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

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

Sponsors

Lead Sponsor: Dova Pharmaceuticals

Source Dova Pharmaceuticals
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. After the follow up visit, all subjects will continue to a long term safety follow-up period

Overall Status Not yet recruiting
Start Date December 31, 2020
Completion Date May 15, 2025
Primary Completion Date May 22, 2023
Phase Phase 3
Study Type Interventional
Primary Outcome
Measure Time Frame
Durable platelet response Last 8 weeks of 12 week treatment regimen
Secondary Outcome
Measure Time Frame
Platelet Counts 12 weeks of treatment
Platelet response Day 8
Platelet percentage 12 weeks
Rescue medications 12 weeks
Bleeding Symptoms Throughout study
Enrollment 72
Condition
Intervention

Intervention Type: Drug

Intervention Name: Avatrombopag

Description: Oral avatrombopag Tablet

Arm Group Label: Experimental: Avatrombopag

Other Name: Doptelet

Intervention Type: Drug

Intervention Name: Placebo

Description: Placebo comparator tablet

Arm Group Label: Placebo Comparator:Placebo

Eligibility

Criteria:

Inclusion Criteria: - Male or female participants ≥1 and <18 years of age at Screening 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 <30×109/L with no single count >35×109/L in the screening period Exclusion Criteria: - Participants must not have a know 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.

Gender: All

Minimum Age: 1 Year

Maximum Age: 18 Years

Healthy Volunteers: No

Overall Contact

Last Name: Dova CClincal

Phone: 919-765-6671

Email: [email protected]

Verification Date

October 2020

Responsible Party

Type: Sponsor

Keywords
Has Expanded Access No
Condition Browse
Number Of Arms 2
Arm Group

Label: Experimental: Avatrombopag

Type: Active Comparator

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

Label: Placebo Comparator:Placebo

Type: Placebo Comparator

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

Patient Data No
Study Design Info

Allocation: Randomized

Intervention Model: Parallel Assignment

Primary Purpose: Supportive Care

Masking: Double (Participant, Investigator)

Source: ClinicalTrials.gov