A Phase II Trial of Regadenoson in Sickle Cell Anemia

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.

Conditions

• Sickle Cell Anemia

Interventions

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.

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.

Sponsors & Collaborators

• Brigham and Women's Hospital

  collaborator OTHER

• Boston Children's Hospital

  collaborator OTHER

• La Jolla Institute for Allergy & Immunology

  collaborator OTHER

• National Heart, Lung, and Blood Institute (NHLBI)

  collaborator NIH

• Washington University School of Medicine

  collaborator OTHER

• Children's Hospital Medical Center, Cincinnati

  collaborator OTHER

• University of Illinois at Chicago

  collaborator OTHER

• Medical College of Wisconsin

  collaborator OTHER

• Duke University

  collaborator OTHER

• Johns Hopkins University

  collaborator OTHER

• Wayne State University

  collaborator OTHER

• Baylor College of Medicine

  collaborator OTHER

• UCSF Benioff Children's Hospital Oakland

  collaborator OTHER

• Dana-Farber Cancer Institute

  lead OTHER

Principal Investigators

• David Nathan, MD · Dana-Farber Cancer Institute

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Model

PARALLEL

Eligibility

Min Age

10 Years

Max Age

70 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2013-07-31

Primary Completion

2016-11-10

Completion

2016-12-12

Countries

• United States

Study Locations