New hope for kids with sickle cell: drug aims to cut painful crises

NCT ID NCT03474965

Completed Disease control Sponsor: Novartis Pharmaceuticals Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tested a drug called crizanlizumab in 117 children aged 2 to 18 with sickle cell disease who have had painful vaso-occlusive crises. The goal was to find the right dose and check safety, with some children also taking hydroxyurea. The drug works by blocking a protein involved in cell clumping, which may reduce crisis frequency.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: Crizanlizumab (with or without hydroxyurea)
What this could lead to: If successful, this could provide a safe and effective treatment option to reduce painful crises in children with sickle cell disease.
What could go wrong: This is a phase 2 study with no placebo group, so results are preliminary. Side effects are possible, and the drug may not work as well in children as in adults.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

COVID-19 sickle cell disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

SICKLE CELL DISEASE (SCD)

Contacts and locations

Locations

Childrens Healthcare of Atlanta

Atlanta, Georgia, 30342, United States
Childrens Hosp Boston Dept of Hematology

Boston, Massachusetts, 02115, United States
Childrens Hospital Of Philadelphia

Philadelphia, Pennsylvania, 19104-4399, United States
Childrens Hospital at Montefiore

The Bronx, New York, 10467, United States
Childrens National Hospital

Washington D.C., District of Columbia, 20010, United States
Cook Childrens Medical Center

Fort Worth, Texas, 76104, United States
Duke University Medical Center

Durham, North Carolina, 27710, United States
East Carolina University

Greenville, North Carolina, 27834, United States
Joe DiMaggio Childrens Hospital

Hollywood, Florida, 33021, United States
Medical Uni of South Carolina

Charleston, South Carolina, 29425, United States
Novartis Investigative Site

Brussels, 1000, Belgium
Novartis Investigative Site

Laken, 1020, Belgium
Novartis Investigative Site

Liège, 4000, Belgium
Novartis Investigative Site

Salvador, Estado de Bahia, 41253-190, Brazil
Novartis Investigative Site

Ribeirão Preto, São Paulo, 14048-900, Brazil
Novartis Investigative Site

São Paulo, São Paulo, 01232-010, Brazil
Novartis Investigative Site

Montreal, Quebec, H3T 1C5, Canada
Novartis Investigative Site

Cali, Valle del Cauca Department, 760012, Colombia
Novartis Investigative Site

Montería, 230004, Colombia
Novartis Investigative Site

Paris, 75015, France
Novartis Investigative Site

Heidelberg, 69120, Germany
Novartis Investigative Site

Nagpur, Maharashtra, 440009, India
Novartis Investigative Site

Padova, PD, 35128, Italy
Novartis Investigative Site

Orbassano, TO, 10043, Italy
Novartis Investigative Site

Beirut, 1107 2020, Lebanon
Novartis Investigative Site

Tripoli, 1434, Lebanon
Novartis Investigative Site

Muscat, 123, Oman
Novartis Investigative Site

Palma de Mallorca, Balearic Islands, 07120, Spain
Novartis Investigative Site

Barcelona, Catalonia, 08035, Spain
Novartis Investigative Site

Madrid, 28009, Spain
Novartis Investigative Site

Adana, 01330, Turkey (Türkiye)
Novartis Investigative Site

Mersin, 33110, Turkey (Türkiye)
Novartis Investigative Site

London, SE1 7EH, United Kingdom
University Of Alabama

Birmingham, Alabama, 35233, United States
University of Florida

Gainesville, Florida, 32610, United States

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