Immune Thrombocytopenia
Conditions
Keywords
ITP
Brief summary
Evaluate the safety and tolerability of avatrombopag given for 90 days after stopping treatment with eltrombopag or romiplostim.
Detailed description
This Phase 4, prospective, multi-center, open-label study will evaluate safety, platelet count, and subject reported medication satisfaction in adult subjects with chronic ITP after switching to avatrombopag from eltrombopag or romiplostim. At least 100 subjects will be enrolled, 50 (±10) who have received eltrombopag and 50 (±10) who have received romiplostim for at least 90 days prior to study entry.
Interventions
Avatrombopag 20 mg given daily for 90 days. Initial dose and dose adjustments will be determined by the physician along with the Doptelet prescribing information
Sponsors
Sobi, Inc.
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
* Subject has been undergoing treatment for primary ITP with eltrombopag or romiplostim for at least 90 days prior to the Screening Visit/Visit 1. * Subject has had a previous response (at any time) to either eltrombopag or romiplostim, defined as at least 2 platelet counts ≥50×10⁹/L. Subject is willing and able to comply with all aspects of the protocol, including completing the self-administered Treatment Satisfaction Medication Questionnaire (TSQM).
Exclusion criteria
* Subject is currently receiving chemotherapy or radiation for any form of cancer. * Subject with conditions that are likely to prevent them from accurately and reliably completing study assessments, including evidence of moderate to severe dementia, and/or severe and progressive medical illness, as determined by the Investigator. * Any previous avatrombopag use. * Previous participation in this study; a subject may not re-enroll after prior discontinuation or completion. * Enrollment in another clinical study with any investigational drug or device within 30 days of Baseline (or 5 half-lives, whichever is longer); however, participation in observational studies within the previous 30 days is permitted.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Safety and Tolerability (Adverse Events) | Screening through Day 90 or End of Study | Safety and Tolerability of Avatrombopag given for 90 days after stopping eltrombopag or romiplostim The incidence and severity of adverse events (AEs), serious adverse events (SAE) and adverse events of special interest (AESIs) will be summarized for all enrolled subjects using counts and percentages. Treatment-emergent AEs and SAEs will be summarized overall, by system organ class, and by preferred term. AESIs will be summarized by event type (thromboembolic events and bleeding events). In addition, treatment-emergent AEs will be summarized by severity and by relationship to study drug. Bleeding events reported during the study will be summarized by WHO grade. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline TSQM Convenience Domain Score | Day 90 | Evaluate the change in subject reported outcomes (TSQM - Treatment Satisfaction Questionnaire for Medication) from Baseline. Convenience domain score ranges from 0 to 100, and a higher score indicates a better outcome. Mean Difference was computed as Day 90 minus Baseline and, therefore, a positive mean difference indicates an increase in the score from Baseline to Day 90. |
| Change From Baseline TSQM Side Effects Domain Score | Day 90 | Evaluate the change in subject reported outcomes (TSQM - Treatment Satisfaction Questionnaire for Medication) from Baseline. Side Effects domain score ranges from 0 to 100, and a higher score indicates a better outcome. Mean Difference was computed as Day 90 minus Baseline and, therefore, a positive mean difference indicates an increase in the score from Baseline to Day 90. |
| Change From Baseline TSQM Effectiveness Domain Score | Day 90 | Evaluate the change in subject reported outcomes (TSQM - Treatment Satisfaction Questionnaire for Medication) from Baseline. Effectiveness domain score ranges from 0 to 100, and a higher score indicates a better outcome. Mean Difference was computed as Day 90 minus Baseline and, therefore, a positive mean difference indicates an increase in the score from Baseline to Day 90. |
| Change From Baseline TSQM Global Satisfaction Domain Score | Day 90 | Evaluate the change in subject reported outcomes (TSQM - Treatment Satisfaction Questionnaire for Medication) from Baseline. Global Satisfaction domain score ranges from 0 to 100, and a higher score indicates a better outcome. Mean Difference was computed as Day 90 minus Baseline and, therefore, a positive mean difference indicates an increase in the score from Baseline to Day 90. |
| Proportion of Subjects Who Have a Platelet Count Between ≥50×10^9/L to ≤200×10^9/L | Day 15 | — |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Avatrombopag Avatrombopag 20 mg oral tablet formulation for 90 days
Avatrombopag Oral Tablet: Avatrombopag 20 mg given daily for 90 days. Initial dose and dose adjustments will be determined by the physician along with the Doptelet prescribing information | 60 |
| Total | 60 |
Baseline characteristics
| Characteristic | Avatrombopag |
|---|---|
| Age, Continuous | 58.0 years STANDARD_DEVIATION 21.7 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 13 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 46 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 1 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 2 Participants |
| Race (NIH/OMB) Black or African American | 5 Participants |
| Race (NIH/OMB) More than one race | 1 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 1 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 8 Participants |
| Race (NIH/OMB) White | 43 Participants |
| Sex: Female, Male Female | 37 Participants |
| Sex: Female, Male Male | 23 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 1 / 60 |
| other Total, other adverse events | 34 / 60 |
| serious Total, serious adverse events | 6 / 60 |
Outcome results
Primary
Safety and Tolerability (Adverse Events)
Safety and Tolerability of Avatrombopag given for 90 days after stopping eltrombopag or romiplostim The incidence and severity of adverse events (AEs), serious adverse events (SAE) and adverse events of special interest (AESIs) will be summarized for all enrolled subjects using counts and percentages. Treatment-emergent AEs and SAEs will be summarized overall, by system organ class, and by preferred term. AESIs will be summarized by event type (thromboembolic events and bleeding events). In addition, treatment-emergent AEs will be summarized by severity and by relationship to study drug. Bleeding events reported during the study will be summarized by WHO grade.
Time frame: Screening through Day 90 or End of Study
Population: All enrolled subjects.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Avatrombopag | Safety and Tolerability (Adverse Events) | TEAEs | 35 Participants |
| Avatrombopag | Safety and Tolerability (Adverse Events) | SAEs | 6 Participants |
| Avatrombopag | Safety and Tolerability (Adverse Events) | AESIs | 1 Participants |
| Avatrombopag | Safety and Tolerability (Adverse Events) | Related TEAEs | 15 Participants |
| Avatrombopag | Safety and Tolerability (Adverse Events) | Severe TEAEs | 8 Participants |
| Avatrombopag | Safety and Tolerability (Adverse Events) | TEAEs leading to study drug discontinuation | 2 Participants |
Secondary
Change From Baseline TSQM Convenience Domain Score
Evaluate the change in subject reported outcomes (TSQM - Treatment Satisfaction Questionnaire for Medication) from Baseline. Convenience domain score ranges from 0 to 100, and a higher score indicates a better outcome. Mean Difference was computed as Day 90 minus Baseline and, therefore, a positive mean difference indicates an increase in the score from Baseline to Day 90.
Time frame: Day 90
Population: All enrolled subjects.
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Avatrombopag | Change From Baseline TSQM Convenience Domain Score | 13.5 Mean difference in convenience score |
p-value: 0.0001t-test, 2 sided
Secondary
Change From Baseline TSQM Effectiveness Domain Score
Evaluate the change in subject reported outcomes (TSQM - Treatment Satisfaction Questionnaire for Medication) from Baseline. Effectiveness domain score ranges from 0 to 100, and a higher score indicates a better outcome. Mean Difference was computed as Day 90 minus Baseline and, therefore, a positive mean difference indicates an increase in the score from Baseline to Day 90.
Time frame: Day 90
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Avatrombopag | Change From Baseline TSQM Effectiveness Domain Score | 14.4 Mean difference in effectiveness score |
p-value: 0.0003t-test, 2 sided
Secondary
Change From Baseline TSQM Global Satisfaction Domain Score
Evaluate the change in subject reported outcomes (TSQM - Treatment Satisfaction Questionnaire for Medication) from Baseline. Global Satisfaction domain score ranges from 0 to 100, and a higher score indicates a better outcome. Mean Difference was computed as Day 90 minus Baseline and, therefore, a positive mean difference indicates an increase in the score from Baseline to Day 90.
Time frame: Day 90
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Avatrombopag | Change From Baseline TSQM Global Satisfaction Domain Score | 14.2 Mean difference in global satisfaction |
p-value: <0.0001t-test, 2 sided
Secondary
Change From Baseline TSQM Side Effects Domain Score
Evaluate the change in subject reported outcomes (TSQM - Treatment Satisfaction Questionnaire for Medication) from Baseline. Side Effects domain score ranges from 0 to 100, and a higher score indicates a better outcome. Mean Difference was computed as Day 90 minus Baseline and, therefore, a positive mean difference indicates an increase in the score from Baseline to Day 90.
Time frame: Day 90
| Arm | Measure | Value (MEAN) |
|---|---|---|
| Avatrombopag | Change From Baseline TSQM Side Effects Domain Score | 8.3 Mean difference in side effects score |
p-value: 0.0093t-test, 2 sided
Secondary
Proportion of Subjects Who Have a Platelet Count Between ≥50×10^9/L to ≤200×10^9/L
Time frame: Day 15
Population: All enrolled subjects.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Avatrombopag | Proportion of Subjects Who Have a Platelet Count Between ≥50×10^9/L to ≤200×10^9/L | 19 Participants |
Secondary
Proportion of Subjects Who Have a Platelet Count Between ≥50×10^9/L to ≤200×10^9/L
Time frame: Day 30
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Avatrombopag | Proportion of Subjects Who Have a Platelet Count Between ≥50×10^9/L to ≤200×10^9/L | 33 Participants |
Secondary
Proportion of Subjects Who Have a Platelet Count Between ≥50×10^9/L to ≤200×10^9/L
Time frame: Day 60
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Avatrombopag | Proportion of Subjects Who Have a Platelet Count Between ≥50×10^9/L to ≤200×10^9/L | 36 Participants |
Secondary
Proportion of Subjects Who Have a Platelet Count Between ≥50×10^9/L to ≤200×10^9/L
Time frame: Day 90
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Avatrombopag | Proportion of Subjects Who Have a Platelet Count Between ≥50×10^9/L to ≤200×10^9/L | 33 Participants |