Exploratory Multi-centre Trial In Patients With ET Treated With XAGRID® (EMIX)

An Exploratory, Observational, Multicentre Study to Investigate the Impact of the Presence of JAK2 (V617F) Mutation on Treatment Response in Patients With Essential Thrombocythaemia Treated With XAGRID® (Anagrelide Hydrochloride)

This study is hypothesis-generating to explore the impact of JAK2 (V617F) mutation status on the treatment response to anagrelide hydrochloride

Study Overview

• Status: Completed
• Conditions: Essential Thrombocythemia (ET)
• Intervention / Treatment: Drug: Anagrelide hydrochloride

• Study Type: Observational
• Enrollment (Actual): 47

Contacts and Locations

Study Locations

• Italy
  • Bari, Italy
    • Azienda Ospedaliera Policlinico Di Bari
  • Bologna, Italy
    • Istituto Seragnoli-Policlinico S.Orsola-Malpighi
  • Brescia, Italy
    • A.O. Spedali Civili di Brescia
  • Firenze, Italy
    • Azienda Ospedaliero-Universitaria Careggi
  • Genova, Italy
    • Ospedale San Martino
  • Milan, Italy, 20125
    • Fondazione IRCCS Ca' Granda
  • Napoli, Italy, 80131
    • Azienda Ospedaliera Universitaria "Federico II"
  • Novara, Italy
    • Ospedale Maggiore della Carita
  • Roma, Italy
    • Policlinico A. Gemelli
  • Terni, Italy
    • Padiglione ex-oncologico Ospedale S.Maria

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: ADULT; OLDER_ADULT; CHILD
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

ET patients in Italy

Description

Inclusion Criteria:

1. Patients must be able to understand and willing to participate in the study, and provide a personally dated and signed written informed consent form.
2. Patients must have a confirmed diagnosis of ET according to the World health Organisation's criteria.
3. ET patients who are uncontrolled, in the Investigator's opinion, by first-line (or previous) cytoreductive treatment for efficacy or tolerance reasons.
4. Patients who have either commenced treatment with anagrelide hydrochloride in the last 7 days or for whom a decision has been documented to commence treatment with anagrelide hydrochloride

Exclusion Criteria:

1. Patients for whom treatment with anagrelide hydrochloride is contraindicated, according to the current XAGRID SmPC.
2. Known or suspected intolerance or hypersensitivity to the product, closely related compounds, or any of the stated ingredients.
3. Patients participating in an interventional research study.
4. Patients on combination therapy or for whom there is an intention to treat with other cytoreductive agents e.g., hydroxyurea, interferon. Patients can however use aspirin and other anti-aggregatory products at the Investigator's discretion.

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
JAK2 Positive Participants	Drug: Anagrelide hydrochloride; 0.5 mg hard capsules, dosing decisions will be made by the treating physician; Other Names: Xagrid
JAK2 Negative Participants	Drug: Anagrelide hydrochloride; 0.5 mg hard capsules, dosing decisions will be made by the treating physician; Other Names: Xagrid

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Patients With Platelet Count ≤600x10^9/L After 12 Months	A platelet count of ≤600x10^9/L after 12 months is considered at least a partial response.	1 year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Patients With Platelet Count ≤400x10^9/L After 12 Months	A platelet count of ≤400x10^9/L after 12 months is considered a complete response.	1 year
Platelet Count		1 year
Red Blood Cell (RBC) Count		1 year
White Blood Cell (WBC) Count		1 year
Differential WBC Count - Neutrophils, Lymphocytes, Monocytes, and Basophils.		1 year
Differential WBC Count - Eosinophils		1 year
Hemoglobin Concentration		1 year
Hematocrit Level	The proportion of the volume of red blood cells to the total volume of blood.	1 year

Collaborators and Investigators

• Sponsor: Shire

Publications and helpful links

General Publications

• Cascavilla N, De Stefano V, Pane F, Pancrazzi A, Iurlo A, Gobbi M, Palandri F, Specchia G, Liberati AM, D'Adda M, Gaidano G, Fjerza R, Achenbach H, Smith J, Wilde P, Vannucchi AM. Impact of JAK2(V617F) mutation status on treatment response to anagrelide in essential thrombocythemia: an observational, hypothesis-generating study. Drug Des Devel Ther. 2015 May 18;9:2687-94. doi: 10.2147/DDDT.S79576. eCollection 2015.

Study record dates

Study Major Dates

• Study Start (ACTUAL): July 19, 2011
• Primary Completion (ACTUAL): September 19, 2013
• Study Completion (ACTUAL): September 19, 2013

Study Registration Dates

• First Submitted: May 10, 2011
• First Submitted That Met QC Criteria: May 11, 2011
• First Posted (ESTIMATE): May 12, 2011

Study Record Updates

• Last Update Posted (ACTUAL): July 30, 2021
• Last Update Submitted That Met QC Criteria: July 6, 2021
• Last Verified: July 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Bone Marrow Diseases; Hematologic Diseases; Hemorrhagic Disorders; Myeloproliferative Disorders; Blood Coagulation Disorders; Blood Platelet Disorders; Thrombocytosis; Thrombocythemia, Essential; Molecular Mechanisms of Pharmacological Action; Fibrinolytic Agents; Fibrin Modulating Agents; Platelet Aggregation Inhibitors; Anagrelide
• Other Study ID Numbers: SPD422-703