Do you have essential thrombocythemia (ET)?
You may be eligible to join EXCEED ET, a Phase 2b clinical research study in the US and Canada that evaluates the potential of a new, investigational ET therapy to control the number of platelets and white blood cells in the blood.
What is ET?
Essential thrombocythemia (ET) is a rare blood cancer that is characterized by several genetic mutations that lead to an increase in blood counts (the number of platelets and, in some cases, white blood cells). This increase in blood counts can cause blood clots and vascular complications. Some people with ET have no symptoms while others may develop many health problems. ET may progress to myelofibrosis or secondary acute myeloid leukemia.
Symptoms can include the following:
Headaches
Excessive bleeding (like nosebleeds or bleeding gums)
Weakness and fatigue
Burning, tingling, or prickling skin sensations
An enlarged spleen
Pain, redness, and swelling of the hands and feetET can also cause more serious health problems because of the increased risk of blood clots. These can include strokes and heart attacks, although these are rare events.
The goal of treatments for ET is to reduce the number of platelets and, in some cases, white blood cells in the blood to help reduce the symptoms and potential health risks.
This study is investigating a specific type of interferon alfa, ropeginterferon alfa-2b-njft, which reduces the number of platelets and white blood cells in the blood.
Why take part in a clinical research study?
Patient participation in clinical research studies is important in helping us learn more about potential new treatments for the community.
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 conducted
- Patient participation in any study is voluntary. Patients may leave a study at any time without penalty
Study details
The EXCEED ET study is for adults who have been diagnosed with ET who:
Have not received any treatment for their ET in the past
Have received treatments such as hydroxyurea or anagrelide for their ET, but they may have stopped working or had unbearable side effects
The purpose of this study is to evaluate the effectiveness and safety of ropeginterferon alfa-2b-njft in adult patients with ET.
Ropeginterferon alfa-2b-njft
- Type of interferon alfa
- Administered as an injection under the skin every 2 weeks
Ropeginterferon alfa-2b-njft
- Type of interferon alfa
- Administered as an injection under the skin every 2 weeks
The main study will last approximately 15 months, including a screening period, titration period, and a core treatment period. After completion of the core study period, study participants who benefitted from treatment will continue on to an extension period of the study. This portion of the study will allow investigators to evaluate the effectiveness of ropeginterferon alfa-2b-njft in the long term.
Who can take part?
Question 1 of 3
Did your doctor diagnose your ET because of elevated blood counts (platelets and, in some cases, white blood cells), mutational testing (JAK2 V617F, CALR, or MPL), and bone marrow biopsy/evaluation?
Yes
No
Question 2 of 3
Have you had any prior treatment with an interferon?
Yes
No
Question 3 of 3
Have you had any prior treatment with ruxolitinib?
Yes
No
18 years of age or older
Diagnosed with ET according to criteria, including elevated platelets, the presence of a genetic mutation (eg, JAK2 V617F, CALR, or MPL), and a bone marrow biopsy
Treated with hydroxyurea and/or anagrelide for their ET, but these may have stopped working or had unbearable side effects
Never received interferon alfa and/or ruxolitinib for their ET in the past
Study participants will need to meet additional eligibility criteria to be accepted to enroll in the study.
What to expect
If you agree to participate in the clinical study, you will visit the study clinic and undergo a series of assessments to find out if you meet all the study requirements before beginning participation. If you meet all the requirements, you can then begin the study treatment period. Once you have completed the main study treatment period (up to 15 months), you may continue on to the extension period of the study.
Up to 4 weeks
Screening period
- Attend screening visits and provide a medical history
- Undergo assessments and evaluations to determine if you meet the study requirements to participate in EXCEED ET
4 weeks
Titration period
- Undergo assessments and evaluations to determine if changes to dose are required
- Receive dose adjustments to ropeginterferon alfa-2b-njft to reach your optimal dose or switch treatments (eg, hydroxyurea or anagrelide) to ropeginterferon alfa-2b-njft
12 months*
Core study period
- Receive ropeginterferon alfa-2b-njft every 2 weeks
- Attend study visits to monitor your health and how you are responding to treatment. Some of these will be in-person visits to your study clinic while others may be phone visits
Up to 2 years*
Extension study period
- Continue to receive ropeginterferon alfa-2b-njft every 2 weeks
- Attend study visits to monitor your health and how you are responding to treatment. Some of these will be in-person visits to your study clinic while others may be phone visits
Up to 4 weeks after last treatment visit
Study follow-up
- Attend the final study visit to monitor your health and how you responded to treatment
*
The length of time for which you receive treatment will vary depending on how you respond to the study drug.
*
For more information about the EXCEED ET clinical study, please visit ClinicalTrials.gov (trial identifier: NCT05482971).
Where are the clinical study sites in the US and Canada?
Find out if there’s a study site in your area using the table below. For additional information, please contact us by emailing clinicaltrials@pharmaessentia-us.com or call 1-800-999-2449.
- Juravinski Cancer Centre
- Hamilton, ON
- Princess Margaret Hospital
- Toronto, ON
- Yale University School of Medicine - Yale Cancer Center
- New Haven, CT
- John Theurer Cancer Center at Hackensack UMC
- Hackensack, NJ
- Montefiore Medical Center
- Bronx, NY
- Fox Chase Cancer Center
- Philadelphia, PA
- The Winship Cancer Institute Emory University
- Atlanta, GA
- East Carolina University
- Greenville, NC
- MD Anderson Cancer Center
- Houston, TX
- University of Texas Health Science Center at San Antonio
- San Antonio, TX
- University of Virginia - Emily Couric Cancer Center
- Charlottesville, VA
- Mayo Clinic - Scottsdale
- Scottsdale, AZ
- Marin Cancer Care
- Greenbrae, CA
- Tulane University Medical Center
- New Orleans, LA
- University of Utah
- Salt Lake City, UT
- University of Michigan
- Ann Arbor, MI
- Washington University School of Medicine - Division of Oncology
- St. Louis, MO
- Cleveland Clinic
- Cleveland, OH
How can I enroll in EXCEED ET?
Discuss with your doctor
If you think you may be eligible to enroll in EXCEED ET, print this page or email it to your doctor and discuss it at your next visit.
Questions?
For more information, contact our Medical Information team at medinfo@pharmaessentia-us.com.
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Updated 01/2023