Lower starting dose of avatrombopag for immune thrombocytopenia (ITP)

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

This pilot will try a lower starting dose of avatrombopag to raise platelet counts in adults with primary ITP who need second-line treatment.

Quick facts

• Phase: Phase 4
• Study type: Interventional
• Enrollment: 25 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Al-Mustansiriyah University (Academic / other)
• Drugs / interventions: prednisone
• Locations: 1 site (Baghdad)
• Trial ID: NCT07133659 on ClinicalTrials.gov

What this trial studies

This is an open-label, single-arm pilot conducted at a single center in Baghdad where adults with primary ITP who require second-line therapy will receive avatrombopag starting at 20 mg every other day instead of the standard 20 mg daily. Doses will be adjusted based on platelet response through a dose-adjustment phase, followed by a maintenance phase and a tapering/off-treatment follow-up phase. Treatment with avatrombopag is planned for up to six months with monitoring for platelet overshoot and adverse events. The trial aims to gather experience on dosing and to explore whether a lower starting dose improves safety while maintaining efficacy.

Who should consider this trial

Good fit: Adults (≥18 years) with primary ITP, platelet count <30 x10^9/L, who failed or relapsed after at least one course of corticosteroids and who have not previously received a thrombopoietin receptor agonist are the intended candidates.

Not a fit: Patients previously treated with TPO receptor agonists, those with secondary ITP, pregnant or breastfeeding individuals, those with active serious bleeding or high bleeding risk, or those unwilling to follow contraceptive guidance are unlikely to benefit or are excluded.

Why it matters

Potential benefit: If successful, a lower starting dose could reduce the frequency of excessive platelet rises and related side effects while keeping platelet counts controlled.

How similar studies have performed: Avatrombopag is approved and effective for chronic ITP, but formally testing a reduced starting dose is novel and not well studied in prior trials.

Eligibility criteria

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.

   \-

Where this trial is running

Baghdad

• hematology center / Medical City — Baghdad, Iraq (Recruiting)

Study contacts

• Study coordinator: Alaa Alwan, MD

Conditions ITP - Immune Thrombocytopenia; ITP; thrombocytopenia; Avatrombopag; thrombopoietin receptor agonist