Trial Outcomes & Findings for A Study of RO4929097 in Patients With Advanced Renal Cell Carcinoma That Have Failed Vascular Endothelial Growth Factor (VEGF)/Vascular Endothelial Growth Factor Receptor (VEGFR) Therapy (NCT NCT01141569)

Last Updated: 2017-03-10

Results Overview

Complete Response (CR): Disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \<10 mm. Partial Response (PR): At least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

12 participants

Primary outcome timeframe

Up to 12 months

Results posted on

2017-03-10

Participant Flow

Participant milestones

Participant milestones
Measure	Treatment (RO4929097) Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO on days 1-3, 8-10, and 15-17. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity. Gamma-Secretase Inhibitor RO4929097: Given PO Laboratory Biomarker Analysis: Correlative studies
Overall Study STARTED	12
Overall Study COMPLETED	12
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Study of RO4929097 in Patients With Advanced Renal Cell Carcinoma That Have Failed Vascular Endothelial Growth Factor (VEGF)/Vascular Endothelial Growth Factor Receptor (VEGFR) Therapy

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment (RO4929097) n=12 Participants Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO on days 1-3, 8-10, and 15-17. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity. Gamma-Secretase Inhibitor RO4929097: Given PO Laboratory Biomarker Analysis: Correlative studies
Age, Categorical <=18 years	0 Participants n=39 Participants
Age, Categorical Between 18 and 65 years	11 Participants n=39 Participants
Age, Categorical >=65 years	1 Participants n=39 Participants
Age, Continuous	60 years n=39 Participants
Sex: Female, Male Female	3 Participants n=39 Participants
Sex: Female, Male Male	9 Participants n=39 Participants
Region of Enrollment Canada	12 participants n=39 Participants

PRIMARY outcome

Timeframe: Up to 12 months

Population: Patients that had PR or CR (Objective response)

Outcome measures

Outcome measures
Measure	Treatment (RO4929097) n=12 Participants Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO on days 1-3, 8-10, and 15-17. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity. Gamma-Secretase Inhibitor RO4929097: Given PO Laboratory Biomarker Analysis: Correlative studies
Objective Response Rate (PR + CR) Using RECIST	0 participants

SECONDARY outcome

Timeframe: Up to 12 months

Time to Progression is duration of time from start of treatment to time of progression

Outcome measures

Outcome measures
Measure	Treatment (RO4929097) n=12 Participants Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO on days 1-3, 8-10, and 15-17. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity. Gamma-Secretase Inhibitor RO4929097: Given PO Laboratory Biomarker Analysis: Correlative studies
Time to Progression	1.2 months Interval 0.8 to 2.6

SECONDARY outcome

Timeframe: Up to 12 months

Population: Number analyzed is number of participants who experienced the adverse event.

Tabulated using counts and proportions detailing frequently occurring, serious and severe events of interest.

Outcome measures

Outcome measures
Measure	Treatment (RO4929097) n=12 Participants Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO on days 1-3, 8-10, and 15-17. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity. Gamma-Secretase Inhibitor RO4929097: Given PO Laboratory Biomarker Analysis: Correlative studies
Frequency and Severity of Adverse Events Edema limbs · Grade 3-4	0 Participants
Frequency and Severity of Adverse Events Hyponatremia · Grade 1-2	4 Participants
Frequency and Severity of Adverse Events Hyponatremia · Grade 3-4	2 Participants
Frequency and Severity of Adverse Events Hypophosphatemia · Grade 1-2	3 Participants
Frequency and Severity of Adverse Events Hypophosphatemia · Grade 3-4	1 Participants
Frequency and Severity of Adverse Events Dyspnea · Grade 1-2	5 Participants
Frequency and Severity of Adverse Events Dyspnea · Grade 3-4	0 Participants
Frequency and Severity of Adverse Events Bone Pain · Grade 1-2	4 Participants
Frequency and Severity of Adverse Events Bone Pain · Grade 3-4	2 Participants
Frequency and Severity of Adverse Events AST increased · Grade 1-2	5 Participants
Frequency and Severity of Adverse Events AST increased · Grade 3-4	0 Participants
Frequency and Severity of Adverse Events ALP Increased · Grade 1-2	5 Participants
Frequency and Severity of Adverse Events ALP Increased · Grade 3-4	0 Participants
Frequency and Severity of Adverse Events Hypertension · Grade 1-2	3 Participants
Frequency and Severity of Adverse Events Hypertension · Grade 3-4	2 Participants
Frequency and Severity of Adverse Events Flank Pain · Grade 1-2	4 Participants
Frequency and Severity of Adverse Events Flank Pain · Grade 3-4	0 Participants
Frequency and Severity of Adverse Events Vomiting · Grade 1-2	4 Participants
Frequency and Severity of Adverse Events Vomiting · Grade 3-4	0 Participants
Frequency and Severity of Adverse Events ALT increased · Grade 1-2	4 Participants
Frequency and Severity of Adverse Events Fatigue · Grade 1-2	12 Participants
Frequency and Severity of Adverse Events Fatigue · Grade 3-4	0 Participants
Frequency and Severity of Adverse Events Insomnia · Grade 1-2	10 Participants
Frequency and Severity of Adverse Events Insomnia · Grade 3-4	0 Participants
Frequency and Severity of Adverse Events Anorexia · Grade 1-2	9 Participants
Frequency and Severity of Adverse Events Anorexia · Grade 3-4	0 Participants
Frequency and Severity of Adverse Events Anemia · Grade 1-2	8 Participants
Frequency and Severity of Adverse Events Anemia · Grade 3-4	0 Participants
Frequency and Severity of Adverse Events Nausea · Grade 1-2	7 Participants
Frequency and Severity of Adverse Events Nausea · Grade 3-4	0 Participants
Frequency and Severity of Adverse Events Creatinine increased · Grade 1-2	6 Participants
Frequency and Severity of Adverse Events Creatinine increased · Grade 3-4	0 Participants
Frequency and Severity of Adverse Events Pruritis · Grade 1-2	6 Participants
Frequency and Severity of Adverse Events Pruritis · Grade 3-4	0 Participants
Frequency and Severity of Adverse Events Lymphocyte count decreased · Grade 1-2	7 Participants
Frequency and Severity of Adverse Events Lymphocyte count decreased · Grade 3-4	2 Participants
Frequency and Severity of Adverse Events Hypoalbuminemia · Grade 1-2	6 Participants
Frequency and Severity of Adverse Events Hypoalbuminemia · Grade 3-4	0 Participants
Frequency and Severity of Adverse Events Edema limbs · Grade 1-2	5 Participants
Frequency and Severity of Adverse Events ALT increased · Grade 3-4	0 Participants

SECONDARY outcome

Timeframe: From start of treatment to time of progression or death, whichever occurs first, assessed up to 12 months

Progression-free survival is defined as the duration of time from start of treatment to time of progression or death, whichever occurs first.

Outcome measures

Outcome measures
Measure	Treatment (RO4929097) n=12 Participants Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO on days 1-3, 8-10, and 15-17. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity. Gamma-Secretase Inhibitor RO4929097: Given PO Laboratory Biomarker Analysis: Correlative studies
Progression-free Survival Rate	1.2 months Interval 0.8 to 2.6

SECONDARY outcome

Timeframe: Up to 12 months

Population: Number of patients who had Stable disease

Stable Disease (SD): Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum diameters while on study

Outcome measures

Outcome measures
Measure	Treatment (RO4929097) n=12 Participants Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO on days 1-3, 8-10, and 15-17. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity. Gamma-Secretase Inhibitor RO4929097: Given PO Laboratory Biomarker Analysis: Correlative studies
Rate of Disease Stabilizations	4 participants

SECONDARY outcome

Timeframe: Up to 12 months

Tumor control rate is defined as the sum of objective response rate (CR+PR) and stable disease (SD) rate.

Outcome measures

Outcome measures
Measure	Treatment (RO4929097) n=12 Participants Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO on days 1-3, 8-10, and 15-17. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity. Gamma-Secretase Inhibitor RO4929097: Given PO Laboratory Biomarker Analysis: Correlative studies
Tumor Control Rate (CR + PR + SD)	4 Participants

Adverse Events

Treatment (RO4929097)

Serious events: 1 serious events

Other events: 12 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Treatment (RO4929097) n=12 participants at risk Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO on days 1-3, 8-10, and 15-17. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity. Gamma-Secretase Inhibitor RO4929097: Given PO Laboratory Biomarker Analysis: Correlative studies
Musculoskeletal and connective tissue disorders Bone Pain	8.3% 1/12 • Number of events 1

Other adverse events

Other adverse events
Measure	Treatment (RO4929097) n=12 participants at risk Patients receive gamma-secretase/Notch signalling pathway inhibitor RO4929097 PO on days 1-3, 8-10, and 15-17. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity. Gamma-Secretase Inhibitor RO4929097: Given PO Laboratory Biomarker Analysis: Correlative studies
General disorders Fatigue	100.0% 12/12 • Number of events 12
Psychiatric disorders Insomnia	83.3% 10/12 • Number of events 10
Metabolism and nutrition disorders Anorexia	75.0% 9/12 • Number of events 9
Blood and lymphatic system disorders Anemia	66.7% 8/12 • Number of events 8
Gastrointestinal disorders Nausea	58.3% 7/12 • Number of events 7
Investigations Creatinine increased	50.0% 6/12 • Number of events 6
Skin and subcutaneous tissue disorders Pruritus	50.0% 6/12 • Number of events 6
Investigations Lymphocyte count decreased	75.0% 9/12 • Number of events 9
Metabolism and nutrition disorders Hypoalbuminemia	50.0% 6/12 • Number of events 6
General disorders Edema limbs	41.7% 5/12 • Number of events 5
Metabolism and nutrition disorders Hyponatremia	50.0% 6/12 • Number of events 6
Metabolism and nutrition disorders Hypophosphatemia	33.3% 4/12 • Number of events 4
Respiratory, thoracic and mediastinal disorders Dyspnea	41.7% 5/12 • Number of events 5
Musculoskeletal and connective tissue disorders Bone pain	41.7% 5/12 • Number of events 5
Investigations AST increased	41.7% 5/12 • Number of events 5
Investigations ALP increased	41.7% 5/12 • Number of events 5
Vascular disorders Hypertension	41.7% 5/12 • Number of events 5
Musculoskeletal and connective tissue disorders Flank pain	33.3% 4/12 • Number of events 4
General disorders Vomiting	33.3% 4/12 • Number of events 4
Investigations ALT increased	33.3% 4/12 • Number of events 4

Additional Information

Dr. Christian Kollmannsberger

BC Cancer Agency Vancouver Cancer Centre

Phone: 604-877-6000

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60