Trial Outcomes & Findings for Study to Allow Access to Nilotinib for Patients Who Are on Nilotinib Treatment in a Novartis-sponsored Study (NCT NCT01735955)
NCT ID: NCT01735955
Last Updated: 2024-02-08
Results Overview
An Adverse Event (AE) is any untoward medical occurrence (eg any unfavorable and unintended sign \[including abnormal laboratory findings\], symptom or disease) in a clinical investigation participant after providing written informed consent for participation in the study. Therefore, an AE may or may not be temporally or causally associated with the use of a medicinal (investigational) product. Serious adverse event (SAE) case data were collected in the Safety Database. Adverse event (AE) data (both non-serious and serious) were collected in the Clinical database. Max. = Maximum Yrs = Years Approx. = Approximately eCRF = electronic Case Report Form Time = timeframe
Recruitment status
COMPLETED
Study phase
PHASE4
Target enrollment
57 participants
Primary outcome timeframe
SAE case data were collected the entire study duration after first dose of study treatment up to a max. time of approx. 10 yrs. AEs (both non-serious and serious) were collected in the eCRF 3 yrs after study initiation up to a max. time of approx. 7 yrs.
Results posted on
2024-02-08
Participant Flow
The study had no screening period. In total, 57 patients were enrolled and treated with nilotinib on this study. Patients were rolled over from 5 parent studies with the following indications: Chronic myelogenous leukemia (CML), Metastatic gastrointestinal stromal tumors (GIST), Acute lymphoblastic leukemia (ALL), and Receptor tyrosine kinase (KIT) mutated melanoma.
Participant milestones
| Measure |
Nilotinib
Adult patients: ≤ 800mg/day; Pediatric patients: 230mg/m2 twice daily (BID) and ≤ 800mg/day
|
|---|---|
|
Overall Study
STARTED
|
57
|
|
Overall Study
COMPLETED
|
20
|
|
Overall Study
NOT COMPLETED
|
37
|
Reasons for withdrawal
| Measure |
Nilotinib
Adult patients: ≤ 800mg/day; Pediatric patients: 230mg/m2 twice daily (BID) and ≤ 800mg/day
|
|---|---|
|
Overall Study
Administrative problems
|
1
|
|
Overall Study
Patient/guardian decision
|
1
|
|
Overall Study
Patient withdrew consent
|
2
|
|
Overall Study
Physician Decision
|
4
|
|
Overall Study
Disease progression
|
24
|
|
Overall Study
Adverse Event
|
5
|
Baseline Characteristics
Race and Ethnicity were not collected from any participant.
Baseline characteristics by cohort
| Measure |
Nilotinib
n=57 Participants
Adult patients: ≤ 800mg/day; Pediatric patients: 230mg/m2 twice daily (BID) and ≤ 800mg/day
|
|---|---|
|
Age, Categorical
<=18 years
|
7 Participants
n=57 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
30 Participants
n=57 Participants
|
|
Age, Categorical
>=65 years
|
20 Participants
n=57 Participants
|
|
Age, Continuous
|
53.02 years
STANDARD_DEVIATION 19.278 • n=57 Participants
|
|
Sex: Female, Male
Female
|
30 Participants
n=57 Participants
|
|
Sex: Female, Male
Male
|
27 Participants
n=57 Participants
|
PRIMARY outcome
Timeframe: SAE case data were collected the entire study duration after first dose of study treatment up to a max. time of approx. 10 yrs. AEs (both non-serious and serious) were collected in the eCRF 3 yrs after study initiation up to a max. time of approx. 7 yrs.Population: safety set
An Adverse Event (AE) is any untoward medical occurrence (eg any unfavorable and unintended sign \[including abnormal laboratory findings\], symptom or disease) in a clinical investigation participant after providing written informed consent for participation in the study. Therefore, an AE may or may not be temporally or causally associated with the use of a medicinal (investigational) product. Serious adverse event (SAE) case data were collected in the Safety Database. Adverse event (AE) data (both non-serious and serious) were collected in the Clinical database. Max. = Maximum Yrs = Years Approx. = Approximately eCRF = electronic Case Report Form Time = timeframe
Outcome measures
| Measure |
Nilotinib
n=57 Participants
Adult patients: ≤ 800mg/day; Pediatric patients: 230mg/m2 twice daily (BID) and ≤ 800mg/day
|
|---|---|
|
Number of Participants With Adverse Events and Serious Adverse Events
Adverse Events - total
|
29 Participants
|
|
Number of Participants With Adverse Events and Serious Adverse Events
Adverse Events - Treatment-related adverse events
|
15 Participants
|
|
Number of Participants With Adverse Events and Serious Adverse Events
SAEs - total
|
17 Participants
|
|
Number of Participants With Adverse Events and Serious Adverse Events
SAEs - Treatment-related SAEs
|
2 Participants
|
|
Number of Participants With Adverse Events and Serious Adverse Events
AEs leading to discontinuation - total
|
3 Participants
|
|
Number of Participants With Adverse Events and Serious Adverse Events
AEs leading to discontinuation - Treatment-related AEs leading to discontinuation
|
3 Participants
|
SECONDARY outcome
Timeframe: Clinical benefit data were first collected 3 years after study initiation and are reported at baseline, Weeks 24, 48, 72, 96, 144, 192, 240, 288, 336, 384, 432, 480, and 528.Population: Safety set
Number of patients (pts) with clinical benefit as assessed by investigator. Clinical benefit data were first collected 3 years after study initiation and up to a maximum timeframe of approx. 7 years and 3 months at a patient level (up to Week 528 total at the study level). Pts who discontinued in the first 3 years after study initiation didn't have any clinical benefit data collected. Pts who enrolled in the first 3 years after study initiation only had clinical benefit data collected starting at approx. the third year of the study until the end of the patient's participation in the study. Pts who enrolled after the first 3 years after study initiation had all clinical benefit data collected until the end of the patient's participation in the study. Data for the earlier time points are provided only for later enrolled pts. Data for the later time points are provided only for the earlier enrolled pts. The time point per patient was calculated from the date of first drug intake.
Outcome measures
| Measure |
Nilotinib
n=33 Participants
Adult patients: ≤ 800mg/day; Pediatric patients: 230mg/m2 twice daily (BID) and ≤ 800mg/day
|
|---|---|
|
Number of Participants With Clinical Benefit From Nilotinib
Patients with clinical benefit - Baseline
|
15 Participants
|
|
Number of Participants With Clinical Benefit From Nilotinib
Patients with clinical benefit - Week 24
|
11 Participants
|
|
Number of Participants With Clinical Benefit From Nilotinib
Patients with clinical benefit - Week 48
|
17 Participants
|
|
Number of Participants With Clinical Benefit From Nilotinib
Patients with clinical benefit - Week 72
|
11 Participants
|
|
Number of Participants With Clinical Benefit From Nilotinib
Patients with clinical benefit - Week 96
|
19 Participants
|
|
Number of Participants With Clinical Benefit From Nilotinib
Patients with clinical benefit - Week144
|
23 Participants
|
|
Number of Participants With Clinical Benefit From Nilotinib
Patients with clinical benefit - Week 192
|
26 Participants
|
|
Number of Participants With Clinical Benefit From Nilotinib
Patients with clinical benefit - Week 240
|
20 Participants
|
|
Number of Participants With Clinical Benefit From Nilotinib
Patients with clinical benefit - Week 288
|
9 Participants
|
|
Number of Participants With Clinical Benefit From Nilotinib
Patients with clinical benefit - Week 336
|
9 Participants
|
|
Number of Participants With Clinical Benefit From Nilotinib
Patients with clinical benefit - Week 384
|
7 Participants
|
|
Number of Participants With Clinical Benefit From Nilotinib
Patients with clinical benefit - Week 432
|
6 Participants
|
|
Number of Participants With Clinical Benefit From Nilotinib
Patients with clinical benefit - Week 480
|
6 Participants
|
|
Number of Participants With Clinical Benefit From Nilotinib
Patients with clinical benefit - Week 528
|
4 Participants
|
Adverse Events
Nilotinib
Serious events: 17 serious events
Other events: 20 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Nilotinib
n=57 participants at risk
Adult patients: ≤ 800mg/day; Pediatric patients: 230mg/m2 twice daily (BID) and ≤ 800mg/day
|
|---|---|
|
Cardiac disorders
Myocardial infarction
|
1.8%
1/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Cardiac disorders
Myocardial ischaemia
|
1.8%
1/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Cardiac disorders
Tachycardia
|
1.8%
1/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Gastrointestinal disorders
Abdominal pain
|
1.8%
1/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
1.8%
1/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Gastrointestinal disorders
Intestinal obstruction
|
1.8%
1/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Gastrointestinal disorders
Melaena
|
1.8%
1/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Gastrointestinal disorders
Pancreatitis acute
|
1.8%
1/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Hepatobiliary disorders
Cholelithiasis
|
1.8%
1/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Infections and infestations
COVID-19
|
1.8%
1/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Infections and infestations
Dengue fever
|
1.8%
1/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Infections and infestations
Infection
|
1.8%
1/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Infections and infestations
Pneumonia
|
1.8%
1/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Infections and infestations
Pyelonephritis acute
|
1.8%
1/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Infections and infestations
Urinary tract infection
|
1.8%
1/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Metabolism and nutrition disorders
Dehydration
|
1.8%
1/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
1.8%
1/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
1.8%
1/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Musculoskeletal and connective tissue disorders
Spinal osteoarthritis
|
1.8%
1/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Gastrointestinal stromal tumour
|
1.8%
1/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Malignant neoplasm progression
|
1.8%
1/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Nervous system disorders
Cerebral ischaemia
|
1.8%
1/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Nervous system disorders
Ischaemic stroke
|
1.8%
1/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Psychiatric disorders
Delirium
|
1.8%
1/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Renal and urinary disorders
Hydronephrosis
|
1.8%
1/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Surgical and medical procedures
Small intestinal resection
|
1.8%
1/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Vascular disorders
Deep vein thrombosis
|
1.8%
1/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Vascular disorders
Peripheral arterial occlusive disease
|
1.8%
1/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Vascular disorders
Peripheral artery occlusion
|
1.8%
1/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
Other adverse events
| Measure |
Nilotinib
n=57 participants at risk
Adult patients: ≤ 800mg/day; Pediatric patients: 230mg/m2 twice daily (BID) and ≤ 800mg/day
|
|---|---|
|
Gastrointestinal disorders
Abdominal pain upper
|
5.3%
3/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
General disorders
Fatigue
|
5.3%
3/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Infections and infestations
COVID-19
|
7.0%
4/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Infections and infestations
Nasopharyngitis
|
5.3%
3/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
7.0%
4/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
5.3%
3/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Nervous system disorders
Headache
|
8.8%
5/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Nervous system disorders
Paraesthesia
|
5.3%
3/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
5.3%
3/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
7.0%
4/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
|
Vascular disorders
Hypertension
|
5.3%
3/57 • Serious adverse event (SAE) case data were collected in the Safety Database the entire study duration after first dose of study treatment, up to a maximum timeframe of approx. 10 years. Adverse Event (AE) data (both non-serious and serious AEs) were collected in the Clinical Database 3 years after study initiation up to a maximum timeframe of approx. 7 years.
|
Additional Information
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.
- Publication restrictions are in place
Restriction type: OTHER