Essential Thrombocythemia (ET)
Conditions
Brief summary
The purpose of this study is to investigate how effective and safe SPD422 (Anagrelide Hydrochloride) is in Japanese subjects, diagnosed with Essential Thrombocythemia, who's previously treatment has either not been effective or has caused unacceptable adverse reactions. The study will aim to show that platelet counts can be safely reduced in treated patients to below 600 x 10\^9/L after a minimum of three months treatment. To demonstrate an positive effect platelet levels will need to remain below 600 x 10\^9/L for at least 4 weeks.
Interventions
Subjects will be started at 1.0 mg per day and titrated as necessary.
Sponsors
Shire
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
20 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Subjects must have previously been treated with a cytoreductive therapy and been intolerant or refractory to that therapy.
Exclusion criteria
* Subjects should not have any other underlying conditions or medications that would confound the study analysis or interact with the study medication.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Subjects Who Responded in Platelet Count | 12 months | A response was defined as platelet counts to \<600x10\^9/L across consecutive visits for at least 4 weeks following at least 3 months of treatment. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Subjects With at Least 50% Reduction in Platelet Count | 12 months | Subjects who achieved at least 50% reduction in platelet count from their baseline level across consecutive visits for at least 4 weeks and following 3 months of treatment. |
| Percentage of Subjects With Normalization in Platelet Count | 12 months | Normalization was defined as platelet counts ≤400x10\^9/L across consecutive visits for at least 4 weeks following at least 3 months of treatment. |
| Percentage of Subjects Who Responded in Platelet Count Whose Baseline Platelet Count Was ≥600x10^9/L | 12 months | A response was defined as platelet counts to \<600x10\^9/L across consecutive visits for at least 4 weeks following at least 3 months of treatment. |
| Percentage of Subjects Who Responded in Platelet Count Whose Baseline Platelet Count Was <600x10^9/L | 12 months | A response was defined as platelet counts to \<600x10\^9/L across consecutive visits for at least 4 weeks following at least 3 months of treatment. |
Countries
Japan
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Anagrelide Hydrochloride | 53 |
| Total | 53 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 8 |
| Overall Study | Lack of Efficacy | 3 |
Baseline characteristics
| Characteristic | Anagrelide Hydrochloride |
|---|---|
| Age, Continuous | 61.6 Years STANDARD_DEVIATION 13.1 |
| Age, Customized 50 - 59 years | 5 Participants |
| Age, Customized < 50 years | 12 Participants |
| Age, Customized 60 - 69 years | 18 Participants |
| Age, Customized 70 - 79 years | 17 Participants |
| Age, Customized >= 80 years | 1 Participants |
| Region of Enrollment JAPAN | 53 Participants |
| Sex: Female, Male Female | 30 Participants |
| Sex: Female, Male Male | 23 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 53 / 53 |
| serious Total, serious adverse events | 15 / 53 |
Outcome results
Primary
Percentage of Subjects Who Responded in Platelet Count
A response was defined as platelet counts to \<600x10\^9/L across consecutive visits for at least 4 weeks following at least 3 months of treatment.
Time frame: 12 months
Population: Full Analysis Set defined as subjects who had taken at least 1 dose of study medication and had at least 1 post-baseline platelet measurement assessment.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Anagrelide Hydrochloride | Percentage of Subjects Who Responded in Platelet Count | 67.9 percentage of subjects |
Secondary
Percentage of Subjects Who Responded in Platelet Count Whose Baseline Platelet Count Was <600x10^9/L
A response was defined as platelet counts to \<600x10\^9/L across consecutive visits for at least 4 weeks following at least 3 months of treatment.
Time frame: 12 months
Population: Full Analysis Set defined as subjects who had taken at least 1 dose of study medication and had at least 1 post-baseline platelet measurement assessment.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Anagrelide Hydrochloride | Percentage of Subjects Who Responded in Platelet Count Whose Baseline Platelet Count Was <600x10^9/L | 66.7 percentage of subjects |
Secondary
Percentage of Subjects Who Responded in Platelet Count Whose Baseline Platelet Count Was ≥600x10^9/L
A response was defined as platelet counts to \<600x10\^9/L across consecutive visits for at least 4 weeks following at least 3 months of treatment.
Time frame: 12 months
Population: Full Analysis Set defined as subjects who had taken at least 1 dose of study medication and had at least 1 post-baseline platelet measurement assessment.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Anagrelide Hydrochloride | Percentage of Subjects Who Responded in Platelet Count Whose Baseline Platelet Count Was ≥600x10^9/L | 68.1 percentage of subjects |
Secondary
Percentage of Subjects With at Least 50% Reduction in Platelet Count
Subjects who achieved at least 50% reduction in platelet count from their baseline level across consecutive visits for at least 4 weeks and following 3 months of treatment.
Time frame: 12 months
Population: Full Analysis Set defined as subjects who had taken at least 1 dose of study medication and had at least 1 post-baseline platelet measurement assessment.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Anagrelide Hydrochloride | Percentage of Subjects With at Least 50% Reduction in Platelet Count | 50.9 percentage of subjects |
Secondary
Percentage of Subjects With Normalization in Platelet Count
Normalization was defined as platelet counts ≤400x10\^9/L across consecutive visits for at least 4 weeks following at least 3 months of treatment.
Time frame: 12 months
Population: Full Analysis Set defined as subjects who had taken at least 1 dose of study medication and had at least 1 post-baseline platelet measurement assessment.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Anagrelide Hydrochloride | Percentage of Subjects With Normalization in Platelet Count | 45.3 percentage of subjects |