Clinical trial NCT04516967: Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia for ≥6 Months
Official title: 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
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Immune Thrombocytopenia
Keywords: ITP
Lead sponsor: Sobi, Inc.
Sponsor class: INDUSTRY
First posted: 2020-08-18
Enrollment: 75
Sex: ALL
Minimum age (years): 1
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Durable Platelet Response
timeFrame: Last 8 weeks of 12 week treatment regimen
measure: Alternative Primary: Platelet Response
timeFrame: 12 weeks of treatment
Secondary outcomes:
measure: Percentage of Weeks Platelet Count ≥50×10^9/L
timeFrame: 12 weeks of treatment
measure: Platelet Response at Day 8
timeFrame: Day 8
measure: Percentage of Weeks Platelet Count Between ≥50×10^9/L and ≤150×10^9/L
timeFrame: 12 weeks of treatment
measure: Rescue Medications
timeFrame: 12 weeks of treatment
measure: Incidence of Any Bleeding Event (WHO Grade 1-4)
timeFrame: 12 weeks of treatment
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Site 112
state: Arizona
city: Long Beach
country: United States
facility: Site 103
state: California
city: Orange
country: United States
facility: Site 119
state: California
city: Sacramento
country: United States
facility: Site 109
state: California
city: San Francisco
country: United States
facility: Site 101
state: California
city: Aurora
country: United States
facility: Site 111
state: Colorado
city: Wilmington
country: United States
facility: Site 120
state: Delaware
city: Gainesville
country: United States
facility: Site 117
state: Florida
city: Atlanta
country: United States
facility: Site 116
state: Georgia
city: Peoria
country: United States
facility: Site 107
state: Illinois
city: Boston
country: United States
facility: Site 104
state: Massachusetts
city: Minneapolis
country: United States
facility: Site 105
state: Minnesota
city: Jackson
country: United States
facility: Site 114
state: Mississippi
city: Las Vegas
country: United States
facility: Site 102
state: Nevada
city: Morristown
country: United States
facility: Site 108
state: New Jersey
city: Charlotte
country: United States
facility: Site 118
state: North Carolina
city: Durham
country: United States
facility: Site 113
state: North Carolina
city: Columbus
country: United States
facility: Site 106
state: Ohio
city: Philadelphia
country: United States
facility: Site 121
state: Pennsylvania
city: Providence
country: United States
facility: Site 110
state: Rhode Island
city: Houston
country: United States
facility: Site 115
state: Texas
city: Toulouse
country: France
facility: Site 201
state: Occitanie
city: Paris
country: France
facility: Site 202
state: Île-de-France
city: Freiburg
country: Germany
facility: Site 301
state: Baden-Württemberg
city: Kiel
country: Germany
facility: Site 303
state: Schleswig-Holstein
city: Berlin
country: Germany
facility: Site 304
city: Hamburg
country: Germany
facility: Site 302
city: Budapest
country: Hungary
facility: Site 801
city: Miskolc
country: Hungary
facility: Site 802
city: Pécs
country: Hungary
facility: Site 803
city: Bydgoszcz
country: Poland
facility: Site 502
state: Kujawsko-Pomorskie
city: Warszawa
country: Poland
facility: Site 503
state: Mazowieckie
city: Olsztyn
country: Poland
facility: Site 501
state: Warmińsko-Mazurskie
city: Łódź
country: Poland
facility: Site 504
state: Łódzkie
city: Zabrze
country: Poland
facility: Site 505
state: Śląskie
city: Chelyabinsk
country: Russian Federation
facility: Site 907
city: Moscow
country: Russian Federation
facility: Site 904
city: Moscow
country: Russian Federation
facility: Site 906
city: Moscow
country: Russian Federation
facility: Site 901
city: Nizhny Novgorod
country: Russian Federation
facility: Site 902
city: Saint-Petersburg
country: Russian Federation
facility: Site 905
city: Volgograd
country: Russian Federation
facility: Site 903
city: Adana
country: Turkey
facility: Site 701
city: Ankara
country: Turkey
facility: Site 704
city: Antalya
country: Turkey
facility: Site 706
city: Antalya
country: Turkey
facility: Site 702
city: Denizli
country: Turkey
facility: Site 705
city: Istanbul
country: Turkey
facility: Site 709
city: Istanbul
country: Turkey
facility: Site 710
city: Istanbul
country: Turkey
facility: Site 703
city: Izmir
country: Turkey
facility: Site 707
city: Mersin
country: Turkey
facility: Site 708
city: Kharkiv
country: Ukraine
facility: Site 954
state: Kharkiv Region
city: Vinnytsia
country: Ukraine
facility: Site 952
city: Birmingham
country: United Kingdom
facility: Site 405
state: England
city: Liverpool
country: United Kingdom
facility: Site 408
state: England
city: London
country: United Kingdom
facility: Site 403
state: England
city: London
country: United Kingdom
facility: Site 410
state: England
city: London
country: United Kingdom
facility: Site 407
state: England
city: Manchester
country: United Kingdom
facility: Site 402
state: England
city: Sheffield
country: United Kingdom
facility: Site 401
state: England
city: Cardiff
country: United Kingdom
facility: Site 406
state: Wales
Study officials:
References: