Clinical trials: Non-transfusion-dependent Thalassemia
Thalassemia
Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Sever Variants of Beta 0/+ Thalassemia, THALLO
Patients have severe beta-thalassemia or one of the thalassemia variants. Thalassemia is a hereditary disease in which the bone marrow produces abnormal red blood cells that have a shorter life span than normal red blood cells.
- Trial name
- THALLO (NCT00578292)
- Testing
- Busulfan, Fludarabine, Campath 1H
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Baylor College of Medicine
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
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
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
Thalassemia
Assessment of Pain in People With Thalassemia
Thalassemia is an inherited blood disorder that can result in mild to severe anemia. People with thalassemia often experience pain, but the exact sources and prevalence of pain remain unknown.
- Trial name
- Pain (NCT00872339)
- Where
- 8 sites in United States, Canada
- Volunteers
- 252 people
- Sponsor
- Carelon Research
Beta-Thalassemia, Non-transfusion-dependent Thalassemia
Safety, Tolerability, Pharmacokinetics (PK), Pharmacodynamics (PD) and Preliminary Efficacy of VIT-2763 in β-thalassaemia
This is a randomised, double-blind, placebo-controlled parallel group trial to investigate the safety, tolerability and efficacy of multiple doses of VIT-2763 versus placebo in participants with non-transfusion-dependent…
- Trial name
- VITHAL (NCT04364269)
- Testing
- VIT-2763 once a day (QD), VIT-2763 twice a day (BID)
- Where
- 16 sites in Greece, Israel and 3 more
- Volunteers
- 35 people
- Sponsor
- Vifor (International) Inc.
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.
Transfusion-dependent Thalassemia, Non-transfusion-dependent Thalassemia
Open-label, Multicenter Study Assessing Preference for Deferasirox Film-coated Tablet Compared to Dispersible Tablet
Study to evaluate patient preference of deferasirox film-coated tablet (FCT) or deferasirox dispersible tablet (DT) in patient with transfusion - dependent thalassemia or non-transfusion -dependent thalassemia as measured by…
- Trial name
- Jupiter (NCT02993224)
- Testing
- Deferasirox dispersable tablet (DT), Deferasirox film coated tablet (FCT)
- Where
- 17 sites in Egypt, Lebanon and 5 more
- Volunteers
- 148 people
- Sponsor
- Novartis Pharmaceuticals
Thalassemia
A Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Participants With Non-transfusion-dependent Thalassemia
Study AG348-C-010 is a multicenter study to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of treatment with AG-348 in adult participants with non-transfusion-dependent thalassemia (NTDT).
- Trial name
- NCT03692052
- Testing
- AG-348
- Where
- 4 sites in United States, Canada and 1 more
- Volunteers
- 20 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Sickle Cell Disease, Thalassemia
Stem Cell Transplant for Hemoglobinopathy
This study tests the clinical outcomes of one of two preparative regimens (determined by available donor source) in patients with non-malignant hemoglobinopathies.
- Conditions
- Sickle Cell Disease, Thalassemia, Severe Congenital Neutropenia, Diamond-Blackfan Anemia
- Trial name
- NCT00176852
- Testing
- Busulfan, Fludarabine, ATG, TLI, Busulfan, Cyclophosphamide, ATG, GCSF, Campath, Fludarabine, Cyclophosphamide
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
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
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
Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)
The primary purpose of this study was to compare the effect of mitapivat versus placebo on hemolytic anemia in participants with alpha- or beta-non-transfusion dependent thalassemia (NTDT).
- Trial name
- NCT04770753
- Testing
- Mitapivat
- Where
- 68 sites in United States, Brazil and 16 more
- Volunteers
- 194 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Non-transfusion Dependent Thalassemia
Efficacy and Safety Study of Deferasirox in Patients With Non-transfusion Dependent Thalassemia
Assessed the efficacy of deferasirox in patients with non-transfusion dependent thalassemia based on change in liver iron concentration from baseline after 52 weeks of treatment.
- Trial name
- THETIS (NCT01709838)
- Testing
- deferasirox
- Where
- 11 sites in China, Greece and 6 more
- Volunteers
- 134 people
- Sponsor
- Novartis Pharmaceuticals