Clinical trials: Transfusion-dependent Anemia, open to volunteers
Severe Aplastic Anemia (SAA), Transfusion-dependent Non-severe Aplastic Anemia
CsA+EPAG/HPAG+Romiplostim N01 in Newly-diagnosed SAA/TD-NSAA
This study aimed to explore the efficacy and safety of cyclosporine (CsA) +eltrombopag (EPAG)/hetrombopag (HPAG)+romiplostim N01 in the treatment of newly-diagnosed transfusion-dependent aplastic anemia (TD-NSAA) and severe…
- Trial name
- NCT07558434
- Testing
- Cyclosporine (CsA), thrombopoietin receptor agonist (TPO-RA), Romiplostim N01
- Volunteers
- 43 people
- Sponsor
- Peking Union Medical College Hospital
Transfusion-dependent Non-severe Aplastic Anemia
Enarodustat + CsA vs CsA in Newly Diagnosed TD-NSAA
This study aimed to compare the efficacy and safety of enarodustat combined with cyclosporine versus cyclosporine monotherapy in the treatment of newly-diagnosed tansfusion-dependent non-severe aplastic anemia (TD-NSAA).
- Trial name
- NCT07548671
- Testing
- Enarodustat, Cyclosporine (CsA)
- Volunteers
- 90 people
- Sponsor
- Peking Union Medical College Hospital
Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat his or her own disease.
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0), Beta-Thalassemia, Transfusion Dependent Beta-Thalassaemia
- Trial name
- NCT06647979
- Testing
- autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, Sequencing Assay for Variant rs114518452
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Daniel Bauer
Transfusion-dependent Beta-Thalassemia, Blood Transfusion
Use of the Hemanext One® Hypoxic Red Blood Cell Storage System for Transfusion in Thalassemia Patients
Transfusion-dependent thalassemia (TDT) requires lifelong, regular red blood cell (RBC) transfusions. Conventionally stored RBCs develop biochemical and structural "storage lesions," driven largely by oxidative stress, which may…
- Trial name
- NCT07055503
- Where
- Greece
- Volunteers
- 30 people
- Sponsor
- University Research Institute for the Study of Genetic & Malignant Disorders in Childhood
Hemoglobinopathies (Transfusion-dependent β-thalassemia and Sickle Cell Disease)
YOLT-204 in Patients With Hemoglobinopathies
This is a single-arm, open-label, single-dose, dose-escalation trial that plans to enrol 3-18 patients with transfusion-dependent β-thalassaemia (TDT) or sickle-cell disease (SCD).
- Trial name
- NCT07190001
- Testing
- YOLT-204
- Where
- China
- Volunteers
- 18 people
- Sponsor
- Guangzhou Women and Children's Medical Center
Transfusion-dependent Non-severe Aplastic Anemia
Treatment of Transfusion-dependent Nonsevere Aplastic Anemia With Luspatercept: a Multicenter Prospective Clinical Study
The goal of this clinical trial is to learn whether Luspatercept alone or in combination with Deferasirox can promote hematopoietic function in patients with transfusion-dependent non-severe aplastic anemia, as well as to assess…
- Trial name
- NCT06964971
- Testing
- Luspatercept, Luspatercept combine with Deferasirox
- Where
- China
- Volunteers
- 90 people
- Sponsor
- The First Affiliated Hospital of Zhejiang Chinese Medical University
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.
Myelodysplastic Syndromes, Del(5Q)
Efficacy and Safety of Luspatercept for the Treatment of Anemia Due to MDS With del5q, Refractory/Resistant/Intolerant to Prior Treatments, RBC-TD
Myelodysplastic syndromes, primarily affecting older adults, are a heterogeneous group of clonal disorders of hematopoietic stem cells characterized by ineffective hematopoiesis that manifest clinically as anemia, neutropenia…
- Conditions
- Myelodysplastic Syndromes, Del(5Q), Anemia, Transfusion-dependent Anemia
- Trial name
- Phoenix (NCT05924100)
- Testing
- Luspatercept Injection [Reblozyl]
- Where
- 23 sites in Italy
- Volunteers
- 22 people
- Sponsor
- Associazione Qol-one
Transfusion Dependent Beta Thalassemia, Sickle Cell Disease
Decision-Making and Quality of Life Surrounding Hematologic Disease and Gene Therapy
Determine knowledge, attitudes, and beliefs among adult patients, and parents of pediatric patients, with transfusion dependent beta-thalassemia and sickle cell disease toward gene therapy to treat their or their child's illness…
- Trial name
- NCT06664541
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Georgetown University