Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia for ≥6 Months
- Conditions
- Immune Thrombocytopenia
- Interventions
- Drug: Placebo
- Registration Number
- NCT04516967
- Lead Sponsor
- Sobi, Inc.
- 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.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 75
- 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
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Experimental: Avatrombopag Double Blind Avatrombopag Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks Placebo Comparator:Placebo Double Blind Placebo Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks Avatrombopag Open Label Extension Avatrombopag Investigational product administered orally for up to 2 years.
- Primary Outcome Measures
Name Time Method Durable Platelet Response 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 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 Outcome Measures
Name Time Method Percentage of Weeks Platelet Count ≥50×10^9/L 12 weeks of treatment The percentage of weeks subjects have a platelet count ≥50×10\^9/L during 12 weeks of treatment in the Core Phase, in the absence of rescue therapy.
Platelet Response at Day 8 Day 8 The proportion of subjects with a platelet count ≥50×10\^9/L at day 8, in the absence of rescue therapy.
Percentage of Weeks Platelet Count Between ≥50×10^9/L and ≤150×10^9/L 12 weeks of treatment The percentage of weeks subjects have a platelet count between ≥50×10\^9/L and ≤150×10\^9/L, during 12 weeks of treatment in the Core Phase, in the absence of rescue therapy.
Rescue Medications 12 weeks of treatment The proportion of subjects who require rescue medications during 12 weeks of treatment in the Core Phase of the study.
Incidence of Any Bleeding Event (WHO Grade 1-4) 12 weeks of treatment Overall incidence of bleeding events associated with ITP measured using the WHO Bleeding Scale (Grade 1-4).
Trial Locations
- Locations (62)
Site 116
🇺🇸Atlanta, Georgia, United States
Site 114
🇺🇸Jackson, Mississippi, United States
Site 121
🇺🇸Philadelphia, Pennsylvania, United States
Site 407
🇬🇧London, England, United Kingdom
Site 402
🇬🇧Manchester, England, United Kingdom
Site 118
🇺🇸Charlotte, North Carolina, United States
Site 106
🇺🇸Columbus, Ohio, United States
Site 110
🇺🇸Providence, Rhode Island, United States
Site 115
🇺🇸Houston, Texas, United States
Site 105
🇺🇸Minneapolis, Minnesota, United States
Site 111
🇺🇸Aurora, Colorado, United States
Site 303
🇩🇪Kiel, Schleswig-Holstein, Germany
Site 803
🇭🇺Pécs, Hungary
Site 117
🇺🇸Gainesville, Florida, United States
Site 952
🇺🇦Vinnytsia, Ukraine
Site 120
🇺🇸Wilmington, Delaware, United States
Site 707
🇹🇷Izmir, Turkey
Site 954
🇺🇦Kharkiv, Kharkiv Region, Ukraine
Site 104
🇺🇸Boston, Massachusetts, United States
Site 112
🇺🇸Phoenix, Arizona, United States
Site 405
🇬🇧Birmingham, England, United Kingdom
Site 406
🇬🇧Cardiff, Wales, United Kingdom
Site 710
🇹🇷Istanbul, Turkey
Site 401
🇬🇧Sheffield, England, United Kingdom
Site 802
🇭🇺Miskolc, Hungary
Site 502
🇵🇱Bydgoszcz, Kujawsko-Pomorskie, Poland
Site 708
🇹🇷Mersin, Turkey
Site 801
🇭🇺Budapest, Hungary
Site 503
🇵🇱Warszawa, Mazowieckie, Poland
Site 102
🇺🇸Las Vegas, Nevada, United States
Site 108
🇺🇸Morristown, New Jersey, United States
Site 501
🇵🇱Olsztyn, Warmińsko-Mazurskie, Poland
Site 907
🇷🇺Chelyabinsk, Russian Federation
Site 905
🇷🇺Saint-Petersburg, Russian Federation
Site 704
🇹🇷Ankara, Turkey
Site 904
🇷🇺Moscow, Russian Federation
Site 703
🇹🇷Istanbul, Turkey
Site 202
🇫🇷Paris, Île-de-France, France
Site 302
🇩🇪Hamburg, Germany
Site 504
🇵🇱Łódź, Łódzkie, Poland
Site 906
🇷🇺Moscow, Russian Federation
Site 505
🇵🇱Zabrze, Śląskie, Poland
Site 902
🇷🇺Nizhny Novgorod, Russian Federation
Site 702
🇹🇷Antalya, Turkey
Site 901
🇷🇺Moscow, Russian Federation
Site 903
🇷🇺Volgograd, Russian Federation
Site 701
🇹🇷Adana, Turkey
Site 706
🇹🇷Antalya, Turkey
Site 705
🇹🇷Denizli, Turkey
Site 709
🇹🇷Istanbul, Turkey
Site 403
🇬🇧London, England, United Kingdom
Site 410
🇬🇧London, England, United Kingdom
Site 103
🇺🇸Long Beach, California, United States
Site 109
🇺🇸Sacramento, California, United States
Site 119
🇺🇸Orange, California, United States
Site 101
🇺🇸San Francisco, California, United States
Site 107
🇺🇸Peoria, Illinois, United States
Site 113
🇺🇸Durham, North Carolina, United States
Site 201
🇫🇷Toulouse, Occitanie, France
Site 304
🇩🇪Berlin, Germany
Site 301
🇩🇪Freiburg, Baden-Württemberg, Germany
Site 408
🇬🇧Liverpool, England, United Kingdom