Trial Outcomes & Findings for Vismodegib in Treating Patients With Advanced Chondrosarcomas (NCT NCT01267955)

NCT ID: NCT01267955

Last Updated: 2025-08-03

Results Overview

CBR was defined as the percentage of participants with complete or partial responses (CR, PR) or stable disease (SD) per RECIST 1.1. CR was defined as disappearance of all non-nodal target lesions. PR was defined as at least a 30% decrease in the sum of diameter of all target lesions, taking as reference the baseline sum of diameters. Analysis of response was performed based on radiological centralized review. A 2-stage optimal Simon's design with 37 participants (first stage: 17 participants) was used. If 3 or less non-progressions (CR + PR + SD) at 6 months were observed during stage 1 (out of 17 participants), the trial would stopped early. Otherwise, 20 additional patients would be accrued for stage 2. If 11 or more non-progressions (out of 37 participants) were observed at the end of recruitment, further investigation of this therapy would be warranted.

• Recruitment status: ACTIVE_NOT_RECRUITING
• Study phase: PHASE2
• Target enrollment: 45 participants
• Primary outcome timeframe: At 6 months after inclusion
• Results posted on: 2025-08-03

Participant Flow

Participant milestones

Measure	Treatment (Vismodegib) Patients receive vismodegib PO on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Pharmacogenomic Study: Correlative studies Vismodegib: Given PO This is a single-arm phase 2 trial, based on a two-stage Simon's optimal design. Assuming 20% (H0: null hypothesis) and 40% (H1: alternative hypothesis) six-month CBR, 10% type I error rate and 90% power, 37 eligible and assessable patients were necessary (17 in the first stage and 20 in the second stage). At the second stage/final stage, GDC-0449 would be considered promising if at least eleven patients were progression-free at 6 months.
Stage 1 (6 Months) STARTED	20
Stage 1 (6 Months) COMPLETED	17
Stage 1 (6 Months) NOT COMPLETED	3
Stage 2 (6 Months) STARTED	45
Stage 2 (6 Months) COMPLETED	39
Stage 2 (6 Months) NOT COMPLETED	6

Reasons for withdrawal

Measure	Treatment (Vismodegib) Patients receive vismodegib PO on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Pharmacogenomic Study: Correlative studies Vismodegib: Given PO This is a single-arm phase 2 trial, based on a two-stage Simon's optimal design. Assuming 20% (H0: null hypothesis) and 40% (H1: alternative hypothesis) six-month CBR, 10% type I error rate and 90% power, 37 eligible and assessable patients were necessary (17 in the first stage and 20 in the second stage). At the second stage/final stage, GDC-0449 would be considered promising if at least eleven patients were progression-free at 6 months.
Stage 1 (6 Months) Death	3
Stage 2 (6 Months) Death	5
Stage 2 (6 Months) Protocol Violation	1

Baseline Characteristics

Weight not available for 1 patient

Baseline characteristics by cohort

Measure	Treatment (Vismodegib) n=45 Participants Patients receive vismodegib PO on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Pharmacogenomic Study: Correlative studies Vismodegib: Given PO
Age, Continuous	58.0 years n=45 Participants
Sex: Female, Male Female	14 Participants n=45 Participants
Sex: Female, Male Male	31 Participants n=45 Participants
Region of Enrollment France	45 participants n=45 Participants
ECOG Performance status 0	20 Participants n=45 Participants
ECOG Performance status 1	20 Participants n=45 Participants
ECOG Performance status 2	5 Participants n=45 Participants
Weight	76.5 kilogram n=44 Participants • Weight not available for 1 patient
Height	173.0 millimetres n=45 Participants
Histological subtype Conventional chondrosarcoma	39 Participants n=45 Participants
Histological subtype Dedifferentiated chondrosarcoma	5 Participants n=45 Participants
Histological subtype Clear cell chondrosarcoma	1 Participants n=45 Participants
Histological subtype Mesenchymal chondrosarcoma	0 Participants n=45 Participants
Stage of the disease Locally advanced	13 Participants n=45 Participants
Stage of the disease Metastatic	32 Participants n=45 Participants
Prior lines of chemotherapy 0	25 Participants n=45 Participants
Prior lines of chemotherapy 1	12 Participants n=45 Participants
Prior lines of chemotherapy 2	3 Participants n=45 Participants
Prior lines of chemotherapy >2	5 Participants n=45 Participants

PRIMARY outcome

Timeframe: At 6 months after inclusion

Population: Eligible participants with at least one complete or two incomplete cycles of treatment and for whom at least one disease measurement has been recorded not less than 8 weeks after treatment onset.

CBR was defined as the percentage of participants with complete or partial responses (CR, PR) or stable disease (SD) per RECIST 1.1. CR was defined as disappearance of all non-nodal target lesions. PR was defined as at least a 30% decrease in the sum of diameter of all target lesions, taking as reference the baseline sum of diameters. Analysis of response was performed based on radiological centralized review. A 2-stage optimal Simon's design with 37 participants (first stage: 17 participants) was used. If 3 or less non-progressions (CR + PR + SD) at 6 months were observed during stage 1 (out of 17 participants), the trial would stopped early. Otherwise, 20 additional patients would be accrued for stage 2. If 11 or more non-progressions (out of 37 participants) were observed at the end of recruitment, further investigation of this therapy would be warranted.

Outcome measures

Measure	Treatment (Vismodegib) n=39 Participants Patients receive vismodegib PO on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Pharmacogenomic Study: Correlative studies Vismodegib: Given PO
Clinical Benefit Rate (CBR) Based on Centralized Imaging Review as Per RECIST 1.1 First stage	23.5 percentage of participants Interval 6.8 to 49.9
Clinical Benefit Rate (CBR) Based on Centralized Imaging Review as Per RECIST 1.1 Second stage (Overall)	25.6 percentage of participants Interval 13.0 to 42.1

SECONDARY outcome

Timeframe: Time from start of treatment to time of progression or death, whichever occurs first, assessed up to 3 years

Will be analyzed using the Kaplan-Meier method. The median survival rates will be reported with a 95% confidence interval. Median follow-up will be calculated using the reverse Kaplan-Meier method.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Time from start of treatment to the time of death, assessed up to 3 years

Overall survival will be analyzed using the Kaplan-Meier method. The median survival rates will be reported with a 95% confidence interval. Median follow-up will be calculated using the reverse Kaplan-Meier method.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: From the time measurement criteria are met for complete response or partial response (whichever is first recorded) until the first date that recurrent or progressive disease is objectively documented, assessed up to 3 years

Will be described in responding subjects using descriptive statistics (median, extreme values, etc.).

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline

The 6-months clinical benefit rate will be correlated with the mutational status of patched and smoothened in order to identify predictive factors of clinical benefit from vismodegib.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Baseline

The 6-months clinical benefit rate will be correlated with the expression score of hedgehog signaling molecules in order to identify predictive factors of clinical benefit from vismodegib.

Outcome measures

Outcome data not reported

Adverse Events

Treatment (Vismodegib)

Serious events: 22 serious events

Other events: 45 other events

Deaths: 23 deaths

Serious adverse events

Measure	Treatment (Vismodegib) n=45 participants at risk Patients receive vismodegib PO on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Pharmacogenomic Study: Correlative studies Vismodegib: Given PO
Blood and lymphatic system disorders ANEMIA	4.4% 2/45 • Number of events 2 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Gastrointestinal disorders ABDOMINAL PAIN	2.2% 1/45 • Number of events 1 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Gastrointestinal disorders CONSTIPATION	2.2% 1/45 • Number of events 1 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
General disorders General disorders and administration site conditions - Other, specify	4.4% 2/45 • Number of events 2 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Hepatobiliary disorders Hepatobiliary disorders - Other, specify	2.2% 1/45 • Number of events 1 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Infections and infestations Sepsis	6.7% 3/45 • Number of events 3 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Infections and infestations Urinary tract infection	2.2% 1/45 • Number of events 1 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Injury, poisoning and procedural complications Fracture	2.2% 1/45 • Number of events 1 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Investigations GGT increased	2.2% 1/45 • Number of events 1 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Investigations Alanine aminotransferase increased	2.2% 1/45 • Number of events 1 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Tumor pain	4.4% 2/45 • Number of events 2 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, specify	11.1% 5/45 • Number of events 5 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Nervous system disorders Cognitive disturbance	2.2% 1/45 • Number of events 1 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Nervous system disorders Transient ischemic attacks	2.2% 1/45 • Number of events 1 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Respiratory, thoracic and mediastinal disorders Adult respiratory distress syndrome	6.7% 3/45 • Number of events 3 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Respiratory, thoracic and mediastinal disorders Dyspnea	2.2% 1/45 • Number of events 1 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Surgical and medical procedures Surgical and medical procedures - Other, specify	4.4% 2/45 • Number of events 2 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Vascular disorders Thromboembolic event	4.4% 2/45 • Number of events 2 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Vascular disorders Phlebitis	2.2% 1/45 • Number of events 1 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.

Other adverse events

Measure	Treatment (Vismodegib) n=45 participants at risk Patients receive vismodegib PO on days 1-28. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity. Laboratory Biomarker Analysis: Correlative studies Pharmacogenomic Study: Correlative studies Vismodegib: Given PO
Blood and lymphatic system disorders ANEMIA	20.0% 9/45 • Number of events 15 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Blood and lymphatic system disorders Blood and lymphatic system disorders - Other, specify	6.7% 3/45 • Number of events 4 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Gastrointestinal disorders Abdominal pain	20.0% 9/45 • Number of events 9 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Gastrointestinal disorders Constipation	17.8% 8/45 • Number of events 10 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Gastrointestinal disorders Diarrhea	28.9% 13/45 • Number of events 19 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Gastrointestinal disorders Dry mouth	6.7% 3/45 • Number of events 3 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Gastrointestinal disorders Gastroesophageal reflux disease	6.7% 3/45 • Number of events 3 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Gastrointestinal disorders Mucositis oral	6.7% 3/45 • Number of events 3 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Gastrointestinal disorders Nausea	37.8% 17/45 • Number of events 20 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Gastrointestinal disorders Stomach pain	6.7% 3/45 • Number of events 5 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Gastrointestinal disorders Vomiting	15.6% 7/45 • Number of events 7 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
General disorders Chills	6.7% 3/45 • Number of events 4 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
General disorders Edema limbs	6.7% 3/45 • Number of events 3 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
General disorders Fatigue	53.3% 24/45 • Number of events 28 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
General disorders Fever	17.8% 8/45 • Number of events 13 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
General disorders General disorders and administration site conditions - Other, specify	6.7% 3/45 • Number of events 3 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Infections and infestations Bronchial infection	8.9% 4/45 • Number of events 4 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Infections and infestations Sepsis	6.7% 3/45 • Number of events 3 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Infections and infestations Urinary tract infection	6.7% 3/45 • Number of events 4 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Investigations Alanine aminotransferase increased	15.6% 7/45 • Number of events 9 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Investigations Alkaline phosphatase increased	11.1% 5/45 • Number of events 5 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Investigations Aspartate aminotransferase increased	11.1% 5/45 • Number of events 5 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Investigations GGT increased	11.1% 5/45 • Number of events 6 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Investigations Lymphocyte count decreased	8.9% 4/45 • Number of events 5 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Investigations Weight loss	26.7% 12/45 • Number of events 12 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Investigations Investigations - Other, specify	11.1% 5/45 • Number of events 8 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Metabolism and nutrition disorders Anorexia	26.7% 12/45 • Number of events 15 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Metabolism and nutrition disorders Hypercalcemia	15.6% 7/45 • Number of events 11 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Metabolism and nutrition disorders Hyperkalemia	11.1% 5/45 • Number of events 6 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Metabolism and nutrition disorders Hypoalbuminemia	13.3% 6/45 • Number of events 9 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Metabolism and nutrition disorders Hypokalemia	6.7% 3/45 • Number of events 3 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Metabolism and nutrition disorders Hypomagnesemia	8.9% 4/45 • Number of events 6 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Metabolism and nutrition disorders Hyponatremia	6.7% 3/45 • Number of events 6 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Musculoskeletal and connective tissue disorders Back pain	6.7% 3/45 • Number of events 3 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Musculoskeletal and connective tissue disorders Myalgia	51.1% 23/45 • Number of events 27 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Tumor pain	17.8% 8/45 • Number of events 8 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, specify	8.9% 4/45 • Number of events 4 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Nervous system disorders Dizziness	6.7% 3/45 • Number of events 3 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Nervous system disorders Dysesthesia	6.7% 3/45 • Number of events 3 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Nervous system disorders Dysgeusia	64.4% 29/45 • Number of events 30 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Nervous system disorders Headache	13.3% 6/45 • Number of events 8 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Nervous system disorders Paresthesia	6.7% 3/45 • Number of events 4 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Psychiatric disorders Anxiety	6.7% 3/45 • Number of events 3 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Psychiatric disorders Confusion	8.9% 4/45 • Number of events 4 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Psychiatric disorders Insomnia	8.9% 4/45 • Number of events 4 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Renal and urinary disorders Urinary frequency	6.7% 3/45 • Number of events 21 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Respiratory, thoracic and mediastinal disorders Adult respiratory distress syndrome	6.7% 3/45 • Number of events 3 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Respiratory, thoracic and mediastinal disorders Cough	8.9% 4/45 • Number of events 4 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Respiratory, thoracic and mediastinal disorders Dyspnea	15.6% 7/45 • Number of events 7 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Skin and subcutaneous tissue disorders Alopecia	40.0% 18/45 • Number of events 18 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Skin and subcutaneous tissue disorders Rash acneiform	6.7% 3/45 • Number of events 3 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Skin and subcutaneous tissue disorders Skin and subcutaneous tissue disorders - Other, specify	6.7% 3/45 • Number of events 3 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Vascular disorders Lymphedema	6.7% 3/45 • Number of events 4 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.
Vascular disorders Thromboembolic event	8.9% 4/45 • Number of events 4 • 14 months of treatment. At the time of primary analysis, 8 were still on treatment and 37 patients discontinued treatment. Adverse event are reported for all treated patients who received at least one administration of treatment.

Additional Information

Pr Italiano Antoine, Department of Medical Oncology

Institut Bergonie

Phone: 0524071947

Email: a.italiano@bordeaux.unicancer.fr

Results disclosure agreements

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

Restriction type: LTE60