Trial Outcomes & Findings for Pilot Study of Erlotinib for the Treatment of Patients With de Novo Acute Myeloid Leukemia (NCT NCT01174043)
NCT ID: NCT01174043
Last Updated: 2023-10-13
Results Overview
The percent of patients were shown as having a partial remission or better based on definitions of response in AML. Partial remission includes a decrease of at least 50% in the percentage of blasts to 5% to 25% in the bone marrow aspirate. Complete remission includes presence of less than 5% blasts in an aspirate sample with marrow spicules and with a count of at least 200 nucleated cells. The percent and 95% exact confidence intervals will be calculated.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
11 participants
Primary outcome timeframe
3 months of treatment with erlotinib
Results posted on
2023-10-13
Participant Flow
This protocol was based on getting 14 patients. Due to the lack of response, the study was stopped at 11 patients for this pilot study.
Participant milestones
| Measure |
Erlotinib
Erlotinib: Erlotinib will be administered orally at 150 mg once a day, continuously. Each cycle will be 28 days and there will be no break between the cycles.
|
|---|---|
|
Overall Study
STARTED
|
11
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
11
|
Reasons for withdrawal
| Measure |
Erlotinib
Erlotinib: Erlotinib will be administered orally at 150 mg once a day, continuously. Each cycle will be 28 days and there will be no break between the cycles.
|
|---|---|
|
Overall Study
Disease progression
|
5
|
|
Overall Study
Adverse Event
|
3
|
|
Overall Study
Death
|
1
|
|
Overall Study
Withdrawal by Subject
|
2
|
Baseline Characteristics
Pilot Study of Erlotinib for the Treatment of Patients With de Novo Acute Myeloid Leukemia
Baseline characteristics by cohort
| Measure |
Erlotinib
n=11 Participants
Erlotinib: Erlotinib will be administered orally at 150 mg once a day, continuously. Each cycle will be 28 days and there will be no break between the cycles.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
1 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
10 Participants
n=5 Participants
|
|
Age, Continuous
|
76.8 years
STANDARD_DEVIATION 7.04 • n=5 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
11 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 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
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
11 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
|
PRIMARY outcome
Timeframe: 3 months of treatment with erlotinibPopulation: All patients enrolled and received treatment
The percent of patients were shown as having a partial remission or better based on definitions of response in AML. Partial remission includes a decrease of at least 50% in the percentage of blasts to 5% to 25% in the bone marrow aspirate. Complete remission includes presence of less than 5% blasts in an aspirate sample with marrow spicules and with a count of at least 200 nucleated cells. The percent and 95% exact confidence intervals will be calculated.
Outcome measures
| Measure |
Erlotinib
n=11 Participants
Erlotinib: Erlotinib will be administered orally at 150 mg once a day, continuously. Each cycle will be 28 days and there will be no break between the cycles.
|
|---|---|
|
Overall Response Rate (Defined as Partial Remission or Better) to 3 Months of Treatment With Erlotinib
|
0 percentage of participants
Interval 0.0 to 0.0
|
SECONDARY outcome
Timeframe: 1 year after treatment discontinuationPopulation: All patients enrolled and received treatment
The duration of response is from the time of response until failure or until the end of follow-up for the patients who received complete remission. Complete remission includes presence of less than 5% blasts in an aspirate sample with marrow spicules and with a count of at least 200 nucleated cells.
Outcome measures
| Measure |
Erlotinib
n=11 Participants
Erlotinib: Erlotinib will be administered orally at 150 mg once a day, continuously. Each cycle will be 28 days and there will be no break between the cycles.
|
|---|---|
|
Duration of Response (up to One Year Follow up) in Patients Who Achieve a Complete Remission
|
0 months
Standard Deviation 0
|
SECONDARY outcome
Timeframe: up to 15 monthsAdverse events will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE) version 3.0. Grading scale will be from 1 (mild) to 5 (causing death). This will determine the number of unique patients who had a treatment related (possible, probable or definite) adverse event that was graded 3 or greater.
Outcome measures
| Measure |
Erlotinib
n=11 Participants
Erlotinib: Erlotinib will be administered orally at 150 mg once a day, continuously. Each cycle will be 28 days and there will be no break between the cycles.
|
|---|---|
|
Treatment Related Adverse Events Grade 3 or Higher
|
0 participants
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Baseline; days 3, 4, 8, and 29 of course 1; and day 29 of courses 3, 6, 9, and 12Outcome measures
Outcome data not reported
Adverse Events
Erlotinib
Serious events: 5 serious events
Other events: 10 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Erlotinib
n=11 participants at risk
Erlotinib: Erlotinib will be administered orally at 150 mg once a day, continuously. Each cycle will be 28 days and there will be no break between the cycles.
|
|---|---|
|
Blood and lymphatic system disorders
FEBRILE NEUTROPENIA (FEVER OF UNKNOWN ORIGIN WITHOUT CLINICALLY OR MICROBIOLOGICALLY DOCUMENTED INFE
|
18.2%
2/11 • Up to 2 years
|
|
Cardiac disorders
SUPRAVENTRICULAR AND NODAL ARRHYTHMIA - ATRIAL FIBRILLATION
|
9.1%
1/11 • Up to 2 years
|
|
Gastrointestinal disorders
DIARRHEA
|
18.2%
2/11 • Up to 2 years
|
|
Hepatobiliary disorders
HEPATOBILIARY/PANCREAS - OTHER
|
9.1%
1/11 • Up to 2 years
|
|
Investigations
CREATININE
|
9.1%
1/11 • Up to 2 years
|
|
Metabolism and nutrition disorders
SODIUM, SERUM-LOW (HYPONATREMIA)
|
9.1%
1/11 • Up to 2 years
|
|
Metabolism and nutrition disorders
URIC ACID, SERUM-HIGH (HYPERURICEMIA)
|
9.1%
1/11 • Up to 2 years
|
|
Musculoskeletal and connective tissue disorders
MUSCLE WEAKNESS, GENERALIZED OR SPECIFIC AREA (NOT DUE TO NEUROPATHY) - EXTRAOCULAR
|
9.1%
1/11 • Up to 2 years
|
|
Renal and urinary disorders
RENAL FAILURE
|
18.2%
2/11 • Up to 2 years
|
|
Respiratory, thoracic and mediastinal disorders
HYPOXIA
|
9.1%
1/11 • Up to 2 years
|
|
Vascular disorders
HYPOTENSION
|
9.1%
1/11 • Up to 2 years
|
Other adverse events
| Measure |
Erlotinib
n=11 participants at risk
Erlotinib: Erlotinib will be administered orally at 150 mg once a day, continuously. Each cycle will be 28 days and there will be no break between the cycles.
|
|---|---|
|
Blood and lymphatic system disorders
BLOOD/BONE MARROW - OTHER
|
9.1%
1/11 • Up to 2 years
|
|
Blood and lymphatic system disorders
FEBRILE NEUTROPENIA (FEVER OF UNKNOWN ORIGIN WITHOUT CLINICALLY OR MICROBIOLOGICALLY DOCUMENTED INFE
|
18.2%
2/11 • Up to 2 years
|
|
Cardiac disorders
CARDIAC ARRHYTHMIA - OTHER
|
9.1%
1/11 • Up to 2 years
|
|
Cardiac disorders
CARDIAC GENERAL - OTHER
|
9.1%
1/11 • Up to 2 years
|
|
Cardiac disorders
PAIN - CARDIAC/HEART
|
9.1%
1/11 • Up to 2 years
|
|
Eye disorders
VISION-BLURRED VISION
|
9.1%
1/11 • Up to 2 years
|
|
Gastrointestinal disorders
DIARRHEA
|
36.4%
4/11 • Up to 2 years
|
|
Gastrointestinal disorders
DRY MOUTH/SALIVARY GLAND (XEROSTOMIA)
|
9.1%
1/11 • Up to 2 years
|
|
Gastrointestinal disorders
GASTROINTESTINAL - OTHER
|
18.2%
2/11 • Up to 2 years
|
|
Gastrointestinal disorders
MUCOSITIS/STOMATITIS (CLINICAL EXAM) - ORAL CAVITY
|
18.2%
2/11 • Up to 2 years
|
|
Gastrointestinal disorders
MUCOSITIS/STOMATITIS (FUNCTIONAL/SYMPTOMATIC) - ORAL CAVITY
|
9.1%
1/11 • Up to 2 years
|
|
Gastrointestinal disorders
NAUSEA
|
9.1%
1/11 • Up to 2 years
|
|
General disorders
EDEMA: LIMB
|
9.1%
1/11 • Up to 2 years
|
|
General disorders
FATIGUE (ASTHENIA, LETHARGY, MALAISE)
|
45.5%
5/11 • Up to 2 years
|
|
General disorders
FEVER (IN THE ABSENCE OF NEUTROPENIA, WHERE NEUTROPENIA IS DEFINED AS ANC <1.0 X 10E9/L)
|
18.2%
2/11 • Up to 2 years
|
|
General disorders
PAIN - OTHER
|
9.1%
1/11 • Up to 2 years
|
|
General disorders
RIGORS/CHILLS
|
9.1%
1/11 • Up to 2 years
|
|
General disorders
SWEATING (DIAPHORESIS)
|
9.1%
1/11 • Up to 2 years
|
|
Immune system disorders
ALLERGY/IMMUNOLOGY - OTHER
|
9.1%
1/11 • Up to 2 years
|
|
Infections and infestations
INFECTION WITH UNKNOWN ANC - LUNG (PNEUMONIA)
|
9.1%
1/11 • Up to 2 years
|
|
Injury, poisoning and procedural complications
BRUISING (IN ABSENCE OF GRADE 3 OR 4 THROMBOCYTOPENIA)
|
9.1%
1/11 • Up to 2 years
|
|
Investigations
METABOLIC/LABORATORY - OTHER
|
9.1%
1/11 • Up to 2 years
|
|
Investigations
PLATELETS
|
9.1%
1/11 • Up to 2 years
|
|
Investigations
WEIGHT LOSS
|
9.1%
1/11 • Up to 2 years
|
|
Metabolism and nutrition disorders
ANOREXIA
|
18.2%
2/11 • Up to 2 years
|
|
Metabolism and nutrition disorders
CALCIUM, SERUM-LOW (HYPOCALCEMIA)
|
18.2%
2/11 • Up to 2 years
|
|
Metabolism and nutrition disorders
GLUCOSE, SERUM-HIGH (HYPERGLYCEMIA)
|
9.1%
1/11 • Up to 2 years
|
|
Metabolism and nutrition disorders
MAGNESIUM, SERUM-LOW (HYPOMAGNESEMIA)
|
18.2%
2/11 • Up to 2 years
|
|
Metabolism and nutrition disorders
POTASSIUM, SERUM-LOW (HYPOKALEMIA)
|
27.3%
3/11 • Up to 2 years
|
|
Musculoskeletal and connective tissue disorders
FRACTURE
|
9.1%
1/11 • Up to 2 years
|
|
Musculoskeletal and connective tissue disorders
MUSCLE WEAKNESS, GENERALIZED OR SPECIFIC AREA (NOT DUE TO NEUROPATHY) - EXTRAOCULAR
|
9.1%
1/11 • Up to 2 years
|
|
Musculoskeletal and connective tissue disorders
MUSCLE WEAKNESS, GENERALIZED OR SPECIFIC AREA (NOT DUE TO NEUROPATHY) - WHOLE BODY/GENERALIZED
|
9.1%
1/11 • Up to 2 years
|
|
Musculoskeletal and connective tissue disorders
MUSCULOSKELETAL/SOFT TISSUE - OTHER
|
9.1%
1/11 • Up to 2 years
|
|
Musculoskeletal and connective tissue disorders
PAIN - BACK
|
9.1%
1/11 • Up to 2 years
|
|
Musculoskeletal and connective tissue disorders
PAIN - JOINT
|
9.1%
1/11 • Up to 2 years
|
|
Musculoskeletal and connective tissue disorders
PAIN - NECK
|
9.1%
1/11 • Up to 2 years
|
|
Nervous system disorders
DIZZINESS
|
18.2%
2/11 • Up to 2 years
|
|
Nervous system disorders
MOOD ALTERATION - AGITATION
|
9.1%
1/11 • Up to 2 years
|
|
Nervous system disorders
MOOD ALTERATION - ANXIETY
|
9.1%
1/11 • Up to 2 years
|
|
Nervous system disorders
NEUROLOGY - OTHER
|
18.2%
2/11 • Up to 2 years
|
|
Nervous system disorders
PAIN - HEAD/HEADACHE
|
18.2%
2/11 • Up to 2 years
|
|
Nervous system disorders
SEIZURE
|
9.1%
1/11 • Up to 2 years
|
|
Renal and urinary disorders
RENAL/GENITOURINARY - OTHER
|
9.1%
1/11 • Up to 2 years
|
|
Respiratory, thoracic and mediastinal disorders
BRONCHOSPASM, WHEEZING
|
9.1%
1/11 • Up to 2 years
|
|
Respiratory, thoracic and mediastinal disorders
COUGH
|
36.4%
4/11 • Up to 2 years
|
|
Respiratory, thoracic and mediastinal disorders
DYSPNEA (SHORTNESS OF BREATH)
|
45.5%
5/11 • Up to 2 years
|
|
Respiratory, thoracic and mediastinal disorders
HYPOXIA
|
9.1%
1/11 • Up to 2 years
|
|
Skin and subcutaneous tissue disorders
DERMATOLOGY/SKIN - OTHER
|
9.1%
1/11 • Up to 2 years
|
|
Skin and subcutaneous tissue disorders
RASH/DESQUAMATION
|
18.2%
2/11 • Up to 2 years
|
|
Skin and subcutaneous tissue disorders
RASH: ACNE/ACNEIFORM
|
9.1%
1/11 • Up to 2 years
|
|
Vascular disorders
HYPOTENSION
|
9.1%
1/11 • Up to 2 years
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place