Trial Outcomes & Findings for Safety and Treatment Satisfaction in Adults With Chronic ITP After Switching to Avatrombopag From Eltrombopag or Romiplostim (NCT NCT04638829)

Last Updated: 2025-01-16

Results Overview

Safety and Tolerability of Avatrombopag given for 90 days after stopping eltrombopag or romiplostim The incidence and severity of adverse events (AEs), serious adverse events (SAE) and adverse events of special interest (AESIs) will be summarized for all enrolled subjects using counts and percentages. Treatment-emergent AEs and SAEs will be summarized overall, by system organ class, and by preferred term. AESIs will be summarized by event type (thromboembolic events and bleeding events). In addition, treatment-emergent AEs will be summarized by severity and by relationship to study drug. Bleeding events reported during the study will be summarized by WHO grade.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

60 participants

Primary outcome timeframe

Screening through Day 90 or End of Study

Results posted on

2025-01-16

Participant Flow

Participant milestones

Participant milestones
Measure	Avatrombopag Avatrombopag 20 mg oral tablet formulation for 90 days Avatrombopag Oral Tablet: Avatrombopag 20 mg given daily for 90 days. Initial dose and dose adjustments will be determined by the physician along with the Doptelet prescribing information
Overall Study STARTED	60
Overall Study COMPLETED	57
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Safety and Treatment Satisfaction in Adults With Chronic ITP After Switching to Avatrombopag From Eltrombopag or Romiplostim

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Avatrombopag n=60 Participants Avatrombopag 20 mg oral tablet formulation for 90 days Avatrombopag Oral Tablet: Avatrombopag 20 mg given daily for 90 days. Initial dose and dose adjustments will be determined by the physician along with the Doptelet prescribing information
Age, Continuous	58.0 years STANDARD_DEVIATION 21.7 • n=5 Participants
Sex: Female, Male Female	37 Participants n=5 Participants
Sex: Female, Male Male	23 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	13 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	46 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	2 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	1 Participants n=5 Participants
Race (NIH/OMB) Black or African American	5 Participants n=5 Participants
Race (NIH/OMB) White	43 Participants n=5 Participants
Race (NIH/OMB) More than one race	1 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	8 Participants n=5 Participants

PRIMARY outcome

Timeframe: Screening through Day 90 or End of Study

Population: All enrolled subjects.

Outcome measures

Outcome measures
Measure	Avatrombopag n=60 Participants Avatrombopag 20 mg oral tablet formulation for 90 days Avatrombopag Oral Tablet: Avatrombopag 20 mg given daily for 90 days. Initial dose and dose adjustments will be determined by the physician along with the Doptelet prescribing information
Safety and Tolerability (Adverse Events) TEAEs	35 Participants
Safety and Tolerability (Adverse Events) SAEs	6 Participants
Safety and Tolerability (Adverse Events) AESIs	1 Participants
Safety and Tolerability (Adverse Events) Related TEAEs	15 Participants
Safety and Tolerability (Adverse Events) Severe TEAEs	8 Participants
Safety and Tolerability (Adverse Events) TEAEs leading to study drug discontinuation	2 Participants

SECONDARY outcome

Timeframe: Day 90

Population: All enrolled subjects.

Evaluate the change in subject reported outcomes (TSQM - Treatment Satisfaction Questionnaire for Medication) from Baseline. Convenience domain score ranges from 0 to 100, and a higher score indicates a better outcome. Mean Difference was computed as Day 90 minus Baseline and, therefore, a positive mean difference indicates an increase in the score from Baseline to Day 90.

Outcome measures

Outcome measures
Measure	Avatrombopag n=60 Participants Avatrombopag 20 mg oral tablet formulation for 90 days Avatrombopag Oral Tablet: Avatrombopag 20 mg given daily for 90 days. Initial dose and dose adjustments will be determined by the physician along with the Doptelet prescribing information
Change From Baseline TSQM Convenience Domain Score	13.5 Mean difference in convenience score Interval 6.9 to 20.1

SECONDARY outcome

Timeframe: Day 90

Evaluate the change in subject reported outcomes (TSQM - Treatment Satisfaction Questionnaire for Medication) from Baseline. Side Effects domain score ranges from 0 to 100, and a higher score indicates a better outcome. Mean Difference was computed as Day 90 minus Baseline and, therefore, a positive mean difference indicates an increase in the score from Baseline to Day 90.

Outcome measures

Outcome measures
Measure	Avatrombopag n=60 Participants Avatrombopag 20 mg oral tablet formulation for 90 days Avatrombopag Oral Tablet: Avatrombopag 20 mg given daily for 90 days. Initial dose and dose adjustments will be determined by the physician along with the Doptelet prescribing information
Change From Baseline TSQM Side Effects Domain Score	8.3 Mean difference in side effects score Interval 2.1 to 14.4

SECONDARY outcome

Timeframe: Day 90

Evaluate the change in subject reported outcomes (TSQM - Treatment Satisfaction Questionnaire for Medication) from Baseline. Effectiveness domain score ranges from 0 to 100, and a higher score indicates a better outcome. Mean Difference was computed as Day 90 minus Baseline and, therefore, a positive mean difference indicates an increase in the score from Baseline to Day 90.

Outcome measures

Outcome measures
Measure	Avatrombopag n=60 Participants Avatrombopag 20 mg oral tablet formulation for 90 days Avatrombopag Oral Tablet: Avatrombopag 20 mg given daily for 90 days. Initial dose and dose adjustments will be determined by the physician along with the Doptelet prescribing information
Change From Baseline TSQM Effectiveness Domain Score	14.4 Mean difference in effectiveness score Interval 7.0 to 21.8

SECONDARY outcome

Timeframe: Day 90

Evaluate the change in subject reported outcomes (TSQM - Treatment Satisfaction Questionnaire for Medication) from Baseline. Global Satisfaction domain score ranges from 0 to 100, and a higher score indicates a better outcome. Mean Difference was computed as Day 90 minus Baseline and, therefore, a positive mean difference indicates an increase in the score from Baseline to Day 90.

Outcome measures

Outcome measures
Measure	Avatrombopag n=60 Participants Avatrombopag 20 mg oral tablet formulation for 90 days Avatrombopag Oral Tablet: Avatrombopag 20 mg given daily for 90 days. Initial dose and dose adjustments will be determined by the physician along with the Doptelet prescribing information
Change From Baseline TSQM Global Satisfaction Domain Score	14.2 Mean difference in global satisfaction Interval 8.8 to 19.5

SECONDARY outcome

Timeframe: Day 15

Population: All enrolled subjects.

Outcome measures

Outcome measures
Measure	Avatrombopag n=60 Participants Avatrombopag 20 mg oral tablet formulation for 90 days Avatrombopag Oral Tablet: Avatrombopag 20 mg given daily for 90 days. Initial dose and dose adjustments will be determined by the physician along with the Doptelet prescribing information
Proportion of Subjects Who Have a Platelet Count Between ≥50×10^9/L to ≤200×10^9/L	19 Participants

SECONDARY outcome

Timeframe: Day 30

Outcome measures

Outcome measures
Measure	Avatrombopag n=60 Participants Avatrombopag 20 mg oral tablet formulation for 90 days Avatrombopag Oral Tablet: Avatrombopag 20 mg given daily for 90 days. Initial dose and dose adjustments will be determined by the physician along with the Doptelet prescribing information
Proportion of Subjects Who Have a Platelet Count Between ≥50×10^9/L to ≤200×10^9/L	33 Participants

SECONDARY outcome

Timeframe: Day 60

Outcome measures

Outcome measures
Measure	Avatrombopag n=60 Participants Avatrombopag 20 mg oral tablet formulation for 90 days Avatrombopag Oral Tablet: Avatrombopag 20 mg given daily for 90 days. Initial dose and dose adjustments will be determined by the physician along with the Doptelet prescribing information
Proportion of Subjects Who Have a Platelet Count Between ≥50×10^9/L to ≤200×10^9/L	36 Participants

SECONDARY outcome

Timeframe: Day 90

Outcome measures

Outcome measures
Measure	Avatrombopag n=60 Participants Avatrombopag 20 mg oral tablet formulation for 90 days Avatrombopag Oral Tablet: Avatrombopag 20 mg given daily for 90 days. Initial dose and dose adjustments will be determined by the physician along with the Doptelet prescribing information
Proportion of Subjects Who Have a Platelet Count Between ≥50×10^9/L to ≤200×10^9/L	33 Participants

Adverse Events

Avatrombopag

Serious events: 6 serious events

Other events: 34 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	Avatrombopag n=60 participants at risk Avatrombopag 20 mg oral tablet formulation for 90 days Avatrombopag Oral Tablet: Avatrombopag 20 mg given daily for 90 days. Initial dose and dose adjustments will be determined by the physician along with the Doptelet prescribing information
Blood and lymphatic system disorders Thrombocytopenia	3.3% 2/60 • Number of events 2 • 90 days
Gastrointestinal disorders Rectal Haemorrhage	1.7% 1/60 • Number of events 1 • 90 days
General disorders Sudden Death	1.7% 1/60 • Number of events 1 • 90 days
Musculoskeletal and connective tissue disorders Osteoarthritis	1.7% 1/60 • Number of events 1 • 90 days
Renal and urinary disorders Nephrolithiasis	1.7% 1/60 • Number of events 1 • 90 days

Other adverse events

Other adverse events
Measure	Avatrombopag n=60 participants at risk Avatrombopag 20 mg oral tablet formulation for 90 days Avatrombopag Oral Tablet: Avatrombopag 20 mg given daily for 90 days. Initial dose and dose adjustments will be determined by the physician along with the Doptelet prescribing information
Nervous system disorders Headache	13.3% 8/60 • Number of events 13 • 90 days
Injury, poisoning and procedural complications Contusion	11.7% 7/60 • Number of events 7 • 90 days
General disorders Oedema Peripheral	6.7% 4/60 • Number of events 4 • 90 days
Infections and infestations COVID-19	5.0% 3/60 • Number of events 3 • 90 days
Ear and labyrinth disorders Dizziness	5.0% 3/60 • Number of events 3 • 90 days
General disorders Fatigue	5.0% 3/60 • Number of events 5 • 90 days
General disorders Peripheral Swelling	5.0% 3/60 • Number of events 3 • 90 days
Gastrointestinal disorders Constipation	3.3% 2/60 • Number of events 2 • 90 days
Gastrointestinal disorders Diarrhoea	3.3% 2/60 • Number of events 2 • 90 days
Gastrointestinal disorders Nausea	3.3% 2/60 • Number of events 2 • 90 days
Gastrointestinal disorders Vomiting	3.3% 2/60 • Number of events 3 • 90 days
General disorders Pain	3.3% 2/60 • Number of events 2 • 90 days
Injury, poisoning and procedural complications Fall	3.3% 2/60 • Number of events 2 • 90 days
Musculoskeletal and connective tissue disorders Arthralgia	3.3% 2/60 • Number of events 2 • 90 days
Musculoskeletal and connective tissue disorders Back Pain	3.3% 2/60 • Number of events 2 • 90 days
Musculoskeletal and connective tissue disorders Pain in Extremity	3.3% 2/60 • Number of events 2 • 90 days
Respiratory, thoracic and mediastinal disorders Cough	3.3% 2/60 • Number of events 2 • 90 days
Respiratory, thoracic and mediastinal disorders Epistaxis	3.3% 2/60 • Number of events 3 • 90 days

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