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

Primary Purpose

Immune Thrombocytopenia

Status
Recruiting
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Avatrombopag
Placebo
Sponsored by
Sobi, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Immune Thrombocytopenia focused on measuring ITP

Eligibility Criteria

1 Year - 17 Years (Child)All SexesDoes not accept 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.

Sites / Locations

  • Site 112Recruiting
  • Site 103
  • Site 119Recruiting
  • Site 109Recruiting
  • Site 101Recruiting
  • Site 111Recruiting
  • Site 120Recruiting
  • Site 117Recruiting
  • Site 116Recruiting
  • Site 107Recruiting
  • Site 104Recruiting
  • Site 105Recruiting
  • Site 114Recruiting
  • Site 102Recruiting
  • Site 108Recruiting
  • Site 118Recruiting
  • Site 113
  • Site 106Recruiting
  • Site 121Recruiting
  • Site 110Recruiting
  • Site 115Recruiting
  • Site 201Recruiting
  • Site 202Recruiting
  • Site 301Recruiting
  • Site 303Recruiting
  • Site 304Recruiting
  • Site 302Recruiting
  • Site 801Recruiting
  • Site 802Recruiting
  • Site 803Recruiting
  • Site 502Recruiting
  • Site 503Recruiting
  • Site 501
  • Site 504Recruiting
  • Site 505Recruiting
  • Site 907
  • Site 904
  • Site 906Recruiting
  • Site 901
  • Site 902Recruiting
  • Site 905
  • Site 903Recruiting
  • Site 701Recruiting
  • Site 704Recruiting
  • Site 706Recruiting
  • Site 702Recruiting
  • Site 705Recruiting
  • Site 709Recruiting
  • Site 710Recruiting
  • Site 703Recruiting
  • Site 707Recruiting
  • Site 708Recruiting
  • Site 954Recruiting
  • Site 952Recruiting
  • Site 405Recruiting
  • Site 408Recruiting
  • Site 403Recruiting
  • Site 410Recruiting
  • Site 407Recruiting
  • Site 402Recruiting
  • Site 401Recruiting
  • Site 406Recruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Placebo Comparator

Arm Label

Experimental: Avatrombopag

Placebo Comparator:Placebo

Arm Description

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

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

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

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.
Platelet response
The proportion of subjects with a platelet count ≥50×10^9/L at day 8, in the absence of rescue therapy.
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.
Rescue medications
The proportion of subjects who require rescue medications during 12 weeks of treatment in the Core Phase of the study.
Bleeding Symptoms
Incidence and severity of bleeding symptoms associated with Immune thrombocytopenia (ITP) measured using the World Health Organization (WHO) Bleeding Scale.

Full Information

First Posted
August 14, 2020
Last Updated
July 19, 2023
Sponsor
Sobi, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT04516967
Brief Title
Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia for ≥6 Months
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
Study Type
Interventional

2. Study Status

Record Verification Date
July 2023
Overall Recruitment Status
Recruiting
Study Start Date
March 2, 2021 (Actual)
Primary Completion Date
August 14, 2023 (Anticipated)
Study Completion Date
November 28, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Sobi, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Immune Thrombocytopenia
Keywords
ITP

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
72 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Experimental: Avatrombopag
Arm Type
Active Comparator
Arm Description
Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks
Arm Title
Placebo Comparator:Placebo
Arm Type
Placebo Comparator
Arm Description
Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks
Intervention Type
Drug
Intervention Name(s)
Avatrombopag
Other Intervention Name(s)
Doptelet
Intervention Description
Oral avatrombopag Tablet
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo comparator tablet
Primary Outcome Measure Information:
Title
Durable platelet response
Description
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
Secondary Outcome Measure Information:
Title
Platelet Counts
Description
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
Title
Platelet response
Description
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
Title
Platelet percentage
Description
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
Title
Rescue medications
Description
The proportion of subjects who require rescue medications during 12 weeks of treatment in the Core Phase of the study.
Time Frame
12 weeks
Title
Bleeding Symptoms
Description
Incidence and severity of bleeding symptoms associated with Immune thrombocytopenia (ITP) measured using the World Health Organization (WHO) Bleeding Scale.
Time Frame
Throughout study

10. Eligibility

Sex
All
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Sobi Inc
Phone
919-765-6671
Email
NAClincal@Sobi.com
Facility Information:
Facility Name
Site 112
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85016
Country
United States
Individual Site Status
Recruiting
Facility Name
Site 103
City
Long Beach
State/Province
California
ZIP/Postal Code
90806
Country
United States
Individual Site Status
Withdrawn
Facility Name
Site 119
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Individual Site Status
Recruiting
Facility Name
Site 109
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Individual Site Status
Recruiting
Facility Name
Site 101
City
San Francisco
State/Province
California
ZIP/Postal Code
94158
Country
United States
Individual Site Status
Recruiting
Facility Name
Site 111
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Individual Site Status
Recruiting
Facility Name
Site 120
City
Wilmington
State/Province
Delaware
ZIP/Postal Code
19803
Country
United States
Individual Site Status
Recruiting
Facility Name
Site 117
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
Country
United States
Individual Site Status
Recruiting
Facility Name
Site 116
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Individual Site Status
Recruiting
Facility Name
Site 107
City
Peoria
State/Province
Illinois
ZIP/Postal Code
61615
Country
United States
Individual Site Status
Recruiting
Facility Name
Site 104
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Individual Site Status
Recruiting
Facility Name
Site 105
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55404
Country
United States
Individual Site Status
Recruiting
Facility Name
Site 114
City
Jackson
State/Province
Mississippi
ZIP/Postal Code
39216
Country
United States
Individual Site Status
Recruiting
Facility Name
Site 102
City
Las Vegas
State/Province
Nevada
ZIP/Postal Code
89135
Country
United States
Individual Site Status
Recruiting
Facility Name
Site 108
City
Morristown
State/Province
New Jersey
ZIP/Postal Code
07960
Country
United States
Individual Site Status
Recruiting
Facility Name
Site 118
City
Charlotte
State/Province
North Carolina
ZIP/Postal Code
28203
Country
United States
Individual Site Status
Recruiting
Facility Name
Site 113
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Individual Site Status
Withdrawn
Facility Name
Site 106
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205
Country
United States
Individual Site Status
Recruiting
Facility Name
Site 121
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Individual Site Status
Recruiting
Facility Name
Site 110
City
Providence
State/Province
Rhode Island
ZIP/Postal Code
02903
Country
United States
Individual Site Status
Recruiting
Facility Name
Site 115
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Individual Site Status
Recruiting
Facility Name
Site 201
City
Toulouse
State/Province
Occitanie
ZIP/Postal Code
31059
Country
France
Individual Site Status
Recruiting
Facility Name
Site 202
City
Paris
State/Province
Île-de-France
ZIP/Postal Code
75019
Country
France
Individual Site Status
Recruiting
Facility Name
Site 301
City
Freiburg
State/Province
Baden-Württemberg
ZIP/Postal Code
79106
Country
Germany
Individual Site Status
Recruiting
Facility Name
Site 303
City
Kiel
State/Province
Schleswig-Holstein
ZIP/Postal Code
24105
Country
Germany
Individual Site Status
Recruiting
Facility Name
Site 304
City
Berlin
ZIP/Postal Code
13353
Country
Germany
Individual Site Status
Recruiting
Facility Name
Site 302
City
Hamburg
ZIP/Postal Code
20246
Country
Germany
Individual Site Status
Recruiting
Facility Name
Site 801
City
Budapest
ZIP/Postal Code
1094
Country
Hungary
Individual Site Status
Recruiting
Facility Name
Site 802
City
Miskolc
ZIP/Postal Code
3526
Country
Hungary
Individual Site Status
Recruiting
Facility Name
Site 803
City
Pécs
ZIP/Postal Code
7623
Country
Hungary
Individual Site Status
Recruiting
Facility Name
Site 502
City
Bydgoszcz
State/Province
Kujawsko-Pomorskie
ZIP/Postal Code
85-667
Country
Poland
Individual Site Status
Recruiting
Facility Name
Site 503
City
Warszawa
State/Province
Mazowieckie
ZIP/Postal Code
02-091
Country
Poland
Individual Site Status
Recruiting
Facility Name
Site 501
City
Olsztyn
State/Province
Warmińsko-Mazurskie
ZIP/Postal Code
10-561
Country
Poland
Individual Site Status
Withdrawn
Facility Name
Site 504
City
Łódź
State/Province
Łódzkie
ZIP/Postal Code
91-738
Country
Poland
Individual Site Status
Recruiting
Facility Name
Site 505
City
Zabrze
State/Province
Śląskie
ZIP/Postal Code
41-800
Country
Poland
Individual Site Status
Recruiting
Facility Name
Site 907
City
Chelyabinsk
ZIP/Postal Code
454076
Country
Russian Federation
Individual Site Status
Withdrawn
Facility Name
Site 904
City
Moscow
ZIP/Postal Code
117198
Country
Russian Federation
Individual Site Status
Withdrawn
Facility Name
Site 906
City
Moscow
ZIP/Postal Code
117997
Country
Russian Federation
Individual Site Status
Recruiting
Facility Name
Site 901
City
Moscow
ZIP/Postal Code
119049
Country
Russian Federation
Individual Site Status
Withdrawn
Facility Name
Site 902
City
Nizhny Novgorod
ZIP/Postal Code
603136
Country
Russian Federation
Individual Site Status
Recruiting
Facility Name
Site 905
City
Saint-Petersburg
ZIP/Postal Code
197022
Country
Russian Federation
Individual Site Status
Withdrawn
Facility Name
Site 903
City
Volgograd
ZIP/Postal Code
400138
Country
Russian Federation
Individual Site Status
Recruiting
Facility Name
Site 701
City
Adana
ZIP/Postal Code
01330
Country
Turkey
Individual Site Status
Recruiting
Facility Name
Site 704
City
Ankara
ZIP/Postal Code
06230
Country
Turkey
Individual Site Status
Recruiting
Facility Name
Site 706
City
Antalya
ZIP/Postal Code
07059
Country
Turkey
Individual Site Status
Recruiting
Facility Name
Site 702
City
Antalya
ZIP/Postal Code
07160
Country
Turkey
Individual Site Status
Recruiting
Facility Name
Site 705
City
Denizli
ZIP/Postal Code
20160
Country
Turkey
Individual Site Status
Recruiting
Facility Name
Site 709
City
Istanbul
ZIP/Postal Code
34093
Country
Turkey
Individual Site Status
Recruiting
Facility Name
Site 710
City
Istanbul
ZIP/Postal Code
34093
Country
Turkey
Individual Site Status
Recruiting
Facility Name
Site 703
City
Istanbul
ZIP/Postal Code
34764
Country
Turkey
Individual Site Status
Recruiting
Facility Name
Site 707
City
Izmir
ZIP/Postal Code
35100
Country
Turkey
Individual Site Status
Recruiting
Facility Name
Site 708
City
Mersin
ZIP/Postal Code
33110
Country
Turkey
Individual Site Status
Recruiting
Facility Name
Site 954
City
Kharkiv
State/Province
Kharkiv Region
ZIP/Postal Code
61000
Country
Ukraine
Individual Site Status
Recruiting
Facility Name
Site 952
City
Vinnytsia
ZIP/Postal Code
21029
Country
Ukraine
Individual Site Status
Recruiting
Facility Name
Site 405
City
Birmingham
State/Province
England
ZIP/Postal Code
B4 6NH
Country
United Kingdom
Individual Site Status
Recruiting
Facility Name
Site 408
City
Liverpool
State/Province
England
ZIP/Postal Code
L12 2AP
Country
United Kingdom
Individual Site Status
Recruiting
Facility Name
Site 403
City
London
State/Province
England
ZIP/Postal Code
SE5 9RS
Country
United Kingdom
Individual Site Status
Recruiting
Facility Name
Site 410
City
London
State/Province
England
ZIP/Postal Code
W12 0HS
Country
United Kingdom
Individual Site Status
Recruiting
Facility Name
Site 407
City
London
State/Province
England
ZIP/Postal Code
WC1N 3JH
Country
United Kingdom
Individual Site Status
Recruiting
Facility Name
Site 402
City
Manchester
State/Province
England
ZIP/Postal Code
M13 9WL
Country
United Kingdom
Individual Site Status
Recruiting
Facility Name
Site 401
City
Sheffield
State/Province
England
ZIP/Postal Code
S10 2TH
Country
United Kingdom
Individual Site Status
Recruiting
Facility Name
Site 406
City
Cardiff
State/Province
Wales
ZIP/Postal Code
CF14 4XW
Country
United Kingdom
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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