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

Sobi

Status and phase: Active, not recruiting; Phase 3

Conditions: Immune Thrombocytopenia

Treatments: Drug: Placebo; Drug: Avatrombopag

Study type: Interventional

Funder types: Industry

Identifiers

NCT04516967

AVA-PED-301

Details and patient eligibility

About

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

Full 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.

Enrollment: 75 patients

Sex: All

Ages: 1 to 17 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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75 participants in 3 patient groups, including a placebo group

Experimental: Avatrombopag Double Blind

Experimental group

Description:

Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks

Treatment:

Drug: Avatrombopag

Placebo Comparator:Placebo Double Blind

Placebo Comparator group

Description:

Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks

Treatment:

Drug: Placebo

Avatrombopag Open Label Extension

Experimental group

Description:

Investigational product administered orally for up to 2 years.

Treatment:

Drug: Avatrombopag