Clinical trials: Transfusion-dependent Anemia
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
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
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.
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
Transfusion-dependent Anemia
Study to Evaluate Treatment Compliance, Efficacy and Safety of an Improved Deferasirox Formulation (Granules) in Pediatric Patients (2-<18 Years Old) With Iron Overload
This was a randomized, open-label, multicenter, two arm, phase II study to evaluate treatment compliance and change in serum ferritin of a deferasirox granule formulation and a deferasirox dispersible tablet (DT) formulation in…
- Trial name
- NCT02435212
- Testing
- Deferasirox granule formulation, Deferasirox DT formulation
- Where
- 43 sites in United States, Belgium and 15 more
- Volunteers
- 224 people
- Sponsor
- Novartis Pharmaceuticals
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
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
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.
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
Thalassemia, Iron Overload
Combined Chelation Therapy in Patients With Transfusion Dependent Thalassemia and Iron Overload
This is a pilot study looking at the safety and efficacy of giving combination chelation with deferasirox and deferoxamine. The hypothesis is that combination chelation is safe in decreasing overall iron in patients with…
- Trial name
- NCT00901199
- Testing
- Combo Chelation with Deferasirox (Exjade) and Deferoxamine (DFO)
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Elliott Vichinsky
Iron Overload
Safety and Efficacy of Ferriprox™ (Deferiprone) Oral Solution in Iron Overloaded Pediatric Patients
The primary objective is to assess the safety of Ferriprox oral solution for the treatment of iron overload in pediatric patients with transfusion-dependent anemia.
- Trial name
- NCT00529152
- Testing
- Deferiprone
- Where
- 4 sites in Egypt, Indonesia and 1 more
- Volunteers
- 100 people
- Sponsor
- ApoPharma
Beta-Thalassemia
A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia
This is a single-arm, multi-site, single-dose, Phase 3 study in approximately 18 participants less than or equal to (<=) 50 years of age with transfusion-dependent β-thalassemia (TDT), who have a β0/β0, β0/IVS-I-110, or…
- Trial name
- NCT03207009
- Testing
- LentiGlobin BB305 Drug Product
- Where
- 9 sites in United States, France and 4 more
- Volunteers
- 19 people
- Sponsor
- Genetix Biotherapeutics Inc.
Transfusional Iron Overload, β-thalassemia Major
A 4-year Extension Study to Core 1-year Study of Iron Chelation Therapy With Deferasirox in β-thalassemia Major Pediatric Patients With Transfusional Iron Overload.
In this 4-year extension study the safety, efficacy and and pharmacokinetics of deferasirox in regularly transfused pediatric patients with β-thalassemia major was assessed.
- Conditions
- Transfusional Iron Overload, β-thalassemia Major, Pediatric Rare Anemia
- Trial name
- NCT00390858
- Testing
- Deferasirox
- Where
- 4 sites in France, Italy
- Volunteers
- 40 people
- Sponsor
- Novartis Pharmaceuticals
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
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
Beta Thalassemia Major Anemia, Iron Overload
Safety and Efficacy of Early Treatment With Deferiprone in Infants and Young Children
This study is looking at the effects of giving early treatment of deferiprone to young children with beta thalassemia who have started receiving regular blood transfusions but have not yet reached the criteria for starting on…
- Trial name
- START (NCT03591575)
- Testing
- Deferiprone oral solution
- Where
- 4 sites in Egypt, Indonesia
- Volunteers
- 64 people
- Sponsor
- Chiesi Canada Corp
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.
Chronic Iron Overload
Pharmacokinetic Study of Deferiprone in Paediatric Patients
Deferiprone (DFP) is the most extensively studied oral iron chelator to date. It has been authorised in Europe in 1999 for the treatment of iron overload in patients with beta-thalassaemia major when DFO is contraindicated or…
- Trial name
- DEEP-1 (NCT01740713)
- Testing
- Deferiprone, dose level 1, Deferiprone, dose level 2, Deferiprone, dose level 3
- Where
- 7 sites in Cyprus, Egypt and 1 more
- Volunteers
- 23 people
- Sponsor
- Consorzio per Valutazioni Biologiche e Farmacologiche
Diamond Blackfan Anemia, Blackfan Diamond Syndrome
Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia
This study will determine the safety and possibility of giving the amino acid, leucine, in patients with Diamond Blackfan anemia(DBA)who are on dependent on red blood cell transfusions.
- Conditions
- Diamond Blackfan Anemia, Blackfan Diamond Syndrome, DBA, Congenital Hypoplastic Anemia
- Trial name
- LeucineDBA (NCT01362595)
- Testing
- leucine
- Where
- 11 sites in United States
- Volunteers
- 55 people
- Sponsor
- Northwell Health