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Open-Label Study to Evaluate the Safety and Efficacy of Avatrombopag and Remission Rates in Adults With ITP of ≤6 Months

Primary Purpose

Immune Thrombocytopenia

Status

Withdrawn

Phase

Phase 3

Locations

Study Type

Interventional

Intervention

Avatrombopag 20 mg Oral Tablet

About this trial

This is an interventional treatment trial for Immune Thrombocytopenia focused on measuring ITP

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Male or female subjects ≥18 years of age at Screening.
Subject must be able to provide informed consent.
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.
Subject has at least one platelet count <30×10⁹/L at any time during the screening period or at the Baseline visit.
Females of childbearing potential must have a negative pregnancy test at Screening and Baseline.
Female subjects of childbearing potential who are sexually active and male subjects who are sexually active must agree to use effective methods of contraception.
Subject is willing and able to comply with all aspects of the protocol.

Exclusion Criteria:

Thrombocytopenia due to a known condition other than primary ITP (e.g., systemic lupus erythematosus, H. pylori infection, splenomegaly, chronic liver disease).
Any history of arterial or venous thrombosis, including partial or complete thrombosis (history of superficial thrombophlebitis is not exclusionary).
Subjects with known inherited thrombocytopenia (e.g., MYH-9 disorders).
History of myelodysplastic syndrome (MDS) or other hematologic malignancies.
Current history of significant cardiac arrhythmias or decompensated congestive heart failure.
History of hepatitis B (HBV), hepatitis C (HCV), or human immunodeficiency virus (HIV).
Previous use of eltrombopag, romiplostim, recombinant human TPO or other platelet-producing agents.
Surgical resection of the spleen.
Previous use of mycophenolate mofetil (MMF), rituximab (or other B-cell lymphocyte depleting agents), mercaptopurine (6-MP) or alkylating agents.
Concurrent malignant disease, other than non-melanoma skin cancer or cervical cancer in-situ.
Known allergy to avatrombopag or any of its excipients.
Subject is unable to take oral medication or has a malabsorption syndrome or any other uncontrolled gastrointestinal condition.
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.
Any clinically relevant abnormality which makes the subject unsuitable for participation in the study, in the opinion of the Investigator.
Considered unable or unwilling to comply with the study protocol requirements.

Sites / Locations

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

20 mg Avatrombopag daily

Arm Description

Avatrombopag

Outcomes

Primary Outcome Measures

Cumulative Number of Weeks of Platelet Response

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

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

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

Incidence of subjects achieving a platelet count response (≥50×10⁹/L) during the active treatment period of the study.

Full Information

NCT ID

NCT05046327

First Posted

September 8, 2021

Last Updated

October 15, 2021

Sponsor

Sobi, Inc.

1. Study Identification

Unique Protocol Identification Number

NCT05046327

Brief Title

Open-Label Study to Evaluate the Safety and Efficacy of Avatrombopag and Remission Rates in Adults With ITP of ≤6 Months

Official Title

A Multicenter, Open-Label Study to Evaluate the Safety and Efficacy of Avatrombopag and Remission Rates in Adults With Immune Thrombocytopenia (ITP) of ≤6 Months Duration

Study Type

Interventional

2. Study Status

Record Verification Date

October 2021

Overall Recruitment Status

Withdrawn

Why Stopped

Corporate Decision

Study Start Date

October 14, 2021 (Anticipated)

Primary Completion Date

April 6, 2024 (Anticipated)

Study Completion Date

February 19, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Sobi, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. 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.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Immune Thrombocytopenia

Keywords

ITP

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

0 (Actual)

8. Arms, Groups, and Interventions

Arm Title

20 mg Avatrombopag daily

Arm Type

Experimental

Arm Description

Avatrombopag

Intervention Type

Drug

Intervention Name(s)

Avatrombopag 20 mg Oral Tablet

Other Intervention Name(s)

Doptelet

Intervention Description

Avatrombopag administered at a frequency to maintain a target platelet count between ≥50 and ≤150×10⁹/L

Primary Outcome Measure Information:

Title

Cumulative Number of Weeks of Platelet Response

Description

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.

Time Frame

6 Months of Active Treatment

Secondary Outcome Measure Information:

Title

Durable Platelet Response

Description

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.

Time Frame

8 Weeks of Treatment

Title

Incidence of ITP remission

Description

Incidence of ITP remission as defined by platelet count ≥50×10⁹/L for 24 consecutive weeks with no ITP treatments (concomitant or rescue).

Time Frame

24 Consecutive Weeks

Title

Incidence of subjects achieving a platelet count response

Description

Incidence of subjects achieving a platelet count response (≥50×10⁹/L) during the active treatment period of the study.

Time Frame

6 Months of Active Treatment

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Male or female subjects ≥18 years of age at Screening. Subject must be able to provide informed consent. 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. Subject has at least one platelet count <30×10⁹/L at any time during the screening period or at the Baseline visit. Females of childbearing potential must have a negative pregnancy test at Screening and Baseline. Female subjects of childbearing potential who are sexually active and male subjects who are sexually active must agree to use effective methods of contraception. Subject is willing and able to comply with all aspects of the protocol. Exclusion Criteria: Thrombocytopenia due to a known condition other than primary ITP (e.g., systemic lupus erythematosus, H. pylori infection, splenomegaly, chronic liver disease). Any history of arterial or venous thrombosis, including partial or complete thrombosis (history of superficial thrombophlebitis is not exclusionary). Subjects with known inherited thrombocytopenia (e.g., MYH-9 disorders). History of myelodysplastic syndrome (MDS) or other hematologic malignancies. Current history of significant cardiac arrhythmias or decompensated congestive heart failure. History of hepatitis B (HBV), hepatitis C (HCV), or human immunodeficiency virus (HIV). Previous use of eltrombopag, romiplostim, recombinant human TPO or other platelet-producing agents. Surgical resection of the spleen. Previous use of mycophenolate mofetil (MMF), rituximab (or other B-cell lymphocyte depleting agents), mercaptopurine (6-MP) or alkylating agents. Concurrent malignant disease, other than non-melanoma skin cancer or cervical cancer in-situ. Known allergy to avatrombopag or any of its excipients. Subject is unable to take oral medication or has a malabsorption syndrome or any other uncontrolled gastrointestinal condition. 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. Any clinically relevant abnormality which makes the subject unsuitable for participation in the study, in the opinion of the Investigator. Considered unable or unwilling to comply with the study protocol requirements.

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

Open-Label Study to Evaluate the Safety and Efficacy of Avatrombopag and Remission Rates in Adults With ITP of ≤6 Months

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