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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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Red Cell Half Life Determination in Patients With and Without Sickle Cell Disease

Background: Sickle cell disease (SCD) is an inherited blood disorder.

Locations

1 United States site

Age

18 to 99 Years

Genotypes

HbSS

Phase

Early Phase 1

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A Test of the Safety, Effectiveness, and Acceptability of an Improvised Dressing for Sickle Cell Leg Ulcers in a Tropical Climate

One in 300 Jamaicans have HbSS sickle cell disease, and of these, up to 70% will suffer from sickle cell leg ulcers (SCLUs).

Locations

1 Jamaica site

Age

13 to 70 Years

Genotypes

HbSS

Phase

N/A

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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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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.

Locations

1 United States site

Age

11 to 17 Years

Phase

N/A

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A First-in-patient Phase I/II Clinical Study to Investigate the Safety, Tolerability and Efficacy of Genome-edited Hematopoietic Stem and Progenitor Cells in Subjects With Severe Complications of Sickle Cell Disease

This study will evaluate two genome-edited, autologous, hematopoietic stem and progenitor cell (HSPC) products - OTQ923 and HIX763 - each reducing the biologic activity of BCL11A, increasing fetal hemoglobin (HbF) and reducing complications of sickle cell disease.

Age

2 to 40 Years

Genotypes

HbSS, HbSC

Phase

Phase 1/Phase 2

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The Role of Oxygen Reserve in Brain Growth and Cortical Thickness in Children With and Without Sickle Cell Anemia

The purpose of this research study is to better understand how blood flow and metabolism change can influence brain development in the early decades of life.

Age

4 to 21 Years

Phase

N/A

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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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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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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

3 Egypt sites

1 Saudi Arabia site

Age

5 to 15 Years

Phase

Phase 2/Phase 3

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