Trial Outcomes & Findings for Stem Cell Monitoring for CML Patients Undergoing Nilotinib Therapy (NCT NCT02353728)
NCT ID: NCT02353728
Last Updated: 2022-03-25
Results Overview
The data obtained from these bone marrow samples, from these patients, may identify stem cell variables that can more accurately predict the success of discontinuation of tyrosine kinase inhibitor (TKI) therapy.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
16 participants
Primary outcome timeframe
1 month, 3 months, 12 months
Results posted on
2022-03-25
Participant Flow
Participant milestones
| Measure |
All Patients
Nilotinib at a dose of 300 mg P.O. twice a day (BID) daily
Nilotinib
|
|---|---|
|
Overall Study
STARTED
|
16
|
|
Overall Study
COMPLETED
|
15
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
All Patients
Nilotinib at a dose of 300 mg P.O. twice a day (BID) daily
Nilotinib
|
|---|---|
|
Overall Study
Subject non-compliance
|
1
|
Baseline Characteristics
Stem Cell Monitoring for CML Patients Undergoing Nilotinib Therapy
Baseline characteristics by cohort
| Measure |
All Patients
n=16 Participants
Nilotinib at a dose of 300 mg P.O. twice a day (BID) daily
Nilotinib
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
2 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
14 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
8 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 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
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
5 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
10 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
16 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 1 month, 3 months, 12 monthsPopulation: 16 patients were treated, 7 patients were not evaluable due to bone marrow samples not being available. Not all timepoints for each patient are reported due to bone marrow samples not being available. Bone marrow samples were not available due to: samples not being collected from a bone marrow procedure; bone marrow procedures not being performed so no sample could be obtained; or collected or sample was not evaluable.
The data obtained from these bone marrow samples, from these patients, may identify stem cell variables that can more accurately predict the success of discontinuation of tyrosine kinase inhibitor (TKI) therapy.
Outcome measures
| Measure |
All Patients
n=9 Participants
Nilotinib at a dose of 300 mg P.O. twice a day (BID) daily
Nilotinib
|
|---|---|
|
Percentage of Leukemic Stem Cells Present in Bone Marrow Aspirate Samples, in This Patient Population
WCMC-017 (1 month)
|
10.9 percentage of leukemic stem cells
|
|
Percentage of Leukemic Stem Cells Present in Bone Marrow Aspirate Samples, in This Patient Population
WCMC-007 (3 months)
|
37.4 percentage of leukemic stem cells
|
|
Percentage of Leukemic Stem Cells Present in Bone Marrow Aspirate Samples, in This Patient Population
WCMC-010 (1 month)
|
2.32 percentage of leukemic stem cells
|
|
Percentage of Leukemic Stem Cells Present in Bone Marrow Aspirate Samples, in This Patient Population
WCMC-011 (12 months)
|
0.7 percentage of leukemic stem cells
|
|
Percentage of Leukemic Stem Cells Present in Bone Marrow Aspirate Samples, in This Patient Population
WCMC-013 (1 month)
|
7.04 percentage of leukemic stem cells
|
|
Percentage of Leukemic Stem Cells Present in Bone Marrow Aspirate Samples, in This Patient Population
WCMC-013 (12 months)
|
24.2 percentage of leukemic stem cells
|
|
Percentage of Leukemic Stem Cells Present in Bone Marrow Aspirate Samples, in This Patient Population
WCMC-014 (1 month)
|
9.57 percentage of leukemic stem cells
|
|
Percentage of Leukemic Stem Cells Present in Bone Marrow Aspirate Samples, in This Patient Population
WCMC-014 (3 month)
|
14.6 percentage of leukemic stem cells
|
|
Percentage of Leukemic Stem Cells Present in Bone Marrow Aspirate Samples, in This Patient Population
WCMC-014 (12 months)
|
5.37 percentage of leukemic stem cells
|
|
Percentage of Leukemic Stem Cells Present in Bone Marrow Aspirate Samples, in This Patient Population
WCMC-015 (1 month)
|
10.9 percentage of leukemic stem cells
|
|
Percentage of Leukemic Stem Cells Present in Bone Marrow Aspirate Samples, in This Patient Population
WCMC-015 (3 months)
|
4.33 percentage of leukemic stem cells
|
|
Percentage of Leukemic Stem Cells Present in Bone Marrow Aspirate Samples, in This Patient Population
WCMC-017 (3 month)
|
13.7 percentage of leukemic stem cells
|
|
Percentage of Leukemic Stem Cells Present in Bone Marrow Aspirate Samples, in This Patient Population
WCMC-020 (3 months)
|
11.7 percentage of leukemic stem cells
|
|
Percentage of Leukemic Stem Cells Present in Bone Marrow Aspirate Samples, in This Patient Population
WCMC-020 (12 months)
|
19.3 percentage of leukemic stem cells
|
|
Percentage of Leukemic Stem Cells Present in Bone Marrow Aspirate Samples, in This Patient Population
WCMC-021 (3 month)
|
3.67 percentage of leukemic stem cells
|
Adverse Events
All Patients
Serious events: 5 serious events
Other events: 16 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
All Patients
n=16 participants at risk
Nilotinib at a dose of 300 mg P.O. twice a day (BID) daily
Nilotinib
|
|---|---|
|
Gastrointestinal disorders
Abdominal pain/vomiting/nausea
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Gastrointestinal disorders
Diarrheal illness
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
General disorders
Fever
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
General disorders
Intermittent left upper quadrant pain
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Pregnancy, puerperium and perinatal conditions
Pregnancy
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Skin and subcutaneous tissue disorders
Livido reticularis
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Gastrointestinal disorders
Small bowel obstruction
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
Other adverse events
| Measure |
All Patients
n=16 participants at risk
Nilotinib at a dose of 300 mg P.O. twice a day (BID) daily
Nilotinib
|
|---|---|
|
Skin and subcutaneous tissue disorders
Dry skin
|
12.5%
2/16 • Number of events 2 • Up to 5 years and 6 months.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
25.0%
4/16 • Number of events 4 • Up to 5 years and 6 months.
|
|
Investigations
Elevated TSH
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Gastrointestinal disorders
Epigastric pain
|
12.5%
2/16 • Number of events 2 • Up to 5 years and 6 months.
|
|
General disorders
Fall
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
General disorders
Fatigue
|
62.5%
10/16 • Number of events 14 • Up to 5 years and 6 months.
|
|
General disorders
Fever
|
18.8%
3/16 • Number of events 4 • Up to 5 years and 6 months.
|
|
Skin and subcutaneous tissue disorders
Folliculitis
|
12.5%
2/16 • Number of events 4 • Up to 5 years and 6 months.
|
|
Gastrointestinal disorders
Gastric reflux syndrome
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Gastrointestinal disorders
Gastritis
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Gastrointestinal disorders
Gastroenteritis
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
General disorders
Generalized pain
|
18.8%
3/16 • Number of events 3 • Up to 5 years and 6 months.
|
|
General disorders
Hair darkening
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
General disorders
Hair thinning
|
6.2%
1/16 • Number of events 2 • Up to 5 years and 6 months.
|
|
Gastrointestinal disorders
Heart burn
|
12.5%
2/16 • Number of events 2 • Up to 5 years and 6 months.
|
|
Investigations
Hematocrit decreased
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Investigations
Hemoglobin decrease
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Gastrointestinal disorders
Hemorrhoids
|
12.5%
2/16 • Number of events 2 • Up to 5 years and 6 months.
|
|
Vascular disorders
Hypertension
|
12.5%
2/16 • Number of events 2 • Up to 5 years and 6 months.
|
|
Investigations
Hypocalcemia
|
25.0%
4/16 • Number of events 12 • Up to 5 years and 6 months.
|
|
Investigations
Hypoalbunemia
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Investigations
Hypoglycemia
|
12.5%
2/16 • Number of events 2 • Up to 5 years and 6 months.
|
|
Investigations
Hyponatremia
|
12.5%
2/16 • Number of events 2 • Up to 5 years and 6 months.
|
|
Investigations
Hypophosphatemia
|
12.5%
2/16 • Number of events 2 • Up to 5 years and 6 months.
|
|
Psychiatric disorders
Insomnia
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Gastrointestinal disorders
Intermittent foul breath
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Investigations
Iron deficiency
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Infections and infestations
Kidney infection
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
General disorders
Hand/wrist pain
|
12.5%
2/16 • Number of events 2 • Up to 5 years and 6 months.
|
|
General disorders
Rib pain
|
12.5%
2/16 • Number of events 2 • Up to 5 years and 6 months.
|
|
General disorders
Lesion
|
12.5%
2/16 • Number of events 2 • Up to 5 years and 6 months.
|
|
Investigations
Leukocytosis
|
31.2%
5/16 • Number of events 5 • Up to 5 years and 6 months.
|
|
Investigations
Leukopenia
|
37.5%
6/16 • Number of events 11 • Up to 5 years and 6 months.
|
|
Investigations
Low MCV
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Investigations
Lipase increased
|
12.5%
2/16 • Number of events 3 • Up to 5 years and 6 months.
|
|
Investigations
Lymphocyte count decreased
|
18.8%
3/16 • Number of events 9 • Up to 5 years and 6 months.
|
|
Renal and urinary disorders
Mid renal insufficiency
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Reproductive system and breast disorders
Missed period
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Skin and subcutaneous tissue disorders
Moles
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Musculoskeletal and connective tissue disorders
Muscle cramp
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Gastrointestinal disorders
Nausea
|
18.8%
3/16 • Number of events 3 • Up to 5 years and 6 months.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neurofibroma
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Investigations
Neutropenia
|
31.2%
5/16 • Number of events 8 • Up to 5 years and 6 months.
|
|
General disorders
Night sweats
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Metabolism and nutrition disorders
Overweight
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
General disorders
Leg/Knee pain
|
18.8%
3/16 • Number of events 3 • Up to 5 years and 6 months.
|
|
Injury, poisoning and procedural complications
Pain at needle entry site
|
12.5%
2/16 • Number of events 2 • Up to 5 years and 6 months.
|
|
Renal and urinary disorders
Pain with urination
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
General disorders
Palpable spleen
|
12.5%
2/16 • Number of events 2 • Up to 5 years and 6 months.
|
|
Cardiac disorders
Palpitations
|
12.5%
2/16 • Number of events 6 • Up to 5 years and 6 months.
|
|
Eye disorders
Periorbital edema
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Investigations
Thrombocytopenia
|
62.5%
10/16 • Number of events 25 • Up to 5 years and 6 months.
|
|
Investigations
Platelet count increased
|
12.5%
2/16 • Number of events 2 • Up to 5 years and 6 months.
|
|
Skin and subcutaneous tissue disorders
Pruritis
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Nervous system disorders
Restless legs
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Musculoskeletal and connective tissue disorders
Broken/fractured arm
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
General disorders
Heel pain
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
General disorders
Neck pain
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Ear and labyrinth disorders
Ringing in ears
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Skin and subcutaneous tissue disorders
Scalp irritation/itching
|
12.5%
2/16 • Number of events 2 • Up to 5 years and 6 months.
|
|
Immune system disorders
Seasonal allergies
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Scalp nodule
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Skin and subcutaneous tissue disorders
Seborrheic Keratoses
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
General disorders
Sensation of bugs crawling on left shoulder
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Respiratory, thoracic and mediastinal disorders
Sore throat
|
25.0%
4/16 • Number of events 5 • Up to 5 years and 6 months.
|
|
General disorders
Splenic pain
|
6.2%
1/16 • Number of events 2 • Up to 5 years and 6 months.
|
|
General disorders
Stomach pain
|
12.5%
2/16 • Number of events 2 • Up to 5 years and 6 months.
|
|
Infections and infestations
Stomach virus
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Infections and infestations
Strep throat
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
General disorders
Swelling of upper lip
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Musculoskeletal and connective tissue disorders
Tenderness behind knees
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
General disorders
Toothache
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Infections and infestations
Upper respiratory infection/symptoms
|
18.8%
3/16 • Number of events 4 • Up to 5 years and 6 months.
|
|
Investigations
Uric acid elevated
|
18.8%
3/16 • Number of events 3 • Up to 5 years and 6 months.
|
|
Gastrointestinal disorders
Vomiting
|
12.5%
2/16 • Number of events 2 • Up to 5 years and 6 months.
|
|
Metabolism and nutrition disorders
Weight gain
|
12.5%
2/16 • Number of events 2 • Up to 5 years and 6 months.
|
|
Infections and infestations
Yeast infection
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Gastrointestinal disorders
Abdominal discomfort (pain or cramps)
|
31.2%
5/16 • Number of events 10 • Up to 5 years and 6 months.
|
|
Skin and subcutaneous tissue disorders
Acne
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Investigations
ALT elevated
|
25.0%
4/16 • Number of events 4 • Up to 5 years and 6 months.
|
|
Investigations
ALP elevated
|
31.2%
5/16 • Number of events 7 • Up to 5 years and 6 months.
|
|
Immune system disorders
Allergic rhinitis
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Investigations
Amylase increased
|
12.5%
2/16 • Number of events 3 • Up to 5 years and 6 months.
|
|
Investigations
Anemia
|
68.8%
11/16 • Number of events 18 • Up to 5 years and 6 months.
|
|
General disorders
Chest pain/tightness
|
31.2%
5/16 • Number of events 8 • Up to 5 years and 6 months.
|
|
Psychiatric disorders
Anxiety
|
12.5%
2/16 • Number of events 2 • Up to 5 years and 6 months.
|
|
Gastrointestinal disorders
Appetite change
|
12.5%
2/16 • Number of events 2 • Up to 5 years and 6 months.
|
|
Investigations
AST increased
|
18.8%
3/16 • Number of events 3 • Up to 5 years and 6 months.
|
|
General disorders
Back pain
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Investigations
Basophilia
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Nervous system disorders
Headaches
|
31.2%
5/16 • Number of events 6 • Up to 5 years and 6 months.
|
|
General disorders
Pain - knee
|
18.8%
3/16 • Number of events 3 • Up to 5 years and 6 months.
|
|
Investigations
Bilirubin elevated
|
12.5%
2/16 • Number of events 9 • Up to 5 years and 6 months.
|
|
Skin and subcutaneous tissue disorders
Rash
|
43.8%
7/16 • Number of events 16 • Up to 5 years and 6 months.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Breasts cysts
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Injury, poisoning and procedural complications
Bruising
|
12.5%
2/16 • Number of events 2 • Up to 5 years and 6 months.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
12.5%
2/16 • Number of events 3 • Up to 5 years and 6 months.
|
|
Cardiac disorders
Bradycardia
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Cardiac disorders
Coronary artery disease
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
General disorders
Cold/cold symptoms
|
25.0%
4/16 • Number of events 5 • Up to 5 years and 6 months.
|
|
General disorders
Chipped tooth
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Investigations
Elevated cholesterol
|
25.0%
4/16 • Number of events 4 • Up to 5 years and 6 months.
|
|
Investigations
Hyperglycemia
|
31.2%
5/16 • Number of events 19 • Up to 5 years and 6 months.
|
|
Investigations
Hyperlipidemia
|
18.8%
3/16 • Number of events 3 • Up to 5 years and 6 months.
|
|
General disorders
Nasal discharge
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Eye disorders
Conjunctiva injected
|
6.2%
1/16 • Number of events 2 • Up to 5 years and 6 months.
|
|
Gastrointestinal disorders
Constipation
|
25.0%
4/16 • Number of events 5 • Up to 5 years and 6 months.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
37.5%
6/16 • Number of events 8 • Up to 5 years and 6 months.
|
|
Infections and infestations
COVID/COVID Symptoms
|
12.5%
2/16 • Number of events 2 • Up to 5 years and 6 months.
|
|
General disorders
Diabetes
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Gastrointestinal disorders
Diarrhea
|
25.0%
4/16 • Number of events 6 • Up to 5 years and 6 months.
|
|
General disorders
Dizziness/Lightheadedness
|
12.5%
2/16 • Number of events 2 • Up to 5 years and 6 months.
|
|
General disorders
Dog bite
|
6.2%
1/16 • Number of events 1 • Up to 5 years and 6 months.
|
|
Eye disorders
Dry/Red eyes
|
31.2%
5/16 • Number of events 6 • Up to 5 years and 6 months.
|
|
General disorders
Dry mouth/perioral dryness
|
12.5%
2/16 • Number of events 2 • Up to 5 years and 6 months.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place