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Hemoglobin Desaturation and RBC Adhesion: Potential Therapeutic Targets in Sickle Cell Disease

As part of routine care for SCD, some people are found to have low oxygen levels (≤ 88%) while sleeping, at rest, or with exercise.

Locations

1 United States site

Age

> 18 Years

Genotypes

SS

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A Phase II, Multicenter, Randomized, Open Label Two Arm Study Comparing the Effect of Crizanlizumab + Standard of Care to Standard of Care Alone on Renal Function in Sickle Cell Disease Patients ≥ 16 Years With Chronic Kidney Disease Due to Sickle Cell Nephropathy

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

Age

> 16 Years

Genotypes

SS, Sβ0-thal

Phase

Phase 2

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A Prospective Multicenter Trial Comparing Allogeneic Matched Related Haematopoietic Stem Cell Transplantation After a Reduced Intensity Conditioning Regimen, With Standard of Care in Adolescents and Adults With Severe Sickle Cell Disease

Although the survival of children with sickle cell disease (SCD) has dramatically improved over the last decades in the US and Europe, mortality remains high in adults.

Age

15 to 45 Years

Phase

Phase 3

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Collect of Cord Blood From Subjects at Risk for Sickle Cell Disease, for the Purpose of Laboratory Research

The study consists in collecting umbilical cord blood cells from newborns at risk of sickle cell disease, to perform laboratory experiments aiming to characterize the cells with HbS/HbS mutation, to develop methods to prepare, to gene-modify and to preserve these cells.

Age

18 to 45 Years

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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.

Locations

3 France sites

Age

< 27 Years

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Safety and Feasibility of High-Dose Topical Capsaicin for the Treatment of Neuropathic Pain in Pediatric Sickle Cell Disease

This study evaluates the safety and feasibility of using high dose topical capsaicin patches for the treatment of neuropathic pain in pediatric patients with sickle cell disease, as well as the feasibility of using a number of tests for the evaluation and monitoring of neuropathic pain.

Locations

1 United States site

Age

14 to 21 Years

Genotypes

SS, SC

Phase

Phase 1

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A Comparison of Individualized vs. Weight Based Protocols to Treat Vaso-Occlusive Episodes in Sickle Cell Disease

The purpose of this research study is to compare two different ways to give opioid pain medicine to treat sickle cell disease pain that is bad enough to go to the emergency department for treatment.

Locations

6 United States sites

Age

> 18 Years

Phase

Phase 3

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An Open-Label 12-Week Food Study to Assess the Safety, Tolerability, And Physiological Effects of An Amino Acid Food Product, AXA4010, In Subjects With Sickle Cell Disease.

This is an open-label study to understand the safety and tolerability of AXA4010, a novel composition of amino acids in adult and adolescent subjects with sickle cell disease over 12 weeks.

Locations

4 United States sites

Age

> 12 Years

Genotypes

SS

Phase

N/A

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Implications of a Paediatrician-psychologist Tandem for Sickle Cell Disease Care and Impact on Cognitive Functioning

Sickle cell disease (SCD) necessitates a paediatric treatment plan that considers the influence of psychological, family and intercultural factors.

Age

< 18 Years

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