Anemia clinical trials at University of California Health
45 in progress, 18 open to eligible people
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…
at UC Irvine UCSF
Research Study to Evaluate How Well Etavopivat Works in People With Sickle Cell Disease
open to eligible people ages 12 years and up
This study is conducted to confirm whether etavopivat works well at reducing the number of Vaso-occlusive crisis VOCs (sickle cell pain crises) caused by obstructions in blood vessels in adults and adolescents living with sickle cell disease. The…
at UC Irvine UCLA
Tebapivat (AG-946) in Participants With Anemia Due to Lower-Risk Myelodysplastic Syndromes (LR-MDS)
open to eligible people ages 18 years and up
This purpose of this study is to establish proof of concept of tebapivat in participants with LR-MDS in Phase 2a and to evaluate the effect of tebapivat on transfusion independence (TI) in participants with LR-MDS in phase 2b.
at UCLA
Comparing Unrelated Donor BMT with IST for Pediatric and Young Adult Patients with Severe Aplastic Anemia (TransIT, BMT CTN 2202)
open to eligible people ages 0-25
Severe Aplastic Anemia (SAA) is a rare condition in which the body stops producing enough new blood cells. SAA can be cured with immune suppressive therapy or a bone marrow transplant. Regular treatment for patients with aplastic anemia who have a…
at UCLA UCSF
Acupressure in Patients With Sickle Cell Disease
open to eligible people ages 14-80
The proposed research is to determine the clinical efficacy and neurobiological mechanisms of acupressure analgesia in patients with sickle cell disease (SCD).
at UC Irvine
Alendronate for Osteonecrosis in Adults With Sickle Cell Disease
open to eligible people ages 18-80
A prospective, single-arm, intervention study of oral alendronate in adults with sickle cell disease and osteonecrosis
at UC Davis
Pharmacodynamics of Tuspetinib (HM43239) in Patients with Relapsed or Refractory Acute Myeloid Leukemia
open to eligible people ages 18 years and up
The main purpose of this study is to identify a safe and potentially effective dose of tuspetinib to be used in future studies in study participants diagnosed with acute myeloid leukemia (AML), myelodysplastic syndromes with increased blasts grade 2 …
at UC Davis UC Irvine UCSD
ELEMENT-MDS: A Study to Compare the Efficacy and Safety of Luspatercept in Participants With Myelodysplastic Syndrome (MDS) and Anemia Not Receiving Blood Transfusions
open to eligible people ages 18 years and up
The purpose of the study is to compare the efficacy and safety of Luspatercept vs epoetin alfa in the treatment of anemia in adults due to IPSS-R very low, low, intermediate-risk MDS in ESA-naïve participants who are non-transfusion dependent (NTD).
at UCLA
Exercise in Child Health
open to eligible people ages 10-17
This study is a cooperative investigation funded by the NIH. The project is a collaboration among three major NIH Clinical Translational Science Awardees: 1) UCI (lead site with its affiliate CHOC), 2) Northwestern University (with its affiliate…
at UC Irvine
Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease
open to eligible people ages 6 months to 34 years
This is a follow-up trial to NYMC 526 (NCT01461837) to assess the safety, efficacy and toxicity of administering Defibrotide prophylaxis for high-risk sickle cell or beta thalassemia patients undergoing a familial haploidentical allogeneic stem cell …
at UCLA
Research Study Investigating How Well NDec Works in People With Sickle Cell Disease
open to eligible people ages 18 years and up
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…
at UCLA UCSF
CSL889 in Adults and Adolescents With Sickle Cell Disease During Vaso-Occlusive Crisis
open to eligible people ages 12 years and up
This study consists of two parts: phase 2 (Part A) and phase 3 (Part B). It is a multicenter study designed to evaluate the safety, effectiveness, and pharmacokinetics (PK) of CSL889 (human hemopexin) when given intravenously (IV) to adults and…
at UC Irvine
Pharmacodynamics of Pociredir
open to eligible people ages 18-65
This is a study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of Pociredir in participants with sickle cell disease.
at UCLA
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 …
at UCLA UCSF
Venetoclax in Combination With ASTX727 for the Treatment of Chronic Myelomonocytic Leukemia and Other Myelodysplastic Syndrome/Myeloproliferative Neoplasm
open to eligible people ages 18 years and up
This phase II trial tests whether decitabine and cedazuridine (ASTX727) in combination with venetoclax work better than ASTX727 alone at decreasing symptoms of bone marrow cancer in patients with chronic myelomonocytic leukemia (CMML),…
at UC Davis UC Irvine
Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
“Assessing new blood cells growth after transplant using cord blood units that do not meet FDA guidelines but meet NMDP guidelines”
open to all eligible people
This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients with hematologic malignancies and other indications.
at UCLA UCSD UCSF
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…
at UC Davis UCLA UCSD UCSF
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 …
at UCSF
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,…
at UC Davis UCLA UCSF
Adult and Pediatric Participants With SCD
Sorry, in progress, not accepting new patients
The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of osivelotor.
at UCLA
Fostamatinib Disodium in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia
Sorry, accepting new patients by invitation only
The primary objective of this study is: • To evaluate the long-term safety of fostamatinib in subjects with warm antibody autoimmune hemolytic anemia (wAIHA).
at UCLA
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 …
at UCLA UCSD UCSF
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).
at UCSF
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…
at UC Irvine UCSF
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…
at UC Davis UCSF
Long-term Safety of Inclacumab Administered to Participants With Sickle Cell Disease
Sorry, in progress, not accepting new patients
This study is an open-label study to evaluate the safety of long-term administration of inclacumab in participants with sickle cell disease (SCD). Participants in this study will have completed a prior study of inclacumab.
at UC Irvine
Luspatercept (ACE-536) Versus Placebo in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis on Concomitant JAK2 Inhibitor Therapy and Who Require Red Blood Cell Transfusions
Sorry, in progress, not accepting new patients
The purpose of this Phase 3 study is to evaluate the efficacy and safety of Luspatercept compared with placebo in subjects with myeloproliferative neoplasm (MPN)-associated Myelofibrosis (MF) and anemia on concomitant Janus kinase 2 (JAK2) inhibitor …
at UCLA
Etavopivat in Adults and Adolescents With Sickle Cell Disease (HIBISCUS)
“We are looking for people age 12 to 65 years old with SCD to help test a drug trying to reduce the number of vaso-occlusive crises”
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…
at UC Davis UC Irvine UCSF
Upfront Haploidentical or Unrelated Donor BMT to Restore Normal Hematopoiesis in Aplastic Anemia
Sorry, not yet accepting patients
BMT CTN 2207 will investigate the use of marrow transplantation for treatment of severe aplastic anemia that has not previously been treated.
at UCLA
Pazopanib on Hereditary Hemorrhagic Telangiectasia Related Epistaxis and Anemia (Paz)
Sorry, in progress, not accepting new patients
During the Efficacy Study (Part B), the investigators will study whether Pazopanib, taken daily for 24 weeks, will reduce the severity of nose bleeds in patients with hereditary hemorrhagic telangiectasia (HHT). Patients will either be provided…
at UCLA
Gene Transfer for Sickle Cell Disease
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 that uses genetic material (mostly DNA) from the patient to treat his or her own disease. In gene therapy, the…
at UCLA
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…
at UCLA UCSF
Ibrutinib and Rituximab Compared With Fludarabine Phosphate, Cyclophosphamide, and Rituximab in Treating Patients With Untreated Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma
Sorry, in progress, not accepting new patients
This phase III trial studies ibrutinib and rituximab to see how well they work compared to fludarabine phosphate, cyclophosphamide, and rituximab in treating patients with untreated chronic lymphocytic leukemia or small lymphocytic lymphoma.…
at UC Irvine
Iron Deficiency Anemia (IDA) and the Brain
Sorry, not currently recruiting here
This is a trial with an observational and an interventional arm, in patients with moderate to severe anemia and control subjects. The main purposes of this study is to phenotype the scope of neurocognitive deficits from iron deficiency anemia (IDA)…
at UCLA
Lenalidomide With or Without Epoetin Alfa in Treating Patients With Myelodysplastic Syndrome and Anemia
Sorry, in progress, not accepting new patients
This randomized phase III trial studies lenalidomide to see how well it works with or without epoetin alfa in treating patients with myelodysplastic syndrome and anemia. Lenalidomide may stop the growth of myelodysplastic syndrome by blocking blood…
at UC Davis
Sickle Cell Disease (SCD) Bone Pain Study
Sorry, in progress, not accepting new patients
A prospective study to determine how low bone mineral density and/or vertebral compression fractures associate with pain in adults with sickle cell disease
at UC Davis
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 …
at UCSF
Stem Cell Gene Therapy for Sickle Cell Disease
Sorry, in progress, not accepting new patients
This Phase I clinical trial will assess the safety and initial evidence for efficacy of an autologous transplant of lentiviral vector modified peripheral blood for adults with severe sickle cell disease.
at UCLA
Transfusion of Prematures Trial
Sorry, in progress, not accepting new patients
The objective of the TOP trial is to determine whether higher hemoglobin thresholds for transfusing ELBW infants resulting in higher hemoglobin levels lead to improvement in the primary outcome of survival and rates of neurodevelopmental impairment…
at UCLA
Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)
Sorry, in progress, not accepting new patients
This phase II trial tests whether treosulfan, fludarabine, and rabbit antithymocyte globulin (rATG) work when given before a blood or bone marrow transplant (conditioning regimen) to cause fewer complications for patients with bone marrow failure…
at UCSD UCSF
Unrelated Donor Transplant Versus Immune Therapy in Pediatric Severe Aplastic Anemia
Sorry, in progress, not accepting new patients
The purpose of this study is to determine the feasibility of comparing outcomes of patients treated de novo with immunosuppressive therapy (IST) versus matched unrelated donor (MUD) hematopoietic stem cell transplant (HSCT) for pediatric acquired…
at UCSF
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 …
at UCSF
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.
at UCSF
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…
at UC Davis UCSF
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 …
at UCSF
Our lead scientists for Anemia research studies include Gary J. Schiller, MD Oyebimpe O Adesina, MD, MS Justin P. McWilliams, MD Dan Cooper Deepa Jeyakumar Alison Matsunaga, MD Theodore B. Moore, MD Wanxing Chai-Ho, MD Ellen Fung, PhD Mary E. Sehl, MD Steven Tsai Brian A. Jonas Donald Kohn, MD Annette Von Drygalski, MD.
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