Clinical trials: Thalassemia
Thalassemia in Children
Observational Study: Hetrombopag for Platelet Recovery in Haploidentical HSCT
The objective of this observational study is to investigate the long-term effects of Hetrombopag in promoting platelet engraftment during haploidentical hematopoietic stem cell transplantation (HSCT) in children with thalassemia…
- Trial name
- NCT07003269
- Testing
- Hetrombopag
- Where
- China
- Volunteers
- 30 people
- Sponsor
- Haikou Affiliated Hospital of Central South University Xiangya School of Medicine
Beta-Thalassemia
Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector
This is a Phase I/II clinical trial of gene transfer for treating Beta-thalassemia using a self-inactivating lentiviral vector to functionally correct the defective gene(s).
- Trial name
- NCT03351829
- Testing
- Gene-modified autologous hematopoeitic stem cells
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute
Thalassemia Intermedia, Thalassemia Major
Growth Evaluation, Health Promotion, and Clinical Management in Children and Adolescents With Thalassemia
There are nearly 300,000 patients with severe or intermediate thalassemia in China. Growth retardation is the most significant health issue for children and adolescents with transfusion-dependent thalassemia (TDT), placing a…
- Conditions
- Thalassemia Intermedia, Thalassemia Major, Growth Delay, Puberty, Delayed
- Trial name
- NCT06931912
- Testing
- MENBS clinical interventions
- Where
- China
- Volunteers
- 385 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Thalassemia
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Healthy Volunteers
The purpose is to assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS TMPRSS6-Lrx Administered Subcutaneously for up to 44 Healthy Volunteers
- Trial name
- NCT03165864
- Testing
- IONIS TMPRSS6-Lrx
- Where
- Australia
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Ionis Pharmaceuticals, Inc.
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
Thalassemia, Iron Overload
Renal Function Among Thalassemia Patients Treated by a Oral Chelator Deferasirox
Thalassemia Major patients developed Iron Overload due to blood transfusions and intestinal iron absorption. Renal function caused by Iron overload was studied in a previous study and shows principally tubular disfunction.
- Trial name
- NCT01905774
- Testing
- Deferasirox
- Where
- Israel
- Volunteers
- 36 people
- Sponsor
- HaEmek Medical Center, Israel
Thalassemia
Iron Balance Study of Deferasirox, Deferoxamine and the Combination of Both
Subjects with thalassemia major require regular transfusion therapy to sustain life. The iron present in the transfused blood remains in the body where it can cause a variety of organ dysfunctions.
- Trial name
- NCT00738413
- Testing
- Deferoxamine, Deferasirox
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Weill Medical College of Cornell University
Beta-Thalassemia Major
A Safety and Efficacy Study Evaluating CS-101 in Subjects 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 anemia.
- Trial name
- NCT06291961
- Testing
- CS-101 injection
- Where
- 3 sites in China
- Volunteers
- 9 people
- Sponsor
- CorrectSequence Therapeutics Co., Ltd
β Thalassemia
A Study of IMR-687 in Subjects With Beta Thalassemia
A Study to Evaluate the Safety and Tolerability of IMR-687 in Subjects with Beta Thalassemia
- Trial name
- NCT04411082
- Testing
- IMR-687
- Where
- 36 sites in Denmark, France and 11 more
- Volunteers
- 122 people
- Sponsor
- Cardurion Pharmaceuticals, Inc.
Anemia, Hemosiderosis
Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Rare Chronic Anemia Patients
A 1-year randomized Phase II core trial was conducted to investigate the efficacy of deferasirox in regularly transfused patients with β-thalassemia and other rare chronic anemia 2 years of age and older.
- Trial name
- NCT00303329
- Testing
- Deferasirox
- Where
- 28 sites in United States, Belgium and 5 more
- Volunteers
- 184 people
- Sponsor
- Novartis Pharmaceuticals
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)
Leukemia, Lymphoma
Dexamethasone, Intravenous Injection of Human Immunoglobulin, and Increased Infusion of Mononuclear Cells to Reduce Donor Specific Antibodies in Haploidentical Hematopoietic Stem Cell Transplantation
This study tests whether a combination of three treatments - dexamethasone (a steroid), intravenous immunoglobulin (IVIG, a protein that helps the immune system), and an extra dose of donor mononuclear cells - can safely lower…
- Conditions
- Leukemia, Lymphoma, Thalassemia, Aplastic Anemia
- Trial name
- NCT07698080
- Testing
- Intravenous Immunoglobulin, Dexamethasone, Mononuclear Cells
- Volunteers
- 60 people
- Sponsor
- Hematology department of the 920th hospital
HbE Trait, Hemoglobinopathies
Prevalence of Abnormal Haemoglobin Variants in Metropolitan Chattogram, Bangladesh
Hemoglobinopathies are inherited disorders that affect haemoglobin and pose a considerable global health challenge. This research addresses the knowledge gap regarding the prevalence of a typical haemoglobin fraction within urban…
- Trial name
- Hb Variants (NCT07305181)
- Where
- Bangladesh
- Volunteers
- 8,400 people
- Sponsor
- Bangladesh Bioscience Research Group
Hemoglobin Mutations, Erythrocyte Variants
Collection of Blood From Persons With Hemoglobin and Erythrocyte Polymorphisms for Laboratory Malaria Research
This study will collect blood samples for use in laboratory studies of malaria. The World Health Organization reports that 40 percent of the world's population is at risk for malaria, mostly in the poorest countries.
- Trial name
- NCT00277849
- Where
- United States
- Volunteers
- 130 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
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)
Thalassemia Major
Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study
The only curative therapy for thalassemia major remains the replacement of the defective erythropoiesis by allogeneic hematopoietic stem cell transplantation(allo-HSCT).
- Trial name
- NCT04009525
- Testing
- Busulfan, Cyclophosphamide, Fludarabine
- Where
- China
- Volunteers
- 823 people
- Sponsor
- First Affiliated Hospital of Guangxi Medical University
Beta-Thalassemia
Safety & Efficacy of ICL670 vs. Deferoxamine in Beta-thalassemia Patients With Iron Overload Due to Blood Transfusions
The purpose of this study is to deterimine if the new orally active iron chelator, ICL670, is as effective and as safe as deferoxamine in preventing accumulation of iron in the body while a patient is undergoing repeated blood…
- Trial name
- NCT00061750
- Testing
- ICL670, deferoxamine
- Where
- 7 sites in United States
- Volunteers
- 595 people
- Sponsor
- Novartis Pharmaceuticals
Diabetes Mellitus, Heart Disease
Deferoxamine for the Treatment of Hemochromatosis
When patients receive repeated blood transfusions the level of iron in the patient s blood can rise. When iron is processed in the body a protein known as hemosiderin can begin collecting in the organs.
- Conditions
- Diabetes Mellitus, Heart Disease, Hemochromatosis, Thalassemia
- Trial name
- NCT00001203
- Where
- United States
- Volunteers
- 151 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Thalassemia, Beta, Thalassemia
Impact of Virtual Reality on Pain and Anxiety During Intravenous Cannulation Procedures Among Thalassemia Patients in UAE.
The goal of this trial is to evaluate the effects of therapeutic Virtual Reality (VR) on pain, anxiety, fatigue and boredom during intravenous (IV) cannulations in comparison to standard of care (SOC).The main question it aims to…
- Conditions
- Thalassemia, Beta, Thalassemia, Pain, Anxiety
- Trial name
- NCT07099196
- Testing
- Headset-based Virtual Reality Simulation
- Where
- United Arab Emirates
- Volunteers
- 115 people
- Sponsor
- Mohammed Bin Rashid University of Medicine and Health Sciences