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RAD001 + BEZ235

Phase 1

Advanced Solid Tumors | Small molecule | Oncology |Novartis AG|Last Updated: Dec 21, 2020

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment46
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01482156Dose Finding Study of RAD001 (Everolimus, Afinitor®) in Combination With BEZ235 in Patients With Advanced Solid TumorsPHASE1 COMPLETED 46Jan 1, 2012Feb 1, 2015Dec 21, 202010 United States, Belgium +5
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Study Endpoints
Primary Endpoints
Probability of a Dose Limiting Toxicity (DLT) by the end of the first treatment cycle (DLT)
First treatment cycle (28 days)

The maximum tolerated dose (MTD) and the dose limiting toxicities during the first cycle of treatment

Incidence of DLT in patients by the end of the first treatment cycle in the co-administration of RAD001 and BEZ235
First treatment cycle (28 days)

Frequency of DLTs during the first cycle of treatment

Number of participants with adverse events and serious adverse events.
12 months

Measured by abnormal safety laboratory parameters, changes in electrocardiograms (ECGs), changes in vital signs and changes in physical examination parameters.

Secondary Endpoints
Time versus blood concentration profiles
First treatment cycle ( 28 days)
Overall Response Rate (ORR) (Complete Response (CR) + Partial Response (PR)) according to local assessments by RECIST 1.0 for renal cell carcinoma (RCC) and metastatic breast cancer (MBC) in dose expansion phase
8 weeks
Progresive Free Survival (PFS) according to local assessments by RECIST 1.0 for renal cell carcinoma (RCC) and metastatic breast cancer (MBC) in dose expansion phase
8 weeks
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
RAD001 + BEZ235EXPERIMENTALPatients will receive first dose of RAD001 at 2.5mg/5mg/10 mg weekly or 2.5mg/5mg daily in combination of BEZ235 at 50 mg/100 mg/200 mg/300 mg/400 mg twice a day. In the initial cohort of the dose finding phase, patients will receive a single 2.5 mg dose of RAD001 on Cycle 1 Day 1 and the combination therapy of RAD001 2.5 mg/week and BEZ235 200 mg bid starting on Cycle 1 Day 8. Dose escalation phase: patients will start RAD001 and BEZ235 on Cycle 1 Day 1 with both study drugs being administered at the center. Dose expansion phase: the first 15 patients enrolled at selected sites will take RAD001 as monotherapy from Day 1 to Day 7 (for PK sampling). The combination therapy of RAD001 and BEZ235 will start on Day 8. All remaining patients will receive the combination therapy of RAD001 and BEZ235 starting on Cycle 1 Day 1.
Interventions
NameTypeDescription
RAD001 + BEZ235DRUGRAD001 is formulated as tablets of 2.5 mg and 5 mg strength, blistered in units of 10 tablets (for oral use) each. Blisters should be opened only at the time of dministration as the drug is both hygroscopic and light-sensitive. RAD001 should be administered immediately after a meal with a large glass of water. BEZ235 is supplied as 50-mg, 200-mg, 300-mg and 400-mg sachets (for oral use). BEZ235 is packaged in aluminum foil bags. Bags are packaged in a box. Patients will receive RAD001 in combination with BEZ235.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites10

Inclusion Criteria: * Male and female patients age 18 years or older * In the dose finding phase, patients with histologically or cytologically confirmed advanced solid malignancies that are metastatic or unresectable * In the dose expansion phase, the enrollment will be limited to patients with: ...

Countries:United StatesBelgiumFranceItalyNew ZealandSpainUnited Kingdom
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