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

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)

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT01352585

Acronym

EMIX

Enrollment

Registered

2011-05-12

Start date

2011-07-19

Completion date

2013-09-19

Last updated

2021-07-30

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Essential Thrombocythemia (ET)

Brief summary

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

Interventions

DRUGAnagrelide hydrochloride

0.5 mg hard capsules, dosing decisions will be made by the treating physician

Study design

Observational model

CASE_ONLY

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Healthy volunteers

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.

Design outcomes

Primary

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

Secondary

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

Countries

Italy

Participant flow

Participants by arm

Arm	Count
JAK2 Positive Participants Anagrelide hydrochloride: 0.5 mg hard capsules, dosing decisions will be made by the treating physician	23
JAK2 Negative Participants Anagrelide hydrochloride: 0.5 mg hard capsules, dosing decisions will be made by the treating physician	24
Total	47

Withdrawals & dropouts

Period	Reason	FG000	FG001
Overall Study	Adverse Event	2	1
Overall Study	Lost to Follow-up	1	0
Overall Study	Other	5	2

Baseline characteristics

Characteristic	JAK2 Positive Participants	JAK2 Negative Participants	Total
Age, Continuous	57.4 years STANDARD_DEVIATION 14	58.6 years STANDARD_DEVIATION 15.94	58.0 years STANDARD_DEVIATION 14.87
Region of Enrollment Italy	23 Participants	24 Participants	47 Participants
Sex: Female, Male Female	14 Participants	14 Participants	28 Participants
Sex: Female, Male Male	9 Participants	10 Participants	19 Participants

Adverse events

Event type	EG000 affected / at risk	EG001 affected / at risk
deaths Total, all-cause mortality	— / —	— / —
other Total, other adverse events	6 / 23	9 / 24
serious Total, serious adverse events	0 / 23	0 / 24

Outcome results

Primary

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.

Time frame: 1 year

Population: The Full Analysis Set (FAS) in each JAK2 status group who had a viable platelet sample during the 12-month follow-up window. For this Outcome Measure 35 patients in the FAS had a viable laboratory sample.

Arm	Measure	Value (NUMBER)
JAK2 Positive Participants	Number of Patients With Platelet Count ≤600x10^9/L After 12 Months	12 participants
JAK2 Negative Participants	Number of Patients With Platelet Count ≤600x10^9/L After 12 Months	14 participants

Secondary

Differential WBC Count - Eosinophils

Time frame: 1 year

Population: The Full Analysis Set (FAS) in each JAK2 status group who had a viable platelet sample during the 12-month follow-up window. For this Outcome Measure 27 patients in the FAS had a viable laboratory sample.

Arm	Measure	Value (MEAN)	Dispersion
JAK2 Positive Participants	Differential WBC Count - Eosinophils	2.95 percent	Standard Deviation 1.958
JAK2 Negative Participants	Differential WBC Count - Eosinophils	2.77 percent	Standard Deviation 1.906

Secondary

Differential WBC Count - Neutrophils, Lymphocytes, Monocytes, and Basophils.

Time frame: 1 year

Population: The Full Analysis Set (FAS) in each JAK2 status group who had a viable platelet sample during the 12-month follow-up window. For this Outcome Measure 28 patients in the FAS had a viable laboratory sample.

Arm	Measure	Group	Value (MEAN)	Dispersion
JAK2 Positive Participants	Differential WBC Count - Neutrophils, Lymphocytes, Monocytes, and Basophils.	Neutrophils	62.05 percent	Standard Deviation 4.429
JAK2 Positive Participants	Differential WBC Count - Neutrophils, Lymphocytes, Monocytes, and Basophils.	Lymphocytes	27.53 percent	Standard Deviation 4.412
JAK2 Positive Participants	Differential WBC Count - Neutrophils, Lymphocytes, Monocytes, and Basophils.	Monocytes	6.50 percent	Standard Deviation 2.235
JAK2 Positive Participants	Differential WBC Count - Neutrophils, Lymphocytes, Monocytes, and Basophils.	Basophils	1.75 percent	Standard Deviation 2.425
JAK2 Negative Participants	Differential WBC Count - Neutrophils, Lymphocytes, Monocytes, and Basophils.	Basophils	0.81 percent	Standard Deviation 0.486
JAK2 Negative Participants	Differential WBC Count - Neutrophils, Lymphocytes, Monocytes, and Basophils.	Neutrophils	60.07 percent	Standard Deviation 13.515
JAK2 Negative Participants	Differential WBC Count - Neutrophils, Lymphocytes, Monocytes, and Basophils.	Monocytes	7.21 percent	Standard Deviation 2.68
JAK2 Negative Participants	Differential WBC Count - Neutrophils, Lymphocytes, Monocytes, and Basophils.	Lymphocytes	28.76 percent	Standard Deviation 12.381

Secondary

Hematocrit Level

The proportion of the volume of red blood cells to the total volume of blood.

Time frame: 1 year

Population: The Safety Analysis Set (SAS) consisted of all subjects enrolled in the study who took at least 1 dose of anagrelide hydrochloride. 27 patients in the SAS had a viable laboratory sample.

Arm	Measure	Value (MEAN)	Dispersion
JAK2 Positive Participants	Hematocrit Level	0.412 Hematocrit (fraction of 1)	Standard Deviation 0.0335
JAK2 Negative Participants	Hematocrit Level	0.367 Hematocrit (fraction of 1)	Standard Deviation 0.0608

Secondary

Hemoglobin Concentration

Time frame: 1 year

Population: The Safety Analysis Set (SAS) consisted of all subjects enrolled in the study who took at least 1 dose of anagrelide hydrochloride. 28 patients in the SAS had a viable laboratory sample.

Arm	Measure	Value (MEAN)	Dispersion
JAK2 Positive Participants	Hemoglobin Concentration	133.6 g/L	Standard Deviation 13.09
JAK2 Negative Participants	Hemoglobin Concentration	120.2 g/L	Standard Deviation 16.85

Secondary

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.

Time frame: 1 year

Arm	Measure	Value (NUMBER)
JAK2 Positive Participants	Number of Patients With Platelet Count ≤400x10^9/L After 12 Months	7 participants
JAK2 Negative Participants	Number of Patients With Platelet Count ≤400x10^9/L After 12 Months	8 participants

Secondary

Platelet Count

Time frame: 1 year

Arm	Measure	Value (MEAN)	Dispersion
JAK2 Positive Participants	Platelet Count	458.9 Platelets (x10^9/L)	Standard Deviation 163.25
JAK2 Negative Participants	Platelet Count	503.0 Platelets (x10^9/L)	Standard Deviation 254.04

Secondary

Red Blood Cell (RBC) Count

Time frame: 1 year

Population: The Safety Analysis Set (SAS) consisted of all subjects enrolled in the study who took at least 1 dose of anagrelide hydrochloride. 27 patients in the SAS met this requirement and had a viable laboratory sample.

Arm	Measure	Value (MEAN)	Dispersion
JAK2 Positive Participants	Red Blood Cell (RBC) Count	4.85 RBC Count (x10^12/L)	Standard Deviation 0.43
JAK2 Negative Participants	Red Blood Cell (RBC) Count	4.24 RBC Count (x10^12/L)	Standard Deviation 0.797

Secondary

White Blood Cell (WBC) Count

Time frame: 1 year

Arm	Measure	Value (MEAN)	Dispersion
JAK2 Positive Participants	White Blood Cell (WBC) Count	7.75 WBC Count (x10^9/L)	Standard Deviation 1.317
JAK2 Negative Participants	White Blood Cell (WBC) Count	8.77 WBC Count (x10^9/L)	Standard Deviation 3.77

Source: ClinicalTrials.gov · Data processed: Mar 5, 2026

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

Conditions

Brief summary

Related papers

Interventions

Sponsors

Study design

Eligibility

Inclusion criteria

Exclusion criteria

Design outcomes

Primary

Secondary

Countries

Participant flow

Participants by arm

Withdrawals & dropouts

Baseline characteristics

Adverse events

Outcome results

Number of Patients With Platelet Count ≤600x10^9/L After 12 Months

Differential WBC Count - Eosinophils

Differential WBC Count - Neutrophils, Lymphocytes, Monocytes, and Basophils.

Hematocrit Level

Hemoglobin Concentration

Number of Patients With Platelet Count ≤400x10^9/L After 12 Months

Platelet Count

Red Blood Cell (RBC) Count

White Blood Cell (WBC) Count