Clinical trials: Thalassemia Major
Transfusional Iron Overload, Beta-Thalassemia
Safety, Efficacy and Pharmacokinetics of an Oral Iron Chelator Given for a Year to Pediatric Patients With Iron Overload
This is an open-label study to assess the pharmacokinetics, safety, efficacy and tolerability of SSP-004184AQ. The study consists of two phases: the pharmacokinetic phase, using a single 16 mg/kg dose of SSP-004184AQ; and the…
- Trial name
- NCT01363908
- Testing
- SPD602
- Where
- 9 sites in United States, Canada and 3 more
- Volunteers
- 30 people
- Sponsor
- Shire
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
Transfusional Iron Overload, Beta-thalassemia
Safety and Pharmacodynamic Study of an Oral Iron Chelator Given for 6 Months to Patients With Iron Overload
The purpose of this research study is to evaluate the safety of two doses of FBS0701, a new oral iron chelator, and its effectiveness in clearing iron from the liver.
- Trial name
- NCT01186419
- Testing
- SPD602 (FBS0701, SSP-004184)
- Where
- 9 sites in United States, Italy and 3 more
- Volunteers
- 51 people
- Sponsor
- Shire
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
β-thalassemia Major
A Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in β-Thalassemia Major Participants
This is a non-randomized, open label, multi-site, single-dose, phase 1/2 study in up to 18 participants (including at least 3 adolescents between 12 and 17 years of age, inclusive) with β-thalassemia major.
- Trial name
- NCT01745120
- Testing
- LentiGlobin BB305 Drug Product
- Where
- 6 sites in United States, Australia and 1 more
- Volunteers
- 19 people
- Sponsor
- Genetix Biotherapeutics Inc.
Thalassemia Major With Severe Transfusional Iron Overload
Combination Deferasirox and Deferiprone for Severe Iron Overload in Thalassemia
We hypothesize that the combination treatment with deferasirox and deferiprone will be well tolerated and will result in significant improvement in cardiac and liver iron levels.
- Trial name
- NCT01709032
- Testing
- Deferasirox and deferiprone
- Where
- 2 sites in United States
- Volunteers
- 9 people
- Sponsor
- Children's Hospital of Philadelphia
Skin Ulcers, Sickle Cell Anemia
Phase II Randomized Trial:Arginine Butyrate Plus Standard Local Therapy in Patients With Refractory Sickle Cell Ulcers
OBJECTIVES: I. Compare the efficacy of local care alone vs local care plus arginine butyrate in terms of healing rate in patients with refractory sickle cell ulcers. II.
- Trial name
- NCT00004412
- Testing
- Arginine Butyrate, Standard local care dressing
- Where
- 4 sites in United States
- Volunteers
- 23 people
- Sponsor
- Susan P. Perrine
Sickle Cell Disease, Hemoglobin SC
Bone Marrow Transplantation, Hemoglobinopathies, SCALLOP
Patients are being asked to participate in this study because they have severe sickle cell anemia (SCD) with or without the beta thalassemia trait.
- Trial name
- SCALLOP (NCT00578344)
- Testing
- Busulfan, Campath 1H, Cyclophosphamide and MESNA
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- Tami D. John
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission
Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer
This phase II trial studies how well fludarabine phosphate, cyclophosphamide, total body irradiation, and donor stem cell transplant work in treating patients with blood cancer.
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
- Trial name
- NCT03333486
- Testing
- Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Roswell Park Cancer Institute
Transfusion Dependent Beta-thalassemia
A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)
This is a single-arm, multi-site, single-dose, Phase 1/2 study to assess ST-400 in 6 subjects with transfusion-dependent β-thalassemia (TDT) who are ≥18 and ≤40 years of age.
- Trial name
- NCT03432364
- Testing
- ST-400 Investigational product
- Where
- 6 sites in United States
- Volunteers
- 5 people
- Sponsor
- Sangamo Therapeutics
Bone Marrow Failure Syndrome, Thalassemia
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum…
- Conditions
- Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia
- Trial name
- NCT03513328
- Testing
- Thiotepa--single daily dose, Thiotepa--escalated dose
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Florida
Hemoglobinopathies, Sickle Cell Disease
Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies
The purpose of this study is to determine if treatment with reduced-dose busulfex, fludarabine and alemtuzumab (CAMPATH) followed by sten cell infusion will allow for donor stem cells to grow in patients with hemoglobinopathies…
- Conditions
- Hemoglobinopathies, Sickle Cell Disease, Thalassemia
- Trial name
- NCT00153985
- Testing
- Busulfex, Fludarabine, Alemtuzumab
- Where
- 6 sites in United States
- Volunteers
- 2 people
- Sponsor
- Dana-Farber Cancer Institute