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Anemia clinical trials at UCSF
20 in progress, 11 open to new patients

  • A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)

    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.

    Oakland, California and other locations

  • A Safety Study of SYNT001 in Subjects With Chronic, Stable Warm Autoimmune Hemolytic Anemia (WAIHA)

    open to eligible people ages 18 years and up

    This study is being conducted to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), immunogenicity, and effects on warm autoimmune hemolytic anemia (WAIHA) disease activity markers of intravenous (IV) SYNT001.

    San Francisco, California and other locations

  • Age of Blood in Children in Pediatric Intensive Care Units

    open to eligible people ages up to 15 years

    ABC PICU is a randomized clinical trial that will compare the clinical consequences of RBC storage duration in 1538 critically ill children. Laboratory and observational evidence points to serious concerns about the lack of safety and effectiveness of older RBCs, especially in more vulnerable populations. Physicians and institutions have been systematically transfusing fresh RBCs to some pediatric patients primarily because of beliefs that the use of fresh RBCs improve outcomes. Conversely, the standard practice of blood banks is to deliver the oldest RBC unit in order to decrease blood wastage. To provide much needed high quality evidence to answer the question "do RBCs of reduced storage duration improve outcomes?" The ABC PICU Trial will conduct a RCT comparing development of New or Progressive Multiple Organ Dysfunction Syndrome (NPMODS) in critically ill children transfused with either RBCs stored ≤ 7 days or standard issue RBCs (expected mean RBC storage duration of 17-21 days).

    San Francisco, California and other locations

  • Efficacy and Safety of Rivipansel (GMI-1070) in the Treatment of Vaso-Occlusive Crisis in Hospitalized Subjects With Sickle Cell Disease

    open to eligible people ages 6 years and up

    This is a clinical study evaluating the efficacy and safety of rivipansel (GMI-1070) in treating subjects with sickle cell disease (SCD) who are 6 years of age or older experiencing a pain crisis necessitating hospitalization.

    Oakland, California and other locations

  • Ibrutinib and Azacitidine for Treatment of Higher Risk Myelodysplastic Syndrome

    open to eligible people ages 18 years and up

    This phase Ib trial studies the side effects and best dose of ibrutinib when given together with azacitidine in treating patients with myelodysplastic syndrome that is likely to occur or spread (higher risk) and who were previously treated or untreated and unfit for or refused intense therapy. Ibrutinib and azacitidine may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth.

    San Francisco, California and other locations

  • In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM)

    open to all eligible people

    The investigators aims to evaluate the safety of in utero hematopoietic stem cell transplantation in fetuses with alpha-thalassemia major performed at the time of in utero transfusion of red blood cells.

    San Francisco, California

  • STRIDE Biorepository

    open to eligible people ages 15-40

    The STRIDE Biorepository is an optional substudy available to participants in "Bone Marrow Transplantation vs Standard of Care in Patients with Severe Sickle Cell Disease (BMT CTN 1503) (STRIDE)".

    Oakland, California and other locations

  • Study of Clofarabine and Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation (HCT)

    open to eligible people ages up to 17 years

    Fludarabine and clofarabine are chemotherapy drugs used extensively in bone marrow transplantation. The goal of this study is to determine what causes some children to have different drug concentrations of clofarabine and fludarabine in their bodies and if drug levels are related to whether or not a child experiences severe side-effects during their bone marrow transplant. The hypothesis is that clinical and individual factors cause changes in clofarabine and fludarabine drug levels in pediatric bone marrow transplant patients and that high levels may cause severe side-effects.

    San Francisco, California

  • Study of Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation

    open to eligible people ages up to 17 years

    Fludarabine is a chemotherapy drug used extensively in bone marrow transplantation. The goal of this study is to determine what causes some children to have different drug concentrations of fludarabine in their bodies and if drug levels are related to whether or not a child experiences severe side-effects during their bone marrow transplant. The hypothesis is that clinical and genetic factors cause changes in fludarabine drug levels in pediatric bone marrow transplant patients and that high levels may cause severe side-effects.

    San Francisco, California

  • Study of Melphalan Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients

    open to eligible people ages up to 17 years

    Melphalan is a chemotherapy drug used extensively in bone marrow transplantation. The goal of this study is to determine what causes some children to have different drug concentrations of melphalan in their bodies and if drug levels are related to whether or not a child experiences severe side-effects during their bone marrow transplant. The hypothesis is that certain clinical and individual factors cause changes in melphalan drug levels in pediatric bone marrow transplant patients and that high levels may cause severe side-effects.

    San Francisco, California

  • Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients

    open to eligible people ages up to 17 years

    Thiotepa is a chemotherapy drug used extensively in bone marrow transplantation. Thiotepa is a prodrug that undergoes metabolic conversion in the liver by CYP2B6 and CYP3A4 to its primary active metabolite, TEPA. The goal of this study is to determine what causes some children to have different drug concentrations of thiotepa and TEPA in their bodies and if drug levels are related to whether or not a child experiences severe side-effects during their bone marrow transplant. The hypothesis is that certain clinical and genetic factors cause changes in thiotepa and TEPA drug levels in pediatric bone marrow transplant patients and that high levels may cause severe side-effects.

    San Francisco, California

  • A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)

    Sorry, not currently recruiting here

    Study AG348-C-007 is a multicenter study designed to evaluate the efficacy and safety of treatment with AG-348 in approximately 15-20 adult participants with pyruvate kinase deficiency (PKD), who are regularly receiving blood transfusions. The study is comprised of two parts. During the Part 1 Dose Optimization Period of the study, all participants will start on a dose of 5 mg AG-348 administered twice daily. Over the course of Part 1 each participant's dose will be optimized individually, up to a maximum dose of 50 milligrams (mg), twice daily. During the Part 2 Fixed-Dose Period, participants will receive AG-348 at their optimized dose from Part 1.

    Oakland, California and other locations

  • A Study to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)

    Sorry, not yet accepting patients

    Study AG348-C-006 will evaluate the efficacy and safety of orally administered AG-348 as compared with placebo in participants with pyruvate kinase deficiency (PKD), who are not regularly receiving blood transfusions. Participants will be randomized 1:1 to receive either AG-348 or matching placebo. The study is comprised of two parts. During the Part 1 Dose Optimization Period of the study, participants will start on a dose of 5 mg AG-348 administered twice daily. Over the course of Part 1 each participant's dose will be optimized individually, up to a maximum dose of 50 milligrams (mg), twice daily. During the Part 2 Fixed-Dose Period, participants will receive AG-348 at their optimized dose from Part 1.

    Oakland, California and other locations

  • Epidemiology of Silent and Overt Strokes in Sickle Cell Anemia

    Sorry, not currently recruiting here

    Sickle cell anemia is a rare disease occurring in an estimated 66,000 children and adults, often poor and underserved, in the United States. Strokes and silent strokes contribute significantly to illness burden in adults with sickle cell anemia, resulting in impairment, challenges with school and job performance, and premature death. Five NIH studies have identified therapies to prevent overt and silent strokes in children with sickle cell anemia, including monthly blood transfusion therapy (for preventing initial and recurrent strokes) and hydroxyurea (for preventing initial strokes). However, no stroke trials have established therapeutic approaches for adults with sickle cell anemia, despite the observation that at least 99% of children with sickle cell anemia in high-income countries reach adulthood, and approximately 60% of adults will experience one or more strokes (~50% with silent strokes and ~10% with overt strokes). Strokes in adults with sickle cell anemia have simply not been well studied. Therapies applied in children may not be effective for stroke prevention in adults with sickle cell anemia. Identifying subgroups of adults with sickle cell anemia and higher incidence coupled with the contribution of established stroke risk factors in the general population (smoking, diabetes, obesity, renal disease) will provide the prerequisite data required for the first ever phase III clinical trials focused on secondary stroke prevention in adults. In six adult sickle cell disease centers, the investigators will conduct a prospective cohort study to test the primary hypothesis that the incidence of stroke recurrence in adults with silent strokes treated with hydroxyurea will be greater than in those without strokes treated with hydroxyurea. We will test two secondary hypotheses: 1) adults with sickle cell anemia and silent strokes have cognitive deficits when compared to adults with sickle cell anemia without silent strokes; and 2) adults with sickle cell anemia and strokes receiving regular blood transfusion will have a higher incidence of stroke recurrence than adults with sickle cell anemia without strokes. We will recruit at least 72 individuals with sickle cell anemia and history of silent stroke receiving hydroxyurea therapy, at least 72 individuals with sickle cell anemia and no history of stroke, and at least 50 individuals with sickle cell anemia and history of overt stroke receiving transfusion therapy.

    Oakland, California and other locations

  • Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell Transplant

    Sorry, not accepting new patients

    This protocol provides expanded access to bone marrow transplants for children who lack a histocompatible (tissue matched) stem cell or bone marrow donor when an alternative donor (unrelated donor or half-matched related donor) is available to donate. In this procedure, some of the blood forming cells (the stem cells) are collected from the blood of a partially human leukocyte antigen (HLA) matched (haploidentical) donor and are transplanted into the patient (the recipient) after administration of a "conditioning regimen". A conditioning regimen consists of chemotherapy and sometimes radiation to the entire body (total body irradiation, or TBI), which is meant to destroy the cancer cells and suppress the recipient's immune system to allow the transplanted cells to take (grow). A major problem after a transplant from an alternative donor is increased risk of Graft-versus-Host Disease (GVHD), which occurs when donor T cells (white blood cells that are involved with the body's immune response) attack other tissues or organs like the skin, liver and intestines of the transplant recipient. In this study, stem cells that are obtained from a partially-matched donor will be highly purified using the investigational CliniMACS® stem cell selection device in an effort to achieve specific T cell target values. The primary aim of the study is to help improve overall survival with haploidentical stem cell transplant in a high risk patient population by limiting the complication of GVHD.

    San Francisco, California

  • Shared-Decision Making for Hydroxyurea

    Sorry, not currently recruiting here

    The goal of the study is to understand how best to help parents of young children with sickle cell disease and their clinicians have a shared discussion about hydroxyurea (one that takes into account medical evidence and parent values and preferences). The study will compare two methods to help clinicians facilitate this—a clinician pocket guide and a clinician hydroxyurea shared decision making toolkit—in a group of parents of children ages 0-5 with sickle cell disease. The investigators hope that both methods lead to parents reaching a high-quality, well-informed decision. In addition, the team hopes to demonstrate that parents who experience a shared decision will have lower anxiety and decisional uncertainty. The researchers also expect these parents to be more likely to choose hydroxyurea and that their children will have less pain, fewer hospitalizations, better developmental outcomes, and higher quality of life. The project team hopes to show that the toolkit method is easy for clinicians to use and gives parents the support needed to make an informed decision.

    Oakland, California and other locations

  • A Safety and Efficacy Study of R935788 in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia (AIHA)

    Sorry, in progress, not accepting new patients

    The purpose of this study is to evaluate whether fostamatinib is safe and effective in the treatment of Warm Antibody Autoimmune Hemolytic Anemia (AIHA).

    San Francisco, California and other locations

  • Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant, Mycophenolate Mofetil, and Cyclosporine in Treating Patients With Fanconi Anemia

    Sorry, in progress, not accepting new patients

    This phase II trial studies how well total-body irradiation (TBI) works when given together with fludarabine phosphate and cyclophosphamide followed by donor bone marrow transplant, mycophenolate mofetil, and cyclosporine in treating patients with Fanconi anemia (FA). Giving low doses of chemotherapy, such as fludarabine phosphate and cyclophosphamide, and TBI before or after a donor bone marrow transplant helps stop the growth of abnormal cells. It may also stop the patient's immune system from rejecting the donor's stem cells. The donated stem cells may replace the patient's immune cells and help destroy any remaining cancer cells (graft-versus-tumor effect). Sometimes the transplanted cells from a donor can also make an immune response against the body's normal cells. Giving mycophenolate mofetil and cyclosporine after the transplant may stop this from happening.

    Oakland, California and other locations

  • Long-term Safety and Efficacy of Ferriprox® in Iron Overloaded Patients With Sickle Cell Disease or Other Anemias

    Sorry, accepting new patients by invitation only

    This is a long-term follow-up to an earlier study, LA38-0411. Its purpose is to gather more information about the safety and efficacy of deferiprone in patients with sickle cell disease or other anemias who suffer from iron overload caused by regular blood transfusions.

    Oakland, California and other locations

  • Safety and Efficacy Study of Roxadustat to Treat Anemia in Patients With Chronic Kidney Disease (CKD), Not on Dialysis

    Sorry, in progress, not accepting new patients

    The purpose of the study is to evaluate the safety and efficacy of roxadustat for treatment of anemia in patients with chronic kidney disease not on dialysis.

    San Francisco, California and other locations