Thalassemia
This proof-of-mechanism study is being performed to investigate the safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of multiple oral doses of bitopertin in adults with NTD beta-thalassemia.
- Trial name
- NCT03271541
- Testing
- Bitopertin
- Where
- 4 sites in Italy, Lebanon and 1 more
- Volunteers
- 12 people
- Sponsor
- Hoffmann-La Roche
Hemoglobin SC Disease, Sickle Cell Anemia
A Phase I/II Trial of Recombinant-Methionyl Human Stem Cell Factor (SCF) in Adult Patients With Sickling Disorders
Sickle cell anemia is a genetic disorder that results from a single nucleotide substitution in codon 6 of the beta-globin gene which, in the homozygous state, produces an abnormal hemoglobin that is prone to polymer formation…
- Trial name
- NCT00005783
- Testing
- Recombinant-methionyl human stem cell factor
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Sickle Cell Disease, Sickle-Cell; Hb-SC
A Study of IMR-687 in Healthy Adult Volunteers
The purpose of this Phase 1a, first in human, randomized, double-blind, placebo-controlled study is to evaluate the safety, tolerability, PK and PD profile of the orally administered IMR-687 in healthy adult subjects.
- Conditions
- Sickle Cell Disease, Sickle-Cell; Hb-SC, Sickle Beta 0 Thalassemia
- Trial name
- NCT02998450
- Testing
- IMR-687
- Where
- United States
- Volunteers
- 66 people, healthy volunteers welcome
- Sponsor
- Cardurion Pharmaceuticals, Inc.
β-thalassemia Major
A Phase 2 Safety and Efficacy Study Evaluating CS-101 in Participants With β-Thalassemia Major
The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 in treating patients with β-Thalassemia Major
- Trial name
- NCT07489196
- Testing
- CS-101 injection
- Where
- 3 sites in China
- Volunteers
- 20 people
- Sponsor
- CorrectSequence Therapeutics Co., Ltd
Beta Thalassemia
Evaluation of Heart Status in Patients of Beta Thalassemia Using Echocardiogram
Heart failure from myocardial iron deposition is a severe complication for patients with transfusion-dependent beta thalassemia .
- Trial name
- NCT04929574
- Testing
- Echocardiogram and Hematological Analysis
- Where
- China
- Volunteers
- 1,200 people
- Sponsor
- Nanfang Hospital, Southern Medical University
Hemoglobinopathies, Sickle Cell Disease
Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies
The purpose of this study is to determine if treatment with reduced-dose busulfex, fludarabine and alemtuzumab (CAMPATH) followed by sten cell infusion will allow for donor stem cells to grow in patients with hemoglobinopathies…
- Conditions
- Hemoglobinopathies, Sickle Cell Disease, Thalassemia
- Trial name
- NCT00153985
- Testing
- Busulfex, Fludarabine, Alemtuzumab
- Where
- 6 sites in United States
- Volunteers
- 2 people
- Sponsor
- Dana-Farber Cancer Institute
β-thalassemia
A Phase 1 Study of Gene-modified Autologous Hematopoietic Stem Cell (BD211) Treating β-thalassemia Major
This study will be intented to evaluate the safety, tolerability, and engraftment efficacy after myeloablative preconditioning and transplantation of autologous CD34+ hematopoietic stem cells transduced with a lentiviral vector…
- Trial name
- NCT06465550
- Testing
- BD211
- Where
- 3 sites in China
- Volunteers
- 9 people
- Sponsor
- Shanghai BDgene Co., Ltd.
Leukemia, Lymphoma
CD34+Selection for Partially Matched Family or Matched Unrelated Adult Donor Transplant
CD34+ stem cell selection in children, adolescents and young adults receiving partially matched family donor or matched unrelated adult donor allogeneic bone marrow or peripheral blood stem cell transplant will be safe and well…
- Conditions
- Leukemia, Lymphoma, Bone Marrow Failure, Immunodeficiencies
- Trial name
- NCT01049854
- Testing
- Full Intensity with TBI, Full Intensity, Reduced Intensity
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- New York Medical College
Beta Thalassemia Major
Iron Accumulation in Erythrocytes in Patients
Thalassemia refers to a group of blood disorders characterized by a decrease or absence of the synthesis of one or more normal globin chains.
- Trial name
- NCT07486531
- Where
- Turkey (Türkiye)
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Ankara City Hospital Bilkent
Thalassemia Major
Unrelated Umbilical Cord Blood Following HLA-haploidentical Hematopoietic Stem Cell Transplantation in Patients With β-thalassemia Major
Allo-hematopoietic stem cell transplantation(HSCT) is the only way to cure β-thalassemia major at present. To expand donor pool，we developed a haplo-identical HSCT (Hi-HSCT) platform.
- Trial name
- NCT02126046
- Testing
- unrelated CB following haplo-identical hematopoietic stem cells transplantation
- Where
- China
- Volunteers
- 30 people
- Sponsor
- Nanfang Hospital, Southern Medical University
Beta-Thalassemia
A Long-term Follow-up Study in Participants Who Received CS-101
This is a study to evaluate the long-term safety and efficacy of CS-101 in participants who received CS-101 in study CS -101-03 (NCT06065189)
- Trial name
- NCT06479616
- Testing
- CS-101
- Where
- China
- Volunteers
- 5 people
- Sponsor
- Children's Hospital of Fudan University