Active clinical trials for "Anemia, Sickle Cell"

OBJECTIVES: I. Phase II trial to determine surgical morbidity of decompression coring, including any adverse events in the perioperative period and the rate of secondary medical or surgical interventions. II. Collect preliminary data to determine if decompression coring results in a substantial improvement in pain and mobility compared to conservative therapy in patients with avascular necrosis of the hip related to sickle cell disease.

Many patients with Sickle Cell Disease (SCD) may develop Acute Chest Syndrome (ACS). ACS is usually caused by a Lower respiratory tract infection (LRTI) which may be caused by either a bacterium or a virus. Antibiotics are usually used for 7 to 10 days with no microbiological workup. The hypothesis of the study is that the identification of the microorganisms might lead to a reduction of antibiotics exposure and a better care of the patients. We speculate that an early pathogen-directed strategy (respiratory broad panel multiplex PCR and early antibiotics interruption based on the PCT values decrease) might reduce the antibiotics exposure in SCD patients with ACS who are hospitalized and for whom an antibiotic treatment is indicated, as compared with usual care

This is a single center, open label Phase 1 clinical trial in normal adult subjects to assess safety, tolerability, pharmacokinetics, and pharmacodynamics of HBI-002, an orally administered liquid containing carbon monoxide (CO), with single ascending doses (SAD), followed by multiple dose with doses daily for 7 days.

FT-4202 is an oral small-molecule agonist of pyruvate kinase red blood cell isozyme (PKR) being developed for the treatment of hemolytic anemias. This initial study will characterize the safety, tolerability and the pharmacokinetics/pharmacodynamics (PK/PD) of a single ascending dose and multiple ascending doses of FT-4202 in the context of Phase 1 studies in healthy volunteers and sickle cell disease patients. The effects of food on the absorption of FT-4202 will also be evaluated in healthy volunteers.

The goal of the study is to evaluate descriptively the effect of crizanlizumab + standard of care and standard of care alone on renal function in sickle cell disease patients ≥ 16 years with chronic kidney disease due to sickle cell nephropathy.

Sickle cell disease (SCD) is a group of inherited blood disorders affecting 100,000 individuals in the United States. SCD often leads to complications, including pain crises and organ damage. Many individuals with SCD require medications (e.g., Hydroxyurea or Endari) that research has demonstrated reduce risk of complications and improve quality of life. Despite the need for strong medication adherence, adolescents and young adults (AYAs; 13-25 years) have the lowest adherence rates compared to other age groups. Efforts to reduce AYA non-adherence risk should include youth in earlier childhood and persist throughout the AYA developmental period, with the goal of maintaining adherence throughout childhood and young adulthood. Motivational Interviewing (MI) has been effective in increasing pediatric and adult medication adherence via in-person or telehealth delivery; however, researchers have yet to empirically evaluate MI for feasibility, acceptability, and/or efficacy in improving pediatric/AYA SCD medication adherence. The proposed feasibility trial will provide preliminary feasibility data for a newly developed MI+education intervention targeting medication adherence for pediatric and adolescents and young adults (AYA) patients who have sickle cell disease. This trial will also evaluate study design feasibility to inform a future randomized controlled trial (RCT). The investigators are interested in delivering the intervention to AYA patients and to parents of younger children who have sickle cell disease because the investigators anticipate that establishing strong adherence in younger childhood could prevent future non-adherence during the AYA developmental period. Participants will include 13-22 year-old patients with sickle cell disease as well as parents of 0-22 year-old patients with sickle cell disease. The investigators will randomize ten families to a 4-session telehealth MI+education intervention and five families to a one-session education-only control condition. All participants will complete assessments at three times. Intervention participants will complete the T2 assessment at their last intervention session (week 4-8), and the T3 assessment 16-20 weeks after study enrollment. Education arm participants will complete T2 assessments 4-8 weeks after study enrollment and will complete T3 assessments 16-20 weeks after study enrollment. Primary outcomes include intervention feasibility and acceptability and study design feasibility.

This study is aimed to assess the genetic and haematological modifiers of disease severity among patients with Sickle Cell Disease (SCD) in Kaduna State, northern Nigeria. It is composed by two separate study designs: a cross-sectional study and a longitudinal study. The cross-sectional study will evaluate clinical and laboratory parameters in paediatric Sickle Cell Anaemia (SCA) patients (ages 2-18 years) in steady state and during Vaso-Occlusive Crisis (VOCs) to determine the parameters that can be used as a guide to monitor the course of the disease towards early recognition and management of sickle cell crises. In addition, the study will explore genotype-phenotype correlations in SCA patients by targeted Next-Generation Sequencing (NGS) of genetic modifiers for haemoglobinopathies. The longitudinal study will collect clinical and laboratory data over time for a paediatric cohort of SCD patients (9 months old; followed up to 2 years of age) and parental samples will be collected to determine the βS-globin haplotype in family trios. The aim is to determine the temporal relationships among foetal haemoglobin (HbF) levels, haematological parameters and frequency of sickle cell crises in SCD patients in relation to the type of the βS-globin haplotype and the sickle genotype. In addition, samples collected at 24 months of age will also be analysed by NGS to identify genetic modifiers of clinical manifestations and severity of SCA. Participants from the following centre will be involved: Ahmadu Bello University Teaching Hospital (ABUTH) Zaria. Consent from all the study parents/legally designated representatives as well as assent from minors will be sought. Consent for genetic analyses will be sought as well. Clinical and haematological analyses will be performed at ABUTH while genetic analyses will be performed at the Cyprus Institute of Neurology and Genetics (CING).

To study the expression pattern of PUM1 gene in patients with sickle cell anemia and β-thalassemia intermedia. To detect PUM1 protein levels in sickle cell anemia and β-thalassemia intermedia patients. To correlate PUM1 gene expression pattern and protein levels with HbF levels in sickle cell anemia and β-thalassemia intermedia patients.

The purpose of this Phase 1a, first in human, randomized, double-blind, placebo-controlled study is to evaluate the safety, tolerability, PK and PD profile of the orally administered IMR-687 in healthy adult subjects.

Disease-related neurocognitive deficits are common in pediatric sickle cell disease (SCD). These deficits can significantly disrupt otherwise normal trajectories toward academic and vocational achievement and negatively impact psychosocial outcomes. Despite widespread recognition of neurocognitive deficits, there are no treatments shown to maintain or recover functioning once a child with SCD endures neuronal damage. Cognitive training (CT) has been a standard intervention used to stabilize and recover functioning in individuals with accidental or disease-related brain injury. Recent advances in technology have led to the development of computerized CT programs. This study seeks to assess the feasibility and efficacy of using computerized CT with pediatric patients with SCD. Children and adolescents with SCD between the ages of 7 and 16 years old (n = 80) will be recruited to complete a randomized (intervention or waitlist-control) home-based computerized CT program (Cogmed). Feasibility will be assessed by examining participation, retention, and program completion rates, as well as feedback from a feasibility and acceptability questionnaire and a brief qualitative interview. Participants will also complete assessments of attention, working memory, and academic fluency at baseline and immediately following the intervention. A final assessment will be conducted 6 months after the conclusion of the intervention to evaluate the stability of treatment effects.