CompletedNCT01626209Updated Dec 9, 2020

A Phase I Study of BKM120 in Adult Chinese Patients With Advanced Solid Tumors

A Phase 1 interventional study of BKM120 in Advanced Breast Cancer, Advanced Carcinomas With Squamous Cell Histology, sponsored by Novartis Pharmaceuticals. Completed at 3 sites in China. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2020-12-09.

Sponsored by Novartis Pharmaceuticals · Phase 1, Interventional, and Treatment

Phase: Phase 1
Study type: Interventional
Enrollment: 32
Allocation: Non-randomized
Ages: 18 Years and older
Sex: All

Study summary

Dose escalation study with a dose expansion phase, to evaluate safety, tolerability, pharmacokinetics and preliminary efficacy of two dose levels of BKM120 when administered orally.

Read the detailed description

This is a single arm study, with a starting dose of BKM120 at 80mg/day. Two dose levels: 80mg/day and 100mg /day will be tested in the dose escalation phase. At least 3 patients will be enrolled at each dose level and at least 6 evaluable patients required to be treated at the recommended Phase II dose(RP2D)/MTD dose. After dose escalation the 80mg/day and the 100mg /day dose levels will be expanded to evaluate up to approximately a total of 15 patients each (if 100mg is determined as the RP2D/MTD).

Conditions studied

Advanced Breast Cancer, Advanced Carcinomas With Squamous Cell Histology

Browse trials for

Breast Neoplasms 12,544

Keywords

Breast cancer, NSCLC, SCCHN, esophageal cancer

In context

Breast Neoplasms

12,544 studies on the registry are indexed under Breast Neoplasms; 2,892 are open to participants now.

This study's enrollment of 32 is below the median of 72 across 9,303 interventional studies indexed under Breast Neoplasms.

Browse Breast Neoplasms studies →

Lead sponsor

Novartis Pharmaceuticals is the lead sponsor of 2,673 studies on the registry; 228 are open to participants now.

Of its 576 completed or terminated interventional studies of FDA-regulated products, 431 (75%) have results posted.

Counted across the registry records on this site, refreshed daily.

Who can participate

Ages eligible: 18 Years and older
Sexes eligible: All
Accepts healthy volunteers: No

Inclusion criteria

Patients with histologically-confirmed, advanced unresectable breast cancer or advanced carcinoma with squamous cell histology (including NSCLC, SCCHN, and esophageal) who have progressed on (or not been able to tolerate) standard therapy or for whom no standard anticancer therapy exists
Patient must provide a representative archival or fresh tumor biopsy for shipping to a Novartis designated laboratory for profiling. Note: one block or ≥ 15 unstained slides is required to determine the PI3K activation status. Whenever possible ≥ 20 unstained slides is preferred.
Patient has measurable and/or non-measurable disease as per RECIST v1.1 guidelines for solid tumors
Patient is an adult (female or male) ≥ 18 years of age on the day of consent signature
Patient has an Eastern Cooperative Oncology Group performance status (ECOG PS) ≤ 2

Exclusion criteria

Exclusion Criteria:

Patient has received previous treatment with a PI3K inhibitor
Patient has symptomatic CNS metastases
Patients with asymptomatic CNS metastases may participate in this trial. The patient must have completed any prior local treatment for CNS metastases ≥ 14 days prior to the start of study treatment (including radiotherapy and/or surgery). If the patient is receiving ongoing corticosteroid therapy, the following criteria must be met:
The patient must be receiving a stable or decreasing dose ≤ dexamethasone 4 mg/day or equivalent anti-inflammatory potency of another corticosteroid
The dose of corticosteroid may not have been escalated for at least 14 days before the start of study treatment
Patient is currently receiving increasing or chronic treatment with corticosteroids (>dexamethasone 4 mg or equivalent anti-inflammatory potency of another corticosteroid) or another immunosuppressive agent.
Note: Topical applications (e.g., rash), inhaled sprays (e.g., obstructive airways diseases), eye drops or local injections (e.g., intra-articular) are allowed. Patients with previously treated and asymptomatic brain metastases, are permitted to use corticosteroids as per specific protocol criteria
Patient is currently receiving treatment with drugs known to be moderate or strong inhibitors or inducers of isoenzyme CYP3A. The patient must have discontinued strong inducers for at least one week and must have discontinued strong inhibitors before the treatment is initiated. Switching to a different medication prior to starting study treatment is allowed.
- Other protocol-defined inclusion/exclusion criteria may apply.

Study design

Phase: Phase 1
Primary purpose: Treatment
Allocation: Non-randomized
Intervention model: Single group
Masking: None (open label)
Enrollment: 32 participants (actual)

Study arms

Experimental
BKM120 at: 80 and 100mg/day dose levels
Drug: BKM120

Interventions

DrugBKM120
This is a single arm study, with a starting dose of BKM120 at 80mg/day. Two dose levels: 80mg/day and 100mg /day will be tested in the dose escalation phase. At least 3 patients will be enrolled at each dose level and at least 6 evaluable patients required to be treated at the recommended Phase II dose(RP2D)/MTD dose. After dose escalation the 80mg/day and the 100mg /day dose levels will be expanded to evaluate up to approximately a total of 15 patients each (if 100mg is determined as the RP2D/MTD).

What researchers measure

Primary outcomes

Dose Limiting Toxicity (DLT)
An adaptive Bayesian logistic regression model (BLRM) for dose escalation with overdose control will guide the dose escalation. The recommended dose is the one with the highest posterior probablity of DLT in the target interval(16%,33%) among the doses fulfilling the overdose criterion that there is less than 25 % chance of excessive toxicity. A clinical synthesis of the available toxicity information including adverse event that are not DLTs, Pharmacokinetics, Pharmacodynamics, efficacy as well as the recommnendations from the BLRM will be used to determine the dose.
Time frame: During Cycle 1 (28 days)

Secondary outcomes

Type, frequency and severity of of Adverse Events (AEs) (based on CTCAE version 4.03
Time frame: On a continous basis up to when patient discontinues for progression or until any discontinuation criteria are met e.g AE, patient withdraws consent, Investigator decision; up to 30 days post last study dose
Laboratory and vital sign parameters
Time frame: Every week cycle 1 & 2 then monthly up to when patient discontinues for progression or until any discontinuation criteria are met e.g AE, patient withdraws consent, Investigator decision,
Pharmacokinetics: plasma concentration-time profiles of BKM120 for Cmax
Time frame: Day 1 (predose, postdose : 0.5,1,1.5,2,3,4,6,8,24 hr) Day 8 (predose, postdose : 0.5,1,1.5,2,3,4,6,8,24 hr) and Day 28 (predose, postdose: 0.5,1,1.5,2,3,4,6,8,24 hr)
Pharmacokinetics: plasma concentration-time profiles of BKM120 for Tmax
Time frame: Day 1 (predose, postdose : 0.5,1,1.5,2,3,4,6,8,24 hr) Day 8 (predose, postdose : 0.5,1,1.5,2,3,4,6,8,24 hr) and Day 28 (predose, postdose: 0.5,1,1.5,2,3,4,6,8,24 hr)
Pharmacokinetics: plasma concentration-time profiles of BKM120 for AUCtlast
Time frame: Day 1 (predose, postdose : 0.5,1,1.5,2,3,4,6,8,24 hr) Day 8 (predose, postdose : 0.5,1,1.5,2,3,4,6,8,24 hr) and Day 28 (predose, postdose: 0.5,1,1.5,2,3,4,6,8,24 hr)
Pharmacokinetics: plasma concentration-time profiles of BKM120 for AUCtau
Time frame: Day 1 (predose, postdose : 0.5,1,1.5,2,3,4,6,8,24 hr) Day 8 (predose, postdose : 0.5,1,1.5,2,3,4,6,8,24 hr) and Day 28 (predose, postdose: 0.5,1,1.5,2,3,4,6,8,24 hr)
Pharmacokinetics: plasma concentration-time profiles of BKM120 for AUCinf
Time frame: Day 1 (predose, postdose : 0.5,1,1.5,2,3,4,6,8,24 hr) Day 8 (predose, postdose : 0.5,1,1.5,2,3,4,6,8,24 hr) and Day 28 (predose, postdose: 0.5,1,1.5,2,3,4,6,8,24 hr)
Pharmacokinetics: plasma concentration-time profiles of BKM120 for AUC%Extrap
Time frame: Day 1 (predose, postdose : 0.5,1,1.5,2,3,4,6,8,24 hr) Day 8 (predose, postdose : 0.5,1,1.5,2,3,4,6,8,24 hr) and Day 28 (predose, postdose: 0.5,1,1.5,2,3,4,6,8,24 hr)
Pharmacokinetics: plasma concentration-time profiles of BKM120 for CL/F
Time frame: Day 1 (predose, postdose : 0.5,1,1.5,2,3,4,6,8,24 hr) Day 8 (predose, postdose : 0.5,1,1.5,2,3,4,6,8,24 hr) and Day 28 (predose, postdose: 0.5,1,1.5,2,3,4,6,8,24 hr)
Pharmacokinetics: plasma concentration-time profiles of BKM120 for Racc
Time frame: Day 1 (predose, postdose : 0.5,1,1.5,2,3,4,6,8,24 hr) Day 8 (predose, postdose : 0.5,1,1.5,2,3,4,6,8,24 hr) and Day 28 (predose, postdose: 0.5,1,1.5,2,3,4,6,8,24 hr)
Pharmacokinetics: plasma concentration-time profiles of BKM120 for T1/2acc
Time frame: Day 1 (predose, postdose : 0.5,1,1.5,2,3,4,6,8,24 hr) Day 8 (predose, postdose : 0.5,1,1.5,2,3,4,6,8,24 hr) and Day 28 (predose, postdose: 0.5,1,1.5,2,3,4,6,8,24 hr)
Pharmacokinetics: plasma concentration-time profiles of BKM120 for Vss/F
Time frame: Day 1 (predose, postdose : 0.5,1,1.5,2,3,4,6,8,24 hr) Day 8 (predose, postdose : 0.5,1,1.5,2,3,4,6,8,24 hr) and Day 28 (predose, postdose: 0.5,1,1.5,2,3,4,6,8,24 hr)
Pharmacokinetics: plasma concentration-time profiles of BKM120 for Rsqadj
Time frame: Day 1 (predose, postdose : 0.5,1,1.5,2,3,4,6,8,24 hr) Day 8 (predose, postdose : 0.5,1,1.5,2,3,4,6,8,24 hr) and Day 28 (predose, postdose: 0.5,1,1.5,2,3,4,6,8,24 hr)
Pharmacokinetics: plasma concentration-time profiles of BKM120 for other PK parameters
Time frame: Day 1 (predose, postdose : 0.5,1,1.5,2,3,4,6,8,24 hr) Day 8 (predose, postdose : 0.5,1,1.5,2,3,4,6,8,24 hr) and Day 28 (predose, postdose: 0.5,1,1.5,2,3,4,6,8,24 hr)
Objective Response Rate (ORR)
Evaluated with CT/MRI according to RECIST criteria (RECIST guidelines version 1.1)
Time frame: Every 8 weeks up to when patient discontinues for progression or until any other discontinuation criteria are met e.g AE, patient withdraws consent, Investigator decision
Time to Progression (TTP)
Evaluated with CT/MRI according to RECIST criteria (RECIST guidelines version 1.1)
Time frame: Every 8 weeks up to when patient discontinues for progression or until any other discontinuation criteria are met e.g AE, patient withdraws consent, Investigator decision

Study locations

3 sites

Novartis Investigative Site
Guangzhou, Guangdong 510030, China
Novartis Investigative Site
Beijing, 100021, China
Novartis Investigative Site
Guangzhou, 510060, China

References and documents

Updates

Tracking since Sep 25, 2026

No changes since tracking began. The registry record was last updated on Dec 9, 2020, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗

Registry details

Key details

Study ID: NCT01626209
Lead sponsor: Novartis Pharmaceuticals
Responsible party: Sponsor
First posted: Jun 22, 2012
Start date: Jul 2012
Primary completion: Apr 2014
Completion: Apr 2014
Last update: Dec 9, 2020

Study contacts

Novartis Pharmaceuticals

study director · Novartis Pharmaceuticals

Oversight

Data monitoring committee: Yes

View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Feb 2015. You cannot join it, but the record below documents what was studied.

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Other studies in this condition

Breast Neoplasms→

Other studies from this sponsor