Clinical trials: β-thalassemia Intermedia
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
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.
Thalassemia Majors (Beta-Thalassemia Major), Haplo-identical Donors
Clinical Study of Super Transplantation in the Treatment of Severe β-thalassemia
This is a single-arm, open label, phase 1 study in subjects with beta-thalassemia. This study will evaluate the safety and efficacy of preconditioning-free super-transplantation on beta-thalassemia pediatric patients.
- Trial name
- NCT06734520
- Testing
- super-transplantation in 3 severe β -thalassemia pediatric patients.
- Where
- 2 sites in China
- Volunteers
- 3 people
- Sponsor
- Guangzhou Women and Children's Medical Center
Beta-thalassemia Major Complicated With Diabetes
Zinc Supplementation in Patients With β-Thalassemia Major Complicated With Diabetes Mellitus
Beta-thalassemia represents a group of recessive inherited hemoglobin disorders characterized by reduced synthesis of β-globin chain.
- Trial name
- NCT03851055
- Testing
- Zinc
- Where
- Egypt
- Volunteers
- 80 people
- Sponsor
- Ain Shams University
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.
Beta-Thalassemia
A Study of EPEG in Beta Thalassemia Patients
An open-label study in which 6 patients will receive once-weekly subcutaneous injections of EPEG for 4 weeks. Final visit will occur 60 days after study entry
- Trial name
- NCT02950857
- Testing
- EPEG
- Where
- Thailand
- Volunteers
- 6 people
- Sponsor
- Prolong Pharmaceuticals
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
A Long-term Follow-up Study in Participants Who Received CS-101
This is a study to evaluate the long-term safety and efficacy of CS-101 in participants who received CS-101 in study CS -101-03 (NCT06065189)
- Trial name
- NCT06479616
- Testing
- CS-101
- Where
- China
- Volunteers
- 5 people
- Sponsor
- Children's Hospital of Fudan University
Beta-Thalassemia
A Follow-up Study to Evaluate the Long-term Safety and Efficacy in Participants Who Received CS-101 Injection
This is a study to evaluate the long-term safety and efficacy of CS-101 in participants who received CS-101 in study CS -101-01 (NCT06291961),No investigational drug product will be administered in the study.
- Trial name
- NCT06717932
- Where
- China
- Volunteers
- 8 people
- Sponsor
- CorrectSequence Therapeutics Co., Ltd
Thalassemia, Beta
Thalidomide and Hydroxyurea Combination in β-Thalassemia Patients
Objectives Primary objective: To determine the efficacy and safety of the combination therapy of Hydroxyurea and thalidomide in beta-thalassemia patients.
- Trial name
- NCT06153784
- Testing
- Hydroxyurea and Thalidomide
- Where
- Pakistan
- Volunteers
- 603 people
- Sponsor
- Children's Hospital Karachi
Beta-Thalassemia, Thalassemia
Combination Therapy With Danazol, Prednisolone, and Thalidomide for Thalidomide-Refractory Transfusion-Dependent β-Thalassemia
This study will compare two treatments for children with beta-thalassemia who need regular blood transfusions and have not responded well to thalidomide. One group will continue thalidomide alone.
- Trial name
- NCT07884773
- Testing
- Thalidomide, Danazol, Prednisolone
- Where
- Pakistan
- Volunteers
- 172 people
- Sponsor
- Khyber Medical University Peshawar
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
β-Thalassemia Major
A Real-World Study of β-Thalassemia Major Treatment With Luspatercept in Taiwan
This real-world study will assess the efficacy and safety of luspatercept treatment for β-thalassemia major in Taiwan as well as the impact on quality of life.
- Trial name
- NCT06596642
- Testing
- Luspatercept
- Where
- 5 sites in Taiwan
- Volunteers
- 61 people
- Sponsor
- Bristol-Myers Squibb
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.
Transfusion Dependent Beta-Thalassaemia
Safety and Efficacy Evaluation of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia
This is an open label, multi-center study to evaluate the safety and Efficacy of ET-01 Transplantation in subjects with Transfusion Dependent β-Thalassaemia.
- Trial name
- NCT04390971
- Testing
- ET-01
- Where
- 3 sites in China
- Volunteers
- 3 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Beta-Thalassemia
CS-101 in Patients With β-thalassemia
The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 in treating β-thalassemia.
- Trial name
- NCT06328764
- Testing
- CS-101
- Where
- China
- Volunteers
- 10 people
- Sponsor
- CorrectSequence Therapeutics Co., Ltd
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
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
Myelodysplastic Syndrome, Beta Thalassemia
A Post-Marketing Surveillance Study to Assess Safety of Luspatercept in Korean Patients With Myelodysplastic Syndrome or β-thalassemia
The purpose of this observational study is to assess the real-world safety of luspatercept in Korean participants with myelodysplastic syndrome (MDS) or beta thalassemia.
- Trial name
- NCT06073860
- Testing
- Luspatercept
- Where
- 2 sites in South Korea
- Volunteers
- 104 people
- Sponsor
- Bristol-Myers Squibb
Beta-thalassemia
A Study to Evaluate Luspatercept (ACE-536) in Chinese Participants Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia.
The purpose of this study is to evaluate the efficacy, safety and pharmacokinetics of luspatercept plus best supportive care (BSC) versus placebo plus BSC in participants who require regular red blood cell transfusions due to…
- Trial name
- NCT05567458
- Testing
- Luspatercept
- Where
- 10 sites in China
- Volunteers
- 94 people
- Sponsor
- Bristol-Myers Squibb