Trial Outcomes & Findings for Open-label, Multi-center Protocol to Provide QTI571 to PAH Patients Who Participated in the Extension Study (A2301E1) in Japan (NCT NCT02042014)

NCT ID: NCT02042014

Last Updated: 2019-06-18

Results Overview

All Serious Adverse Events were evaluated and reported for all participants receiving QTI571. 16 individual SAEs were observed in 5 subjects.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 8 participants
• Primary outcome timeframe: Approximately 2.9 years
• Results posted on: 2019-06-18

Participant Flow

8 participants entered this study after completing the CQTI571A2301E1 study. All 8 participants discontinued study prematurely.

Participant milestones

Measure	QTI571 Participants received QTI571 during 3 years
Overall Study STARTED	8
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	8

Reasons for withdrawal

Measure	QTI571 Participants received QTI571 during 3 years
Overall Study Adverse Event	1
Overall Study Physician Decision	7

Baseline Characteristics

Open-label, Multi-center Protocol to Provide QTI571 to PAH Patients Who Participated in the Extension Study (A2301E1) in Japan

Baseline characteristics by cohort

Measure	QTI571 n=8 Participants Participants received QTI571 during 3 years.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	7 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants
Sex: Female, Male Female	8 Participants n=5 Participants
Sex: Female, Male Male	0 Participants n=5 Participants

PRIMARY outcome

Timeframe: Approximately 2.9 years

All Serious Adverse Events were evaluated and reported for all participants receiving QTI571. 16 individual SAEs were observed in 5 subjects.

Outcome measures

Measure	QTI571 n=8 Participants Participants will receive QTI571 during 3 years.
Serious Adverse Events Cardiac disorders (5 events)	5 participants
Serious Adverse Events Infections and infestations (5 events)	3 participants
Serious Adverse Events Resp, thoracic,mediastinal & disorder (2 events)	1 participants
Serious Adverse Events Product issues (3 events)	1 participants
Serious Adverse Events Death (none)	0 participants
Serious Adverse Events Skin & subcutaneous tissue disorder(1 event)	1 participants

Adverse Events

QTI571

Serious events: 5 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Measure	QTI571 n=8 participants at risk QTI571
Cardiac disorders Acute myocardial infarction	12.5% 1/8 • Adverse events were collected up to approximately 2.9 years AEs and SAEs were collected for the maximum duration of treatment and follow up for a participant per protocol for approximately 2.9 years. All cause mortality (deaths) was collected from First Patient First Visit (FPFV) to Last Patient Last Visit (LPLV) up to a maximum of 2.9 years
Cardiac disorders Right ventricular failure	37.5% 3/8 • Adverse events were collected up to approximately 2.9 years AEs and SAEs were collected for the maximum duration of treatment and follow up for a participant per protocol for approximately 2.9 years. All cause mortality (deaths) was collected from First Patient First Visit (FPFV) to Last Patient Last Visit (LPLV) up to a maximum of 2.9 years
Cardiac disorders Supraventricular tachycardia	12.5% 1/8 • Adverse events were collected up to approximately 2.9 years AEs and SAEs were collected for the maximum duration of treatment and follow up for a participant per protocol for approximately 2.9 years. All cause mortality (deaths) was collected from First Patient First Visit (FPFV) to Last Patient Last Visit (LPLV) up to a maximum of 2.9 years
Infections and infestations Cellulitis	12.5% 1/8 • Adverse events were collected up to approximately 2.9 years AEs and SAEs were collected for the maximum duration of treatment and follow up for a participant per protocol for approximately 2.9 years. All cause mortality (deaths) was collected from First Patient First Visit (FPFV) to Last Patient Last Visit (LPLV) up to a maximum of 2.9 years
Infections and infestations Gangrene	12.5% 1/8 • Adverse events were collected up to approximately 2.9 years AEs and SAEs were collected for the maximum duration of treatment and follow up for a participant per protocol for approximately 2.9 years. All cause mortality (deaths) was collected from First Patient First Visit (FPFV) to Last Patient Last Visit (LPLV) up to a maximum of 2.9 years
Infections and infestations Respiratory tract infection	12.5% 1/8 • Adverse events were collected up to approximately 2.9 years AEs and SAEs were collected for the maximum duration of treatment and follow up for a participant per protocol for approximately 2.9 years. All cause mortality (deaths) was collected from First Patient First Visit (FPFV) to Last Patient Last Visit (LPLV) up to a maximum of 2.9 years
Infections and infestations Urinary tract infection	12.5% 1/8 • Adverse events were collected up to approximately 2.9 years AEs and SAEs were collected for the maximum duration of treatment and follow up for a participant per protocol for approximately 2.9 years. All cause mortality (deaths) was collected from First Patient First Visit (FPFV) to Last Patient Last Visit (LPLV) up to a maximum of 2.9 years
Product Issues Device connection issue	12.5% 1/8 • Adverse events were collected up to approximately 2.9 years AEs and SAEs were collected for the maximum duration of treatment and follow up for a participant per protocol for approximately 2.9 years. All cause mortality (deaths) was collected from First Patient First Visit (FPFV) to Last Patient Last Visit (LPLV) up to a maximum of 2.9 years
Product Issues Device occlusion	12.5% 1/8 • Adverse events were collected up to approximately 2.9 years AEs and SAEs were collected for the maximum duration of treatment and follow up for a participant per protocol for approximately 2.9 years. All cause mortality (deaths) was collected from First Patient First Visit (FPFV) to Last Patient Last Visit (LPLV) up to a maximum of 2.9 years
Respiratory, thoracic and mediastinal disorders Pulmonary arterial hypertension	12.5% 1/8 • Adverse events were collected up to approximately 2.9 years AEs and SAEs were collected for the maximum duration of treatment and follow up for a participant per protocol for approximately 2.9 years. All cause mortality (deaths) was collected from First Patient First Visit (FPFV) to Last Patient Last Visit (LPLV) up to a maximum of 2.9 years
Skin and subcutaneous tissue disorders Skin ulcer	12.5% 1/8 • Adverse events were collected up to approximately 2.9 years AEs and SAEs were collected for the maximum duration of treatment and follow up for a participant per protocol for approximately 2.9 years. All cause mortality (deaths) was collected from First Patient First Visit (FPFV) to Last Patient Last Visit (LPLV) up to a maximum of 2.9 years

Other adverse events

Adverse event data not reported

Additional Information

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