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Pediatric Cancer & Blood Disorder Clinical Trials & Studies

Access to More Clinical Trials and Innovative Therapies

Innovative clinical trials and drug development are creating new treatment paradigms and changing adolescent and young adult cancer treatment. Because of the strength of our research enterprise, our patients have access to new and promising treatment options. For example, we are one of only a few medical centers globally to offer groundbreaking RUBY CRISPR therapy to young adults with Sickle Cell Disease. This highly advanced therapy modifies a patient’s own cells to reduce harmful sickle cell-related pain crises.

Our collaborative and multi-disciplinary approach makes clinical trials more accessible to pediatric patients. In addition, by working closely with the UH Seidman Cancer Center for adults, we can offer adolescent, and young adult patients access to the same clinical trials typically only available for older patients.

Highlighted Clinical Trials & Studies

ATLAS Neo Study

A Phase 3, Single-arm, Multicenter, Multinational, Open-label, One-way Crossover Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged ≥ 12 Years With Severe Hemophilia A or B With or Without Inhibitory Antibodies to Factor VIII or IX

HOPS Study

Multi-center randomized trial to test whether the dosing of hydroxyurea using a novel precision medicine strategy for children with Sickle Cell Anemia (SCA) is superior to a standard step-wise dose escalation.

RUBY Study

The purpose of this study is to evaluate the safety, performance and tolerability of treatment of an investigational treatment (EDIT-301) to reduce the number of pain crises.

Pediatric Metastatic Osteosarcoma Study

The purpose of this study is to evaluate if Natalizumab can be used safely and effectively as immunotherapy in children, adolescent and young adult patients with pOS.