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124 studies

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  • Sickle cell disease

    Bone Loss, Physical Function and Frailty in Older Women With Sickle Cell Trait Sickle Cell Trait

    Comparing bone loss, physical function, and frailty in older women with sickle cell trait

    📍 UConn Health · Farmington, CT · Get directions →

    This is a cross-sectional, clinical research study comparing postmenopausal women of African Descent (AD) with different hemoglobin genotypes: normal and sickle cell trait (SCT). This research study has two purposes.

    ✓ Study care freeAges 50+WomenLearn more →
  • Sickle cell disease

    Achieving Understanding of the Natural History of Sickle Cell Trait (AUNT)

    Tracking people with sickle cell trait to understand health patterns and complications

    📍 University of Alabama · Birmingham, AL · +9 more US sites · Get directions →

    The main purpose of this study is to create a longitudinal cohort of those with Sickle Cell Trait (SCT) to better understand the hematologic phenotype for those that carry HbS, assess for differences in those with varying quantities of HbS and assess for potential clinical complications of SCT.

    ✓ Study care freeAges 18+Learn more →
  • Sickle cell disease

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

    Testing different doses of DISC-3405 to check safety in sickle cell disease

    📍 University of Alabama at Birmingham · Birmingham, AL · +6 more US sites · Get directions →

    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.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • Sickle cell disease

    Escalating Doses of VAS-101 in Subjects With Stable Sickle Cell Disease

    Testing higher doses of VAS-101 in people with stable sickle cell disease

    💵 May compensate

    📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →

    Background: Sickle cell disease (SCD) is an inherited blood disorder. The disease affects the ability of red blood cells to carry oxygen.

    ✓ Study care freePhase 1Ages 18–90Learn more →
  • Sickle cell disease

    Stress Management and Mindfulness Intervention for Patients With Sickle Cell Disease

    Testing a stress-management and mindfulness program to improve quality of life in people with sickle

    📍 University of Pennsylvania · Philadelphia, PA · Get directions →

    The purpose of this research is to determine whether a new stress management and mindfulness program can improve quality of life in individuals with sickle cell disease. The program is based on a number of empirically supported treatments including techniques from cognitive-behavioral therapy like overcoming motivational barriers to self-care, learning to see things as accurately as possible, practicing assertive communication, relaxation exercises like deep slow breathing, mindfulness meditation, and other stress management strategies.

    ✓ Study care freeAges 18+Learn more →
  • Sickle cell disease

    SMYLS Multi-site Trial

    Testing a mobile app and website to help teens manage sickle cell symptoms

    📍 Medical University of South Carolina · Charleston, SC · Get directions →

    The purpose of this study is to find out whether a web-based intervention using a mobile app is helpful for teens and young adults with sickle cell disease (SCD) in learning how to care for and manage their symptoms.

    ✓ Study care freeAges 14–21Learn more →
  • Sickle cell disease

    A Safety, Efficacy, and Pharmacokinetic (PK) Study of HBI-002, an Oral Carbon Monoxide (CO) Therapeutic, in Subjects With Sickle Cell Disease (SCD)

    Testing the safety and how an oral carbon monoxide liquid works in sickle cell disease

    📍 Cevaxin - The Panama Clinic · Panama City, · Get directions →

    This is a multi-center, open label Phase 2a clinical trial in subjects with sickle cell disease to assess safety, tolerability, pharmacokinetics, and pharmacodynamics of HBI-002, an orally administered liquid containing carbon monoxide (CO), with doses daily for 14 days.

    ✓ Study care freePhase 1Ages 18–55Learn more →
  • Sickle cell disease

    Sickle Cell Improvement: Enhancing Care in the Emergency Department

    Improving emergency care for people with sickle cell disease pain

    📍 Children's Wisconsin · Milwaukee, WI · Get directions →

    Sickle cell disease (SCD) is an inherited blood disorder affecting approximately 36,000 children in the United States, approximately 90% of whom are Black. The disease is characterized by recurrent, severe pain crises which result in high rates of emergency department visits and hospitalizations, and decreased quality of life.

    ✓ Study care freeLearn more →
  • Sickle cell disease

    Integrative Medicine in Pain Management in Sickle Cell Disease, 2.0

    Testing whether acupuncture can reduce pain in people with sickle cell disease

    💵 May compensate

    📍 Indiana University School of Medicine · Indianapolis, IN · Get directions →

    The proposed research is to determine the clinical efficacy and neurobiological mechanisms of acupuncture analgesia in patients with sickle cell disease.

    ✓ Study care freeAges 14–80Learn more →
  • Sickle cell disease

    Verifying Antibodies After Live Immunization Delivery (VALID): A Study of Measles Vaccine Immunogenicity in Children With Sickle Cell Disease

    Checking whether children with sickle cell disease develop protection after measles shots

    📍 Cincinnati Children's Hospital Medical Center · Cincinnati, OH · Get directions →

    The goal of this study is to learn if infants with sickle cell disease (SCD) develop adequate protection after measles vaccines. (not looking at any prolonged duration)

    ✓ Study care freeAges 1–6Learn more →
  • Sickle cell disease

    Sub-dissociative Dose Ketamine in Treatment of Vaso-occlusive Pain Event in Children and Young Adults

    Testing whether a low dose of ketamine helps treat sickle cell pain in children and young adults

    📍 Harbor UCLA Medical Center · Torrance, CA · Get directions →

    The purpose of this research is to see if ketamine is effective and safe in treating children and young adults with sickle cell disease experiencing sickle cell related pain. In this study, we will compare the outcomes (such as pain scores) in persons who receive standard of care pain medicine (an opioid such as morphine) plus a low dose (amount) of ketamine to those who receive only standard of care pain medicine.

    ✓ Study care freePhase 2Ages 5–20Learn more →
  • Sickle cell disease

    Comparing the Effectiveness of Matched Related Donor Hematopoietic Stem Cell Transplantation to Disease Modifying Therapy in Pediatric Patients With Sickle Cell Disease

    Comparing outcomes of transplant vs non-transplant treatments in children with sickle cell disease

    📍 Children's of Alabama (MRD-HCT) · Birmingham, AL · +30 more US sites · Get directions →

    The WeDecide study is a large observational study comparing the long-term effects of matched related donor hematopoietic stem cell transplantation (MRD HCT) and non-transplant disease-modifying therapies (NT-DMT) for pediatric patients with sickle cell disease (SCD). The study aims to assess health-related quality of life (HRQoL), cognitive function, risks, and benefits of both treatments, including survival rates, chronic complications, and organ damage prevention.

    ✓ Study care freeAges 3–20Learn more →
  • Sickle cell disease

    Advancing Feasibility and Acceptability of Digital Cognitive Rehabilitation in Sickle Cell Disease

    Testing digital brain training plus support to help youth with sickle cell use it

    📍 Children's National Hospital · Washington D.C., DC · Get directions →

    This is a single site nonrandomized pilot clinical trial of the feasibility, acceptability, and efficacy of a combination treatment involving digital Cogmed working memory skills training, social support components, and memory strategy training to improve adherence to Cogmed in youth with sickle cell disease ages 7-16 years.

    ✓ Study care freeAges 7–16Learn more →
  • Sickle cell disease

    The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease

    Testing rilzabrutinib for safety and effectiveness in sickle-cell disease

    📍 University of Alabama at Birmingham- Site Number : 8400003 · Birmingham, AL · +15 more US sites · Get directions →

    This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group, flexible-adaptive, group-sequential study (Part A), followed by an open-label LTE period (Part B) to investigate the efficacy, and safety of rilzabrutinib in participants with sickle-cell disease (SCD). Study details include: * Study duration: a 52-week double-blind period (Part A), followed by an open-label LTE period (Part B).

    ✓ Study care freePhase 3Ages 10–65Learn more →
  • Sickle cell disease

    Functional Ovarian Reserve in Sickle Cell Disease

    Measuring hormone levels in girls with sickle cell as they grow

    📍 St. Jude Children's Research Hospital · Memphis, TN · Get directions →

    This study aims to look at AMH levels in female children with SCD as they go through puberty to see if they are at the same level as other children without SCD at the same age and/or pubertal stage and will also look at how treatment exposures and pain crises affect the AMH levels in children with SCD. Primary Objective: * To evaluate whether AMH levels are lower in pre-teens and adolescent females with SCD when compared with healthy female controls (siblings, relatives, non-relatives of similar race/ethnicity) at the same age and pubertal stage.

    ✓ Study care freeAges 10–18WomenLearn more →
  • Sickle cell disease

    Mindfulness-Based Intervention for Pain and Sleep in Adolescents and Young Adults With Sickle Cell Disease

    Testing a mindfulness mobile program to help teens and young adults with sickle cell disease manage

    📍 University of Illinois Chicago · Chicago, IL · Get directions →

    This study examines the feasibility and acceptability of a mindfulness-based mobile intervention designed to support pain and sleep management among adolescents and young adults with sickle cell disease. Chronic pain and sleep problems are common in this population and can negatively affect daily functioning and quality of life.

    ✓ Study care freeAges 15–39Learn more →
  • Sickle cell disease

    Impact of Regional Anesthesia on Inflammatory Mechanisms During Vaso-occlusive Crisis in Sickle Cell Patients

    Testing whether regional anesthesia affects inflammation during sickle cell pain crises

    📍 Chu de La Guadeloupe · Pointe-à-Pitre, · Get directions →

    Sickle cell disease is a chronic disease characterized by multiple vaso-occlusive complications. The basic treatment for patients with a vaso-occlusive crisis (VOC) is based on adequate hydration, oxygen therapy and pain control.

    ✓ Study care freeAges 18+Learn more →
  • Sickle cell disease

    Blood Sampling for Research Related to Sickle Cell Disease

    Collecting blood samples to study sickle cell disease in people

    📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →

    This study will collect representative blood samples from healthy children and adults and from children and adults who have unique red blood cell features that are related to sickle cell disease. Sickle cell disease is a blood disease that limits the ability of red blood cells to carry oxygen throughout the body.

    ✓ Study care freeAges 18–100Learn more →
  • Sickle cell disease

    Predictors of Pain in Sickle Cell Disease

    Looking for factors that predict severe, long-lasting pain in sickle cell disease

    📍 Duke University Medical Center · Durham, NC · Get directions →

    Sickle cell disease is a painful inherited disorder that affects approximately 100,000 people in the United States, and more than half of these individuals develop chronic or persistent pain that is often severe and disabling. The factors that predict whether an individual with sickle cell disease will develop severe, disabling pain are unclear.

    ✓ Study care freeAges 15–40Learn more →
  • Sickle cell disease

    A Real-World Registry of Chronic Wounds and Ulcers

    Tracking the real-life course of chronic wounds and ulcers in people

    📍 US Wound Registry · The Woodlands, TX · Get directions →

    WOUNDJOURNEY is a longitudinal, real-world, observational registry designed to capture the full clinical course and patient journey associated with chronic wounds and ulcers. Data are collected during routine care from over 1,400 clinicians across the USA and Puerto Rico using a highly structured certified electronic health record (EHR) or electronic data capture (EDC) system.

    ✓ Study care freeLearn more →
  • Sickle cell disease

    Arginine Therapy for the Treatment of Pain in Children With Sickle Cell Disease

    Testing whether extra arginine lowers pain in children with sickle cell

    📍 Children's Healthcare fo Atlanta at Hughes Spalding · Atlanta, GA · +1 more US site · Get directions →

    The purpose of this study is to determine whether giving extra arginine to patients with sickle cell disease seeking treatment for vaso-occlusive painful events (VOE) will decrease pain scores, decrease need for pain medications or decrease length of hospital stay or emergency department visit.

    ✓ Study care freePhase 1Ages 7–21Learn more →
  • Sickle cell disease

    ATHNdataset Registry

    Studying the quality of life of people with sickle cell and other blood disorders

    📍 American Thrombosis and Hemostasis Network · Hickory, NC · Get directions →

    The Hemophilia Treatment Center (HTC) where you receive care is working with The American Thrombosis and Hemostasis Network (ATHN) to look at the quality of life of people with blood disorders and problems. Doctors, scientists, policymakers, and other health care providers need a large amount of information from a lot of people to answer scientific, public health, and policy questions about better ways to treat blood disorders.

    ✓ Study care freeLearn more →
  • Sickle cell disease

    Virtual Reality Devices as an Adjunct to Usual Care for Patients With Sickle Cell Disease Experiencing Vaso-Occlusive Crises

    Testing virtual reality along with usual care for sickle cell pain crises

    📍 University of Maryland Medical Systems · Baltimore, MD · Get directions →

    This study aims to evaluate the use of virtual reality as an adjunct to standard care for patients with sickle cell disease experiencing vaso-occlusive crises.

    ✓ Study care freeAges 18+Learn more →
  • Sickle cell disease

    A Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell Disease

    Testing a once-daily mouth medicine in teens with sickle cell disease

    📍 The Hospital for Sick Children · Toronto, Ontario · Get directions →

    This study is being done to learn about etavopivat, a once a day medicine taken by mouth in adolescents with sickle cell disease. The main goals are to study safety and how long etavopivat stays in the bloodstream, while also studying if there are benefits from taking etavopivat.

    ✓ Study care freePhase 2Ages 1–18Learn more →
  • Sickle cell disease

    Trans-auricular Nerve Stimulation as an Innovative Approach to the Treatment of Pain in Pediatric Patients Suffering From Sickle Cell Disease

    Testing a device that stimulates nerves through the ear to help treat pain in children with sickle

    📍 Children's Hospital of Atlanta: Eagelston · Atlanta, GA · Get directions →

    The goal of this study is to evaluate the effects of transauricular vagus and trigeminal nerve stimulation on the treatment of pain in children with Sickle Cell Disease. * The primary objective is to determine the feasibility of using a tAN device to treat pain in pediatric participants with SCD by assessing the completion rate, stimulation tolerability, and acceptability of the intervention measures.

    ✓ Study care freeAges 7–18Learn more →
  • Sickle cell disease

    Genotype -Phenotype Correlation of PKLR Variants With Pyruvate Kinase, 2,3-Diphosphglycerate and Adenosine Triphosphate Activities in Red Blood Cells of People With Sickle Cell Disease

    Checking whether differences in the PKLR gene relate to blood changes in people with sickle cell

    📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →

    Background: Some people with the same disorder on a genetic level have more complications than others. Researchers want to look for a link between the PKLR gene and sickle cell disease (SCD) symptoms.

    ✓ Study care freeAges 18–80Learn more →
  • Sickle cell disease

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

    Testing a program to improve self-management in teens with sickle cell

    📍 Nemours Children's Health · Wilmington, DE · +3 more US sites · Get directions →

    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?

    ✓ Study care freeAges 13–21Learn more →
  • Sickle cell disease

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

    Long-term follow-up of people with sickle cell who received BEAM-101

    📍 University of Alabama at Birmingham · Birmingham, AL · +16 more US sites · Get directions →

    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.

    ✓ Study care freeAges 14–37Learn more →
  • Sickle cell disease

    SickleFit Exercise and Nutrition Study

    Testing an exercise and nutrition program for adults with sickle cell disease

    📍 Duke University · Durham, NC · Get directions →

    To goal of this study is to pilot the SickleFit exercise and nutrition intervention in adults with sickle cell disease in a randomized control trial

    ✓ Study care freeAges 35+Learn more →
  • Sickle cell disease

    Metabolic and Hemodynamic Reserve in Pediatric SCA

    Studying how blood flow and metabolism affect brain development in children with sickle cell

    📍 Washington University in St. Louis · St Louis, MO · Get directions →

    The purpose of this research study is to better understand how blood flow and metabolism change can influence brain development in the early decades of life. SCA participants and healthy controls are age and sex-matched for comparison.

    ✓ Study care freeAges 4–21Learn more →
  • Sickle cell disease

    School Readiness Intervention for Preschool Children With Sickle Cell Disease

    Testing a preschool program to help children with sickle cell disease get ready for school

    📍 St. Jude Children's Research Hospital · Memphis, TN · Get directions →

    The study participant is being asked to take part in this clinical trial, a type of research study, because the participant is a young child with sickle cell disease or the caregiver of a child with sickle cell disease. This study is being done to test a school readiness program for children with sickle cell disease (ages 3.5-6,5 years old).

    ✓ Study care freeAges 4–7Learn more →
  • Sickle cell disease

    RH Genotype Matched RBC Transfusions

    Testing whether matching donor red blood cells by RH type helps people with sickle cell who need

    📍 Children's Hospital of Philadelphia · Philadelphia, PA · Get directions →

    To determine the feasibility and efficacy of matching donor red cells by RH genotype for a cohort of chronically transfused patients with SCD.

    ✓ Study care freePhase 1Ages 1+Learn more →
  • Sickle cell disease

    A Stress and Pain Self-management m-Health App for Adult Outpatients With Sickle Cell Disease

    Testing a phone app that teaches stress and pain coping skills for adults with sickle cell disease

    📍 University of Florida · Gainesville, FL · Get directions →

    Our long-term goal is to reduce stress and improve sickle cell disease (SCD) pain control with less opioid use through an intervention with self-management relaxation/distraction exercises (RDE), named You Cope, We Support (YCWS). Americans living with SCD suffer recurrent episodes of acute and chronic pain, both of which are exacerbated by stress.

    ✓ Study care freeAges 18–99Learn more →
  • Sickle cell disease

    Sickle Cell, Pain and Mediterranean Diet

    Comparing pain levels in people with sickle cell disease on a Mediterranean diet vs their usual diet

    📍 University of Illinois Chicago · Chicago, IL · Get directions →

    The goal of this study is to compare pain levels in individuals with Sickle Cell Disease while following the Mediterranean Diet to pain levels while following their usual diet.

    ✓ Study care freeAges 18+Learn more →
  • Sickle cell disease

    Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease

    Testing a one-time blood stem cell transplant using gene editing for severe sickle cell disease

    📍 University of California, Los Angeles · Los Angeles, CA · +1 more US site · Get directions →

    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 severe Sickle Cell Disease (SCD). The study will evaluate the hematopoietic stem cell transplantation (HSCT) using CRISPR/Cas9 edited red blood cells (known as CRISPR\_SCD001 Drug Product).

    ✓ Study care freePhase 1Ages 12–35Learn more →
  • Sickle cell disease

    Integrative Training Program for Pediatric Sickle Cell Pain

    Testing a group training program for teens with sickle cell pain

    📍 Connecticut Children's Medical Center · Hartford, CT · +3 more US sites · Get directions →

    This research aims to answer the question: does a group training program specifically for teens with chronic sickle cell disease (SCD) pain that teaches skills to strengthen the mind and body help improve everyday functioning and reduce pain symptoms? The program will be tailored to address challenges related to frequent or chronic sickle cell pain and may improve participants' physical and emotional health.

    ✓ Study care freeAges 12–18Learn more →
  • Sickle cell disease

    MULTIsite Feasibility of MUSIc Therapy to Address Quality Of Life in Sickle Cell Disease

    Testing whether music therapy can improve quality of life in people with sickle cell disease

    📍 UH Seidman Cancer Center Adult SCD Clinic or UH Rainbow Babies and Children's Hospital Sickle Cell Anemia Center · Cleveland, OH · +1 more US site · Get directions →

    This is a multi-site, multi-visit feasibility RCT of music therapy (MT) among adolescent and adult patients (aged 14 and older) with sickle cell disease (SCD). Subjects will be randomized into one of three groups, either (1) 6 visits of in- person MT (InMT:); (2) 1 visit of in-person MT and 5 visits of virtual MT (HybMT); or (3) 1 visit of in-person health education and 5 visits of virtual health education (HybHE).

    ✓ Study care freeAges 14+Learn more →
  • Sickle cell disease

    Cannabidiol in Sickle Cell Disease

    Testing twice-daily cannabidiol doses for people with sickle cell disease

    📍 Icahn School of Medicine at Mount Sinai · Manhattan, NY · Get directions →

    Randomized, placebo-controlled, double masked, dose finding study of twice daily cannabidiol given at 3 dose levels, 200mg, 400mg, and 600mg, compared to placebo for 4 weeks.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • Kidney diseaseSickle cell disease

    Cooperative Assessment of Late Effects for SCD Curative Therapies

    Studying long-term effects in people with sickle cell disease after curative treatments

    📍 Children's National Medical Center · Washington D.C., DC · +4 more US sites · Get directions →

    Sickle Cell Disease is one of the most common genetic diseases in the United States, occurring in approximately 1 in 400 births. Approximately 100,000 individuals are diagnosed with SCD in the United States.

    ✓ Study care freeAges 4–65Learn more →
  • Sickle cell disease

    Promoting Resilience Among Adolescents and Young Adults With Sickle Cell Disease

    Testing whether a care program can help teens and young adults with sickle cell build resilience

    📍 Boston Children's Hospital · Boston, MA · Get directions →

    Adolescents and young adults with sickle cell disease (SCD) face challenges managing their illness and maintaining their well-being. This study proposes to test the feasibility and acceptability of a resilience-promoting intervention through a Collaborative Care Model.

    ✓ Study care freeAges 8–25Learn more →
  • Sickle cell disease

    Observational Study to Deeply Phenotype Major Organs in Sickle Cell Disease After Curative Therapies

    Studying how major organs in sickle cell disease change after curative treatments

    📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →

    Background: People with sickle cell disease (SCD) have problems with their heart, brain, kidneys, liver, and lungs as they age. These problems may improve after transplant.

    ✓ Study care freeAges 18–100Learn more →
  • Sickle cell disease

    Early Screening and Treatment of Heart Complication in Sickle Cell Disease

    Testing early heart screening and iron overload treatment in people with sickle cell disease

    📍 Inova Schar Cancer · Fairfax, VA · +1 more US site · Get directions →

    This study tests whether early heart screening and treatment for iron overload in subjects with sickle cell disease can prevent heart problems and reduce hospitalizations.

    ✓ Study care freePhase 2Ages 18+Learn more →
  • Sickle cell disease

    Prophylactic Transfusion In Pregnant in Women With Sickle Cell Disease

    Testing whether routine red blood cell transfusions in pregnancy reduce sickle cell flare-ups or

    📍 Grady Health System · Atlanta, GA · Get directions →

    The goal of this study is to determine if there is a positive effect of prophylactic red blood cell (RBC) transfusion of leukoreduced, ABO, Rh (D/Cc/Ee) and Kell matched blood compared to standard of care on the number of episodes of acute sickle cell disease (SCD) manifestations or pregnancy-related complications requiring acute health care encounters (acute care/ER/Hospital visits) or resulting in death over the entirety of pregnancy until 2 months post-partum in women with SCD. RBC transfusion is the only disease-modifying therapy for pregnant women with SCD, and it is considered a standard treatment option however, there exists no consensus on the role of transfusion therapy in preventing SCD-related pregnancy complications.

    ✓ Study care freePhase 4Ages 18+WomenLearn more →
  • Sickle cell disease

    Zinc Supplementation in Sickle Cell Disease: A Precursor to the Think Zinc for Bones Trial

    Testing two daily doses of zinc in people with sickle cell disease to see changes in bone-related

    📍 UCSF Benioff Children's Hospital Oakland · Oakland, CA · +5 more US sites · Get directions →

    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 15-40 years old. The main question it aims to answer is: Which biomarkers are most responsive to zinc supplementation, and what is the maximum tolerated zinc dose that induces the desired changes in biomarkers of bone turnover?

    ✓ Study care freePhase 2Ages 15–40Learn more →
  • Sickle cell disease

    Gene Editing For Sickle Cell Disease

    Testing whether gene editing is safe for people with sickle cell disease

    📍 St. Jude Children's Research Hospital · Memphis, TN · Get directions →

    This study is being done to test the safety of a new treatment called gene editing in Sickle Cell Disease (SCD) patients and to see if a single dose of this genetically modified cellular product will increase the amount of a certain hemoglobin called fetal hemoglobin (HbF) and help reduce the symptoms of SCD. Primary Objective * To assess the safety of autologous infusion of clustered regularly interspaced palindromic repeats (CRISPR)/ CRISPR associated protein (Cas9)-edited CD34+ hematopoietic stem and progenitor cells (HSPCs) in patients with severe SCD.

    ✓ Study care freePhase 1Ages 18–24Learn more →
  • Sickle cell disease

    Allo HSCT for High Risk Hemoglobinopathies

    Testing donor stem cell transplant for people with high-risk sickle cell

    📍 Masonic Cancer Center · Minneapolis, MN · Get directions →

    A single center, open label, interventional, phase II trial for donor transplant for high risk hemoglobinopathies and other red cell transfusion dependent disorders utilizing allogeneic hematopoietic stem cell transplantation (HSCT) regimens.

    ✓ Study care freePhase 2Learn more →
  • Sickle cell disease

    Recruitment and Engagement in Care to Impact Practice Enhancement (RECIPE) for Sickle Cell Disease

    Testing ways to reach people with sickle cell disease not in specialty care

    📍 University of Alabama, Birmingham (UAB) · Birmingham, AL · +8 more US sites · Get directions →

    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 specialist care use three distinct pathways?

    ✓ Study care freeAges 18+Learn more →
  • Sickle cell disease

    Study of a Single Dose of a 21-valent Pneumococcal Conjugate Vaccine in Children and Adolescents With Sickle Cell Disease

    Testing a single pneumococcal vaccine dose in children with sickle cell disease

    📍 Site # 8400006 · Atlanta, GA · +4 more US sites · Get directions →

    The purpose of this study is to measure whether PCV21 vaccine (investigational pneumococcal vaccine) is safe and can help the body to develop germ-fighting agents called "antibodies" (immunogenicity) compared with 20vPCV (licensed pneumococcal vaccine) when given as a single dose to children aged 2 to 17 years with sickle cell disease who had received or not a previous vaccination with pneumococcal conjugate or pneumococcal polysaccharide vaccine.

    ✓ Study care freePhase 3Ages 2–17Learn more →
  • Sickle cell disease

    Applying Directly Observed Therapy to Hydroxyurea to Realize Effectiveness

    Testing video check-ins to help people stay on hydroxyurea for sickle cell

    📍 Lurie Children's Hospital · Chicago, IL · +2 more US sites · Get directions →

    This study is for caregivers of young children with sickle cell disease and adolescents with sickle cell disease who are currently prescribed hydroxyurea and are receiving care at one of the study sites. The study will assess retention and engagement during a pilot randomized control trial comparing video directly observed therapy (VDOT) to attention control.

    ✓ Study care freeAges 11+Learn more →
  • Sickle cell disease

    Exercise in Child Health

    Testing exercise approaches in children with sickle cell disease

    📍 University of California, Irvine · Irvine, CA · Get directions →

    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 Lurie Children's Hospital), and 3) USC (with its affiliate Children's Hospital of Los Angeles).

    ✓ Study care freeAges 10–17Learn more →
  • Sickle cell disease

    Molecular and Cellular Basis of Severe Forms of Dengue in Sickle Cell Patients

    Studying why severe dengue happens more in people with sickle cell

    📍 Chu de La Guadeloupe · Pointe-à-Pitre, · Get directions →

    Dengue virus (DENV) belongs to the genus of Flavivirus transmitted by the mosquito Aedes aegypti and is responsible for an infectious disease associated with different forms and severities such as dengue hemorrhagic fever or shock syndrome. Several recent reports have shown that sickle cell patients exhibited an increased risk of developing severe forms of dengue episodes compared to non-sickle cell subjects.

    ✓ Study care freeAges 6–25Learn more →
  • Sickle cell disease

    Peripheral Arterial Tonometry and Neurocognition in Sickle Cell Disease

    Testing how sleep problems in teens and young adults with sickle cell affect sleep quality using a

    📍 St. Jude Children's Research Hospital · Memphis, TN · Get directions →

    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.

    ✓ Study care freeAges 12–25Learn more →
  • Sickle cell disease

    A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)

    Testing immune-suppressing treatment before stem cell transplant for people with sickle cell disease

    📍 Memorial Sloan Kettering at Basking Ridge (Consent only) · Basking Ridge, NJ · +5 more US sites · Get directions →

    Hematopoietic Cell Transplantation/HCT involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in participants' bone marrow. The researchers think giving participants treatment with fludarabine and dexamethasone, drugs that lower the activity of the body's immune system (immune suppression), before standard conditioning therapy and HCT may help prevent serious side effects, including graft failure and GvHD.

    ✓ Study care freePhase 2Ages 2–50Learn more →
  • Sickle cell disease

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

    Testing long-term treatment with a new medicine for sickle cell or thalassaemia

    📍 Univ of Alabama Birmingham · Birmingham, AL · +38 more US sites · Get directions →

    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.

    ✓ Study care freePhase 3Ages 2+Learn more →
  • Sickle cell disease

    A Study to Evaluate BMS-986470 in Healthy Volunteers and Participants With Sickle Cell Disease

    Testing how BMS-986470 affects the body and safety in sickle cell disease

    📍 University of Alabama at Birmingham · Birmingham, AL · +18 more US sites · Get directions →

    The purpose of this study is to evaluate the safety and tolerability, pharmacokinetics and pharmacodynamics, pH and food effect, and preliminary efficacy of BMS-986470 in healthy volunteers and participants with sickle cell disease.

    ✓ Study care freePhase 1Ages 18+Learn more →
  • Sickle cell disease

    Determination of Red Cell Survival in Sickle Cell Disease and Other Hemoglobinopathies Using Biotin Labeling

    Measuring how long red blood cells last in sickle cell disease

    📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →

    Background: Sickle cell disease (SCD) is an inherited disorder of the blood. SCD causes red blood cells (RBCs) to die early.

    ✓ Study care freeEarly Phase 1Ages 18–100Learn more →
  • Sickle cell disease

    Study to Understand the Genetic Risk of Developing an Immune Response After Blood Transfusions Among Individuals With Sickle Cell Disease

    Studying how genes may affect immune reactions to blood transfusions in people with sickle cell

    📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →

    The purpose of this research study is to look at genes and determine how they interact with each other to find changes that could explain why some people's immune systems may respond to blood transfusions. This response is called an alloimmune response.

    ✓ Study care freeAges 2–99Learn more →
  • Sickle cell disease

    Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors

    Testing a partial stem cell transplant from matched donors for severe sickle cell disease or

    📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →

    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.

    ✓ Study care freePhase 1Ages 4–65Learn more →
  • Sickle cell disease

    Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease

    Testing how safe escalating doses of fostamatinib are in people with stable sickle cell disease

    📍 National Institutes of Health Clinical Center · Bethesda, MD · Get directions →

    Background: Sickle cell disease (SCD) is a genetic disease that causes the body to produce abnormal ( sickled ) red blood cells. SCD can cause anemia and life-threatening complications in the lungs, heart, kidney, and nerves.

    ✓ Study care freePhase 1Ages 18–65Learn more →
  • Sickle cell disease

    Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies

    Testing gene editing in stem cells for severe sickle cell disease

    📍 Boston Children's Hospital · Boston, MA · Get directions →

    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.

    ✓ Study care freePhase 1Ages 13–40Learn more →
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