Clinical trials: Transfusion
Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
The primary objective of this study is to compare the effect of mitapivat versus placebo on transfusion burden in pediatric participants with α- or β-transfusion-dependent thalassemia.
- Trial name
- ENERGIZEKids-T (NCT07506863)
- Testing
- Mitapivat
- Where
- 4 sites in United States
- Volunteers
- 54 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia
The primary objective of this study is to compare the effect of mitapivat versus placebo on anemia in pediatric participants with alpha- or beta-non-transfusion-dependent thalassemia.
- Trial name
- ENERGIZEKids (NCT07517133)
- Testing
- Mitapivat
- Where
- 5 sites in United States
- Volunteers
- 45 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Transfusion Reaction, Hemolytic
Challenge Transfusion of INTERCEPT Pathogen Reduced Red Blood Cells (RBCs) in Subjects With or Without Pre-existing Antibodies to INTERCEPT RBCs
This study aims to determine whether transfusion of INTERCEPT RBCs into patients with pre-existing antibodies to INTERCEPT RBCs will result in increased antibody titer indicative of a secondary immune response, and whether these…
- Trial name
- Challenge (NCT07152379)
- Testing
- 30-40 mL of INTERCEPT RBCs, ~30 mL of INTERCEPT RBCs plus plus 10 mL biotinylated INTERCEPT RBCs
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Cerus Corporation
Transfusion-dependent Beta-Thalassemia
Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy
This is a multi-center, long-term safety and efficacy follow-up study for subjects with transfusion-dependent β-thalassemia (TDT) who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored parent…
- Trial name
- NCT02633943
- Testing
- Safety and efficacy assessments
- Where
- 15 sites in United States, Australia and 6 more
- Volunteers
- 66 people
- Sponsor
- Genetix Biotherapeutics Inc.
Sickle Cell Disease, Transfusion-dependent Beta-Thalassemia
A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301
The purpose of this study is to evaluate the long-term safety and efficacy of EDIT-301 in participants with severe sickle cell disease (SCD) or transfusion-dependent β-thalassemia (TDT) who have received EDIT-301.
- Conditions
- Sickle Cell Disease, Transfusion-dependent Beta-Thalassemia, Hemoglobinopathies
- Trial name
- NCT06363760
- Testing
- Safety and efficacy assessments
- Where
- 18 sites in United States, Canada
- Volunteers
- 54 people
- Sponsor
- Editas Medicine, Inc.
Transfusion Dependent Beta-Thalassaemia
A Safety and Efficacy Study Evaluating ET-01 in Subjects with Transfusion Dependent Β-Thalassaemia
This is a single-arm, open label, multi-center, single-dose phase 1 study in subjects with transfusion dependent β-thalassaemia.
- Trial name
- ET-01 (NCT04925206)
- Testing
- ET-01
- Where
- 4 sites in China
- Volunteers
- 8 people
- Sponsor
- EdiGene (GuangZhou) Inc.
Transfusion Dependent Beta-Thalassaemia
A Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects with Transfusion Dependent Β-Thalassaemia.
This is an open label, single site study to evaluate the safety and Efficacy of ET-01 Transplantation in subjects with Transfusion Dependent β-Thalassaemia.
- Trial name
- NCT05752123
- Testing
- ET-01
- Where
- China
- Volunteers
- 2 people
- Sponsor
- EdiGene (GuangZhou) Inc.
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.
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
Transfusion Reaction
Clinical Performance Study of the ABTest Card® Device.
The study aims to confirm the performance equivalence of the in vitro diagnostic device ABTest Card®REF with the ABTest Card®NEW device.
- Trial name
- NCT06557031
- Testing
- ABTest Card®REF and ABTest Card®NEW
- Where
- France
- Volunteers
- 200 people
- Sponsor
- Diagast
Transfusion Reaction
Clinical Performance Study of Safety Test ABO Device
The study aims to confirm the performance equivalence of the in vitro diagnostic device Safety Test ABO with the ABTest Card device.
- Trial name
- NCT06543771
- Testing
- Safety Test ABO and ABTest Card®
- Where
- France
- Volunteers
- 100 people
- Sponsor
- Diagast
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, 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.
Anemia, Sickle Cell, Complex and Transfusion-dependent Hemoglobinopathies
Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies
The goal of this research study is to establish chimerism and avoid graft-versus-host disease in patients with hemoglobinopathies.
- Conditions
- Anemia, Sickle Cell, Complex and Transfusion-dependent Hemoglobinopathies, Thalassemia, Diamond-Blackfan Anemia
- Trial name
- NCT01419704
- Testing
- Enriched Hematopoetic Stem Cell Infusion
- Where
- 3 sites in United States
- Sponsor
- Talaris Therapeutics Inc.
Veinous, Chemotherapy
A Retrospective, Observational, Multicenter Study to Evaluate the Safety and Performance of POLYSITE® and SEESITE® Implantable Ports
The purpose of this study is to evaluate performance and safety of POLYSITE® and SEESITE® implantable ports allowing repeated and prolonged access to the venous system for the administration of chemotherapy, antibiotics…
- Conditions
- Veinous, Chemotherapy, Antibiotics, Antiviral Drugs
- Trial name
- NCT05519761
- Testing
- Implantation of veinous access ports
- Where
- 3 sites in France
- Volunteers
- 250 people
- Sponsor
- Perouse Medical
Venous System, Venous Access
A Retrospective, Observational, Monocenter, Study to Describe the Utilization and Overall Safety and Performance of POLYSITE®/SEESITE® Implantable Ports
This study is a retrospective, observational, monocenter case series. The purpose of this study is to collect clinical data to describe the use of POLYSITE® and SEESITE® devices and verify overall safety and performance.
- Conditions
- Venous System, Venous Access, Chemotherapy, Antibiotics
- Trial name
- NCT05519787
- Testing
- Central Veinous access devices' insertion
- Where
- France
- Volunteers
- 70 people
- Sponsor
- Perouse Medical
Transfusion Related Acute Lung Injury
A Open Label, Post Marketing Surveillance Study Following Transfusion of INTERCEPT Platelet Components
This study is a prospective, non-randomized sequential cohort, open label, multi-center, non-inferiority, Phase IV surveillance study following transfusion of INTERCEPT PCs.
- Trial name
- PIPER (NCT02549222)
- Testing
- Standard of Care
- Where
- 15 sites in United States
- Volunteers
- 2,291 people
- Sponsor
- Cerus Corporation
Transfusion Reaction
ABTEST© Card Clinical Perfomance Study
The study confirms the performance equivalence of the in vitro Diagnostic reagent ABTEST card used with the new reformulated WS solution
- Trial name
- NCT05140395
- Testing
- ABTEST card
- Where
- France
- Volunteers
- 100 people
- Sponsor
- Diagast
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
Iron Overload Due to Repeated Red Blood Cell Transfusions
Study for Transfusionally Iron Overloaded Children, Adolescents and Adults Using FBS0701 (SSP-004184)
The purpose of this extension study is to evaluate SSP-004184AQ in patients with transfusional iron overload and to provide data on long term safety and efficacy.
- Trial name
- NCT01671111
- Testing
- SSP-004184AQ
- Where
- 9 sites in United States, Canada and 3 more
- Volunteers
- 30 people
- Sponsor
- Shire