Rare Cancers and Blood Disorders Clinical Studies

Some of the many Hematology/Oncology 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 Acute Chest Syndrome Treatment with Prasugrel – Phase III

A Comparison of Prasugrel and Placebo in Pediatric Patients with Sickle Cell Disease

Read more

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

Read more

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

Read more

Sickle Cell Pain Crisis Management Study (SUSTAIN) – Phase II

Study to Assess Safety and Efficacy of SelG1 With or Without Hydroxyurea Therapy in Sickle Cell Disease Patients with Sickle Cell-Related Pain Crises (SUSTAIN)

Read more

Sickle Cell Vaso-Occlusive Pain Crisis Treatment Study (EPIC) – Phase II

Evaluation of Purified Poloxamer 188 (MST 188) in Vaso-Occlusive Crisis of Sickle Cell Disease (EPIC)

Read more

Solid Tumor or ALCL Treatment with Crizotinib and Conventional Chemotherapy – Phase I

Crizotinib in Combination with Conventional Chemotherapy for Relapsed or Refractory Solid Tumors or Anaplastic Large Cell Lymphoma

Read more

Transient Myeloproliferative Disorder Blood Study

Biology Study of Transient Myeloproliferative Disorder in Children with Down Syndrome

Read more