UCL-ONCO2012-01

Cliniques universitaires Saint-Luc- Université Catholique de Louvain

Avenue Hippocrate 10, Brussels, Belgium, 1200

Jean-Pascal Machiels, Centre du Cancer, Cliniques universitaires Saint-Luc, 0032 27645457, jean-pascal.machiels@uclouvain.be

Jean-Pascal Machiels, Centre du Cancer, Cliniques universitaires Saint-Luc, 0032 27645457, jean-pascal.machiels@uclouvain.be

No

No

No

Final

16 Jun 2015

No

Yes

16 Jun 2015

No

To assess, in a multicentre phase II trial, the safety and efficacy of BEZ235, an oral pan-class I phosphoinositol-3- kinase (PI3K) and mammalian target of rapamycin (mTOR) complex1/2 inhibitor, in locally advanced or metastatic transitional cell carcinoma (TCC) after failure of platinumbased therapy.

All observed or volunteered adverse events regardless of treatment group or suspected causal relationship to the investigational product(s) were reported. For all adverse events, the investigator must pursue and obtain information adequate both to determine the outcome of the adverse event and to assess whether it meets the criteria for classification as a serious adverse event requiring immediate notification to the study coordinator or its designated representative. For all adverse events, sufficient information should be obtained by the investigator to determine the causality of the adverse event. The investigator is required to assess causality.

15 Nov 2012

Yes

No

Luxembourg: 2

Belgium: 18

20

20

0

0

0

0

0

0

9

11

0

BEZ235 (overall period)

Not applicable

BEZ235

BEZ235

Powder for oral solution in sachet

Oral use

BEZ235, supplied in sachets of 200 mg, 300 mg and 400 mg. The starting dose is 300 mg PO bid. It is administered orally on the same day on a continuous basis twice a day and should be taken immediately after a meal (e.g. breakfast / dinner), 12 ± 2 hours apart (dinner in the evening), at about the same time every day, except cycle 1, day 1. The full cycle is 28 days. Patients were treated until progression or until criteria for discontinuation were met.

BEZ235

BEZ235

The PFS rate was defined as the proportion of patients alive and progression-free at 16 weeks. Patients who did not progress were considered as having stable disease, a partial response or a complete response at 16 weeks, according to RECIST 1.1. Patients who were unable to be evaluated at 16 weeks, because of rapid clinical deterioration or death from any cause, or the start of an additional anti-tumour therapy, were considered as having progressive disease.

Primary

16 weeks

The definition used for PFS was the time interval between the date of inclusion and the date of progressive disease or death from any cause.

Primary

8 weeks

Secondary

Progression free survival (PFS) will be measured from the date of registration to the date of progression or death, whatever the cause. Patients who are still alive and without progression at the time of the analyse will be censored at the date of last fo

Secondary

Overall survival (OS) is defined as the time from the date of registration to the date of death from any cause.

All patients will have a follow-up visit scheduled 28 days after the last dose of the study drug to follow for AEs and SAEs that may have occurred after discontinuation from the study.

For adverse events with a causal relationship to the investigational product, follow-up by the investigator is required until the event or its sequelae resolve or stabilize at a level acceptable to the investigator, and the study coordinator concurs with that assessment.

4.03

BEZ235