Clinical trials: Beta-thalassemia
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.
Sickle Cell Disease
Single-dose Pharmacokinetics of Pociredir in Participants With Sickle Cell Disease
This clinical trial is a study to evaluate the pharmacokinetics of the tablet formulation Pociredir in fasted and fed state participants with Sickle Cell Disease (SCD).
- Trial name
- NCT07431398
- Testing
- Pociredir
- Where
- 6 sites in United States
- Volunteers
- 13 people
- Sponsor
- Fulcrum Therapeutics
Beta-Thalassemia, Thalassemia
A Long-term Follow-up Study in Participants Who Received CTX001
This is a multi-site, open- label rollover study to evaluate the long-term safety and efficacy of CTX001 in pediatric and adult participants who received CTX001 in parent studies 111 (NCT03655678) 141 (NCT05356195) or 161…
- Conditions
- Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases
- Trial name
- NCT04208529
- Testing
- CTX001
- Where
- 20 sites in United States, Belgium and 4 more
- Volunteers
- 160 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Blood and Lymphatic Diseases
Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.
Primary Objectives: Long-term safety of BIVV003 in participants with severe sickle cell disease (SCD) and ST- 400 in participants with transfusion-dependent beta-thalassemia (TDT) Secondary Objectives: Long-term efficacy of the…
- Trial name
- NCT05145062
- Testing
- BIVV003, ST-400
- Where
- 7 sites in United States
- Volunteers
- 8 people
- Sponsor
- Sangamo Therapeutics
Non-Transfusion Dependent Beta-Thalassemia (NTDT)
A Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body
This study is researching an experimental drug called REGN7999 (called "study drug"). The study is focused on patients with non-transfusion dependent beta-thalassemia.
- Trial name
- FERVENT-1 (NCT06421636)
- Testing
- REGN7999
- Where
- 26 sites in United States, Georgia and 7 more
- Volunteers
- 19 people
- Sponsor
- Regeneron Pharmaceuticals
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
Safety & Efficacy of ICL670 vs. Deferoxamine in Beta-thalassemia Patients With Iron Overload Due to Blood Transfusions
The purpose of this study is to deterimine if the new orally active iron chelator, ICL670, is as effective and as safe as deferoxamine in preventing accumulation of iron in the body while a patient is undergoing repeated blood…
- Trial name
- NCT00061750
- Testing
- ICL670, deferoxamine
- Where
- 7 sites in United States
- Volunteers
- 595 people
- Sponsor
- Novartis Pharmaceuticals
Beta-Thalassemia
A Clinical Study Evaluating the Safety and Efficacy of CS-101 in Treating Subjects 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
- NCT06024876
- Testing
- CS-101
- Where
- China
- Volunteers
- 5 people
- Sponsor
- CorrectSequence Therapeutics Co., Ltd
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
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.
Hematologic Diseases
A Study Evaluating the Safety and Efficacy of the BD211 Drug Product in β-Thalassemia Major Participants
This is a Phase 1,open label,safety,and efficacy study in subjects with non-β0/β0 TDT β-thalassemia Major by transplanting BD211 drug product which is for autologous use only,via a single IV administration.
- Trial name
- NCT05015920
- Testing
- BD211 Drug Product
- Where
- China
- Volunteers
- 2 people
- Sponsor
- Shanghai BDgene Co., Ltd.
Thalassemia Major
Safety and Efficacy Evaluation of γ-globin Reactivated Autologous Hematopoietic Stem Cells
This is a single arm, open label, single-dose, phase 1/2 study in up to 5 participants with β-thalassemia major.The study will evaluate the safety and efficacy of the treatment with γ-globin reactivated autologous hematopoietic…
- Trial name
- NCT05442346
- Testing
- γ-globin reactivated autologous hematopoietic stem cells
- Where
- China
- Volunteers
- 5 people
- Sponsor
- Bioray Laboratories
Non-transfusion-dependent Thalassemia, Low Risk Myelodysplastic Syndrome
A Study Investigate the Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Response of SLN124 in Adults With Alpha/Beta-thalassaemia and Very Low- and Low-risk Myelodysplastic Syndrome
This study will investigate the safety and tolerability of SLN124 in patients with Thalassaemia or patients with Very Low- and Low-risk Myelodysplastic Syndrome (MDS) after single ascending s.c.
- Conditions
- Non-transfusion-dependent Thalassemia, Low Risk Myelodysplastic Syndrome, Very-Low Risk Myelodysplastic Syndrome
- Trial name
- NCT04718844
- Testing
- SLN124
- Where
- 20 sites in Germany, Israel and 5 more
- Volunteers
- 44 people
- Sponsor
- Silence Therapeutics plc
β-thalassemia
Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients
This is an open label study to evaluate the safety and efficacy of β-globin Restored Autologous Hematopoietic Stem Cells in ß-Thalassemia Major Patients
- Trial name
- NCT05745532
- Testing
- β-globin restored autologous hematopoietic stem cells
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Shenzhen Hemogen
Myelodysplastic Syndromes, Beta-thalassemia
Study to Evaluate Additional Risk Minimisation Measures (aRMMs) for REBLOZYL Among Healthcare Professionals (HCPs)
This is a non-interventional post-authorization safety study (PASS) employing a cross sectional design to evaluate the effectiveness of the additional risk minimization measures (aRMMs) for REBLOZYL.
- Trial name
- NCT04973280
- Where
- 4 sites in Austria, Belgium and 2 more
- Volunteers
- 187 people, healthy volunteers welcome
- Sponsor
- Celgene
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
Non-transfusion-dependent Thalassemia, Low Risk Myelodysplastic Syndrome
Study in Beta-thalassaemia or Myelodysplastic Syndrome Patients to Investigate the Safety and Tolerability of SLN124
The primary purpose of this study is to determine the safety and tolerability of SLN124 for the treatment of non-transfusion-dependent (NTD) β-thalassaemia and low risk myelodysplastic syndrome.
- Trial name
- NCT04176653
- Where
- 5 sites in Bulgaria, United Kingdom
- Sponsor
- Silence Therapeutics plc
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.
All Diagnosed Health Conditions, ADD/ADHD
At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions
We are the missing link in clinical trials, connecting patients and researchers seamlessly and conveniently using a mobile health platform to advance medical research.
- Conditions
- All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis
- Trial name
- NCT01931644
- Where
- United States
- Volunteers
- 17,667 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
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