Phase 3
N=75
Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia for ≥6 Months
Immune Thrombocytopenia
Bottom Line
View on ClinicalTrials.gov: NCT04516967 ↗Enrolled (actual)
75
Serious AEs
18.9%
Results posted
Jan 2025
Primary outcome: Primary: Durable Platelet Response — 15; 0 Participants — p=0.0077
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Avatrombopag (Drug); Placebo (Drug)
- Age
- Pediatric · 1+ yrs
- Sex
- All
- Sponsor
- Sobi, Inc.
- Primary completion
- Nov 2023
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 |
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
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). 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.