Clinical Trials

Completed

Hydroxyurea for the Treatment of Patients With Sickle Cell Anemia

A total of fifty severely affected patients with homozygous sickle cell disease or other sickling disorders (e.g. B negative or B positive Thalassemia/Sickle) who are greater than 18 years of age will be eligible for treatment. Such patients must be able to tolerate an extensive period without blood transfusion and have relatively well preserved renal and hepatic function (creatinine less than 1.5 mg/dl and normal liver function test with exception of a mild elevation in transaminase).

Learn More

Recruiting

Phenotypic and Transcriptomic Description of Megakaryocytes in Sickle Cell Patient

Sickle cell disease is the most common inherited blood disorder in the world. Chronic hemolysis induces platelet activation and chronic inflammation. Platelets and megakaryocyte, as medullar platelets precursors, are known to play a role in innate immunity. Little is known about the role of megakaryocytes at basal state and during acute complication in sickle cell disease patients. The aim of this study is to evaluate the role of megakaryocytes in sickle cell disease.

Learn More

Completed

Hydroxyurea Management in Kids: Intensive Versus Stable Dosage Strategies

This is a pilot study, single-blind, randomized, multicenter, therapeutic clinical trial designed to evaluate the feasibility of enrolling infants and toddlers (9 months to 36 months) with sickle cell anemia (SCA; HbSS or HbSβ\^0thalassemia), regardless of disease severity, to a therapeutic trial. A prior clinical trial at St.

Learn More

Completed

DREPAMASSE Study - Evaluation of a Newborn Screening for Sickle Cell Disease by Tandem Mass Spectrometry

Three methods are actually used in newborn screening for sickle cell disease (SCD) in France: isoelectric focusing, high performance liquid chromatography and capillary electrophoresis. New technologies are currently under development such as Matrix Assisted Laser Desorption Ionisation - Time of Flight (MALDI-TOF) and tandem mass spectrometry (MS/MS) using the SpOtOn Diagnostics Reagent Kit available in United Kingdom only.

Learn More

Completed

Phosphodiesterase Type-5 Inhibitor Therapy in Sickle Cell People With Pulmonary Hypertension

Background:

Sickle cell disease (SCD) is a common inherited blood disorder. Many people with SCD are at risk to get pulmonary hypertension (PH). PH means that the blood pressure in the blood vessels to the lungs is high, and is a serious disease and. Very few studies have looked at the success of treatments for PH in people with SCD. Researchers want to learn more about treating PH with a type of drug called phosphodiesterase type 5 inhibitors (PDE5-I). They will look at the records of people who have already joined other studies.

Objective:

Learn More

Completed

Assessment of Algorithm-Based Hydroxyurea Dosing on Fetal Hemoglobin Response, Acute Complications, and Organ Function in People With Sickle Cell Disease

Background:

\- Sickle cell disease (SCD) is a blood disease. The drug hydroxyurea (HU) is approved to prevent pain crises in people with SCD. Researchers want to see how higher doses of HU affect the blood. This will help them learn about the right dosage of HU to give to people with SCD.

Objective:

\- To improve hydroxyurea dosing in people with SCD.

Eligibility:

\- People age 15 or older with homozygous SCD (HbSS).

Design:

Learn More