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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.

Dr. Enrico Novelli's current research interests

Dr. Novelli's current research project explores the role Thrombospondin-1 (TSP1), a glycoprotein found in certain platelet granules, plays in regulating hemostasis and thrombosis. This research will help reveal the link between hemolysis and thrombosis in sickle cell disease patients suffering from hemolytic anemia and acute episodes of pain crises. Establishing this link will help in the development of possible preventive and therapeutic protocols for people with sickle cell disease.

Other research seeks to explore various mechanisms leading to vaso-occlusion and hemolysis in patients with sickle cell disease. Vaso-occlusion and hemolysis lead to pulmonary hypertension and vascular disease in sickle cell disease patients. This research will lead to a clearer understanding of the role certain proteins (like TSP1 and others) play in leading to dangerous conditions like pulmonary hypertension. Understanding these mechanisms will help researchers develop effective preventive and therapeutic medications for people with sickle cell disease.

Dr. Mark Gladwin's current research interests

Dr. Gladwin’s clinical interests, research, and expertise focus on pulmonary hypertension and other pulmonary complications of sickle cell disease. Most recently, he served as the principal investigator (PI) on two multi-center trials: Walk-PHASST (Pulmonary Hypertension and Sickle Sildenafil Therapy Trial) and DeNOVO (Delivery of NO for Vaso-Occlusive pain crisis in sickle cell disease).

Gladwin has served as the PI or associate investigator on over 25 human subject protocols and holds seven FDA INDs for the use of investigational therapeutic medications, including nitrite, carbon monoxide, L-NMMA, and sildenafil.

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.

Adult Sickle Cell Disease Research and Clinical Trials

Collection of blood for studies of endothelial dysfunction and systemic inflammation
Who can participate Sickle cell disease patients and volunteers.
About the study The ultimate goal of this study is to develop 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.
Who to contact Suchitra Barge, MPH, Clinical Research Coordinator — barges@upmc.edu or 412-864-3290 — to learn about eligibility requirements.

 

Treatment of pulmonary hypertension in sickle cell disease with sildenafil treatment (walk-PHaSST)
Who can participate People with sickle cell disease who have pulmonary hypertension (high blood pressure in the lungs).
About the study 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.
Who to 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
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 Suchitra Barge, MPH, Clinical Research Coordinator — barges@upmc.edu or 412-864-3290 — to learn about eligibility requirements.

 

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MRI of long bones in arms and legs during pain crisis
Who can participate 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.
About the study 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.
Who to contact Diana Ross, MSN, Research Nurse Coordinator — Diana.Ross@chp.edu or 412-692-7857 — 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 Melissa Byrne (Jones), MPH, Research Nurse Coordinator — Melissa.Jones@chp.edu or 412-692-7336 —to learn about eligibility requirements for this study.

Pediatric Sickle Cell Disease Research and Clinical Trials

The following studies are for children and young adults with sickle cell disease.

Contact Diana Ross, MSN, Research Nurse Coordinator — Diana.Ross@chp.edu or 412-692-7857 — to learn about eligibility for the following studies:

  • Sickle Cell Research Registry — Researchers are currently collecting information about the care young 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.
  • Effects of Different Doses of Prasugrel in Children with Sickle Cell Disease — This 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.
  • Web-based Monitoring of Pain in Children with Sickle Cell Disease — The health care team will use Internet technology to monitor pain levels in children with sickle cell disease.

Contact Melissa Byrne (Jones), MPH, Research Nurse Coordinator — Melissa.Jones@chp.edu or 412-692-7336 — to learn about eligibility requirements for the studies below:

  • Safety and Effectiveness of Bone Marrow Transplants in Children with Sickle Cell Disease (BMT CTN #0601, The SCURT Study) — This multicenter study investigates a unique approach to pre-conditioning that uses reduced intensity conditioning prior to bone marrow transplantation with unrelated donor material. Youths aged 3 to 19 with severe sickle cell disease may be eligible to participate.
  • Severe Blood Disorder Treatment with Allogenic Stem Cell Transplant Study — This pilot study investigates new nontoxic 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. View the NIH information about this allogenic stem cell transplant research.
  • Study of GMI-1070 for the Treatment of Vaso-Occlusive Crises (VOC) in People with Sickle Cell Disease — Investigators participating in this inpatient study are evaluating a drug called GMI-1070 to determine its safety and effectiveness for treating VOC in people aged 12 to 23.
  • Intravenous Magnesium for Sickle Cell Vaso-Occlusive Crises (Magnesium in Crisis – MAGIC) — This inpatient 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.
  • 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.
  • 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.

Not Qualified to Participate in a Clinical Trial?

Logo for Ryan Clark's Cure LeagueEven 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.

The UPMC Adult Sickle Cell Disease Program doctors and researchers invite you to visit Ryan Clark's Cure League — an exciting initiative conceived and championed by Pittsburgh Steelers’ safety and leading tackle, Ryan Clark.

Clark, who carries the sickle cell trait, suffered life-threatening complications after playing a game in Denver, Colorado. The Cure League raises money to fund sickle cell disease research, clinical trials, community outreach, and more.

Learn how you can share your time, talent, and personal resources to help us find new treatments and a cure for this devastating inherited blood disorder.

» Join Ryan Clark's Cure League today!

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