Trial Outcomes & Findings for RAPTOR: RAD001 as Monotherapy in the Treatment of Advanced Papillary Renal Cell Tumors Program in Europe (NCT NCT00688753)
NCT ID: NCT00688753
Last Updated: 2016-09-02
Results Overview
PFSR at 6 months based on central review
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
92 participants
Primary outcome timeframe
6 mos
Results posted on
2016-09-02
Participant Flow
Participant milestones
| Measure |
RAD001
two 5 mg tablets orally, once daily at the same time every day immediately after a meal
|
|---|---|
|
Overall Study
STARTED
|
92
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
92
|
Reasons for withdrawal
| Measure |
RAD001
two 5 mg tablets orally, once daily at the same time every day immediately after a meal
|
|---|---|
|
Overall Study
Disease progression
|
55
|
|
Overall Study
Missing
|
1
|
|
Overall Study
Death
|
9
|
|
Overall Study
Withdrawal by Subject
|
3
|
|
Overall Study
Adverse Event
|
20
|
|
Overall Study
Protocol Violation
|
1
|
|
Overall Study
Lost to Follow-up
|
3
|
Baseline Characteristics
RAPTOR: RAD001 as Monotherapy in the Treatment of Advanced Papillary Renal Cell Tumors Program in Europe
Baseline characteristics by cohort
| Measure |
RAD001 10 mg
n=92 Participants
two 5 mg tablets of everolimus orally, once daily
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
76 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
16 Participants
n=5 Participants
|
|
Age, Continuous
|
59.9 years
STANDARD_DEVIATION 14.9 • n=5 Participants
|
|
Sex: Female, Male
Female
|
20 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
72 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 6 mosPopulation: PP, PPFF, ITT
PFSR at 6 months based on central review
Outcome measures
| Measure |
RAD001
n=92 Participants
10 mg/day
|
|---|---|
|
To Evaluate Efficacy of RAD001 as Monotherapy for the Treatment of Papillary Renal Cancer. Efficacy is Defined as the Percentage of Patients Progression-free at 6 Months.
(PPFF Set, N=44)
|
34.1 % participants
Interval 24.5 to 44.8
|
|
To Evaluate Efficacy of RAD001 as Monotherapy for the Treatment of Papillary Renal Cancer. Efficacy is Defined as the Percentage of Patients Progression-free at 6 Months.
(PPSet, N=66)
|
33.3 % participants
Interval 25.6 to 41.8
|
|
To Evaluate Efficacy of RAD001 as Monotherapy for the Treatment of Papillary Renal Cancer. Efficacy is Defined as the Percentage of Patients Progression-free at 6 Months.
(ITT Set, N=86)
|
32.6 % participants
Interval 25.9 to 39.9
|
SECONDARY outcome
Timeframe: 6 mosPopulation: PP,ITT
DCR was defined as the proportion of patients with a best overall response of CR, PR or SD and ORR as the percentage of patients with CR or PR
Outcome measures
| Measure |
RAD001
n=92 Participants
10 mg/day
|
|---|---|
|
Disease Control Rate (SD + PR + CR)
PP set
|
65.2 % Participants
Interval 54.4 to 74.9
|
|
Disease Control Rate (SD + PR + CR)
ITT set
|
65.1 % Participants
Interval 55.8 to 73.6
|
SECONDARY outcome
Timeframe: End of trialORR is defined as the proportion of patients with tumor size reduction of a predefined amount and for a minimum time period. Response duration usually is measured from the time of initial response until documented tumor progression
Outcome measures
| Measure |
RAD001
n=92 Participants
10 mg/day
|
|---|---|
|
Objective Response Rate
PP Set
|
1.5 % participants
Interval 0.1 to 7.0
|
|
Objective Response Rate
ITT Set
|
1.2 % participants
Interval 0.1 to 5.4
|
SECONDARY outcome
Timeframe: End of trialPopulation: In the final analysis, for central review, the DOR could not be calculated as only 1 patient in the PP and ITT sets met the criteria
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=92 Participants
10 mg/day
|
|---|---|
|
Duration of Response
local review PP set
|
169 days
Interval 64.0 to 169.0
|
|
Duration of Response
local review ITT set
|
226 days
Interval 64.0 to 928.0
|
SECONDARY outcome
Timeframe: End of trialPFS was defined as the time from first study drug administration to objective tumor progression or death from any cause.
Outcome measures
| Measure |
RAD001
n=92 Participants
10 mg/day
|
|---|---|
|
Median Progression Free Survival
PP set
|
118 days
Interval 65.0 to 174.0
|
|
Median Progression Free Survival
ITT set
|
113 days
Interval 77.0 to 167.0
|
SECONDARY outcome
Timeframe: End of trialPopulation: Safety Set
Outcome measures
| Measure |
RAD001
n=92 Participants
10 mg/day
|
|---|---|
|
Incidence of Adverse Events, Serious Adverse Events, and Death.
Patients with any AE
|
100 % participants
|
|
Incidence of Adverse Events, Serious Adverse Events, and Death.
AE with suspected relation to study drug
|
97.83 % participants
|
|
Incidence of Adverse Events, Serious Adverse Events, and Death.
AE leading to dose adjustment or interruption
|
53.26 % participants
|
|
Incidence of Adverse Events, Serious Adverse Events, and Death.
AE leading to permanent discontinuation
|
27.17 % participants
|
|
Incidence of Adverse Events, Serious Adverse Events, and Death.
AE requiring concomitant medication
|
90.22 % participants
|
|
Incidence of Adverse Events, Serious Adverse Events, and Death.
Patients with serious adverse event (SAE)
|
46.74 % participants
|
|
Incidence of Adverse Events, Serious Adverse Events, and Death.
SAE suspected relation to study drug
|
23.91 % participants
|
|
Incidence of Adverse Events, Serious Adverse Events, and Death.
SAE leading to permanent discontinuation
|
10.87 % participants
|
|
Incidence of Adverse Events, Serious Adverse Events, and Death.
Patients died
|
10.87 % participants
|
Adverse Events
All Patients
Serious events: 43 serious events
Other events: 90 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
All Patients
n=92 participants at risk
two 5 mg tablets orally, once daily at the same time every day immediately after a meal
|
|---|---|
|
Renal and urinary disorders
Renal failure
|
3.3%
3/92
|
|
Renal and urinary disorders
Renal failure acute
|
4.3%
4/92
|
|
Reproductive system and breast disorders
Vaginal haemorrhage
|
1.1%
1/92
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
1.1%
1/92
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
3.3%
3/92
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea exertional
|
1.1%
1/92
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
1.1%
1/92
|
|
Respiratory, thoracic and mediastinal disorders
Interstitial lung disease
|
1.1%
1/92
|
|
Respiratory, thoracic and mediastinal disorders
Lung disorder
|
1.1%
1/92
|
|
Respiratory, thoracic and mediastinal disorders
Nasal septum deviation
|
1.1%
1/92
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
1.1%
1/92
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
4.3%
4/92
|
|
Blood and lymphatic system disorders
Anaemia
|
4.3%
4/92
|
|
Cardiac disorders
Atrial fibrillation
|
1.1%
1/92
|
|
Cardiac disorders
Atrial flutter
|
1.1%
1/92
|
|
Cardiac disorders
Cardiac arrest
|
1.1%
1/92
|
|
Cardiac disorders
Cor pulmonale
|
1.1%
1/92
|
|
Cardiac disorders
Tricuspid valve incompetence
|
1.1%
1/92
|
|
Gastrointestinal disorders
Abdominal pain
|
2.2%
2/92
|
|
Gastrointestinal disorders
Ascites
|
2.2%
2/92
|
|
Gastrointestinal disorders
Colitis
|
1.1%
1/92
|
|
Gastrointestinal disorders
Diarrhoea
|
1.1%
1/92
|
|
Gastrointestinal disorders
Ileus
|
1.1%
1/92
|
|
Gastrointestinal disorders
Large intestine polyp
|
1.1%
1/92
|
|
Gastrointestinal disorders
Oesophagitis
|
1.1%
1/92
|
|
Gastrointestinal disorders
Subileus
|
1.1%
1/92
|
|
Gastrointestinal disorders
Vomiting
|
1.1%
1/92
|
|
General disorders
Asthenia
|
4.3%
4/92
|
|
General disorders
Chest pain
|
1.1%
1/92
|
|
General disorders
Condition aggravated
|
2.2%
2/92
|
|
General disorders
General physical health deterioration
|
2.2%
2/92
|
|
General disorders
Mucosal inflammation
|
1.1%
1/92
|
|
General disorders
Multi-organ failure
|
1.1%
1/92
|
|
General disorders
Oedema peripheral
|
1.1%
1/92
|
|
General disorders
Pyrexia
|
4.3%
4/92
|
|
Hepatobiliary disorders
Cholestasis
|
1.1%
1/92
|
|
Infections and infestations
Anal infection
|
1.1%
1/92
|
|
Infections and infestations
Bacteraemia
|
1.1%
1/92
|
|
Infections and infestations
Cellulitis
|
1.1%
1/92
|
|
Infections and infestations
Erysipelas
|
1.1%
1/92
|
|
Infections and infestations
Gastrointestinal infection
|
1.1%
1/92
|
|
Infections and infestations
Herpes zoster
|
1.1%
1/92
|
|
Infections and infestations
Pneumonia
|
2.2%
2/92
|
|
Infections and infestations
Pneumonia bacterial
|
1.1%
1/92
|
|
Infections and infestations
Postoperative abscess
|
1.1%
1/92
|
|
Infections and infestations
Respiratory tract infection
|
1.1%
1/92
|
|
Infections and infestations
Upper respiratory tract infection
|
1.1%
1/92
|
|
Injury, poisoning and procedural complications
Lumbar vertebral fracture
|
1.1%
1/92
|
|
Investigations
Blood creatinine increased
|
1.1%
1/92
|
|
Metabolism and nutrition disorders
Dehydration
|
2.2%
2/92
|
|
Metabolism and nutrition disorders
Fluid overload
|
1.1%
1/92
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
1.1%
1/92
|
|
Metabolism and nutrition disorders
Hyponatraemia
|
1.1%
1/92
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
1.1%
1/92
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
1.1%
1/92
|
|
Musculoskeletal and connective tissue disorders
Fistula
|
1.1%
1/92
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasm progression
|
4.3%
4/92
|
|
Nervous system disorders
Aphasia
|
1.1%
1/92
|
|
Nervous system disorders
Hemiparesis
|
1.1%
1/92
|
|
Nervous system disorders
Spinal cord compression
|
1.1%
1/92
|
|
Nervous system disorders
Tremor
|
1.1%
1/92
|
|
Psychiatric disorders
Depression
|
1.1%
1/92
|
|
Psychiatric disorders
Hallucination
|
1.1%
1/92
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
1.1%
1/92
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary arterial hypertension
|
1.1%
1/92
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
|
1.1%
1/92
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
1.1%
1/92
|
|
Skin and subcutaneous tissue disorders
Rash erythematous
|
2.2%
2/92
|
|
Vascular disorders
Deep vein thrombosis
|
2.2%
2/92
|
|
Vascular disorders
Hypertension
|
1.1%
1/92
|
|
Vascular disorders
Varicose vein
|
1.1%
1/92
|
|
Vascular disorders
Venous thrombosis
|
1.1%
1/92
|
Other adverse events
| Measure |
All Patients
n=92 participants at risk
two 5 mg tablets orally, once daily at the same time every day immediately after a meal
|
|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
27.2%
25/92
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
10.9%
10/92
|
|
Cardiac disorders
Tachycardia
|
6.5%
6/92
|
|
Gastrointestinal disorders
Abdominal pain
|
25.0%
23/92
|
|
Gastrointestinal disorders
Aphthous stomatitis
|
12.0%
11/92
|
|
Gastrointestinal disorders
Ascites
|
5.4%
5/92
|
|
Gastrointestinal disorders
Constipation
|
16.3%
15/92
|
|
Gastrointestinal disorders
Diarrhoea
|
39.1%
36/92
|
|
Gastrointestinal disorders
Dry mouth
|
8.7%
8/92
|
|
Gastrointestinal disorders
Nausea
|
29.3%
27/92
|
|
Gastrointestinal disorders
Stomatitis
|
25.0%
23/92
|
|
Gastrointestinal disorders
Vomiting
|
17.4%
16/92
|
|
General disorders
Asthenia
|
42.4%
39/92
|
|
General disorders
Chest pain
|
7.6%
7/92
|
|
General disorders
Fatigue
|
32.6%
30/92
|
|
General disorders
Mucosal inflammation
|
39.1%
36/92
|
|
General disorders
Oedema peripheral
|
31.5%
29/92
|
|
General disorders
Pain
|
7.6%
7/92
|
|
General disorders
Pyrexia
|
27.2%
25/92
|
|
Infections and infestations
Bronchitis
|
5.4%
5/92
|
|
Infections and infestations
Rhinitis
|
9.8%
9/92
|
|
Infections and infestations
Urinary tract infection
|
6.5%
6/92
|
|
Investigations
Alanine aminotransferase increased
|
7.6%
7/92
|
|
Investigations
Aspartate aminotransferase increased
|
6.5%
6/92
|
|
Investigations
Blood creatinine increased
|
8.7%
8/92
|
|
Investigations
Platelet count decreased
|
6.5%
6/92
|
|
Investigations
Weight decreased
|
13.0%
12/92
|
|
Metabolism and nutrition disorders
Decreased appetite
|
39.1%
36/92
|
|
Metabolism and nutrition disorders
Hypercholesterolaemia
|
16.3%
15/92
|
|
Metabolism and nutrition disorders
Hyperglycaemia
|
13.0%
12/92
|
|
Metabolism and nutrition disorders
Hypertriglyceridaemia
|
10.9%
10/92
|
|
Metabolism and nutrition disorders
Hypocalcaemia
|
5.4%
5/92
|
|
Metabolism and nutrition disorders
Hypophosphataemia
|
8.7%
8/92
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
12.0%
11/92
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
17.4%
16/92
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
5.4%
5/92
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
5.4%
5/92
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
10.9%
10/92
|
|
Nervous system disorders
Dysgeusia
|
25.0%
23/92
|
|
Nervous system disorders
Headache
|
17.4%
16/92
|
|
Psychiatric disorders
Insomnia
|
9.8%
9/92
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
39.1%
36/92
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
31.5%
29/92
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
28.3%
26/92
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
7.6%
7/92
|
|
Skin and subcutaneous tissue disorders
Acne
|
5.4%
5/92
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
13.0%
12/92
|
|
Skin and subcutaneous tissue disorders
Nail disorder
|
14.1%
13/92
|
|
Skin and subcutaneous tissue disorders
Palmar-plantar erythrodysaesthesia syndrome
|
5.4%
5/92
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
21.7%
20/92
|
|
Skin and subcutaneous tissue disorders
Rash
|
57.6%
53/92
|
|
Vascular disorders
Hypertension
|
6.5%
6/92
|
Additional Information
Clinical Disclosure Office
Novartis Pharmaceuticals
Phone: 862-778-8300
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 (ie, data from all sites) in the clinical trial.
- Publication restrictions are in place
Restriction type: OTHER