Trial Outcomes & Findings for A Trial of Everolimis in Patients With Advanced Renal Cell Carcinoma. (NCT NCT01206764)
NCT ID: NCT01206764
Last Updated: 2019-06-24
Results Overview
the time from the date of the start of RAD001 treatment to the date of the first documented disease progression or death due to any cause
Recruitment status
COMPLETED
Study phase
PHASE4
Target enrollment
143 participants
Primary outcome timeframe
Approximately 4 years
Results posted on
2019-06-24
Participant Flow
Participant milestones
| Measure |
RAD001
Two 5 mg RAD001 tablets
|
|---|---|
|
Overall Study
STARTED
|
142
|
|
Overall Study
COMPLETED
|
4
|
|
Overall Study
NOT COMPLETED
|
138
|
Reasons for withdrawal
| Measure |
RAD001
Two 5 mg RAD001 tablets
|
|---|---|
|
Overall Study
Adverse Event
|
20
|
|
Overall Study
Abnormal laboratory value
|
2
|
|
Overall Study
Abnormal test procedure result
|
1
|
|
Overall Study
Patient withdrew consent
|
5
|
|
Overall Study
Lost to Follow-up
|
6
|
|
Overall Study
Death
|
11
|
|
Overall Study
New cancer therapy
|
2
|
|
Overall Study
Disease progression
|
88
|
|
Overall Study
Protocol deviation
|
3
|
Baseline Characteristics
A Trial of Everolimis in Patients With Advanced Renal Cell Carcinoma.
Baseline characteristics by cohort
| Measure |
RAD001
n=142 Participants
Two 5 mg RAD001 tablets
|
|---|---|
|
Age, Continuous
|
55.5 Years
STANDARD_DEVIATION 12.63 • n=5 Participants
|
|
Sex: Female, Male
Female
|
55 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
87 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
42 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
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
71 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
27 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Approximately 4 yearsPopulation: The Intent-to-Treat population (ITT population) consisted of all patients treated with RAD001.
the time from the date of the start of RAD001 treatment to the date of the first documented disease progression or death due to any cause
Outcome measures
| Measure |
RAD001
n=142 Participants
Two 5 mg RAD001 tablets
|
|---|---|
|
PFS (Progression-Free Survival)
|
27.71 Weeks
Interval 21.86 to 35.29
|
SECONDARY outcome
Timeframe: Approximately 4 yearsPopulation: The Intent-to-Treat population (ITT population) consisted of all patients treated with RAD001.
The disease control rate was based on the data as per local radiological review following the RECIST criteria. The disease control rate is defined as the proportion of patients with CR, PR, or SD and was summarized in terms of percentage with 95% exact Clopper-Pearson CIs
Outcome measures
| Measure |
RAD001
n=142 Participants
Two 5 mg RAD001 tablets
|
|---|---|
|
Disease Control Rate (Stable Disease [SD] + Partial Response [PR] + Complete Response [CR]);
|
74.6 Percentage of Participants
Interval 66.7 to 81.6
|
SECONDARY outcome
Timeframe: Approximately 4 yearsPopulation: The Intent-to-Treat population (ITT population) consisted of all patients treated with RAD001.
The overall tumor response was based on the data as per local radiological review, following RECIST criteria. The ORR is defined as the proportion of patients with CR or PR and was summarized in terms of percentage with 95% exact Clopper-Pearson CIs
Outcome measures
| Measure |
RAD001
n=142 Participants
Two 5 mg RAD001 tablets
|
|---|---|
|
Objective Response Rate (ORR; Where ORR = CR + PR)
|
17 Percentage of participants
Interval 7.1 to 18.5
|
SECONDARY outcome
Timeframe: Approximately 4 yearsPopulation: The Intent-to-Treat population (ITT population) consisted of all patients treated with RAD001.
The DOR analysis applied only to patients whose overall response was CR or PR and was defined as the time from onset of response (CR/PR) to progression or death from any cause
Outcome measures
| Measure |
RAD001
n=142 Participants
Two 5 mg RAD001 tablets
|
|---|---|
|
Duration of Response (DOR)
|
169 Weeks
Interval 52.71 to
NA= level of detection, insufficient number of participants with events
|
SECONDARY outcome
Timeframe: Approximately 4 yearsPopulation: The Intent-to-Treat population (ITT population) consisted of all patients treated with RAD001.
Overall survival is defined as the time from date of start of treatment to date of death due to any cause. If a patient is not known to have died, survival will be censored at the date of last contact.
Outcome measures
| Measure |
RAD001
n=142 Participants
Two 5 mg RAD001 tablets
|
|---|---|
|
Overall Survival
|
NA Weeks
Interval 116.86 to
NA=level of detection, insufficient number of participants with events
|
Adverse Events
RAD001
Serious events: 48 serious events
Other events: 125 other events
Deaths: 33 deaths
Serious adverse events
| Measure |
RAD001
n=142 participants at risk
Two 5 mg RAD001 tablets
|
|---|---|
|
Blood and lymphatic system disorders
ANAEMIA
|
3.5%
5/142 • Approximately 4 years
AE additional description
|
|
Blood and lymphatic system disorders
FEBRILE NEUTROPENIA
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Blood and lymphatic system disorders
HYPOCHROMIC ANAEMIA
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Cardiac disorders
CARDIO-RESPIRATORY ARREST
|
1.4%
2/142 • Approximately 4 years
AE additional description
|
|
Cardiac disorders
MYOCARDIAL INFARCTION
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Cardiac disorders
TACHYCARDIA
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Endocrine disorders
HYPERCALCAEMIA OF MALIGNANCY
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Gastrointestinal disorders
ABDOMINAL DISTENSION
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Gastrointestinal disorders
ABDOMINAL PAIN
|
1.4%
2/142 • Approximately 4 years
AE additional description
|
|
Gastrointestinal disorders
COLONIC FISTULA
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Gastrointestinal disorders
CONSTIPATION
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Gastrointestinal disorders
DIARRHOEA
|
2.1%
3/142 • Approximately 4 years
AE additional description
|
|
Gastrointestinal disorders
DYSPHAGIA
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Gastrointestinal disorders
INTESTINAL OBSTRUCTION
|
1.4%
2/142 • Approximately 4 years
AE additional description
|
|
Gastrointestinal disorders
NAUSEA
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Gastrointestinal disorders
STOMATITIS
|
1.4%
2/142 • Approximately 4 years
AE additional description
|
|
Gastrointestinal disorders
UPPER GASTROINTESTINAL HAEMORRHAGE
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Gastrointestinal disorders
VOMITING
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
General disorders
ASTHENIA
|
2.1%
3/142 • Approximately 4 years
AE additional description
|
|
General disorders
FATIGUE
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
General disorders
MUCOSAL INFLAMMATION
|
2.8%
4/142 • Approximately 4 years
AE additional description
|
|
General disorders
MULTI-ORGAN DISORDER
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
General disorders
PYREXIA
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Infections and infestations
ABSCESS
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Infections and infestations
ANAL ABSCESS
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Infections and infestations
BACTERAEMIA
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Infections and infestations
DIVERTICULITIS
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Infections and infestations
GASTROENTERITIS
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Infections and infestations
GENITAL ABSCESS
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Infections and infestations
LOWER RESPIRATORY TRACT INFECTION
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Infections and infestations
PNEUMONIA
|
4.2%
6/142 • Approximately 4 years
AE additional description
|
|
Infections and infestations
SEPSIS
|
1.4%
2/142 • Approximately 4 years
AE additional description
|
|
Infections and infestations
TOOTH ABSCESS
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Infections and infestations
URINARY TRACT INFECTION
|
2.1%
3/142 • Approximately 4 years
AE additional description
|
|
Injury, poisoning and procedural complications
FEMUR FRACTURE
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Injury, poisoning and procedural complications
PROCEDURAL PAIN
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Injury, poisoning and procedural complications
ROAD TRAFFIC ACCIDENT
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Investigations
ASPARTATE AMINOTRANSFERASE INCREASED
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Investigations
BLOOD CREATININE INCREASED
|
1.4%
2/142 • Approximately 4 years
AE additional description
|
|
Investigations
URINE OUTPUT DECREASED
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Metabolism and nutrition disorders
DECREASED APPETITE
|
2.1%
3/142 • Approximately 4 years
AE additional description
|
|
Metabolism and nutrition disorders
DEHYDRATION
|
2.1%
3/142 • Approximately 4 years
AE additional description
|
|
Metabolism and nutrition disorders
DIABETES MELLITUS
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Metabolism and nutrition disorders
HYPERCALCAEMIA
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Metabolism and nutrition disorders
HYPERCHOLESTEROLAEMIA
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Metabolism and nutrition disorders
HYPERNATRAEMIA
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Metabolism and nutrition disorders
HYPOGLYCAEMIA
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Metabolism and nutrition disorders
HYPOKALAEMIA
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Metabolism and nutrition disorders
HYPOMAGNESAEMIA
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Metabolism and nutrition disorders
HYPOPHOSPHATAEMIA
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Metabolism and nutrition disorders
KETOACIDOSIS
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Musculoskeletal and connective tissue disorders
MUSCULOSKELETAL CHEST PAIN
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Musculoskeletal and connective tissue disorders
MUSCULOSKELETAL PAIN
|
1.4%
2/142 • Approximately 4 years
AE additional description
|
|
Musculoskeletal and connective tissue disorders
NECK PAIN
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
BONE CANCER METASTATIC
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
MALIGNANT NEOPLASM PROGRESSION
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
METASTASES TO BONE
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
METASTASES TO LUNG
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
NEOPLASM PROGRESSION
|
2.1%
3/142 • Approximately 4 years
AE additional description
|
|
Nervous system disorders
DEPRESSED LEVEL OF CONSCIOUSNESS
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Nervous system disorders
HEMIPARESIS
|
1.4%
2/142 • Approximately 4 years
AE additional description
|
|
Nervous system disorders
LETHARGY
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Nervous system disorders
MENTAL IMPAIRMENT
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Nervous system disorders
NERVOUS SYSTEM DISORDER
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Nervous system disorders
NEUROPATHY PERIPHERAL
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Psychiatric disorders
CONFUSIONAL STATE
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Renal and urinary disorders
HAEMATURIA
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Renal and urinary disorders
RENAL FAILURE
|
1.4%
2/142 • Approximately 4 years
AE additional description
|
|
Renal and urinary disorders
RENAL FAILURE ACUTE
|
2.1%
3/142 • Approximately 4 years
AE additional description
|
|
Renal and urinary disorders
RENAL IMPAIRMENT
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Respiratory, thoracic and mediastinal disorders
COUGH
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Respiratory, thoracic and mediastinal disorders
DYSPNOEA
|
2.8%
4/142 • Approximately 4 years
AE additional description
|
|
Respiratory, thoracic and mediastinal disorders
LUNG DISORDER
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Respiratory, thoracic and mediastinal disorders
PLEURAL EFFUSION
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Respiratory, thoracic and mediastinal disorders
PNEUMONITIS
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Respiratory, thoracic and mediastinal disorders
PULMONARY ARTERY THROMBOSIS
|
1.4%
2/142 • Approximately 4 years
AE additional description
|
|
Respiratory, thoracic and mediastinal disorders
PULMONARY EMBOLISM
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Respiratory, thoracic and mediastinal disorders
RESPIRATORY FAILURE
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Vascular disorders
ARTERIAL THROMBOSIS LIMB
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Vascular disorders
HYPOTENSION
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
|
Vascular disorders
THROMBOPHLEBITIS
|
0.70%
1/142 • Approximately 4 years
AE additional description
|
Other adverse events
| Measure |
RAD001
n=142 participants at risk
Two 5 mg RAD001 tablets
|
|---|---|
|
Gastrointestinal disorders
STOMATITIS
|
28.9%
41/142 • Approximately 4 years
AE additional description
|
|
Blood and lymphatic system disorders
THROMBOCYTOPENIA
|
12.7%
18/142 • Approximately 4 years
AE additional description
|
|
Gastrointestinal disorders
ABDOMINAL PAIN
|
10.6%
15/142 • Approximately 4 years
AE additional description
|
|
Gastrointestinal disorders
CONSTIPATION
|
10.6%
15/142 • Approximately 4 years
AE additional description
|
|
Gastrointestinal disorders
DIARRHOEA
|
12.7%
18/142 • Approximately 4 years
AE additional description
|
|
Gastrointestinal disorders
NAUSEA
|
10.6%
15/142 • Approximately 4 years
AE additional description
|
|
Blood and lymphatic system disorders
ANAEMIA
|
43.7%
62/142 • Approximately 4 years
AE additional description
|
|
Blood and lymphatic system disorders
LEUKOPENIA
|
15.5%
22/142 • Approximately 4 years
AE additional description
|
|
Blood and lymphatic system disorders
NEUTROPENIA
|
16.2%
23/142 • Approximately 4 years
AE additional description
|
|
Gastrointestinal disorders
VOMITING
|
6.3%
9/142 • Approximately 4 years
AE additional description
|
|
General disorders
ASTHENIA
|
15.5%
22/142 • Approximately 4 years
AE additional description
|
|
General disorders
FATIGUE
|
21.1%
30/142 • Approximately 4 years
AE additional description
|
|
General disorders
MUCOSAL INFLAMMATION
|
16.9%
24/142 • Approximately 4 years
AE additional description
|
|
General disorders
OEDEMA PERIPHERAL
|
10.6%
15/142 • Approximately 4 years
AE additional description
|
|
General disorders
PYREXIA
|
9.9%
14/142 • Approximately 4 years
AE additional description
|
|
Infections and infestations
URINARY TRACT INFECTION
|
6.3%
9/142 • Approximately 4 years
AE additional description
|
|
Investigations
ALANINE AMINOTRANSFERASE INCREASED
|
9.9%
14/142 • Approximately 4 years
AE additional description
|
|
Investigations
ASPARTATE AMINOTRANSFERASE INCREASED
|
11.3%
16/142 • Approximately 4 years
AE additional description
|
|
Investigations
BLOOD ALKALINE PHOSPHATASE INCREASED
|
7.7%
11/142 • Approximately 4 years
AE additional description
|
|
Investigations
BLOOD CHOLESTEROL INCREASED
|
9.2%
13/142 • Approximately 4 years
AE additional description
|
|
Investigations
BLOOD CREATININE INCREASED
|
14.8%
21/142 • Approximately 4 years
AE additional description
|
|
Investigations
BLOOD TRIGLYCERIDES INCREASED
|
8.5%
12/142 • Approximately 4 years
AE additional description
|
|
Investigations
GAMMA-GLUTAMYLTRANSFERASE INCREASED
|
13.4%
19/142 • Approximately 4 years
AE additional description
|
|
Investigations
PROTHROMBIN TIME PROLONGED
|
5.6%
8/142 • Approximately 4 years
AE additional description
|
|
Metabolism and nutrition disorders
ABNORMAL LOSS OF WEIGHT
|
20.4%
29/142 • Approximately 4 years
AE additional description
|
|
Metabolism and nutrition disorders
DECREASED APPETITE
|
22.5%
32/142 • Approximately 4 years
AE additional description
|
|
Metabolism and nutrition disorders
HYPERCHOLESTEROLAEMIA
|
17.6%
25/142 • Approximately 4 years
AE additional description
|
|
Metabolism and nutrition disorders
HYPERGLYCAEMIA
|
26.1%
37/142 • Approximately 4 years
AE additional description
|
|
Metabolism and nutrition disorders
HYPERMAGNESAEMIA
|
7.0%
10/142 • Approximately 4 years
AE additional description
|
|
Metabolism and nutrition disorders
HYPERTRIGLYCERIDAEMIA
|
14.8%
21/142 • Approximately 4 years
AE additional description
|
|
Metabolism and nutrition disorders
HYPERURICAEMIA
|
7.0%
10/142 • Approximately 4 years
AE additional description
|
|
Metabolism and nutrition disorders
HYPOALBUMINAEMIA
|
7.7%
11/142 • Approximately 4 years
AE additional description
|
|
Metabolism and nutrition disorders
HYPOCALCAEMIA
|
12.7%
18/142 • Approximately 4 years
AE additional description
|
|
Metabolism and nutrition disorders
HYPOKALAEMIA
|
6.3%
9/142 • Approximately 4 years
AE additional description
|
|
Musculoskeletal and connective tissue disorders
ARTHRALGIA
|
6.3%
9/142 • Approximately 4 years
AE additional description
|
|
Musculoskeletal and connective tissue disorders
BACK PAIN
|
8.5%
12/142 • Approximately 4 years
AE additional description
|
|
Musculoskeletal and connective tissue disorders
PAIN IN EXTREMITY
|
8.5%
12/142 • Approximately 4 years
AE additional description
|
|
Nervous system disorders
DYSGEUSIA
|
6.3%
9/142 • Approximately 4 years
AE additional description
|
|
Nervous system disorders
HEADACHE
|
6.3%
9/142 • Approximately 4 years
AE additional description
|
|
Respiratory, thoracic and mediastinal disorders
COUGH
|
21.8%
31/142 • Approximately 4 years
AE additional description
|
|
Respiratory, thoracic and mediastinal disorders
DYSPNOEA
|
14.1%
20/142 • Approximately 4 years
AE additional description
|
|
Respiratory, thoracic and mediastinal disorders
EPISTAXIS
|
11.3%
16/142 • Approximately 4 years
AE additional description
|
|
Respiratory, thoracic and mediastinal disorders
PNEUMONITIS
|
7.7%
11/142 • Approximately 4 years
AE additional description
|
|
Skin and subcutaneous tissue disorders
PRURITUS
|
7.7%
11/142 • Approximately 4 years
AE additional description
|
|
Skin and subcutaneous tissue disorders
RASH
|
23.2%
33/142 • Approximately 4 years
AE additional description
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial or disclosure of trial results in their entirety.
- Publication restrictions are in place
Restriction type: OTHER