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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. RecruitingObservationalNCT07373639

    A Long-term Follow-up Study in Patients Who Received BEAM-101

    Sickle Cell Disease, Hemoglobinopathy

    This is a Long-term Follow-up (LTFU) study in patients who received BEAM-101 in the parent study (Study BTX-AUT-001). Eligible patients who received BEAM-101 will be asked to participate in this LTFU study prior to completing Study BTX-AUT-001. Patients who enter into this study will be followed for 13 years (a total of 15 years after receiving BEAM-101).

    Sites
    17
    Target
    50
    Study sites
    Birmingham, US +16 more

    Sponsor: Beam Therapeutics Inc.

    View on ClinicalTrials.gov(opens in a new tab)
  2. RecruitingPhase 1InterventionalNCT07187973

    A Phase 1b, Open-Label Study of DISC-3405 in Participants With Sickle Cell Disease (SCD)

    Sickle Cell Disease

    This is an open-label, multicenter, within-participant dose-escalation study examining up to 3 dose levels of DISC-3405 and will assess the safety, tolerability, PK, and PD of DISC 3405 in participants with sickle cell disease.

    Sites
    11
    Target
    24
    Study sites
    Birmingham, US +10 more

    Sponsor: Disc Medicine, Inc

    View on ClinicalTrials.gov(opens in a new tab)
  3. RecruitingPhase 2InterventionalNCT05953584

    A Phase 2 Open-label Study to Evaluate the Activity of Etavopivat on Transcranial Doppler Velocities in Pediatric Patients With Sickle Cell Disease Who Are at Increased Risk for Primary Stroke

    Sickle Cell Disease

    The study will test a new medicine, etavopivat, for sickle cell disease and see if it is safe and help-ful for participants with sickle cell disease who are at an increased risk of stroke. Participants will be divided into two cohorts depending on their transcranial doppler (TCD) ultrasound results and whether or not they receive hydroxyurea (medication that they may already be taking). In one cohort, participants with conditional transcranial doppler (TCD) or participants with abnormal TCD who are not able to receive hydroxyurea will be included. The study doctor will determine if the TCD result is conditional or abnormal. In another cohort, participants with conditional TCD or participants with abnormal TCD who are receiving a stable dose of hydroxyurea will be included. The study doctor will determine if the TCD result is conditional or abnormal. The participant will start a 52-week (1 year) treatment period. The participant will take 400 milligrams (mg) of etavopivat once a day for the 52 weeks. The dose of 400 mg will be taken as 2 tablets by mouth, each containing 200 mg of etavopivat. Etavopivat may be taken with or without food. Each dose should be taken with a glass of water. As part of the study, the participants will be asked to visit the clinic frequently. The participant will have the opportunity to participate in a 48-week optional extension treatment period. The optional extension treatment period will allow continued as-sessment of safety of etavopivat in paediatric patients. At the end of the study, if deemed appro-priate the participant, the caregiver, and the study doctor, the participant may be offered the op-portunity to participate in a separate study to continue receiving etavopivat. If/when this separate study becomes available, the participant may only transfer to the new study after completion of the 52-week primary treatment period and at any time during the 48-week optional extension treatment period.

    Sites
    9
    Target
    27
    Study sites
    Raipur, IN +8 more

    Sponsor: Forma Therapeutics, Inc.

    View on ClinicalTrials.gov(opens in a new tab)
  4. RecruitingPhase 2, Phase 3InterventionalNCT05431088

    A Phase 2/3 Study of Osivelotor in Adult and Adolescent Participants With SCD

    Sickle Cell Disease

    The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of osivelotor.

    Sites
    80
    Target
    389
    Study sites
    Mobile, US +79 more

    Sponsor: Pfizer

    View on ClinicalTrials.gov(opens in a new tab)
  5. RecruitingPhase 1, Phase 2InterventionalNCT06546670

    A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease

    Sickle Cell Disease

    The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary food effect of ITU512 as well as the fetal hemoglobin (HbF)-inducing capacity of ITU512. This will be the first evaluation of the potential therapeutic effect of ITU512 in healthy participants and patients with sickle cell disease (SCD).

    Sites
    14
    Target
    161
    Study sites
    Birmingham, US +13 more

    Sponsor: Novartis Pharmaceuticals

    View on ClinicalTrials.gov(opens in a new tab)
  6. RecruitingObservationalNCT04166526

    A Pilot Study on Neuroimaging in SCD: Part of The Boston Consortium to Cure Sickle Cell Disease

    Sickle Cell Disease

    Sickle Cell Disease (SCD) impairs oxygen transport to tissue and causes endothelial injury. Thus, therapeutic interventions aim to improve both, but there is an unmet need for biomarkers to determine when intervention is necessary and evaluate the effectiveness of the chosen intervention in individual patients. This study proposes to monitor SCD and its treatment through their impact on cerebral hemodynamics, as the brain is one of the most vulnerable and consequential targets of the disease. Specifically, this study will optimize quantitative magnetic resonance imaging (MRI) and advanced optical spectroscopy techniques such as frequency-domain near-infrared and diffuse correlation spectroscopies (FDNIRS-DCS) to monitor 1) cerebral oxygen transport with measures of cerebral blood flow (CBF), cerebral oxygen extraction fraction (OEF) and cerebral metabolic rate of oxygen consumption (CMRO2) and 2) endothelial function with cerebrovascular reactivity (CVR). Additionally, this study aims to monitor baseline cerebral oxygen transport and CVR, as well as changes that occur with treatment (transfusion or genetic therapy to induce fetal hemoglobin) and assess hemoglobinopathy patients with known genotypes and phenotypes. The ultimate goal is to demonstrate the potential of this monitoring approach to select individual SCD subjects for interventions and evaluate individual responses to treatment. Success will help justify inclusion of these modalities in ongoing and future clinical trials of novel SCD therapies.

    Sites
    1
    Target
    8
    Study sites
    Boston, US

    Sponsor: Boston Children's Hospital

    View on ClinicalTrials.gov(opens in a new tab)
  7. RecruitingPhase 3InterventionalNCT06609226

    A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia

    Sickle Cell Disease, Thalassemia

    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 carries oxygen through the body. This study is looking into how safe treatment with etavopivat is and how well it works over a long period of time. The study will last for up to 264 weeks, but it will end earlier if etavopivat is approved in the participant's country.

    Sites
    107
    Target
    480
    Study sites
    Birmingham, US +106 more

    Sponsor: Novo Nordisk A/S

    View on ClinicalTrials.gov(opens in a new tab)
  8. RecruitingInterventionalNCT06290401

    A Socio-ecological Approach for Improving Self-management in Adolescents With SCD

    Anemia, Sickle Cell

    The goal of this clinical trial is to evaluate the impact of SCThrive (a behavioral self-management intervention) on patient activation, self-management behaviors, daily functioning, and emergency room visits in 260 adolescents and young adults with sickle cell disease (SCD) ages 13-21 receiving care at 1 of 4 pediatric SCD clinics. The main question[s]it aims to answer are: * Does SCThrive improve patient activation? * Does SCThrive improve self-management behaviors, daily functioning, and decrease emergency room visits? * Are any improvements maintained 3 months after treatment? Participants will complete self-management related surveys before, after, and 3 months following their participation in an 8- week, virtual group intervention with an accompanying mobile app (SCThrive). Researchers will compare outcomes for participants who receive SCThrive and participants who receive uniform standard care (SCHealthED which = standard of care plus SCD educational text messages) to see if there are differences in patient activation, self-management behaviors, daily functioning, and emergency room visits.

    Sites
    4
    Target
    310
    Study sites
    Wilmington, US +3 more

    Sponsor: Children's Hospital Medical Center, Cincinnati

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