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

Sobi, Inc.75 enrolled

Overview

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

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 Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

Conditions

Immune Thrombocytopenia

Interventions

AvatrombopagDRUG

Oral avatrombopag tablet or sprinkle capsule

PlaceboDRUG

Placebo comparator tablet or sprinkle capsule

Eligibility

Sex: ALLMin age: 1 YearMax age: 17 Years

Medical Language ↔ Plain English

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.

Locations (62)

Outcomes

Primary Outcomes

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

Time frame: 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.

Time frame: 12 weeks of treatment

Secondary Outcomes

Percentage of Weeks Platelet Count ≥50×10^9/L

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.

Time frame: 12 weeks of treatment

Platelet Response at Day 8

The proportion of subjects with a platelet count ≥50×10\^9/L at day 8, in the absence of rescue therapy.

Time frame: Day 8

Percentage of Weeks Platelet Count Between ≥50×10^9/L and ≤150×10^9/L

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.

Time frame: 12 weeks of treatment

Rescue Medications

The proportion of subjects who require rescue medications during 12 weeks of treatment in the Core Phase of the study.

Time frame: 12 weeks of treatment

Incidence of Any Bleeding Event (WHO Grade 1-4)

Data from ClinicalTrials.gov

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