Exploring the Efficacy, Safety of a Modified Starting Dosage of Avatrombopag in Immune Throbocytopenia (ITP) - a Pilot Study (Ava dosing)

August 14, 2025 updated by: Alaa Fadhil Alwan, Al-Mustansiriyah University
This is a single-center, open label pilot trial where patients with primary ITP who require second line treatment will be offered avatrombopag at a reduced starting dose, adjusted thereafter according to the response and continued for up to 24 weeks. The study aims to acquire experience on use of avatrombopag and explore the efficacy and safety of lower starting dose of avatrombopag

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

Avatrombopag is an oral thrombopoietin receptor agonist that is licensed for chronic ITP. Avatrombopag is administered at a starting dose of 20 mg daily. Overshooting of platelet count is a frequent problem that occurs in 20 to 40% of the patients after initiating avatrombopag as recommended. In this open label, single arm, pilot study, we will start avatrombopag at a reduced starting dose of 20 mg every other day. The dose will be adjusted thereafter according to the platelet response. The study consists of 3 phases: Dose adjustment phase, a maintenance phase, and dose tapering/disconsolation and follow-up phase. The study aims to acquire experience on use of avatrombopag and explore the efficacy and safety of lower starting avatrombopag-dose, and assess the rate of sustained response off-treatment. The duration of treatment with avatrombopag is 6 months.

The study is an investigator-initiated trial sponsored by Center for Transplantation and Blood Diseases. Medical City Complex, Baghdad Iraq.

Study Type

Interventional

Enrollment (Estimated)

25

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Iraq
      • Baghdad, Iraq, 10016
        • Recruiting
        • hematology center / Medical City
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Male or female aged ≥18 years.
  2. Diagnosis of primary ITP and having a platelet count of < 30 x109/L measured within two weeks prior to inclusion with failure to achieve response or relapse after at least one cycle of dexamethasone (20-40 mg daily for 4 days) or prednisone /prednisolone (1 mg/kg for at least two weeks). Shorter courses or lower doses are allowed if discontinued or modified due to side effects.
  3. Clinical need for second (subsequent) line treatment with a platelet elevating therapy assessed by the physician in charge.
  4. Signed and dated written informed consent.

Exclusion Criteria:

  1. Previous treatment with TPO-RA.
  2. Pregnancy or lactation.
  3. Patients with active serious bleeding or at high risk of bleeding as judged by physician in charge.
  4. Females of child-bearing potential refusing to follow effective contraceptive methods (as described in SmPC) during treatment with Avatrombopag.
  5. Secondary ITP defined as ITP secondary to lymphoma or chronic lymphocytic leukemia; ITP secondary to the following autoimmune disorders Systemic Lupus Erythematosus or Antiphospholipid Syndrome; ITP secondary to Common Variable Immune Deficiency; ITP secondary to the following viral infections eg Human Immunodeficiency Virus.
  6. Concomitant autoimmune hemolytic anemia, Evans syndrome.
  7. Presence of any serious comorbidity where the condition may worsen the study drugs.

  8. Presence of active malignancy unless deemed cured by adequate treatment. Participants with the following neoplastic conditions can be included:

    • Monoclonal gammopathy of undetermined significance (MGUS) or monoclonal B lymphocytosis of undetermined significance (MBUS)
    • Basal/squamous cell carcinoma of the skin
    • Carcinoma in situ of the cervix
    • Carcinoma in situ of the breast
    • Incidental histological finding of prostate cancer (TNM stage T1a or T1b)

  9. Patients with history of poor compliance or history of alcohol/drug abuse or excessive alcohol beverage consumption that would interfere with the ability to comply with the study protocol, or current or past psychiatric disease that might interfere with the ability to comply with the study protocol or give informed consent.

    -

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Avatrombopag
Description: Patients will start on avatrombopag 20 mg every other day for a week then the dose will be readjusted according to the platelet count.
Drug: Avatrombopag 20 mg Oral Tablet
Description: Patients will start on avatrombopag 20 mg every other day for a week then the dose will be readjusted according to the platelet count.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Total Response Time
Time Frame: 20 weeks
Median cumulative number of weeks with platelet count >50 x 109/L from time of Avatrombopag initiation to end of week 20. A period of 2 week will be deducted after IVIG and 4 weeks after dexamethasone.
20 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
TTR
Time Frame: 20 weeks
Time from initiation of dose to first platelet count > 50 x 109/L without any rescue therapy during the 2 weeks prior to the blood sampling.
20 weeks
Response by day 8
Time Frame: 8 days
Number of patients achieving platelet count > 50 x 109/L by day 8 without any rescue therapy during the 2 weeks prior to the blood sampling.
8 days
Rates of platelet overshoot.
Time Frame: 6 weeks
Number of episode with platelet count > 200 and >400 x 109/L during the first 6 weeks after the initiation of avatrombopag.
6 weeks
Durable response rate.
Time Frame: 20 weeks
Number of patients achieving 4 consecutive platelet counts > 50 x 109/L between weeks 12 and 20 including the last count, without the use of rescue therapy, corticosteroids or any platelet elevating agent after week 6.
20 weeks
The rates of treatment failure.
Time Frame: 20 weeks

Occurrence of treatment failure is defined as:

Discontinuation of Avatrombopag and switching to another platelet elevating agent between weeks 6 and 20 due to non-response or intolerance to Avatrombopag or administration of rescue therapy after week 6 and Thrombocytopenia (platelet count <30 x 109/L), high risk of bleeding or intolerance to avatrombopag.
20 weeks
Bleeding complications during the study.
Time Frame: 52 weeks
Number and severity of WHO bleeding events.
52 weeks
The safety of treatment with Avatrombopag.
Time Frame: 52 weeks
Occurrence and severity of treatment emergent adverse events. Occurrence and severity of adverse events of special interest including arterial and venous thrombosis and bone marrow fibrosis (bone marrow biopsy showing MF2 or higher).
52 weeks
The changes in HRQoL during the study.
Time Frame: 20 weeks
Change in of SF-36 (v1) questionnaires from baseline to weeks 20.
20 weeks
The rates of Sustained Response Off-Treatment (SROT) at week 52.
Time Frame: 52 weeks

Occurrence of SROT defined as:

A platelet count > 30 x 109/L in all planned visits between the time of discontinuation of Avatrombopag and week 52 including week 52 and no administration of platelet elevating agent between weeks 20 and 52.
52 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Al-Mustansiriyah University

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 8, 2025

Primary Completion (Estimated)

July 8, 2026

Study Completion (Estimated)

July 8, 2027

Study Registration Dates

First Submitted

August 14, 2025

First Submitted That Met QC Criteria

August 14, 2025

First Posted (Actual)

August 21, 2025

Study Record Updates

Last Update Posted (Actual)

August 21, 2025

Last Update Submitted That Met QC Criteria

August 14, 2025

Last Verified

August 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • RGHC007

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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