Clinical trials: Transfusion-dependent Anemia
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.
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.
Beta-Thalassemia
Efficacy and Safety Study of Multiple Doses of VIT-2763 in Adults With Transfusion-dependent Beta-thalassemia
The main purpose of this study is to evaluate the efficacy of 3 multiple doses of VIT-2763 as measured by the reduction in red blood cell (RBC) transfusion burden from Week 13 to Week 24, to identify the most efficacious and safe…
- Trial name
- NCT04938635
- Testing
- VIT-2763 60 mg QD, VIT-2763 60 mg BID, VIT-2763 120 mg BID
- Where
- 7 sites in United States, Bulgaria and 1 more
- Sponsor
- Vifor (International) Inc.
Bone Marrow Failure Syndromes, Aplastic Anemias
A Phase 1/2 Study to Evaluate the Safety and Efficacy of Dibotatug (DR-01) in Adults With Bone Marrow Failure Syndromes
This is a multicenter, open-label, Phase 1/2 basket study to evaluate the safety and efficacy of Dibotatug (DR-01) in adults with Bone Marrow Failure syndromes.
- Conditions
- Bone Marrow Failure Syndromes, Aplastic Anemias, Severe Aplastic Anemia (SAA), Severe Aplastic Anemia, Relapse
- Trial name
- BMF (NCT07712562)
- Testing
- Dibotatug (DR-01)
- Where
- 10 sites in United States
- Volunteers
- 60 people
- Sponsor
- Dren Bio
Blood and Lymphatic Diseases
Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.
Primary Objectives: Long-term safety of BIVV003 in participants with severe sickle cell disease (SCD) and ST- 400 in participants with transfusion-dependent beta-thalassemia (TDT) Secondary Objectives: Long-term efficacy of the…
- Trial name
- NCT05145062
- Testing
- BIVV003, ST-400
- Where
- 7 sites in United States
- Volunteers
- 8 people
- Sponsor
- Sangamo Therapeutics
Iron Overload, Beta-Thalassemia
Safety and Pharmacokinetic Study of Escalating Doses of SP-420, an Iron Chelator, in Patients With β-Thalassemia
The purpose of this study is to assess safety and amount of the study drug in the blood after increasing doses of SP-420. The study will be conducted in patients with β-thalassemia.
- Trial name
- NCT02274233
- Testing
- SP-420
- Where
- 7 sites in United States, Canada and 3 more
- Volunteers
- 24 people
- Sponsor
- Sideris Pharmaceuticals
Thalassemia
A Study of SNH-119014 in Adult Participants With Non-transfusion-dependent Thalassemia (NTDT)
This study is a multicenter study to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of treatment with SNH-119014 in adult participants with non-transfusion-dependent thalassemia.
- Trial name
- NCT07579949
- Testing
- SNH-119014 dose 1, SNH-119014 dose 2
- Where
- 8 sites in China
- Volunteers
- 30 people
- Sponsor
- ScinnoHub Pharmaceutical Co., Ltd.
Beta-Thalassemia, Thalassemia
A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in participans with transfusion-dependent β-thalassemia (TDT).
- Conditions
- Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases
- Trial name
- NCT03655678
- Testing
- CTX001
- Where
- 14 sites in United States, Canada and 3 more
- Volunteers
- 59 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Non-Transfusion Dependent Beta-Thalassemia (NTDT)
A Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body
This study is researching an experimental drug called REGN7999 (called "study drug"). The study is focused on patients with non-transfusion dependent beta-thalassemia.
- Trial name
- FERVENT-1 (NCT06421636)
- Testing
- REGN7999
- Where
- 26 sites in United States, Georgia and 7 more
- Volunteers
- 19 people
- Sponsor
- Regeneron Pharmaceuticals
Myelodysplastic Syndrome
APG101 in Myelodysplastic Syndrome
It has been shown in preclinical experiments with bone marrow from patients with myelodysplastic syndrome that APG101 rescues erythrocytes from premature cell death.
- Trial name
- APG101 in MDS (NCT01736436)
- Testing
- Treatment with APG101, Bone marrow collection, Blood drawings
- Where
- 2 sites in Germany
- Volunteers
- 20 people
- Sponsor
- Apogenix GmbH
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.
Sickle Cell Disease, Thalassemia
A Study of Etavopivat in Patients With Thalassemia or Sickle Cell Disease
This clinical trial is a Phase 2 study that will evaluate the safety and clinical activity of etavopivat in patients with thalassemia or sickle cell disease and test how well etavopivat works to lower the number of red blood cell…
- Trial name
- GLADIOLUS (NCT04987489)
- Testing
- Etavopivat tablets
- Where
- 32 sites in United States, Canada and 4 more
- Volunteers
- 53 people
- Sponsor
- Forma Therapeutics, Inc.
Myelodysplastic Syndromes
A Study of Aezea® (Cenersen) in Transfusion Dependent Anemia Associated With Myelodysplastic Syndrome (MDS)
The purpose of the study is to test the safety of six cycles of cenersen treatment and to begin to test the hypothesis that intermittent administration of cenersen may lead to a reduced dependence on transfusion.
- Trial name
- NCT02243124
- Testing
- cenersen, Dexamethasone
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Eleos, Inc.
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
Myelodysplastic Syndromes
A Study to Assess Luspatercept in Lower-risk Myelodysplastic Syndrome Participants
The purpose of this study is to evaluate the efficacy and safety of Luspatercept when administered at the maximum approved dose in low-risk Myelodysplastic Syndrome participants who require red blood cell transfusions.
- Trial name
- MAXILUS (NCT06045689)
- Testing
- Luspatercept
- Where
- 52 sites in United States, Belgium and 7 more
- Volunteers
- 106 people
- Sponsor
- Bristol-Myers Squibb
Myelodysplastic Syndromes
Lenalidomide Versus Placebo in Myelodysplastic Syndromes With a Deletion 5q[31] Abnormality
The purpose of this study was to compare 2 doses (10 mg and 5 mg) of lenalidomide to that of placebo in subjects with red blood cell (RBC) transfusion-dependent low- or intermediate-1-risk IPSS MDS associated with a deletion…
- Trial name
- NCT00179621
- Testing
- Lenalidomide 5 mg, Lenalidomide 10 mg
- Where
- 38 sites in Belgium, France and 7 more
- Volunteers
- 205 people
- Sponsor
- Celgene Corporation
Myelodysplastic Syndromes, Leukemia, Myelomonocytic, Chronic
Study of Lonafarnib Versus Placebo in Subjects With Either Myelodysplastic Syndrome (MDS) or Chronic Myelomonocytic Leukemia (CMML) (Study P02978AM3)(TERMINATED)
The purpose of this study is to assess the benefit of lonafarnib (versus placebo) in patients with myelodysplastic syndrome (MDS) or chronic myelomonocytic leukemia (CMML).
- Conditions
- Myelodysplastic Syndromes, Leukemia, Myelomonocytic, Chronic, Myelodysplasia, Myelomonocytic
- Trial name
- NCT00109538
- Testing
- Lonafarnib
- Volunteers
- 47 people
- Sponsor
- Merck Sharp & Dohme LLC
Beta-Thalassemia
An Open-label Study of a Gene Therapy Product (Vebeglogene Autotemcel) in Transfusion Dependent Beta-Thalassemia
This is an interventional study to evaluate the safety and efficacy of autologous Hematopoietic Stem and Progenitor Cells (HSPCs) transduced with lentiviral vector encoding functional hemoglobin subunit beta (HBB) gene in…
- Trial name
- NCT06308159
- Testing
- Vebeglogene autotemcel
- Where
- 2 sites in China
- Volunteers
- 6 people
- Sponsor
- Lantu Biopharma
Transfusion-dependent Iron Overload
A Study Assessing the Efficacy and Safety of Deferasirox in Patients With Transfusion-dependent Iron Overload
This study uses a single arm, multi-center, open-label trial design. The study will assess the efficacy and safety of 52 weeks of treatment with deferasirox (ICL670) in patients with evidence of transfusion induced iron overload.
- Trial name
- NCT00171821
- Testing
- Deferasirox
- Where
- 97 sites in Australia, Austria and 21 more
- Volunteers
- 1,784 people
- Sponsor
- Novartis Pharmaceuticals
Myelodysplastic Syndromes, Beta-Thalassemia
Evaluating the Efficacy of Deferasirox in Transfusion Dependent Chronic Anaemias (Myelodysplastic Syndrome, Beta-thalassaemia Patients) With Chronic Iron Overload
This study will evaluate the safety and efficacy of deferasirox in transfusion dependent Myelodysplastic Syndrome, Beta-thalassaemia major patients with chronic iron overload
- Trial name
- NCT00564941
- Testing
- deferasirox
- Where
- 4 sites in Hungary
- Volunteers
- 309 people
- Sponsor
- Novartis Pharmaceuticals