Clinical trials: Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
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
Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Sickle Cell Disease
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 deoxyribonucleic acid (DNA)) from the patient to treat his…
- Trial name
- NCT07708350
- Testing
- autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, Sequencing Assay for Variant rs114518452
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Daniel Bauer
Sickle Cell Disease (SCD), Sickle Cell Disease Vaso-occlusive Crisis
Buprenorphine for the Management of Pain in Children With Sickle Cell Disease
The goal of this clinical trial is to learn whether a single dose of buccal buprenorphine (BELBUCA) is safe when used in place and/or with full agonist opioids that would otherwise be started during a sickle cell pain crisis…
- Conditions
- Sickle Cell Disease (SCD), Sickle Cell Disease Vaso-occlusive Crisis, Sickle Cell Anemia Crisis, Sickle Cell Anemia in Children
- Trial name
- NCT07799571
- Testing
- Buprenorphine Buccal Film [Belbuca]
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Natasha Archer
Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
Phase 2 Study of Montelukast for the Treatment of Sickle Cell Anemia
In this feasibility trial, the investigators will compare participants treated with montelukast and hydroxyurea to those treated with placebo and hydroxyurea for a total of 8 weeks.
- Trial name
- NCT01960413
- Testing
- Montelukast added to Hydroxyurea
- Where
- 2 sites in United States
- Volunteers
- 46 people
- Sponsor
- Vanderbilt University Medical Center
Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0), Albuminuria
Targeting the Pathophysiology of Sickle Cell-Related Kidney Disease Using the SGLT2 Inhibitors, Empagliflozin
Sickle cell anemia (SCA) is an inherited red blood disorder. The kidneys are among the most commonly affected organ systems in SCA.
- Trial name
- EMPA-CKD (NCT07175051)
- Testing
- Empagliflozin (oral)
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of Illinois Chicago
Sickle Cell Anaemia, Sickle Cell Anemia Crisis
Hypoxic Red Blood Cells in Sickle Cell Anemia
The overall objective of this study is to evaluate the effectiveness and safety of transfusing hypoxic red blood cells manufactured with the Hemanext ONE system in patients with sickle cell anemia.
- Conditions
- Sickle Cell Anaemia, Sickle Cell Anemia Crisis, Sickle Cell Anemia in Children, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
- Trial name
- NCT06743113
- Testing
- Hemanext ONE System, Conventional RBCs
- Where
- 5 sites in United States
- Volunteers
- 48 people
- Sponsor
- Hemanext