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Preliminary Feasibility and Efficacy of The Balance Program to Reduce the Impact of Pain on Daily Functioning in Pediatric Sickle Cell Disease

Pain is the primary complication of sickle cell disease (SCD), including vaso-occlusive crises and more persistent, chronic pain.

Age

8 to 17 Years

Genotypes

HbSS, HbSC

Phase

N/A

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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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Low Dose Ketamine for Acute Pain Crisis in Patients With Sickle Cell Disease

BACKGROUND: Current treatment standard for acute pain crisis in sickle cell disease (SCD) is largely supportive care: opioid analgesics, hydration, oxygen, and blood transfusion.

Age

> 18 Years

Phase

Phase 4

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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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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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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 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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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 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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