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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.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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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