A Phase II Trial of Regadenoson in Sickle Cell Anemia

• Status: Completed
• Trial end date: 2016-12-12
• Target enrollment: 0
• Participant gender: All

Summary

This research study is a Phase II clinical trial, which tests the safety and effectiveness of an investigational drug called Regadenoson (or Lexiscan) to learn whether the drug works in treating a specific disease, in this case Sickle Cell Disease (SCD). "Investigational" means that the drug is being studied. It also means that the FDA has not yet approved the drug for your type of disease.

SCD is an inherited blood disorder that causes the red blood cells to change their shape from a round shape to a half-moon/crescent or sickled shape. People who have SCD have a different type of protein that carries oxygen in their blood (hemoglobin) than people without SCD. This different type of hemoglobin makes the red blood cells change into crescent shape under certain conditions. Sickle-shaped cells are a problem because they often get stuck in the blood vessels blocking the flow of blood, and cause inflammation and injury to important areas in the body.

Regadenoson (trade name Lexiscan) is a drug that may prevent this inflammation and injury caused by the sickle shaped cells. This drug is approved by the FDA to be used as a fast infusion during a heart stress test in people who are unable to exercise enough to put stress on their heart by making the heart beat faster. Regadenoson has been studied as a long infusion at this dose in adults, and no safety issues have been identified (ClinicalTrials.gov Identifier: NCT01085201). This is the first study to look at patient benefit with the long infusion of the drug. This drug has been used in laboratory experiments and information from those other research studies suggests that this drug may help to protect the body from damage caused by sickle-shaped cells in this research study.

In this research study, the investigators are specifically looking to see if Regadenoson is an effective treatment for pain crises and acute chest syndrome in SCD.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Dana-Farber Cancer Institute
• Collaborators: Baylor College of Medicine; Boston Children's Hospital; Boston Children’s Hospital; Brigham and Women's Hospital; Children's Hospital & Research Center Oakland; Children's Hospital Medical Center, Cincinnati; Duke University; Johns Hopkins University; La Jolla Institute for Allergy & Immunology; Medical College of Wisconsin; National Heart, Lung, and Blood Institute (NHLBI); UCSF Benioff Children's Hospital Oakland; University of Illinois at Chicago; Washington University School of Medicine; Wayne State University
• Treatments: Regadenoson

Criteria

Inclusion Criteria:

• Must have sickle cell anemia confirmed by hemoglobin analysis

• Must be admitted to hospital for pain or ACS

• Reliable IV access as determined by the study physician

• Participants must have the laboratory indices as defined below:

• Hemoglobin ≥ 5 g/dL

• Platelets > 100,000/mcL

• ALT (SGPT) < 3 X institutional upper limit of normal

• Serum creatinine ≤ 1.5 mg/dL

• INR ≤2.0, PTT ≤ 48 seconds

Exclusion Criteria:

• Pregnant or breastfeeding

• Current physician diagnosis of asthma defined by treatment with systemic corticosteroids within the last 12 months or predicted/current use of asthma controller medications

• 10 or more hospitalizations for pain in the last 12 months

• Receiving regularly scheduled transfusions

• Severe ACS

• Second or third degree AV block or sinus node dysfunction

• History of a bleeding diathesis

• History of clinically overt stroke within 3 years

• History of severe hypertension not adequately controlled with anti-hypertensive medications

• Receiving chronic anti-coagulation or anti-platelet therapy

• History of metastatic cancer

• Receiving any other study agents or have received a study agent in the past 30 days

• Uncontrolled intercurrent illness

• Known HIV

• Have previously enrolled and received the investigational agent as part of this study

• Taking medications that may interact with the investigational agent

• Have previously undergone a hematopoietic stem cell transplant or solid organ transplant