Head Neck Cancer Squamous Cell MetastaticPhase 2NCT01527877
Study of BKM120 in Advanced Squamous Cell Carcinoma of Head and Neck
A Phase 2 study testing BKM120 in people with Head Neck Cancer Squamous Cell Metastatic and Head Neck Cancer Squamous Cell Recurrent, run by Yonsei University at 1 site in South Korea.
Trial summary
This study is to evaluate disease control rate (DCR) at 8 weeks of BKM120 administered as therapy for patient with recurrent/metastatic head and neck squamous cell carcinoma. Read more about this study
- Who is it for?
- Ages 20 to 80 years
- Conditions the study is for
- Head Neck Cancer Squamous Cell Metastatic, Head Neck Cancer Squamous Cell Recurrent
- What is being tested?
- BKM120
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 53 participants
- Where
- 1 siteSouth Korea
- Study dates
- Sep 2012to Aug 2014
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 20 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Histologically or cytologically confirmed recurrent or metastatic squamous-cell carcinoma of head and neck (SCCHN), except nasopharyngeal carcinoma
- Disease not amenable to curative treatment (surgery or radiation for curative intent)
- 20 years of age or older
- Progressive disease defined as follows
- after one or two prior chemotherapy regimens including platinum-based chemotherapy given for palliation
- within 6 months after concurrent chemoradiotherapy (including induction chemotherapy) delivered as part of primary treatment.
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- Life expectancy of at least 12 weeks
- At least one measurable lesion according to the RECIST 1.1 criteria.
- ECOG performance score of 0 ~ 2
- Adequate organ function
- Absolutely Neutrophil Count (ANC) ≥ 1.5 x 109/L, Platelets ≥ 100 x 109/L, Hemoglobin ≥ 9.0 g/dL
- Serum Creatinine ≤ 1.5 x ULN
- Adequate liver function (total bilirubin ≤ 2.0 x ULN, AST and ALT ≤ 2.0 x ULN or < 5.0 x ULN if liver metastases are present)
- Availability of tissue samples (archival tissue or rebiopsied tissues) for molecular analysis (representative paraffin block or unstained sections from tumor diagnostic specimen are mandatory)
- Patients who have will and ability to comply with the scheduled visits, the treatment plan, laboratory tests and any other trial procedures
- Patient's informed consent
Who cannot join
- Nasopharyngeal carcinoma
- More than two prior lines of chemotherapy in the palliative setting.
- Uncontrolled, untreated brain metastasis Patients with controlled and asymptomatic CNS metastases may participate in this trial. The patient must have completed any prior treatment for CNS metastases ≥ 28 days (must include radiotherapy and/or surgery) and, if on corticosteroid therapy, should be receiving a stable low dose (e.g. dexamethasone 4 mg or equivalent dose of another corticosteroid for at least 14 days before start of study treatment)
- Surgery, chemotherapy or irradiation within 4 weeks of study entry
- Prior treatment with any investigational drug within the preceding 4 weeks
- Concomitant chemotherapy, hormonal therapy or immunotherapy
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- Previous or concomitant malignant disease, except adequately treated basal cell cancer of the skin or cervical cancer in situ, superficial bladder tumors (Ta, Tis \& T1) or any cancer curatively treated > 5 years prior study entry
- Patient who cannot take the oral drug
- Patient is pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test (> 5 mIU/mL).
- Clinically significant psychological disorders including mood and anxiety disorders judged by psychiatry physician
- Patient who have not recovered to grade 1 or better from any adverse events (except alopecia) related to previous antineoplastic therapy before screening procedures are initiated
- Severe acute or chronic medical condition or laboratory abnormality that may increase the risk associated with trial participation or investigational product administration or may interfere with the interpretation of trial results and, in the judgment of the investigator, would make the patient inappropriate for entry into this trial.
- Patient has poorly controlled diabetes mellitus (HbA1c> 8 %)
- Patient has history of cardiac dysfunction including history of documented congestive heart failure (New York Heart Association functional classification III-IV) and documented cardiomyopathy
- Patient is currently receiving treatment with medication that has a known risk to prolong the QT interval or inducing Torsades de Pointes. * Active infection, inflammatory bowel disease
- Inadequate liver function (total bilirubin ≥ 2.0 x ULN, AST and ALT ≥ 2.0 x ULN or ≥ 5.0 x ULN if liver metastases are present)
Trial locations and contacts
1 site in 1 country.
South Korea1 site, 1 recruiting
- Recruiting
Severance Hospital
Seoul
Byoung Chul Cho, MD, 82-2-2228-8126, cbc1971@yuhs.ac
Byoung Chul Cho, M.D., Ph.D.
About this study
Official title: An Open Label, Single Arm, Multicenter Phase II Study of BKM120 in Patients With Recurrent or Metastatic Squamous Cell Carcinoma of Head and Neck Who Failed to Respond to Platinum-based Therapy
This study is to evaluate disease control rate (DCR) at 8 weeks of BKM120 administered as therapy for patient with recurrent/metastatic head and neck squamous cell carcinoma.
Read the detailed description
One promising approach to the treatment of cancer is inhibition or modulating the crucial signal transduction pathway of PI3K-Akt-mTOR. Several PI3K inhibitors are being tested in the clinical trials for cancer treatment but not for the head and neck cancer yet. BKM120 is a specific Pan-class I PI3K inhibitor. We suggest multicenter single arm phase II study to determine anti-tumor effects of BKM120 in patients with recurrent and/or metastatic SCCHN who failed to prior platinum-based chemotherapy regimens.
- Being tested
- BKM120
- Keywords
- BKM120, head and neck cancer, squamous cell carcinoma
- Medical subject headings
- Head and Neck Neoplasms, Carcinoma, Squamous Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BKM120Drug
Also called NVP-BKM120, BKM-120
Patients will be instructed to take BKM120 orally at a dose of 100 mg with a glass of water once daily, in a fasting state or with a light fat-free meal, and as close as possible to the same time each day. The patient will be dosed on a flat scale of mg/day and not be adjusted to body weight or body surface area. If vomiting occurs no attempt should be made to replace the dose.
- BKM120 should be taken 1-hour following a light meal. Please note that patients must avoid consumption of Seville orange (and juice), grapefruit or grapefruit juice, grapefruit hybrids, pummelos and exotic citrus fruits from 7 days prior to the first dose of study drug and during the entire study treatment period due to potential CYP3A4 interaction. Regular orange juice is allowed.
| Study group | Role | What they receive |
|---|---|---|
| BKM120 | Experimental | Not described Receives: Drug: BKM120 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Disease control rate at 8 weeks
The disease control rate (DCR) is defined as the proportion of randomized patients achieving a best overall response of PR or CR or SD, defined by RECIST criteria (version 1.1), relative to the total number of patients in the considered analysis population (ITT).
Measured: Eight weeks after administration of the drug
Secondary outcomes (6)
Overall response rate (ORR)
Overall objective response rate (ORR) is the best response rate stipulated as complete response (CR) or partial response (PR) (target lesion and tumor response defined according to RECIST guideline version 1.1) and identified as percentage of the confirmed patients.
Measured: Every 8 weeks from date of first treatment until date of last treatment up to 24 months
Toxicity profile
From C1D1 to 1 months after the last dose adminitration
Overall safety profile verified as relevance of adverse events and laboratory abnormality in the study and grades granted based on (USA National Cancer Center) Common Terminology Criteria for Adverse Events such as the type, frequency and severity (CTCAE), v4.0.
Measured: Every 4 weeks from date of first treatment until date of last treatment up to 24 months
Overall survival
From C1D1 to death
Measured: Every 8 weeks from date of first treatment until the date of death from any cause, assessed approximately up to 24 months
Progression-free survival
From C1D1 until confirmed disease progression or death
Measured: Every 8 weeks from date of first treatment until the date of first documented progression or date of death from any cause, whichever came first, assessed approximately up to 24 months
Quality of life assessment
Quality of life assessment will be performed using FACT-HN\& questionnaire
FACT-H\&N questionnaire includes physical well-being (PWB), social/family well-being (SWB), emotional well-being (EWB), functional well-being (FWB), and head \& neck cancer subscale (HNCS).
Patients will be evaluation on baseline, day 1 of every cycle (4 weeks), and end of treatment.
Measured: Every 4 weeks from date of first treatment until the date of death from any cause, assessed approximately up to 24 months
Time to progression (TTP)
From C1D1 until confirmed disease progression.
Measured: Every 8 weeks from date of first treatment until the date of first documented progression, assessed approximately up to 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Feb 2012 (estimated)
- Study startSep 2012
- Primary completionDec 2013 (estimated)
- Study completionAug 2014 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Head Neck Cancer Squamous Cell MetastaticStudies of the same condition, open and under way.
- More trials run by Yonsei UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 53 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Yonsei University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Byoung Chul Cho, Yonsei University
- Investigators
- Byoung Chul Cho, M.D., Ph.D., Principal investigator, Yonsei University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01527877
- Sponsor's protocol ID
- 2011-0828-001
- Status last verified
- Oct 2012
- First submitted
- 26 Jan 2012
- Last updated
- 8 Oct 2012
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Kim HR, Kang HN, Yun MR, Ju KY, Choi JW, Jung DM, Pyo KH, Hong MH, Ahn MJ, Sun JM, Kim HS, Kim J, Yoo J, Kim KR, Koh YW, Kim SH, Choi EC, Yoon SO, Shim HS, Paik S, Kim TM, Cho BC. Mouse-human co-clinical trials demonstrate superior anti-tumour effects of buparlisib (BKM120) and cetuximab combination in squamous cell carcinoma of head and neck. Br J Cancer. 2020 Dec;123(12):1720-1729. doi: 10.1038/s41416-020-01074-2. Epub 2020 Sep 23.
PubMed 32963347
Study at a glance
From the registry record, last updated by the sponsor on 8 Oct 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label, Single Arm, Multicenter Phase II Study of BKM120 in Patients With Recurrent or Metastatic Squamous Cell Carcinoma of Head and Neck Who Failed to Respond to Platinum-based Therapy
- ClinicalTrials.gov ID
- NCT01527877
- Acronym / sponsor ID
- 2011-0828-001
- Keywords
- BKM120, head and neck cancer, squamous cell carcinoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 53 participants (estimated)
- Lead sponsor
- Yonsei UniversityUniversity, hospital or other
- Study start
- Sep 2012
- Primary completion
- Dec 2013 (estimated)
- Study completion
- Aug 2014 (estimated)
- First posted
- 7 Feb 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is to evaluate disease control rate (DCR) at 8 weeks of BKM120 administered as therapy for patient with recurrent/metastatic head and neck squamous cell carcinoma.
Detailed description
One promising approach to the treatment of cancer is inhibition or modulating the crucial signal transduction pathway of PI3K-Akt-mTOR. Several PI3K inhibitors are being tested in the clinical trials for cancer treatment but not for the head and neck cancer yet. BKM120 is a specific Pan-class I PI3K inhibitor. We suggest multicenter single arm phase II study to determine anti-tumor effects of BKM120 in patients with recurrent and/or metastatic SCCHN who failed to prior platinum-based chemotherapy regimens.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| BKM120 | Experimental | Drug: BKM120 |
Who may qualify
- Ages
- 20 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (16 listed)
- Histologically or cytologically confirmed recurrent or metastatic squamous-cell carcinoma of head and neck (SCCHN), except nasopharyngeal carcinoma
- Disease not amenable to curative treatment (surgery or radiation for curative intent)
- 20 years of age or older
- Progressive disease defined as follows
- Life expectancy of at least 12 weeks
Key exclusion criteria (16 listed)
- Nasopharyngeal carcinoma
- More than two prior lines of chemotherapy in the palliative setting.
- Uncontrolled, untreated brain metastasis Patients with controlled and asymptomatic CNS metastases may participate in this trial. The patient must have completed any prior treatment for CNS metastases ≥ 28 days (must include radiotherapy and/or surgery) and, if on corticosteroid therapy, should be receiving a stable low dose (e.g. dexamethasone 4 mg or equivalent dose of another corticosteroid for at least 14 days before start of study treatment)
- Surgery, chemotherapy or irradiation within 4 weeks of study entry
- Prior treatment with any investigational drug within the preceding 4 weeks
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BKM120Also: NVP-BKM120, BKM-120Patients will be instructed to take BKM120 orally at a dose of 100 mg with a glass of water once daily, in a fasting state or with a light fat-free meal, and as close as possible to the same time each day. The patient will be dosed on a flat scale of mg/day and not be adjusted to body weight or body surface area. If vomiting occurs no attempt should be made to replace the dose. • BKM120 should be taken 1-hour following a light meal. Please note that patients must avoid consumption of Seville orange (and juice), grapefruit or grapefruit juice, grapefruit hybrids, pummelos and exotic citrus fruits from 7 days prior to the first dose of study drug and during the entire study treatment period due to potential CYP3A4 interaction. Regular orange juice is allowed. | Drug | BKM120 |
Primary outcome
Disease control rate at 8 weeks
Time frame: Eight weeks after administration of the drug
The disease control rate (DCR) is defined as the proportion of randomized patients achieving a best overall response of PR or CR or SD, defined by RECIST criteria (version 1.1), relative to the total number of patients in the considered analysis population (ITT).
6 secondary outcomes
Overall response rate (ORR)
Time frame: Every 8 weeks from date of first treatment until date of last treatment up to 24 months
Toxicity profile
Time frame: Every 4 weeks from date of first treatment until date of last treatment up to 24 months
Overall survival
Time frame: Every 8 weeks from date of first treatment until the date of death from any cause, assessed approximately up to 24 months
Progression-free survival
Time frame: Every 8 weeks from date of first treatment until the date of first documented progression or date of death from any cause, whichever came first, assessed approximately up to 24 months
Quality of life assessment
Time frame: Every 4 weeks from date of first treatment until the date of death from any cause, assessed approximately up to 24 months
Time to progression (TTP)
Time frame: Every 8 weeks from date of first treatment until the date of first documented progression, assessed approximately up to 24 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Oct 2012.
- Derived from this study: PubMed 32963347Kim HR, Kang HN, Yun MR, Ju KY, Choi JW, Jung DM, Pyo KH, Hong MH, Ahn MJ, Sun JM, Kim HS, Kim J, Yoo J, Kim KR, Koh YW, Kim SH, Choi EC, Yoon SO, Shim HS, Paik S, Kim TM, Cho BC. Mouse-human co-clinical trials demonstrate superior anti-tumour effects of buparlisib (BKM120) and cetuximab combination in squamous cell carcinoma of head and neck. Br J Cancer. 2020 Dec;123(12):1720-1729. doi: 10.1038/s41416-020-01074-2. Epub 2020 Sep 23.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Byoung Chul Cho, M.D., Ph.D. principal investigator
Yonsei University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing BKM120 in people with Head Neck Cancer Squamous Cell Metastatic and Head Neck Cancer Squamous Cell Recurrent, run by Yonsei University at 1 site in South Korea.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 16 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 8 Oct 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Byoung Chul Cho, M.D., Ph.D. (82-2-2228-8126, cbc1971@yuhs.ac), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Disease control rate at 8 weeks (time frame: Eight weeks after administration of the drug). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01527877, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Oct 2012.