Clinical trial NCT01352585: Exploratory Multi-centre Trial In Patients With ET Treated With XAGRID®
Official title: 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)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Essential Thrombocythemia (ET)
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2011-05-12
Enrollment: 47
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: ET patients in Italy

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


Eligibility criteria: 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.

Primary outcomes:
measure: Number of Patients With Platelet Count ≤600x10^9/L After 12 Months
timeFrame: 1 year
Secondary outcomes:
measure: Number of Patients With Platelet Count ≤400x10^9/L After 12 Months
timeFrame: 1 year
measure: Platelet Count
timeFrame: 1 year
measure: Red Blood Cell (RBC) Count
timeFrame: 1 year
measure: White Blood Cell (WBC) Count
timeFrame: 1 year
measure: Differential WBC Count - Neutrophils, Lymphocytes, Monocytes, and Basophils.
timeFrame: 1 year
measure: Differential WBC Count - Eosinophils
timeFrame: 1 year
measure: Hemoglobin Concentration
timeFrame: 1 year
measure: Hematocrit Level
timeFrame: 1 year
Other outcomes:
Locations:
city: Bari
country: Italy
facility: Azienda Ospedaliera Policlinico di Bari
city: Bologna
country: Italy
facility: Istituto Seragnoli-Policlinico S.Orsola-Malpighi
city: Brescia
country: Italy
facility: A.O. Spedali Civili di Brescia
city: Firenze
country: Italy
facility: Azienda Ospedaliero-Universitaria Careggi
city: Genova
country: Italy
facility: Ospedale San Martino
city: Milan
country: Italy
facility: Fondazione IRCCS Ca' Granda
city: Napoli
country: Italy
facility: Azienda Ospedaliera Universitaria "Federico II"
city: Novara
country: Italy
facility: Ospedale Maggiore della Carita
city: Roma
country: Italy
facility: Policlinico A. Gemelli
city: Terni
country: Italy
facility: Padiglione ex-oncologico Ospedale S.Maria
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References:
citation: 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.