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Sickle Cell Disease clinical trials

View clinical trials related to Sickle Cell Disease.

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NCT ID: NCT05228821 Withdrawn - Sickle Cell Disease Clinical Trials

Voxelotor Brain Oxygenation and Neurocognitive Study

Start date: August 9, 2023
Phase: Phase 4
Study type: Interventional

This is an open label, single arm multicenter trial to evaluate the effect of voxelotor treatment on cerebral blood flow (CBF) and neurocognitive function in adolescent and young adult participants (12-30 years of age) with sickle cell disease (SCD).

NCT ID: NCT05228756 Withdrawn - Clinical trials for Cardiovascular Diseases

SCD Fit Homebase Program

Start date: November 1, 2022
Phase: N/A
Study type: Interventional

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.

NCT ID: NCT04817345 Withdrawn - Sickle Cell Disease Clinical Trials

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

Start date: April 2023
Phase: Phase 2
Study type: Interventional

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.

NCT ID: NCT04776850 Withdrawn - Sickle Cell Disease Clinical Trials

Pre-transplant Immunosuppression and Donor Stem Cell Transplant for the Treatment of Severe Hemoglobinopathies

Start date: December 29, 2020
Phase: Early Phase 1
Study type: Interventional

This clinical trial studies the effect of pre-transplant immunosuppression (PTIS) and donor stem cell transplant in treating patients with severe blood diseases (hemoglobinopathies). PTIS helps prepare the body for the transplant and lowers the risk of developing graft versus host disease (GVHD). Hematopoietic cells are found in the bone marrow and produce blood cells. Hematopoietic cell transplantation (HCT) injects healthy hematopoietic cells into the body to support blood cell production. PTIS and HCT may help to control severe hemoglobinopathies.

NCT ID: NCT04594031 Withdrawn - Sickle Cell Disease Clinical Trials

US Phase I Study of ECT-001-CB in Patients With Sickle-Cell Disease

Start date: July 13, 2021
Phase: Phase 1
Study type: Interventional

The application of experimental hematopoietic cell transplantation (HCT) therapy in sickle-cell disease (SCD) must strike a balance between the underlying disease severity and the possibility of a direct benefit of the treatment, particularly in pediatric populations. Clinical studies in adults with SCD have focused on interventions that prolong survival and improve the quality of life. Unlike children, adults with SCD are much more likely to have a debilitating complication. As a result, the risk/benefit ratio of HCT is very favorable in adults, particularly if an approach to HCT that defines an acceptable level of toxicity can be established. Whereas hematopoietic stem cell transplantation (HSCT) remains the only curative treatment currently available for patients with SCD, the morbidity, the frequent irreversible damage in target organs and the mortality reported in the natural course of patients with severe SCD are strong incentives to perform HSCTs in younger age groups. For those who lack a matched related donor, CB transplant is an appealing option, but despite been less problematic, CB accessibility related to cell dose of appropriately matched cord blood unit (CBU) remains a significant issue. Through a 7-day culture process of a CBU's hematopoietic stem cell HSCs with the UM171 compound, the total cell dose is increased mitigating this limitation. UM171-CB expansion (ECT-001-CB) allows a greater CB accessibility, the selection of better matched cords that might translate into favourable clinical outcomes as reported in previous trials, including a lower risk of graft-versus-host disease. After CB selection and ex-vivo expansion, ECT-001-CB transplant will follow a myeloablative reduced-toxicity conditioning regimen consisting of rATG, busulfan and fludarabine with doses of all agents optimized to the individual using model-based dosing and will be followed by standard supportive care and GVHD prophylaxis consisting of tacrolimus and MMF.

NCT ID: NCT04541875 Withdrawn - Cystic Fibrosis Clinical Trials

Medication Adherence and Non-adherence in Adults With Rare Disease

Start date: January 2021
Phase:
Study type: Observational

The purpose of this study is to use the Medication Adherence Reasons Scale (MAR-Scale) to determine the extent of non-adherence to specific medications indicated to treat cystic fibrosis, hemophilia (A or B), idiopathic pulmonary fibrosis, myasthenia gravis, and sickle cell disease, and to identify the top patient-reported reasons for non-adherence. Internal reliability of the MAR-Scale will also be assessed in each condition.

NCT ID: NCT04474626 Withdrawn - Sickle Cell Disease Clinical Trials

Isoquercetin in Sickle Cell Anemia

Start date: December 2020
Phase: Phase 2
Study type: Interventional

This research study is being done to assess the safety and effectiveness of isoquercetin to reduce levels of soluble P-Selectin in patients with sickle cell disease. Isoquercetin is a naturally occurring flavonoid-or vitamin. You will find quercetin and isoquercetin in fruits and vegetables. The names of the study drug involved in this study are/is: - Isoquercetin

NCT ID: NCT04293172 Withdrawn - Sickle Cell Disease Clinical Trials

Study to Evaluate the Effect of Ticagrelor Versus Placebo in Reducing Vaso-Occlusive Crises Rate in Pediatric Patients With Sickle Cell Disease.

HESTIA5
Start date: June 30, 2020
Phase: Phase 3
Study type: Interventional

The purpose of this study is to compare the effect of ticagrelor vs placebo for the reduction of Vaso-Occlusive crises in paediatric patients with Sickle Cell Disease

NCT ID: NCT04058197 Withdrawn - Sickle Cell Disease Clinical Trials

Deferoxamine for Sickle Cell Chronic Leg Ulcer Treatment

D-SCOUT
Start date: July 21, 2020
Phase: Phase 1/Phase 2
Study type: Interventional

Approximately 60 subjects will be enrolled into this double-blind, placebo-controlled study for the Deferoxamine Intradermal Delivery Patch (DIDP). Those subjects who pass Screening will enter into the 2-week Standard of Care (SOC) Run-In period. During this time, ulcers will be assessed to check healing based on digital planimetry, and qualitative features of the ulcer. Subjects who meet eligibility criteria at the end of the 2-week Run-in Period will be randomized into active and control groups (2 active to 1 placebo) and enter the 12-week Treatment Period. At each visit during the Treatment Period, the target ulcer will be measured by digital photographic planimetry, the Principal Investigator will assess the wound qualitative attributes, and the DIDP (or placebo patch) will be placed as the primary wound dressing. At each visit the subject will also receive/review a daily diary to document pain , study drug compliance, and analgesic use.

NCT ID: NCT04031521 Withdrawn - Sickle Cell Disease Clinical Trials

Near Infrared Spectroscopy in Sickle Cell Pediatric Patients

Start date: June 26, 2019
Phase:
Study type: Observational

Endothelial dysfunction contributes to vaso-occlusion and acute pain in sickle cell disease. Near infrared spectroscopy (NIRS) technology can measure tissue oxygenation and endothelial function. The main objective of this study is to study the natural history of tissue muscle oxygenation using NIRS in pediatric sickle cell subjects experiencing acute pain and pediatric sickle cell patients in steady-state.