Clinical trials: Thalassemia, Beta
Myelodysplastic Syndrome (MDS), Aplastic Anaemia (AA)
Traditional Chinese Medicine in the Supportive Management of Anaemic and Cytopenic (Leukopenia, Thrombocytopenia) Haematological Disorders
The purpose of this study is to study the efficacy of Traditional Chinese Medicine (TCM) on anaemic and cytopenic haematological disorders including myelodysplastic syndrome (MDS), aplastic anaemia (AA), myelofibrosis (MF) and…
- Conditions
- Myelodysplastic Syndrome (MDS), Aplastic Anaemia (AA), Myelofibrosis (MF), Thalassemia Intermedia
- Trial name
- NCT01224496
- Testing
- Chinese herbal concoction twice a day for 6 months
- Where
- Singapore
- Volunteers
- 38 people
- Sponsor
- Singapore General Hospital
Transfusion-dependent Beta-Thalassemia
Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy
This is a multi-center, long-term safety and efficacy follow-up study for subjects with transfusion-dependent β-thalassemia (TDT) who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored parent…
- Trial name
- NCT02633943
- Testing
- Safety and efficacy assessments
- Where
- 15 sites in United States, Australia and 6 more
- Volunteers
- 66 people
- Sponsor
- Genetix Biotherapeutics Inc.
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
Thalassemia Major
Observing the Changes of Endocrine and Metabolism in Patients With Thalassemia Major
Patient with thalassemia major have many endocrine diseases, such as hypopituitarism, hypothyroidism, hypoparathyroidis, osteoporosis, and etc.. These problems may be due to anemia itself or related to iron deposition.
- Trial name
- NCT03951818
- Where
- Taiwan
- Volunteers
- 100 people
- Sponsor
- National Taiwan University Hospital
Alpha-Thalassemia, Beta-Thalassemia
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases.
- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis
- Trial name
- NCT06539169
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- xCures
B Thalassemia Major
Oral Health Status in Children With β-Thalassemia Major
To evaluate the effectiveness of a structured oral health educational program on oral hygiene status and oral health-related quality of life (OHRQoL) among children diagnosed with β-thalassemia.
- Trial name
- NCT07614217
- Testing
- oral health educational program for Immediate Intervention Group, routine oral hygiene instructions
- Volunteers
- 46 people
- Sponsor
- Cairo University
Sickle Cell Disease, Sickle Cell Anemia
Decision Aid for Therapeutic Options In Sickle Cell Disease
Sickle cell disease (SCD) is an inherited disorder with chronic multi-system manifestations affecting 100,000 individuals in the US, largely of minority origin and associated with substantial morbidity, premature mortality…
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia, Hemoglobin SS, Hemoglobin SC
- Trial name
- NCT02326597
- Testing
- Decision Aid Tool, Standard Practice
- Where
- United States
- Volunteers
- 134 people, healthy volunteers welcome
- Sponsor
- Emory University
Beta Thalassemia Intermedia
Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx)
The purpose was to evaluate the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics of sapablursen administered subcutaneously to participants with non-transfusion dependent β-Thalassemia Intermedia.
- Trial name
- NCT04059406
- Testing
- sapablursen
- Where
- 19 sites in Australia, Greece and 3 more
- Volunteers
- 29 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Leukemia, Myeloid, Acute, Leukemia, B-cell
Precision Exercise in Children With Malignant Hemopathies
In the early years of life and during adolescence, physical activity is crucial for good development of motor skills. It is even more so for those children and young people who are forced to undergo anti-cancer therapies and…
- Conditions
- Leukemia, Myeloid, Acute, Leukemia, B-cell, Leukemia, T Cell, Lymphoma, Hodgkin
- Trial name
- SportTherapy (NCT04090268)
- Testing
- Sport Therapy
- Where
- Italy
- Volunteers
- 380 people, healthy volunteers welcome
- Sponsor
- University of Milano Bicocca
Transfusion Dependent Beta Thalassemia
Glucagon-Like Peptide 1 Receptor Agonist in Diabetes Mellitus Management in Children and Adolescents With Transfusion-Dependent Thalassemia
Blood transfusion and iron-chelation therapy have prolonged and improved the quality of life in patients with β-thalassemia. The improvement was mainly due to the decrease in mortality from heart failure Such a treatment…
- Trial name
- NCT07370922
- Testing
- Dulaglutide 0.75Mg/0.5Ml Inj Pen, control group (insulin)
- Where
- Egypt
- Volunteers
- 80 people
- Sponsor
- Ain Shams University
Red Cell Alloantibodies, Blood Transfusion-Dependent
Evaluation of Micro RNA-155 Expression in Relation to Alloantibody Formation in Transfusion-Dependent Patients
Transfusion-dependent patients, particularly those with β-thalassemia major, require lifelong regular red blood cell (RBC) transfusions to maintain adequate hemoglobin levels and prevent severe anemia.
- Conditions
- Red Cell Alloantibodies, Blood Transfusion-Dependent, Beta Thalassemia
- Trial name
- NCT07480980
- Volunteers
- 100 people
- Sponsor
- Assiut University
β Thalassemia Major
Safety and Efficacy Evaluation of γ-globin Reactivated Autologous Hematopoietic Stem Cells
This is a non-randomized, open label, single-dose, phase 1/2 study in up to 12 participants with β-thalassemia major.This study aims to evaluate the safety and efficacy of the treatment with γ-globin reactivated autologous…
- Trial name
- NCT04211480
- Testing
- γ-globin reactivated autologous hematopoietic stem cells
- Where
- China
- Volunteers
- 6 people
- Sponsor
- Bioray Laboratories
Beta-Thalassemia
The Prevelence of HBB c.93-21 G-A in β Thalassemia Patients
To design an amplification-refractory mutation system (ARMS) for the DNA diagnosis of the IVS I-110 (G>A) [HBB:c.93-21G˃A] mutation. To detect the prevelence of the mutation among Assiut University Hospital patients.
- Trial name
- NCT05133388
- Testing
- ARMS PCR
- Where
- Egypt
- Volunteers
- 150 people
- Sponsor
- Assiut University
β-thalassemia Intermedia
Apotransferrin in Patients With β-thalassemia
The aim of the trial is to study the effect of apotransferrin administration in patients suffering from β-thalassemia intermedia in order to restore the erythropoiesis as reflected by enhanced haemoglobin levels or reduced…
- Trial name
- NCT03993613
- Testing
- human apotransferrin
- Where
- Netherlands
- Volunteers
- 10 people
- Sponsor
- Prothya Biosolutions
Thalassemia Major
Monitoring of Erythroid Lineage Specific Chimerism Following Allogeneic Hematopoietic Transplantation for Thalassemia Major
The purpose of this study is to collect peripheral blood and bone marrow aspirate samples from thalassemia patients in Tehran, in a collaborative effort to develop an erythroid lineage specific chimerism assay applicable to…
- Trial name
- NCT00480506
- Where
- 2 sites in United States, Iran
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Dana-Farber Cancer Institute
β-thalassemia, Ineffective Erythropoiesis
Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects With Chronic Anemia
This is a Phase 2 open-label, single-arm design study with dose escalation by subject cohort . The study is designed to monitor the PTG-300 safety profile, to obtain preliminary evidence of efficacy of PTG-300 for the treatment…
- Conditions
- β-thalassemia, Ineffective Erythropoiesis, Chronic Anemia
- Trial name
- TRANSCEND (NCT03802201)
- Testing
- PTG-300
- Where
- 33 sites in United States, Greece and 7 more
- Volunteers
- 63 people
- Sponsor
- Protagonist Therapeutics, Inc.
Thalassemia, Beta
The Efficacy and Safety of HYDROXYUREA in Management of Beta Thalassemia Patients in Karachi Pakistan
Objectives Primary objectives: To determine the efficacy of Hydroxyurea in the study participants. Hypothesis: The study will result in either maintenance or rise in hemoglobin as compared to the control treatment.
- Trial name
- NCT03183375
- Testing
- Hydroxyurea
- Where
- Pakistan
- Volunteers
- 100 people
- Sponsor
- Dr.Saqib Hussain Ansari
Hemoglobinopathies
Prevalence of Hemoglobinopathies Associated With Significant Hb Variants in the Chattogram Region of Bangladesh
Hemoglobinopathies represent a collection of genetic conditions that influence the structure or synthesis of haemoglobin, the protein found in red blood cells that facilitates oxygen transport from the lungs throughout the body.
- Trial name
- Hb variants (NCT07207551)
- Where
- Bangladesh
- Volunteers
- 760 people
- Sponsor
- Bangladesh Bioscience Research Group
Beta Thalassemia Major
An Algorithm to Start Iron Chelation in Minimally Transfused Young Beta-thalassemia Major Patients
A prospective randomized study on Safety, Tolerability and Efficacy of oral Low dose DFP (50 mg/kg/day) in minimally transfused B-TM after 5 transfusions when SF reaches 500 ng/m and with either appearance of LPI > 0.2 or TSAT…
- Trial name
- NCT02173951
- Testing
- Deferiprone
- Where
- Egypt
- Volunteers
- 64 people
- Sponsor
- Ain Shams University
Thalassemia Major, Thalassemia Intermedia
Prevalence of Pulmonary Hypertension (PAH) in Patients With Thalassemia
This is a multicenter observational case-control analysis lasting 12 months aimed at determining the prevalence of pulmonary hypertension (PAH) in patients with Thalassemia Major and Intermedia.
- Conditions
- Thalassemia Major, Thalassemia Intermedia, Pulmonary Arterial Hypertension
- Trial name
- PAH2010 (NCT01496963)
- Testing
- Physician standard-of-care according to ESC/ERS Guidelines
- Where
- 8 sites in Italy
- Volunteers
- 1,500 people
- Sponsor
- Ente Ospedaliero Ospedali Galliera