Trial Outcomes & Findings for Erlotinib Study for Myelodysplastic Syndrome (MDS) (NCT NCT00977548)
NCT ID: NCT00977548
Last Updated: 2013-09-16
Results Overview
Best Response Categories: Marrow complete response (CR), Bone marrow: ≤ 5% myeloblasts and decrease by ≥ 50% over pretreatment; Hematological improvement (HI), Hgb increase by ≥ 1.5 g/dL, Absolute increase of ≥ 30 x 10\^9/L for patients starting with \> 20 x 10\^9/L, At least 100% increase and an absolute increase of \> 0.5 x 10\^9/L, as defined by the International Working Group (IWG) 2006 criteria.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
39 participants
Primary outcome timeframe
Up to 21 Months
Results posted on
2013-09-16
Participant Flow
Between September 2009 and January 2011, 39 patients signed consent at Moffitt Cancer Center.
4 of the initial 39 patients were found to be ineligible after signing informed consent.
Participant milestones
| Measure |
Erlotinib Treatment
Erlotinib was given as an oral 150 mg daily dose for 16 weeks. The dose was adjusted for diarrhea, rash and pulmonary toxicity.
|
|---|---|
|
Overall Study
STARTED
|
39
|
|
Overall Study
COMPLETED
|
26
|
|
Overall Study
NOT COMPLETED
|
13
|
Reasons for withdrawal
| Measure |
Erlotinib Treatment
Erlotinib was given as an oral 150 mg daily dose for 16 weeks. The dose was adjusted for diarrhea, rash and pulmonary toxicity.
|
|---|---|
|
Overall Study
Ineligible
|
4
|
|
Overall Study
Adverse Event
|
8
|
|
Overall Study
Withdrawal by Subject
|
1
|
Baseline Characteristics
Erlotinib Study for Myelodysplastic Syndrome (MDS)
Baseline characteristics by cohort
| Measure |
Erlotinib Treatment
n=35 Participants
Erlotinib was given as an oral 150 mg daily dose for 16 weeks. The dose was adjusted for diarrhea, rash and pulmonary toxicity.
|
|---|---|
|
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
|
34 Participants
n=5 Participants
|
|
Age Continuous
|
73 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
7 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
28 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
35 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to 21 MonthsPopulation: All evaluable participants. Nine patients were not evaluable for response (withdrew consent or off study due to adverse event before first evaluation).
Best Response Categories: Marrow complete response (CR), Bone marrow: ≤ 5% myeloblasts and decrease by ≥ 50% over pretreatment; Hematological improvement (HI), Hgb increase by ≥ 1.5 g/dL, Absolute increase of ≥ 30 x 10\^9/L for patients starting with \> 20 x 10\^9/L, At least 100% increase and an absolute increase of \> 0.5 x 10\^9/L, as defined by the International Working Group (IWG) 2006 criteria.
Outcome measures
| Measure |
Erlotinib Treatment
n=26 Participants
Erlotinib was given as an oral 150 mg daily dose for 16 weeks. The dose was adjusted for diarrhea, rash and pulmonary toxicity.
|
|---|---|
|
Combined Overall Response Rate (ORR)
Marrow Complete Response (CR)
|
3 participants
|
|
Combined Overall Response Rate (ORR)
Hematological Improvement (HI)
|
2 participants
|
|
Combined Overall Response Rate (ORR)
Combined Overall Response
|
5 participants
|
SECONDARY outcome
Timeframe: Up to 21 MonthsPopulation: All evaluable participants. Nine patients were not evaluable for response (withdrew consent or off study due to adverse event before first evaluation).
OS: The time from randomization until death from any cause. Kaplan-Meier estimates were used for secondary endpoint analysis.
Outcome measures
| Measure |
Erlotinib Treatment
n=26 Participants
Erlotinib was given as an oral 150 mg daily dose for 16 weeks. The dose was adjusted for diarrhea, rash and pulmonary toxicity.
|
|---|---|
|
Median Overall Survival (OS)
|
6.8 months
Interval 4.9 to 13.2
|
SECONDARY outcome
Timeframe: Up to 21 MonthsPopulation: All evaluable participants. Nine patients were not evaluable for response (withdrew consent or off study due to adverse event before first evaluation).
PFS: The time elapsed between treatment initiation and tumor progression or death from any cause. Kaplan-Meier estimates were used for secondary endpoint analysis. Disease Progression is defined using International Working Group (IWG) Response Criteria for MDS, as at least 50% decrement from maximum remission/response levels in granulocytes or platelets; reduction in hemoglobin (Hgb) concentration by ≥ 2 g/dL; transfusion dependence.
Outcome measures
| Measure |
Erlotinib Treatment
n=26 Participants
Erlotinib was given as an oral 150 mg daily dose for 16 weeks. The dose was adjusted for diarrhea, rash and pulmonary toxicity.
|
|---|---|
|
Median Progression Free Survival (PFS)
|
3.6 months
Interval 2.0 to 4.8
|
SECONDARY outcome
Timeframe: Up to 21 MonthsPopulation: All evaluable participants. Nine patients were not evaluable for response (withdrew consent or off study due to adverse event before first evaluation).
LFS: Survival without evidence of relapse at any time post-transplant. Kaplan-Meier estimates were used for secondary endpoint analysis.
Outcome measures
| Measure |
Erlotinib Treatment
n=26 Participants
Erlotinib was given as an oral 150 mg daily dose for 16 weeks. The dose was adjusted for diarrhea, rash and pulmonary toxicity.
|
|---|---|
|
Leukemia Free Survival (LFS)
|
5 months
Interval 3.4 to 7.3
|
Adverse Events
Erlotinib Treatment
Serious events: 24 serious events
Other events: 32 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Erlotinib Treatment
n=35 participants at risk
Erlotinib was given as an oral 150 mg daily dose for 16 weeks. The dose was adjusted for diarrhea, rash and pulmonary toxicity.
|
|---|---|
|
Blood and lymphatic system disorders
Hemoglobin
|
28.6%
10/35 • 1 year, 9 months
|
|
Blood and lymphatic system disorders
Neutrophils/granulocytes (ANC/AGC)
|
2.9%
1/35 • 1 year, 9 months
|
|
Blood and lymphatic system disorders
Platelets
|
14.3%
5/35 • 1 year, 9 months
|
|
Cardiac disorders
Cardiac Arrhythmia - Other
|
2.9%
1/35 • 1 year, 9 months
|
|
Cardiac disorders
Cardiac ischemia/infarction
|
2.9%
1/35 • 1 year, 9 months
|
|
Cardiac disorders
Hypotension
|
2.9%
1/35 • 1 year, 9 months
|
|
Blood and lymphatic system disorders
Coagulation - Other
|
2.9%
1/35 • 1 year, 9 months
|
|
General disorders
Fatigue (asthenia, lethargy, malaise)
|
11.4%
4/35 • 1 year, 9 months
|
|
General disorders
Fever (in the absence of neutropenia)
|
5.7%
2/35 • 1 year, 9 months
|
|
General disorders
Death not associated with CTCAE term - Death NOS
|
8.6%
3/35 • 1 year, 9 months
|
|
General disorders
Death not associated with CTCAE term - Disease progression NOS
|
5.7%
2/35 • 1 year, 9 months
|
|
General disorders
Death not associated with CTCAE term - Sudden death
|
2.9%
1/35 • 1 year, 9 months
|
|
Skin and subcutaneous tissue disorders
Rash: acne/acneiform
|
2.9%
1/35 • 1 year, 9 months
|
|
Gastrointestinal disorders
Anorexia
|
8.6%
3/35 • 1 year, 9 months
|
|
Gastrointestinal disorders
Diarrhea
|
14.3%
5/35 • 1 year, 9 months
|
|
Gastrointestinal disorders
Ileus, GI (functional obstruction of bowel, i.e., neuroconstipation)
|
2.9%
1/35 • 1 year, 9 months
|
|
Nervous system disorders
Hemorrhage, CNS
|
5.7%
2/35 • 1 year, 9 months
|
|
Gastrointestinal disorders
Hemorrhage, GI - Colon
|
5.7%
2/35 • 1 year, 9 months
|
|
Gastrointestinal disorders
Hemorrhage, GI - Lower GI NOS
|
2.9%
1/35 • 1 year, 9 months
|
|
General disorders
Hemorrhage/Bleeding - Other
|
2.9%
1/35 • 1 year, 9 months
|
|
Infections and infestations
Infection (documented clinically or microbiologically) with Grade 3 or 4 neutrophils - Abdomen NOS
|
2.9%
1/35 • 1 year, 9 months
|
|
Infections and infestations
Infection (documented clinically or microbiologically) with Grade 3 or 4 neutrophils -Blood
|
2.9%
1/35 • 1 year, 9 months
|
|
Infections and infestations
Infection (documented clinically or microbiologically) w/Grade 3 or 4 neutrophils - Lung (pneumonia)
|
5.7%
2/35 • 1 year, 9 months
|
|
Infections and infestations
Infection (documented clinically or microbiologically) with Grade 3 or 4 neutrophils - Mucosa
|
2.9%
1/35 • 1 year, 9 months
|
|
Infections and infestations
Infection - Other
|
22.9%
8/35 • 1 year, 9 months
|
|
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils - Blood
|
8.6%
3/35 • 1 year, 9 months
|
|
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils - Lung (pneumonia)
|
5.7%
2/35 • 1 year, 9 months
|
|
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils - Sinus
|
2.9%
1/35 • 1 year, 9 months
|
|
Infections and infestations
Infection with unknown ANC - Lung (pneumonia)
|
2.9%
1/35 • 1 year, 9 months
|
|
Metabolism and nutrition disorders
Creatinine
|
2.9%
1/35 • 1 year, 9 months
|
|
Metabolism and nutrition disorders
Sodium, serum-high (hypernatremia)
|
2.9%
1/35 • 1 year, 9 months
|
|
Metabolism and nutrition disorders
Uric acid, serum-high (hyperuricemia)
|
2.9%
1/35 • 1 year, 9 months
|
|
Psychiatric disorders
Mental status
|
2.9%
1/35 • 1 year, 9 months
|
|
Psychiatric disorders
Mood alteration - Depression
|
2.9%
1/35 • 1 year, 9 months
|
|
General disorders
Neurology - Other
|
5.7%
2/35 • 1 year, 9 months
|
|
General disorders
Syncope (fainting)
|
2.9%
1/35 • 1 year, 9 months
|
|
General disorders
Pain - Abdomen NOS
|
2.9%
1/35 • 1 year, 9 months
|
|
General disorders
Pain - Back
|
2.9%
1/35 • 1 year, 9 months
|
|
General disorders
Pain - Chest wall
|
2.9%
1/35 • 1 year, 9 months
|
|
General disorders
Pain - Head/headache
|
2.9%
1/35 • 1 year, 9 months
|
|
General disorders
Pain - Other
|
2.9%
1/35 • 1 year, 9 months
|
|
General disorders
Pain - Throat/pharynx/larynx
|
2.9%
1/35 • 1 year, 9 months
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
5.7%
2/35 • 1 year, 9 months
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea (shortness of breath)
|
17.1%
6/35 • 1 year, 9 months
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary/Upper Respiratory - Other
|
2.9%
1/35 • 1 year, 9 months
|
|
Renal and urinary disorders
Renal failure
|
8.6%
3/35 • 1 year, 9 months
|
|
Vascular disorders
Thrombosis/embolism (vascular access-related)
|
2.9%
1/35 • 1 year, 9 months
|
|
Vascular disorders
Thrombosis/thrombus/embolism
|
2.9%
1/35 • 1 year, 9 months
|
|
Vascular disorders
Vascular - Other
|
2.9%
1/35 • 1 year, 9 months
|
Other adverse events
| Measure |
Erlotinib Treatment
n=35 participants at risk
Erlotinib was given as an oral 150 mg daily dose for 16 weeks. The dose was adjusted for diarrhea, rash and pulmonary toxicity.
|
|---|---|
|
Gastrointestinal disorders
Diarrhea
|
65.7%
23/35 • 1 year, 9 months
|
|
Gastrointestinal disorders
Anorexia
|
40.0%
14/35 • 1 year, 9 months
|
|
Gastrointestinal disorders
Nausea
|
17.1%
6/35 • 1 year, 9 months
|
|
Gastrointestinal disorders
Gastrointestinal - Other
|
8.6%
3/35 • 1 year, 9 months
|
|
Gastrointestinal disorders
Vomiting
|
8.6%
3/35 • 1 year, 9 months
|
|
Gastrointestinal disorders
Dry mouth/salivary gland (xerostomia)
|
5.7%
2/35 • 1 year, 9 months
|
|
Gastrointestinal disorders
Hemorrhoids
|
5.7%
2/35 • 1 year, 9 months
|
|
Skin and subcutaneous tissue disorders
Pruritus/itching
|
45.7%
16/35 • 1 year, 9 months
|
|
Skin and subcutaneous tissue disorders
Rash: acne/acneiform
|
42.9%
15/35 • 1 year, 9 months
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
25.7%
9/35 • 1 year, 9 months
|
|
Skin and subcutaneous tissue disorders
Dermatology/Skin - Other
|
22.9%
8/35 • 1 year, 9 months
|
|
Skin and subcutaneous tissue disorders
Rash/desquamation
|
14.3%
5/35 • 1 year, 9 months
|
|
Skin and subcutaneous tissue disorders
Rash: erythema multiforme (e.g., Stevens-Johnson syndrome, toxic epidermal necrolysis)
|
5.7%
2/35 • 1 year, 9 months
|
|
Skin and subcutaneous tissue disorders
Ulceration
|
5.7%
2/35 • 1 year, 9 months
|
|
General disorders
Fatigue (Asthenia, lethargy, malaise)
|
48.6%
17/35 • 1 year, 9 months
|
|
General disorders
Fever (in the absence of neutropenia, where neutropenia is defined as ANC <1.0 x 10^9/L)
|
8.6%
3/35 • 1 year, 9 months
|
|
General disorders
Sweating (diaphoresis)
|
5.7%
2/35 • 1 year, 9 months
|
|
General disorders
Weight loss
|
5.7%
2/35 • 1 year, 9 months
|
|
General disorders
Hemorrhage/Bleeding - Other
|
17.1%
6/35 • 1 year, 9 months
|
|
Respiratory, thoracic and mediastinal disorders
Hemorrhage, pulmonary/upper respiratory - Nose
|
11.4%
4/35 • 1 year, 9 months
|
|
Gastrointestinal disorders
Hemorrhage, GI - Colon
|
5.7%
2/35 • 1 year, 9 months
|
|
Gastrointestinal disorders
Hemorrhage, GI - Lower GI NOS
|
5.7%
2/35 • 1 year, 9 months
|
|
Infections and infestations
Infection - Other
|
5.7%
2/35 • 1 year, 9 months
|
|
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils - Lung (pneumonia)
|
5.7%
2/35 • 1 year, 9 months
|
|
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils - Blood
|
5.7%
2/35 • 1 year, 9 months
|
|
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils - Eye NOS
|
5.7%
2/35 • 1 year, 9 months
|
|
General disorders
Pain - Chest/thorax NOS
|
5.7%
2/35 • 1 year, 9 months
|
|
General disorders
Pain - Other
|
5.7%
2/35 • 1 year, 9 months
|
|
General disorders
Pain - Scalp
|
5.7%
2/35 • 1 year, 9 months
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
17.1%
6/35 • 1 year, 9 months
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea (shortness of breath)
|
20.0%
7/35 • 1 year, 9 months
|
|
General disorders
Dizziness
|
11.4%
4/35 • 1 year, 9 months
|
|
Blood and lymphatic system disorders
Platelets
|
11.4%
4/35 • 1 year, 9 months
|
|
Blood and lymphatic system disorders
Hemoglobin
|
5.7%
2/35 • 1 year, 9 months
|
|
Blood and lymphatic system disorders
Neutrophils/granulocytes (ANC/AGC)
|
5.7%
2/35 • 1 year, 9 months
|
|
Blood and lymphatic system disorders
Edema: limb
|
5.7%
2/35 • 1 year, 9 months
|
|
Blood and lymphatic system disorders
Lymphatics - Other
|
5.7%
2/35 • 1 year, 9 months
|
|
Eye disorders
Dry eye syndrome
|
5.7%
2/35 • 1 year, 9 months
|
Additional Information
Rami Komrokji, Principal Investigator
H. Lee Moffitt Cancer Center and Research Institute
Phone: 813-745-4692
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place