Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia for ≥6 Months

Immune Thrombocytopenia Clinical Trial

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04516967
• Other study ID #: AVA-PED-301
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: March 5, 2021
• Est. completion date: November 28, 2025

Study information

• Verified date: January 2024
• Source: Sobi, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

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 information / eligibility

• Status: Active, not recruiting
• Enrollment: 75
• Est. completion date: November 28, 2025
• Est. primary completion date: November 8, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year to 17 Years

Eligibility

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.

Related Conditions & MeSH terms

• Immune Thrombocytopenia
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenia

Intervention

Drug:
• Avatrombopag
Oral avatrombopag Tablet
• Placebo
Placebo comparator tablet

Locations

Country	Name	City	State
France	Site 202	Paris	Île-de-France
France	Site 201	Toulouse	Occitanie
Germany	Site 304	Berlin
Germany	Site 301	Freiburg	Baden-Württemberg
Germany	Site 302	Hamburg
Germany	Site 303	Kiel	Schleswig-Holstein
Hungary	Site 801	Budapest
Hungary	Site 802	Miskolc
Hungary	Site 803	Pécs
Poland	Site 502	Bydgoszcz	Kujawsko-Pomorskie
Poland	Site 504	Lódz	Lódzkie
Poland	Site 501	Olsztyn	Warminsko-Mazurskie
Poland	Site 503	Warszawa	Mazowieckie
Poland	Site 505	Zabrze	Slaskie
Russian Federation	Site 907	Chelyabinsk
Russian Federation	Site 901	Moscow
Russian Federation	Site 904	Moscow
Russian Federation	Site 906	Moscow
Russian Federation	Site 902	Nizhny Novgorod
Russian Federation	Site 905	Saint-Petersburg
Russian Federation	Site 903	Volgograd
Turkey	Site 701	Adana
Turkey	Site 704	Ankara
Turkey	Site 702	Antalya
Turkey	Site 706	Antalya
Turkey	Site 705	Denizli
Turkey	Site 703	Istanbul
Turkey	Site 709	Istanbul
Turkey	Site 710	Istanbul
Turkey	Site 707	Izmir
Turkey	Site 708	Mersin
Ukraine	Site 954	Kharkiv	Kharkiv Region
Ukraine	Site 952	Vinnytsia
United Kingdom	Site 405	Birmingham	England
United Kingdom	Site 406	Cardiff	Wales
United Kingdom	Site 408	Liverpool	England
United Kingdom	Site 403	London	England
United Kingdom	Site 407	London	England
United Kingdom	Site 410	London	England
United Kingdom	Site 402	Manchester	England
United Kingdom	Site 401	Sheffield	England
United States	Site 116	Atlanta	Georgia
United States	Site 111	Aurora	Colorado
United States	Site 104	Boston	Massachusetts
United States	Site 118	Charlotte	North Carolina
United States	Site 106	Columbus	Ohio
United States	Site 113	Durham	North Carolina
United States	Site 117	Gainesville	Florida
United States	Site 115	Houston	Texas
United States	Site 114	Jackson	Mississippi
United States	Site 102	Las Vegas	Nevada
United States	Site 103	Long Beach	California
United States	Site 105	Minneapolis	Minnesota
United States	Site 108	Morristown	New Jersey
United States	Site 119	Orange	California
United States	Site 107	Peoria	Illinois
United States	Site 121	Philadelphia	Pennsylvania
United States	Site 112	Phoenix	Arizona
United States	Site 110	Providence	Rhode Island
United States	Site 109	Sacramento	California
United States	Site 101	San Francisco	California
United States	Site 120	Wilmington	Delaware

Sponsors (1)

Lead Sponsor	Collaborator
Sobi, Inc.

Countries where clinical trial is conducted

United States,  France,  Germany,  Hungary,  Poland,  Russian Federation,  Turkey,  Ukraine,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Secondary	Platelet Counts	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.	12 weeks of treatment
Secondary	Platelet response	The proportion of subjects with a platelet count =50×10^9/L at day 8, in the absence of rescue therapy.	Day 8
Secondary	Platelet percentage	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.	12 weeks
Secondary	Rescue medications	The proportion of subjects who require rescue medications during 12 weeks of treatment in the Core Phase of the study.	12 weeks
Secondary	Bleeding Symptoms	Incidence and severity of bleeding symptoms associated with Immune thrombocytopenia (ITP) measured using the World Health Organization (WHO) Bleeding Scale.	Throughout study