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Study of a Red Blood Cell Deformability Parameter in Patients With Sickle Cell Disease, at Basal State and During Vaso-occlusive Crisis

Sickle cell disease is the most common genetic disease in the world.

Locations

1 France site

Age

> 18 Years

Genotypes

SS

Phase

N/A

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A Phase 1/2 Randomized, Double-blind, Placebo-controlled, Multicenter, Ascending Dose, Safety and PK/PD Study of SHP655 (rADAMTS13) in Sickle Cell Disease at Baseline Health and During Acute Vaso-Occlusive Crisis

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and efficacy of SHP655 in participants with baseline health sickle cell disease (SCD) and SCD with acute vaso-occlusive crisis (VOC).

Age

18 to 65 Years

Genotypes

SS

Phase

Phase 1/Phase 2

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Integration of mHEALTH Into the Care of Patients With Sickle Cell Disease to Increase Hydroxyurea Utilization- mESH Study

This project proposes to develop, test and evaluate targeted interventions to improve clinical provider prescribing of and patient adherence to hydroxyurea (HU).

Locations

8 United States sites

Age

15 to 45 Years

Phase

N/A

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A Phase III, Multicenter, Randomized, Double-blind Study to Assess Efficacy and Safety of Two Doses of Crizanlizumab Versus Placebo, With or Without Hydroxyurea/ Hydroxycarbamide Therapy, in Adolescent and Adult Sickle Cell Disease Patients With Vaso-Occlusive Crises (STAND)

The purpose of this study is to compare the efficacy and safety of 2 doses of crizanlizumab (5.

Locations

3 United States sites, 4 Belgium sites, 2 Brazil sites, 3 France sites, 3 Germany sites, 1 Greece site, 1 Italy site, 1 Lebanon site, 1 Netherlands site, 1 Spain site, 4 United Kingdom sites

Age

> 12 Years

Phase

Phase 3

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An Open-label Extension Study of IMR-687 in Adult Patients With Sickle Cell Anemia (Homozygous HbSS or Sickle-β0 Thalassemia) Who Participated in Study IMR-SCD-102

This is an open-label extension study of IMR-687 in adult patients who completed Imara's blinded Phase 2a study (IMR-SCD-102).

Locations

1 United States site

Age

> 18 Years

Genotypes

SS

Phase

Phase 2

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Folic Acid Supplementation in Children With Sickle-Cell Disease: A Randomized Double-Blind Cross-Over Trial

Folic acid supplementation (1mg/d) is the standard recommendation for Canadian children with Sickle cell disease (SCD), even though it can provide up to six times the recommended intake amount for healthy children.

Age

2 to 19 Years

Phase

N/A

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A Comprehensive Program to Increase Sickle Cell Trait Knowledge and Awareness Among Parents of Infants Identified in Newborn Screening

This is a study for parents of infants with Sickle Cell Trait (SCT) identified by newborn screening who are referred and present for in person SCT education at the Institution.

Locations

1 United States site

Age

> 18 Years

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A Randomized, Double-Blind, Placebo-Controlled Pilot Study of the Safety and Efficacy of Deferoxamine Intradermal Delivery Patch (DIDP) in Chronic Sickle Cell Leg Ulcers

Approximately 60 subjects will be enrolled into this double-blind, placebo-controlled study for the Deferoxamine Intradermal Delivery Patch (DIDP).

Age

> 18 Years

Phase

Phase 1/Phase 2

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Metabolic and Vascular Response to Exercise in Sickle Cell Trait Carriers: Effect of Hot Environment

The heterozygous form of sickle cell disease is clinically asymptomatic.

Locations

1 Guadeloupe site

Age

18 to 30 Years

Phase

N/A

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An Open-Label, Expanded Access Protocol for Patient With Sickle Cell Disease Who Have No Alternative Treatment Options

The intent of this open-label, multicenter expanded access program (EAP) is to provide early access to voxelotor prior to market authorization .

Locations

14 United States sites

Age

> 12 Years

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