Clinical trials: Thalassemia, Beta
Sickle Cell Disease, Thalassemia, Beta
A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)
Hematopoietic Cell Transplantation/HCT involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in participants' bone marrow.
- Conditions
- Sickle Cell Disease, Thalassemia, Beta, Thalassemia
- Trial name
- NCT05736419
- Testing
- Fludarabine, Cyclophosphamide, Tacrolimus
- Where
- 6 sites in United States
- Volunteers
- 24 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
β-thalassemia Major
A Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in β-Thalassemia Major Participants
This is a non-randomized, open label, multi-site, single-dose, phase 1/2 study in up to 18 participants (including at least 3 adolescents between 12 and 17 years of age, inclusive) with β-thalassemia major.
- Trial name
- NCT01745120
- Testing
- LentiGlobin BB305 Drug Product
- Where
- 6 sites in United States, Australia and 1 more
- Volunteers
- 19 people
- Sponsor
- Genetix Biotherapeutics Inc.
Beta Thalassemia Transfusion Dependent
Effect of Deferasirox on Patients With Cardiac MRI T2* < 20 Msec
This study will assess the safety and efficacy of deferasirox in patients with cardiac MRI T2* < 20 msec.
- Trial name
- NCT00879242
- Testing
- Deferasirox
- Where
- 2 sites in Italy
- Volunteers
- 20 people
- Sponsor
- Novartis Pharmaceuticals
Beta-Thalassemia, Non-transfusion-dependent Thalassemia
Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD) and Preliminary Efficacy of VIT-2763 in β-thalassaemia
This is a randomised, double-blind, placebo-controlled parallel group trial to investigate the safety, tolerability and efficacy of multiple doses of VIT-2763 versus placebo in participants with non-transfusion-dependent…
- Trial name
- VITHAL (NCT04364269)
- Testing
- VIT-2763 once a day (QD), VIT-2763 twice a day (BID)
- Where
- 16 sites in Greece, Israel and 3 more
- Volunteers
- 35 people
- Sponsor
- Vifor (International) Inc.
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, 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
Preterm Birth, Oxygen Toxicity
Fetal Haemoglobin and Cerebral and Peripheral Oxygenation.
The aim of this study is to investigate the relationship between cerebral and peripheral oxygenation and oxygen extraction, as measured by NIRS (near-infrared spectroscopy ), and the FHbF (fraction of fetal hemoglobin) and…
- Conditions
- Preterm Birth, Oxygen Toxicity, Fetal Hemoglobin
- Trial name
- HbFIN (NCT04802629)
- Testing
- Near-infrared spectroscopy
- Where
- Austria
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Medical University of Graz
Transfusion Dependent Beta Thalassemia
A Pilot Study on the Safety and Efficacy of YOLT-204 for Transfusion-Dependent Beta-Thalassemia
This study is a single-arm, open-label, dose-escalation trial, planning to enroll 3-9 patients with transfusion-dependent β-thalassemia, aimed at assessing the safety and tolerability of a single-dose of YOLT-204 in patients with…
- Trial name
- NCT06678165
- Testing
- YOLT-204
- Where
- China
- Volunteers
- 3 people
- Sponsor
- First Affiliated Hospital of Guangxi Medical University
Beta Thalassemia Major
Phase 2a Pilot Study of NBMI Treatment in Patients With Beta Thalassemia Major, Requiring Iron Chelation
A pilot study to explore safety and efficacy of NBMI treatment in patients with Beta Thalassemia Major requiring iron chelation Investigational product: NBMI (N1,N3-bis(2-mercaptoethyl) isophthalamide), INN: Emeramide Indication…
- Trial name
- EMERA007 (NCT04092205)
- Testing
- Emeramide
- Where
- Albania
- Volunteers
- 13 people
- Sponsor
- EmeraMed
EKLF Mutations in Beta Thalassemia Patients
Detection of Krupple Like Factor -1(KLF1/ EKLF) DNA Mutations in Beta Thalassemia Patients
Detection of KLF1 gene mutations in patients with beta thalassemia considering the alpha and beta molecular status of these patients.
- Trial name
- NCT06433570
- Testing
- multiplex PCR
- Volunteers
- 100 people
- Sponsor
- Assiut University
Thalassemia Major
Amlodipine Use in the Prevention and Treatment of Iron Overload in Patients With Thalassemia Major
This study aims to investigate the use of amlodipine, a drug that blocks the uptake of calcium into cells, in the prevention and treatment of iron overload in patients with thalassemia major.
- Trial name
- AnloThal (NCT01125254)
- Testing
- Amlodipine
- Where
- Brazil
- Volunteers
- 10 people
- Sponsor
- University of Campinas, Brazil
β-Thalassemia Intermedia
Combination Therapy of Hydroxyurea With L-Carnitine and Magnesium Chloride in Thalassemia Intermedia
The purpose of this study is determination of the efficacy of combination of hydroxyurea with L-carnitine or magnesium in improving hematologic parameters and cardiac status in patients with β-thalassemia intermedia in comparison…
- Trial name
- NCT00809042
- Testing
- hydroxyurea, L-carnitine and hydroxyurea, hydroxyurea and magnesium chloride
- Where
- Iran
- Volunteers
- 120 people
- Sponsor
- Shiraz University of Medical Sciences
Beta-Thalassemia
Gene Therapy for Transfusion Dependent Beta-thalassemia
This is a phase I/II study evaluating safety and efficacy of autologous hematopoietic stem cells genetically modified with GLOBE lentiviral vector encoding for the human beta-globin gene for the treatment of patients affected by…
- Trial name
- TIGET-BTHAL (NCT02453477)
- Testing
- Autologous hematopoietic stem cells genetically modified with GLOBE lentiviral vector encoding for the human beta-globin gene
- Where
- Italy
- Volunteers
- 10 people
- Sponsor
- IRCCS San Raffaele
Thalassemia
Safety and Efficacy of Hemoglobin F Inducers in Patients With Beta Thalassemia
The aim of this study is to determine the safety and therapeutic effect of HbF inducers (combination therapy: thalidomide and hydroxyurea) on beta thalassemia patients.
- Trial name
- NCT07673302
- Testing
- Thalidomide, Hydroxy Urea
- Where
- Pakistan
- Volunteers
- 240 people
- Sponsor
- Riphah International University
Myelodysplastic Syndromes, Beta-Thalassemia
Evaluating the Efficacy of Deferasirox in Transfusion Dependent Chronic Anaemias (Myelodysplastic Syndrome, Beta-thalassaemia Patients) With Chronic Iron Overload
This study will evaluate the safety and efficacy of deferasirox in transfusion dependent Myelodysplastic Syndrome, Beta-thalassaemia major patients with chronic iron overload
- Trial name
- NCT00564941
- Testing
- deferasirox
- Where
- 4 sites in Hungary
- Volunteers
- 309 people
- Sponsor
- Novartis Pharmaceuticals
Beta-Thalassemia
Vitamin E Supplementation for Children With Transfusion Dependent Beta Thalassemia on Different Iron Chelation Regimen
repeated transfusions are the mainstay of disease management in most patients with transfusion dependent beta thalassemia.iron overload predispose to oxidative stress and tissue injury.
- Trial name
- NCT06509581
- Testing
- vitamin E
- Where
- Egypt
- Volunteers
- 180 people
- Sponsor
- Ain Shams University
Hemoglobinopathies
Interferon and Ribavirin Treatment in Patients With Hemoglobinopathies
Worldwide, several studies report that 4.4% to 85.4% of thalassemia patients were positive for anti hepatitis C antibodies. Recently, three different studies reported the efficacy and the safety of combination therapy with…
- Trial name
- IFNRIBAHE (NCT00887081)
- Testing
- PEG-IFN alpha2a or PEG-IFN alpha2b and Ribavirin
- Where
- Italy
- Volunteers
- 150 people
- Sponsor
- Azienda Ospedaliera V. Cervello
Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
The primary purpose of this study was to compare the effect of mitapivat versus placebo on hemolytic anemia in participants with alpha- or beta-non-transfusion dependent thalassemia (NTDT).
- Trial name
- NCT04770753
- Testing
- Mitapivat
- Where
- 68 sites in United States, Brazil and 16 more
- Volunteers
- 194 people
- Sponsor
- Agios Pharmaceuticals, Inc.
β-thalassemia
A Real-World Study to Evaluate Luspatercept in Adults With Transfusion-Dependent Beta-Thalassemia in the Middle East
The purpose of this study is to evaluate luspatercept treatment in adults with transfusion-dependent beta-Thalassemia in the Middle East
- Trial name
- NCT07215975
- Testing
- Luspatercept
- Where
- 13 sites in Kuwait, Oman and 2 more
- Volunteers
- 200 people
- Sponsor
- Bristol-Myers Squibb
Endocrine Disease, Fertility Decreased Male
Sperm DNA Damage in β-thalassemia Major: Is There a Role for Antioxidants?
Accumulation of iron in patients with beta thalassemia major causes free radical formation which leads to damage of biological membranes. Sperm DNA damage may result from these generated antioxidants.
- Conditions
- Endocrine Disease, Fertility Decreased Male, DNA Damage
- Trial name
- NCT03014882
- Testing
- L-Carnitine and N-acetyl cysteine
- Where
- 3 sites in Egypt
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Ain Shams University