Trial Outcomes & Findings for Phase II Nilotinib With Newly Diagnosed Chronic Phase Chronic Myelogenous Leukemia (CML) (NCT NCT00129740)
NCT ID: NCT00129740
Last Updated: 2019-09-24
Results Overview
Polymerase chain reaction (PCR) Ratio BCR-Abl/Abl of 0% after 12 months of therapy with Nilotinib by international standard.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
148 participants
Primary outcome timeframe
12 months
Results posted on
2019-09-24
Participant Flow
Participant milestones
| Measure |
Nilotinib
400 mg orally twice daily
Nilotinib: 400 mg orally twice daily
|
|---|---|
|
Overall Study
STARTED
|
148
|
|
Overall Study
COMPLETED
|
148
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Phase II Nilotinib With Newly Diagnosed Chronic Phase Chronic Myelogenous Leukemia (CML)
Baseline characteristics by cohort
| Measure |
Nilotinib
n=148 Participants
400 mg orally twice daily
Nilotinib: 400 mg orally twice daily
|
|---|---|
|
Age, Categorical
<=18 years
|
3 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
124 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
21 Participants
n=5 Participants
|
|
Age, Continuous
|
51 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
60 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
88 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
10 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
134 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
148 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 12 monthsPolymerase chain reaction (PCR) Ratio BCR-Abl/Abl of 0% after 12 months of therapy with Nilotinib by international standard.
Outcome measures
| Measure |
Nilotinib
n=148 Participants
400 mg orally twice daily
Nilotinib: 400 mg orally twice daily
|
|---|---|
|
Participants With Complete Molecular Response (Molecular CR)
|
23 Participants
|
SECONDARY outcome
Timeframe: 6 monthsComplete hematologic remission classified according to suppression of Philadelphia chromosome (Ph) by cytogenetics or i Fluorescence in situ hybridization (FISH) 1. No cytogenetic response - Ph positive 100% 2. Minor cytogenetic response - Ph positive 35-90% 3. Partial cytogenetic response - Ph positive 1-34% 4. Complete cytogenetic response - Ph positive 0% Major cytogenetic response = complete + partial (Ph positive \<35%)
Outcome measures
| Measure |
Nilotinib
n=148 Participants
400 mg orally twice daily
Nilotinib: 400 mg orally twice daily
|
|---|---|
|
Number of Participants With Complete Cytogenetic Response (CCyR)
|
131 Participants
|
Adverse Events
Nilotinib
Serious events: 47 serious events
Other events: 148 other events
Deaths: 7 deaths
Serious adverse events
| Measure |
Nilotinib
n=148 participants at risk
400 mg orally twice daily
Nilotinib: 400 mg orally twice daily
|
|---|---|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Left Buccal Squamous Cell Carcinoma
|
0.68%
1/148 • Number of events 1 • Up to 11.5 years
|
|
Cardiac disorders
Cardiac Chest Pain
|
4.1%
6/148 • Number of events 7 • Up to 11.5 years
|
|
Cardiac disorders
Cardiac ischemia/infarction
|
3.4%
5/148 • Number of events 6 • Up to 11.5 years
|
|
Blood and lymphatic system disorders
CNS Hemorrhage
|
0.68%
1/148 • Number of events 1 • Up to 11.5 years
|
|
Nervous system disorders
CNS Ischemia
|
2.0%
3/148 • Number of events 3 • Up to 11.5 years
|
|
Gastrointestinal disorders
Colitis
|
0.68%
1/148 • Number of events 1 • Up to 11.5 years
|
|
Investigations
Elevated Creatinine
|
0.68%
1/148 • Number of events 1 • Up to 11.5 years
|
|
General disorders
Death
|
1.4%
2/148 • Number of events 2 • Up to 11.5 years
|
|
Surgical and medical procedures
Revision right hip arthroplasty
|
0.68%
1/148 • Number of events 1 • Up to 11.5 years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
2.0%
3/148 • Number of events 3 • Up to 11.5 years
|
|
General disorders
Flu like Syndrome
|
0.68%
1/148 • Number of events 1 • Up to 11.5 years
|
|
Musculoskeletal and connective tissue disorders
Fracture
|
1.4%
2/148 • Number of events 3 • Up to 11.5 years
|
|
Gastrointestinal disorders
GI Pain
|
1.4%
2/148 • Number of events 2 • Up to 11.5 years
|
|
Vascular disorders
Hematoma
|
0.68%
1/148 • Number of events 1 • Up to 11.5 years
|
|
Gastrointestinal disorders
Gastrointestinal Hemorrhage
|
1.4%
2/148 • Number of events 2 • Up to 11.5 years
|
|
Vascular disorders
Hypertension
|
1.4%
2/148 • Number of events 2 • Up to 11.5 years
|
|
Infections and infestations
Cellulitis
|
1.4%
2/148 • Number of events 2 • Up to 11.5 years
|
|
Surgical and medical procedures
Hysterectomy
|
1.4%
2/148 • Number of events 2 • Up to 11.5 years
|
|
Surgical and medical procedures
Knee Replacement
|
0.68%
1/148 • Number of events 1 • Up to 11.5 years
|
|
General disorders
Pain
|
2.0%
3/148 • Number of events 3 • Up to 11.5 years
|
|
Gastrointestinal disorders
Pancreatitis
|
0.68%
1/148 • Number of events 2 • Up to 11.5 years
|
|
Respiratory, thoracic and mediastinal disorders
Pleural Effusion
|
1.4%
2/148 • Number of events 2 • Up to 11.5 years
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
0.68%
1/148 • Number of events 1 • Up to 11.5 years
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.68%
1/148 • Number of events 1 • Up to 11.5 years
|
|
Renal and urinary disorders
Urethra Stricture
|
0.68%
1/148 • Number of events 1 • Up to 11.5 years
|
|
Cardiac disorders
Atrial Fibrillation
|
0.68%
1/148 • Number of events 1 • Up to 11.5 years
|
|
Cardiac disorders
Valvular heart disease
|
0.68%
1/148 • Number of events 2 • Up to 11.5 years
|
|
Vascular disorders
Vascular Stenosis
|
0.68%
1/148 • Number of events 1 • Up to 11.5 years
|
|
Gastrointestinal disorders
Abdoninal Pain
|
5.4%
8/148 • Number of events 9 • Up to 11.5 years
|
|
Gastrointestinal disorders
Acute Gastroenteritis
|
0.68%
1/148 • Number of events 1 • Up to 11.5 years
|
|
Surgical and medical procedures
Angioplasty with Stent Placement
|
0.68%
1/148 • Number of events 1 • Up to 11.5 years
|
|
General disorders
Back Pain
|
1.4%
2/148 • Number of events 2 • Up to 11.5 years
|
|
Hepatobiliary disorders
Cholecyctitis
|
0.68%
1/148 • Number of events 1 • Up to 11.5 years
|
|
Gastrointestinal disorders
Constipation
|
0.68%
1/148 • Number of events 1 • Up to 11.5 years
|
|
Metabolism and nutrition disorders
Dehydration
|
1.4%
2/148 • Number of events 2 • Up to 11.5 years
|
|
Psychiatric disorders
Depression
|
0.68%
1/148 • Number of events 1 • Up to 11.5 years
|
|
Blood and lymphatic system disorders
Hemorrhage
|
0.68%
1/148 • Number of events 1 • Up to 11.5 years
|
|
Surgical and medical procedures
Hip Replacement
|
0.68%
1/148 • Number of events 1 • Up to 11.5 years
|
|
Endocrine disorders
Hypothyroidism
|
0.68%
1/148 • Number of events 1 • Up to 11.5 years
|
|
Investigations
Elevated Transaminases
|
0.68%
1/148 • Number of events 1 • Up to 11.5 years
|
|
Infections and infestations
Infection
|
2.0%
3/148 • Number of events 4 • Up to 11.5 years
|
|
Gastrointestinal disorders
Nausea/Vomiting
|
2.0%
3/148 • Number of events 3 • Up to 11.5 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasm squamous cell carcinoma
|
0.68%
1/148 • Number of events 1 • Up to 11.5 years
|
|
Gastrointestinal disorders
Small Bowel Obstruction
|
0.68%
1/148 • Number of events 1 • Up to 11.5 years
|
|
Psychiatric disorders
Persomality/behavioral -alcohol treatment
|
0.68%
1/148 • Number of events 1 • Up to 11.5 years
|
|
Infections and infestations
Pneumonia
|
1.4%
2/148 • Number of events 2 • Up to 11.5 years
|
|
Psychiatric disorders
Psychosis
|
0.68%
1/148 • Number of events 1 • Up to 11.5 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Secondary Malignancy - Prostate Cancer
|
0.68%
1/148 • Number of events 1 • Up to 11.5 years
|
Other adverse events
| Measure |
Nilotinib
n=148 participants at risk
400 mg orally twice daily
Nilotinib: 400 mg orally twice daily
|
|---|---|
|
Skin and subcutaneous tissue disorders
Rash
|
51.4%
76/148 • Number of events 76 • Up to 11.5 years
|
|
General disorders
Fatigue
|
43.9%
65/148 • Number of events 65 • Up to 11.5 years
|
|
Investigations
Elevated bilirubin
|
56.8%
84/148 • Number of events 84 • Up to 11.5 years
|
|
Investigations
Elevated Aminotransferases
|
48.0%
71/148 • Number of events 71 • Up to 11.5 years
|
|
Vascular disorders
Hypertension
|
17.6%
26/148 • Number of events 30 • Up to 11.5 years
|
|
Cardiac disorders
Cardiovascular Ischemia
|
7.4%
11/148 • Number of events 11 • Up to 11.5 years
|
|
Cardiac disorders
Arrhythmia
|
11.5%
17/148 • Number of events 17 • Up to 11.5 years
|
Additional Information
Cortes,Jorge E, MD./Professor
The University of Texas MD Anderson Cancer Center
Phone: 713-794-5783
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place