Trial Outcomes & Findings for RO4929097 in Treating Patients With Metastatic Colorectal Cancer (NCT NCT01116687)

Last Updated: 2014-05-20

Results Overview

To determine the objective radiographic response rate associated with RO4929097 in patients with metastatic colorectal cancer who have progressed following at least 2 prior treatments in the metastatic setting. Radiologic assessment of tumor burden (CT scans of the chest, abdomen and pelvis, or MRI of the abdomen and pelvis and CT of the chest) was scheduled every 8 weeks. Response Evaluation Criteria in Solid Tumors (RECIST version 1.1) were used for evaluation of the primary endpoint.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

37 participants

Primary outcome timeframe

2 months from enrollment for each participant

Results posted on

2014-05-20

Participant Flow

Patients with metastatic colorectal cancer who had received at least two prior lines of systemic chemotherapy were enrolled on the study.

Participant milestones

Participant milestones
Measure	Investigational Drug Therapy RO4929097 20 mg by mouth 3 days on 4 days off continuously.
Overall Study STARTED	37
Overall Study COMPLETED	33
Overall Study NOT COMPLETED	4

Reasons for withdrawal

Reasons for withdrawal
Measure	Investigational Drug Therapy RO4929097 20 mg by mouth 3 days on 4 days off continuously.
Overall Study Withdrawal by Subject	4

Baseline Characteristics

RO4929097 in Treating Patients With Metastatic Colorectal Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Investigational Drug Therapy n=37 Participants RO4929097 20 mg by mouth 3 days on 4 days off continuously.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	28 Participants n=5 Participants
Age, Categorical >=65 years	9 Participants n=5 Participants
Age, Customized	60 years n=5 Participants
Sex: Female, Male Female	15 Participants n=5 Participants
Sex: Female, Male Male	22 Participants n=5 Participants
Region of Enrollment United States	37 participants n=5 Participants

PRIMARY outcome

Timeframe: 2 months from enrollment for each participant

Population: All evaluable participants

Outcome measures

Outcome measures
Measure	Investigational Drug Therapy n=33 Participants RO4929097 20 mg by mouth 3 days on 4 days off continuously.
Number of Participants With Objective Radiographic Response (ORR)	0 participants

SECONDARY outcome

Timeframe: Study duration of 12 months

Population: All evaluable participants

Overall Survival defined as the time from start of treatment until death as a result of any cause, with patients censored at the date of last follow-up if still alive. The Kaplan-Meier method was used to estimate all time-to-event functions. Statistical analysis was performed using Stata SE 9.0 software and SAS 9.2 software.

Outcome measures

Outcome measures
Measure	Investigational Drug Therapy n=33 Participants RO4929097 20 mg by mouth 3 days on 4 days off continuously.
Participant Overall Survival (OS) Rate	6 months Interval 3.9 to 9.1

SECONDARY outcome

Timeframe: Study duration of 12 months

Population: All evaluable participants

Progression-Free Survival defined as the time from start of treatment until disease progression or death as a result of any cause. Patients were re-evaluated for response every 8 weeks. Response and progression were evaluated using the new international criteria proposed by the revised Response Evaluation Criteria in Solid Tumors (RECIST) guideline (version 1.1) \[Eur J Ca 45:228-247, 2009\]. Changes in the largest diameter (unidimensional measurement) of the tumor lesions and the shortest diameter in the case of malignant lymph nodes are used in the RECIST criteria.

Outcome measures

Outcome measures
Measure	Investigational Drug Therapy n=33 Participants RO4929097 20 mg by mouth 3 days on 4 days off continuously.
Participant Progression Free Survival (PFS) Rate	1.8 months Interval 1.8 to 1.86

SECONDARY outcome

Timeframe: Study duration of 12 months

Population: All evaluable participants

Study drug related grade 3-4 toxicities. To measure Adverse Events, investigators used the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.

Outcome measures

Outcome measures
Measure	Investigational Drug Therapy n=33 Participants RO4929097 20 mg by mouth 3 days on 4 days off continuously.
Number of Related Serious Adverse Events (SAEs)	0 events

Adverse Events

Investigational Drug Therapy

Serious events: 12 serious events

Other events: 29 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Investigational Drug Therapy n=33 participants at risk RO4929097 20 mg by mouth 3 days on 4 days off continuously.
Metabolism and nutrition disorders Dehydration - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Eye disorders Eye disorder - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Nervous system disorders Headache - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Hepatobiliary disorders Hepatic failure - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Infections and infestations Hepatic infection - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Gastrointestinal disorders Ileus - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Blood and lymphatic system disorders Leukocytosis - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Gastrointestinal disorders Nausea - unrelated	12.1% 4/33 • Number of events 4 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Gastrointestinal disorders Abdominal pain - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Metabolism and nutrition disorders Anorexia - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Gastrointestinal disorders Constipation - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
General disorders Death - unrelated	24.2% 8/33 • Number of events 8 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Investigations Platelet count decreased - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Respiratory, thoracic and mediastinal disorders Pleural effusion - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Respiratory, thoracic and mediastinal disorders Pneumonitis - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Gastrointestinal disorders Rectal pain - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Renal and urinary disorders Renal and urinary disorders - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Gastrointestinal disorders Small intestinal obstruction - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Gastrointestinal disorders Vomiting - unrelated	6.1% 2/33 • Number of events 2 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.

Other adverse events

Other adverse events
Measure	Investigational Drug Therapy n=33 participants at risk RO4929097 20 mg by mouth 3 days on 4 days off continuously.
Gastrointestinal disorders Abdominal pain - unrelated	21.2% 7/33 • Number of events 8 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Investigations Alkaline phosphatase increased - unrelated	3.0% 1/33 • Number of events 2 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Metabolism and nutrition disorders Anorexia - unrelated	30.3% 10/33 • Number of events 10 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Musculoskeletal and connective tissue disorders Back pain - unrelated	12.1% 4/33 • Number of events 4 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Investigations Blood bilirubin increased - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Respiratory, thoracic and mediastinal disorders Bronchopulmonary hemorrhage - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
General disorders Chills - unrelated	6.1% 2/33 • Number of events 3 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Gastrointestinal disorders Constipation - unrelated	12.1% 4/33 • Number of events 4 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Respiratory, thoracic and mediastinal disorders Cough - unrelated	6.1% 2/33 • Number of events 2 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Gastrointestinal disorders Diarrhea - unrelated	6.1% 2/33 • Number of events 2 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Nervous system disorders Dizziness - possibly related	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Nervous system disorders Dizziness - unrelated	6.1% 2/33 • Number of events 2 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Eye disorders Dry eye - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Nervous system disorders Dysgeusia - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Respiratory, thoracic and mediastinal disorders Dyspnea - unrelated	15.2% 5/33 • Number of events 5 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
General disorders Fatigue - possibly related	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
General disorders Fatigue - unrelated	9.1% 3/33 • Number of events 3 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
General disorders Fever - unrelated	12.1% 4/33 • Number of events 6 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Gastrointestinal disorders Gastroesophageal reflux disease - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Gastrointestinal disorders Gastrointestinal pain - unrelated	3.0% 1/33 • Number of events 2 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Nervous system disorders Headache - unrelated	15.2% 5/33 • Number of events 5 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Vascular disorders Hot flashes - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Vascular disorders Hypertension - unrelated	6.1% 2/33 • Number of events 2 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Metabolism and nutrition disorders Hypoglycemia - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Infections and infestations Infections and infestations - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Psychiatric disorders Insomnia - unrelated	6.1% 2/33 • Number of events 2 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Nervous system disorders Lethargy - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Gastrointestinal disorders Mucositis oral - possibly related	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Gastrointestinal disorders Nausea - possibly related	9.1% 3/33 • Number of events 3 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Gastrointestinal disorders Nausea - unrelated	27.3% 9/33 • Number of events 9 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Musculoskeletal and connective tissue disorders Neck pain - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Investigations Neutrophil count decreased - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
General disorders Non-cardiac chest pain - unrelated	6.1% 2/33 • Number of events 2 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Gastrointestinal disorders Oral pain - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
General disorders Pain - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Musculoskeletal and connective tissue disorders Pain in extremity - unrelated	6.1% 2/33 • Number of events 2 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Reproductive system and breast disorders Pelvic pain - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Respiratory, thoracic and mediastinal disorders Productive cough - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Skin and subcutaneous tissue disorders Pruritus - possibly related	6.1% 2/33 • Number of events 2 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Skin and subcutaneous tissue disorders Pruritus - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Skin and subcutaneous tissue disorders Rash acneiform - possibly related	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Gastrointestinal disorders Rectal hemorrhage - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Skin and subcutaneous tissue disorders Skin hyperpigmentation - possibly related	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Renal and urinary disorders Urine discoloration - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Investigations Urine output decreased - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Gastrointestinal disorders Vomiting - possibly related	6.1% 2/33 • Number of events 2 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Gastrointestinal disorders Vomiting - unrelated	27.3% 9/33 • Number of events 9 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.
Investigations Weight loss - unrelated	3.0% 1/33 • Number of events 1 • 12 months The toxicities considered possibly related to treatment are designated as such in the events listed below.

Additional Information

Jonathan Strosberg, M.D.

H. Lee Moffitt Cancer Center and Research Institute

Phone: 813-745-7257

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60