Trial Outcomes & Findings for Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia for ≥6 Months (NCT NCT04516967)

Last Updated: 2026-01-15

Results Overview

The proportion of subjects for whom at least 2 consecutive platelet assessments are ≥50×10\^9/L over the 12 weeks of treatment in the Core Phase in the absence of rescue medication.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

75 participants

Primary outcome timeframe

12 weeks of treatment

Results posted on

2026-01-15

Participant Flow

Open label extension optional and not all subjects entered this phase. 73 of 75 entered this phase and 2 dropped out after the core phase.

Participant milestones

Participant milestones
Measure	Experimental: Avatrombopag Double Blind Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks Avatrombopag: Oral avatrombopag tablet or sprinkle capsule	Placebo Comparator:Placebo Double Blind Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks Placebo: Placebo comparator tablet or sprinkle capsule	Avatrombopag Open Label Extension Subjects receive investigational drug up to 2 years.
Core Phase STARTED	54	21	0
Core Phase COMPLETED	44	1	0
Core Phase NOT COMPLETED	10	20	0
Extension Phase STARTED	0	0	73
Extension Phase COMPLETED	0	0	4
Extension Phase NOT COMPLETED	0	0	69

Reasons for withdrawal

Reasons for withdrawal
Measure	Experimental: Avatrombopag Double Blind Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks Avatrombopag: Oral avatrombopag tablet or sprinkle capsule	Placebo Comparator:Placebo Double Blind Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks Placebo: Placebo comparator tablet or sprinkle capsule
Core Phase Adverse Event	2	1
Core Phase Physician Decision	1	1
Core Phase Lack of Efficacy	7	18

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Experimental: Avatrombopag n=54 Participants Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks Avatrombopag: Oral avatrombopag tablet or sprinkle capsule	Placebo Comparator:Placebo n=21 Participants Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks Placebo: Placebo comparator tablet or sprinkle capsule	Total n=75 Participants Total of all reporting groups
Age, Continuous	8.9 years STANDARD_DEVIATION 4.36 • n=14 Participants	9.9 years STANDARD_DEVIATION 4.13 • n=10 Participants	9.2 years STANDARD_DEVIATION 4.29 • n=24 Participants
Sex: Female, Male Female	24 Participants n=14 Participants	12 Participants n=10 Participants	36 Participants n=24 Participants
Sex: Female, Male Male	30 Participants n=14 Participants	9 Participants n=10 Participants	39 Participants n=24 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	3 Participants n=14 Participants	2 Participants n=10 Participants	5 Participants n=24 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	50 Participants n=14 Participants	15 Participants n=10 Participants	65 Participants n=24 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=14 Participants	4 Participants n=10 Participants	5 Participants n=24 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=14 Participants	0 Participants n=10 Participants	0 Participants n=24 Participants
Race (NIH/OMB) Asian	3 Participants n=14 Participants	1 Participants n=10 Participants	4 Participants n=24 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=14 Participants	0 Participants n=10 Participants	0 Participants n=24 Participants
Race (NIH/OMB) Black or African American	0 Participants n=14 Participants	0 Participants n=10 Participants	0 Participants n=24 Participants
Race (NIH/OMB) White	48 Participants n=14 Participants	15 Participants n=10 Participants	63 Participants n=24 Participants
Race (NIH/OMB) More than one race	0 Participants n=14 Participants	0 Participants n=10 Participants	0 Participants n=24 Participants
Race (NIH/OMB) Unknown or Not Reported	3 Participants n=14 Participants	5 Participants n=10 Participants	8 Participants n=24 Participants
Region of Enrollment Turkey	20 participants n=14 Participants	9 participants n=10 Participants	29 participants n=24 Participants
Region of Enrollment Hungary	4 participants n=14 Participants	1 participants n=10 Participants	5 participants n=24 Participants
Region of Enrollment United States	8 participants n=14 Participants	5 participants n=10 Participants	13 participants n=24 Participants
Region of Enrollment Ukraine	2 participants n=14 Participants	0 participants n=10 Participants	2 participants n=24 Participants
Region of Enrollment Poland	3 participants n=14 Participants	1 participants n=10 Participants	4 participants n=24 Participants
Region of Enrollment United Kingdom	11 participants n=14 Participants	2 participants n=10 Participants	13 participants n=24 Participants
Region of Enrollment France	0 participants n=14 Participants	1 participants n=10 Participants	1 participants n=24 Participants
Region of Enrollment Germany	2 participants n=14 Participants	1 participants n=10 Participants	3 participants n=24 Participants
Region of Enrollment Russia	4 participants n=14 Participants	1 participants n=10 Participants	5 participants n=24 Participants

PRIMARY outcome

Timeframe: Last 8 weeks of 12 week treatment regimen

Population: Full Analysis Set

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

Outcome measures

Outcome measures
Measure	Experimental: Avatrombopag n=54 Participants Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks Avatrombopag: Oral avatrombopag tablet or sprinkle capsule	Placebo Comparator:Placebo n=21 Participants Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks Placebo: Placebo comparator tablet or sprinkle capsule
Durable Platelet Response	15 Participants	0 Participants

PRIMARY outcome

Timeframe: 12 weeks of treatment

Population: Full Analysis Set

The proportion of subjects for whom at least 2 consecutive platelet assessments are ≥50×10\^9/L over the 12 weeks of treatment in the Core Phase in the absence of rescue medication.

Outcome measures

Outcome measures
Measure	Experimental: Avatrombopag n=54 Participants Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks Avatrombopag: Oral avatrombopag tablet or sprinkle capsule	Placebo Comparator:Placebo n=21 Participants Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks Placebo: Placebo comparator tablet or sprinkle capsule
Alternative Primary: Platelet Response	44 Participants	0 Participants

SECONDARY outcome

Timeframe: 12 weeks of treatment

Population: Full Analysis Set

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.

Outcome measures

Outcome measures
Measure	Experimental: Avatrombopag n=54 Participants Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks Avatrombopag: Oral avatrombopag tablet or sprinkle capsule	Placebo Comparator:Placebo n=21 Participants Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks Placebo: Placebo comparator tablet or sprinkle capsule
Percentage of Weeks Platelet Count ≥50×10^9/L	48.9 percentage of weeks Standard Deviation 25.22	1.2 percentage of weeks Standard Deviation 3.92

SECONDARY outcome

Timeframe: Day 8

Population: Full Analysis Set

The proportion of subjects with a platelet count ≥50×10\^9/L at day 8, in the absence of rescue therapy.

Outcome measures

Outcome measures
Measure	Experimental: Avatrombopag n=54 Participants Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks Avatrombopag: Oral avatrombopag tablet or sprinkle capsule	Placebo Comparator:Placebo n=21 Participants Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks Placebo: Placebo comparator tablet or sprinkle capsule
Platelet Response at Day 8	30 Participants	0 Participants

SECONDARY outcome

Timeframe: 12 weeks of treatment

Population: Full Analysis Set

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.

Outcome measures

Outcome measures
Measure	Experimental: Avatrombopag n=54 Participants Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks Avatrombopag: Oral avatrombopag tablet or sprinkle capsule	Placebo Comparator:Placebo n=21 Participants Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks Placebo: Placebo comparator tablet or sprinkle capsule
Percentage of Weeks Platelet Count Between ≥50×10^9/L and ≤150×10^9/L	29.2 percentage of weeks Standard Deviation 19.64	1.2 percentage of weeks Standard Deviation 3.92

SECONDARY outcome

Timeframe: 12 weeks of treatment

Population: Full Analysis Set

The proportion of subjects who require rescue medications during 12 weeks of treatment in the Core Phase of the study.

Outcome measures

Outcome measures
Measure	Experimental: Avatrombopag n=54 Participants Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks Avatrombopag: Oral avatrombopag tablet or sprinkle capsule	Placebo Comparator:Placebo n=21 Participants Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks Placebo: Placebo comparator tablet or sprinkle capsule
Rescue Medications	4 Participants	9 Participants

SECONDARY outcome

Timeframe: 12 weeks of treatment

Population: Full Analysis Set

Overall incidence of bleeding events associated with ITP measured using the WHO Bleeding Scale (Grade 1-4).

Outcome measures

Outcome measures
Measure	Experimental: Avatrombopag n=54 Participants Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks Avatrombopag: Oral avatrombopag tablet or sprinkle capsule	Placebo Comparator:Placebo n=21 Participants Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks Placebo: Placebo comparator tablet or sprinkle capsule
Incidence of Any Bleeding Event (WHO Grade 1-4)	45 Participants	19 Participants

Adverse Events

Experimental: Avatrombopag

Serious events: 5 serious events

Other events: 50 other events

Deaths: 0 deaths

Placebo Comparator:Placebo

Serious events: 1 serious events

Other events: 16 other events

Deaths: 0 deaths

Ongoing Open Label Extension Phase

Serious events: 16 serious events

Other events: 62 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Additional Information

Medicine Development Leader

Sobi Inc.

Phone: 1-781-786-7370

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee PIs are not allowed to publish clinical trial data on their own after trial completion.
Publication restrictions are in place

Restriction type: OTHER

Measure	Experimental: Avatrombopag n=54 participants at risk Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks Avatrombopag: Oral avatrombopag tablet or sprinkle capsule	Placebo Comparator:Placebo n=21 participants at risk Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks Placebo: Placebo comparator tablet or sprinkle capsule	Ongoing Open Label Extension Phase n=73 participants at risk All subjects receive investigational drug for up to 2 years.
Blood and lymphatic system disorders Immune Thrombocytopenia	1.9% 1/54 • Number of events 2 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	1.4% 1/73 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Blood and lymphatic system disorders Thrombocytosis	1.9% 1/54 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	1.4% 1/73 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Gastrointestinal disorders Mouth Haemorrhage	1.9% 1/54 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	4.8% 1/21 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/73 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Gastrointestinal disorders Oral Purpura	1.9% 1/54 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/73 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Gastrointestinal disorders Rectal Haemorrhage	1.9% 1/54 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/73 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Nervous system disorders Headache	1.9% 1/54 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/73 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Respiratory, thoracic and mediastinal disorders Epistaxis	1.9% 1/54 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	5.5% 4/73 • Number of events 5 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Skin and subcutaneous tissue disorders Skin Haemorrhage	0.00% 0/54 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	4.8% 1/21 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/73 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Blood and lymphatic system disorders Aplastic Anemia	0.00% 0/54 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	1.4% 1/73 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Blood and lymphatic system disorders Bone Marrow Disorder	0.00% 0/54 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	1.4% 1/73 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Blood and lymphatic system disorders Bone Marrow Reticulin fibrosis	0.00% 0/54 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	1.4% 1/73 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Nervous system disorders Dysarthria	0.00% 0/54 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	1.4% 1/73 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Nervous system disorders Paresthesia	0.00% 0/54 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	1.4% 1/73 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Reproductive system and breast disorders Vaginal Haemorrhage	0.00% 0/54 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	1.4% 1/73 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Skin and subcutaneous tissue disorders Petechiae	0.00% 0/54 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	1.4% 1/73 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Skin and subcutaneous tissue disorders Urticaria	0.00% 0/54 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	1.4% 1/73 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Vascular disorders Deep Vein Thrombosis	0.00% 0/54 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	1.4% 1/73 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Blood and lymphatic system disorders Febrile Neutropenia	0.00% 0/54 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	1.4% 1/73 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Blood and lymphatic system disorders Thrombocytopenia	0.00% 0/54 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	5.5% 4/73 • Number of events 7 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Gastrointestinal disorders Dental Caries	0.00% 0/54 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	1.4% 1/73 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Gastrointestinal disorders Gastritis	0.00% 0/54 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	1.4% 1/73 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
General disorders Mucosal Haemorrhage	0.00% 0/54 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	2.7% 2/73 • Number of events 3 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Immune system disorders Hypogammaglobulinaemia	0.00% 0/54 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	1.4% 1/73 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Infections and infestations Appendicitis	0.00% 0/54 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	1.4% 1/73 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Infections and infestations Gastroenteritis	0.00% 0/54 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	1.4% 1/73 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Injury, poisoning and procedural complications Abdominal Injury	0.00% 0/54 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	1.4% 1/73 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Injury, poisoning and procedural complications Head Injury	0.00% 0/54 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	1.4% 1/73 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)

Measure	Experimental: Avatrombopag n=54 participants at risk Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks Avatrombopag: Oral avatrombopag tablet or sprinkle capsule	Placebo Comparator:Placebo n=21 participants at risk Study is 3:1 randomization ratio (avatrombopag to placebo). Investigational product administered orally once daily for 12 weeks Placebo: Placebo comparator tablet or sprinkle capsule	Ongoing Open Label Extension Phase n=73 participants at risk All subjects receive investigational drug for up to 2 years.
General disorders Toothache	0.00% 0/54 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	5.5% 4/73 • Number of events 4 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Infections and infestations Influenza	0.00% 0/54 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	6.8% 5/73 • Number of events 5 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Metabolism and nutrition disorders Iron Deficiency	1.9% 1/54 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	5.5% 4/73 • Number of events 4 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Infections and infestations Pharyngitis	1.9% 1/54 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	5.5% 4/73 • Number of events 5 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Infections and infestations Nasopharyngitis	7.4% 4/54 • Number of events 5 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	15.1% 11/73 • Number of events 13 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Infections and infestations Viral Upper Respiratory Tract Infection	5.6% 3/54 • Number of events 5 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	6.8% 5/73 • Number of events 11 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Injury, poisoning and procedural complications Contusion	5.6% 3/54 • Number of events 7 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	4.8% 1/21 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	5.5% 4/73 • Number of events 8 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Musculoskeletal and connective tissue disorders Arthralagia	7.4% 4/54 • Number of events 6 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	4.1% 3/73 • Number of events 6 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Musculoskeletal and connective tissue disorders Pain in Extremity	5.6% 3/54 • Number of events 4 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	1.4% 1/73 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Nervous system disorders Headache	18.5% 10/54 • Number of events 22 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	19.0% 4/21 • Number of events 4 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	13.7% 10/73 • Number of events 32 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Respiratory, thoracic and mediastinal disorders Epistaxis	22.2% 12/54 • Number of events 30 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	19.0% 4/21 • Number of events 9 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	17.8% 13/73 • Number of events 89 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Respiratory, thoracic and mediastinal disorders Cough	16.7% 9/54 • Number of events 11 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	17.8% 13/73 • Number of events 27 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Respiratory, thoracic and mediastinal disorders Oropharyngeal Pain	13.0% 7/54 • Number of events 7 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	13.7% 10/73 • Number of events 13 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	5.6% 3/54 • Number of events 3 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	8.2% 6/73 • Number of events 7 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Skin and subcutaneous tissue disorders Petechiae	25.9% 14/54 • Number of events 20 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	28.6% 6/21 • Number of events 6 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	11.0% 8/73 • Number of events 11 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Skin and subcutaneous tissue disorders Ecchymosis	18.5% 10/54 • Number of events 19 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	4.8% 1/21 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	6.8% 5/73 • Number of events 13 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Vascular disorders Haematoma	5.6% 3/54 • Number of events 3 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	14.3% 3/21 • Number of events 6 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	5.5% 4/73 • Number of events 4 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Respiratory, thoracic and mediastinal disorders Nasal Congestion	3.7% 2/54 • Number of events 3 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	9.6% 7/73 • Number of events 11 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Gastrointestinal disorders Nausea	3.7% 2/54 • Number of events 5 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	8.2% 6/73 • Number of events 9 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Respiratory, thoracic and mediastinal disorders Rhinitis	0.00% 0/54 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	8.2% 6/73 • Number of events 7 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Infections and infestations Gastroenteritis	0.00% 0/54 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	5.5% 4/73 • Number of events 4 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Gastrointestinal disorders Diarrhoea	7.4% 4/54 • Number of events 5 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	11.0% 8/73 • Number of events 11 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Gastrointestinal disorders Vomiting	7.4% 4/54 • Number of events 6 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	5.5% 4/73 • Number of events 10 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Gastrointestinal disorders Abdominal Pain	5.6% 3/54 • Number of events 3 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	5.5% 4/73 • Number of events 12 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Gastrointestinal disorders Gingival Bleeding	3.7% 2/54 • Number of events 2 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	9.5% 2/21 • Number of events 3 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	2.7% 2/73 • Number of events 2 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Gastrointestinal disorders Rectal Haemorrhage	0.00% 0/54 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	9.5% 2/21 • Number of events 2 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	1.4% 1/73 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
General disorders Pyrexia	16.7% 9/54 • Number of events 9 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	0.00% 0/21 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	17.8% 13/73 • Number of events 17 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Infections and infestations Upper Respiratory Tract Infection	11.1% 6/54 • Number of events 6 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	9.5% 2/21 • Number of events 2 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	30.1% 22/73 • Number of events 36 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)
Infections and infestations COVID 19	7.4% 4/54 • Number of events 4 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	4.8% 1/21 • Number of events 1 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)	4.1% 3/73 • Number of events 4 • Up to 12 weeks (Core Phase) and up to 2 years (ongoing Extension Phase)