Immune Thrombocytopenic Purpura
Conditions
Brief summary
The primary objective of this study was to assess the long term safety of fostamatinib in subjects with persistent/chronic ITP
Interventions
Fostamatinib Disodium tablet 100 mg or 150 mg by mouth twice a day
Sponsors
Rigel Pharmaceuticals
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Completed week 24 evaluation of Study C935788-047 or Study C935788-048 or discontinued early due to lack of response. * Able and willing to give written informed consent
Exclusion criteria
* Discontinued participation in Study C935788-047 or Study C935788-048 for any reason other than lack of response * Poorly controlled hypertension during Study C935788-047 or Study C935788-048 * Significant infection, an acute infection such as influenza, or known inflammatory process
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Subjects Who Achieved Platelet Count of at Least 50,000/µL Within 12 Weeks of Beginning Treatment up to 12 Months (Fostamatinib in 047/048 or 049):Version 1 | Up to 12 months | Percentage of subjects who achieved platelet count of at least 50,000/µL within 12 Weeks of beginning treatment up to 12 months was analyzed among all subjects who received active treatment in one of the prior studies (C788-047 or C788-048), in the current extension study (C788-049), or in both prior and current studies. Treated Population was defined as all enrolled and treated subjects. |
| Percentage of Subjects Who Achieved Platelet Count of at Least 50,000/µL Within 12 Weeks of Beginning Treatment up to 12 Months (Placebo in 047/048 and Fostamatinib 049): Version 2 | Up to 12 months | A within-subject, between-study comparison of platelet counts for subjects who were previously treated with placebo in one of the prior studies (C788-047 or C788-048) was prospectively defined in the protocol (version 2). Achievement of platelet response by 12 weeks and maintenance of platelet response for 12 weeks was analyzed among subjects who had been randomized to placebo in one of the prior studies (C788-047 or C788-048). Treated Population was defined as all enrolled and treated subjects. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Duration of Platelet Response Based on Platelet Count and Rescue Medication | Up to 12 months | The duration of platelet response was defined as the time from when the subject first achieved a platelet count of at least 50,000/µL, until the first of 2 visits with platelet counts \< 50,000/µL that were at least 4 weeks apart without an intervening visit with a platelet count less than equal to (\<=) 50,000/µL unrelated to rescue therapy. Duration of platelet response was analyzed using the Kaplan-Meier method. Treated Population was defined as all enrolled and treated subjects. Here, a number of subjects analyzed included all subjects evaluable for this endpoint. |
| Percentage of Subjects in Whom a Reduction in the Dose of Concomitant ITP Therapy Can be Achieved While Maintaining an Adequate Platelet Count | Up to 12 months | The percentage of subjects in whom a reduction in the dose of concomitant ITP therapy could be achieved while maintaining an adequate platelet count, the reduction event was clarified to apply only to reductions in the dose of concomitant ITP therapy occurring within a period of platelet response and the reduction event was not be prompted by an adverse event. |
Countries
Australia, Austria, Bulgaria, Canada, Czechia, Denmark, Hungary, Italy, Netherlands, Norway, Poland, Romania, Spain, United Kingdom, United States
Participant flow
Recruitment details
A total of 124 subjects were screened from studies C788-047/ C788-048 for this extension study. Out of which, 123 subjects were enrolled and treated with study drug.
Participants by arm
| Arm | Count |
|---|---|
| Overall Study Baseline characteristics are analyzed with treated population data analysis set which was defined as all enrolled and treated subjects. | 123 |
| Total | 123 |
Baseline characteristics
| Characteristic | Overall Study |
|---|---|
| Age, Continuous | 52.0 years |
| Ethnicity (NIH/OMB) Hispanic or Latino | 2 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 121 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 4 Participants |
| Race (NIH/OMB) Black or African American | 4 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 2 Participants |
| Race (NIH/OMB) White | 113 Participants |
| Region of Enrollment Australia | 10 Participants |
| Region of Enrollment Austria | 1 Participants |
| Region of Enrollment Bulgaria | 12 Participants |
| Region of Enrollment Canada | 4 Participants |
| Region of Enrollment Czechia | 10 Participants |
| Region of Enrollment Denmark | 1 Participants |
| Region of Enrollment Hungary | 2 Participants |
| Region of Enrollment Italy | 6 Participants |
| Region of Enrollment Netherlands | 1 Participants |
| Region of Enrollment Norway | 2 Participants |
| Region of Enrollment Poland | 34 Participants |
| Region of Enrollment Romania | 1 Participants |
| Region of Enrollment Spain | 4 Participants |
| Region of Enrollment United Kingdom | 18 Participants |
| Region of Enrollment United States | 17 Participants |
| Sex: Female, Male Female | 74 Participants |
| Sex: Female, Male Male | 49 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 4 / 123 |
| other Total, other adverse events | 98 / 123 |
| serious Total, serious adverse events | 34 / 123 |
Outcome results
Primary
Percentage of Subjects Who Achieved Platelet Count of at Least 50,000/µL Within 12 Weeks of Beginning Treatment up to 12 Months (Fostamatinib in 047/048 or 049):Version 1
Percentage of subjects who achieved platelet count of at least 50,000/µL within 12 Weeks of beginning treatment up to 12 months was analyzed among all subjects who received active treatment in one of the prior studies (C788-047 or C788-048), in the current extension study (C788-049), or in both prior and current studies. Treated Population was defined as all enrolled and treated subjects.
Time frame: Up to 12 months
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Fostamatinib Disodium | Percentage of Subjects Who Achieved Platelet Count of at Least 50,000/µL Within 12 Weeks of Beginning Treatment up to 12 Months (Fostamatinib in 047/048 or 049):Version 1 | 15.4 Percentage of Subjects |
Primary
Percentage of Subjects Who Achieved Platelet Count of at Least 50,000/µL Within 12 Weeks of Beginning Treatment up to 12 Months (Placebo in 047/048 and Fostamatinib 049): Version 2
A within-subject, between-study comparison of platelet counts for subjects who were previously treated with placebo in one of the prior studies (C788-047 or C788-048) was prospectively defined in the protocol (version 2). Achievement of platelet response by 12 weeks and maintenance of platelet response for 12 weeks was analyzed among subjects who had been randomized to placebo in one of the prior studies (C788-047 or C788-048). Treated Population was defined as all enrolled and treated subjects.
Time frame: Up to 12 months
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Fostamatinib Disodium | Percentage of Subjects Who Achieved Platelet Count of at Least 50,000/µL Within 12 Weeks of Beginning Treatment up to 12 Months (Placebo in 047/048 and Fostamatinib 049): Version 2 | 2.3 Percentage of Subjects |
Secondary
Duration of Platelet Response Based on Platelet Count and Rescue Medication
The duration of platelet response was defined as the time from when the subject first achieved a platelet count of at least 50,000/µL, until the first of 2 visits with platelet counts \< 50,000/µL that were at least 4 weeks apart without an intervening visit with a platelet count less than equal to (\<=) 50,000/µL unrelated to rescue therapy. Duration of platelet response was analyzed using the Kaplan-Meier method. Treated Population was defined as all enrolled and treated subjects. Here, a number of subjects analyzed included all subjects evaluable for this endpoint.
Time frame: Up to 12 months
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Fostamatinib Disodium | Duration of Platelet Response Based on Platelet Count and Rescue Medication | 127.0 Days |
Secondary
Percentage of Subjects in Whom a Reduction in the Dose of Concomitant ITP Therapy Can be Achieved While Maintaining an Adequate Platelet Count
The percentage of subjects in whom a reduction in the dose of concomitant ITP therapy could be achieved while maintaining an adequate platelet count, the reduction event was clarified to apply only to reductions in the dose of concomitant ITP therapy occurring within a period of platelet response and the reduction event was not be prompted by an adverse event.
Time frame: Up to 12 months
Population: insufficient of number participants with events to perform the analysis