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

NCT05046327 | Withdrawn Phase 3 FDA Drug

• 1 other identifier: AVA-ITP-306
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Immune Thrombocytopenia

Keywords

ITP

Outcome Measures

Primary Outcomes (1)

• 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.

  6 Months of Active Treatment

Secondary Outcomes (3)

• Durable Platelet Response

  8 Weeks of Treatment

• Incidence of ITP remission

  24 Consecutive Weeks

• Incidence of subjects achieving a platelet count response

  6 Months of Active Treatment

Study Arms (1)

20 mg Avatrombopag daily

EXPERIMENTAL

Avatrombopag

Drug: Avatrombopag 20 mg Oral Tablet

Interventions

Avatrombopag 20 mg Oral Tablet DRUG

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

Also known as: Doptelet

20 mg Avatrombopag daily

Eligibility Criteria

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

You may qualify if:

• 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.

You may not qualify if:

• Thrombocytopenia due to a known condition other than primary ITP (e.g., systemic lupus erythematosus, H. pylori infection, splenomegaly, chronic liver disease).
• 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.

Sponsors & Collaborators

• Sobi, Inc.: lead

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

avatrombopag; Tablets

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

Intervention Hierarchy (Ancestors)

Dosage Forms; Pharmaceutical Preparations

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 8, 2021
• First Posted: September 16, 2021
• Study Start: October 14, 2021
• Primary Completion: April 6, 2024
• Study Completion: February 19, 2025
• Last Updated: October 22, 2021

Record last verified: 2021-10

Data Sharing

• IPD Sharing: Will not share