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.
  • Sickle Cell Research Registry — Researchers are currently collecting information about the care sickle cell disease patients receive, the medications they are prescribed, as well as the complications they experience. People with sickle cell disease, newborn to age 21, may qualify for enrollment. More about the sickle cell research registry. Contact Diana Ross, MSN, Research Nurse Coordinator — Diana.Ross@chp.edu or 412-692-7857 — to learn about eligibility 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.
  • MRI of Long Bones in Arms and Legs During Pain Crisis — This study accepts people with sickle cell disease between the ages of 12 and 65 who are admitted to the hospital with a pain crisis. Pain must occur in the long bones of the arms and legs to meet enrollment criteria. Patients receive an MRI during the pain crisis to evaluate the activity of the bone marrow during the event. The participant will receive additional MRIs 1 and 2 months after the initial MRI to evaluate and compare the activity of bone marrow to the first MRI. Researchers intend to gain a better understanding of bone marrow activity during pain crisis in hopes that it will seed new research into acute changes in the bone marrow during pain. Contact Diana Ross, MSN, Research Nurse Coordinator — Diana.Ross@chp.edu or 412-692-7857 — to learn about eligibility for this study.
  • Web-based Monitoring of Pain in Children with Sickle Cell Disease — The UPMC health care team will use Internet technology to monitor pain levels in children with sickle cell disease. Contact Diana Ross, MSN, Research Nurse Coordinator — Diana.Ross@chp.edu or 412-692-7857 — to learn about eligibility for this study.
  • Study Investigating Effects of Different Doses of Prasugrel in Children with Sickle Cell Disease — Prasugrel is a drug that helps prevent platelets in the blood stream from sticking together to form clots. The study is accepting children between the ages of 2 and 17 with sickle cell disease and will investigate the best dose of prasugrel to prevent clot formation. Researchers suspect that preventing clot formation or clumping of platelets will reduce the frequency and severity of pain in children with sickle cell disease. Researchers hope to determine the safest and most effective dose of prasugrel in children. More about prasugrel dosing study. Contact Diana Ross, MSN, Research Nurse Coordinator — Diana.Ross@chp.edu or 412-692-7857 — to learn about eligibility for this study.

Bone marrow transplantation studies in sickle cell disease

  • Sickle Cell Transplantation to Prevent Disease Exacerbation (STRIDE) Study — STRIDE is a multicenter study focused on young adults (aged 16 to 40) with severe sickle cell disease with the end goal of gaining a better understanding of the safety and feasibility of bone marrow transplantation (BMT). 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. Contact Melissa Byrne (Jones), MPH, Research Nurse Coordinator — Melissa.Jones@chp.edu or 412-692-7336 — to learn about eligibility requirements for this study.
  • Evaluating the Safety and Effectiveness of Bone Marrow Transplants in Children with Sickle Cell Disease (BMT CTN #0601, The SCURT Study) — This multi-center study investigates the safety and effectiveness of a unique approach to pre-conditioning that uses reduced intensity conditioning prior to a BMT with unrelated donor material. Youths aged 3 to 19 with severe sickle cell disease may be eligible to participate. More about the SCURT study. Contact Melissa Byrne (Jones), MPH, Research Nurse Coordinator — Melissa.Jones@chp.edu or 412-692-7336 — to learn about eligibility requirements for this study.
  • Severe Blood Disorder Treatment with Allogenic Stem Cell Transplant Study — This pilot study investigates new non-toxic approaches to stem cell transplantation with a goal of determining the best immunosuppressive regimen for post transplant therapy. People aged 3 to 35 who have severe blood disorders, such as sickle cell disease or thalassemia, and have a related donor may enroll in this study. Read more about this study and view the NIH information about the research. Contact Melissa Byrne (Jones), MPH, Research Nurse Coordinator — Melissa.Jones@chp.edu or 412-692-7336 — to learn about eligibility requirements for this study.

Novel approaches for treating vaso-occlusive crises

  • Study of GMI-1070 for the Treatment of Vaso-Occlusive Crises (VOC) in People with Sickle Cell Disease — Investigators participating in this in-patient study are evaluating a drug called GMI-1070 to determine its safety and effectiveness for treating VOC in people aged 12 to 23. A member of the health care team approaches patients about enrollment in the study when they enter the hospital with a VOC. Those who agree to participate receive the study drug only during this particular hospital admission. They must then return 2 or 3 more times over the next 30 days for follow up. More about the GMI-1070 study. Contact Melissa Byrne (Jones), MPH, Research Nurse Coordinator — Melissa.Jones@chp.edu or 412-692-7336 — to learn about eligibility requirements for this study.
  • Intravenous Magnesium for Sickle Cell Vaso-Occlusive Crises (Magnesium in Crisis – MAGIC) — Another in-patient research study seeks to determine the safety and efficacy of using IV magnesium therapy in the treatment of VOC. A member of the health care team approaches people aged 4 to 21 about enrollment upon admission to the hospital with a pain crisis. They receive up to six doses of magnesium, but only while hospitalized during this particular admission. They then follow up with the research team for 1 to 3 months via telephone or in-person visit. Contact Melissa Byrne (Jones), MPH, Research Nurse Coordinator — Melissa.Jones@chp.edu or 412-692-7336 — to learn about eligibility requirements for this study.

Hydroxyurea in sickle cell disease

  • Patient and Caregiver Reported Barriers to Hydroxyurea Use in Pediatric Sickle Cell Disease — Investigators in this study interview parents and caregivers to determine why they chose to use, or not to use, hydroxyurea for reducing frequency of pain crises in their child with sickle cell disease. Although this study is no longer enrolling participants, investigators will use the data collected to develop a larger scale, similar study. Contact Melissa Byrne (Jones), MPH, Research Nurse Coordinator — Melissa.Jones@chp.edu or 412-692-7336 — to learn about eligibility requirements for this study.
  • Mobile Phone Directly Observed Hydroxyurea Therapy for Pediatric Sickle Cell Patients — This study looks at the use of technology to monitor compliance in taking hydroxyurea. Patients or their caregivers receive daily reminders to take or administer the medicine and they submit videos of the patient taking the medicine. Contact Melissa Byrne (Jones), MPH, Research Nurse Coordinator — Melissa.Jones@chp.edu or 412-692-7336 — to learn about eligibility requirements for 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.