A Phase II Trial of Regadenoson in Sickle Cell Anemia
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ClinicalTrials.gov Identifier: NCT01788631 |
Recruitment Status :
Completed
First Posted : February 11, 2013
Results First Posted : February 7, 2018
Last Update Posted : February 7, 2018
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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.
Condition or disease | Intervention/treatment | Phase |
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Sickle Cell Anemia | Drug: Regadenoson Drug: Placebo | Phase 2 |

Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 100 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Triple (Participant, Care Provider, Investigator) |
Primary Purpose: | Treatment |
Official Title: | A Phase II, Randomized, Placebo-Controlled Trial of Regadenoson in Sickle Cell Anemia |
Study Start Date : | July 2013 |
Actual Primary Completion Date : | November 10, 2016 |
Actual Study Completion Date : | December 12, 2016 |

Arm | Intervention/treatment |
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Active Comparator: Regadenoson Arm
1.44 mcg/kg/hour infused over 48 hours
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Drug: Regadenoson
Regadenoson is an A2AR agonist that is a coronary vasodilator. It is chemically described as adenosine, 2-[4-[(methylamino)carbonyl]-1H-pyrazol-1-yl]-, monohydrate. Its molecular formula is C15H18N8O5. Regadenoson has an FDA indication for use in radionuclide myocardial perfusion imaging in patients unable to undergo adequate exercise stress. It has lower affinity for non-A2A adenosine receptor subtypes thought to be associated with some of the adverse effects associated with non-selective adenosine receptor agonists, which increase extracellular adenosine by blocking its uptake into cells. The maximal plasma concentration of regadenoson is achieved within 1 to 4 minutes after injection and parallels the onset of the pharmacodynamic response. Its half-life is approximately 2 to 4 minutes.
Other Name: Lexiscan |
Placebo Comparator: Placebo Arm
Placebo infused over 48 hours
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Drug: Placebo
This study uses 0.9% Normal Saline (NS) as placebo. This is a sterile sodium chloride solution usually used to replenish fluids and electrolytes. It contains no additives, and is a standard solution used as placebo in clinical trials where the study drug is administration intravenously. NS will be prepared by investigational pharmacy.
Other Name: Regadenoson Placebo, Lexiscan Placebo, 0.9% Normal Saline |
- Number of Participants With a Reduction in Invariant Natural-Killer T-Cell (iNKT Cell) Activation by 70% or More [ Time Frame: Baseline-End of study infusion over 48 hours ]To determine if infusional Regadenoson reduced iNKT cell activation among individuals with sickle cell anemia (SCA) and pain or acute chest syndrome (ACS) compared to placebo by 70% or greater.
- Length of Hospital Stay [ Time Frame: Hospital Presentation- Hospital Discharge, assessed up to 1 month ]To determine if regadenoson reduces length of hospital stay among individuals admitted with SCA and pain or ACS compared to placebo
- Number of Participants With an Improvement in Respiratory Symptoms [ Time Frame: Baseline-End of study infusion over 48 hours ]To determine if regadenoson improved respiratory symptoms among individuals with sickle cell anemia (SCA) and pain or acute chest syndrome (ACS) compared to placebo. Patients were classified as having an improvement in respiratory symptoms if they experienced any of the following outcomes:(1) respiratory rate decreased by 25% from baseline or normalized (≤20 bpm) or (2) degree of hypoxia (SpO2) on room air increased by 10% from baseline or normalized (≥92%) or (3) thoracic pain improved by 3 points from baseline on a 10-point visual analog scale.
- Opioid Use [ Time Frame: Baseline-End of study infusion over 48 hours ]To determine if regadenoson reduces opioid use among individuals with SCA and pain or ACS compared to placebo.
- Level of Inflammatory Markers (A2A) [ Time Frame: Baseline-End of study infusion over 48 hours ]To determine if regadenoson reduces levels of inflammatory markers among individuals with SCA and pain or ACS compared to placebo.
- Level of Inflammatory Markers (IL-4) [ Time Frame: Baseline-End of study infusion over 48 hours ]To determine if regadenoson reduces levels of inflammatory markers among individuals with SCA and pain or ACS compared to placebo.
- Level of Inflammatory Markers (IFN-gamma) [ Time Frame: Baseline-End of study infusion over 48 hours ]To determine if regadenoson reduces levels of inflammatory markers among individuals with SCA and pain or ACS compared to placebo.

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 10 Years to 70 Years (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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
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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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01788631
United States, California | |
Children's Hospital and Research Center at Oakland | |
Oakland, California, United States, 94609 | |
United States, Illinois | |
University of Illinois at Chicago | |
Chicago, Illinois, United States, 60612 | |
United States, Maryland | |
Johns Hopkins University | |
Baltimore, Maryland, United States, 21205 | |
United States, Massachusetts | |
Boston Children's Hospital | |
Boston, Massachusetts, United States, 02215 | |
Brigham and Women's Hospital | |
Boston, Massachusetts, United States, 02215 | |
Dana-Farber Cancer Institute | |
Boston, Massachusetts, United States, 02215 | |
United States, Michigan | |
Wayne State University/Karmanos Cancer Institute | |
Detroit, Michigan, United States, 48201 | |
United States, Missouri | |
Washington University in St. Louis | |
Saint Louis, Missouri, United States, 63110 | |
United States, North Carolina | |
Duke University | |
Durham, North Carolina, United States, 27705 | |
United States, Ohio | |
Cincinnati Children's Hospital Medical Center | |
Cincinnati, Ohio, United States, 45229 | |
United States, Texas | |
Baylor College of Medicine | |
Houston, Texas, United States, 77030 | |
United States, Wisconsin | |
Medical College of Wisconsin | |
Milwaukee, Wisconsin, United States, 53226 |
Principal Investigator: | David Nathan, MD | Dana-Farber Cancer Institute |
Responsible Party: | David G. Nathan, MD, Principal Investigator, Dana-Farber Cancer Institute |
ClinicalTrials.gov Identifier: | NCT01788631 |
Other Study ID Numbers: |
13-005 1P50HL110790-01 ( U.S. NIH Grant/Contract ) |
First Posted: | February 11, 2013 Key Record Dates |
Results First Posted: | February 7, 2018 |
Last Update Posted: | February 7, 2018 |
Last Verified: | January 2018 |
Anemia Anemia, Sickle Cell Hematologic Diseases Anemia, Hemolytic, Congenital Anemia, Hemolytic Hemoglobinopathies Genetic Diseases, Inborn Regadenoson |
Adenosine A2 Receptor Agonists Purinergic P1 Receptor Agonists Purinergic Agonists Purinergic Agents Neurotransmitter Agents Molecular Mechanisms of Pharmacological Action Physiological Effects of Drugs |