Trial Outcomes & Findings for Extension to CQTI571A2102 to Evaluate Long-term Safety, Tolerability and Efficacy of Imatinib in Severe Pulmonary Arterial Hypertension (PAH) (NCT NCT01392495)
NCT ID: NCT01392495
Last Updated: 2015-08-10
Results Overview
Adverse event monitoring was conducted throughout the trial.
Recruitment status
TERMINATED
Study phase
PHASE3
Target enrollment
17 participants
Primary outcome timeframe
144 weeks
Results posted on
2015-08-10
Participant Flow
Participant milestones
| Measure |
QTI571
Participants received 200 mg or 400 mg every day (qd) based on their highest tolerated dose in CQTI571A2102 (NCT01392469).
|
|---|---|
|
Overall Study
STARTED
|
17
|
|
Overall Study
Received 200 mg
|
4
|
|
Overall Study
Received 400 mg
|
13
|
|
Overall Study
COMPLETED
|
1
|
|
Overall Study
NOT COMPLETED
|
16
|
Reasons for withdrawal
| Measure |
QTI571
Participants received 200 mg or 400 mg every day (qd) based on their highest tolerated dose in CQTI571A2102 (NCT01392469).
|
|---|---|
|
Overall Study
Death
|
3
|
|
Overall Study
Administrative problems
|
8
|
|
Overall Study
Withdrawal by Subject
|
2
|
|
Overall Study
Adverse Event
|
3
|
Baseline Characteristics
Extension to CQTI571A2102 to Evaluate Long-term Safety, Tolerability and Efficacy of Imatinib in Severe Pulmonary Arterial Hypertension (PAH)
Baseline characteristics by cohort
| Measure |
QTI571
n=17 Participants
Participants received 200 mg or 400 mg qd based on their highest tolerated dose in CQTI571A2102.
|
|---|---|
|
Age, Continuous
|
53.5 Years
STANDARD_DEVIATION 14.3 • n=5 Participants
|
|
Sex: Female, Male
Female
|
11 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 144 weeksPopulation: Safety Analysis Set: The safety analysis set included all participants who received at least one dose of study drug during the extension and had at least one post-baseline safety assessment.
Adverse event monitoring was conducted throughout the trial.
Outcome measures
| Measure |
QTI571 200 mg
n=4 Participants
Participants received 200 mg or 400 mg qd based on their highest tolerated dose in CQTI571A2102.
|
QTI571 400 mg
n=13 Participants
Participants received 200 mg or 400 mg qd based on their highest tolerated dose in CQTI571A2102.
|
|---|---|---|
|
Number of Patients With Adverse Events, Serious Adverse Events and Deaths
Adverse Events (serious and non-serious)
|
4 Participants
|
13 Participants
|
|
Number of Patients With Adverse Events, Serious Adverse Events and Deaths
Serious Adverse Events
|
4 Participants
|
5 Participants
|
|
Number of Patients With Adverse Events, Serious Adverse Events and Deaths
Deaths
|
2 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: baseline, 144 weeksPopulation: The study terminated early. No statistical analysis was performed on the efficacy outcomes.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 144 weeksPopulation: The study terminated early. No statistical analysis was performed on the efficacy outcomes.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 144 weeksPopulation: The study terminated early. No statistical analysis was performed on the efficacy outcomes.
Outcome measures
Outcome data not reported
Adverse Events
QTI571 200mg
Serious events: 4 serious events
Other events: 4 other events
Deaths: 0 deaths
QTI571 400mg
Serious events: 5 serious events
Other events: 13 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
QTI571 200mg
n=4 participants at risk
Participants received 200 mg or 400 mg qd based on their highest tolerated dose in CQTI571A2102.
|
QTI571 400mg
n=13 participants at risk
Participants received 200 mg or 400 mg qd based on their highest tolerated dose in CQTI571A2102.
|
|---|---|---|
|
Blood and lymphatic system disorders
Iron deficiency anaemia
|
25.0%
1/4
|
0.00%
0/13
|
|
Cardiac disorders
Right ventricular failure
|
25.0%
1/4
|
0.00%
0/13
|
|
Gastrointestinal disorders
Abdominal pain upper
|
0.00%
0/4
|
7.7%
1/13
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/4
|
7.7%
1/13
|
|
Gastrointestinal disorders
Gastrointestinal haemorrhage
|
25.0%
1/4
|
7.7%
1/13
|
|
Gastrointestinal disorders
Inguinal hernia
|
25.0%
1/4
|
0.00%
0/13
|
|
Gastrointestinal disorders
Mesenteric panniculitis
|
0.00%
0/4
|
7.7%
1/13
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/4
|
7.7%
1/13
|
|
Gastrointestinal disorders
Salivary gland cyst
|
0.00%
0/4
|
7.7%
1/13
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/4
|
7.7%
1/13
|
|
General disorders
Death
|
0.00%
0/4
|
7.7%
1/13
|
|
Infections and infestations
Bacteraemia
|
25.0%
1/4
|
0.00%
0/13
|
|
Infections and infestations
Gastroenteritis
|
0.00%
0/4
|
7.7%
1/13
|
|
Infections and infestations
Peritonitis
|
25.0%
1/4
|
0.00%
0/13
|
|
Infections and infestations
Septic shock
|
25.0%
1/4
|
0.00%
0/13
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
25.0%
1/4
|
7.7%
1/13
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Adenocarcinoma of colon
|
25.0%
1/4
|
0.00%
0/13
|
|
Psychiatric disorders
Catatonia
|
25.0%
1/4
|
0.00%
0/13
|
|
Renal and urinary disorders
Renal failure acute
|
25.0%
1/4
|
7.7%
1/13
|
|
Renal and urinary disorders
Urethral haemorrhage
|
25.0%
1/4
|
0.00%
0/13
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary mass
|
0.00%
0/4
|
7.7%
1/13
|
Other adverse events
| Measure |
QTI571 200mg
n=4 participants at risk
Participants received 200 mg or 400 mg qd based on their highest tolerated dose in CQTI571A2102.
|
QTI571 400mg
n=13 participants at risk
Participants received 200 mg or 400 mg qd based on their highest tolerated dose in CQTI571A2102.
|
|---|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
50.0%
2/4
|
23.1%
3/13
|
|
Blood and lymphatic system disorders
Leukopenia
|
25.0%
1/4
|
0.00%
0/13
|
|
Blood and lymphatic system disorders
Lymphopenia
|
0.00%
0/4
|
7.7%
1/13
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
25.0%
1/4
|
0.00%
0/13
|
|
Eye disorders
Cataract
|
0.00%
0/4
|
7.7%
1/13
|
|
Eye disorders
Periorbital oedema
|
0.00%
0/4
|
7.7%
1/13
|
|
Gastrointestinal disorders
Abdominal discomfort
|
25.0%
1/4
|
7.7%
1/13
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/4
|
7.7%
1/13
|
|
Gastrointestinal disorders
Abdominal pain upper
|
0.00%
0/4
|
7.7%
1/13
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/4
|
7.7%
1/13
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/4
|
46.2%
6/13
|
|
Gastrointestinal disorders
Gastritis
|
25.0%
1/4
|
0.00%
0/13
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
25.0%
1/4
|
0.00%
0/13
|
|
Gastrointestinal disorders
Large intestine polyp
|
0.00%
0/4
|
7.7%
1/13
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/4
|
23.1%
3/13
|
|
Gastrointestinal disorders
Toothache
|
25.0%
1/4
|
7.7%
1/13
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/4
|
30.8%
4/13
|
|
General disorders
Oedema peripheral
|
25.0%
1/4
|
23.1%
3/13
|
|
General disorders
Pyrexia
|
0.00%
0/4
|
15.4%
2/13
|
|
Infections and infestations
Bacteriuria
|
0.00%
0/4
|
7.7%
1/13
|
|
Infections and infestations
Bronchitis
|
0.00%
0/4
|
7.7%
1/13
|
|
Infections and infestations
Ear infection
|
0.00%
0/4
|
7.7%
1/13
|
|
Infections and infestations
Gastroenteritis
|
0.00%
0/4
|
15.4%
2/13
|
|
Infections and infestations
Haemophilus infection
|
0.00%
0/4
|
7.7%
1/13
|
|
Infections and infestations
Nasopharyngitis
|
0.00%
0/4
|
46.2%
6/13
|
|
Infections and infestations
Otitis externa
|
25.0%
1/4
|
0.00%
0/13
|
|
Infections and infestations
Respiratory tract infection
|
0.00%
0/4
|
7.7%
1/13
|
|
Infections and infestations
Staphylococcal skin infection
|
25.0%
1/4
|
0.00%
0/13
|
|
Infections and infestations
Upper respiratory tract infection
|
25.0%
1/4
|
7.7%
1/13
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/4
|
23.1%
3/13
|
|
Investigations
Blood creatinine increased
|
25.0%
1/4
|
7.7%
1/13
|
|
Investigations
Blood potassium decreased
|
0.00%
0/4
|
15.4%
2/13
|
|
Investigations
Blood sodium decreased
|
25.0%
1/4
|
0.00%
0/13
|
|
Investigations
International normalised ratio increased
|
0.00%
0/4
|
7.7%
1/13
|
|
Investigations
Platelet count decreased
|
0.00%
0/4
|
7.7%
1/13
|
|
Metabolism and nutrition disorders
Decreased appetite
|
0.00%
0/4
|
7.7%
1/13
|
|
Metabolism and nutrition disorders
Gout
|
0.00%
0/4
|
15.4%
2/13
|
|
Metabolism and nutrition disorders
Hypercholesterolaemia
|
0.00%
0/4
|
7.7%
1/13
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
0.00%
0/4
|
7.7%
1/13
|
|
Metabolism and nutrition disorders
Hypokalaemia
|
0.00%
0/4
|
7.7%
1/13
|
|
Metabolism and nutrition disorders
Iron deficiency
|
25.0%
1/4
|
0.00%
0/13
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.00%
0/4
|
7.7%
1/13
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/4
|
7.7%
1/13
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
0.00%
0/4
|
15.4%
2/13
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
0.00%
0/4
|
7.7%
1/13
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
0.00%
0/4
|
15.4%
2/13
|
|
Musculoskeletal and connective tissue disorders
Scleroderma
|
0.00%
0/4
|
7.7%
1/13
|
|
Nervous system disorders
Dizziness
|
0.00%
0/4
|
7.7%
1/13
|
|
Nervous system disorders
Neuropathy peripheral
|
0.00%
0/4
|
7.7%
1/13
|
|
Nervous system disorders
Sciatica
|
0.00%
0/4
|
7.7%
1/13
|
|
Nervous system disorders
Syncope
|
0.00%
0/4
|
7.7%
1/13
|
|
Psychiatric disorders
Depression
|
0.00%
0/4
|
7.7%
1/13
|
|
Psychiatric disorders
Insomnia
|
0.00%
0/4
|
15.4%
2/13
|
|
Psychiatric disorders
Sleep disorder
|
0.00%
0/4
|
7.7%
1/13
|
|
Renal and urinary disorders
Haematuria
|
0.00%
0/4
|
7.7%
1/13
|
|
Renal and urinary disorders
Proteinuria
|
0.00%
0/4
|
7.7%
1/13
|
|
Renal and urinary disorders
Renal failure
|
0.00%
0/4
|
7.7%
1/13
|
|
Renal and urinary disorders
Renal impairment
|
25.0%
1/4
|
0.00%
0/13
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
0.00%
0/4
|
15.4%
2/13
|
|
Respiratory, thoracic and mediastinal disorders
Dysphonia
|
0.00%
0/4
|
7.7%
1/13
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
0.00%
0/4
|
23.1%
3/13
|
|
Respiratory, thoracic and mediastinal disorders
Haemoptysis
|
25.0%
1/4
|
0.00%
0/13
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
0.00%
0/4
|
7.7%
1/13
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
0.00%
0/4
|
7.7%
1/13
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory distress
|
25.0%
1/4
|
0.00%
0/13
|
|
Skin and subcutaneous tissue disorders
Night sweats
|
0.00%
0/4
|
7.7%
1/13
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/4
|
7.7%
1/13
|
|
Vascular disorders
Hypotension
|
0.00%
0/4
|
15.4%
2/13
|
Additional Information
Study Director
Novartis Pharmaceuticals
Phone: 862-778-8300
Results disclosure agreements
- Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial or disclosure of trial results in their entirety.
- Publication restrictions are in place
Restriction type: OTHER