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

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ClinicalTrials.gov Identifier: NCT04516967
Recruitment Status : Recruiting
First Posted : August 18, 2020
Last Update Posted : October 27, 2021
Sponsor:
Information provided by (Responsible Party):
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

Condition or disease Intervention/treatment Phase
Immune Thrombocytopenia Drug: Avatrombopag Drug: Placebo Phase 3

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 72 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Supportive Care
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
Actual Study Start Date : March 2, 2021
Estimated Primary Completion Date : May 22, 2023
Estimated Study Completion Date : May 15, 2025


Arm Intervention/treatment
Active Comparator: Experimental: Avatrombopag
Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks
Drug: Avatrombopag
Oral avatrombopag Tablet
Other Name: Doptelet

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




Primary Outcome Measures :
  1. Durable platelet response [ Time Frame: Last 8 weeks of 12 week treatment regimen ]
    The proportion of subjects achieving at least 6 out of 8 weekly platelet counts ≥50×109/L during the last 8 weeks of the 12 week Treatment Period in the Core Phase, in the absence of rescue medication


Secondary Outcome Measures :
  1. Platelet Counts [ Time Frame: 12 weeks of treatment ]
    The percentage of weeks subjects have a platelet count ≥50×109/L during 12 weeks of treatment in the Core Phase, in the absence of rescue therapy.

  2. Platelet response [ Time Frame: Day 8 ]
    The proportion of subjects with a platelet count ≥50×109/L at day 8, in the absence of rescue therapy.

  3. Platelet percentage [ Time Frame: 12 weeks ]
    The percentage of weeks subjects have a platelet count between ≥50×109/L and ≤150×109/L, during 12 weeks of treatment in the Core Phase, in the absence of rescue therapy.

  4. Rescue medications [ Time Frame: 12 weeks ]
    The proportion of subjects who require rescue medications during 12 weeks of treatment in the Core Phase of the study.

  5. Bleeding Symptoms [ Time Frame: Throughout study ]
    Incidence and severity of bleeding symptoms associated with Immune thrombocytopenia ( ITP measured using the World Health Organization (WHO) Bleeding Scale.



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Ages Eligible for Study:   1 Year to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04516967


Contacts
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Contact: Dova Clincal 919-765-6671 clinical@dova.com

Locations
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Sponsors and Collaborators
Dova Pharmaceuticals
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Responsible Party: Dova Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04516967    
Other Study ID Numbers: AVA-PED-301
First Posted: August 18, 2020    Key Record Dates
Last Update Posted: October 27, 2021
Last Verified: August 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Dova Pharmaceuticals:
ITP
Additional relevant MeSH terms:
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Thrombocytopenia
Purpura, Thrombocytopenic, Idiopathic
Blood Platelet Disorders
Hematologic Diseases
Purpura, Thrombocytopenic
Purpura
Blood Coagulation Disorders
Thrombotic Microangiopathies
Hemorrhagic Disorders
Autoimmune Diseases
Immune System Diseases
Hemorrhage
Pathologic Processes
Skin Manifestations