Sickle Cell Anemia clinical trials at UCSF
17 in progress, 4 open to eligible people
Sickle cell anemia is a blood condition where cells become sickle-shaped, blocking blood flow. UCSF studies include testing Etavopivat for treating sickle cell disease. Another study records natural changes in blood disorders over time. Researchers are also testing CRISPR-modified cells for severe sickle cell cases.
Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
open to all eligible people
Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood disorders that affect haemoglobin. Haemoglobin is the protein that…
Oakland, California and other locations
Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients with Severe Sickle Cell Disease
open to eligible people ages 12-35
This is an open label, non-randomized, 2-center, phase 1/2 trial of a single infusion of sickle allele modified cluster of differentiation (CD34+) hematopoietic stem progenitor cells (HSPCs) in subjects with in subjects ≥12 years old to 35 years old …
Oakland, California and other locations
ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
open to all eligible people
In parallel with the growth of American Thrombosis and Hemostasis Network's (ATHN) clinical studies, the number of new therapies for all congenital and acquired hematologic conditions, not just those for bleeding and clotting disorders, is…
Oakland, California and other locations
Recruitment and Engagement in Care to Impact Practice Enhancement (RECIPE) for Sickle Cell Disease
open to eligible people ages 18 years and up
The goal of this observational study is to help us understand more about the best ways to help individuals living with Sickle Cell Disease (SCD) get the best care. The main question it aims to answer is: How to find individuals unaffiliated from SCD …
Oakland, California and other locations
Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001)
Sorry, in progress, not accepting new patients
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine in which genetic material (mostly DNA) in the patient is changed to treat his or her own disease. In gene therapy,…
Oakland, California and other locations
Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)
Sorry, in progress, not accepting new patients
This clinical trial is a Phase 2/3 study that will determine the recommended dose of mitapivat and evaluate the efficacy and safety of mitapivat in sickle cell disease by testing how well mitapivat works compared to placebo to increase the amount of …
Oakland, California and other locations
EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY)
Sorry, in progress, not accepting new patients
The purpose of this study is to evaluate the efficacy, safety and tolerability of treatment with EDIT-301 in adult and adolescent participants with severe sickle cell disease (SCD).
Oakland, California and other locations
Etavopivat in Adults and Adolescents With Sickle Cell Disease (HIBISCUS)
Sorry, in progress, not accepting new patients
This clinical trial is a Phase 2/3 study that will evaluate the efficacy and safety of etavopivat and test how well etavopivat works compared to placebo to improve the amount of hemoglobin in the blood and to reduce the number of vaso-occlusive…
Oakland, California and other locations
Etavopivat in Patients With Thalassemia or Sickle Cell Disease
Sorry, in progress, not accepting new patients
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 transfusions…
Oakland, California and other locations
BIVV003 for Autologous Hematopoietic Stem Cell Transplantation in Patients With Severe Sickle Cell Disease
Sorry, in progress, not accepting new patients
This is an open label, multicenter, Phase 1/2 study in approximately eight adults with severe Sickle Cell Disease (SCD). The study will evaluate the safety, tolerability, and efficacy of autologous hematopoietic stem cell transplantation using…
Oakland, California and other locations
Haplo T-Cell Depleted Transplantation in High-Risk Sickle Cell Disease
Sorry, in progress, not accepting new patients
This study is being done to determine the safety and outcome (long-term control) of a high-dose chemotherapy regimen followed by an infusion of CD34 selected (immune cells) stem cells from a partially matched adult family member donor, called…
Oakland, California and other locations
Research Study Investigating How Well NDec Works in People With Sickle Cell Disease
Sorry, in progress, not accepting new patients
This study examines how well a new, potential medicine called NDec works and is tolerated in people with sickle cell disease. NDec is a combination of two medicines (decitabine-tetrahydrouridine). Both medicines are new for the treatment of sickle…
Oakland, California and other locations
Sickle Cell Disease and CardiovAscular Risk - Red Cell Exchange Trial (SCD-CARRE)
Sorry, in progress, not accepting new patients
The SCD-CARRE trial is a Phase 3, prospective, randomized, multicenter, controlled, parallel two-arm study aimed to determine if automated exchange blood transfusion and standard of care administered to high mortality risk adult SCD patients reduces …
Oakland, California and other locations
Zinc Supplementation in Sickle Cell Disease: A Precursor to the Think Zinc for Bones Trial
Sorry, not yet accepting patients
The goal of this short term prospective Phase II study is to compare the effects of two alternate daily doses of zinc (25 and 40 mg/day) in 34 randomly assigned homozygous Sickle Cell Disease (SCD-SS) patients aged 15-35 years old. The main question …
Oakland, California
Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301
Sorry, accepting new patients by invitation only
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.
Oakland, California and other locations
Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.
Sorry, accepting new patients by invitation only
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 biological…
Oakland, California and other locations
Sickle Cell Disease Treated with Ex Vivo Gene Therapy
Sorry, accepting new patients by invitation only
This is a multi-center, long-term safety and efficacy follow-up study for subjects with sickle cell disease who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored clinical studies. After completing the parent clinical …
Oakland, California and other locations
Our lead scientists for Sickle Cell Anemia research studies include Alison Matsunaga, MD Ellen Fung, PhD Mark Walters, MD.
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