Rare Cancers and Blood Disorders Clinical Studies

Some of the many Cancer and Blood Disorders research opportunities available related to rare cancers and blood disorders, such as sickle cell disease, are listed below. Select a study to review a brief description and requirements. Parents or guardians of children who might be eligible are encouraged to inquire about enrollment. Contact information is provided at the end of each study.

Please note that Pilot and Phase I studies are the most experimental and should be considered only after other treatments or trials have been attempted without success.

Thank you for exploring these opportunities and helping us to find cures and prevent childhood disease.

Clinical Studies


Sickle Cell Disease and Vaso-occlusive Crisis Treatment With Rivipansel – Phase III

Study to Evaluate the Efficacy and Safety of Rivipansel (GMI-1070) in the Treatment of Vaso-occlusive Crisis in Hospitalized Subjects With Sickle Cell Disease

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Sickle Cell Disease Treatment with BMT and Conditioning (STRIDE) – Phase II

A Study of Hematopoietic Stem Cell Therapy for Young Adults with Severe Sickle Cell Disease

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Sickle Cell Medication Management Study

Patient-Centered Comprehensive Medication Adherence Management System to Improve Effectiveness of Disease Modifying Therapy with Hydroxyurea in Patients with Sickle Cell Disease

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Sickle Cell Treatment with GBT440 – Phase IIa

Study to Evaluate the Pharmacokinetics, Safety, Tolerability, and Treatment Effect of GBT440 in Pediatric Participants With Sickle Cell Disease

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Thrombosis Therapy Duration: The Kids-DOTT Study – Phase III

Prospective Evaluation of the Duration of Therapy for Thrombosis in Children

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Transient Myeloproliferative Disorder Blood Study

AAML08B1: Biology Study of Transient Myeloproliferative Disorder in Children with Down Syndrome

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Venous Thromboembolism Prevention with Dabigatran – Phase III

Study of Dabigatran Etexilate for Secondary Prevention of Venous Thromboembolism in Children

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Venous Thromboembolism Treatment with Dabigatran: The DIVERSITY Study – Phase III

Study of Dabigatran Etexilate Versus Standard of Care for Venous Thromboembolism Treatment in Children

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