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Anemia, Sickle Cell clinical trials

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

Pharmacokinetics of Oral Hydroxyurea Solution (SHORT TITLE)

HUPK
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 Not yet recruiting - Sickle Cell Disease Clinical Trials

Inhaled Mometasone to Promote Reduction in Vasoocclusive Events 2

IMPROVE2
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

DREPAPNEE
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 patients with OSA may 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.

NCT ID: NCT03733249 Enrolling by invitation - Clinical trials for Myelodysplastic Syndromes

Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study

Start date: January 2017
Phase: Phase 1/Phase 2
Study type: Interventional

This is a long-term follow up study evaluating the safety of BPX-501 T cells (rivogenlecleucel) and infused in pediatric patients previously enrolled on the BP-004 study.

NCT ID: NCT03731182 Not yet recruiting - Clinical trials for Pneumococcal Infections

A Study to Evaluate the Safety, Tolerability, and Immunogenicity of V114 in Children With Sickle Cell Disease (V114-023/PNEU-SICKLE)

Start date: January 23, 2019
Phase: Phase 3
Study type: Interventional

This study is designed to describe the safety, tolerability, and immunogenicity of V114 in children with sickle cell disease.

NCT ID: NCT03720626 Available - Sickle Cell Disease Clinical Trials

MAP to Provide Access to Crizanlizumab, for Sickle Cell Disease Patients

Start date: n/a
Phase:
Study type: Expanded Access

The purpose of this Cohort Treatment Plan is to allow access to crizanlizumab (SEG101) for eligible patients diagnosed with sickle cell disease (SCD) to prevent or reduce the frequency of vaso-occlusive crises (VOC). The patient's Treating Physician should follow the suggested treatment guidelines and comply with all local health authority regulations.

NCT ID: NCT03719729 Recruiting - Sickle Cell Disease Clinical Trials

Rifaximin to Modify the Disease Course in Sickle Cell Disease

Start date: August 22, 2018
Phase: Phase 2
Study type: Interventional

In this single-arm, one-stage Phase II study, we hypothesize that gut decontamination with rifaximin will reduce the frequency of hospital admission due to painful crisis in patients with SCD. We will accrue 20 SCD patients who had at least two hospital admissions in the previous 12 months. These patients will receive rifaximin 550 mg twice a day for a total of 12 months. This following clinical parameters will be measured: 1. Changes in the annual rate of hospital admissions due to painful crisis; 2. Changes in the annual rate of days hospitalized; 3. Annual rates of uncomplicated crises; 4. Annual rate of acute chest syndrome; 5. Changes in the quality of life; and 6). Toxicities. The following laboratory parameters will be measured: 1. Changes in the number of circulating activated neutrophils; 2. Changes in the intestinal microbiome diversity; 3. Changes in the urinary 3-indoxyl sulfate levels; 4. Changes in the serum biomarkers of intestinal permeability (lipopolysaccharides; zonulin, citrulline, and fatty acid binding proteins).

NCT ID: NCT03716726 Not yet recruiting - Sickle Cell Disease Clinical Trials

Sickle Cell Anemia WE CARE

SCAWECARE
Start date: April 2019
Phase: N/A
Study type: Interventional

This mixed-methods study aims to understand the implementation of a previously tested, efficacious SDOH screening and referral intervention in the outpatient pediatric hematology setting; qualitatively assess possible mechanisms for such interventions on improving child health; and obtain population-specific empirical estimates to plan a large-scale clinical trial.

NCT ID: NCT03715972 Recruiting - Sickle Cell Disease Clinical Trials

Cerebrovascular Reserve and White Matter Disease in Patients With Chronic Anemia

CVR
Start date: July 15, 2018
Phase:
Study type: Observational

This is primarily an observational trial in patients with chronic anemia syndromes (sickle cell disease and thalassemia) and control subjects. The key purpose is to understand how brain blood flow reserve (the ability of the brain to increase its flow in response to stress) is altered in patients with chronic anemia. Since this parameter may depend on anemia severity, we will perform the MRI monitoring prior to and following clinically indicated transfusions in a subset of patients. Most patients will already be prescribed hydroxyurea as part of their standard of care. Since hydroxyurea could impact brain blood flow, there is also a small pilot study (20 patients, nonrandomized, open label) where MRI imaging will be performed prior to and following administration of hydroxyurea up to maximum tolerated dose. The study will enroll 90 adult subjects with transfusion independent sickle cell disease (70 SS, 10 SC, 10 Sβ0) and 60 patients with transfusion-dependent sickle cell disease. It will also include 10 transfusion independent thalassemia patients and 20 transfusion dependent thalassemia patients as well as 40 control subjects recruited from first degree relatives of the sickle cell disease population. All eligible subjects will be asked to provide informed consent before participating in the study.