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Sickle Cell Anemia clinical trials at UC Health
19 in progress, 15 open to new patients

  • A Study of the Safety Of Experimental Medicine Rivipansel (GMI-1070) For Hospitalized Patients With Sickle Cell Disease

    open to eligible people ages 6 years and up

    This is an open label extension study in subjects with Sickle Cell Disease (SCD) who have completed the double blind Phase 3 study (B5201002).

    at UC Davis

  • Adjuvant Low-dose Ketamine in Pediatric Sickle Cell Vaso-occlusive Crisis

    open to eligible people ages 8-25

    Acute vaso-occlusive episodes (VOEs) in sickle cell disease (SCD) are primarily managed with opioids. Tolerance and hyperalgesia to opioids develops due to N-methyl-D-aspartate (NMDA)-receptor mediated activation of the nociceptive system, and as a receptor antagonist, ketamine mitigates this. Intravenous (IV) ketamine has demonstrated efficacy in reducing post-operative, chronic, and cancer-related pain in pediatrics, as well as in reducing time to pain control in the emergency department (ED) in adults. Limited studies suggest efficacy in adult opioid-refractory SCD patients. This study is investigating the safety and tolerability of adjuvant low-dose IV ketamine bolus for pediatric SCD VOE in the ED, as well as its efficacy in improving pain control and reducing hospitalization.

    at UCSF

  • Allogeneic SCT of CordIn™, in Patients With Hemoglobinopathies

    open to eligible people ages 2-25

    CordIn™ is a cryopreserved stem/progenitor cell-based product of purified CD133+ cells composed of ex vivo expanded allogeneic UCB cells. The overall study objectives are to evaluate the safety and efficacy of CordIn™.

    at UCSF

  • Bone Marrow Transplantation vs Standard of Care in Patients With Severe Sickle Cell Disease (BMT CTN 1503)

    open to eligible people ages 15-40

    This is a clinical trial that will compare survival and sickle related outcomes in adolescents and young adults with severe sickle cell disease after bone marrow transplantation and standard of care. The primary outcome is 2-year overall survival.

    at UCSF

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

    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.

    at UCSF UC Davis

  • Haplo T-Cell Depleted Transplantation in High-Risk Sickle Cell Disease

    open to eligible people ages 2-20

    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 haploidentical stem cell transplantation, in high-risk sickle cell disease patients. Funding Source - FDA OOPD

    at UCLA UCSF

  • Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMT CTN 1507)

    open to eligible people ages 5-45

    This is a Phase II, single arm, multi-center trial, designed to estimate the efficacy and toxicity of haploidentical bone marrow transplantation (BMT) in patients with sickle cell disease (SCD). Based on their age and entry criteria patients are stratified into two groups: (1) children with SCD with strokes; and (2) adults with severe SCD.

    at UCSF

  • Laboratory-based Hypnosis Intervention on Pain Responsivity in Adolescents With Sickle Cell Disease

    open to eligible people ages 12-18

    The purpose of this study is to test the effects of a laboratory-based hypnosis session compared to an attention control condition on peripheral blood flow, autonomic stress responses, and acute pain responses in adolescents (ages 12-18) with sickle cell disease, and examine how perceived disease-related stigma may affect these responses.

    at UCLA

  • Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease

    open to eligible people ages 2-34

    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 patients undergoing a familial haploidentical allogeneic stem cell transplantation with CD34 enrichment and T-cell addback. This patient population historically has a risk of developing sinusoidal obstructive syndrome (SOS) and Defibrotide has demonstrated efficacy in treatment of SOS. The Funding Source is FDA OOPD.

    at UCLA

  • Stem Cell Gene Therapy for Sickle Cell Disease

    open to eligible people ages 18 years and up

    This Phase I clinical trial will assess the safety and initial evidence for efficacy of an autologous transplant of lentiviral vector modified bone marrow for adults with severe sickle cell disease.

    at UCLA

  • 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)".

    at UCSF

  • 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.

    at UCSF

  • 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.

    at UCSF

  • 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.

    at UCSF

  • 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.

    at UCSF

  • Dose-Finding Study of SC411 in Children With Sickle Cell Disease

    Sorry, in progress, not accepting new patients

    This is a Phase 2, randomized, double-blind, placebo-controlled, parallel-group, dose-finding study of SC411 in children with sickle cell disease (SCD). The primary objective of the study is to evaluate the safety and tolerability of three different doses of SC411 compared to a placebo. All patients will undergo eight weeks of oral study treatment and a four-week safety follow-up period. Patients will be randomized to one of three dose levels of SC411 or placebo.

    at UCSF

  • 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.

    at UCSF

  • 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.

    at UCSF

  • 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.

    at UCSF

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