What are clinical research studies?

Clinical research studies explore whether a therapy, device, or test is safe and improves people’s health.

Before any therapy is
approved and made available to the public, it goes through several phases of clinical research.

Every country has strict rules about how clinical research is completed.

Patient participation helps elevate the knowledge on potential new treatments for the community.

Patient participation in a study is voluntary. Patients may leave a study at any time without penalty.

Has your essential thrombocythemia (ET) therapy stopped working?

You may be eligible to join a Phase 3 clinical research study in the U.S. that evaluates the potential of a new, investigational ET therapy to reduce the number of platelets in the blood.

About the Trial

This Phase 3 clinical trial will assess the long-term safety and efficacy of ropeginterferon alfa-2b (P1101)

Where are the clinical study sites in the U.S. and Canada?

(Rollover locations to view contact information)

MD Anderson Cancer Center
Principal Investigator: Dr Srdan Verstovsek
Study Coordinator: Nichole Ard
Site Phone: 713-745-4657
Site Email: nard@mdanderson.org

University of Texas Health Science Center at San Antonio
Principal Investigator: Dr Ruben Mesa
Study Coordinator: Cynthia Kirk
Site Phone: 210-450-8508 (office)
Site Email: kirkc1@uthscsa.edu

Weill Medical College of Cornell University
Principal Investigator: Dr Ghaith Abu-Zeinah
Study Coordinator: Roland Babmatee
Site Phone: N/A
Site Email: rob4010@med.cornell.edu

Mount Sinai Medical Center
Principal Investigator: Dr John Mascarenhas
Study Coordinator: Mikaela Dougherty
Site Phone: N/A
Site Email: mikaela.dougherty@mssm.edu

Froedtert Hospital and the Medical College of Wisconsin
Principal Investigator: Dr Laura Michaelis
Study Coordinator: Deepa Pereira
Site Phone: N/A
Site Email: dpereira@mcw.edu

Washington University School of Medicine
Principal Investigator: Dr Stephen Oh
Study Coordinator: Nicole Gaudin
Site Phone: 314-747-7960
Site Email: nrgaudin@wustl.com

Mayo Clinic - Scottsdale
Principal Investigator: Dr Jeanne Palmer
Study Coordinator: Chris Reed Peace
Site Phone: 480-342-6068
Site Email: reedpease.christine@mayo.edu

University of Michigan
Principal Investigator: Dr Kristen Pettit
Study Coordinator: Stephen Tryban
Site Phone: 734-615-0052
Site Email: stephtho@med.umich.edu

University of Utah
Principal Investigator: Dr Tsewang Tashi
Study Coordinator: Karen Pena
Site Phone: 801-213-5601
Site Email: karen.pena@hci.utah.edu

University of Kansas Cancer Center
Principal Investigator: Dr Abdulraheem Yacoub
Study Coordinator: Marc Morrison
Site Phone: 913-588-7585
Site Email: mmorrison3@kumc.edu

MD Anderson Cancer Center
Principal Investigator: Srdan Verstovsek, MD
Study Coordinator: Nichole Ard
Site Phone:
Site Email: nard@mdanderson.org

Princess Margaret Cancer Centre
Principal Investigator: DawnMaze
Study Coordinator: Nicole Sokolsky
Site Phone: 416-946-4501 ext. 5834
Site Email: dawn.maze@uhn.ca.

St. Paul’s Hospital
Principal Investigator: Linda Foltz
Study Coordinator: Tathiana Ruiz
Site Phone: 604-682-2344 ext. 64987
Site Email: N/A

Jewish General Hospital
Principal Investigator: Shireen Sirhan
Study Coordinator: Chadi Zakaria
Site Phone: 514-340-8222 Ext 28326
Site Email: chadi.zakaria.ccomtl@ssss.gouv.qc.ca

What is Essential Thrombocythemia?

Essential Thrombocythemia

Essential thrombocythemia (ET) is a condition characterized by an excessive increase of the number of platelets in your blood. Platelets cause blot clots and vascular complications. Some people with ET have no symptoms, while others develop many health problems due to additional platelets in the blood.

Symptoms can include the following:

Weakness and fatigue
Burning, tingling, or prickling skin sensations
Excessive bleeding (like nosebleeds or bleeding gums)
Headaches
Pain, redness, and swelling of the hands and feet
An enlarged spleen

ET can also cause more serious problems from increased risk of blood clots, including strokes and heart attacks, although these are rare events.
Controlling the number of platelets in the blood can help to reduce these
symptoms and health risks.

The P1101 ET Study

The P1101 ET study is for people with ET, who have tried other therapy options, like hydroxyurea, that are no longer effective at controlling their ET or that have such extreme side effects that people stop using them. The goal of this study is to compare the new study therapy, ropeginterferon alfa-2b (P1101), to the approved ET therapy, anagrelide.

The study therapy (P1101) is considered investigational because it has not been approved for public use in ET and can only be used in clinical research studies like this P1101 ET study. The study therapy (P1101) is designed to reduce the number of platelets in the blood. It is given as an injection under the skin every 2 weeks.

Who can participate in the study?

You may qualify to participate in the study if you meet basic study requirements*:

18 years of age or older
Diagnosed with high-risk ET (defined as having disease-related blood clots or bleeding in the past and/or being over 60 years of age and JAK2 positive)
Received hydroxyurea for ET
Never received interferon therapy
*Other study requirements will apply.

What is the study therapy?

PharmaEssentia

Study Overview—What To Expect

Study participation will last about 14 months and is divided into 3 study periods:

You will be randomly assigned to receive either the study therapy (P1101) or the approved ET therapy (anagrelide). You will have an equal chance of receiving either therapy. You and your study doctor will know your therapy assignment.
You will come to the study center every 2 weeks. You will have study tests and procedures (like those during Study Qualification) to monitor your health during the study. If you are assigned the study therapy (P1101), you will receive an injection at each study visit. If you are assigned the approved ET therapy (anagrelide), you will take this daily at home and bring your bottle to each 2-week visit, so the study staff can make sure you are following instructions and find out if your dose needs to change.
In addition to the study therapy (P1101) or approved ET therapy (anagrelide), you will also take low-dose aspirin as prescribed by your doctor unless you are medically unable to take aspirin.

Where are the clinical study sites in the U.S. and Canada?

(Rollover locations to view contact information)

MD Anderson Cancer Center
Principal Investigator: Dr Srdan Verstovsek
Study Coordinator: Nichole Ard
Site Phone: 713-745-3429
Site Email: nard@mdanderson.org

University of Texas Health Science Center at San Antonio
Principal Investigator: Dr Ruben Mesa
Study Coordinator: Epp Goodwin
Site Phone: 210-450-5798
Site Email: goodwine@uthscsa.edu

Weill Medical College of Cornell University
Principal Investigator: Dr Ghaith Abu-Zeinah
Study Coordinator: Niamh Savage
Site Phone: 212-746-0284
Site Email: nis2049@med.cornell.edu

Mount Sinai Medical Center
Principal Investigator: Dr John Mascarenhas
Study Coordinator: Lonette Sandy
Site Phone: 212-241-4546
Site Email: lonette.sandy@mssm.edu

Froedtert Hospital and the Medical College of Wisconsin
Principal Investigator: Dr Laura Michaelis
Study Coordinator: Paulette Jacobs
Site Phone: 414-805-4196
Site Email: pjacobs@mcw.edu

Washington University School of Medicine
Principal Investigator: Dr Stephen Oh
Study Coordinator: Karyn Gordon
Site Phone: 314-362-0156
Site Email: kdgordon@wustl.edu

Mayo Clinic - Scottsdale
Principal Investigator: Dr Jeanne Palmer
Study Coordinator: Crystal Anderson
Site Phone: 855-776-0015
Site Email: anderson.crystal2@mayo.edu