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

NCT00625443 | Completed Phase 2 Results

• 1 other identifier: AKR-501-CL-004
• Study Type: interventional
• Target: 53
• Locations: 1 country
• Sites: 12

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).

Conditions

Idiopathic Thrombocytopenic Purpura

Keywords

Idiopathic Thrombocytopenic Purpura; ITP; Chronic Idiopathic Thrombocytopenic Purpura

Outcome Measures

Primary Outcomes (3)

• Number of Participants With Treatment-Emergent Adverse Events (TEAEs)

  A TEAE was defined as 1) the AE started on or after the date of first dose of study drug up to and including 30 days after the last dose of study drug, or 2) the AE started before the first dose of study drug but worsened in severity on or after the date of first dose of study drug up to and including 3 days after the last dose of study drug. For participants having a tapering period of study drug, the last dose day is the last day of study drug during the tapering period. Related AEs included those whose relationship was categorized as possible or probably by the investigator. Dose interruption includes dose decreased, dose previously stopped, permanently stopped, or temporarily stopped. The safety population was divided into three subgroups based on the mean daily dose of active study drug taken; lower 1/3 (less than 8.85 mg avatrombopag), middle 1/3 (greater than or equal to 8.85 mg but less than 13.5 mg avatrombopag), and upper 1/3 (greater than or equal to 13.5 mg avatrombopag).

  Day 1 through Month 6 while receiving treatment and at Month 7 after discontinuation of treatment.

• Incidence of Severe (Grade 3 or 4) TEAEs

  A TEAE was defined as 1) the AE started on or after the date of first dose of study drug up to and including 30 days after the last dose of drug, or 2) the AE started before the first dose of study drug but worsened in severity on or after the date of first dose of study drug up to and including 3 days after the last dose of study drug. For participants having a tapering period of study drug, the last dose day is the last day of study drug during the tapering period. The safety population was divided into three subgroups based on the mean daily dose of active study drug taken; lower 1/3 (less than 8.85 mg avatrombopag), middle 1/3 (greater than or equal to 8.85 mg but less than 13.5 mg avatrombopag), and upper 1/3 (greater than or equal to 13.5 mg avatrombopag).

  Day 1 through Month 6 while receiving treatment and at Month 7 after discontinuation of treatment.

• Incidence of Drug-Related TEAEs

  Drug-related TEAEs included those whose relationship was categorized as possible or probably by the investigator. A TEAE was defined as 1) the AE started on or after the date of first dose of study drug up to and including 30 days after the last dose of drug, or 2) the AE started before the first dose of study drug but worsened in severity on or after the date of first dose of study drug up to and including 3 days after the last dose of study drug. For participants having a tapering period of study drug, the last dose day is the last day of study drug during the tapering period. The safety population was divided into three subgroups based on the mean daily dose of active study drug taken; lower 1/3 (less than 8.85 mg avatrombopag), middle 1/3 (greater than or equal to 8.85 mg but less than 13.5 mg avatrombopag), and upper 1/3 (greater than or equal to 13.5 mg avatrombopag).

  Day 1 through Month 6 while receiving treatment and at Month 7 after discontinuation of treatment.

Secondary Outcomes (7)

• Median Platelet Counts at Selected Analysis Timepoints

  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

• Percentage of Participants Who Achieved a Platelet Count of 100,000/mm^3 or Higher by Response Status and Selected Study Visit

  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

• Percentage of Participants Who Maintained a Platelet Count of 100,000/mm^3 or Higher by Response Status

  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

• Percentage of Participants Who Achieved Response-Level Platelet Count by Selected Study Visit

  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

• Percentage of Participants Who Maintained Response-Level Platelet Count

  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

Study Arms (3)

Placebo (double-blind)

EXPERIMENTAL

Drug: Blinded (placebo)

Avatrombopag tablets (open-label)

EXPERIMENTAL

Drug: Open Label (Avatrombopag tablets)

Avatrombopag tablets (double-blind)

EXPERIMENTAL

Drug: Blinded (Avatrombopoag tablets)

Interventions

Blinded (placebo) DRUG

Placebo Orally, once daily administered under fasting conditions (at least 1 hr prior to or at least 2 hours after a meal or snack) Duration - 6 months

Placebo (double-blind)

Open Label (Avatrombopag tablets) DRUG

Dose 10 mg Orally, once daily administered under fasting conditions (at least 1 hr prior to or at least 2 hours after a meal or snack) Duration - 6 months

Also known as: AKR-501, E5501, YM477

Avatrombopag tablets (open-label)

Blinded (Avatrombopoag tablets) DRUG

Dose: 2.5, 5, 10, or 20 mg Orally, once daily administered under fasting conditions (at least 1 hr prior to or at least 2 hours after a meal or snack) Duration - 6 months

Also known as: AKR-501, E5501, YM477

Avatrombopag tablets (double-blind)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients who completed 28 days of study treatment in Protocol 501-CL-003.
• No significant safety or tolerability concerns from the patient's participation of Protocol 501-CL-003 as determined by the Investigator.
• Received medical monitor approval for enrollment into this study.
• 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.
• 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).
• 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).
• Willing and able to provide written informed consent.

You may not qualify if:

• Women who are pregnant and/or lactating.
• 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).
• Inability to comply with protocol requirements or give informed consent, as determined by the Investigator.

Sponsors & Collaborators

• Eisai Inc.: lead

Study Sites (12)

Pacific Cancer Medical Center, Inc

Anaheim, California, 92801, United States

Location

Comprehensive Blood and Cancer Center

Bakersfield, California, 93309, United States

Location

Davis, Posteraro and Wasser, MDs, LLP

Manchester, Connecticut, 06105, United States

Location

Florida Cancer Institute

New Port Richey, Florida, 34655, United States

Location

John H. Stroger, Jr. Hospital of Cook County, Div. of Hematology and Oncology

Chicago, Illinois, 60612, United States

Location

Cancer Care Center, Inc.

New Albany, Indiana, 47150, United States

Location

Capitol Comprehensive Cancer Care Clinic

Jefferson City, Missouri, 65109, United States

Location

Kansas City Cancer Center, LLC

Kansas City, Missouri, 64131, United States

Location

Mount Sinai Medical Center

New York, New York, 10029, United States

Location

New York Presbyterian Hospital, Weill Medical College of Cornell University

New York, New York, 10032, United States

Location

Emerywood Oncology and Hematology

High Point, North Carolina, 27262, United States

Location

Mid Ohio Oncology/Hematology, Inc., dba The Mark H. Zangmeister Center

Columbus, Ohio, 43219, United States

Location

Related Publications (1)

• 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.

  PMID: 24802775 DERIVED

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

avatrombopag

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Results Point of Contact

• Title: Eisai Medical Services
• Organization: Eisai Inc.

1-888-422-4743

Study Officials

• Pei-Ran Ho, MD

  Eisai Inc.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 19, 2008
• First Posted: February 28, 2008
• Study Start: May 1, 2007
• Primary Completion: June 1, 2009
• Study Completion: October 1, 2009
• Last Updated: March 16, 2018
• Results First Posted: March 16, 2018

Record last verified: 2018-03

Locations

US (12)