Trial Outcomes & Findings for Exploratory Multi-centre Trial In Patients With ET Treated With XAGRID® (NCT NCT01352585)

NCT ID: NCT01352585

Last Updated: 2021-07-30

Results Overview

A platelet count of ≤600x10\^9/L after 12 months is considered at least a partial response.

• Recruitment status: COMPLETED
• Target enrollment: 47 participants
• Primary outcome timeframe: 1 year
• Results posted on: 2021-07-30

Participant Flow

Participant milestones

Measure	JAK2 Positive Participants Anagrelide hydrochloride: 0.5 mg hard capsules, dosing decisions will be made by the treating physician	JAK2 Negative Participants Anagrelide hydrochloride: 0.5 mg hard capsules, dosing decisions will be made by the treating physician
Overall Study STARTED	23	24
Overall Study COMPLETED	15	21
Overall Study NOT COMPLETED	8	3

Reasons for withdrawal

Measure	JAK2 Positive Participants Anagrelide hydrochloride: 0.5 mg hard capsules, dosing decisions will be made by the treating physician	JAK2 Negative Participants Anagrelide hydrochloride: 0.5 mg hard capsules, dosing decisions will be made by the treating physician
Overall Study Adverse Event	2	1
Overall Study Lost to Follow-up	1	0
Overall Study Other	5	2

Baseline Characteristics

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

Baseline characteristics by cohort

Measure	JAK2 Positive Participants n=23 Participants Anagrelide hydrochloride: 0.5 mg hard capsules, dosing decisions will be made by the treating physician	JAK2 Negative Participants n=24 Participants Anagrelide hydrochloride: 0.5 mg hard capsules, dosing decisions will be made by the treating physician	Total n=47 Participants Total of all reporting groups
Age, Continuous	57.4 years STANDARD_DEVIATION 14.00 • n=5 Participants	58.6 years STANDARD_DEVIATION 15.94 • n=7 Participants	58.0 years STANDARD_DEVIATION 14.87 • n=5 Participants
Sex: Female, Male Female	14 Participants n=5 Participants	14 Participants n=7 Participants	28 Participants n=5 Participants
Sex: Female, Male Male	9 Participants n=5 Participants	10 Participants n=7 Participants	19 Participants n=5 Participants
Region of Enrollment Italy	23 Participants n=5 Participants	24 Participants n=7 Participants	47 Participants n=5 Participants

PRIMARY outcome

Timeframe: 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.

A platelet count of ≤600x10^9/L after 12 months is considered at least a partial response.

Outcome measures

Measure	JAK2 Positive Participants n=14 Participants Anagrelide hydrochloride: 0.5 mg hard capsules, dosing decisions will be made by the treating physician	JAK2 Negative Participants n=21 Participants Anagrelide hydrochloride: 0.5 mg hard capsules, dosing decisions will be made by the treating physician
Number of Patients With Platelet Count ≤600x10^9/L After 12 Months	12 participants	14 participants

SECONDARY outcome

Timeframe: 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.

A platelet count of ≤400x10^9/L after 12 months is considered a complete response.

Outcome measures

Measure	JAK2 Positive Participants n=14 Participants Anagrelide hydrochloride: 0.5 mg hard capsules, dosing decisions will be made by the treating physician	JAK2 Negative Participants n=21 Participants Anagrelide hydrochloride: 0.5 mg hard capsules, dosing decisions will be made by the treating physician
Number of Patients With Platelet Count ≤400x10^9/L After 12 Months	7 participants	8 participants

SECONDARY outcome

Timeframe: 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.

Outcome measures

Measure	JAK2 Positive Participants n=11 Participants Anagrelide hydrochloride: 0.5 mg hard capsules, dosing decisions will be made by the treating physician	JAK2 Negative Participants n=17 Participants Anagrelide hydrochloride: 0.5 mg hard capsules, dosing decisions will be made by the treating physician
Platelet Count	458.9 Platelets (x10^9/L) Standard Deviation 163.25	503.0 Platelets (x10^9/L) Standard Deviation 254.04

SECONDARY outcome

Timeframe: 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.

Outcome measures

Measure	JAK2 Positive Participants n=11 Participants Anagrelide hydrochloride: 0.5 mg hard capsules, dosing decisions will be made by the treating physician	JAK2 Negative Participants n=16 Participants Anagrelide hydrochloride: 0.5 mg hard capsules, dosing decisions will be made by the treating physician
Red Blood Cell (RBC) Count	4.85 RBC Count (x10^12/L) Standard Deviation 0.430	4.24 RBC Count (x10^12/L) Standard Deviation 0.797

SECONDARY outcome

Timeframe: 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.

Outcome measures

Measure	JAK2 Positive Participants n=11 Participants Anagrelide hydrochloride: 0.5 mg hard capsules, dosing decisions will be made by the treating physician	JAK2 Negative Participants n=17 Participants Anagrelide hydrochloride: 0.5 mg hard capsules, dosing decisions will be made by the treating physician
White Blood Cell (WBC) Count	7.75 WBC Count (x10^9/L) Standard Deviation 1.317	8.77 WBC Count (x10^9/L) Standard Deviation 3.770

SECONDARY outcome

Timeframe: 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.

Outcome measures

Measure	JAK2 Positive Participants n=11 Participants Anagrelide hydrochloride: 0.5 mg hard capsules, dosing decisions will be made by the treating physician	JAK2 Negative Participants n=17 Participants Anagrelide hydrochloride: 0.5 mg hard capsules, dosing decisions will be made by the treating physician
Differential WBC Count - Neutrophils, Lymphocytes, Monocytes, and Basophils. Neutrophils	62.05 percent Standard Deviation 4.429	60.07 percent Standard Deviation 13.515
Differential WBC Count - Neutrophils, Lymphocytes, Monocytes, and Basophils. Lymphocytes	27.53 percent Standard Deviation 4.412	28.76 percent Standard Deviation 12.381
Differential WBC Count - Neutrophils, Lymphocytes, Monocytes, and Basophils. Monocytes	6.50 percent Standard Deviation 2.235	7.21 percent Standard Deviation 2.680
Differential WBC Count - Neutrophils, Lymphocytes, Monocytes, and Basophils. Basophils	1.75 percent Standard Deviation 2.425	0.81 percent Standard Deviation 0.486

SECONDARY outcome

Timeframe: 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.

Outcome measures

Measure	JAK2 Positive Participants n=11 Participants Anagrelide hydrochloride: 0.5 mg hard capsules, dosing decisions will be made by the treating physician	JAK2 Negative Participants n=16 Participants Anagrelide hydrochloride: 0.5 mg hard capsules, dosing decisions will be made by the treating physician
Differential WBC Count - Eosinophils	2.95 percent Standard Deviation 1.958	2.77 percent Standard Deviation 1.906

SECONDARY outcome

Timeframe: 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.

Outcome measures

Measure	JAK2 Positive Participants n=11 Participants Anagrelide hydrochloride: 0.5 mg hard capsules, dosing decisions will be made by the treating physician	JAK2 Negative Participants n=17 Participants Anagrelide hydrochloride: 0.5 mg hard capsules, dosing decisions will be made by the treating physician
Hemoglobin Concentration	133.6 g/L Standard Deviation 13.09	120.2 g/L Standard Deviation 16.85

SECONDARY outcome

Timeframe: 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.

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

Outcome measures

Measure	JAK2 Positive Participants n=11 Participants Anagrelide hydrochloride: 0.5 mg hard capsules, dosing decisions will be made by the treating physician	JAK2 Negative Participants n=16 Participants Anagrelide hydrochloride: 0.5 mg hard capsules, dosing decisions will be made by the treating physician
Hematocrit Level	0.412 Hematocrit (fraction of 1) Standard Deviation 0.0335	0.367 Hematocrit (fraction of 1) Standard Deviation 0.0608

Adverse Events

JAK2 Positive Participants

Serious events: 0 serious events

Other events: 6 other events

Deaths: 0 deaths

JAK2 Negative Participants

Serious events: 0 serious events

Other events: 9 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	JAK2 Positive Participants n=23 participants at risk Anagrelide hydrochloride: 0.5 mg hard capsules, dosing decisions will be made by the treating physician	JAK2 Negative Participants n=24 participants at risk Anagrelide hydrochloride: 0.5 mg hard capsules, dosing decisions will be made by the treating physician
Blood and lymphatic system disorders anemia	4.3% 1/23 • Number of events 1	8.3% 2/24 • Number of events 3
Cardiac disorders Palpitations	4.3% 1/23 • Number of events 1	4.2% 1/24 • Number of events 1
Cardiac disorders Tachycardia	4.3% 1/23 • Number of events 1	4.2% 1/24 • Number of events 1
Eye disorders Scotoma	0.00% 0/23	4.2% 1/24 • Number of events 1
Gastrointestinal disorders Diarrhea	0.00% 0/23	4.2% 1/24 • Number of events 1
Metabolism and nutrition disorders Hyperuricemia	0.00% 0/23	4.2% 1/24 • Number of events 1
Nervous system disorders Headache	8.7% 2/23 • Number of events 2	16.7% 4/24 • Number of events 4
Nervous system disorders Presyncope	4.3% 1/23 • Number of events 1	0.00% 0/24
Skin and subcutaneous tissue disorders Dermatits	4.3% 1/23 • Number of events 1	0.00% 0/24
Skin and subcutaneous tissue disorders Pruritus	0.00% 0/23	4.2% 1/24 • Number of events 1

Additional Information

Study Director

Shire

Phone: +1 866 842 5335

Email: ClinicalTransparency@shire.com

Results disclosure agreements

• Principal investigator is a sponsor employee If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
• Publication restrictions are in place

Restriction type: OTHER