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Buparlisib

Phase 2

Diffuse Large B-cell Lymphoma, Mantle Cell Lymphoma, Follicular Lymphoma | Small molecule | Oncology |Novartis AG|Last Updated: Dec 9, 2020

Success Probability

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Market & Valuation

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Trial Design

CONTROLLED
Total Trials1
Total Enrollment72

FDA Designations

No designations recorded

Clinical trial landscape

Buparlisib · 3 trials · 3 indications

Phase 2 1Phase 1 2
NCT01693614Safety and Efficacy of BKM120 in Relapsed and Refractory NHLDiffuse Large B-cell Lymphoma, Mantle Cell Lymphoma, Follicular Lymphoma
COMPLETED72 Analytics
PHASE2COMPLETED
Safety and Efficacy of BKM120 in Relapsed and Refractory NHL
Diffuse Large B-cell Lymphoma, Mantle Cell Lymphoma, Follicular LymphomaUnlock trial analytics

Study Endpoints

Primary Endpoints

Overall Response Rate (ORR) and Disease Control Rate (DCR) Per Investigator at 6 Months (FAS)
Baseline up to 6 months

Overall Response rate is the percentage of patients in a cohort who experienced either complete response (CR) or partial response (PR) during their follow-up after treatment start divided by the total percentage of patients included in the corresponding cohort according to Cheson criteria The analysis for each cohort was based on an exact binomial test comparing the ORR to the reference level of 10% (null hypothesis) in the FAS. The test for each cohort used a significance level of 5%. The ORR was presented together with an exact 95% Clopper- Pearson confidence interval. Disease Control Rate (DCR progressive. Disease Control Rate (DCR) was the percentage of patients with CR, PR or SD (stable disease). Patients for whom the best response after treatment start was missing, unknown (UNK) or progressive disease (PD) were considered non-responders and were counted in the denominator for the estimation of the ORR

Plasma pharmacokinetic (PK) parameter Cmax
predose, 10, 30 min, 1, 1.5, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144, 192, 240 hours post-dose

Measurement of effect of renal impairment on PK of buparlisib by assessment of the maximum plasma concentration (PK parameter Cmax). Cmax directly determined from the plasma concentration-time profiles.

Plasma PK parameter AUCinf
predose, 10, 30 min, 1, 1.5, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144, 192, 240 hours post-dose

Measurement of effect of renal impairment on PK of buparlisib by assessment of the PK parameter AUCinf (area under the plasma concentration-time curve from time 0 to infinity). AUC determined from the plasma concentration-time profile using non-compartmental analysis.

Plasma PK parameter AUC0-t
predose, 10, 30 min, 1, 1.5, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144, 192, 240 hours post-dose

Measurement of effect of renal impairment on PK of buparlisib by assessment of the PK parameter AU0-t (area under the plasma-concentration time curve from timepoint 0 to time t). AUC determined from the plasma concentration-time profile using non-compartmental analysis.

Plasma PK parameter CL/F
predose, 10, 30 min, 1, 1.5, 2, 3, 4, 6, 8, 12, 24, 48, 72, 96, 144, 192, 240 hours post-dose

Measurement of effect of renal impairment on PK of buparlisib by assessment of the PK parameter CL/F (clearance).

Urine PK parameter CLR
predose, 0-4 h, 4-8 h, 8-12 h, 12-24 h, 24-48 h, 48-72 h, 72-96 h, 96-120 h, 120-144 h

Measurement of effect of renal impairment on PK of buparlisib by assessment of the urine clearance CLR.

Number of Total Dose-limiting Toxicity (DLT) during Dose Escalation part to determine Maximum Tolerated Dose (MTD) [Phase Ib]
Cycle 1 (21 days carboplatin combination or 42 days lomustine combination )

Maximum Tolerated Dose (MTD) is defined as the highest BKM120 dosage that does not cause medically unacceptable Dose Limiting Toxicities (DLTs) in more than 35% of the treated patients during the first cycle of treatment. DLT is defined as treatment-related toxicity occurring during the phase Ib cycle 1 and meeting specific protocol-predefined criteria. The information will be integrated in a Bayesian logistic regression model with overdose control to estimate the MTD.

12 week Progression Free Survival (PFS) rate (Phase II- Carboplatin combination)
12 weeks

12-week Progression Free Survival (PFS) is defined as the percentage of patients who are progression free 12 weeks after the date of the start of the treatment ("success"). Patients who progressed, died or discontinued within the 12 weeks of observation are counted as "failure".

Progression Free Survival (PFS) [phase II lomustine combinations]
Randomization until date of the event (expected average 3 months).

Progression Free Survival (PFS) is defined as the time from date of randomization to the date of the event, which is the first radiologically documented disease progression \[per local investigator assessment according to Response Assessment in Neuro-Oncology (RANO) criteria\] or death due to any cause.

Secondary Endpoints

Progression- Free Survival (PFS) Based on Investigator Assessment (FAS)
Baseline up to approximately 44 months
Duration of Response for Diffuse Large B-cell Lymphoma (DLBCL), and Follicular Lymphoma (FL) Cohorts (FAS)
Baseline up to approximately 18 months
Overall Survival (OS) - Percentage of Participants With OS Events (FAS)
Baseline up to approximately 44 months
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
DLBCL CohortEXPERIMENTALDiffuse large B-cell lymphoma cohort
MCL CohortEXPERIMENTALMantle cell lymphoma cohort
FL CohortEXPERIMENTALFollicular lymphoma cohort
Moderate renal impairment groupEXPERIMENTALSubjects with moderate renal impairment defined as eGFR of 30-59 mL/min/1.73m2 at screening can be enrolled in this group
Severe renal impairment groupEXPERIMENTALSubjects with severe renal impairment defined as eGFR of 15-29 mL/min/1.73m2 at screening can be enrolled in this group
Matching healthy control groupEXPERIMENTALSubjects with normal renal function defined as eGFR ≥ 90 mL/min/1.73m2 at screening and matching to the renal impaired subject based on gender, race, age, and weight can be enrolled in this group.
Lomustine+ buparlisib (Phase Ib)EXPERIMENTAL -
carboplatin+ buparlisib (Phase Ib)EXPERIMENTAL -
lomustine+ buparlisib (Phase II)EXPERIMENTAL -
lumustine + placebo (Phase II)PLACEBO_COMPARATOR -
carboplatin+ buparlisib (Phase II)EXPERIMENTAL -

Interventions

NameTypeDescription
BuparlisibDRUG100 mg hard gelatin capsules administered orally, once daily in cycles of 28 days
carboplatinDRUGCarboplatin intravenous infusion will be administered at a dose of AUC 5 in a 21 day cycle (every 3 weeks).
lomustineDRUGLomustine will be administered as a single oral dose of 100 mg/m² every 6 weeks in a 42 day cycles.
placeboDRUGPlacebo will be administered orally on a continuous QD dosing schedule for cycles of 42 days. Buparlisib matching placebo is manufactured as 10 mg and 50 mg hard gelatin capsules.
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites20

Inclusion Criteria: 1. Patient had a histologically confirmed diagnosis of mantle cell lymphoma, follicular lymphoma, or diffuse large B cell lymphoma. 2. Patient had relapsed or refractory disease and received at least one prior therapy. 3. Patient with diffuse large B cell lymphoma had received o...

Countries:United StatesBelgiumFranceGermanyItalySouth KoreaSpainTurkey (Türkiye)BulgariaCzechiaRomaniaAustraliaCanada
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Frequently asked questions about Buparlisib

What is Buparlisib used for?

Buparlisib is an investigational small molecule being studied for use in oncology. It has been evaluated in clinical trials for recurrent glioblastoma multiforme, diffuse large B-cell lymphoma, mantle cell lymphoma, follicular lymphoma, and renal impairment. It is not approved by the FDA and remains in clinical development.

What does Buparlisib target?

Buparlisib is a small molecule that targets PI3K, a key enzyme in the PI3K/AKT/mTOR signaling pathway involved in cell growth and survival. By inhibiting PI3K, it aims to disrupt cancer cell proliferation. This mechanism is being studied across several cancer types.

Who makes Buparlisib?

Buparlisib is being developed by Novartis AG, a multinational pharmaceutical company traded on the New York Stock Exchange under the ticker symbol NVS. Novartis is conducting clinical trials to evaluate the drug's safety and efficacy in various oncology indications.

What phase is Buparlisib in?

Buparlisib has completed Phase 1 and Phase 2 clinical trials. The most advanced study, a Phase 2 trial in relapsed and refractory non-Hodgkin lymphoma, is completed. It is not FDA approved and remains an investigational drug in clinical development.

What clinical trials is Buparlisib in?

Buparlisib has been studied in three completed trials: NCT01693614, a Phase 2 study in diffuse large B-cell lymphoma, mantle cell lymphoma, and follicular lymphoma; NCT01934361, a Phase 1b/2 study in recurrent glioblastoma multiforme; and NCT02048787, a Phase 1 pharmacokinetic study in renal impairment.

Is Buparlisib the same as BKM120?

Yes, Buparlisib is also known as BKM120. In clinical trial NCT01693614, the drug is referred to as BKM120. This alternative name is used in some research contexts, so patients and researchers may encounter either designation for the same investigational compound.