Avatrombopag for the Treatment of Thrombocytopenia in Adults Scheduled for a Surgical Procedure

• Sponsor: Sobi, Inc. (Industry)
• Overall Status: Terminated
• CT.gov ID: NCT03326843
• Collaborator: (none)
• Enrollment: 4
• Locations: 27
• Arm: 1
• Actual Duration (Months): 11
• Patients Per Site: 0.1
• Patients Per Site Per Month: 0

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Thrombocytopenia	Drug: Avatrombopag 60 mg	Phase 3

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.

Study Design

• Study Type: Interventional
• Actual Enrollment: 4 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: An Open-Label Study to Evaluate the Efficacy and Safety of Avatrombopag for the Treatment of Subjects With Thrombocytopenia Scheduled for a Surgical Procedure
• Actual Study Start Date: Mar 26, 2018
• Actual Primary Completion Date: Feb 25, 2019
• Actual Study Completion Date: Feb 25, 2019

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Avatrombopag 60 mg Open-label: oral avatrombopag	Drug: Avatrombopag 60 mg; Oral avatrombopag administered once daily for 5 days prior to procedure.

Outcome Measures

Primary Outcome Measures

1. 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 Outcome Measures

1. Evaluate Safety of Avatrombopag: Incidence of Treatment Emergent Adverse Events [Up to 35 days]

   Incidence of treatment emergent adverse events

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Dova Site	Little Rock	Arkansas	United States	72205
2	Dova Site	Coronado	California	United States	92118
3	Dova Site	Aurora	Colorado	United States	80045
4	Dova Site	Jacksonville	Florida	United States	32209
5	Dova Site	Jacksonville	Florida	United States	32224
6	Dova Site	Miami	Florida	United States	33135
7	Dova Site	Miami	Florida	United States	33136
8	Dova Site	Pensacola	Florida	United States	32503
9	Dova Site	Sarasota	Florida	United States	34232
10	Dova Site	Tamarac	Florida	United States	33321
11	Dova Site	Tampa	Florida	United States	33615
12	Dova Site	New Orleans	Louisiana	United States	70112
13	Dova Site	Baltimore	Maryland	United States	21202
14	Dova Site	Boston	Massachusetts	United States	02115
15	Dova Site	Detroit	Michigan	United States	48202
16	Dova Site	Saint Louis	Missouri	United States	63110
17	Dova Site	Springfield	Missouri	United States	65807
18	Dova Site	Reno	Nevada	United States	89503
19	Dova Site	Englewood	New Jersey	United States	07631
20	Dova Site	Buffalo	New York	United States	14203
21	Dova Site	Durham	North Carolina	United States	27710
22	Dova Site	Winston-Salem	North Carolina	United States	27157
23	Dova Site	Toledo	Ohio	United States	43606
24	Dova Site	Bethlehem	Pennsylvania	United States	18015
25	Dova Site	Austin	Texas	United States	78731
26	Dova Site	Newport News	Virginia	United States	23602
27	Dova Site	Milwaukee	Wisconsin	United States	53226

Sponsors and Collaborators

• Sobi, Inc.

Investigators

None specified.

Study Documents (Full-Text)

• Study Protocol - Sep 17, 2018
• Statistical Analysis Plan - Jan 22, 2019

More Information

Publications

None provided.

• Responsible Party: Sobi, Inc.
• ClinicalTrials.gov Identifier: NCT03326843
• Other Study ID Numbers: AVA-PST-320
• First Posted: Oct 31, 2017
• Last Update Posted: Apr 15, 2020
• Last Verified: May 1, 2018

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by Sobi, Inc.: low platelet count; ITP
• Additional relevant MeSH terms: Thrombocytopenia

Study Results

Participant Flow

Arm/Group Title	Avatrombopag 60 mg
Arm/Group Description	Open-label: oral avatrombopag Avatrombopag 60 mg: Oral avatrombopag administered once daily for 5 days prior to procedure.
Period Title: Overall Study
STARTED	4
COMPLETED	4
NOT COMPLETED	0

Baseline Characteristics

Arm/Group Title	Avatrombopag 60 mg
Arm/Group Description	Open-label: oral avatrombopag Avatrombopag 60 mg: Oral avatrombopag administered once daily for 5 days prior to procedure.
Overall Participants	4
Age (Count of Participants)
<=18 years	0 0%
Between 18 and 65 years	1 25%
>=65 years	3 75%
Age (years) [Mean (Full Range) ]
Mean (Full Range) [years]	66
Sex: Female, Male (Count of Participants)
Female	2 50%
Male	2 50%
Ethnicity (NIH/OMB) (Count of Participants)
Hispanic or Latino	1 25%
Not Hispanic or Latino	3 75%
Unknown or Not Reported	0 0%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%
Asian	0 0%
Native Hawaiian or Other Pacific Islander	0 0%
Black or African American	1 25%
White	3 75%
More than one race	0 0%
Unknown or Not Reported	0 0%
Region of Enrollment (participants) [Number]
United States	4 100%
Platelet Counts (10^9 platelets/L) [Mean (Full Range) ]
Mean (Full Range) [10^9 platelets/L]	68

Outcome Measures

1. Primary Outcome

Title	Evaluate Efficacy of Avatrombopag in Increasing Platelet Counts in Subjects With Thrombocytopenia Scheduled for Operations
Description	Proportion of subjects that achieve a platelet count >100 x 10^9 platelets/L on procedure day
Time Frame	Baseline to 10-13 days

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Avatrombopag 60 mg
Arm/Group Description	Open-label: oral avatrombopag Avatrombopag 60 mg: Oral avatrombopag administered once daily for 5 days prior to procedure.
Measure Participants	4
Number [proportion of total participants]	0.75 18.8%

2. Secondary Outcome

Title	Evaluate Safety of Avatrombopag: Incidence of Treatment Emergent Adverse Events
Description	Incidence of treatment emergent adverse events
Time Frame	Up to 35 days

Outcome Measure Data

Analysis Population Description

Study was early terminated due to low enrollment

Arm/Group Title	Avatrombopag 60 mg
Arm/Group Description	Open-label: oral avatrombopag Avatrombopag 60 mg: Oral avatrombopag administered once daily for 5 days prior to procedure.
Measure Participants	0

Adverse Events

Time Frame	From the time of consent through study follow up visit, up to 35 days.
Arm/Group Title	Avatrombopag 60 mg
Arm/Group Description	Open-label: oral avatrombopag Avatrombopag 60 mg: Oral avatrombopag administered once daily for 5 days prior to procedure.
All Cause Mortality
	Avatrombopag 60 mg
	Affected / at Risk (%)	# Events
Total	0/4 (0%)
Serious Adverse Events
	Avatrombopag 60 mg
	Affected / at Risk (%)	# Events
Total	0/4 (0%)
Other (Not Including Serious) Adverse Events
	Avatrombopag 60 mg
	Affected / at Risk (%)	# Events
Total	4/4 (100%)
Infections and infestations
Influenza	1/4 (25%)	1
Urinary Tract Infection	1/4 (25%)	1
Injury, poisoning and procedural complications
Subcutaneous hematoma	1/4 (25%)	1
Metabolism and nutrition disorders
Gout	1/4 (25%)	1
Renal and urinary disorders
Urinary Retention	1/4 (25%)	1

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

Following a multicenter publication, the Institution and/or Principal Investigator may publish data or results from the Study, provided, however, that the Institution and/or Principal Investigator submits the proposed publication to the Sponsor for review at least sixty (60) days prior to the date of the proposed publication. Institution shall remove from the proposed publication any information that is considered confidential other than Study data and results.