Clinical trial NCT01195376: A Study of BEZ235 in Adult Japanese Patients With Advanced Solid Tumors
Official title: A Phase I Study of BEZ235, Administered Orally in Adult Japanese Patients With Advanced Solid Tumors
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Advanced Solid Tumor
Keywords: PI3K
mTOR
Advanced solid tumor
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2010-09-06
Enrollment: 35
Sex: ALL
Minimum age (years): 20
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: In this study, BEZ235 will be administered to adult patients with advanced solid tumors whose disease has progressed despite standard therapy or for whom no standard therapy exists. The trial will confirmed the safety and tolerability and determine the MTD of BEZ235 in Japanese patients.


Eligibility criteria: Inclusion Criteria:

1. Patients with histologically-confirmed, advanced unresectable solid tumors who have progressed on (or not been able to tolerate) standard therapy or for whom no standard anticancer therapy exists.
2. At least one measurable lesion as defined by RECIST criteria for solid tumors.
3. Age ≥ 20
4. Eastern Cooperative Oncology Group Performance Status (ECOG P.S.) of ≤ 2
5. Life expectancy of ≥ 12 weeks
6. Patients must have the laboratory values Patients in the expansion part of this study have to meet the following criteria in addition to the criteria described above.
7. Availability of a representative tumor tissue specimen (either archival tumor or fresh tumor biopsy) for pre-screening.
8. Patients whose molecular status proved to meet the criteria (PIK3CA mutation/amplification and/or PTEN mutation and/or low/null PTEN expression) during pre-screening.

Exclusion Criteria:

1. Patients who have brain metastases or who have signs/symptoms attributable and have not been assessed with radiologic imaging to rule out the presence of brain metastases
2. Patients with any peripheral neuropathy ≥ CTCAE grade 2
3. Patients with unresolved diarrhea ≥ CTCAE grade 2
4. Patients with a history of photosensitivity reactions to other drugs
5. Women of child-bearing potential who are pregnant or breast feeding or adults of reproductive potential not employing an effective method of birth control. Barrier contraceptives must be used throughout the trial in both sexes. Women of child-bearing potential, defined as sexually mature women who have not undergone a hysterectomy or who have not been naturally postmenopausal for at least consecutive 1 years (i.e., who has had menses any time in the preceding consecutive 2 years), must have a negative serum pregnancy test ≤ 7 days prior to starting BEZ235.

Other protocol-defined inclusion/exclusion criteria may apply

Primary outcomes:
measure: To establish Maximum tolerate dose (MTD)
timeFrame: Every week
Secondary outcomes:
measure: Safety assessed by type, frequency and severity of adverse events
timeFrame: Every week
measure: Efficacy assessed by RECIST
timeFrame: Every 2 months
measure: To characterize the PK profiles
timeFrame: Every 2 weeks
measure: To assess the biomarkers
timeFrame: Every 2 months
Other outcomes:
Locations:
city: Kobe-city
country: Japan
facility: Novartis Investigative Site
state: Hyogo
city: Yufu
country: Japan
facility: Novartis Investigative Site
state: Oita
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References:
citation: Toyoda M, Watanabe K, Amagasaki T, Natsume K, Takeuchi H, Quadt C, Shirao K, Minami H. A phase I study of single-agent BEZ235 special delivery system sachet in Japanese patients with advanced solid tumors. Cancer Chemother Pharmacol. 2019 Feb;83(2):289-299. doi: 10.1007/s00280-018-3725-2. Epub 2018 Nov 16.