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NeoplasmsPhase 2NCT01286753

A Study of Vemurafenib (RO5185426) in Participants With Metastatic or Unresectable Papillary Thyroid Cancer Positive for the BRAF V600 Mutation

A Phase 2 study testing Vemurafenib in people with Neoplasms, run by Hoffmann-La Roche at 16 sites in 4 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This open-label, multi-center study will evaluate the safety and efficacy of Vemurafenib (RO5185426) in participants with metastatic or unresectable papillary thyroid cancer (PTC) positive for the BRAF V600 mutation and resistant to radioactive iodine therapy. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Neoplasms
What is being tested?
Vemurafenib
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
51 participants
Where
16 sitesin 4 countries
Study dates
Jun 2011to May 2015
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

  • Adult participants. >/= 18 years of age
  • Histologically confirmed metastatic or unresectable papillary thyroid cancer for which standard curative or palliative measures do not exist or are no longer effective; participants whose tumors exhibit areas of "other histology" may be enrolled, provided the tumor histology remains predominantly papillary
  • Positive for BRAF V600 mutation (Roche Cobas 4800 BRAF V600 Mutation Test)
  • Radioactive Iodine resistant disease
  • Prior therapy excluding (Cohort 1, TKI Naive) or including (Cohort 2, TKI Experienced) TKI
  • Clinically relevant disease progression according to RECIST criteria within the prior 14 months
  • Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1
  • Adequate hematological, renal and liver function

Who cannot join

  • Histological diagnosis other than papillary PTC, including squamous cell variants of PTC or PTC with areas of squamous metaplasia
  • Active or untreated central nervous system metastases
  • History of or known carcinomatous meningitis
  • Anticipated or ongoing administration of any anti-cancer therapies other than those administered in the study
  • Active squamous cell skin cancer that has not been excised or adequately healed post excision
  • Previous treatment with any agent that specifically and selectively targets the MEK or BRAF pathway
  • Prior radiotherapy to the only measurable lesion
  • Clinically relevant cardio-vascular disease or event within the prior 6 months

Trial locations and contacts

16 sites in 4 countries.

16 sites in 4 countries

United States9 sites

  • Study site

    Baltimore, Maryland, 21231

  • Study site

    Boston, Massachusetts, 02114

  • Study site

    Boston, Massachusetts, 02215

  • Study site

    Chicago, Illinois, 60637

  • Study site

    Houston, Texas, 77030

  • Study site

    New Haven, Connecticut, 06510

  • Study site

    New York, New York, 10017

  • Study site

    Philadelphia, Pennsylvania, 19104

  • Study site

    Torrance, California, 90502

Other trials in United States

France3 sites

  • Study site

    Lyon, 69008

  • Study site

    Paris, 75651

  • Study site

    Villejuif, 94805

Other trials in France

Italy2 sites

  • Study site

    Milan, Lombardy, 20133

  • Study site

    Pisa, Tuscany, 56124

Other trials in Italy

Netherlands2 sites

  • Study site

    Groningen, 9700 RB

  • Study site

    Leiden, 2300 RC

Other trials in Netherlands

About this study

Official title: An Open-Label, Multi-Center Phase II Study of the BRAF Inhibitor Vemurafenib in Patients With Metastatic or Unresectable Papillary Thyroid Cancer (PTC) Positive for the BRAF V600 Mutation and Resistant to Radioactive Iodine

This open-label, multi-center study will evaluate the safety and efficacy of Vemurafenib (RO5185426) in participants with metastatic or unresectable papillary thyroid cancer (PTC) positive for the BRAF V600 mutation and resistant to radioactive iodine therapy. Participants will receive vemurafenib 960 milligrams (mg) orally twice daily until progressive disease or unacceptable toxicity occurs.

Conditions
Neoplasms
Being tested
Vemurafenib
Medical subject headings
Neoplasms

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • VemurafenibDrug

    Also called Zelboraf®, RO5185426

    Vemurafenib 960 mg orally twice daily.

Study groups in NCT01286753
Study groupRoleWhat they receive
Tyrosine Kinase Inhibitor (TKI) NaiveExperimentalVemurafenib in participants naive to any prior systemic TKI therapy.
Receives: Drug: Vemurafenib
TKI ExperiencedExperimentalVemurafenib in participants previously treated with TKI therapy active against vascular endothelial growth factor receptor 2 (VEGFR).
Receives: Drug: Vemurafenib

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Best Overall Response Rate in TKI-Naive Participants

    Best overall response rate was assessed by the investigators according to the Response Evaluation Criteria in Solid Tumors (RECIST) v1.1. Best overall response rate: the percentage of participants with best objective response of complete response (CR) or partial response (PR) (calculated as the number of participants with best response CR or PR divided by the total number of efficacy-evaluable participants). CR: disappearance of all target lesions with reduction in target/non-target pathological lymph nodes to < 10 millimeters (mm). PR: ≥ 30% decrease in the sum of diameters of target lesions, compared to the baseline sum diameters.

    Measured: Up to approximately 4 years

Secondary outcomes (7)

  1. Best Overall Response Rate in TKI-Experienced Participants

    Best overall response rate was assessed by the investigators according to RECIST v1.1. Best overall response rate: the percentage of participants with best objective response of CR or PR (calculated as the number of participants with best response CR or PR divided by the total number of efficacy-evaluable participants). CR: disappearance of all target lesions with reduction in target/non-target pathological lymph nodes to < 10 mm. PR: ≥ 30% decrease in the sum of diameters of target lesions, compared to the baseline sum diameters.

    Measured: Up to approximately 4 years

  2. Clinical Benefit Rate

    Clinical benefit rate: the percentage of participants with confirmed CR, PR, or stable disease (SD; maintained for at least 6 months) as assessed by investigators according to RECIST v1.1. CR: disappearance of all target lesions with reduction in target/non-target pathological lymph nodes to < 10 mm. PR: ≥ 30% decrease in the sum of diameters of target lesions, compared to the baseline sum diameters. SD: neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, compared to the baseline sum diameters.

    Measured: Up to approximately 4 years

  3. Duration of Response

    Duration of response (for participants with confirmed best response CR or PR): the interval between earliest qualifying response and date of progression of disease (PD) or death for any cause, whichever occurred first; participants with no documented progression after CR or PR were censored at the date of last known CR or PR, respectively. CR: disappearance of all target lesions with reduction in target/non-target pathological lymph nodes to < 10 mm. PR: ≥ 30% decrease in the sum of diameters of target lesions, compared to the baseline sum diameters. PD: ≥ 20% increase in the sum of diameters of target lesions, compared to the smallest sum on study.

    Measured: From the date of first qualifying response to the date of PD or death for any cause (up to approximately 4 years)

  4. Progression-Free Survival

    Progression-free survival: the interval between the day of first treatment and the first documentation of PD or death; participants who were withdrawn from the study without documented progression were censored at the date of the last tumor assessment when the participant was known to be progression-free; participants without post baseline tumor assessments were censored at the time of enrollment. PD: ≥ 20% increase in the sum of diameters of target lesions, compared to the smallest sum on study.

    Measured: From the day of first treatment until the first documented PD or death (up to approximately 4 years)

  5. Overall Survival

    Overall survival: the interval between the date of first treatment to the date of death, regardless of the cause of death; participants who were alive at the time of the analysis were censored at the date of the last known alive; participants with no post baseline information were censored at the time of enrollment.

    Measured: From the date of first treatment to the date of death for any cause (up to approximately 4 years)

  6. Percentage of Participants With Adverse Events

    An adverse event was defined as any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product.

    Measured: Baseline until 28 days after the last dose of study treatment or until initiation of another anti-cancer therapy, whichever occurred first (up to approximately 4 years)

  7. Pharmacokinetics of Vemurafenib: Area Under the Concentration-Time Curve (AUC)

    AUC is a measure of the drug or biologic concentration in the body following administration.

    Measured: Up to approximately 4 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted31 Jan 2011 (estimated)
  2. Study startJun 2011
  3. Primary completionNov 2014
  4. Study completionMay 2015

Resources

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
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
51 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01286753
Sponsor's protocol ID
NO25530
Other IDs
2010-024133-23
Status last verified
Jul 2016
First submitted
28 Jan 2011
Last updated
7 Sep 2016
Data monitoring committee
Yes

Publications

  • Brose MS, Cabanillas ME, Cohen EE, Wirth LJ, Riehl T, Yue H, Sherman SI, Sherman EJ. Vemurafenib in patients with BRAF(V600E)-positive metastatic or unresectable papillary thyroid cancer refractory to radioactive iodine: a non-randomised, multicentre, open-label, phase 2 trial. Lancet Oncol. 2016 Sep;17(9):1272-82. doi: 10.1016/S1470-2045(16)30166-8. Epub 2016 Jul 23.

    PubMed 27460442

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Vemurafenib in people with Neoplasms, run by Hoffmann-La Roche at 16 sites in 4 countries.

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 8 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 7 Sep 2016. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 16 sites in United States, France, Italy, Netherlands. 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 7 Sep 2016). 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?

Best Overall Response Rate in TKI-Naive Participants (time frame: Up to approximately 4 years). 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 NCT01286753, 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 7 Sep 2016.

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