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195 active trials for Sickle Cell Disease

Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases

This is a Phase II study for the use of T-cell replete reduced intensity conditioning (RIC) haploidentical donor allogeneic hematopoietic cell transplantation (HaploHCT) for individuals with high-risk non-malignant diseases who lack a suitable HLA-matched sibling donor.

Start: July 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

Study to Evaluate the Effect of GBT440 on TCD in Pediatrics With Sickle Cell Disease

This study is a Phase 3, randomized, double-blind, placebo-controlled study of voxelotor in pediatric participants, aged ? 2 to < 15 years old, with Sickle Cell Disease. The primary objective is to evaluate the effect of voxelotor on the TCD (Transcranial Doppler Ultrasound) measurements in SCD participants in this age range.

Start: April 2021

Peripheral Blood Stem Cell Collection From Patients With Sickle Cell Disease (SCD) Using Plerixafor

With recent advances in gene editing, gene therapy is becoming a viable curative treatment option for sickle cell disease. In order to do genetic manipulation, investigators need to collect hematopoietic stem cells from patients with sickle cell disease. In this study, investigators want to study the safety and feasibility of collecting peripheral blood stem cells from pediatric and young adult patients with sickle cell disease after administering plerixafor. Studying these peripheral blood stem cells will help in optimizing the yield of peripheral CD34+ cells from pediatric and young adult patients with sickle cell disease, which in turn will help to develop better gene therapies for these patients. Primary Objectives Determine the safety profile associated with administration of plerixafor in pediatric and young adult patients with sickle cell disease (SCD). To estimate the number of CD34+ cells/kg of body weight that can be collected with peripheral apheresis after administration of plerixafor in pediatric and young adult patients with SCD. Exploratory Objectives To describe the kinetics of CD34+ cell mobilization in peripheral blood after - + cells obtained from pediatric and young adult patients with SCD. To study the effect of hydroxyurea therapy on senescence in plerixafor-mobilized CD34+ cells obtained from pediatric and young adult patients with SCD.

Start: June 2021

Study of Safety and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Sickle Cell Disease (SCD)

This study will evaluate two genome-edited, autologous, hematopoietic stem and progenitor cell (HSPC) products - OTQ923 and HIX763 - each reducing the biologic activity of BCL11A, increasing fetal hemoglobin (HbF) and reducing complications of sickle cell disease.

Start: August 2020

A Study of FT-4202 in Adults and Adolescents With Sickle Cell Disease (HIBISCUS)

This clinical trial is a Phase 2/3 study that will evaluate the efficacy and safety of FT-4202 and test how well FT-4202 works compared to placebo to improve the amount of hemoglobin in the blood and to reduce the number of vaso-occlusive crises (times when the blood vessels become blocked and cause pain).

Start: March 2021

Clinical Transplant-Related Long-term Outcomes of Alternative Donor Allogeneic Transplantation

The purpose of this study is to determine if a search strategy of searching for an HLA-matched unrelated donor for allogeneic transplantation if possible then an alternative donor if an HLA-matched unrelated donor is not available versus proceeding directly to an alternative donor transplant will result in better survival for allogeneic transplant recipients within 2 years after study enrollment.

Start: June 2019

Best Noninvasive Predictor of Renal Function in Assessing Adult Sickle Nephropathy

Background: Sickle cell disease is a common inherited blood disorder. Kidney disease is a major cause of problems in people with sickle cell disease. In order to identify kidney problems early and stop the progression of kidney disease, doctors need the most accurate tests to check kidney function. Researchers hope to understand more about how to test for kidney disease in people with sickle cell disease. Objective: To determine which of two different lab tests is the best to measure kidney function in adults with sickle cell disease. Eligibility: People 18 years and older who have sickle cell disease Design: Participants will be screened with a medical history and blood tests. Participants will have up to 3 visits. Participants will collect their urine in a special container over 24 hours. At the first visit, participants will have blood tests. They will bring their container of urine to the visit. They will have an iothalamate test. For the test, they will get a catheter: a small tube will be inserted into a vein. A special contract agent will be injected into the vein. Blood will be collected over the next 4 hours to test kidney function. Participants will return the next day for a second visit. They will have blood tests. They will have an MRI. For the MRI, they will like on a table that slides into a machine that takes pictures of the kidneys. They may have the MRI in a third visit. ...

Start: May 2019

Pediatric Open-Label Extension of Voxelotor

Open-label extension study of voxelotor for pediatric participants ages 4 to 18 years old with Sickle Cell Disease who have participated in voxelotor clinical trials.

Start: November 2019

Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell Disease

This study consists of four parts, Parts A, B, C, and D. Part A is a single dose pharmacokinetic (PK) study in pediatric participants with Sickle Cell Disease. Part B is a multiple dose, safety, exploratory, efficacy, and PK study in adolescent Sickle Cell Disease participants who were 12-17 years of age. Part C is a multiple dose, safety, tolerability, and PK study, which includes the assessment of hematological effects and the effect on TCD flow velocity of voxelotor in pediatric participants with Sickle Cell Disease who are 4 to 17 years of age. Part D is a multiple dose, safety, tolerability, and PK study, which will examine the hematological effects of voxelotor in pediatric participants with Sickle Cell Disease who are between 9 months to < 4 years of age.

Start: May 2016

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

The long-term goal is to reduce stress and improve pain control with less opioid use in patients with sickle cell disease by promoting an intervention with self-management relaxation/distraction exercises, named You Cope, We Support (YCWS).

Start: April 2021

Cerebrovascular Reserve and White Matter Disease in Patients With Chronic Anemia

This is primarily an observational trial in patients with chronic anemia syndromes (sickle cell disease and thalassemia) and control subjects. The key purpose is to understand how brain blood flow reserve (the ability of the brain to increase its flow in response to stress) is altered in patients with chronic anemia. Since this parameter may depend on anemia severity, we will perform the MRI monitoring prior to and following clinically indicated transfusions in a subset of patients. Most patients will already be prescribed hydroxyurea as part of their standard of care. Since hydroxyurea could impact brain blood flow, there is also a small pilot study (20 patients, nonrandomized, open label) where MRI imaging will be performed prior to and following administration of hydroxyurea up to maximum tolerated dose. The study will enroll 90 adult subjects with transfusion independent sickle cell disease (70 SS, 10 SC, 10 S?0) and 60 patients with transfusion-dependent sickle cell disease. It will also include 10 transfusion independent thalassemia patients and 20 transfusion dependent thalassemia patients as well as 40 control subjects recruited from first degree relatives of the sickle cell disease population. All eligible subjects will be asked to provide informed consent before participating in the study.

Start: July 2018

A Long-term Follow-up Study in Subjects Who Received CTX001

This is a multi-site, observational study to evaluate the long-term safety and efficacy of CTX001 in subjects who received CTX001 in Study CTX001-111 (NCT03655678) or Study CTX001-121 (NCT03745287).

Start: January 2021

Non-Myeloablative Conditioning and Bone Marrow Transplantation

Allogeneic blood or marrow transplantation (alloBMT) is a curative therapy for a variety of hematologic disorders, including sickle cell disease and thalassemia. Even when it is clear that alloBMT can give to these patients an improvement in their disease, myeloablative transplants have important toxicities and mortalities associated. The lack of suitable donors continues to be a limit to access to transplantation. Substantial progress has been made recently in the development of pre-treatment regimens that facilitate the sustained engraftment of donor marrow with reduced toxicity. Most of these regimens incorporate highly immunosuppressive drugs, which allow the reduction or elimination of myeloablative agents or total body irradiation without endangering the sustained engraftment of HLA-identical allogeneic stem cells. Preliminary results of non-myeloablative allogeneic stem cell transplantation suggest that the procedure can be performed in patients who are ineligible for myeloablative alloBMT, and that sustained remissions of several hematologic malignancies can be obtained.

Start: May 2013

Reduced Intensity Transplantation for Severe Sickle Cell Disease

This study is being done to test a transplant method that may have fewer side effects (or less toxic, less harmful) than conventional high dose chemotherapy conditioning-based transplants for children and young adults with Sickle Cell Disease (SCD). Patients less than or equal to 25 years old with SCD who would likely benefit from allogeneic hematopoietic cell transplantation (HCT) will be included in this study. Patients with a suitable HLA matched sibling donor (MSD) will be enrolled on the MSD arm while patients without an eligible MSD who have a suitable haploidentical (HAPLO) donor available will be enrolled on the HAPLO arm of the study. Primary Objective To assess the donor T-cell chimerism at 1-year post transplant in each respective arm (MSD, HAPLO) of the trial. Secondary Objectives Assess the overall survival and 1-year, 2-year and 3-year post-transplant graft versus host disease (GVHD)-free SCD-free survival. Estimate the primary and secondary graft rejection rate at 1-year, 2-year and 3-year post- transplant. Estimate the incidence and severity of acute and chronic (GVHD). Estimate the incidence of SCD recurrence after transplant Assess the neutrophil and platelet recovery kinetics post-transplant. Exploratory Objectives Record immune reconstitution parameters, including chimerism analysis, quantitative lymphocyte subsets, T cell receptor excision circle (TREC) analysis, V-beta spectratyping, and lymphocyte phenotype and function. Conduct longitudinal examination of impact of HCT on patient health-related quality of life (HRQL) and adjustment, and parental adjustment. Examine impact of HCT on patient cognitive and academic function. Determine factors that influenced the decision to undergo HCT, explore perceptions of the HCT experience, and assess decisional satisfaction/regret. Develop and evaluate an objective/quantitative imaging biomarker to assess organ (liver and heart) function/disease status and changes following HCT. Develop and evaluate an objective/quantitative imaging biomarker to determine cerebral blood flow and oxygen extraction fraction following HCT.

Start: April 2020

A Study of SHP655 (rADAMTS13) in Sickle Cell Disease

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and efficacy of SHP655 in participants with baseline health sickle cell disease (SCD) and SCD with acute vaso-occlusive crisis (VOC).

Start: October 2019

Collection of Human Biospecimens for Basic and Clinical Research Into Alpha Globin Variants

Background: Blood disorders like sickle cell disease and malaria affect many people around the world. Researchers want to learn more about blood disorders. To do this, they need to collect biological samples from people with blood disorders. They also need to collect samples from healthy people. Objective: To collect samples to use for research on blood disorders. Eligibility: People ages 18-70 who have blood disorders. Healthy volunteers without blood disorders are also needed. Design: Participants will be screened with a medical history, physical exam, and blood and urine tests. Participants will give one or more samples. They will give them over 5 years. They can choose not to give any of the samples: Saliva: Participants will spit into a tube. They may also have the inside of their mouth swabbed. Urine: Participants will urinate into a cup. Blood: Blood will be taken through a needle in the participant s arm. Fat samples: An area on the participant s belly or buttock will be numbed. A small cut will be made into the skin and a small piece of fat removed. Mucus and cells from the lungs: The participant will be sedated. A flexible tube will be inserted through the nose or mouth into the lung airways. These participants will also have a physical exam, chest x-ray, and heart tests after the procedure. ...

Start: September 2019

Red Blood Cell Survival in Sickle Cell Disease

This is a single-arm, mechanistic clinical trial to measure predictors of senescence and the in vivo survival of transfused red blood cells (RBCs) in individuals with sickle cell disease (SCD) receiving chronic transfusion therapy (CTT). Chronic transfusion in patients with SCD is a common treatment. The efficacy of RBC transfusion therapy to treat or prevent complications of SCD may be hampered by variable survival of the transfused donor RBC. The overall aim is to see how long RBC survive in SCD patients who are chronically transfused. When a study participant has a regular blood transfusion the researchers will label a small portion of the RBCs that are transfused with biotin. The participant will return at Day 1, weekly for 3 months and monthly for 3 months to measure how long those RBCs survive.

Start: August 2021

Integration of mHEALTH Into the Care of Patients With Sickle Cell Disease to Increase Hydroxyurea Utilization

This project proposes to develop, test and evaluate targeted interventions to improve clinical provider prescribing of and patient adherence to hydroxyurea (HU). Using a stepped-wedge design, The investigators will test two innovative interventions utilizing mobile health to address both patients' and providers' needs: 1) an mHealth application for patients (InCharge Health app) that includes multi-component features to address the memory, motivation, and knowledge barriers to hydroxyurea use, and 2) an mHealth toolbox application for providers (HU Toolbox app) that addresses clinical knowledge barriers in prescribing and monitoring hydroxyurea use. These two interventions will be tested through the following aims: Aim 1. Improve Patient Adherence to Hydroxyurea: Addressing Memory, Motivation, and Knowledge Barriers to Hydroxyurea Use. Primary hypothesis: The investigators hypothesize that among adolescents and adults with SCD, the adherence to hydroxyurea, as measured by proportion of daily coverage (PDC), will increase by at least 20% at 24 weeks after receiving the InCharge Health app, compared to their hydroxyurea adherence at baseline. Sub-aim 1.a. To examine and assess both patient engagement and behaviors related to use of the InCharge Health app, the investigators will evaluate consistent use of the app among enrolled patients, patient satisfaction, and continued use of the app beyond the study period. Sub-Aim 1.b. To examine the clinical influence of the use of the InCharge Health app on PDC, patients' clinical outcomes, perceived health literacy, health related quality of life, and perceived self-efficacy between baseline and 24 weeks. Aim 2. Improve Provider Hydroxyurea Awareness, Prescribing and Monitoring Behaviors. Sub-Aim 2.a. To examine and assess provider engagement and behaviors related to use of the HU Toolbox, the investigators will evaluate consistent use of the app among enrolled providers, providers' satisfaction, and continued use of the app beyond the study period. Sub-Aim 2.b. To assess the combined effects of the patient and provider mHealth interventions on hydroxyurea and health care utilization, the investigators will examine if the changes in hydroxyurea adherence are enhanced by the use of both provider and patient interventions compared to those not exposed to one or both interventions. Aim 3. Identify and Evaluate the Barriers and Facilitators to the use of mHealth Interventions.

Start: November 2019

Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Escalating Multiple Oral Doses of AG-348 in Subjects With Stable Sickle Cell Disease

Background: Sickle Cell Disease (SCD) is an inherited blood disorder. People with SCD have abnormal hemoglobin in their red blood cells. Researchers are investigating the safety and efficacy of an investigational medicine called AG-348 (mitapivat sulfate) to determine if it will help people with SCD. Objective: To test the tolerability and safety of AG-348 in people with SCD. Eligibility: People ages 18 and older with SCD. Design: Participants will have 8 visits over approximately 14 weeks. At the first visit participants will be screened with a medical history; a physical exam; and blood, urine, and heart tests. At the following 5 visits participants will stay at the clinic for 1 night each. Participants will take study drug in increasing doses upto visit 6, after which the drug will be tapered off. All visits will include physical exam, blood, and urine tests. The last visit will occur 4 weeks after stopping the drug and also includes a heart test. Participants will provide DNA from the blood samples they provide. The DNA will be tested for an inherited gene that can cause differences in response to the study drug. Researchers may also test other genes to see if they can find any genes that interact with SCD.

Start: July 2019