View clinical trials related to Anemia, Sickle Cell.
Filter by:This is a phase 1, first-in-human, multi-center, open-label, single dose 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 sickle cell disease (SCD). The study involves sequential dose escalation of cohorts with between-group assessments of key safety and PK variables.
Children with sickle cell disease systematically receive a transfusion 2 to 5 days before scheduled surgery (with the exception of minor surgeries) in order to avoid post-operative complications of which the vaso-occlusive crisis and acute thoracic syndrome are the most frequent. This standardized preoperative protocol was established on the basis of the results of large-scale randomized studies, most of which date back over ten years, and which have demonstrated the beneficial effects of transfusion (or transfusion exchange) preoperatively. To date, several other more recent studies (but not controlled) have questioned this type of systematic management. The purpose of this study is to review retrospectively data of sickle cell children who have undergone elective surgery at the Huderf in the last ten years and to identify the eventual complications encountered. The most common procedures in these patients are: tonsillectomy with or without associated adenoids, splenectomy and cholecystectomy. General data on sickle cell disease (history, genotype, G6PD deficiency, biology and previous complications), pre-surgical preparation, surgery and post surgical management and complications will be collected and analyzed. This retrospective analysis will allow an objective assessment of the current quality of care and will provide useful data to improve patient management.
This Phase 1 first-in-human, first-in-patient, single ascending dose and multiple dose study will be a randomized, double-blind, placebo-controlled investigation of the safety, tolerability, and pharmacokinetics of PF-07209326 in healthy participants and participants with sickle cell disease.
Study participants will undergo up to four periods of voxelotor administered orally at progressively higher dose levels from 1500 mg until either a maximum tolerated dose (MTD) or 3000 mg/day dose is reached, whichever occurs first
Sickle cell disease (SCD) is an inherited blood disorder. Symptoms include acute and chronic complications. Due to progress in SCD care, patients with SCD are living longer than before and we focus more attention in chronic complications. Children with SCD experience worse cognitive functions than healthy children, and fewer is known about cognitive functions in adults. Studies suggest lower cognitive performance in SCD, mostly in executive functions and processing speed, but the biological and anatomical substrates of cognitive decline are not yet well established in SCD. Often times, cognitive impairments and cerebral disorders are not diagnosed and treated in adults with SCD. The main objective of this study is to propose a deep neuropsychological assessment in adults with SCD and cognitive complaints and to highlight links between cognitive functions and clinical, biological and neuroradiological markers. The hypothesis of this study is that cognitive functions are associated with severity of the SCD, with bood abnormalities, with MRI markers and Transcranial Doppler (TCD) markers of cerebrovascular disease. The secondary objective of this study is to validate a brief cognitive assessment tool (BEARNI tool) in adults with SCD. This study is an observational cross-sectional study that will enroll adults with SCD and cognitive complaint.
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.
This is a multi-site, open- label rollover study to evaluate the long-term safety and efficacy of CTX001 in pediatric and adult participants who received CTX001 in parent studies 111 (NCT03655678) 141 (NCT05356195) or 161 (NCT05477563) (transfusion-dependent β-thalassemia [TDT] studies) or Study 121 (NCT03745287) or 151 (NCT05329649), 161(NCT05477563),171 (NCT05951205) (severe sickle cell disease [SCD] studies).
The purpose of this study is to develop a safe and curative stem cell transplant approach to treating sickle cell disease by assessing the safety of haploidentical hematopoietic stem cell transplantation using αβ+ T-cell depletion for children and adolescents with severe sickle cell disease (SCD).
The objective of the study is to refine our knowledge on the physiopathology of the symptoms and the complications for the patients affected by a drepanocytic syndrome. The establishment of risk factors and indicators of severity will allow to target better the patients requiring an adequate strategy in order to prevent the installation of some complications or to limit their worsening.
HSCT is currently the only curative option for SCD but less than 20% of SCD patients have a MD donor available. So far, all curative approaches beyond a MSD HSCT at young age are non-satisfactory. With the lack of a suitable donor for the vast majority of patients, the major question of this trial is, if a haploidentical αß/CD19+ T-cell depleted HSCT can be a valid alternative to a MSD HSCT. The main challenge in non-malignant diseases is to offer a safe and GvHD-free HSCT without rejection.