Randomized, Double-blind, Placebo-controlled Study With Open-label Extension Phase to Evaluate the Efficacy and Safety of Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia for ≥6 Months
Trial Snapshot
- Phase
- Phase 3
- Status
- Completed
- Sponsor
- Sobi, Inc.
- Enrollment
- 75
- Locations
- 67
- Primary Endpoint
- Durable Platelet Response
Study Overview
Brief 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
Detailed Description
Subjects will be randomized in to blinded therapy of avatrombopag or placebo in a 3:1 ratio for a period of 12 weeks. Subjects who complete the 12 week treatment period and are eligible may continue to the open label extension phase which will last 2 years.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Double (Participant, Investigator)
Eligibility Criteria
- Ages
- 1 Year to 17 Years (Child)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Male or female participants ≥1 and <18 years of age at Screening and Baseline with a diagnosis of primary ITP for ≥6 months duration and has had an insufficient response to a previous treatment, in the opinion of the Investigator.
- •Participant has an average of 2 platelet counts <30×10^9/L with no single count >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.
Arms & Interventions
Experimental: Avatrombopag Double Blind
Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks
Intervention: Avatrombopag (Drug)
Avatrombopag Open Label Extension
Investigational product administered orally for up to 2 years.
Intervention: Avatrombopag (Drug)
Placebo Comparator:Placebo Double Blind
Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks
Intervention: Placebo (Drug)
Outcomes
Primary Outcomes
Durable Platelet Response
Time Frame: Last 8 weeks of 12 week treatment regimen
The proportion of subjects achieving at least 6 out of 8 weekly platelet counts ≥50×10\^9/L during the last 8 weeks of the 12 week Treatment Period in the Core Phase, in the absence of rescue medication
Alternative Primary: Platelet Response
Time Frame: 12 weeks of treatment
The proportion of subjects for whom at least 2 consecutive platelet assessments are ≥50×10\^9/L over the 12 weeks of treatment in the Core Phase in the absence of rescue medication.
Secondary Outcomes
- Percentage of Weeks Platelet Count ≥50×10^9/L(12 weeks of treatment)
- Platelet Response at Day 8(Day 8)
- Percentage of Weeks Platelet Count Between ≥50×10^9/L and ≤150×10^9/L(12 weeks of treatment)
- Rescue Medications(12 weeks of treatment)
- Incidence of Any Bleeding Event (WHO Grade 1-4)(12 weeks of treatment)