Clinical trials: Transfusion Dependent Beta-Thalassaemia
Non-transfusion Dependent Thalassemia
Efficacy and Safety of Deferasirox in Non-transfusion Dependent Thalassemia Patients With Iron Overload and a One Year Open-label Extension Study
CICL670A2209: This study will evaluate the safety and efficacy of deferasirox in non-transfusion dependent thalassemia patients with iron overload.
- Trial name
- THALASSA (NCT00873041)
- Testing
- deferasirox
- Where
- 21 sites in United States, Greece and 7 more
- Volunteers
- 166 people
- Sponsor
- Novartis Pharmaceuticals
Transfusional Iron Overload, β-thalassemia Major
A 4-year Extension Study to Core 1-year Study of Iron Chelation Therapy With Deferasirox in β-thalassemia Major Pediatric Patients With Transfusional Iron Overload.
In this 4-year extension study the safety, efficacy and and pharmacokinetics of deferasirox in regularly transfused pediatric patients with β-thalassemia major was assessed.
- Conditions
- Transfusional Iron Overload, β-thalassemia Major, Pediatric Rare Anemia
- Trial name
- NCT00390858
- Testing
- Deferasirox
- Where
- 4 sites in France, Italy
- Volunteers
- 40 people
- Sponsor
- Novartis Pharmaceuticals
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.
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
Transfusion Dependent Beta Thalassemia, Hemoglobinopathies
EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)
The purpose of this study is to evaluate the safety, tolerability, and efficacy of treatment with EDIT-301 in adult participants with Transfusion Dependent beta Thalassemia
- Conditions
- Transfusion Dependent Beta Thalassemia, Hemoglobinopathies, Thalassemia Major, Thalassemia Intermedia
- Trial name
- NCT05444894
- Testing
- EDIT-301
- Where
- 8 sites in United States, Canada
- Volunteers
- 9 people
- Sponsor
- Editas Medicine, Inc.
Thalassemia Major, Chronic Blood Transfusion Related Iron Overload
MR Imaging of Diffuse Myocardial Fibrosis in Transfusion-Dependent Anemias
Cardiac failure is the major cause of death in patients with thalassemia and chronic blood transfusion-related iron overload. The treatment of thalassemia has been revolutionized over the past decade with the implementation of…
- Trial name
- MAFIO (NCT02090699)
- Where
- Canada
- Volunteers
- 42 people, healthy volunteers welcome
- Sponsor
- University Health Network, Toronto
Beta-Thalassemia, Thalassemia
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
This is a single-dose, open-label study in pediatric participants with TDT. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) (CTX001).
- Conditions
- Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases
- Trial name
- NCT05356195
- Testing
- CTX001
- Where
- 6 sites in United States, Canada and 3 more
- Volunteers
- 16 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Thalassemia
A Study to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta (β)-Thalassemia
This is a Phase 2, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept (ACE-536) versus placebo in adults with non-transfusion dependent beta (β)-thalassemia.
- Trial name
- BEYOND (NCT03342404)
- Testing
- Luspatercept, Best Supportive Care (BSC)
- Where
- 16 sites in United States, Greece and 4 more
- Volunteers
- 145 people
- Sponsor
- Celgene
Sickle Cell Anemia, Beta-thalassemia Major
T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias
The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent…
- Conditions
- Sickle Cell Anemia, Beta-thalassemia Major, Diamond-blackfan Anemia
- Trial name
- NCT03653338
- Testing
- CD3/CD19 depleted leukocytes, CD45RA depleted leukocytes, Hydroxyurea
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Paul Szabolcs
Beta Thalassemia Transfusion Dependent
Gene Therapy for Patients With Transfusion-Dependent β-Thalassemia
Thalassemia is a group of recessively inherited hemoglobin disorders characterized by reduced or no production of hemoglobin and chronic anemia of varying severity.
- Trial name
- NCT07721480
- Testing
- HGI-001 Injection
- Volunteers
- 6 people
- Sponsor
- Prince of Wales Hospital, Shatin, Hong Kong
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
Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
The primary objective of this study was to compare the effect of mitapivat versus placebo on transfusion burden in participants with α- or β-transfusion-dependent thalassemia.
- Trial name
- ENERGIZE-T (NCT04770779)
- Testing
- Mitapivat
- Where
- 75 sites in United States, Brazil and 17 more
- Volunteers
- 258 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Beta-Thalassemia
To Document the Burden of Illness on the Quality of Life and the Impact on Healthcare Utilization in (Beta) β-thalassemia Subjects Who Are Transfusion Dependent (TD) and Non-transfusion Dependent (NTD) Receiving Standard of Care
This is a multi-site, prospective, observational study implemented in β-thalassemia treatment centers from 5 countries (Italy, Turkey, Greece, Lebanon, and Thailand).
- Trial name
- NCT02626689
- Testing
- Quality of Life (QOL) questionnaires, Healthcare Resource Utilization
- Where
- 5 sites in Greece, Italy and 3 more
- Volunteers
- 100 people
- Sponsor
- Celgene
Hereditary Anemias
Utility of Fibroscan in Estimating Hepatic Iron Concentration
In patients with hereditary anemias (e.g. thalassemias), defective red blood cells are produced due to an error in the genes, or DNA, that provide the instructions for their synthesis.
- Trial name
- NCT02067130
- Testing
- Fibroscan
- Where
- Canada
- Volunteers
- 30 people
- Sponsor
- University of British Columbia
Chronic Iron Overload
Pharmacokinetic Study of Deferiprone in Paediatric Patients
Deferiprone (DFP) is the most extensively studied oral iron chelator to date. It has been authorised in Europe in 1999 for the treatment of iron overload in patients with beta-thalassaemia major when DFO is contraindicated or…
- Trial name
- DEEP-1 (NCT01740713)
- Testing
- Deferiprone, dose level 1, Deferiprone, dose level 2, Deferiprone, dose level 3
- Where
- 7 sites in Cyprus, Egypt and 1 more
- Volunteers
- 23 people
- Sponsor
- Consorzio per Valutazioni Biologiche e Farmacologiche
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
Beta-Thalassemia
A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia
This is a single-arm, multi-site, single-dose, Phase 3 study in approximately 18 participants less than or equal to (<=) 50 years of age with transfusion-dependent β-thalassemia (TDT), who have a β0/β0, β0/IVS-I-110, or…
- Trial name
- NCT03207009
- Testing
- LentiGlobin BB305 Drug Product
- Where
- 9 sites in United States, France and 4 more
- Volunteers
- 19 people
- Sponsor
- Genetix Biotherapeutics Inc.
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
Chronic Iron Overload
Efficacy/Safety Study of Deferiprone Compared to Deferasirox in Paediatric Patients
Multicentre, randomised, open label, non-inferiority active-controlled trial to evaluate efficacy and safety of a 12-months treatment with deferiprone (DFP) at dose of 75-100 mg/kg/day versus deferasirox (DFX) at dose of 20-40…
- Trial name
- NCT01825512
- Testing
- Deferiprone, Deferasirox
- Where
- 22 sites in Albania, Cyprus and 5 more
- Volunteers
- 435 people
- Sponsor
- Consorzio per Valutazioni Biologiche e Farmacologiche
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