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BKM120 + temozolomide

Phase 1

Glioblastoma | Small molecule | Oncology |Novartis AG|Last Updated: Dec 9, 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
CONTROLLED
Total Trials1
Total Enrollment38
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01473901A Phase I Dose Escalation Study of BKM120 With Radiation Therapy and Temozolomide in Patients With Newly Diagnosed GlioblastomaPHASE1 COMPLETED 38Dec 30, 2011May 17, 2017Dec 9, 20208 United States, Australia +2
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Study Endpoints
Primary Endpoints
Dose Limiting Toxicity (DLT)
Concomitant phase (42 days), adjuvant phase cycle 1 (28-day cycle), adjuvant phase cycles 2 (28-day cycle)

Per DLT criteria as defined in protocol

Secondary Endpoints
No of participants with Adverse events based on abnormal laboratory results, abnormal electrocardiogram (ECG) findings
Baseline, 30 days post the last BKM120 treatment
Objective response rate (ORR)
Baseline, 18 months after first BKM120 treatment
Progression free survival (PFS)
at 12 months and at 18 months
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
BKM120 + Temozolomide (Concomitant Phase)EXPERIMENTALCranial radiation: Days 1 - 5 every 7 days for 42 days60 Gy in 30 fractions; Temozolomide: 75 mg/m2 Daily, orally; BKM120: 0, or 40, or 60, or 80 mg/d Daily, orally or Days 1-5 every 7 days, orally
BKM120 + temozolomide with/without radiotherapyEXPERIMENTALAdjuvant phase cycle 1: Temozolomide 150 mg/m2 - Days 1 - 5 every 28 days Daily; BKM120 60, or 80, or 100 mg/d; Adjuvant phase cycle 2+: Temozolomide 200\* mg/m2 - Day 1 \~ 5 every 28 days Daily BKM120 0, or 40, or 60, or 80 or 100 mg/d
Interventions
NameTypeDescription
BKM120 + temozolomideDRUGThe investigational drug, BKM120, will be supplied as 10-mg and 50-mg hard gelatin capsules. BKM120 will be administered on a once daily dosing schedule at a dose of 40 mg, or 60 mg, or 80 mg, or 100 mg (p.o.), in combination with the approved dosing of temozolomide and SoC delivery of cranial irradiation for GBM. The patient will be dosed with BKM120 on a flat scale of mg/day and the dose of BKM120 will not be adjusted to body weight or body surface area. Patients should not eat for 2 hours after the administration of BKM120. Temozolomide in 5 mg, 20 mg, 100 mg, 140 mg, 180 mg or 250 mg capsules will be administered in combination with the investigational drug BKM120.
BKM120 +temozolomide with/without radiotherapyDRUGThe investigational drug, BKM120, will be supplied as 10-mg and 50-mg hard gelatin capsules. BKM120 will be administered on a continuous once daily dosing schedule at a dose of 40 mg, or 60 mg, or 80 mg, or 100 mg (p.o.), in combination with the approved dosing of temozolomide and SoC delivery of cranial irradiation for GBM. The patient will be dosed with BKM120 on a flat scale of mg/day and the dose of BKM120 will not be adjusted to body weight or body surface area. Patients should not eat for 2 hours after the administration of BKM120. Temozolomide in 5 mg, 20 mg, 100 mg, 140 mg, 180 mg or 250 mg capsules will be administered in combination with the investigational drug BKM120.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites8

Inclusion Criteria: * Patient is ≥ 18 years of age on the day of consent signature * Patient with histologically demonstrated, previously untreated glioblastoma * Patient may have received initial treatment for GBM as follows: * For patients enrolled into Stage I, they must have received at leas...

Countries:United StatesAustraliaCanadaSpain
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