Thrombocytopenia
Conditions
Keywords
low platelet count, ITP
Brief summary
Phase 3b open-label, multicenter study to evaluate the safety and efficacy of avatrombopag in subjects with thrombocytopenia scheduled for operations to critical sites or operations with a high risk of bleeding.
Detailed description
Subjects will receive oral avatrombopag once daily for 5 days beginning on Day 1, followed by a wait period prior to the procedure, which will occur on Day 10 to 13. The Follow-up Period will include 2 visits; 7 days post-procedure and 30 days after last dose.
Interventions
DRUGAvatrombopag 60 mg
Oral avatrombopag administered once daily for 5 days prior to procedure.
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
* Men and women greater than or equal to 18 years of age; * A mean baseline platelet count between: * 50 × 10\^9/L and \<100 × 10\^9/L for non-chronic liver disease participants * 50 × 10\^9/L and \<75 × 10\^9/L for participants with chronic liver disease; * Participant is scheduled to undergo operations to critical sites (eg, eye surgery, neurosurgery) or operations with a high risk of bleeding (eg, major abdominal surgery), or, in the opinion of the Investigator, would otherwise require a pre-operative platelet transfusion to prevent bleeding
Exclusion criteria
* Participant with a history of arterial or venous thrombosis within 6 months of baseline; * Participant with known portal vein blood flow velocity rate \<10 cm/second or previous portal vein thrombosis within 6 months of baseline; * Participant plans to have a platelet transfusion or plans to receive blood products containing platelets within 7 days of the Baseline Visit; * Use of erythropoietin-stimulating agents; * Participant has a known medical history of genetic prothrombotic syndromes; or * Participant has abnormal hemoglobin levels or prothrombin time/international normalized ratio
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Evaluate Efficacy of Avatrombopag in Increasing Platelet Counts in Subjects With Thrombocytopenia Scheduled for Operations | Baseline to 10-13 days | Proportion of subjects that achieve a platelet count \>100 x 10\^9 platelets/L on procedure day |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Evaluate Safety of Avatrombopag: Incidence of Treatment Emergent Adverse Events | Up to 35 days | Incidence of treatment emergent adverse events |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Avatrombopag 60 mg Open-label: oral avatrombopag
Avatrombopag 60 mg: Oral avatrombopag administered once daily for 5 days prior to procedure. | 4 |
| Total | 4 |
Baseline characteristics
| Characteristic | Avatrombopag 60 mg |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 3 Participants |
| Age, Categorical Between 18 and 65 years | 1 Participants |
| Age, Continuous | 66 years |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 3 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Platelet Counts | 68 10^9 platelets/L |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 1 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 | 0 Participants |
| Race (NIH/OMB) White | 3 Participants |
| Region of Enrollment United States | 4 participants |
| Sex: Female, Male Female | 2 Participants |
| Sex: Female, Male Male | 2 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 4 |
| other Total, other adverse events | 4 / 4 |
| serious Total, serious adverse events | 0 / 4 |
Outcome results
Primary
Evaluate Efficacy of Avatrombopag in Increasing Platelet Counts in Subjects With Thrombocytopenia Scheduled for Operations
Proportion of subjects that achieve a platelet count \>100 x 10\^9 platelets/L on procedure day
Time frame: Baseline to 10-13 days
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Avatrombopag 60 mg | Evaluate Efficacy of Avatrombopag in Increasing Platelet Counts in Subjects With Thrombocytopenia Scheduled for Operations | 0.75 proportion of total participants |
Secondary
Evaluate Safety of Avatrombopag: Incidence of Treatment Emergent Adverse Events
Incidence of treatment emergent adverse events
Time frame: Up to 35 days
Population: Study was early terminated due to low enrollment