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Prevalence of Osteoporosis in Sickle Cell Disease

Sickle cell disease is the most common single-gene disease in the world.

Locations

1 France site

Age

20 to 40 Years

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A Phase 1, Multi-Center, Open Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of CSL889 in Adult Patients With Stable Sickle Cell Disease

This is a phase 1, first-in-human, multi-center, open-label, single ascending dose (SAD) cohort study to evaluate the safety and tolerability, pharmacokinetics (PK), exploratory pharmacodynamics (PD), and biomarkers of target engagement of CSL889 following single intravenous (IV) doses in subjects with stable sickle cell disease (SCD).

Age

18 to 60 Years

Genotypes

HbSS

Phase

Phase 1

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Is the Preoperative Preparation of Sickle Cell Patients Optimal: Retrospective Assessment of Practices and Post-operative Complications in a Cohort of Children Followed at Hôpital Universitaire Des Enfants Reine Fabiola (HUDERF) and Who Have Been Managed According Local Guidelines Including Transfusion or Exchange Transfusion Before Surgery

Children with sickle cell disease systematically receive a transfusion 2 to 5 days before scheduled surgery (with the exception of minor surgeries) in order to avoid post-operative complications of which the vaso-occlusive crisis and acute thoracic syndrome are the most frequent.

Locations

1 Belgium site

Age

< 18 Years

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SMYLS: A Self-management Program for Youth Living With Sickle Cell Disease

The purpose of this study is to find out whether a web-based intervention using a mobile device is helpful for teens learning to care for and manage symptoms of sickle cell disease.

Age

11 to 17 Years

Phase

N/A

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Comparative Effectiveness of the Different Treatment Modalities for Management of Vaso-occlusive Painful Crisis in Pediatric Sickle Cell Disease

The aim of the present study is comparing the effectiveness of different treatment regimens for investigating the therapeutic potential for each one in management of Vaso-occlusive pain in pediatric sickle cell disease.

Locations

2 Egypt sites

1 Saudi Arabia site

Age

5 to 15 Years

Phase

Phase 2/Phase 3

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A Randomised, Double Blind, Parallel Group, Multicentre, Phase III Study to Evaluate the Effect of Ticagrelor Versus Placebo in Reducing the Number of Vaso-Occlusive Crises in Paediatric Patients Aged 6 Months to <18 Years With Sickle Cell Disease (HESTIA5)

The purpose of this study is to compare the effect of ticagrelor vs placebo for the reduction of Vaso-Occlusive crises in paediatric patients with Sickle Cell Disease .

Age

6 to 17 Years

Genotypes

HbSS

Phase

Phase 3

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A Pilot Study of Voxelotor for Sickle Cell Anemia Patients at Highest Risk for Progression of Chronic Kidney Disease

This study is a single center, prospective exploratory pilot study of Sickle Cell Anemia (SCA) participants.

Age

> 18 Years

Genotypes

HbSS

Phase

Phase 1/Phase 2

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A Phase 3 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo With the LentiGlobin BB305 Lentiviral Vector in Subjects With Sickle Cell Disease

This is a non-randomized, open-label, multi-site, single-dose, Phase 3 study in approximately 35 adults and pediatric subjects ≥2 and ≤50 years of age with sickle cell disease (SCD).

Locations

2 United States sites

Age

2 to 50 Years

Phase

Phase 3

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The Effect of Exercise on Resting Biomarkers in Subjects With Sickle Cell Trait

This study measures the effect of exercise on a variety of biomarkers in blood and urine selected to evaluate the physiological pathways of hemolysis, myolysis, thrombosis, inflammation, and renal function in subjects with sickle cell trait.

Age

18 to 70 Years

Phase

N/A

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A Phase 2, Open Label, Multiple Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Voxelotor in Patients With Sickle Cell Disease

This is a dose escalation study to evaluate the safety and tolerability of voxelotor at daily doses of 1500 mg to 3000 mg in participants with sickle cell disease (SCD).

Locations

6 United Kingdom sites

Age

18 to 60 Years

Genotypes

HbSS

Phase

Phase 2

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