Clinical trials: Thalassemia Major
Thalassemia
Endocrine and Nutritional Assessment in B Thalassemia Major
B Thalassemia patients developed short stature and low weight in spite no evident endocrine abnormalities. One hypothesis is that they developed some degree of malnutrition,then the purpose of this study is to assess the…
- Trial name
- NCT00456690
- Testing
- Nutritional recommendations
- Where
- Israel
- Volunteers
- 133 people
- Sponsor
- HaEmek Medical Center, Israel
Pateints of β-thalassemia Major With Iron Overload
Comparison of Deferasirox and Desferoxamine in Patients of β-Thalassemia Major With Iron Overload
This study was planned to compare deferasirox and desferrioxamine in terms of mean serum ferritin levels in patients of β-thalassemia major having Iron overload.
- Trial name
- NCT06468423
- Testing
- Deferasirox (DFX), Deferoxamine (DFO)
- Where
- Pakistan
- Volunteers
- 142 people
- Sponsor
- RESnTEC, Institute of Research
Myeloid Chimerism
Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels
Background: Sickle cell disease can often be treated with blood stem cell transplants. But for some people the disease returns. This study will give a second transplant to people whose disease has returned but still have some…
- Trial name
- NCT04008368
- Testing
- CliniMACS CD34 Reagent
- Where
- United States
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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
Beta-Thalassemia
A Multiple Ascending Dose Study of 9MW3011 in Patients With Non-transfusion-dependent β-thalassemia
This is a phase Ib, randomized, double-blind, placebo-controlled, multiple ascending dose study . The objectives of the study are to evaluate the safety , tolerability, pharmacokinetics(PK), pharmacodynamics(PD), and…
- Trial name
- NCT06772766
- Testing
- 9MW3011
- Where
- 2 sites in China
- Volunteers
- 40 people
- Sponsor
- Mabwell (Shanghai) Bioscience Co., Ltd.
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 Intermedia
Study of SDMB (2,2 Dimethylbutyrate, Sodium Salt) in Beta Thalassemia Intermedia in Thailand
Beta thalassemia intermedia is an inherited blood disease caused by molecular mutations which reduce the beta globin protein chain of adult hemoglobin A, the protein in red blood cells which carries oxygen throughout the body.
- Trial name
- ST20-P2T (NCT01609595)
- Testing
- sodium 2,2 dimethylbutyrate
- Where
- Thailand
- Volunteers
- 10 people
- Sponsor
- Boston University
Transfusion-dependent Beta-Thalassemia
Evaluation the Safety and Efficacy of KL003 Cell Injection in the Treatment of Transfusion-dependent β-thalassemia.
This is a non-randomized, open-label, single-dose study. The aim of this study is to evaluate the safety and efficacy of the treatment with lentiviral vector encoding βA-T87Q-globin gene transduced autologous hematopoietic stem…
- Trial name
- NCT05860595
- Testing
- KL003 cell injection Drug Product
- Where
- China
- Volunteers
- 3 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia
Pharmacokinetics of Oral Hydroxyurea Solution
An open label, safety and pharmacokinetic study of oral hydroxyurea solution administered to children from 6 months to 17.99 years (i.e.
- Conditions
- Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia, Sickle Cell-beta-thalassemia, Sickle Cell Hemoglobin C
- Trial name
- HUPK (NCT03763656)
- Testing
- Oral Hydroxyurea (100 mg/mL) Solution
- Where
- 6 sites in Jamaica, United Kingdom
- Volunteers
- 33 people
- Sponsor
- Nova Laboratories Limited
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
Myelodysplastic Syndromes, Beta-thalassemia
Study to Evaluate Additional Risk Minimisation Measures (aRMMs) for REBLOZYL Among Healthcare Professionals (HCPs)
This is a non-interventional post-authorization safety study (PASS) employing a cross sectional design to evaluate the effectiveness of the additional risk minimization measures (aRMMs) for REBLOZYL.
- Trial name
- NCT04973280
- Where
- 4 sites in Austria, Belgium and 2 more
- Volunteers
- 187 people, healthy volunteers welcome
- Sponsor
- Celgene
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis
Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.
- Conditions
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies
- Trial name
- NCT02231710
- Testing
- BPX-501 and Rimiducid
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Bellicum Pharmaceuticals
Fetal Hemoglobin, Thalassemia Major
Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia
The project "Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia" investigates the safety and efficacy of low-dose thalidomide in managing beta thalassemia, a genetic disorder causing…
- Conditions
- Fetal Hemoglobin, Thalassemia Major, Genetic Disease
- Trial name
- BTM (NCT06490627)
- Testing
- Thalidomide
- Where
- Pakistan
- Volunteers
- 54 people
- Sponsor
- National Institute of Blood and Marrow Transplant (NIBMT), Pakistan
Immune Abnormalities
Immunomodulatory Effects of Silymarin in Patients With Beta-Thalassemia Major
A wide spectrum of immune abnormalities has been described by numerous studies involving β-thalassemic patients with multiple transfusions.
- Trial name
- NCT01752153
- Testing
- Desferrioxamine, Legalon® (Silymarin), Silymarin (Legalon)
- Where
- Iran
- Volunteers
- 25 people
- Sponsor
- Shiraz University of Medical Sciences
Thalassemia
Demographic, Clinical, Laboratory and Genetical Characteristics of Patients With Beta Thalassemia Intermedia
The definition of Thalassemia Intermedia is not generally accepted and the specific clinical and laboratory characteristics varies between patients.
- Trial name
- NCT01443312
- Testing
- Laboratory analysis.
- Where
- Israel
- Volunteers
- 28 people
- Sponsor
- HaEmek Medical Center, Israel
Thalassemia Major, Hemosiderosis
Efficacy Study in Removing Excess Iron From the Heart
The purpose of this study is to determine whether deferiprone has superior efficacy in removing excess iron from the heart when compared with deferoxamine.
- Trial name
- NCT00105495
- Testing
- Ferriprox (deferiprone), Desferal (deferoxamine)
- Where
- 4 sites in Greece, Italy
- Volunteers
- 60 people
- Sponsor
- ApoPharma
Bone Marrow Failure Syndrome, Severe Aplastic Anemia
CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation
This study's goal is to determine the frequency and severity of acute graft versus host disease, to evaluate incidence of primary and secondary graft rejection, to assess event free survival and overall survival, to determine the…
- Conditions
- Bone Marrow Failure Syndrome, Severe Aplastic Anemia, Severe Congenital Neutropenia, Amegakaryocytic Thrombocytopenia
- Trial name
- NCT01966367
- Testing
- CD34 Stem Cell Selection Therapy
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- Diane George
Thalassemia, Beta
Clinical Experience of Thalidomide in Thalassemic Patients
Objectives Primary objective: To determine the efficacy and safety of the combination therapy of Hydroxyurea and thalidomide in beta-thalassemia patients.
- Trial name
- NCT05132270
- Testing
- hydroxyurea and thalidomide
- Where
- Pakistan
- Volunteers
- 135 people
- Sponsor
- Children's Hospital Karachi
Alpha-Thalassemia, Alpha Thalassemia Major
International Registry of Patients With Alpha Thalassemia
This is an international prospective registry of patients with Alpha thalassemia to understand the natural history of the disease and the outcomes of fetal therapies, with the overall goal of improving the prenatal management of…
- Conditions
- Alpha-Thalassemia, Alpha Thalassemia Major, Alpha Thalassemia Minor
- Trial name
- ATM Registry (NCT04872179)
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- University of California, San Francisco
Beta-thalassemia, Iron Overload
Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment
The purpose of this trial is to evaluate changes in cardiac iron as measured by MRI T2* in beta-thalassemia patients with deferasirox treatment.
- Trial name
- NCT00447694
- Testing
- Deferasirox
- Where
- 3 sites in United States
- Volunteers
- 30 people
- Sponsor
- Novartis