Clinical Trials

This is a prospective, interventional, phase II, open-label, multicentre, national, non-comparative study of a single administration of the new dispersible form of hydroxycarbamide at the usual dose in children with sickle cell disease who are already treated with the current form of hydroxycarbamide (Siklos® 100 mg and/or 1000 mg film-coated tablets).

Since 1987, a screening for β Thalassemia in pregnant women is carried on in northern Israel, and from 1999 all the samples were tested also for Hgb S, Hgb C, Hgb D, Hgb O Arab and others.

In this study, the investigators intend to summarize the results of this preventive program aiming to detect couples at risk for having offspring with Thalassemia or SCA, the compliance regard to genetic counseling and prenatal diagnosis and the incidence of new affected babies born.

Secretory phosholipases A2 (sPLA2) are significantly elevated in the plasma of sickle cell disease patients with acute chest syndrome (ACS), and similar enzymes have been measured in exhaled breath condensate (EBC), which is collected easily and non-invasively. The investigators hypothesize that sPLA2 will be measurable in EBC samples from sickle cell patients with acute chest syndrome.

The objective of this study is to evaluate the incidence of ACS within the DREPADOM system and compare it to expected incidences of ACS (historic cohorts of PRESEV1 and PRESEV2)

Background:

\- Small blood vessels (microvessels) in many different organs are affected by diseases such as diabetes and atherosclerosis. These microvessels may also be abnormal in people who have sickle cell disease. Stiffness of the red blood cells leads to problems in the microvessels of the heart and kidneys. However, these problems may not be detected until these organs are severely affected. Researchers want to study problems with microvessels in people with and without sickle cell disease.

Objectives:

ICA-17043 is being developed for the chronic treatment of patients with sickle cell disease (SCD) in both adults and children. ICA-17043 is a potent and specific inhibitor of a channel in human red blood cells (RBCs) that blocks RBC dehydration. ICA-17043 is expected to inhibit RBC dehydration and thus should prevent or delay the sickling process. By reducing sickled cells, an improvement in anemia, a reduction in painful crises, and ultimately, less end-organ disease is anticipated.

This study will determine whether niacin can improve blood flow in people with sickle cell disease, in which abnormal red blood cells interfere with blood flow to cause the disease symptoms. Niacin, a drug that has been used to increase HDL (good cholesterol) levels, improves blood flow in people without sickle cell disease. This study will see if it can do the same in people with the disease.

Patients with sickle cell disease between 18 and 65 years of age may be eligible for this study.

The purpose of this study is to evaluate crovalimab for the treatment of a sickle cell pain crisis (also known as a VOE) that requires hospitalisation in adult and adolescent participants with SCD. The primary objective of this study is safety and will additionally evaluate pharmacokinetics (how crovalimab is processed by your body), pharmacodynamics (how your body reacts to crovalimab) and the preliminary efficacy of crovalimab compared with placebo.

The purpose of this study is to test the efficacy of a peer support coaching intervention to improve activated chronic illness self-management versus an attention control group in 225 adolescents and young adults with childhood onset chronic conditions.

Background:

People with sickle cell disease (SCD) have problems with their heart, brain, kidneys, liver, and lungs as they age. These problems may improve after transplant. Researchers want to learn how and why this happens.

Objective:

To study the benefits of treatments that are intended to cure SCD.

Eligibility:

People aged 18 and older with SCD who are either receiving curative therapy in the next 3 months or don t have any plans to receive a curative therapy in the next 2 years.

Design: