Clinical trials: Thalassemia, Beta
Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia
Pharmacokinetics of Oral Hydroxyurea Solution
An open label, safety and pharmacokinetic study of oral hydroxyurea solution administered to children from 6 months to 17.99 years (i.e.
- Conditions
- Sickle Cell Disease, Sickle-Cell; Hemoglobin Disease, Thalassemia, Sickle Cell-beta-thalassemia, Sickle Cell Hemoglobin C
- Trial name
- HUPK (NCT03763656)
- Testing
- Oral Hydroxyurea (100 mg/mL) Solution
- Where
- 6 sites in Jamaica, United Kingdom
- Volunteers
- 33 people
- Sponsor
- Nova Laboratories Limited
Beta-thalassemia, Iron Overload
Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment
The purpose of this trial is to evaluate changes in cardiac iron as measured by MRI T2* in beta-thalassemia patients with deferasirox treatment.
- Trial name
- NCT00447694
- Testing
- Deferasirox
- Where
- 3 sites in United States
- Volunteers
- 30 people
- Sponsor
- Novartis
Sickle Cell Disease, Thalassemia Major
Pilot Study PBSCT With TCRab Depletion For Hemoglobinopathies
This is a single arm pilot study of peripheral stem cell transplantation (PSCT) with ex vivo t-cell receptor alpha beta+(TCRαβ+) T cell and cluster of differentiation 19+ beta (CD19+ B) cell depletion of unrelated donor (URD)…
- Trial name
- NCT04523376
- Testing
- CliniMACS
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Timothy Olson
Liver Iron Overload
Safety and Efficacy of Deferasirox in Patients With Transfusion Dependent Iron Overload - a Non-comparative Extension Study
The purpose of this study is to assess the safety and the effects on liver iron of Deferasirox when given for a long treatment period in patients with transfusion dependent iron overload.
- Trial name
- NCT01033747
- Testing
- Deferasirox
- Where
- 4 sites in Italy
- Volunteers
- 70 people
- Sponsor
- Novartis Pharmaceuticals
Transfusion-dependent Hemachromatosis, Thalassemia Major
Pilot Study for Patients With Poor Response to Deferasirox
This purpose of this study is to understand the differences between people who have a good response to deferasirox (exjade) compared to people who have a poor response to this medication when used for transfusion-dependent iron…
- Conditions
- Transfusion-dependent Hemachromatosis, Thalassemia Major, Sickle Cell Disease
- Trial name
- NCT00749515
- Testing
- Deferoxamine, Deferasirox, HIDA
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Boston Children's Hospital
Sickle Cell Disease, Sickle Cell Anemia
Decision Aid for Therapeutic Options In Sickle Cell Disease
Sickle cell disease (SCD) is an inherited disorder with chronic multi-system manifestations affecting 100,000 individuals in the US, largely of minority origin and associated with substantial morbidity, premature mortality…
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia, Hemoglobin SS, Hemoglobin SC
- Trial name
- NCT02326597
- Testing
- Decision Aid Tool, Standard Practice
- Where
- United States
- Volunteers
- 134 people, healthy volunteers welcome
- Sponsor
- Emory University
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.
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
B-Thalassemia
Study to Evaluate the Safety and Efficacy of Luspatercept (ACE-536) in Participants With Beta-thalassemia (A536-04/MK-6143-002)
The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics, of ascending doses of luspatercept in participants with β-thalassemia.
- Trial name
- NCT01749540
- Testing
- luspatercept
- Where
- 8 sites in Greece, Italy
- Volunteers
- 64 people
- Sponsor
- Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Premature Infant Disease, Transfusion Related Complication
Cord Blood Transfusion In Preterm Neonates (CB-TrIP)
Repeated transfusions have been associated with very poor outcome of preterm infants. Fetal hemoglobin (HbF) and adult Hb (HbA) have different affinity for oxygen.
- Conditions
- Premature Infant Disease, Transfusion Related Complication, Fetal Hemoglobin
- Trial name
- CB-TrIP (NCT03764813)
- Where
- 2 sites in Italy
- Volunteers
- 25 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
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.
Thalassemia
Evaluating the Safety and Effectiveness of Decitabine in People With Thalassemia Intermedia
Thalassemia intermedia (TI) is an inherited blood disorder that can cause anemia due to low levels of hemoglobin. Decitabine is a medication that may be effective at increasing hemoglobin levels.
- Trial name
- NCT00661726
- Testing
- Decitabine (USAN, INN)
- Where
- 3 sites in United States, Canada
- Volunteers
- 6 people
- Sponsor
- Carelon Research
Beta-Thalassemia
A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype
This is a single-arm, multi-site, single-dose, Phase 3 study in 23 participants less than or equal to (<=) 50 years of age with transfusion-dependent β-thalassemia (TDT), also known as β-thalassemia major, who do not have a β0…
- Trial name
- NCT02906202
- Testing
- LentiGlobin BB305 Drug Product
- Where
- 8 sites in United States, France and 4 more
- Volunteers
- 24 people
- Sponsor
- Genetix Biotherapeutics Inc.
Thalassemia Major
Study of Efficacy and Safety of INC424 in Regularly Transfused Patients With Thalassemia.
Patients with severe thalassemia (thalassemia major) present with severe anemia that required life-long transfusion therapy, spleen enlargement that led to increased transfusion requirement, and other serious complications as…
- Trial name
- NCT02049450
- Testing
- ruxolitinib
- Where
- 7 sites in Greece, Italy and 3 more
- Volunteers
- 30 people
- Sponsor
- Novartis Pharmaceuticals
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
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 Major, Hemosiderosis
Extension Study of the Efficacy and Safety of Deferasirox Treatment in Beta-thalassemia Patients With Transfusional Hemosiderosis (Study Amended to 2-year Duration)
To allow patients treated with deferasirox in the core study to continue iron chelation therapy for 2 years or until the drug became locally commercially available.
- Conditions
- Beta-thalassemia Major, Hemosiderosis, Iron Overload, Rare Anemia
- Trial name
- ESCALATOR (NCT00171301)
- Testing
- Deferasirox
- Where
- 5 sites in Egypt, Lebanon and 3 more
- Volunteers
- 233 people
- Sponsor
- Novartis Pharmaceuticals
Beta Thalassemia Major, Congenital Anemias
Assess the Feasibility and Safety of Granulocyte Colony Stimulating Factor (GCSF) Mobilization of CD34+ Hematopoietic Progenitor Cells in Patients With Betathalassemia Major
Betathalassemia major is a disease of the blood and bone marrow. You were born with it and it has made you unable to make normal hemoglobin and red cells. You have been receiving red blood cell transfusions all your life.
- Trial name
- NCT00658385
- Testing
- GCSF, Central venous line placement, Stem cell Collection (leukapheresis)
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Sickle Cell Anemia
Haploidentical PBMC Transplant for Severe Congenital Anemias
Background: Bone marrow transplantation (BMT), which involves transplanting a donor's marrow stem cells, is capable of curing some congenital anemias.
- Trial name
- NCT00977691
- Testing
- PBSC Transplant, Alemtuzumab, Sirolimus
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Cardiovascular Diseases, Heart Diseases
Combination Therapy Compared With Single-Drug Therapy in Patients With Cardiac Diseases
The purpose of this study is to determine whether left ventricular function improves more rapidly with deferoxamine (DFO) and deferiprone (L1) combination therapy than with DFO monotherapy in patients with thalassemia and…
- Conditions
- Cardiovascular Diseases, Heart Diseases, Beta-Thalassemia
- Trial name
- NCT00115349
- Testing
- Deferoxamine, Deferiprone (L1)
- Where
- 6 sites in United States
- Volunteers
- 20 people
- Sponsor
- Carelon Research