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Anemia, Sickle Cell clinical trials

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NCT ID: NCT03825341 Recruiting - Sickle Cell Disease Clinical Trials

Hydroxyurea Therapy: Optimizing Access in Pediatric Populations Everywhere

Start date: December 21, 2018
Phase: Phase 2
Study type: Interventional

Primary Objective 1. Define the pharmacokinetics of liquid-formulated HU in infants (9 months to <2 years) 2. Assess the relative bioavailability of HU "sprinkles" compared to capsules in children and adolescents (≥2 to 18 years). Secondary Objective Compare PK parameters in infants versus older children on this study and those from our previous "Pharmacokinetics and Bioavailability of a Liquid Formulation of Hydroxyurea in Pediatric Patients with Sickle Cell Anemia" (NCT01506544) trial. Exploratory Objectives Capture information regarding the taste of HU sprinkles using palatability questionnaire. This trial is an open label, single center assessment of the pharmacokinetics of two formulations of hydroxyurea (HU) designed to (1) determine the pharmacokinetic profile of a liquid formulation in infants and to (2) determine the bioavailability of "sprinkles", a novel method of administration for older children. The study aims to generate data to facilitate FDA approval for HU in children and potentially validate a new mode of administration ("sprinkles") that will optimize access and adherence for children in the US and globally.

NCT ID: NCT03814746 Not yet recruiting - Sickle Cell Disease Clinical Trials

Study of Two Doses of Crizanlizumab Versus Placebo in Adolescent and Adult Sickle Cell Disease Patients

Start date: April 29, 2019
Phase: Phase 3
Study type: Interventional

The purpose of this study is to compare the efficacy and safety of 2 doses of crizanlizumab (5.0 mg/kg and 7.5 mg/kg) versus placebo in adolescent and adult sickle cell disease (SCD) patients with history of vaso-occlusive crisis (VOC) leading to healthcare visit.

NCT ID: NCT03806452 Not yet recruiting - Sickle Cell Disease Clinical Trials

SIKAMIC (SIklos on Kidney Function and AlbuMInuria Clinical Trial)

Start date: February 2019
Phase: Phase 2
Study type: Interventional

The purpose of this phase IIb, national (France), multicentre, double-blind, randomised, placebo-controlled study is to determine the effect of hydroxycarbamide on albuminuria after 12 months of treatment in SCD adult patients.

NCT ID: NCT03805581 Recruiting - Sickle Cell Disease Clinical Trials

Defibrotide in Sickle Cell Disease-Related Acute Chest Syndrome

Start date: January 12, 2018
Phase: Phase 2
Study type: Interventional

This study evaluates the safety of defibrotide in subjects with sickle cell disease (SCD)-associated acute chest syndrome (ACS).

NCT ID: NCT03789591 Recruiting - Sickle Cell Disease Clinical Trials

Hydroxyurea Optimization Through Precision Study

Start date: January 17, 2019
Phase: Phase 3
Study type: Interventional

Hydroxyurea Optimization through Precision Study (HOPS) is a prospective, multi-center, randomized trial that will directly compare a novel, individualized dosing strategy of hydroxyurea to standard weight-based dosing for children with SCA. The primary objective of the study is to evaluate whether a pharmacokinetics-based starting hydroxyurea dose thieves superior fetal hemoglobin response to to standard weight-based initial dosing. Patients will be recruited from the pediatric sickle cell clinic at Cincinnati Children's Hospital Medical Center and from additional pediatric sickle cell centers within the United States.

NCT ID: NCT03786549 Not yet recruiting - Sickle Cell Disease Clinical Trials

Pediatric -Adult Care Transition Program of Patients With Sickle Cell Disease

Start date: January 2019
Phase: N/A
Study type: Interventional

Background The pediatric-adult care transition is a risk-disrupting time for patients with chronic disease. This care transition takes place during adolescence; a period of psychological upheavals and adaptations of family roles. During this period, medication adherence is non-optimal and absenteeism at medical appointments is high. Sickle cell disease (SCD) is the first genetic disease detected in France. It is chronic disease characterized by frequent painful vaso-occlusive crises (VOC) requiring emergency hospitalization when they are severe. Other serious complications are acute chest syndromes (ACS) and stroke. In order to improve the health status of teenagers with sickle cell disease, it is necessary to anticipate this care transition and to involve the pediatric and adult sectors. The biopsychosocial health approach and the Social-Ecological Model of Adolescent and Young Adult Readiness to Transition (SMART) describe a care transition integrating bioclinical and psychosocial factors such as integration of the patient's family, education on disease and therapeutics, psychological management of pain and medico-social orientation. The pediatric-adult transition program proposed is based on this biopsychosocial approach. It aims to improve the health status of adolescents with SCD, their quality of life and the use of health care service. Objective of the study To assess the impact of a pediatric-adult transition program on the incidence of sickle-cell-related complications leading to hospitalization on 24-months after transfer to the adult sector. The evaluation focuses on severe complications leading to hospitalization, such as VOC, ACS, and stroke. Study design Multicenter Open-label individual Randomized Controlled Trial Population : Patients aged at least 16 years old with sickle cell disease, and their parents (or legal representatives Number of subject : 196 patients (98 patients by arm) The study will last 24 months Expected results For patients and families Better health and quality of life for patients is expected, including better use of medical care after the transition program. It is also expected a better experience of the pediatric-adult care transition and indirectly a better experience of intrafamilial relations. For health professionals This project is expected to provide solutions to improve the pediatric-adult care transition of patients with chronic disease. Indeed, the methodological quality of the study will make it possible to evaluate the efficiency of the proposed program, to possibly adapt it and test it to other chronic diseases presenting the same care transition problematic. In terms of public health SCD mainly affects populations of sub-Saharan origin, with low visibility and high social vulnerability. By focusing on this population, this project will reduce the social inequalities in health, experienced by patients with SCD and their families. By improving the health, quality of life and care of patients with SCD, this project is expected to decrease the cost of the pediatric-adult care transition period.

NCT ID: NCT03763656 Recruiting - Anemia, Sickle Cell Clinical Trials

Pharmacokinetics of Oral Hydroxyurea Solution (SHORT TITLE)

Start date: November 20, 2018
Phase: Phase 2
Study type: Interventional

An open label, safety and pharmacokinetic study of oral hydroxyurea solution administered to children from 6 months to 17.99 years (i.e. to the day before 18th birthday), with a 12 15 month treatment period for each participant. The study treatment duration will be for 6 months at the maximum tolerated dose [MTD], which is usually reached by 6 months after initiation of treatment. For patients in whom time to MTD is longer than 6 months or not achieved at all, the maximum duration of study treatment will be 15 months.

NCT ID: NCT03758950 Recruiting - Sickle Cell Disease Clinical Trials

Inhaled Mometasone to Promote Reduction in Vasoocclusive Events 2

Start date: November 29, 2018
Phase: Phase 2
Study type: Interventional

The study team proposes a triple-blind, placebo-controlled, phase II clinical trial of once-daily inhaled mometasone for 48 weeks (with 4-week washout at study completion) in individuals with Sickle Cell Disease (SCD) who report episodic cough or wheeze (ECW) but do not have asthma. Patients will be recruited from and followed in SCD clinics at participating sites. The primary endpoint will be a reduction in sVCAM level of 20% or more in comparison to placebo.

NCT ID: NCT03753854 Recruiting - Sickle Cell Disease Clinical Trials

Sleep Apnea in Sickle Cell Disease

Start date: May 28, 2018
Phase: N/A
Study type: Interventional

Despite the fact that obstructive sleep apnoea (OSA) is highly prevalent in the sickle cell population, studies focusing on the associations of the two diseases and their common pathophysiological mechanisms are scarce. OSA is one of the most common conditions responsible for hemoglobin desaturation. The nocturnal hemoglobin desaturation occurring in some sickle cell disease (SCD) patients with OSA could trigger hemoglobin S polymerization and red blood cell (RBC) sickling, leading to further blood rheological alterations, hence increasing the risks for VOC. Moreover, OSA has been demonstrated to increase oxidative stress and inflammation in non Sickle Cell Disease (SCD) patients, which, in SCD patients, could increase the risk for complications. Finally, OSA is accompanied by impaired vascular function and autonomic nervous system dysfunction in the general population. Indeed, the presence of OSA in SCD could increase the clinical severity of patients and the frequency of VOC.

NCT ID: NCT03745287 Recruiting - Sickle Cell Disease Clinical Trials

A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease

Start date: November 27, 2018
Phase: Phase 1/Phase 2
Study type: Interventional

This is a single-arm, open-label, multi-site, single-dose Phase 1/2 study in up to 12 subjects 18 to 35 years of age with severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.