Open-Label Study to Evaluate the Safety and Efficacy of Avatrombopag and Remission Rates in Adults With ITP of ≤6 Months
Study Details
Study Description
Brief Summary
This study will evaluate the safety and efficacy of avatrombopag in subjects with a confirmed diagnosis of primary ITP (≤6 months duration) over 26 weeks of treatment, and also evaluate the incidence of ITP remission.
Condition or Disease | Intervention/Treatment | Phase |
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Phase 3 |
Detailed Description
This phase 3b, multi-center, open-label study will enroll approximately 75 adult subjects with a confirmed diagnoses of primary ITP (≤6 months duration) who have had a previous response to a first line treatment. The study will consist of a 26-week treatment period to evaluate the safety and efficacy of avatrombopag. Subjects with platelet counts ≥50×10⁹/L at Week 26 may enter a dose-tapering period in which the dose of avatrombopag will be decreased for up to 16 weeks until avatrombopag treatment is discontinued and the platelet count is maintained ≥50×10⁹/L. Once avatrombopag treatment has been discontinued, the subjects will enter a remission evaluation period of up to 24 weeks to evaluate whether they have entered a state of remission.
Study Design
Arms and Interventions
Arm | Intervention/Treatment |
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Experimental: 20 mg Avatrombopag daily Avatrombopag |
Drug: Avatrombopag 20 mg Oral Tablet
Avatrombopag administered at a frequency to maintain a target platelet count between ≥50 and ≤150×10⁹/L
Other Names:
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Outcome Measures
Primary Outcome Measures
- Cumulative Number of Weeks of Platelet Response [6 Months of Active Treatment]
Cumulative number of weeks of platelet response in which the platelet count is ≥50×10⁹/L during 6 months of treatment in the absence of rescue therapy.
Secondary Outcome Measures
- Durable Platelet Response [8 Weeks of Treatment]
Durable platelet response as defined by the incidence of subjects who have at least 6 out of 8 weekly platelet counts ≥50×10⁹/L during the last 8 weeks of treatment.
- Incidence of ITP remission [24 Consecutive Weeks]
Incidence of ITP remission as defined by platelet count ≥50×10⁹/L for 24 consecutive weeks with no ITP treatments (concomitant or rescue).
- Incidence of subjects achieving a platelet count response [6 Months of Active Treatment]
Incidence of subjects achieving a platelet count response (≥50×10⁹/L) during the active treatment period of the study.
Eligibility Criteria
Criteria
Inclusion Criteria:
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Male or female subjects ≥18 years of age at Screening.
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Subject must be able to provide informed consent.
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Subject has a confirmed diagnosis of primary ITP according to the International Consensus Report on the Investigation and Management of Primary ITP within the previous 6 months prior to Visit 1 and has had a previous response to a first line treatment (corticosteroids, IVIg, or anti-D), in the opinion of the Investigator.
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Subject has at least one platelet count <30×10⁹/L at any time during the screening period or at the Baseline visit.
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Females of childbearing potential must have a negative pregnancy test at Screening and Baseline.
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Female subjects of childbearing potential who are sexually active and male subjects who are sexually active must agree to use effective methods of contraception.
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Subject is willing and able to comply with all aspects of the protocol.
Exclusion Criteria:
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Thrombocytopenia due to a known condition other than primary ITP (e.g., systemic lupus erythematosus, H. pylori infection, splenomegaly, chronic liver disease).
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Any history of arterial or venous thrombosis, including partial or complete thrombosis (history of superficial thrombophlebitis is not exclusionary).
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Subjects with known inherited thrombocytopenia (e.g., MYH-9 disorders).
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History of myelodysplastic syndrome (MDS) or other hematologic malignancies.
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Current history of significant cardiac arrhythmias or decompensated congestive heart failure.
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History of hepatitis B (HBV), hepatitis C (HCV), or human immunodeficiency virus (HIV).
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Previous use of eltrombopag, romiplostim, recombinant human TPO or other platelet-producing agents.
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Surgical resection of the spleen.
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Previous use of mycophenolate mofetil (MMF), rituximab (or other B-cell lymphocyte depleting agents), mercaptopurine (6-MP) or alkylating agents.
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Concurrent malignant disease, other than non-melanoma skin cancer or cervical cancer in-situ.
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Known allergy to avatrombopag or any of its excipients.
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Subject is unable to take oral medication or has a malabsorption syndrome or any other uncontrolled gastrointestinal condition.
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Enrollment in another clinical study with any investigational drug or device within 30 days of Day 1/Visit 2 (or 5 half-lives, whichever is longer); however, participation in observational studies is permitted.
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Any clinically relevant abnormality which makes the subject unsuitable for participation in the study, in the opinion of the Investigator.
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Considered unable or unwilling to comply with the study protocol requirements.
Contacts and Locations
Locations
No locations specified.Sponsors and Collaborators
- Sobi, Inc.
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- AVA-ITP-306