Combined BRAF-Targeted Therapy & Immunotherapy for Melanoma

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01754376
Recruitment Status : Terminated (changes in available treatments for melanoma)
First Posted : December 21, 2012
Results First Posted : March 9, 2017
Last Update Posted : April 12, 2017
Information provided by (Responsible Party):
Ryan Sullivan, M.D., Massachusetts General Hospital

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition: Melanoma
Interventions: Drug: Aldesleukin
Drug: Vemurafenib

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Patients were recruited at Massachusetts General Hospital (Boston, MA) between February and November 2013

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
Treatment Arm

Oral vemurafenib twice a day IV infusion of aldesleukin

Aldesleukin: Intravenous therapy given every 8 hours for up to 14 doses per week.

Vemurafenib: Tablets given twice daily.

Participant Flow:   Overall Study
    Treatment Arm
Completed 2-week Lead in of Vemurafenib   6 
Adverse Event                1 
Lack of Efficacy                2 

  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
Treatment Arm

Oral vemurafenib twice a day IV infusion of aldesleukin

Aldesleukin: Intravenous therapy given every 8 hours for up to 14 doses per week.

Vemurafenib: Tablets given twice daily.

Baseline Measures
   Treatment Arm 
Overall Participants Analyzed 
[Units: Participants]
[Units: Years]
Median (Full Range)
 (23 to 58) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
Female      3  50.0% 
Male      3  50.0% 
Race (NIH/OMB) 
[Units: Participants]
Count of Participants
American Indian or Alaska Native      0   0.0% 
Asian      0   0.0% 
Native Hawaiian or Other Pacific Islander      0   0.0% 
Black or African American      0   0.0% 
White      6 100.0% 
More than one race      0   0.0% 
Unknown or Not Reported      0   0.0% 
Region of Enrollment 
[Units: Participants]
United States   6 
Brain Metastasis present 
[Units: Participants]
Count of Participants
ECOG Status [1] 
[Units: Participants]
Count of Participants
ECOG Status = 0   3 
ECOG Status = 1   3 
[1] The ECOG Scale of Performance Status describes a patient’s level of functioning in terms of their ability to care for themself, daily activity, and physical ability. A score of 0 indicates fully active, and a score of 5 indicates dead. (A score of one = Restricted in physically strenuous activity but ambulatory and able to carry out work of a light or sedentary nature, e.g., light house work, office work)
Previous treatments 
[Units: Participants]
Count of Participants
Surgery   6 
Adjuvant interferon-alfa   5 

  Outcome Measures

1.  Primary:   Progression Free Survival   [ Time Frame: 3 years ]

2.  Secondary:   Overall Response Rate   [ Time Frame: 2 years ]

3.  Secondary:   Overall Survival   [ Time Frame: 2 years ]

4.  Secondary:   Number of Participants Experiencing Grade 3 (Severe) Adverse Events   [ Time Frame: 2 years ]

5.  Secondary:   Mean Percentage of Aldesleukin Doses Received Per Participant   [ Time Frame: Approximately 9 months from start of treatment ]

6.  Secondary:   Ratio of CD8+ T Cells to Regulatory T Cells   [ Time Frame: Up to 8 weeks from start of treatment ]

  Serious Adverse Events

  Other Adverse Events

  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
The protocol terminated early due to a dramatic change in the treatment landscape for treating BRAF mutant melanoma (e.g. one BRAF/MEK inhibitor combination and two anti-PD1 antibodies were approved by the FDA while this study was open).

  More Information

Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.

Results Point of Contact:  
Name/Title: Ryan Sullivan
Organization: MGH Cancer Center
phone: 617-724-4000

Publications automatically indexed to this study by Identifier (NCT Number):

Responsible Party: Ryan Sullivan, M.D., Massachusetts General Hospital Identifier: NCT01754376     History of Changes
Other Study ID Numbers: 12-343
First Submitted: December 16, 2012
First Posted: December 21, 2012
Results First Submitted: January 18, 2017
Results First Posted: March 9, 2017
Last Update Posted: April 12, 2017