Clinical Trials

What is a Clinical Trial?

Clinical trials help researchers assess the effectiveness of various types of treatments, be they new medications, medical devices or physical therapies – all in a carefully controlled setting. An important goal of the Children’s Sickle Cell Foundation is to help families find effective treatments for SCD. We hope to achieve this goal by connecting families who are interested in participating in clinical trials with researchers who are recruiting patients.

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

The prospect of participating in a clinical treatment trial that has the potential to improve function, prognosis, and quality of life can give families and individuals living with SCD a lot of hope. But with that hope may come an emotional rollercoaster that brings elation and anxiety as families learn about study eligibility and what is involved in participation. A family’s best approach is education about the research goals, eligibility, process, and pros and cons of study participation. Such information comes from resources like CSCF, Inc. and from open communications with the study team.

Much of today’s medical care is based on the results from past clinical studies. Clinical trials help researchers assess the effectiveness of various types of treatments, be they new medications, medical devices or physical therapies – all in a carefully controlled setting.

In addition to helping others by contributing to the advancement of medical knowledge, participants in clinical trials can play a more active role in their own health care. They can also gain access to new treatments before they are widely available.

How to Participate in a Clinical Study

If, after reviewing these studies, you would like to participate in one, please call or send an email to the person listed as the contact or discuss the study with your/your child’s doctor to see if he or she will recommend you/your child for participation. Families are strongly encouraged to read Understanding Clinical Trials on the Nationwide Newborn Screening website before making a decision about participating in clinical trials.

Research studies investigating various aspects of sickle cell disease
  • Sickle Cell Acute Chest Syndrome Treatment with Prasugrel – Phase III — 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. For information about the study or enrollment, please contact: Study Coordinator Angela Martino, BSN, RN 412-692-6467

  • 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.

  • 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.

  • 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.

  • INSIGHTS Study on Sickle Cell Disease and Leg Ulcers — The purpose of this study is to 1) understand the role of the skin microbiome in formation and delay in the healing of leg ulcers in sickle cell disease, 2) understand genomic, environmental, and social factors that may contribute to better health an complications of sickle cell disease. For more information, call 301-451-3997 or email SCDLegUlcerStudy@mail.nih.gov.

  • 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.

  • 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.

  • Conduct a general search for studies. — The Find Studies. section of the ClinicalTrials.gov site describes all the options for finding studies on the site, how to use those options to find the studies you are looking for, and how to read study records.
Acute Chest Syndrome studies in sickle cell disease
Bone marrow transplantation studies in sickle cell disease
  • The Haplo SCD Consortium — The Haplo SCD Consortium offers Parent-To-Child (haplo) bone marrow transplants for sickle cell disease (SCD). The consortium offers a multi-institutional phase II clinical trial that uses half-matched family donors to try and cure sickle cell disease in children ages 2-20. Led by Dr. Mitchell Cairo (Principal investigator) at New York Medical College, the study is enrolling at 5 transplant centers across the country. While using HLA-matched siblings, as transplant donors has been successful in curing sickle cell disease, not everyone has a full HLA matched donor available. The purpose of this study is to repeat that same success for children who do not have fully matched donors. To learn more, please visit www.clinicaltrials.gov (NCT01461837) or our study homepage. Contacts: Dr. Mitchell Cairo mitchell_cairo@nymc.edu at 914-594-2150 or Erin Morris, RN (Study coordinator) at 714-964-5359 to learn more. Participating transplant centers: To find a participating transplant center near you, please visit our website http://www.sicklecelltransplantconsortium.org/transplant-facilities-near-you.html

  • Sickle Cell Transplantation to Prevent Disease ExacerbationSickle Cell Transplantation to Prevent Disease Exacerbation (STRIDE) is a study focused on young adults with severe sickle cell disease (SCD) to better understand the safety and feasibility of bone marrow transplantation (BMT) preceded by a special medication regimen called Reduced Intensity Conditioning (RIC). The use of RIC prior to BMT, a form of stem cell therapy, has been shown to cure SCD, but has been performed almost exclusively in children with matched sibling donors. This study is a first step to help to determine if BMT using RIC can be successful in older patients, and possibly those without matched family donors. For information or to enroll, please contact Mark Vander Lugt, MD 412-692-5055 or Melissa Byrne, MPH, RN 412-692-7336.

  • 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
  • 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.

  • 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 and sickle cell disease
  • 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.

  • 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.
About the Vascular Medicine Institute

The Vascular Medicine Institute at the University of Pittsburgh (VMI) was founded in 2008 with the support of the Institute for Transfusion Medicine and the Hemophilia Center of Western Pennsylvania. It is part of the Department of Medicine in the University of Pittsburgh School of Medicine, and our multidisciplinary team of scientists have appointments in the Division of Pulmonary, Allergy and Critical Care Medicine, the Division of Hematology/Oncology, the Department of Pharmacology & Chemical Biology, and the Department of Pathology.

Our partnership with the Vascular Medicine Institute helps to offer research grants to young researchers to spawn innovation with regard to sickle cell disease. We are raising funds that will support research to find better treatments, medicines and for a universal CURE for sickle cell disease!

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

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.

There are many ways you can donate your financial resources and personal talents to the cause.