Trial Outcomes & Findings for Cisplatin, Paclitaxel, and Everolimus in Treating Patients With Metastatic Breast Cancer (NCT NCT01031446)

NCT ID: NCT01031446

Last Updated: 2018-08-31

Results Overview

The recommended dose for the Phase II trial will be the most prevalent dose delivered per week in Phase I that allows for safe and feasible administration of the medications.The maximum tolerated dose (MTD) is defined as the dose preceding that at which 2 or more of 3 patients experience dose-limiting toxicity (DLT) during the initial cycle of therapy. DLTs include Common Toxicity Criteria (CTC) Grade 4 neutropenia (absolute neutrophil count \[ANC\] \< 0.5 x 10 9/L for \> 5 days), febrile neutropenia (ANC \< 1.0 x 10 0/L with fever \> 38.5 degrees Centigrade) or documented infection associated with Grade 3-4 neutropenia, CTC Grade 4 thrombocytopenia \< 25 x 10 9/L or CTC Grade 3 \< 50-25 x 10 9/L thrombocytopenia with bleeding, and Grade 3-4 non-hematologic toxicity despite symptomatic therapy.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

55 participants

Primary outcome timeframe

at 8 weeks

Results posted on

2018-08-31

Participant Flow

This study opened to accrual in October 2009 and ran to June 2011.

Fifty-seven patients consented for this study, two were determined ineligible to participate.

Participant milestones

Participant milestones
Measure
RAD001 and Cisplatin and Pacletaxel
RAD001 (Everolimus) by mouth once a day. Cisplatin intravenously (IV) weekly for 3 weeks, then 1 week of rest; paclitaxel IV weekly for 3 weeks, then 1 week of rest. One cycle = 4 weeks. All patients began at the same dose level of the study drugs with no de-escalation of dose, thus results for Phase I and II were combined
Overall Study
STARTED
55
Overall Study
COMPLETED
0
Overall Study
NOT COMPLETED
55

Reasons for withdrawal

Reasons for withdrawal
Measure
RAD001 and Cisplatin and Pacletaxel
RAD001 (Everolimus) by mouth once a day. Cisplatin intravenously (IV) weekly for 3 weeks, then 1 week of rest; paclitaxel IV weekly for 3 weeks, then 1 week of rest. One cycle = 4 weeks. All patients began at the same dose level of the study drugs with no de-escalation of dose, thus results for Phase I and II were combined
Overall Study
Disease Progression
38
Overall Study
Adverse Event
9
Overall Study
Physician Decision
4
Overall Study
Withdrawal by Subject
3
Overall Study
Still on treatment
1

Baseline Characteristics

Cisplatin, Paclitaxel, and Everolimus in Treating Patients With Metastatic Breast Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
RAD001 Cisplatin Paclitaxel
n=55 Participants
RAD001 (Everolimus) by mouth once a day. Cisplatin intravenously (IV) weekly for 3 weeks, then 1 week of rest; paclitaxel IV weekly for 3 weeks, then 1 week of rest. One cycle = 4 weeks.
Age, Categorical
<=18 years
0 Participants
n=93 Participants
Age, Categorical
Between 18 and 65 years
47 Participants
n=93 Participants
Age, Categorical
>=65 years
8 Participants
n=93 Participants
Age, Continuous
52 years
STANDARD_DEVIATION 11 • n=93 Participants
Sex: Female, Male
Female
54 Participants
n=93 Participants
Sex: Female, Male
Male
1 Participants
n=93 Participants
Region of Enrollment
United States
55 participants
n=93 Participants

PRIMARY outcome

Timeframe: at 8 weeks

Population: Patients enrolled to determine the safety profile and recommended doses of cisplatin + paclitaxel + everolimus (RAD001) in women with metastatic breast cancer

The recommended dose for the Phase II trial will be the most prevalent dose delivered per week in Phase I that allows for safe and feasible administration of the medications.The maximum tolerated dose (MTD) is defined as the dose preceding that at which 2 or more of 3 patients experience dose-limiting toxicity (DLT) during the initial cycle of therapy. DLTs include Common Toxicity Criteria (CTC) Grade 4 neutropenia (absolute neutrophil count \[ANC\] \< 0.5 x 10 9/L for \> 5 days), febrile neutropenia (ANC \< 1.0 x 10 0/L with fever \> 38.5 degrees Centigrade) or documented infection associated with Grade 3-4 neutropenia, CTC Grade 4 thrombocytopenia \< 25 x 10 9/L or CTC Grade 3 \< 50-25 x 10 9/L thrombocytopenia with bleeding, and Grade 3-4 non-hematologic toxicity despite symptomatic therapy.

Outcome measures

Outcome measures
Measure
RAD001 and Cisplatin and Paclitazel
n=3 Participants
Cisplatin intravenously (IV) weekly for 3 weeks, then 1 week of rest; paclitaxel IV weekly for 3 weeks, then 1 week of rest. Everolimus (RAD001) po daily. One cycle = 4 weeks
Maximum Feasible Dose in Milligrams Per Meter Squared of Body Surface Area (mg/m2) of Cisplatin and Paclitaxel for Women With Metastatic Breast Cancer
Cisplatin
25 mg/m2
Maximum Feasible Dose in Milligrams Per Meter Squared of Body Surface Area (mg/m2) of Cisplatin and Paclitaxel for Women With Metastatic Breast Cancer
Paclitaxel
80 mg/m2

PRIMARY outcome

Timeframe: at 8 weeks

Population: Patients enrolled to determine the safety profile and recommended dose of cisplatin + RAD001 + paclitaxel in women with metastatic breast cancer

The recommended dose for the Phase II trial will be the most prevalent dose delivered per day in Phase I that allows for safe and feasible administration the medication. The MTD is defined as the dose preceding that at which 2 or more of 3 patients experience dose-limiting toxicity (DLT) during the initial cycle of therapy. DLTs include Common Toxicity Criteria (CTC) Grade 4 neutropenia (absolute neutrophil count \[ANC\] \< 0.5 x 10 9/L for \> 5 days), febrile neutropenia (ANC \< 1.0 x 10 0/L with fever \> 38.5 degrees Centigrade) or documented infection associated with Grade 3-4 neutropenia, CTC Grade 4 thrombocytopenia \< 25 x 10 9/L or CTC Grade 3 \< 50-25 x 10 9/L thrombocytopenia with bleeding, and Grade 3-4 non-hematologic toxicity despite symptomatic therapy

Outcome measures

Outcome measures
Measure
RAD001 and Cisplatin and Paclitazel
n=3 Participants
Cisplatin intravenously (IV) weekly for 3 weeks, then 1 week of rest; paclitaxel IV weekly for 3 weeks, then 1 week of rest. Everolimus (RAD001) po daily. One cycle = 4 weeks
Maximum Feasible Dose in mg of RAD001 (Everolimus)for Women With Metastatic Breast Cancer
5 mg

PRIMARY outcome

Timeframe: at 6 months

Population: Patient who received the study drugs. Four patients were not available for measurement of progression at 6 months: toxicity (3), withdrew after beginning treatment (1)

Patients who had not experienced disease progression and who were alive at 6 months after study entry

Outcome measures

Outcome measures
Measure
RAD001 and Cisplatin and Paclitazel
n=51 Participants
Cisplatin intravenously (IV) weekly for 3 weeks, then 1 week of rest; paclitaxel IV weekly for 3 weeks, then 1 week of rest. Everolimus (RAD001) po daily. One cycle = 4 weeks
Patients With Progression-free Survival
21 participants
Interval 0.0 to 23.0

SECONDARY outcome

Timeframe: every 12 weeks

Population: Patients for whom an overall response could be measured. Four patients were not evaluable due to: withdrew after beginning treatment (1) and not evaluable due to only 1 cycle of treatment (3).

Per Response Evaluation Criteria in Solid Tumor (RECIST) criteria v. 1.0: measurable lesions: complete response (CR) disappearance of target lesions, partial response (PR) \> 30% decrease in the sum of the longest diameter (LD) of target lesions, progressive disease (PD) \> 20% increase in the sum of the LD of target lesions or appearance of new lesions, stable disease (SD) neither sufficient decrease nor increase of the sum of smallest sum of the LD of target lesions

Outcome measures

Outcome measures
Measure
RAD001 and Cisplatin and Paclitazel
n=51 Participants
Cisplatin intravenously (IV) weekly for 3 weeks, then 1 week of rest; paclitaxel IV weekly for 3 weeks, then 1 week of rest. Everolimus (RAD001) po daily. One cycle = 4 weeks
Patients With Overall Response
Complete Response
1 participants
Patients With Overall Response
Partial Response
12 participants
Patients With Overall Response
Progressive Disease
11 participants
Patients With Overall Response
Stable Disease
27 participants

SECONDARY outcome

Timeframe: Up to 64 weeks

Population: Patients who were available for determination of duration of time to progressive disease. Time to progression is unknown for 8 Phase II patients due to: on-treatment (1), toxicity (3), withdrew after beginning treatment (1), and no progression (3)

Duration in months from date on-study to date patient exhibited progressive disease

Outcome measures

Outcome measures
Measure
RAD001 and Cisplatin and Paclitazel
n=47 Participants
Cisplatin intravenously (IV) weekly for 3 weeks, then 1 week of rest; paclitaxel IV weekly for 3 weeks, then 1 week of rest. Everolimus (RAD001) po daily. One cycle = 4 weeks
Time to Progression
4.0 months
Interval 0.4 to 23.0

SECONDARY outcome

Timeframe: Up to 64 weeks

Population: Patients who are negative for estrogen, progesterone and HER2 receptors. Time to progression was not determined for 5 patients in this group: toxicity (2), withdrew after beginning treatment (1), no progression (1), and on-treatment (1).

Median duration in months from on-study to disease progression in patients with metastatic basal-like breast cancer. All patients with basal-like breast cancer are negative for estrogen, progesterone, and human epidermal growth factor (HER2) receptors.

Outcome measures

Outcome measures
Measure
RAD001 and Cisplatin and Paclitazel
n=31 Participants
Cisplatin intravenously (IV) weekly for 3 weeks, then 1 week of rest; paclitaxel IV weekly for 3 weeks, then 1 week of rest. Everolimus (RAD001) po daily. One cycle = 4 weeks
Time to Progression in Patients With Metastatic Basal-like Breast Cancer.
4.0 months
Interval 0.4 to 23.0

Adverse Events

RAD001 and Cisplatin and Paclitaxel

Serious events: 9 serious events
Other events: 55 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
RAD001 and Cisplatin and Paclitaxel
n=55 participants at risk
RAD001 (Everolimus) by mouth once a day. Cisplatin intravenously (IV) weekly for 3 weeks, then 1 week of rest; paclitaxel IV weekly for 3 weeks, then 1 week of rest. One cycle = 4 weeks.
Gastrointestinal disorders
pain-abdomen
5.5%
3/55 • Number of events 6
General disorders
death not associated with CTCAE term-disease progression NOS
5.5%
3/55 • Number of events 3
Infections and infestations
Left maxillary odontogenic abscess and cellulitis
1.8%
1/55 • Number of events 1
Metabolism and nutrition disorders
glucose, serum-low (hypoglycemia)
1.8%
1/55 • Number of events 2
Musculoskeletal and connective tissue disorders
pain-back
3.6%
2/55 • Number of events 3
Blood and lymphatic system disorders
neutrophils
3.6%
2/55 • Number of events 4
Blood and lymphatic system disorders
leukocytes
1.8%
1/55 • Number of events 2
Blood and lymphatic system disorders
platelets
1.8%
1/55 • Number of events 2
Hepatobiliary disorders
cholecystitis
1.8%
1/55 • Number of events 2
Respiratory, thoracic and mediastinal disorders
malignant pleural effusion
1.8%
1/55 • Number of events 2
Respiratory, thoracic and mediastinal disorders
dyspnea
9.1%
5/55 • Number of events 6
Infections and infestations
infection with unknown ANC-mucosititis
1.8%
1/55 • Number of events 1
Infections and infestations
infection with unknown ANC-wound, methicillin-resistant Staphylococcus aureus
1.8%
1/55 • Number of events 1
Blood and lymphatic system disorders
febrile neutropenia, ANC < 1.0 x 10e9L, fever ≥ 38.5 degrees Celsius
1.8%
1/55 • Number of events 1
General disorders
fever in absence of neutropenia w/ neutropenia defined as ANC < 1.0 x 10e9L
3.6%
2/55 • Number of events 4
Gastrointestinal disorders
diarrhea
1.8%
1/55 • Number of events 1
Gastrointestinal disorders
nausea
5.5%
3/55 • Number of events 3
Gastrointestinal disorders
vomiting
5.5%
3/55 • Number of events 3
Vascular disorders
thromboembolism (vascular access-related)
1.8%
1/55 • Number of events 1
Gastrointestinal disorders
enteritis, small bowel
1.8%
1/55 • Number of events 1
General disorders
edema, facial
1.8%
1/55 • Number of events 1
Renal and urinary disorders
infection with unknown ANC-urinary tract NOS
1.8%
1/55 • Number of events 1
Metabolism and nutrition disorders
calcium, serum-high (hypercalcemia)
1.8%
1/55 • Number of events 1
Nervous system disorders
hydrocephalus
1.8%
1/55 • Number of events 1
Nervous system disorders
pain-headache
1.8%
1/55 • Number of events 1
Cardiac disorders
ventricular tachycardia
1.8%
1/55 • Number of events 1
Respiratory, thoracic and mediastinal disorders
pain-right-sided thorax (non-cardiac)
1.8%
1/55 • Number of events 1
Respiratory, thoracic and mediastinal disorders
cough
1.8%
1/55 • Number of events 1
Respiratory, thoracic and mediastinal disorders
pneumonia
1.8%
1/55 • Number of events 1
Blood and lymphatic system disorders
anemia
3.6%
2/55 • Number of events 3
Blood and lymphatic system disorders
thrombocytopenia
3.6%
2/55 • Number of events 3

Other adverse events

Other adverse events
Measure
RAD001 and Cisplatin and Paclitaxel
n=55 participants at risk
RAD001 (Everolimus) by mouth once a day. Cisplatin intravenously (IV) weekly for 3 weeks, then 1 week of rest; paclitaxel IV weekly for 3 weeks, then 1 week of rest. One cycle = 4 weeks.
Metabolism and nutrition disorders
albumin, serum-low
12.7%
7/55 • Number of events 8
Investigations
alkaline phosphatase
41.8%
23/55 • Number of events 51
Immune system disorders
allergic reaction\hypersensitivity (including drug fever)
5.5%
3/55 • Number of events 4
Immune system disorders
allergy/immunology-other
5.5%
3/55 • Number of events 3
Metabolism and nutrition disorders
ALT, SGPT (serum glutamic pyruvic transaminase)
40.0%
22/55 • Number of events 44
Metabolism and nutrition disorders
anorexia
21.8%
12/55 • Number of events 18
Metabolism and nutrition disorders
AST, SGOT (serum glutamic oxaloacetic transaminase)
25.5%
14/55 • Number of events 18
Metabolism and nutrition disorders
calcium, serum-high (hypercalcemia)
9.1%
5/55 • Number of events 7
Investigations
cholesterol, serum-high (hypercholesteremia
30.9%
17/55 • Number of events 26
Gastrointestinal disorders
constipation
32.7%
18/55 • Number of events 21
General disorders
constitutional symptoms-other
34.5%
19/55 • Number of events 162
Respiratory, thoracic and mediastinal disorders
cough
20.0%
11/55 • Number of events 12
Investigations
creatinine
7.3%
4/55 • Number of events 9
Skin and subcutaneous tissue disorders
dermatology/skin-other
23.6%
13/55 • Number of events 21
Gastrointestinal disorders
diarrhea
36.4%
20/55 • Number of events 28
Gastrointestinal disorders
dizziness
10.9%
6/55 • Number of events 6
Respiratory, thoracic and mediastinal disorders
dyspnea
18.2%
10/55 • Number of events 10
General disorders
edema limb
20.0%
11/55 • Number of events 17
General disorders
fatigue
58.2%
32/55 • Number of events 45
Gastrointestinal disorders
gastrointestinal-other
12.7%
7/55 • Number of events 9
Metabolism and nutrition disorders
glucose, serum-high (hyperglycemia)
60.0%
33/55 • Number of events 76
Skin and subcutaneous tissue disorders
hair loss/alopecia (scalp or body)
14.5%
8/55 • Number of events 9
Gastrointestinal disorders
heartburn/dyspepsia
5.5%
3/55 • Number of events 3
Blood and lymphatic system disorders
hemoglobin
94.5%
52/55 • Number of events 182
Respiratory, thoracic and mediastinal disorders
hemorrhage, pumonary/upper respiratory-nose
9.1%
5/55 • Number of events 5
Infections and infestations
infection-other
20.0%
11/55 • Number of events 11
Renal and urinary disorders
infection with normal ANC or grade 1 or 2 neutrophils-urinary tract NOS
5.5%
3/55 • Number of events 3
Psychiatric disorders
insomnia
16.4%
9/55 • Number of events 11
Blood and lymphatic system disorders
leukocytes (total WBC)
60.0%
33/55 • Number of events 141
Gastrointestinal disorders
nausea
61.8%
34/55 • Number of events 44
Nervous system disorders
neurology-other
21.8%
12/55 • Number of events 28
Nervous system disorders
neuropathy-sensory
29.1%
16/55 • Number of events 29
Blood and lymphatic system disorders
neutrophils/granulocytes (ANC/AGC)
50.9%
28/55 • Number of events 77
Musculoskeletal and connective tissue disorders
pain back
5.5%
3/55 • Number of events 4
Reproductive system and breast disorders
pain-breast
5.5%
3/55 • Number of events 4
Musculoskeletal and connective tissue disorders
pain-chest wall
5.5%
3/55 • Number of events 3
Musculoskeletal and connective tissue disorders
pain-extremity-limb
7.3%
4/55 • Number of events 6
Nervous system disorders
pain-head/headache
20.0%
11/55 • Number of events 12
General disorders
pain-other
40.0%
22/55 • Number of events 34
Blood and lymphatic system disorders
platelets
70.9%
39/55 • Number of events 85
Metabolism and nutrition disorders
potassium-serum-low (hypokalemia)
36.4%
20/55 • Number of events 33
Respiratory, thoracic and mediastinal disorders
pulmonary/upper respiratory-other
12.7%
7/55 • Number of events 9
Skin and subcutaneous tissue disorders
rash acne/acneiform
12.7%
7/55 • Number of events 8
Metabolism and nutrition disorders
sodium, serum-low (hyponatremia)
25.5%
14/55 • Number of events 25
Nervous system disorders
taste alteration (dysgeusia)
10.9%
6/55 • Number of events 7
Ear and labyrinth disorders
tinnitus
5.5%
3/55 • Number of events 3
Metabolism and nutrition disorders
triglyceride, serum-high (hypertriglyceridemia)
27.3%
15/55 • Number of events 17
Gastrointestinal disorders
vomiting
21.8%
12/55 • Number of events 18
Musculoskeletal and connective tissue disorders
pain-joint
5.5%
3/55 • Number of events 3
Metabolism and nutrition disorders
magnesium, serum-low (hypomagnesemia)
15.4%
8/52 • Number of events 11
Metabolism and nutrition disorders
metabolic/laboratory-other
34.5%
19/55 • Number of events 145
Blood and lymphatic system disorders
blood/bone marrow-other
7.3%
4/55 • Number of events 10
Metabolism and nutrition disorders
calcium, serum-low (hypocalcemia)
25.5%
14/55 • Number of events 30
Metabolism and nutrition disorders
potassium, serum-high (hyperkalemia)
5.5%
3/55 • Number of events 4
Gastrointestinal disorders
mucositis/stomatitis (clinical exam)-oral cavity
18.2%
10/55 • Number of events 12
Gastrointestinal disorders
mucositis/stomatitis (functional/symptomatic)-oral cavity
10.9%
6/55 • Number of events 6
Skin and subcutaneous tissue disorders
nail changes
7.3%
4/55 • Number of events 4
Skin and subcutaneous tissue disorders
pruritis/itching
5.5%
3/55 • Number of events 3
Skin and subcutaneous tissue disorders
rash/desquamation
5.5%
3/55 • Number of events 4
Psychiatric disorders
depression
5.5%
3/55 • Number of events 3
Infections and infestations
infection with normal ANC or Grade 1 or 2 neutrophils-nails
5.5%
3/55 • Number of events 4
Infections and infestations
infection with normal ANC or Grade 1 or 2 neutrophils-upper airway NOS
9.1%
5/55 • Number of events 6
Blood and lymphatic system disorders
lymphatics-other
5.5%
3/55 • Number of events 3
Musculoskeletal and connective tissue disorders
Pain-neck
5.5%
3/55 • Number of events 4
Gastrointestinal disorders
pain-abdomen NOS
7.3%
4/55 • Number of events 5

Additional Information

Ingrid A. Mayer, MD

Vanderbilt-Ingram Cancer Center

Phone: 615-936-2033

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place