Clinical trials: Thalassemia Major
Sickle Cell Disease, Beta Thalassemia
Stem Cell Transplant in Sickle Cell Disease and Thalassemia
The primary purpose of this study is to see if giving lower doses of chemotherapy (moderately ablative) will result in successful bone marrow replacement without as severe side-effects but with permanent control of the disease.
- Trial name
- NCT00408447
- Testing
- Busulfan, Fludarabine, Alemtuzumab
- Where
- United States
- Volunteers
- 53 people
- Sponsor
- Columbia University
Thalassemia Major, Osteoporosis
Denosumab vs Placebo in Patients With Thalassemia Major and Osteoporosis
This is a single-site, randomized, placebo-controlled, double blind phase 2b clinical trial. Patients with Thalassemia will participate in this study and will be treated with Denosumab or placebo.
- Trial name
- NCT02559648
- Testing
- Denosumab
- Where
- Greece
- Volunteers
- 63 people
- Sponsor
- Ersi Voskaridou
Beta-Thalassemia
A Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Patients With Transfusion-Dependent Beta Thalassemia
This study is a Phase 2 multicenter, randomized, open-label, parallel-group study. The primary objective of the study is to evaluate the effect of LJPC-401 (synthetic human hepcidin) on iron levels in patients with…
- Trial name
- NCT03381833
- Testing
- LJPC-401
- Where
- 22 sites in United States, Australia and 6 more
- Volunteers
- 84 people
- Sponsor
- La Jolla Pharmaceutical Company
β-thalassemia
Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent Beta-thalassemia
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
- NCT05773729
- Testing
- BD211
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Shanghai BDgene Co., Ltd.
Transfusion Dependent Beta Thalassemia
Combination of Thalidomide and Hydroxyuria in Transfusion Dependent Thalasemmia
Beta thalassemia Major (BTM) is the most common hemoglobinopathy caused by mutations in the beta-globin gene . Worldwide, approximately 80 million people carry thalassemia gene mutation.
- Trial name
- Thal-H (NCT07292259)
- Testing
- ADDITION OF THALIDOMIDE AND HYDROXYUREA
- Where
- 9 sites in Pakistan
- Volunteers
- 100 people
- Sponsor
- Pakistan Blood and Marrow Transplant (PBMT) Group
Beta-Thalassemia, Iron Overload
Amlodipine as Adjuvant Treatment to Iron Chelation for Prevention of Cardiac Iron Overload in Thalassemia Patients
This is a randomized, open label, two arms superiority trial of a representative population of patients with a primary diagnosis of transfusion dependent thalassemia with evidence of moderate cardiac iron overload, defined as an…
- Trial name
- CANALI (NCT02474420)
- Testing
- Amlodipine, Deferasirox
- Where
- Canada
- Volunteers
- 60 people
- Sponsor
- Kevin H.M. Kuo, MD, MSc, FRCPC
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
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
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
Nigella Sativa With Beta Thalassemia Major
Benefits of Nigella Sativa in Children With Beta Thalassemia Major
Evaluate the therapeutic effects health benefits, and immunological effect of Nigella sativa in children with beta thalassemia major.
- Trial name
- Nigella (NCT02816957)
- Testing
- nigella sativ
- Where
- Egypt
- Volunteers
- 80 people
- Sponsor
- Tanta University
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
Beta Thalassemia Transfusion Dependent
Evaluation of Physiological and Psychological Factors Involved in Exercise Intolerance in Patients With β-TTD
The goal of this clinical trial is to compare physical ability of patients with transfusion-dependent β-thalassemia to control subjects.
- Trial name
- THALEX (NCT07207577)
- Testing
- Maximal incremental exercise test
- Where
- France
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Metropole Savoie
Thalassemia Major
Pilot Study to Assess the Safety, PK and Iron Chelating Activity of DST-0509 (Deferasirox) in Thalassemia Patients Refractory to Chelation
This study is a multicenter, open-label, two-period crossover design that evaluates the safety, tolerability, pharmacokinetics and preliminary evidence of iron chelating activity of DST-0509 as compared to Jadenu and Exjade in…
- Trial name
- NCT03637556
- Testing
- DST-0509, Jadenu, Exjade
- Where
- 4 sites in United States, Thailand
- Volunteers
- 25 people
- Sponsor
- DisperSol Technologies, LLC
Transfusion Dependent Beta-thalassemia
Effects of Hydroxyurea and Metformin in Transfusion Dependent Beta-Thalassemia
This clinical trial aims to evaluate and compare the effects and safety of hydroxyurea and metformin in transfusion-dependent beta-thalassemia major and correlate responders/non-responders with the single nucleotide polymorphism…
- Trial name
- NCT06606886
- Testing
- Tablet Metformin (Group A), Tablet Hydroxyurea (Group B)
- Where
- Pakistan
- Volunteers
- 96 people
- Sponsor
- Bahria University
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission
Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer
This phase II trial studies how well fludarabine phosphate, cyclophosphamide, total body irradiation, and donor stem cell transplant work in treating patients with blood cancer.
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
- Trial name
- NCT03333486
- Testing
- Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Roswell Park Cancer Institute
Beta Thalassemia Major, Thalassemia Majors (Beta-Thalassemia Major)
The Effect of Laughter Yoga in Beta Thalassaemia Major Patients: a Randomised Controlled Trial
The aim of this study was to evaluate the effects of laughter yoga on Fatigue, Depression-Anxiety-Stress, Sleep quality, Haematological values and Blood transfusion number in Beta Thalassemia Major patients.
- Trial name
- NCT06980662
- Testing
- laughter yoga
- Where
- Turkey (Türkiye)
- Volunteers
- 60 people
- Sponsor
- Ataturk University
Transfusion-dependent Beta-Thalassemia
Gene-Modified Stem Cell Therapy for Subjects With Transfusion-dependent Beta-thalassemia
This is a prospective, dual-centre, single dose, Phase IIb, single arm, open label study. The proposed clinical trial involves a single infusion of autologous HSPCs genetically modified with the GLOBE lentiviral vector, using an…
- Trial name
- NCT07680803
- Testing
- FT007
- Where
- 2 sites in Italy
- Volunteers
- 9 people
- Sponsor
- Fondazione Telethon
Beta Thalassemia Major
the Potential Immunomodulatory Effects of Spirulina on Thalassemic Children
The aim of this study is to evaluate the possible immunologic effects of spirulina in children with beta thalassemia.
- Trial name
- NCT02674607
- Testing
- Spirulina
- Where
- Egypt
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Tanta University
Beta Thalassemia Major Anemia, Myelodysplastic Syndrome
A Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia or Low-risk Myelodysplastic Syndromes
The goal of this clinical trial is to learn about SP-420 ability to remove iron from organs in subjects with transfusion-dependent β-thalassemia or transfusion-dependent low-risk myelodysplastic syndrome.
- Trial name
- NCT05693909
- Testing
- SP-420
- Where
- Denmark
- Volunteers
- 90 people
- Sponsor
- Pharmacosmos A/S
Beta-Thalassemia, Thalassemia
A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in participans with transfusion-dependent β-thalassemia (TDT).
- Conditions
- Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases
- Trial name
- NCT03655678
- Testing
- CTX001
- Where
- 14 sites in United States, Canada and 3 more
- Volunteers
- 59 people
- Sponsor
- Vertex Pharmaceuticals Incorporated