Sickle Cell Disease Research and Clinical Trials

At the University of Pittsburgh, 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.

Participating in a clinical trial represents one of the many ways you can help Ryan Clark's Cure League achieve the ultimate mission of finding a cure for sickle cell disease.

Why Are 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

Research studies investigating various aspects of sickle cell disease

Learn more about current and ongoing studies by researchers at the University of Pittsburgh’s Vascular Medicine Institute and UPMC.

  • Collection of Blood from Volunteers and Patients for Studies of Endothelial Dysfunction and Systemic Inflammation — Researchers designed this study with the ultimate goal of developing a new laboratory test to help scientists study the various cells in the blood and lining the blood vessels. By developing an effective way to study these different cells, researchers can identify specific biomarkers for sickle cell disease. Contact Suchitra Barge, MPH, Clinical Research Coordinator — barges@upmc.edu or 412-864-3290 — to learn about eligibility requirements for this study.
  • Treatment of Pulmonary Hypertension in Sickle Cell Disease with Sildenafil Treatment (walk-PHaSST) — The Translational Research Core Laboratory at the Division of Pulmonary, Allergy, and Critical Care Medicine of the University of Pittsburgh serves as a biological specimen repository for the walk-PHaSST study. Funded by the National Institutes of Health (NIH), researchers conducted this study in 10 sites across the United States and United Kingdom. Contact Suchitra Barge, MPH, Clinical Research Coordinator — barges@upmc.edu or 412-864-3290 — to learn more about this study.
  • MRI Correlates of Accelerated Brain Aging in Sickle Cell Disease — 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. Contact Suchitra Barge, MPH, Clinical Research Coordinator — barges@upmc.edu or 412-864-3290 — to learn about eligibility requirements for this study.
  • Sickle Cell Acute Chest Syndrome Treatment with Prasugrel - Phase III - A comparison of Prasugrel and Placebo in Pediatric Patients with Sickle Cell Disease — The purpose of the study is to evaluate the efficacy and safety of the drug prasugrel for the reduction of acute chest syndrome, a severe form of vaso-occlusive crisis (VOC), in children, adolescents, and teens with sickle cell disease. The study will also investigate reduction in daily pain in children who have sickle cell disease.  Contact Angela Martino, BSN, RN, Research Nurse Coordinator  — Angela.Martino2@chp.edu or 412-692-6467 — to learn more about this study.
  • 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 —  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.  Contact Angela Martino, BSN, RN, Research Nurse Coordinator  — Angela.Martino2@chp.edu or 412-692-6467 — to learn more about this study.
  • 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) —  Through this study researchers hope to determine whether the investigational drug SelG1 is effective in preventing or reducing the occurrence of pain crises when given to patients with sickle cell disease. The study will include and compare results for participants who are being treated with hydroxyurea and those who are not. SelG1 prevents various cells in the bloodstream from sticking together. By stopping these cell-cell interactions, SelG1 may prevent small blood vessels from becoming blocked and therefore reduce the occurrence and severity of pain crises, which can be acute and last from hours to days.  Contact Angela Martino, BSN, RN, Research Nurse Coordinator  — Angela.Martino2@chp.edu or 412-692-6467 — to learn more about this study.
  • Sickle Cell Vaso-Occlusive Pain Crisis Treatment Study (EPIC) - Phase III - Evaluation of Purified Poloxamer 188 (MST 188) in Vaso-Occlusive Crisis of Sickle Cell Disease (EPIC) —  The EPIC study evaluates whether purified poloxamer 188 (MST-188) can reduce the duration of vaso-occlusive pain crisis (VOC) in patients with sickle cell disease. The study will also evaluate whether MST-188 can reduce the frequency of rehospitalization of subjects due to a recurrence of VOC. Additionally, this study will compare the development of acute chest syndrome during VOC in patients who receive MST-188 to those who do not receive MST-188. “EPIC” stands for Evaluation of Purified Poloxamer 188 In Crisis.  Contact Angela Martino, BSN, RN, Research Nurse Coordinator  — Angela.Martino2@chp.edu or 412-692-6467 — to learn more about this study.

Not Qualified to Participate in a Clinical Trial?

Even if you don’t have sickle cell disease, or none of our current research fits your health profile, you can still help in a big way.

We need funding from concerned citizens like you to initiate new research studies and get closer to a cure. We encourage you to give what you can as a member of the Cure League sickle cell disease fighting team.

» Learn more about the many ways you can donate your financial resources and personal talents to the cause.