View clinical trials related to Sickle Cell Disease.
Filter by:Rationale: Non-myeloablative allogeneic stem cell transplantation (SCT) has become a feasible curative treatment option for sickle cell disease (SCD) patients with an available matched sibling donor. Chemotherapy free conditioning with alemtuzumab and 3 Gy total body irradiation (TBI) is increasingly being used as preferred conditioning scheme for these patients. This regimen typically results in mixed donor chimerism and has only few toxic effects. However, the risk of graft failure (rejection) is still significant, with an occurrence of 13% in the latest series. Levels of T cell chimerism are crucial for the success of this kind of transplantation. A donor T cell level of at least 50% at 1-year post-transplantation seems to be sufficient to allow the discontinuation of immunosuppressive medication without risk of graft rejection. Low levels of alemtuzumab prior to or shortly after SCT are thought to facilitate rejection of the donor graft. Recently, a positive correlation between alemtuzumab levels on day+14 was found with levels of T cell chimerism +2 and +4 months post-transplantation in adult SCD patients receiving matched sibling donor SCT. However, in this study alemtuzumab levels prior to the infusion of hematopoietic stem cells and beyond day +28 post-transplantation were not measured. Furthermore, the alemtuzumab levels were measured in 2 patient groups undergoing two different conditioning regimens. Here, the investigators aim to thoroughly investigate the correlation of alemtuzumab levels and T cell chimerism. This will be the first study involving SCD patients receiving matched sibling donor SCT with alemtuzumab/TBI conditioning that includes alemtuzumab level measurements before the infusion of hematopoietic stem cells and beyond 1-month post-transplantation. Findings from this study will improve the insights into the etiology of graft failure in these patients and might ultimately lead to a more personalized approach in dosing alemtuzumab in order to achieve a more robust and stable engraftment of donor hematopoietic stem cells. Objectives: To investigate whether serum alemtuzumab concentrations are predictive of the robustness of engraftment in SCD patients undergoing a matched sibling donor transplantation with alemtuzumab/TBI conditioning resulting in mixed chimerism. Study design: Prospective observational laboratory study. Serum alemtuzumab concentration will be measured at various time points before and after stem cell infusion (days -3, 0, +7, +14, +28, +60). Study population: Adult SCD patients that are planned for a matched sibling donor transplantation with alemtuzumab/TBI conditioning at the Amsterdam UMC. Main study parameters/endpoints: The correlation between serum alemtuzumab concentration and levels of donor chimerism. Secondary endpoints: correlation between serum alemtuzumab levels and patients with and without successful engraftment. Correlation of serum alemtuzumab levels and the dosing of alemtuzumab in mg/kg, number of patient lymphocyte count and total number of infused enucleated donor-derived cells.
To assess the pharmacokinetics, pharmacodynamics and safety of Epeleuton capsules in adult SCD patients who are aged ≥18 years.
Vaso-occlusive crisis are highly painful in Sickle-cell patients. Morphine is the treatment of choice for this pain. Various adjuncts have been studied for the treatment of vaso-occlusive crisis. The investigators aimed to study the effect of clonidine associated with morphine in PCIA (patient controlled intravenous analgesia pumps) regimen. The investigators will compare it to the morphine alone in PCIA for the treatment of vaso-occlusive pain. The investigators will measure the morphine consumption of all patient, the impact on the apparition of the morphine secondary effect and on inflammation biomarkers and the biopsychosocial respond. Each patient will be hospitalized and follow by haematologist from the hospital, pain doctors and nurses. It will be a double blind randomised, prospective study. The randomisation will be done by the pharmacy.
This prospective mixed-method interview study aims to qualitatively describe the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases among patients and parents of children with a rare disease targeted for treatment using gene therapy techniques. Using learned insights, the team will develop an online platform providing educational content and patient decision aids for patients and their families.
This study will investigate the role of genetic modifiers in hemoglobinopathies through a large-scale, multi-ethnic genome-wide association study (GWAS).
A total of 170 patients male or female who are carrying SS or Sbeta0 versions of the beta globin gene will be included in the study. The subjects will be assigned with 1:1:1 ratio of either NUV001 Immediate release IR or NUV001 Gastro resistant GR or Placebo. The treatment duration of the study will be 90 days which has in total 5 visits. The primary end point of this study is to check the safety and tolerance of the orally administered nutraceutical supplement. This endpoint will be checked by assessing the Adverse events, Vital signs of the subject and the Change in hematological parameters from Baseline to Final visit.
This is a pilot study of daily dosing of NUV001 as a dietary supplement in 12 sickle cell disease patients with 3 months of follow-up plus 1 month after supplementation.The present study is designed to evaluate, first, the safety and tolerability parameters as well as to measure the plasma and urinary residues of daily oral doses of NUV001. Secondly, the study will evaluate the impact of NUV001 on biological parameters and quality of life of patients.
This study aims to describe and/or searches for, in cohorts of adult sickle cell anemia (SCA) and SC sickle cell patients living in the French West Indies and followed by SCD Reference and Competence Centers: 1-lipids profiles and associations at steady state with occurrence of sickle cell disease (SCD) complications, 2-lipids profile evolution during and after prospective acute complications (vasoocclusive crises (VOC) and priapism), 3-lipids profile variation (inter /intra individuals) during 4 prospective years, 4- Genetic primary modulators of SCD complications, 5- insulin resistance (HOMA), free fatty acids and glycerol dosages, 6- lipids enzymes, lipidome and functionality of HDL in sub-groups of SCD population.
Sickle cell disease (SCD) is associated with significant morbidity and mortality. Pain and many adverse outcomes occurring in sickle cell disease are inflammatory driven. Recent data has shown that gut dysbiosis is present in individuals with sickle cell disease. Gut dysbiosis has been linked to inflammation in certain diseases. Omega -3-fatty acids (fish oil) has been shown to improve pain outcomes in individuals with sickle cell disease, but its acceptance is variable. The aim of this study is to determine if a plant-based omega-3-fatty acids will be more acceptable and also improve outcomes in individuals with sickle cell disease
Structural and functional changes in arteries are increasingly being recognized as significant features of sickle cell disease. This study aims to determine whether there are differences in arterial function parameters between children with sickle cell disease with normal and abnormal transcranial Doppler velocity. After informed consent is obtained, participants will have vascular, Transcranial Doppler, haematological and biochemical parameters measured. Researchers will compare children with sickle cell disease who have normal Transcranial Doppler velocity and no history of stroke with children with those who have an abnormal Transcranial Doppler velocity with or without a history of stroke to see if there are significant differences in arterial function parameters.