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

Sponsor

Sobi, Inc. (Industry)

Overall Status

Recruiting

CT.gov ID

NCT04516967

Collaborator

(none)

Enrollment

Locations

Arms

56.9

Anticipated Duration (Months)

1.1

Patients Per Site

Patients Per Site Per Month

Study Details

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

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.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

72 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Double (Participant, Investigator)

Primary Purpose:

Treatment

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 :

Mar 2, 2021

Anticipated Primary Completion Date :

Jun 2, 2023

Anticipated Study Completion Date :

Nov 28, 2025

Arms and Interventions

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 Names: 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

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 Names:

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

Outcome Measures

Primary Outcome Measures

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

Eligibility Criteria

Criteria

Ages Eligible for Study:

1 Year to 17 Years

Sexes Eligible for Study:

All

Accepts 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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Site 112	Phoenix	Arizona	United States	85016
2	Site 103	Long Beach	California	United States	90806
3	Site 119	Orange	California	United States	92868
4	Site 109	Sacramento	California	United States	95817
5	Site 101	San Francisco	California	United States	94158
6	Site 111	Aurora	Colorado	United States	80045
7	Site 120	Wilmington	Delaware	United States	19803
8	Site 117	Gainesville	Florida	United States	32610
9	Site 116	Atlanta	Georgia	United States	30322
10	Site 107	Peoria	Illinois	United States	61615
11	Site 104	Boston	Massachusetts	United States	02115
12	Site 105	Minneapolis	Minnesota	United States	55404
13	Site 114	Jackson	Mississippi	United States	39216
14	Site 102	Las Vegas	Nevada	United States	89135
15	Site 108	Morristown	New Jersey	United States	07960
16	Site 118	Charlotte	North Carolina	United States	28203
17	Site 113	Durham	North Carolina	United States	27710
18	Site 106	Columbus	Ohio	United States	43205
19	Site 121	Philadelphia	Pennsylvania	United States	19104
20	Site 110	Providence	Rhode Island	United States	02903
21	Site 115	Houston	Texas	United States	77030
22	Site 201	Toulouse	Occitanie	France	31059
23	Site 202	Paris	Île-de-France	France	75019
24	Site 301	Freiburg	Baden-Württemberg	Germany	79106
25	Site 303	Kiel	Schleswig-Holstein	Germany	24105
26	Site 304	Berlin		Germany	13353
27	Site 302	Hamburg		Germany	20246
28	Site 801	Budapest		Hungary	1094
29	Site 802	Miskolc		Hungary	3526
30	Site 803	Pécs		Hungary	7623
31	Site 502	Bydgoszcz	Kujawsko-Pomorskie	Poland	85-667
32	Site 503	Warszawa	Mazowieckie	Poland	02-091
33	Site 501	Olsztyn	Warmińsko-Mazurskie	Poland	10-561
34	Site 504	Łódź	Łódzkie	Poland	91-738
35	Site 505	Zabrze	Śląskie	Poland	41-800
36	Site 907	Chelyabinsk		Russian Federation	454076
37	Site 904	Moscow		Russian Federation	117198
38	Site 906	Moscow		Russian Federation	117997
39	Site 901	Moscow		Russian Federation	119049
40	Site 902	Nizhny Novgorod		Russian Federation	603136
41	Site 905	Saint-Petersburg		Russian Federation	197022
42	Site 903	Volgograd		Russian Federation	400138
43	Site 701	Adana		Turkey	01330
44	Site 704	Ankara		Turkey	06230
45	Site 706	Antalya		Turkey	07059
46	Site 702	Antalya		Turkey	07160
47	Site 705	Denizli		Turkey	20160
48	Site 709	Istanbul		Turkey	34093
49	Site 710	Istanbul		Turkey	34093
50	Site 703	Istanbul		Turkey	34764
51	Site 707	Izmir		Turkey	35100
52	Site 708	Mersin		Turkey	33110
53	Site 954	Kharkiv	Kharkiv Region	Ukraine	61051
54	Site 954	Kharkiv	Kharkiv Region	Ukraine	61075
55	Site 952	Vinnytsia	Vinnytsia Oblast	Ukraine	21000
56	Site 952	Vinnytsia		Ukraine	21029
57	Site 405	Birmingham	England	United Kingdom	B4 6NH
58	Site 408	Liverpool	England	United Kingdom	L12 2AP
59	Site 403	London	England	United Kingdom	SE5 9RS
60	Site 410	London	England	United Kingdom	W12 0HS
61	Site 407	London	England	United Kingdom	WC1N 3JH
62	Site 402	Manchester	England	United Kingdom	M13 9WL
63	Site 401	Sheffield	England	United Kingdom	S10 2TH
64	Site 406	Cardiff	Wales	United Kingdom	CF14 4XW

Sponsors and Collaborators

Sobi, Inc.

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Sobi, Inc.

ClinicalTrials.gov Identifier:

NCT04516967

Other Study ID Numbers:

AVA-PED-301

First Posted:

Aug 18, 2020

Last Update Posted:

Jul 15, 2022

Last Verified:

Jul 1, 2022

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Yes

Studies a U.S. FDA-regulated Device Product:

Keywords provided by Sobi, Inc.

ITP

Additional relevant MeSH terms:

Thrombocytopenia

Purpura, Thrombocytopenic, Idiopathic

Study Results

No Results Posted as of Jul 15, 2022