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195 active trials for Sickle Cell Disease

A Voxelotor for Sickle Cell Anemia Patients at Highest Risk for Progression of Chronic Kidney Disease

This study is a single center, prospective exploratory pilot study of Sickle Cell Anemia (SCA) participants. The study will enroll patients with early stages of sickle cell nephropathy (Chronic Kidney Disease (CKD) stage 1 or 2) who are at the highest risk of CKD progression (presence of both hemoglobinuria and urine albumin concentration ? 30 mg/g creatinin

Start: March 2021

Haploidentical Transplantation With Pre-Transplant Immunosuppressive Therapy for Patients With Sickle Cell Disease

This is a study to evaluate the safety and toxicity of a treatment regimen consisting of 2 cycles of pre-transplant immunosuppressive therapy followed by myeloablative preparative regimen and allogeneic hematopoietic stem cell transplantation from a haploidentical donor in patients with sickle cell disease. The overall goal of this study is to expand the donor pool for hematopoietic stem cell transplantation in sickle cell disease using haploidentical donors, and to develop a non-toxic, myeloablative regimen, with the goal of achieving a consistent donor chimerism utilizing pre-transplant immunosuppressive therapy.

Start: February 2018

A Study Evaluating the Safety and Efficacy of bb1111 in Severe Sickle Cell Disease

This is a non-randomized, open label, multi-site, single dose, Phase 1/2 study in approximately 50 adults and adolescents with severe SCD. The study will evaluate hematopoietic stem cell (HSC) transplantation (HSCT) using bb1111 (also known as LentiGlobin BB305 Drug Product for SCD).

Start: August 2014

A Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease

This is a non-randomized, open-label, multi-site, single-dose, Phase 3 study in approximately 35 adults and pediatric subjects ?2 and ?50 years of age with sickle cell disease (SCD). The study will evaluate hematopoietic stem cell (HSC) transplantation (HSCT) with LentiGlobin BB305 Drug Product for SCD.

Start: February 2020

Intravenous Gammaglobulin for Sickle Cell Pain Crises

The purpose of this study is to determine whether intravenous immune globulin is safe and effective in the acute treatment of pain crises in sickle cell disease. Funding Source: Food and Drug Administration (FDA), Office of Orphan Products Development (OOPD)

Start: November 2008

iPeer2Peer Program for Youth With Sickle Cell Disease

The iPeer2Peer Sickle Cell Disease (SCD) study matches youth (12-18 years of age) with SCD to a mentor (trained young adult) who has learned to manage their SCD well, transitioned to adult care, and can support youth participants emotionally and socially. Participants will be randomly assigned one of two groups, either (1) The intervention group: Study group participants are matched with a mentor for 15 weeks, and are expected to have up to ten calls with one another; (2) The control group: This study group will be on a 15 week waitlist to receive a mentor. This study will first assess the feasibility of conducting this research with youth with SCD. Also, this study will assess the preliminary effectiveness of peer mentorship by comparing various health outcomes of the two study groups post-intervention.

Start: January 2020

Fitness Trial in Adults With Sickle Cell Disease (SCD Fit): A Feasibility Study

The purpose of this project is to develop novel approaches to promote health and longevity while enhancing quality of life among persons with Sickle cell disease (SCD). Therefore, investigators are aiming to adapt an evidence-based exercise intervention for adults with SCD informed by culturally- relevant and biologic factors and pre-test the adapted exercise program in a small sample of adults with SCD.

Start: August 2021

Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure

Background: - Some sickle cell disease or beta-thalassemia can be cured with transplant. Researchers want to test a variation of transplant that uses low dose radiation and a combination of immunosuppressive drugs. They want to know if it helps a body to better accept donor stem cells. Objectives: - To see if low dose radiation (300 rads), oral cyclophosphamide, pentostatin, and sirolimus help a body to better accept donor stem cells. Eligibility: - People 4 and older with beta-thalassemia or sickle cell disease that can be cured with transplant, and their donors. Design: Participants and donors will be screened with medical history, physical exam, blood test, tissue and blood typing, and bone marrow sampling. They will visit a social worker. Donors: may receive an intravenous (IV) tube in their groin vein. will receive a drug injection daily for 5 or 6 days to move the blood stem cells from the bone marrow into general blood circulation. will undergo apheresis: an IV is put into a vein in each arm. Blood is taken from one arm, a machine removes the white blood cells that contain blood stem cells, and the rest is returned through the other arm. Participants: may undergo red cell exchange procedure. will remain in the hospital for about 30 days. will receive a large IV line that can stay in their body from transplant through recovery. will receive a dose of radiation, and transplant related drugs by mouth or IV. will receive blood stem cells over 8 hours by IV. will take neuropsychological tests and may complete questionnaires throughout the transplant process. must stay near NIH for 4 months. They will visit the outpatient clinic weekly. will have 5 follow-up visits for 3 years after transplant, then annually.

Start: April 2014

EDIT-301 for Autologous HSCT in Subjects With Severe Sickle Cell Disease

The purpose of this study is to evaluate the efficacy, safety and tolerability of treatment with EDIT-301 in adult subjects with severe sickle cell disease (SCD).

Start: April 2021

LCI-HEM-SCD-ST3P-UP-001: The Sickle Cell Trevor Thompson Transition Project (ST3P-UP Study)

This multi-center study will compare the effectiveness of adding virtual peer mentoring (PM) to a structured education-based (STE) transition program for emerging adults with sickle cell disease to determine its effect on decreasing the number of acute care visits per year, improving patient-reported outcomes, and reducing healthcare utilization among emerging adults with sickle cell disease (EA-SCD)

Start: January 2018

Living With Sickle Cell Disease in the COVID-19 Pandemic

Background: Sickle cell disease (SCD) is a chronic illness. It affects about 100,000 people in the United States. People with SCD have red blood cells that are sickle-shaped and impaired in their function. This results in a lifetime of complications that affect every organ system. People with SCD also are at greater risk for respiratory infections and lung problems. Researchers want to study how this population s stress, anxiety, fear, pain, sleep, and health care use are being affected by the COVID-19 pandemic. Objective: To study the extent and impact of life changes induced by the COVID-19 pandemic on people living with SCD in the U.S. Eligibility: People age 18 and older with SCD who live in the U.S. Design: Participants will complete a survey online. The questions will focus on the following: Medical history Mental and physical health Demographics Stress Resilience Health care use COVID-19 Beliefs about medical mistrust and participation in research. At the end of the survey, participants will be asked if they would like to take the survey again in the future. If they reply "yes," then they will be contacted by the study team in 6-9 months to take the survey again. They may complete the survey again in 6-8 months, 12-15 months, and 18-21 months. The survey should take less than 40 minutes to complete. Participants' data will be coded to protect their privacy. The coded data may be shared with other researchers.

Start: June 2020

Safety of Single Ascending Doses of CSL889 in Adult Patients With Stable Sickle Cell Disease

This is a phase 1, first-in-human, multi-center, open-label, single ascending dose (SAD) cohort study to evaluate the safety and tolerability, pharmacokinetics (PK), exploratory pharmacodynamics (PD), and biomarkers of target engagement of CSL889 following single intravenous (IV) doses in subjects with stable sickle cell disease (SCD). The study involves sequential dose escalation of cohorts with between-group assessments of key safety and PK variables.

Start: May 2021

SMYLS: A Self-management Program for Youth Living With Sickle Cell Disease

The purpose of this study is to find out whether a web-based intervention using a mobile device is helpful for teens learning to care for and manage symptoms of sickle cell disease. The intervention lasts 12 weeks with a 3-month follow up period, and uses a smartphone or a tablet.

Start: July 2020

Nonmyeloablative Stem Cell Transplant in Children With Sickle Cell Disease and a Major ABO-Incompatible Matched Sibling Donor

The aim of this study to evaluate the safety and efficacy of a nonmyeloablative conditioning regimen for allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric patients with sickle cell disease (SCD) who have a matched related major ABO-incompatible donor. The nonmyeloablative regimen will use alemtuzumab, total body irradiation (TBI) and sirolimus for immune suppression. This study will expand the access of HSCT for patients with SCD who are currently not eligible because of donor restrictions.

Start: July 2017

The BENeFiTS Trial in Beta Thalassemia Intermedia

Beta-thalassemias and hemoglobinopathies are serious inherited blood diseases caused by abnormal or deficiency of beta A chains of hemoglobin, the protein in red blood cells which delivers oxygen throughout the body.The diseases are characterized by hemolytic anemia, organ damage, and early mortality without treatment. Increases in another type of (normal) hemoglobin, fetal globin (HbF), which is normally silenced in infancy, reduces anemia and morbidity. Even incremental augmentation of fetal globin is established to reduce red blood cell pathology, anemia, certain complications, and to improve survival. This trial will evaluate an oral drug discovered in a high throughput screen, which increases fetal globin protein (HbF and red blood cells expressing HbF)and messenger ribonucleic acid (mRNA) to high levels in anemic nonhuman primates and in transgenic mice. The study drug acts by suppressing 4 repressors of the fetal globin gene promoter in progenitor cells from patients. The drug has been used for 50 years in a combination product for different actions - to enhance half-life and reduce side effects of a different active drug- and is considered safe for long-term use. This trial will first evaluate 3 dose levels in small cohorts of nontransfused patients with beta thalassemia intermedia. The most active dose will then be evaluated in larger subject groups with beta thalassemia and other hemoglobinopathies, such as sickle cell disease.

Start: October 2020

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

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

Start: January 2012

Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease

The goal of this clinical research study is to find out about the safety and effects of a drug called panobinostat when given to adults with sickle cell disease. Panobinostat is a pan histone deacetylase (HDAC) inhibitor. HDAC inhibitors have been shown to significantly increase hemoglobin F induction, which is well documented to improve outcomes in sickle cell disease. HDAC inhibitors are also known to potently inhibit cell-specific inflammation, which is a primary contributor to the debilitating effects of sickle cell disease. Given the relevance of these mechanisms of action in SCD, panobinostat may prove to contribute significantly to the management of SCD patients, a population in critical need of further effective treatment options.

Start: November 2010

Neurovascular Determinants of Cognitive Function in Adults With Sickle Cell Disease

Cognitive impairment is a poorly understood, serious, and emerging complication for adult patients with sickle cell disease. Because there is extensive microvascular damage from oxidative damage in sickle cell disease, the investigators hypothesize that this is also present in the cerebral microvasculature to cause cognitive impairment. The investigators plan to test this by correlating markers of inflammation and oxidative damage with cognitive performance and 7 Tesla brain MRI microvascular findings in these patients, with the long term goal of understanding the mechanisms and risk factors of cognitive impairment in sickle cell disease.

Start: April 2016

Study to Assess the Effect of Long-term Treatment With Voxelotor in Participants Who Have Completed Treatment in Study GBT440-031

Open Label Extension Study of Voxelotor Clinical Trial Participants with Sickle Cell Disease Who Participated in Voxelotor Clinical Trials

Start: June 2018

Sickle Cell Disease and the Genomic and Gene Therapy Needs of Stakeholders

The primary objectives of this prospective mixed-method interview study are to use semi-structured interviews in parents of sickle cell disease (SCD) patients to describe parental attitudes of research involving genomic sequencing, including concerns about participation and expectations from researchers and to use surveys to quantitatively measure genetic/genomic knowledge, trust in health care provider, and literacy/numeracy ability in parents of children with SCD and adolescents with SCD. Investigators hope to use the results of the planned surveys and interviews to reduce the risk of misunderstanding about DNA and genetic research and build strong relationships between SCD families and researchers in the future, and to design educational information and study materials that will help parents with children with SCD understand important details about DNA and genetic research.

Start: December 2020