Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia for ≥6 Months
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
1 other identifier
interventional
75
9 countries
62
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
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Mar 2021
Longer than P75 for phase_3
62 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
August 14, 2020
CompletedFirst Posted
Study publicly available on registry
August 18, 2020
CompletedStudy Start
First participant enrolled
March 5, 2021
CompletedPrimary Completion
Last participant's last visit for primary outcome
November 8, 2023
CompletedResults Posted
Study results publicly available
January 17, 2025
CompletedStudy Completion
Last participant's last visit for all outcomes
October 28, 2025
CompletedApril 22, 2026
March 1, 2026
2.7 years
August 14, 2020
November 7, 2024
April 2, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (2)
Durable Platelet Response
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
Last 8 weeks of 12 week treatment regimen
Alternative Primary: Platelet Response
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.
12 weeks of treatment
Secondary Outcomes (5)
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
Study Arms (3)
Experimental: Avatrombopag Double Blind
EXPERIMENTALStudy is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks
Placebo Comparator:Placebo Double Blind
PLACEBO COMPARATORStudy is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks
Avatrombopag Open Label Extension
EXPERIMENTALInvestigational product administered orally for up to 2 years.
Interventions
Oral avatrombopag tablet or sprinkle capsule
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Sobi, Inc.lead
Study Sites (62)
Site 112
Phoenix, Arizona, 85016, United States
Site 103
Long Beach, California, 90806, United States
Site 119
Orange, California, 92868, United States
Site 109
Sacramento, California, 95817, United States
Site 101
San Francisco, California, 94158, United States
Site 111
Aurora, Colorado, 80045, United States
Site 120
Wilmington, Delaware, 19803, United States
Site 117
Gainesville, Florida, 32610, United States
Site 116
Atlanta, Georgia, 30322, United States
Site 107
Peoria, Illinois, 61615, United States
Site 104
Boston, Massachusetts, 02115, United States
Site 105
Minneapolis, Minnesota, 55404, United States
Site 114
Jackson, Mississippi, 39216, United States
Site 102
Las Vegas, Nevada, 89135, United States
Site 108
Morristown, New Jersey, 07960, United States
Site 118
Charlotte, North Carolina, 28203, United States
Site 113
Durham, North Carolina, 27710, United States
Site 106
Columbus, Ohio, 43205, United States
Site 121
Philadelphia, Pennsylvania, 19104, United States
Site 110
Providence, Rhode Island, 02903, United States
Site 115
Houston, Texas, 77030, United States
Site 201
Toulouse, Occitanie, 31059, France
Site 202
Paris, Île-de-France Region, 75019, France
Site 301
Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany
Site 302
Hamburg, Free and Hanseatic City of Hamburg, 20246, Germany
Site 303
Kiel, Schleswig-Holstein, 24105, Germany
Site 304
Berlin, State of Berlin, 13353, Germany
Site 801
Budapest, 1094, Hungary
Site 802
Miskolc, 3526, Hungary
Site 803
Pécs, 7623, Hungary
Site 502
Bydgoszcz, Kuyavian-Pomeranian Voivodeship, 85-667, Poland
Site 503
Warsaw, Masovian Voivodeship, 02-091, Poland
Site 505
Zabrze, Silesian Voivodeship, 41-800, Poland
Site 501
Olsztyn, Warmian-Masurian Voivodeship, 10-561, Poland
Site 504
Lodz, Łódź Voivodeship, 91-738, Poland
Site 907
Chelyabinsk, 454076, Russia
Site 904
Moscow, 117198, Russia
Site 906
Moscow, 117997, Russia
Site 901
Moscow, 119049, Russia
Site 902
Nizhny Novgorod, 603136, Russia
Site 905
Saint Petersburg, 197022, Russia
Site 903
Volgograd, 400138, Russia
Site 701
Adana, 01330, Turkey (Türkiye)
Site 704
Ankara, 06230, Turkey (Türkiye)
Site 706
Antalya, 07059, Turkey (Türkiye)
Site 702
Antalya, 07160, Turkey (Türkiye)
Site 705
Denizli, 20160, Turkey (Türkiye)
Site 709
Istanbul, 34093, Turkey (Türkiye)
Site 710
Istanbul, 34093, Turkey (Türkiye)
Site 703
Istanbul, 34764, Turkey (Türkiye)
Site 707
Izmir, 35100, Turkey (Türkiye)
Site 708
Mersin, 33110, Turkey (Türkiye)
Site 954
Kharkiv, Kharkivs’ka Oblast’, 61000, Ukraine
Site 952
Vinnytsia, 21029, Ukraine
Site 405
Birmingham, England, B4 6NH, United Kingdom
Site 408
Liverpool, England, L12 2AP, United Kingdom
Site 403
London, England, SE5 9RS, United Kingdom
Site 410
London, England, W12 0HS, United Kingdom
Site 407
London, England, WC1N 3JH, United Kingdom
Site 402
Manchester, England, M13 9WL, United Kingdom
Site 401
Sheffield, England, S10 2TH, United Kingdom
Site 406
Cardiff, Wales, CF14 4XW, United Kingdom
Related Publications (1)
Grace RF, Leblebisatan G, Aydinok Y, Unal S, Grainger JD, Zhang J, Smallwood L, de Leon E, Jamieson BD; AVA-PED-301 Study Group. Avatrombopag for the treatment of children and adolescents with immune thrombocytopenia (AVA-PED-301): a multicentre, randomised, double-blind, placebo-controlled, phase 3b study. Lancet Haematol. 2025 Jul;12(7):e494-e504. doi: 10.1016/S2352-3026(25)00107-3. Epub 2025 May 23.
PMID: 40418942DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Medicine Development Leader
- Organization
- Sobi Inc.
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- DOUBLE
- Who Masked
- PARTICIPANT, INVESTIGATOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
August 14, 2020
First Posted
August 18, 2020
Study Start
March 5, 2021
Primary Completion
November 8, 2023
Study Completion
October 28, 2025
Last Updated
April 22, 2026
Results First Posted
January 17, 2025
Record last verified: 2026-03
Data Sharing
- IPD Sharing
- Will not share