MelanomaPhase 2NCT01894672
BRAF Inhibitor, LGX818, Utilizing a Pulsatile Schedule in Patients With Stage IV or Unresectable Stage III Melanoma Characterized by a BRAFV600 Mutation
A Phase 2 study testing LGX818 in people with Melanoma, run by Memorial Sloan Kettering Cancer Center at 5 sites in United States.
Trial summary
The purpose of this phase II study is to find out if an investigational drug called LGX818 can stop the melanoma from growing. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Melanoma
- What is being tested?
- LGX818
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 5 sitesUnited States
- Study dates
- Jul 2013to 17 Mar 2016
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Stage IV, or unresectable stage III melanoma that harbors a BRAFV600 mutation
- Any prior therapy allowed except a BRAF or MEK inhibitor,.
- Patients must provide written informed consent prior to any screening procedures.
- Age 18 years or older.
- Willing and able to comply with scheduled visits, treatment plan and laboratory tests
- Patient is able to swallow and retain oral medication
- Measurable disease according to RECIST v1.1
- ECOG performance status ≤ 1
Who cannot join
- Brain metastasis or leptomeningeal disease
- Known acute or chronic pancreatitis
- Prior colectomy
- Clinically significant cardiac disease including any of the following:
- CHF requiring treatment (NYHA Classification ≥ 2) in which patients have a history of LVEF < 45% as determined by MUGA scan or ECHO, or uncontrolled hypertension (please refer to WHO-ISH guidelines)
- History or presence of clinically significant ventricular arrhythmias or atrial fibrillation
Show all 34Show fewer
- Clinically significant resting bradycardia
- Unstable angina pectoris ≤ 3 months prior to starting study drug
- Acute Myocardial Infarction (AMI) ≤ 3 months prior to starting study drug
- QTcF> 480 msec
- Patients with any of the following laboratory values at Screening/baseline:
- Absolute neutrophil count (ANC) <1,500/mm3 [1.5 x 109/L]
- Platelets <100,000/mm3 [100 x 109/L]
- Hemoglobin < 9.0 g/dL
- Serum creatinine>1.5 x ULN
- Serum total bilirubin >1.5 x ULN
- AST/SGOT and/or ALT/SGPT > 2.5 x ULN
- Impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of oral LGX818 (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, small bowel resection).
- Previous or concurrent malignancy. Exceptions: adequately treated basal cell or squamous cell skin cancer; in situ carcinoma of the cervix, treated curatively and without evidence of recurrence for at least 3 years prior to study entry; or other solid tumor treated curatively, and without evidence of recurrence for at least 3 years prior to study entry.
- 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).
- Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, are not allowed to participate in this study UNLESS they are using highly effective methods of contraception throughout the study and for 3 months after study drug discontinuation. Highly effective contraception methods include:
- Total abstinence or
- Male or female sterilization
- Combination of any two of the following (a+b or a+c or b+c)
- Use of oral, injected, or implanted hormonal methods of contraception
- Placement of an intrauterine device (IUD) or intrauterine system (IUS)
- Barrier methods of contraception: condom or occlusive cap (diaphragm or cervical/vault caps) with spermicidal foam/gel/film/cream/vaginal suppository
- Post-menopausal women are allowed to participate in this study. Women are considered post-menopausal and not of child bearing potential if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (e.g. age appropriate, history of vasomotor symptoms) or six months of spontaneous amenorrhea with serum Follicle-Stimulating Hormone (FSH) levels > 40 mIU/mL or have had surgical bilateral oophorectomy (with or without hysterectomy) or tubal ligation at least six weeks prior to screening. In the case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment is she considered not of child bearing potential.
- Sexually active males must use a condom during intercourse while taking the drug and for 5 T1/2 after stopping treatment and should not father a child in this period. A condom is required to be used also by vasectomized men in order to prevent delivery of the drug via seminal fluid.
- History of thromboembolic or cerebrovascular events within the last 6 months, including transient ischemic attack, cerebrovascular accident, deep vein thrombosis, or pulmonary embolism.
- Patients who have undergone any major surgery within the last 2 weeks prior to starting study drug or who would not have fully recovered from previous surgery.
- Known Human Immunodeficiency Virus (HIV) infection
- Other severe, acute, or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or study drug administration or that may interfere with the interpretation of study results and, in the judgment of the investigator, would make the patient inappropriate for the study.
- Treatment with a prior BRAF or MEK inhibitor
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Memorial Sloan Kettering Cancer Center at Basking Ridge
Basking Ridge, New Jersey
Memorial Sloan Kettering Cancer Center at Commack
Commack, New York, 11725
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
Memorial Sloan Kettering Cancer Center at Mercy Medical Center
Rockville Centre, New York, 11570
Memorial Sloan Kettering Cancer Center at Phelps Memorial Hospital Center
Sleepy Hollow, New York, 10591
About this study
Official title: A Phase 2 Trial of the BRAF Inhibitor, LGX818, Utilizing a Pulsatile Schedule in Patients With Stage IV or Unresectable Stage III Melanoma Characterized by a BRAFV600 Mutation
The purpose of this phase II study is to find out if an investigational drug called LGX818 can stop the melanoma from growing.
- Conditions
- Melanoma
- Being tested
- LGX818
- Keywords
- LGX818, BRAF Inhibitor, Stage IV melanoma, unresectable stage III melanoma, BRAFV600 mutation, 13-053
- Medical subject headings
- Melanoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LGX818Drug
| Study group | Role | What they receive |
|---|---|---|
| LGX818 | Experimental | LGX818 capsules will be administered orally on a once -daily schedule (QD) dosing at a dose of 300 mg/day, 2 weeks on followed by a 2 week break. This schedule of 2 weeks on, followed by 2 weeks off, will continue for the duration of time the patients remains on the clinical trial. After the follow up visit patients will be contacted approximately every 12 weeks until they have received a subsequent therapy or until they have been off treatment for a year to monitor their survival. Receives: Drug: LGX818 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Response According to RECIST v1.1 Criteria
Efficacy for all patients will be evaluated by the study sites using RECIST v1.1 and response criteria based on contrast-enhanced CT. Complete Response (CR): Disappearance of all target lesions. Partial Response (PR): At least a 30% decrease in the sum of the longest diameter (LD) of target lesions, taking as reference the baseline sum LD. Progressive Disease (PD): At least a 20% increase in the sum of the LD of target lesions, taking as reference the smallest sum LD recorded since the treatment started or the appearance of one or more new lesions. Progression must also involve an increase in size of measurable lesions by at least 5 mm, to minimize the possibility that small changes in a small number of target lesions is falsely interpreted as progression.Stable Disease (SD): Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum LD since the treatment started.
Measured: 1.5 years
Secondary outcomes (1)
Response Rate
Response rate (defined as complete + partial response) and 95% confidence interval will be estimated.
Measured: 1.5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jul 2013 (estimated)
- Study startJul 2013
- Primary completion17 Mar 2016
- Study completion17 Mar 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for MelanomaStudies of the same condition, open and under way.
- More trials run by Memorial Sloan Kettering Cancer CenterThe 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
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Memorial Sloan Kettering Cancer Center
- Sponsor type
- University, hospital or other
- Collaborators
- Array BioPharma
- Responsible party
- Sponsor
- Investigators
- Paul Chapman, MD, Principal investigator, Memorial Sloan Kettering Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01894672
- Sponsor's protocol ID
- 13-053
- Status last verified
- Dec 2019
- First submitted
- 3 Jul 2013
- Last updated
- 16 Jul 2024
- Results posted
- Yes, on ClinicalTrials.gov
Study at a glance
From the registry record, last updated by the sponsor on 16 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Trial of the BRAF Inhibitor, LGX818, Utilizing a Pulsatile Schedule in Patients With Stage IV or Unresectable Stage III Melanoma Characterized by a BRAFV600 Mutation
- ClinicalTrials.gov ID
- NCT01894672
- Acronym / sponsor ID
- 13-053
- Conditions
- Melanoma
- Keywords
- LGX818, BRAF Inhibitor, Stage IV melanoma, unresectable stage III melanoma, BRAFV600 mutation, 13-053
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Memorial Sloan Kettering Cancer CenterUniversity, hospital or other
- Collaborators
- Array BioPharma
- Study start
- Jul 2013
- Primary completion
- 17 Mar 2016 (actual)
- Study completion
- 17 Mar 2016 (actual)
- First posted
- 10 Jul 2013 (estimated)
- Results
- Posted 24 Jul 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this phase II study is to find out if an investigational drug called LGX818 can stop the melanoma from growing.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| LGX818 | Experimental | Drug: LGX818LGX818 capsules will be administered orally on a once -daily schedule (QD) dosing at a dose of 300 mg/day, 2 weeks on followed by a 2 week break. This schedule of 2 weeks on, followed by 2 weeks off, will continue for the duration of time the patients remains on the clinical trial. After the follow up visit patients will be contacted approximately every 12 weeks until they have received a subsequent therapy or until they have been off treatment for a year to monitor their survival. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Stage IV, or unresectable stage III melanoma that harbors a BRAFV600 mutation
- Any prior therapy allowed except a BRAF or MEK inhibitor,.
- Patients must provide written informed consent prior to any screening procedures.
- Age 18 years or older.
- Willing and able to comply with scheduled visits, treatment plan and laboratory tests
Key exclusion criteria (34 listed)
- Brain metastasis or leptomeningeal disease
- Known acute or chronic pancreatitis
- Prior colectomy
- Clinically significant cardiac disease including any of the following:
- CHF requiring treatment (NYHA Classification ≥ 2) in which patients have a history of LVEF < 45% as determined by MUGA scan or ECHO, or uncontrolled hypertension (please refer to WHO-ISH guidelines)
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 |
|---|---|---|
| LGX818 | Drug | LGX818 |
Primary outcome
Number of Participants With Response According to RECIST v1.1 Criteria
Time frame: 1.5 years
Efficacy for all patients will be evaluated by the study sites using RECIST v1.1 and response criteria based on contrast-enhanced CT. Complete Response (CR): Disappearance of all target lesions. Partial Response (PR): At least a 30% decrease in the sum of the longest diameter (LD) of target lesions, taking as reference the baseline sum LD. Progressive Disease (PD): At least a 20% increase in the sum of the LD of target lesions, taking as reference the smallest sum LD recorded since the treatment started or the appearance of one or more new lesions. Progression must also involve an increase in size of measurable lesions by at least 5 mm, to minimize the possibility that small changes in a small number of target lesions is falsely interpreted as progression.Stable Disease (SD): Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum LD since the treatment started.
1 secondary outcome
Response Rate
Time frame: 1.5 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jul 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Memorial Sloan Kettering Cancer CenterLink provided by the sponsor.
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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.
Paul Chapman, MD principal investigator
Memorial Sloan Kettering Cancer Center
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing LGX818 in people with Melanoma, run by Memorial Sloan Kettering Cancer Center at 5 sites in United States.
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 8 inclusion and 34 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: finished, as of the sponsor's last update on 16 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. 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?
Yes. Results are posted in the registry (first posted 24 Jul 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Response According to RECIST v1.1 Criteria (time frame: 1.5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 16 Jul 2024; AzTrials last refreshed its copy on 6 Nov 2026.