Sickle Cell Disease Research and Clinical Trials

At Duke, we are dedicated to looking into new and effective therapies for sickle cell disease with the ultimate goal of finding a cure accessible to all. 

The understanding of the underlying issues associated with sickle cell disease grows each day and we are committed to our research efforts to keep this learn even more. This includes studying new treatments, innovative technology and participating in large multi institutional trials. We have investigators conducting research studies across many interest areas in hopes of improving the lives of people with 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.

Adult Sickle Cell Disease Research and Clinical Trials

A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate The Efficacy and Safety of Rivipansel (GMI-1070) in The Treatment of Vaso-Occlusive Crisis in Hospitalized Subjects with Sickle Cell Disease

Who can participate: Patients with sickle cell disease, aged 15 to 45, who have sickle cell disease and are receiving HU therapy.

About the study: This multi-institutional study led at Duke by Drs. Rothman and Telen is evaluating the use of a novel therapeutic agent that specifically focuses on adhesive proteins to help blood flow. Patients admitted for pain crisis are given an infusion of the study drug to determine if it can more rapidly improve pain and discharge from the hospital. Dr. Telen led the preceding phase 2 multicenter study, and Duke hopes to be a leading site for this pivotal phase 3 study.

Who to contact: Angela Braswell, LPN, 919-681-8645, to learn about eligibility requirements for this study.

Rejuvesol® Washed RBC in Sickle Cell Patients Requiring Frequent Transfusions.

Who can participate: Patients with sickle cell disease, aged 18 to 45, who have sickle cell disease and are receiving chronic transfusions.

About the study: This study will evaluate the use of Rejuvesol®, which ‘rejuvenates’ blood prior to transfusions in an effort to improve the life of red blood cells (RBCs) transfused to SCD patients. This study is being performed in conjunction with UNC and will compare patients with ‘rejuvenated’ blood versus standard of care. The goal is to potentially use this to extend the life of RBCs and therefore, allow patients to receive fewer transfusions. The study is ongoing and hopes to complete patient follow up within the next year.

Who to contact: Betty Thames, RN, 919-681-9564, to learn about eligibility requirements for this study.

Sickle Cell Disease Implementation Consortium

Who can participate: Adults and adolescents with sickle cell disease aged 15 or older.

About the study: Duke has been awarded a large 6 year U01 grant as one of eight sites around the US, to examine barriers to care, develop a registry for a total of 2400 patients, and implement a clinical intervention to improve outcomes in SCD. Included in our grant support are two patient advocates as key members to provide input to our study and assist in performing our intervention as peer mentors. Importantly, a key component of our intervention is a self-developed mobile app (SCD toolbox), which was developed in collaboration with CCNC and state based leaders in SCD.

Who to contact: Nirmish Shah, MD, co-Principal Investigator; Paula Tanabe, RN, co-Principal Investigator; Study Coordinator – Sheila Lambert-Adams: 919-684-2406 to learn about eligibility requirements.

Patient-Empowered Mobile Technology in Hospitalized Patients: Technology Resources to Understand Pain Assessment in Patients with Pain (TRU-PAIN).

Who can participate: Patients, aged 8 to 80, with sickle cell disease admitted to the hospital with pain.

About the study: This study led by Dr. Shah was recently funded by Duke to evaluate the use of the self-developed SMART mobile app with wearable technology for patients with SCD admitted for pain crisis. The goal is to follow objective data such as heart rate and activity along with subjective symptoms such as pain. Analysis will be performed to find correlations between subjective and objective data, with the ultimate goal of predicting pain prior to it increasing significantly. The study continues to enroll both pediatric and adult patients and is currently being analyzed by a group of 7 statisticians and mathematicians who are leaders in big data analytics.

Who to contact: Nirmish Shah, MD, 919-684-8111, to learn about eligibility requirements for this study.

Collection of blood from volunteers and patients for studies of vascular 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 understand and develop new laboratory tests to diagnose how red cell diseases such as sickle cell disease affect how your red blood cells, white blood cells, platelets and endothelial cells function.  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 and how we should direct treatment to relieve the diseases’ symptoms.