A Phase II Trial of Regadenoson in Sickle Cell Anemia

This study is currently recruiting participants. (see Contacts and Locations)
Verified August 2015 by Dana-Farber Cancer Institute
Brigham and Women's Hospital
Children's Hospital Boston
La Jolla Institute for Allergy & Immunology
National Heart, Lung, and Blood Institute (NHLBI)
Washington University School of Medicine
Children's Hospital Medical Center, Cincinnati
University of Illinois at Chicago
Medical College of Wisconsin
Duke University
Johns Hopkins University
Wayne State University
Baylor College of Medicine
Information provided by (Responsible Party):
David G. Nathan, MD, Dana-Farber Cancer Institute
ClinicalTrials.gov Identifier:
First received: February 7, 2013
Last updated: August 6, 2015
Last verified: August 2015

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 Intervention Phase
Sickle Cell Anemia
Drug: Regadenoson
Drug: Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator)
Primary Purpose: Treatment
Official Title: A Phase II, Randomized, Placebo-Controlled Trial of Regadenoson in Sickle Cell Anemia

Resource links provided by NLM:

Further study details as provided by Dana-Farber Cancer Institute:

Primary Outcome Measures:
  • Determine iNKT cell reduction with Regadenoson vs. Placebo [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    To determine if infusional regadenoson reduced iNKT cell activation among individuals with SCA and pain or ACS compared to placebo

Secondary Outcome Measures:
  • Impact of Regadenoson on length of hospital stay [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    To determine if regadenoson reduces length of hospital stay among individuals admitted with SCA and pain or ACS compared to placebo

  • Impact of regadenoson on respiratory symptoms [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    To determine if regadenoson improved respiratory symptoms among individuals with SCA and pain or ACS compared to placebo

  • Impact of regadenoson on opioid use [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    To determine if regadenoson reduces opioid use among individuals with SCA and pain or ACS compared to placebo

  • Impact of regadenoson on level of inflammatory markers [ Time Frame: 2 years ] [ Designated as safety issue: No ]
    To determine if regadenoson reduces levels of inflammatory markers among individuals with SCA and pain or ACS compared to placebo

Estimated Enrollment: 96
Study Start Date: May 2013
Estimated Study Completion Date: June 2017
Estimated Primary Completion Date: June 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: Regadenoson Arm
1.44 mcg/kg/hour infused over 48 hours
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
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

  Show Detailed Description


Ages Eligible for Study:   10 Years to 70 Years
Genders Eligible for Study:   Both
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

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
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01788631

Contact: David Nathan, MD 6176322155 dgnathan@partners.org

United States, Illinois
University of Illinois at Chicago Recruiting
Chicago, Illinois, United States, 60612
Contact: Victor Gordeuk, MD    312-996-5461    vgordeuk@uic.edu   
Principal Investigator: Victor Gordeuk, MD         
Sub-Investigator: Robert Molokie, MD         
Sub-Investigator: Johara Hassan, MD         
Sub-Investigator: Michel Gowhari, DO         
Sub-Investigator: Lewis Hsu, MD         
Sub-Investigator: Santosh Saraf, MD         
Sub-Investigator: Chaher Alhandalous, MD         
United States, Maryland
Johns Hopkins University Recruiting
Baltimore, Maryland, United States, 21205
Contact: Jeffrey Keefer, MD, PhD    410-955-6132    jkeefer1@jhmi.edu   
Principal Investigator: Jeffrey Keefer, MD, PhD         
Sub-Investigator: Sophie Lanzkron, MD         
United States, Massachusetts
Brigham and Women's Hospital Recruiting
Boston, Massachusetts, United States, 02215
Contact: Maureen Okam, MD, MPH    617-732-5048    mokam@partners.org   
Principal Investigator: Maureen Okam, MD, MPH         
Sub-Investigator: Ronald McCaffrey, MD         
Sub-Investigator: Nancy Berliner, MD         
Sub-Investigator: Elyse Mandell, NP         
Dana-Farber Cancer Institute Active, not recruiting
Boston, Massachusetts, United States, 02215
Boston Children's Hospital Recruiting
Boston, Massachusetts, United States, 02215
Contact: Matthew Heeney, MD    617-355-7700    mheeney@partners.org   
Principal Investigator: Matthew Heeney, MD         
Sub-Investigator: Ellis Neufeld, MD, PhD         
Sub-Investigator: Venee Tubman, MD         
United States, Michigan
Wayne State University/Karmanos Cancer Institute Recruiting
Detroit, Michigan, United States, 48201
Contact: Paul S Swerdlow, MD    313-576-8731    swerdlow@karmanos.org   
Principal Investigator: Paul Swerdlow, MD         
Sub-Investigator: Indryas Woldie, MD         
United States, Missouri
Washington University in St. Louis Recruiting
St. Louis, Missouri, United States, 63110
Contact: Elaine Majerus, MD, PhD    312-362-8866    EMajerus@dom.wustl.edu   
Principal Investigator: Elaine Majerus, MD, PhD         
United States, North Carolina
Duke University Recruiting
Durham, North Carolina, United States, 27705
Contact: Marilyn Telen, MD    919-684-5378    marilyn.telen@dm.duke.edu   
Principal Investigator: Marilyn J Telen, MD         
Sub-Investigator: Nirmish Shah, MD         
Sub-Investigator: Regina Crawford, MD         
United States, Ohio
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Contact: Charles Quinn, MD, MS    513-803-3086    Charles.quinn@cchmc.org   
Principal Investigator: Charles Quinn, MD, MS         
Sub-Investigator: Theodosia Kalfa, MD         
Sub-Investigator: Karen Kalinyak, MD         
Sub-Investigator: Punam Malik, MD         
Sub-Investigator: Omar Niss, MD         
United States, Texas
Baylor College of Medicine Not yet recruiting
Houston, Texas, United States, 77030
Contact: Alex George, MD    832-822-4583    axgeorge@texaschildrens.org   
Sub-Investigator: Gladstone Airewele, MD         
Sub-Investigator: Vivien Sheehan, MD         
Sub-Investigator: Corrie E Chumpitazi, MD         
Sub-Investigator: Mary Louise Vaughan, MS         
Sub-Investigator: Susan Edwinna Kirk, MS         
Sub-Investigator: Deborah Lynn Shardy, MD, PhD         
Sub-Investigator: Donald Mahoney, Jr., MD         
Sub-Investigator: Amber Meshell Yates, MD         
Sub-Investigator: Victor Mario Gonzales, MD         
Sub-Investigator: Bogdan Dinu, MD         
United States, Wisconsin
Medical College of Wisconsin Recruiting
Milwaukee, Wisconsin, United States, 53226
Contact: Joshua Field, MD, MS    414-937-3848    Joshua.field@bcw.edu   
Principal Investigator: Joshua Field, MD, MS         
Sub-Investigator: Patrick Foy, MD         
Sub-Investigator: Kathryn Koch, NP         
Sub-Investigator: Meenu Singh, MD         
Sponsors and Collaborators
Dana-Farber Cancer Institute
Brigham and Women's Hospital
Children's Hospital Boston
La Jolla Institute for Allergy & Immunology
National Heart, Lung, and Blood Institute (NHLBI)
Washington University School of Medicine
Children's Hospital Medical Center, Cincinnati
University of Illinois at Chicago
Medical College of Wisconsin
Duke University
Johns Hopkins University
Wayne State University
Baylor College of Medicine
Principal Investigator: David Nathan, MD Dana-Farber Cancer Institute
  More Information

No publications provided

Responsible Party: David G. Nathan, MD, Principal Investigator, Dana-Farber Cancer Institute
ClinicalTrials.gov Identifier: NCT01788631     History of Changes
Other Study ID Numbers: 13-005, 1P50HL110790-01
Study First Received: February 7, 2013
Last Updated: August 6, 2015
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Anemia, Sickle Cell
Anemia, Hemolytic
Anemia, Hemolytic, Congenital
Genetic Diseases, Inborn
Hematologic Diseases
Adenosine A2 Receptor Agonists
Molecular Mechanisms of Pharmacological Action
Neurotransmitter Agents
Pharmacologic Actions
Physiological Effects of Drugs
Purinergic Agents
Purinergic Agonists
Purinergic P1 Receptor Agonists

ClinicalTrials.gov processed this record on November 24, 2015