Clinical trials: β-thalassemia
Sickle Cell Disease, Beta Thalassemia Intermedia
Pumilio1 (PUM1) Expression, Sickle Cell Anemia, β-thalassemia Intermedia
1. To study the expression pattern of PUM1 gene in patients with sickle cell anemia and β-thalassemia intermedia. 2. To detect PUM1 protein levels in sickle cell anemia and β-thalassemia intermedia patients. 3.
- Trial name
- NCT05883254
- Testing
- RNA Binding Protein Pumilio1 (PUM1) Expression by reverse transcriptase quantatative PCR
- Volunteers
- 80 people
- Sponsor
- Assiut University
Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
The primary objective of this study is to compare the effect of mitapivat versus placebo on transfusion burden in pediatric participants with α- or β-transfusion-dependent thalassemia.
- Trial name
- ENERGIZEKids-T (NCT07506863)
- Testing
- Mitapivat
- Where
- 4 sites in United States
- Volunteers
- 54 people
- Sponsor
- Agios Pharmaceuticals, Inc.
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 -101-06 (NCT06024876),No investigational drug product will be administered in the study.
- Trial name
- NCT06685536
- Testing
- Safety and efficacy assessments
- Where
- China
- Volunteers
- 5 people
- Sponsor
- CorrectSequence Therapeutics Co., Ltd
Beta-Thalassemia
A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel
The main aim of this study is to collect real-world longitudinal data on participants with β-thalassemia treated with betibeglogene autotemcel (beti-cel) in the post marketing setting.
- Trial name
- NCT06271512
- Testing
- No Intervention
- Where
- 6 sites in United States
- Volunteers
- 150 people
- Sponsor
- Genetix Biotherapeutics Inc.
Beta-Thalassemia
Base-edited Autologous Hematopoietic Stem Cell Transplantation in Treating Patients With β-thalassemia Major
The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of base-edited autologous hematopoietic stem cell transplantation(CS-101) in treating patients with β-thalassemia major.
- Trial name
- NCT06065189
- Testing
- CS-101 injection
- Where
- China
- Volunteers
- 2 people
- Sponsor
- Children's Hospital of Fudan University
Transfusion-dependent Beta-Thalassemia
Deciphering Effects of Thalidomide on Red Blood Cells in Transfusion Dependents Beta Thalassemia Patients
The goal of this Non-Randomized Clinical Trial is to determine the effects of thalidomide on red blood cells in transfusion dependent beta thalassemia patients.
- Trial name
- NCT06146478
- Testing
- Thalidomide
- Where
- Pakistan
- Volunteers
- 200 people
- Sponsor
- Blood Care Clinic
Thalassemia, Beta, Thalassemia Major
Safety and Efficacy Evaluation of Autologous CRISPR-Cas12b Edited Hematopoietic Stem Cells
This is a single-arm, open, single-injection exploratory clinical study with two transfusion-dependent β thalassemia (β-TDT) participants planned to enroll.
- Trial name
- NCT06041620
- Testing
- VGB-Ex01
- Where
- China
- Volunteers
- 2 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
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
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.
β-Thalassemia
Extension Study to Evaluate the Safety and Efficacy of Luspatercept in Participants With β-Thalassemia Previously Enrolled in A536-04 (A536-06/MK-6143-004)
Study A536-06 (MK-6143-004) is an open-label extension study for participants previously enrolled in study A536-04 (NCT01749540), to evaluate the long-term safety and tolerability of luspatercept in adult participants with…
- Trial name
- NCT02268409
- Testing
- luspatercept
- Where
- 7 sites in Greece, Italy
- Volunteers
- 51 people
- Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia
The primary objective of this study is to compare the effect of mitapivat versus placebo on anemia in pediatric participants with alpha- or beta-non-transfusion-dependent thalassemia.
- Trial name
- ENERGIZEKids (NCT07517133)
- Testing
- Mitapivat
- Where
- 5 sites in United States
- Volunteers
- 45 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Thalassemia
The Change of Coagulation Markers in Children With β-thalassemia Disease After Stem Cell Transplantation
Hypercoagulable state is well recognized in patients with β-thalassemia. Evidences of hypercoagulability include abnormal expression of phosphatidylserine on red blood cell (rbc) surface and consequent increased platelet…
- Trial name
- NCT00789516
- Where
- Thailand
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Mahidol University
Erythrocyte Transfusion, Beta-Thalassemia
An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemia
This is a Phase 3, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept (ACE-536) plus Best supportive care (BSC) versus placebo plus BSC in adults who require…
- Trial name
- BELIEVE (NCT02604433)
- Testing
- Luspatercept
- Where
- 76 sites in United States, Australia and 13 more
- Volunteers
- 336 people
- Sponsor
- Celgene
Transfusional Iron Overload in β-thalassemia
An Extension Study of Iron Chelation Therapy With Deferasirox (ICL670) in β-thalassemia Patients With Transfusional Iron Overload
A 1-year randomized Phase III core trial (NCT00061750) using deferoxamine as the comparator was conducted to investigate the efficacy of deferasirox in regularly transfused patients with β-thalassemia 2 years of age and older.
- Trial name
- NCT00171210
- Testing
- Deferasirox
- Where
- 49 sites in United States, Argentina and 10 more
- Volunteers
- 506 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Anemia, Sickle Cell β+ or β0 Thalassemia
Sickle Cell Anemia - A Comparative Study Between Three Ethnical Communities, a Multicenter Study
The purpose of this study is to take advance of the presence of two different cohorts of SCA patients in one country, the first group included SCA patients from Bedouin Arab origin that lives in Israel for more than one century…
- Trial name
- NCT01905787
- Where
- 4 sites in United States, Israel
- Volunteers
- 300 people
- Sponsor
- HaEmek Medical Center, Israel
β-Thalassemia, Pulmonary Function
Correlation Between Pulmonary Functions and Physical Fitness in Children With β-thalassemia
The aims of this study are: 1. To determine the correlation between pulmonary functions and physical fitness in children with β-thalassemia. 2.
- Conditions
- β-Thalassemia, Pulmonary Function, Physical Fitness
- Trial name
- NCT05494333
- Testing
- no intervention
- Where
- Egypt
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Cairo University
Transfusion-dependent Beta-Thalassemia
Β-Thalassemia Treatment with KL003 Cell Injection
This is a non-randomized, open label, single-dose study in up to 41 participants with β-thalassemia major. The goal of this clinical trial is to evaluate the safety and efficacy of KL003 cell injection in subjects with…
- Trial name
- NCT06280378
- Testing
- KL003 Cell Injection Drug Product
- Where
- 2 sites in China
- Volunteers
- 41 people
- Sponsor
- Kanglin Biotechnology (Hangzhou) Co., Ltd.
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
Anemia, Hemosiderosis
Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Rare Chronic Anemia Patients
A 1-year randomized Phase II core trial was conducted to investigate the efficacy of deferasirox in regularly transfused patients with β-thalassemia and other rare chronic anemia 2 years of age and older.
- Trial name
- NCT00303329
- Testing
- Deferasirox
- Where
- 28 sites in United States, Belgium and 5 more
- Volunteers
- 184 people
- Sponsor
- Novartis Pharmaceuticals
Beta-Thalassemia Major
A Safety and Efficacy Study Evaluating CS-101 in Subjects With β-Thalassemia Major
The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 in treating patients with β-thalassemia major anemia.
- Trial name
- NCT06291961
- Testing
- CS-101 injection
- Where
- 3 sites in China
- Volunteers
- 9 people
- Sponsor
- CorrectSequence Therapeutics Co., Ltd