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Adult Sickle Cell Disease Research and Clinical Trials

At UPMC, we're dedicated to developing new and effective therapies for sickle cell disease with the ultimate goal of finding a cure accessible to all.

Since the scientific community now has a greater understanding of the basic molecular processes and issues associated with sickle cell disease, we can devote many of our research efforts to clinical issues, such as specific treatment therapies, and developing gene-based therapeutics that will lead to a cure.

Experts at the UPMC Adult Sickle Cell Program conduct research studies across many interest areas in hopes of improving the lives of people with sickle cell disease.

Why Are Sickle Cell Disease Clinical Trials Important?

Clinical research trials, typically just referred to as clinical trials, play a critical role in the advancement of medical knowledge.

By conducting clinical trials, we can:

  • Learn how a new therapeutic medicine or treatment works in humans.
  • Learn which treatment strategies work well and which do not.
  • Discover new and better ways to prevent, diagnose, and treat complications of sickle cell disease.
  • Get closer to discovering a cure for sickle cell disease and related hemoglobinopathies that will work for a majority of patients.

Adult Sickle Cell Disease Research and Clinical Trials

Patient-centered comprehensive medication adherence management system to improve effectiveness of disease modifying therapy with Hydroxyurea in patients with sickle cell disease (SCD Mobile Dot)

Who can participate Patients with sickle cell disease, aged 2 to 65, who have sickle cell disease and are receiving HU therapy.
About the study Through this study researchers will determine whether an interactive medication management program will help children and adults with sickle cell disease who are taking hydroxyurea (HU) for their condition. HU is the only disease-modifying therapy for SCD, and can be effective in reducing complications such as pain crisis and acute chest syndrome, and improving survival. It is, however, vastly underutilized and poorly adhered to for a variety of reasons. Through this study, structured interventions will be individualized to patient barriers and preferences, and will consist of improved access to care through virtual clinic visits and in-home testing, adherence management via video-based, directly observed therapy (Mobile-DOT), and patient support using text and telephone follow-up.
Who to contact Study Coordinator Jude Jonassaint, RN — 412-623-1110 — to learn about eligibility requirements for this study.

Collection of blood from volunteers and patients for studies of endothelial function and systemic inflammation

Who can participate Both patients seen by the investigators and healthy volunteers, aged 18 or older.
About the study This study is designed to help develop new laboratory tests to help us study the genes your white blood cells are making, the proteins your white blood cells are making, and how well the cells that line your blood vessels (such as veins and arteries) are working. In order to do this, we are asking for donations of blood samples. This will help us develop better strategies of identifying specific diseases in blood.
Who to contact Jude Jonassaint, RN — 412-623-1110 — to learn about eligibility requirements for this study.

MRI correlates of accelerated brain aging in sickle cell disease

Who can participate Adults with sickle cell disease.
About the study This observational study investigates a new brain MRI protocol for detecting early evidence of accelerated brain aging and determining how these changes correlate with cognitive impairment in adult sickle cell patients.
Who to contact Caterina Rosano, MD, MPH, Principal Investigator —, 412-383-1294 — to learn about eligibility requirements.

Sickle cell transplantation to prevent disease exacerbation (STRIDE) study

Who can participate Young adults, aged 16 to 40, with severe sickle cell disease.
About the study STRIDE is a multicenter study focused on gaining a better understanding of the safety and feasibility of bone marrow transplantation (BMT) in young adults. Patients receive a special medication regimen, reduced intensity conditioning (RIC), prior to the BMT procedure. Using RIC prior to a BMT has worked to cure sickle cell disease in young children with matched sibling donors. The STRIDE study aims to determine if this same strategy can be successful in young adult patients and possibly those without matched family donors. More about STRIDE Phase I and II and the NIH information about the study.
Who to contact Mark Vander Lugt, MD — 412-692-5055 — to learn about eligibility requirements for this study.

Pediatric Sickle Cell Studies at UPMC Children's Hospital of Pittsburgh

Learn more about pediatric sickle cell studies at UPMC Children's Hospital of Pittsburgh.