Clinical trial NCT00625443: Phase 2, Parallel Group, Rollover Study of AKR-501 in Patients With ChronicITP Who Completed 28 Days of Study Treatment in Protocol 501-CL-003
Official title: A Phase 2, Parallel Group, Rollover Study of AKR-501 in Patients With Chronic Idiopathic Thrombocytopenic Purpura (ITP) Who Completed 28 Days of Study Treatment in Protocol 501-CL-003
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Idiopathic Thrombocytopenic Purpura
Keywords: Idiopathic Thrombocytopenic Purpura
ITP
Chronic Idiopathic Thrombocytopenic Purpura
Lead sponsor: Eisai Inc.
Sponsor class: INDUSTRY
First posted: 2008-02-28
Enrollment: 53
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to determine the safety and efficacy of AKR-501 (avatrombopag) administered in participants with chronic Idiopathic Thrombocytopenic Purpura (ITP) who were enrolled into and completed 28 days of study treatment in Protocol 501-CL-003 (NCT00441090).

Detailed description: Participants eligible to enroll into this rollover protocol will begin study treatment within 2-5 days of their Day 28 study termination visit in Protocol 501-CL-003 (NCT00441090). Participants who met the primary efficacy response criterion in Protocol 501-CL-003 will continue receiving the same study treatment to which they were assigned in the previous protocol in a double-blinded manner, these being one of the following 5 treatments:

* avatrombopag 2.5 mg daily
* avatrombopag 5 mg daily
* avatrombopag 10 mg daily
* avatrombopag 20 mg daily
* placebo

Participants who did not meet the primary efficacy response criterion in Protocol 501-CL-003 who otherwise meet the eligibility criteria for this rollover protocol will be offered open label avatrombopag 10 mg daily.

This is a parallel group, rollover study.

Eligibility criteria: Inclusion Criteria:

1. Patients who completed 28 days of study treatment in Protocol 501-CL-003.
2. No significant safety or tolerability concerns from the patient's participation of Protocol 501-CL-003 as determined by the Investigator.
3. Received medical monitor approval for enrollment into this study.
4. Patients receiving maintenance corticosteroids may be enrolled, as long as the corticosteroids have been administered at a stable dose and the Investigator does not foresee the need to change the steroid dose during study participation. Patients should remain on this stable corticosteroid dose during study participation.
5. Women of child-bearing potential must have a negative serum pregnancy test at the Day 28 assessment in Protocol 501-CL-003. (Childbearing potential is defined as any woman who has not been surgically sterilized and is pre-menopausal or peri-menopausal i.e., any menstrual flow within 12 months of Screening Visit A for Protocol 501-CL-003).
6. Women of child-bearing potential must agree to practice a medically approved form of contraception (one of the following must be used: condoms (male or female) with a spermicidal agent, diaphragm or cervical cap with a spermicidal agent, IUD,hormonal contraception, abstinence).
7. Willing and able to provide written informed consent.

Exclusion Criteria:

1. Women who are pregnant and/or lactating.
2. Use of the following drugs or treatments:

   * Rituximab
   * Azathioprine, Cyclosporine A, or other immunosuppressant therapy
   * Aspirin, Aspirin-containing compounds, Salicylates,Anticoagulants, Non-steroidal anti-inflammatory drugs(NSAIDs)(including Cyclooxygenase-2 \[COX-2\] specific NSAIDs), clopidogrel; ticlopidine; and any drugs that affect platelet function.
   * Danazol
   * Rh0(D) immune globulin (WinRho®) or intravenous immunoglobulin (IVIG).
3. Inability to comply with protocol requirements or give informed consent, as determined by the Investigator.

For more information regarding inclusion/exclusion criteria, please see record for AKR 501-CL-003 Protocol.

Primary outcomes:
measure: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
timeFrame: Day 1 through Month 6 while receiving treatment and at Month 7 after discontinuation of treatment.
measure: Incidence of Severe (Grade 3 or 4) TEAEs
timeFrame: Day 1 through Month 6 while receiving treatment and at Month 7 after discontinuation of treatment.
measure: Incidence of Drug-Related TEAEs
timeFrame: Day 1 through Month 6 while receiving treatment and at Month 7 after discontinuation of treatment.
Secondary outcomes:
measure: Median Platelet Counts at Selected Analysis Timepoints
timeFrame: Day 1, Weeks 2, 4, 6, 8, 10, 12, 14, 16, 18, 20, 22, 24, and Follow-up Weeks (after last dose of study drug in this study) 1, 2, 3, 4
measure: Percentage of Participants Who Achieved a Platelet Count of 100,000/mm^3 or Higher by Response Status and Selected Study Visit
timeFrame: Baseline (last PC before first dose of study drug in previous study), Weeks 2, 4, 8, 12, 16, 20, 24, and Follow-up Weeks (after last dose of study drug in this study) 1, 2, 3, 4
measure: Percentage of Participants Who Maintained a Platelet Count of 100,000/mm^3 or Higher by Response Status
timeFrame: Baseline (last PC before first dose of study drug in previous study), Weeks 2, 4, 8, 12, 16, 20, 24, and Follow-up Weeks (after last dose of study drug in this study) 1, 2, 3, 4
measure: Percentage of Participants Who Achieved Response-Level Platelet Count by Selected Study Visit
timeFrame: Baseline (last PC before first dose of study drug in previous study), Weeks 2, 4, 8, 12, 16, 20, 24, and Follow-up Weeks (after last dose of study drug in this study) 1, 2, 3, 4
measure: Percentage of Participants Who Maintained Response-Level Platelet Count
timeFrame: Baseline (last PC before first dose of study drug in previous study), Weeks 2, 4, 8, 12, 16, 20, 24, and Follow-up Weeks (after last dose of study drug in this study) 1, 2, 3, 4
measure: Percentage of Participants Who Achieved a Durable, Transient, or Overall Platelet Response
timeFrame: Baseline (last PC before first dose of study drug in previous study), Weeks 2, 4, 8, 12, 16, 20, 24, and Follow-up Weeks (after last dose of study drug in this study) 1, 2, 3, 4
measure: Number of Participants With Changes in Concomitant Steroid Use
timeFrame: Day 1 through last 8 weeks of the Treatment Period
Other outcomes:
Locations:
city: Anaheim
country: United States
facility: Pacific Cancer Medical Center, Inc
state: California
city: Bakersfield
country: United States
facility: Comprehensive Blood and Cancer Center
state: California
city: Manchester
country: United States
facility: Davis, Posteraro and Wasser, MDs, LLP
state: Connecticut
city: New Port Richey
country: United States
facility: Florida Cancer Institute
state: Florida
city: Chicago
country: United States
facility: John H. Stroger, Jr. Hospital of Cook County, Div. of Hematology and Oncology
state: Illinois
city: New Albany
country: United States
facility: Cancer Care Center, Inc.
state: Indiana
city: Jefferson City
country: United States
facility: Capitol Comprehensive Cancer Care Clinic
state: Missouri
city: Kansas City
country: United States
facility: Kansas City Cancer Center, LLC
state: Missouri
city: New York
country: United States
facility: Mount Sinai Medical Center
state: New York
city: New York
country: United States
facility: New York Presbyterian Hospital, Weill Medical College of Cornell University
state: New York
city: High Point
country: United States
facility: Emerywood Oncology and Hematology
state: North Carolina
city: Columbus
country: United States
facility: Mid Ohio Oncology/Hematology, Inc., dba The Mark H. Zangmeister Center
state: Ohio
Study officials:
affiliation: Eisai Inc.
name: Pei-Ran Ho, MD
role: STUDY_DIRECTOR
References:
citation: Bussel JB, Kuter DJ, Aledort LM, Kessler CM, Cuker A, Pendergrass KB, Tang S, McIntosh J. A randomized trial of avatrombopag, an investigational thrombopoietin-receptor agonist, in persistent and chronic immune thrombocytopenia. Blood. 2014 Jun 19;123(25):3887-94. doi: 10.1182/blood-2013-07-514398. Epub 2014 May 6.