Phase 3
Completed N=75
Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia for ≥6 Months
Source: ClinicalTrials.gov NCT04516967 ↗Enrolled (actual)
75
Serious AEs
18.9%
Results posted
Jan 2025
Primary outcomePrimary: Durable Platelet Response — 15; 0 Participants — p=0.0077
◆ Published Evidence
Emerging
7citations · ~7 / year
Avatrombopag for the treatment of children and adolescents with immune thrombocytopenia (AVA-PED-301): a multicentre, randomised, double-blind, placebo-controlled, phase 3b study.
Summary
A Phase 3b Study to Evaluate the Efficacy and Safety of Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects with Immune Thrombocytopenia for ≥6 Months
Linked Publications (2)
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Avatrombopag for the treatment of children and adolescents with immune thrombocytopenia (AVA-PED-301): a multicentre, randomised, double-blind, placebo-controlled, phase 3b study.
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Avatrombopag for treating children and adolescents with immune thrombocytopenia: a plain language summary.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Durable Platelet Response |
15; 0 | 0.0077 sig |
| PRIMARY Alternative Primary: Platelet Response |
44; 0 | <0.0001 sig |
| SECONDARY Percentage of Weeks Platelet Count ≥50×10^9/L |
48.9; 1.2 | <0.0001 sig |
| SECONDARY Platelet Response at Day 8 |
30; 0 | <0.0001 sig |
| SECONDARY Percentage of Weeks Platelet Count Between ≥50×10^9/L and ≤150×10^9/L |
29.2; 1.2 | <0.0001 sig |
| SECONDARY Rescue Medications |
4; 9 | 0.0008 sig |
| SECONDARY Incidence of Any Bleeding Event (WHO Grade 1-4) |
45; 19 | 0.7175 |
Eligibility Criteria
Inclusion Criteria
- Male or female participants ≥1 and 35×10^9/L in the screening period
Exclusion Criteria
- Participants must not have a known history of secondary ITP, any history of arterial or venous thrombosis, including partial or complete thrombosis, known inherited thrombocytopenia, a history of myelodysplastic syndrome (MDS) or known history of congenital heart abnormalities or arrhythmias.
Data sourced from ClinicalTrials.gov (NCT04516967) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.