Clinical trials: Thalassaemia
Beta-thalassemia, Myelodysplastic Syndromes
Study of Deferasirox in Iron Overload From Beta-thalassemia Unable to be Treated With Deferoxamine or Chronic Anemias
The purpose of this study is to determine the effects of the oral iron chelator Deferasirox on liver iron content after one year of treatment in patients with iron overload from repeated blood transfusions.
- Conditions
- Beta-thalassemia, Myelodysplastic Syndromes, Fanconi Syndrome, Anemia, Diamond-Blackfan
- Trial name
- NCT00061763
- Testing
- Deferasirox
- Where
- 6 sites in United States
- Volunteers
- 175 people
- Sponsor
- Novartis Pharmaceuticals
Beta Thalassemia Intermedia
Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx)
The purpose was to evaluate the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics of sapablursen administered subcutaneously to participants with non-transfusion dependent β-Thalassemia Intermedia.
- Trial name
- NCT04059406
- Testing
- sapablursen
- Where
- 19 sites in Australia, Greece and 3 more
- Volunteers
- 29 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
β-thalassemia Intermedia
Apotransferrin in Patients With β-thalassemia
The aim of the trial is to study the effect of apotransferrin administration in patients suffering from β-thalassemia intermedia in order to restore the erythropoiesis as reflected by enhanced haemoglobin levels or reduced…
- Trial name
- NCT03993613
- Testing
- human apotransferrin
- Where
- Netherlands
- Volunteers
- 10 people
- Sponsor
- Prothya Biosolutions
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
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
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
Beta-Thalassemia Major, Sickle Cell Disease
A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease
This is a Phase 1/2, open label, safety, and efficacy study of the administration of LentiGlobin BB305 Drug Product to participants with either transfusion dependent beta-thalassemia (TDT) or sickle cell disease (SCD).
- Trial name
- NCT02151526
- Testing
- LentiGlobin BB305 Drug Product
- Where
- France
- Volunteers
- 7 people
- Sponsor
- Genetix Biotherapeutics Inc.
Sickle Cell Disease
Open-Label Extension Study to Pioneer Study 6058-SCD-101
This is an open-label study to evaluate the safety and tolerability of long-term treatment with pociredir without a comparator in participants with SCD who have previously been treated and shown benefit with pociredir in feeder…
- Trial name
- NCT07401823
- Testing
- Pociredir
- Where
- 8 sites in United States
- Volunteers
- 1 people
- Sponsor
- Fulcrum Therapeutics
Sickle Cell Disease
A Study of IMR-687 in Adult Participants With Sickle Cell Anemia (Homozygous HbSS or Sickle-β0 Thalassemia)
Study of IMR-687 in adult participants with sickle cell anemia (SCA) (homozygous HbSS or sickle-β0 thalassemia).
- Trial name
- NCT03401112
- Testing
- IMR-687
- Where
- 13 sites in United States, United Kingdom
- Volunteers
- 100 people
- Sponsor
- Cardurion Pharmaceuticals, Inc.
Anemia
A Study to Determine the Efficacy and Safety of Luspatercept in Adult Participants and to Evaluate the Safety and Pharmacokinetics in and Adolescent Participants With Alpha (α)-Thalassemia
The purpose of the study is to evaluate the efficacy and safety of luspatercept plus best supportive care (BSC) vs placebo plus BSC on anemia in adult participants with α-thalassemia hemoglobin H (HbH) disease and determine the…
- Trial name
- NCT05664737
- Testing
- Luspatercept
- Where
- 31 sites in China, Greece and 8 more
- Volunteers
- 189 people
- Sponsor
- Bristol-Myers Squibb
Sickle Cell Disease, Sickle-Beta Zero Thalassemia
Dose-Escalation Study of SCD-101 in Sickle Cell Disease
The purpose of this study is to determine the safety and clinical effects of SCD-101 when given to adults with sickle cell disease.
- Trial name
- NCT02380079
- Testing
- SCD-101
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Invenux, LLC
Transfusion-dependent β-thalassemia Patients, Cardiac Iron Overload
Safety and Efficacy of Deferasirox in Combination With Desferoxamine in β-thalassaemia Patients With Severe Cardiac Iron Overload
The primary efficacy endpoint of this interventional study was to evaluate the number of patients achieving a complete response (CR), defined as patients switching from intensive deferasirox -DFO treatment, at any time point…
- Trial name
- NCT01459718
- Testing
- Deferasirox, Deferoxamine (DFO)
- Where
- 4 sites in Greece
- Volunteers
- 32 people
- Sponsor
- Novartis Pharmaceuticals
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, Thalassemia
A Study of Etavopivat in Patients With Thalassemia or Sickle Cell Disease
This clinical trial is a Phase 2 study that will evaluate the safety and clinical activity of etavopivat in patients with thalassemia or sickle cell disease and test how well etavopivat works to lower the number of red blood cell…
- Trial name
- GLADIOLUS (NCT04987489)
- Testing
- Etavopivat tablets
- Where
- 32 sites in United States, Canada and 4 more
- Volunteers
- 53 people
- Sponsor
- Forma Therapeutics, 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, Thalassemia
A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in participans with transfusion-dependent β-thalassemia (TDT).
- Conditions
- Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases
- Trial name
- NCT03655678
- Testing
- CTX001
- Where
- 14 sites in United States, Canada and 3 more
- Volunteers
- 59 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
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.
Sickle Cell Disease
PK Study of Ticagrelor in Children Aged Less Than 24 Months, With Sickle Cell Disease (HESTIA4)
The purpose of this Phase I study is to investigate the pharmacokinetic properties of ticagrelor in pediatric patients from 0 to less than 24 months with sickle cell disease.
- Trial name
- HESTIA4 (NCT03492931)
- Testing
- Ticagrelor
- Where
- 8 sites in Belgium, Italy and 4 more
- Volunteers
- 21 people
- Sponsor
- AstraZeneca
β-thalassemia Major
An Open Label Study to Evaluate the Pharmacokinetics, Safety, Tolerability and Efficacy of Deferasirox Administered to Chinese Patients With β-thalassemia Major Aged From 2 to Less Than 6 Years Old
To characterize the PK of deferasirox in pediatric β-thalassemia major patients aged from 2 to less than 6 years old, when administrated with a fixed starting dose of 20 mg/kg/day.
- Trial name
- MACS2163 (NCT01724138)
- Testing
- Deferasirox
- Sponsor
- Novartis Pharmaceuticals
Transfusion Dependent Thalassemia
Efficacy and Safety of RBCs Derived From Mirasol-treated Whole Blood in Patients Requiring Chronic Transfusion (PRAISE)
This is a prospective, multi-center, randomized, crossover trial to evaluate the clinical effectiveness of red blood cells (RBCs) derived from Mirasol-treated whole blood (WB) versus conventional RBCs in transfusion dependent…
- Trial name
- PRAISE (NCT03329404)
- Testing
- Mirasol Red Blood Cells (MIR RBCs), Reference Red Blood Cells (REF RBCs)
- Where
- 9 sites in United States, Israel and 2 more
- Volunteers
- 9 people
- Sponsor
- Terumo BCTbio