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Clinical Trial Results:
A Phase 3b, Multi-center, Randomized, Double-blind, Placebo-controlled, Parallel-group Trial with an 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

Summary
EudraCT number	2020-003232-24
Trial protocol	FR HU DE PL
Global end of trial date

Results information
Results version number	v1(current)
This version publication date	22 May 2024
First version publication date	22 May 2024
Other versions

Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information

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Trial information

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Sponsor protocol code

AVA-PED-301

ISRCTN number

-

US NCT number

NCT04516967

WHO universal trial number (UTN)

-

Sponsor organisation name

Sobi Inc.

Sponsor organisation address

890 Winter Street, Suite 200, Waltham, United States, 02451

Public contact

Medical Information, Sobi, Inc., +1 781 786 7370, medinfo.us@sobi.com

Scientific contact

Medical Information, Sobi, Inc., +1 781 786 7370, medinfo.us@sobi.com

Is trial part of an agreed paediatric investigation plan (PIP)

Yes

EMA paediatric investigation plan number(s)

EMEA-001136-PIP01-11

Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?

No

Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?

Yes

Analysis stage

Interim

Date of interim/final analysis

18 Dec 2023

Is this the analysis of the primary completion data?

Yes

Primary completion date

08 Nov 2023

Global end of trial reached?

No

Main objective of the trial

To demonstrate that the efficacy of avatrombopag is superior to placebo for the treatment of pediatric subjects with ITP of ≥6 months duration who have had an insufficient response to a previous treatment

Protection of trial subjects

The study was in compliance with the ethical principles derived from the Declaration of Helsinki and the International Conference on Harmonization (ICH) and Good Clinical Practice (GCP) guidelines. All the local regulatory requirements pertinent to safety of trial subjects were also followed during the conduct of the trial.

Background therapy

-

Evidence for comparator

-

Actual start date of recruitment

02 Mar 2021

Long term follow-up planned

No

Independent data monitoring committee (IDMC) involvement?

Yes

Number of subjects enrolled per country

Country: Number of subjects enrolled

Poland: 4

Country: Number of subjects enrolled

United Kingdom: 13

Country: Number of subjects enrolled

France: 1

Country: Number of subjects enrolled

Germany: 3

Country: Number of subjects enrolled

Hungary: 5

Country: Number of subjects enrolled

United States: 13

Country: Number of subjects enrolled

Russian Federation: 5

Country: Number of subjects enrolled

Ukraine: 2

Country: Number of subjects enrolled

Türkiye: 29

Worldwide total number of subjects

75

EEA total number of subjects

13

Number of subjects enrolled per age group

In utero

0

Preterm newborn - gestational age < 37 wk

0

Newborns (0-27 days)

0

Infants and toddlers (28 days-23 months)

1

Children (2-11 years)

45

Adolescents (12-17 years)

29

Adults (18-64 years)

0

From 65 to 84 years

0

85 years and over

0

Subject disposition

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Recruitment details

-

Screening details

To be eligible for the study, the subject must have had a confirmed diagnosis of primary ITP for ≥6 months duration and had an insufficient response to a previous treatment with an average of 2 platelet counts <30×10^9/L with no single count >35×10^9/L.

Period 1 title

Core Phase (overall period)

Is this the baseline period?

Yes

Allocation method

Randomised - controlled

Blinding used

Double blind

Roles blinded

Subject, Investigator, Monitor, Data analyst, Carer, Assessor

Are arms mutually exclusive

Yes

Avatrombopag

Arm description

-

Arm type

Experimental

Investigational medicinal product name

Avatrombopag

Investigational medicinal product code

Other name

Pharmaceutical forms

Powder for oral suspension, Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects received avatrombopag or matching placebo as either the film-coated oral tablet or the powder for oral suspension. The powder for oral suspension was contained in a capsule that was to be opened to sprinkle the contents into an appropriate vehicle to prepare the suspension. No partial dosing from the capsule was allowed; the entire contents were to be used to prepare the suspension. On Day 1, Cohort 1 (≥12 to <18 years old) and Cohort 2 (≥6 to <12 years old) had a starting dose of avatrombopag of 20 mg once daily, administered as an oral tablet, consistent with the approved adult dosing. The starting dose for Cohort 3 (≥1 to <6 years old) was 10 mg once daily administered as an oral suspension. The dose of study drug was to be titrated up or down based on the subject’s platelet count to maintain a platelet count between ≥50 and ≤150×10^9/L.

Placebo

Arm description

-

Arm type

Placebo

Investigational medicinal product name

Placebo to match avatrombopag

Investigational medicinal product code

Other name

Pharmaceutical forms

Powder for oral suspension, Tablet

Routes of administration

Oral use

Dosage and administration details

Subjects received avatrombopag or matching placebo as either the film-coated oral tablet or the powder for oral suspension. The powder for oral suspension was contained in a capsule that was to be opened to sprinkle the contents into an appropriate vehicle to prepare the suspension. No partial dosing from the capsule was allowed; the entire contents were to be used to prepare the suspension. On Day 1, Cohort 1 (≥12 to <18 years old) and Cohort 2 (≥6 to <12 years old) had a starting dose of avatrombopag of 20 mg once daily, administered as an oral tablet, consistent with the approved adult dosing. The starting dose for Cohort 3 (≥1 to <6 years old) was 10 mg once daily administered as an oral suspension. The dose of study drug was to be titrated up or down based on the subject’s platelet count to maintain a platelet count between ≥50 and ≤150×10^9/L.

Number of subjects in period 1	Avatrombopag	Placebo
Started	54	21
Completed	44	1
Not completed	10	20
Physician decision	1	1
Adverse event, non-fatal	2	1
Lack of efficacy	7	18

Baseline characteristics

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Reporting group title

Avatrombopag

Reporting group description

-

Reporting group title

Placebo

Reporting group description

-

Reporting group values	Avatrombopag	Placebo	Total
Number of subjects	54	21	75
Age categorical
Units: Subjects
Infants and toddlers (28 days-23 months)	1	0	1
Children (2-11 years)	32	13	45
Adolescents (12-17 years)	21	8	29
Age continuous
Units: years
arithmetic mean (standard deviation)	8.9 ( 4.36 )	9.9 ( 4.13 )	-
Gender categorical
Units: Subjects
Female	24	12	36
Male	30	9	39

Subject analysis set title

Full Analysis Set

Subject analysis set type

Full analysis

Subject analysis set description

All randomized subjects.

Subject analysis sets values	Full Analysis Set
Number of subjects	75
Age categorical
Units: Subjects
Infants and toddlers (28 days-23 months)	1
Children (2-11 years)	45
Adolescents (12-17 years)	29
Age continuous
Units: years
arithmetic mean (standard deviation)	( )
Gender categorical
Units: Subjects
Female
Male

End points

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Reporting group title

Avatrombopag

Reporting group description

-

Reporting group title

Placebo

Reporting group description

-

Subject analysis set title

Full Analysis Set

Subject analysis set type

Full analysis

Subject analysis set description

All randomized subjects.

Primary: Durable Platelet Response

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End point title

Durable Platelet Response

End point description

The primary efficacy endpoint was durable platelet response as defined by 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.

End point type

Primary

End point timeframe

12 Weeks

End point values	Avatrombopag	Placebo
Number of subjects analysed	54	21
Units: Percentage
Yes	15	0
No	39	21

Durable Platelet Response

Comparison groups

Avatrombopag v Placebo

Number of subjects included in analysis

75

Analysis specification

Pre-specified

Analysis type

equivalence

P-value

= 0.0077

Method

Fisher exact

Confidence interval

Primary: Platelet Response

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End point title

Platelet Response

End point description

Platelet response was defined as having at least 2 consecutive platelet assessments ≥50×10^9/L over the 12 weeks of treatment in the Core Phase in the absence of rescue medication.

End point type

Primary

End point timeframe

12 Weeks

End point values	Avatrombopag	Placebo
Number of subjects analysed	54	21
Units: Percentage
Yes	44	0
No	10	21

Platelet Response - FAS

Comparison groups

Avatrombopag v Placebo

Number of subjects included in analysis

75

Analysis specification

Pre-specified

Analysis type

equivalence

P-value

= 0.0067

Method

Fisher exact

Confidence interval

Adverse events

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Timeframe for reporting adverse events

12 weeks

Assessment type

Systematic

Dictionary name

MedDRA

Dictionary version

26.1

Reporting group title

Avatrombopag

Reporting group description

-

Reporting group title

Placebo

Reporting group description

-

Serious adverse events	Avatrombopag	Placebo
Total subjects affected by serious adverse events
subjects affected / exposed	5 / 54 (9.26%)	1 / 21 (4.76%)
number of deaths (all causes)	0	0
number of deaths resulting from adverse events	0	0
Nervous system disorders
Headache
subjects affected / exposed	1 / 54 (1.85%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	1 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Blood and lymphatic system disorders
Immune thrombocytopenia
subjects affected / exposed	1 / 54 (1.85%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 2	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Thrombocytosis
subjects affected / exposed	1 / 54 (1.85%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	1 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Gastrointestinal disorders
Mouth haemorrhage
subjects affected / exposed	1 / 54 (1.85%)	1 / 21 (4.76%)
occurrences causally related to treatment / all	0 / 1	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0
Oral purpura
subjects affected / exposed	1 / 54 (1.85%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Rectal haemorrhage
subjects affected / exposed	1 / 54 (1.85%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Respiratory, thoracic and mediastinal disorders
Epistaxis
subjects affected / exposed	1 / 54 (1.85%)	0 / 21 (0.00%)
occurrences causally related to treatment / all	0 / 1	0 / 0
deaths causally related to treatment / all	0 / 0	0 / 0
Skin and subcutaneous tissue disorders
Skin haemorrhage
subjects affected / exposed	0 / 54 (0.00%)	1 / 21 (4.76%)
occurrences causally related to treatment / all	0 / 0	0 / 1
deaths causally related to treatment / all	0 / 0	0 / 0

Frequency threshold for reporting non-serious adverse events: 5%

Non-serious adverse events	Avatrombopag	Placebo
Total subjects affected by non serious adverse events
subjects affected / exposed	50 / 54 (92.59%)	16 / 21 (76.19%)
Injury, poisoning and procedural complications
Contusion
subjects affected / exposed	3 / 54 (5.56%)	1 / 21 (4.76%)
occurrences all number	7	1
Vascular disorders
Haematoma
subjects affected / exposed	3 / 54 (5.56%)	3 / 21 (14.29%)
occurrences all number	3	6
Nervous system disorders
Headache
subjects affected / exposed	10 / 54 (18.52%)	4 / 21 (19.05%)
occurrences all number	22	4
General disorders and administration site conditions
Pyrexia
subjects affected / exposed	9 / 54 (16.67%)	0 / 21 (0.00%)
occurrences all number	9	0
Gastrointestinal disorders
Diarrhoea
subjects affected / exposed	4 / 54 (7.41%)	0 / 21 (0.00%)
occurrences all number	5	0
Vomiting
subjects affected / exposed	4 / 54 (7.41%)	0 / 21 (0.00%)
occurrences all number	6	0
Abdominal Pain
subjects affected / exposed	3 / 54 (5.56%)	0 / 21 (0.00%)
occurrences all number	3	0
Gingival bleeding
subjects affected / exposed	2 / 54 (3.70%)	2 / 21 (9.52%)
occurrences all number	2	3
Rectal haemorrhage
subjects affected / exposed	0 / 54 (0.00%)	2 / 21 (9.52%)
occurrences all number	0	2
Respiratory, thoracic and mediastinal disorders
Epistaxis
subjects affected / exposed	12 / 54 (22.22%)	4 / 21 (19.05%)
occurrences all number	30	9
Cough
subjects affected / exposed	9 / 54 (16.67%)	0 / 21 (0.00%)
occurrences all number	11	0
Oropharyngeal pain
subjects affected / exposed	7 / 54 (12.96%)	0 / 21 (0.00%)
occurrences all number	7	0
Rhinorrhoea
subjects affected / exposed	3 / 54 (5.56%)	0 / 21 (0.00%)
occurrences all number	3	0
Skin and subcutaneous tissue disorders
Petechiae
subjects affected / exposed	14 / 54 (25.93%)	6 / 21 (28.57%)
occurrences all number	20	6
Ecchymosis
subjects affected / exposed	10 / 54 (18.52%)	1 / 21 (4.76%)
occurrences all number	19	1
Musculoskeletal and connective tissue disorders
Arthralgia
subjects affected / exposed	4 / 54 (7.41%)	0 / 21 (0.00%)
occurrences all number	6	0
Pain in extremity
subjects affected / exposed	3 / 54 (5.56%)	0 / 21 (0.00%)
occurrences all number	4	0
Infections and infestations
Upper respiratory tract infection
subjects affected / exposed	6 / 54 (11.11%)	2 / 21 (9.52%)
occurrences all number	6	2
COVID-19
subjects affected / exposed	4 / 54 (7.41%)	1 / 21 (4.76%)
occurrences all number	4	1
Nasopharyngitis
subjects affected / exposed	4 / 54 (7.41%)	0 / 21 (0.00%)
occurrences all number	5	0
Viral upper respiratory tract infection
subjects affected / exposed	3 / 54 (5.56%)	0 / 21 (0.00%)
occurrences all number	5	0

More information

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Were there any global substantial amendments to the protocol? Yes

17 Dec 2020

Amendment 1 refined several inclusion and exclusion criteria, clarified the allowable contraception methods, aligned the definition of a lack of treatment effect in the Extension Phase with the Core Phase definition, and confirmed that a protocol amendment would be implemented if two subjects in Cohort 3 require medical monitor approved dose escalations above 20 mg daily.

02 Nov 2021

Amendment 2 updated the sponsor name, and clarified corticosteroid use prior to Baseline, the minimum subject age at Baseline, and the timing of serial PK sampling.

Were there any global interruptions to the trial? No

Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.

None reported

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