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Sickle Cell Disease clinical trials near you

A clinical trial is a research study that tests whether a treatment is safe and works. Taking part is always your choice, and you can stop at any time. Listing a study here is not a recommendation — talk it through with your own team.

138 recruiting studiesFrom ClinicalTrials.gov (opens in a new tab)

Trial results

8 shown of 138

8 shown of 138 clinical trials.

  1. RecruitingPhase 2, Phase 3InterventionalNCT06761560

    Optimizing Hydroxyurea Dosage With Pharmakokinetic in Patients Suffering of Moderate to Severe Sickle Cell Anemia

    Sickle Cell Disease (SCD)

    The goal of this study is to evaluate if patients with sickle cell disease can achieve a maximum tolerate dose of hydroxuyrea (HU) over a period of 12 months faster with pharmacokinetic testing than the standard of care bloodwork follow-up. Pharmacokinetic test is used to evaluate the process by which drugs are absorbed, distributed in the body, localized in the tissues, and is excreted. Patient will be a randomized (coin toss method) into 2 groups. Group A will have an increase of their HU dosage with pharmacokinetic results and Group B will have an increase of their HU dosage following the standard of care bloodwork follow-up. Group C will include patient with sickle cell disease that has been taking HU for at least 12 months and will undergo a pharmacokinetic dosage to check the level of HU only one time.

    Sites
    1
    Target
    29
    Study sites
    Montreal, CA

    Sponsor: Yves Pastore

    View on ClinicalTrials.gov(opens in a new tab)
  2. RecruitingPhase 1, Phase 2InterventionalNCT07708714

    Optimizing Hydroxyurea Therapy in Children With Sickle Cell Anemia In Malaria Endemic Areas: The NOHARM Maximum Tolerated Dose (MTD) Study

    Sickle Cell Disease, Sickle Cell Anemia in Children

    NOHARM MTD is an extension of a previous study for children with Sickle Cell Anemia (SCA) who were enrolled in the NOHARM study. All children enrolled in NOHARM received hydroxyurea treatment at a fixed daily dose of 20 mg/kg/day. This dose was selected as a likely safe dose, but does not escalate hydroxyurea to maximum tolerated dose "MTD" as is commonly done in the US. Without this information, we cannot know whether hydroxyurea treatment at the MTD would be feasible (since it requires closer monitoring to avoid hematological toxicities), safe (since adverse events may be greater with MTD, risk of malaria may be altered by MTD, and risk of infections as a result of neutropenia could also be greater with MTD) or beneficial (MTD is associated with higher hemoglobin and fetal hemoglobin concentration).

    Sites
    1
    Target
    250
    Study sites
    Kampala, UG

    Sponsor: Children's Hospital Medical Center, Cincinnati

    View on ClinicalTrials.gov(opens in a new tab)
  3. RecruitingPhase 1, Phase 2InterventionalNCT07599176

    Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors

    Sickle Cell Disease, Beta-Thalassemia

    This is a non-ablative (partial) stem cell transplant for patients with severe sickle cell disease or beta-thalassemia requiring red cell transfusions. The intensity of the transplant is slightly increased from our previous transplant regimens. The goal is to aim for higher percentage of donor cells to stably remain in the recipients long term.

    Sites
    1
    Target
    90
    Study sites
    Bethesda, US

    Sponsor: National Heart, Lung, and Blood Institute (NHLBI)

    View on ClinicalTrials.gov(opens in a new tab)
  4. RecruitingObservationalNCT06923111

    PASS of Xromi Comparing Safety and Effectiveness in Children Under 2 Years With Sickle Cell Disease [PRECISE PASS]

    Sickle Cell Disease

    This post-authorisation safety and efficacy study (PRECISE PASS) evaluates the use of Xromi® (hydroxycarbamide 100 mg/mL oral solution) in children aged 9 months to under 2 years with sickle cell disease (SCD). The objective is to assess the safety profile and clinical effectiveness of Xromi® under routine clinical conditions. The study includes a prospective cohort of Xromi®-treated patients and a matched retrospective comparator cohort of untreated patients. Participants will be followed for 24 months from treatment initiation or matched index date.

    Sites
    12
    Target
    180
    Study sites
    Hamburg, DE +11 more

    Sponsor: Nova Laboratories Limited

    View on ClinicalTrials.gov(opens in a new tab)
  5. RecruitingInterventionalNCT03786549

    Pediatric -Adult Care Transition Program of Patients With Sickle Cell Disease

    Sickle Cell Disease

    Background The pediatric-adult care transition is a risk-disrupting time for patients with chronic disease. This care transition takes place during adolescence; a period of psychological upheavals and adaptations of family roles. During this period, medication adherence is non-optimal and absenteeism at medical appointments is high. Sickle cell disease (SCD) is the first genetic disease detected in France. It is chronic disease characterized by frequent painful vaso-occlusive crises (VOC) requiring emergency hospitalization when they are severe. Other serious complications are acute chest syndromes (ACS) and stroke. In order to improve the health status of teenagers with sickle cell disease, it is necessary to anticipate this care transition and to involve the pediatric and adult sectors. The biopsychosocial health approach and the Social-Ecological Model of Adolescent and Young Adult Readiness to Transition (SMART) describe a care transition integrating bioclinical and psychosocial factors such as integration of the patient's family, education on disease and therapeutics, psychological management of pain and medico-social orientation. The pediatric-adult transition program proposed is based on this biopsychosocial approach. It aims to improve the health status of adolescents with SCD, their quality of life and the use of health care service. Objective of the study To assess the impact of a pediatric-adult transition program on the incidence of sickle-cell-related complications leading to hospitalization on 24-months after transfer to the adult sector. The evaluation focuses on severe complications leading to hospitalization, such as VOC, ACS, and stroke. Study design Multicenter Open-label individual Randomized Controlled Trial Population : Patients aged at least 16 years old with sickle cell disease, and their parents (or legal representatives Number of subject : 196 patients (98 patients by arm) The study will last 24 months Expected results For patients and families Better health and quality of life for patients is expected, including better use of medical care after the transition program. It is also expected a better experience of the pediatric-adult care transition and indirectly a better experience of intrafamilial relations. For health professionals This project is expected to provide solutions to improve the pediatric-adult care transition of patients with chronic disease. Indeed, the methodological quality of the study will make it possible to evaluate the efficiency of the proposed program, to possibly adapt it and test it to other chronic diseases presenting the same care transition problematic. In terms of public health SCD mainly affects populations of sub-Saharan origin, with low visibility and high social vulnerability. By focusing on this population, this project will reduce the social inequalities in health, experienced by patients with SCD and their families. By improving the health, quality of life and care of patients with SCD, this project is expected to decrease the cost of the pediatric-adult care transition period.

    Sites
    8
    Target
    196
    Study sites
    Fort-de-France-La Martinique, FR +7 more

    Sponsor: Hospices Civils de Lyon

    View on ClinicalTrials.gov(opens in a new tab)
  6. RecruitingInterventionalNCT06374238

    Peer Support for Adolescents and Emerging Adults With Sickle Cell Pain

    Sickle Cell Disease, Pain

    The study, known as the Peer suppoRt for adolescents and Emerging adults with Sickle cell pain: promoting ENgagement in Cognitive behavioral thErapy (PRESENCE), aims to determine the effectiveness of digital CBT in reducing pain, opioid use, and healthcare utilization among AYAs with SCD. It also seeks to understand the role of personalized peer support in enhancing engagement and outcomes of digital CBT interventions. By leveraging existing infrastructure for delivering virtual peer support interventions, tailored digital CBT programs for individuals with SCD, and partnerships with CBOs, the study aims to provide valuable insights into the feasibility and effectiveness of digital CBT as a pain management strategy for this vulnerable population.

    Sites
    14
    Target
    390
    Study sites
    Mobile, US +13 more

    Sponsor: University of Pittsburgh

    View on ClinicalTrials.gov(opens in a new tab)
  7. RecruitingObservationalNCT06477289

    Peripheral Arterial Tonometry and Neurocognition in Sickle Cell Disease

    Sickle Cell Disease

    This study will examine sleep disordered breathing and sleep quality in participants (ages 12-25) diagnosed with sickle cell disease of any genotype. We will utilize remote peripheral arterial tonometry (PAT) and questionnaires to evaluate difficulties with sleep. PAT assessments will occur remotely in the homes of participants. Neurocognitive, behavioral, and neuroimaging evaluations will occur on the same day as a routine clinic visit. Primary Objective: Evaluate the relationship between nocturnal oxyhemoglobin saturation (SpO2) and neurocognitive functioning (working memory and verbal comprehension) in individuals (ages 12-25) diagnosed with sickle cell disease controlling for age, genotype, and social vulnerability. Secondary Objective: Assess differences in white matter integrity, silent cerebral infarcts, neuroinflammation, and functional connectivity among individuals (ages 12-25) diagnosed with sickle cell disease with and without sleep disordered breathing after controlling for age. Assess differences in self- and caregiver-reported mood and pain severity among individuals (ages 12-25) diagnosed with sickle cell disease with and without sleep disordered breathing after controlling for age. Exploratory Objectives: Explore the relationship between nocturnal oxyhemoglobin saturation (SpO2) and neurocognitive functioning (attention, processing speed, verbal memory, visual memory, motor dexterity) in individuals (ages 12-25) diagnosed with sickle cell disease controlling for age, genotype, and social vulnerability. Assess the feasibility of an optical imaging tool (Speckle Contrast Optical Spectroscopy - Open-Motion 3.0) to measure cerebral blood flow and blood volume in patients diagnosed with sickle cell disease (ages 12-25). Assess the concordance between measurement of cerebral blood flow and volume using speckle contrast optical spectroscopy and arterial spin labeling brain MRI.

    Sites
    1
    Target
    65
    Study sites
    Memphis, US

    Sponsor: St. Jude Children's Research Hospital

    View on ClinicalTrials.gov(opens in a new tab)
  8. RecruitingPhase 2InterventionalNCT06578507

    Pharmacokinetics, Efficacy and Safety of Twice Daily Dosing Regimen of Hydroxycarbamide Dispersible Tablets in Children With Sickle Cell Disease

    Sickle Cell Disease

    The purpose of this interventional, phase II, national, multicentric, non-randomised, open-label study is to evaluate the pharmacokinetics (PK), efficacy and safety of Hydroxycarbamide Paediatric dispersible tablets with a twice daily dosing regimen in children with Sickle Celle Disease between 9 months to 11 years of age. Participants will: * Take Hydroxycarbamide twice a day every day for 12 months * Visit the clinic at screening, baseline, 1, 3, 6, 9 and 12 months

    Sites
    7
    Target
    50
    Study sites
    Créteil, FR +6 more

    Sponsor: Theravia

    View on ClinicalTrials.gov(opens in a new tab)