LGX818 for Patients With BRAFV600 Mutated Tumors

NCT01981187 | Terminated Phase 2 Results

• 2 other identifiers: CLGX818AUS03C4221021
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 13

Brief Summary

The purpose of this signal seeking study is to determine whether treatment with LGX818 demonstrates sufficient efficacy in select pathway-activated solid tumors and/or hematologic malignancies to warrant further study

Conditions

Solid Tumor; Hematologic Malignancies

Outcome Measures

Primary Outcomes (1)

• Clinical Benefit Rate (CBR) for Solid Tumors as Per Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1

  CBR for solid tumors was defined as percentage of participants with complete response (CR) or partial response (PR), or stable disease (SD) for greater than or equal to (\>=) 16 weeks. As per RECIST v1.1, CR was defined as disappearance of all target and non-target lesions and normalization of tumor markers. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to less than (\<) 10 millimeter (mm). PR was defined as at least a 30 percent (%) decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters. SD was defined as neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease, taking as reference the smallest sum diameters while on study. PD was defined as at least a 20% increase in the sum of diameter of all measured target lesions, taking as reference the smallest sum of diameter of all target lesions recorded at or after baseline.

  Up to 13.3 months

Secondary Outcomes (13)

• Overall Response Rate (ORR) for Solid Tumors as Per RECIST Version 1.1

  From the first dose study treatment until the first documented CR or PR (maximum up to 13.3 months)

• Progression-Free Survival (PFS) for Solid Tumors as Per RECIST Version 1.1

  From the date of first dose until the first documentation of PD, relapse, censored date or death, whichever occurred first (maximum up to 13.3 months)

• Overall Survival (OS) for Solid Tumors

  From date of the first dose until the date of death, censored date (maximum up to 13.3 months)

• Duration of Response (DOR) for Solid Tumors as Per RECIST Version 1.1

  From first documentation of response to first documentation of PD or relapse or death (maximum up to 13.3 months)

• Number of Participants With Treatment Emergent Adverse Events (TEAEs) Graded According to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), Version 4.03

  Screening up to 30 days after the last dose of study treatment (maximum up to 13.3 months)

Study Arms (1)

LGX818

EXPERIMENTAL

LGX818 will be dosed on a flat scale of 300 mg (e.g., 3 x 100 mg capsules) once daily on a continuous dosing cycle. A complete treatment cycle is defined as 28 days. There will be no breaks between dosing cycles.

Drug: LGX818

Interventions

LGX818 DRUG

LGX818 will be dosed on a flat scale of 300 mg (e.g., 3 x 100 mg capsules) once daily on a continuous dosing cycle. A complete treatment cycle is defined as 28 days. There will be no breaks between dosing cycles.

LGX818

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patient has a confirmed diagnosis of a select solid tumor (except with a primary diagnosis of melanoma and colorectal cancer (CRC)) or hematologic malignancies and is in need of treatment because of progression or relapse.
• Patient's tumor has been evaluated and pre-identified as having a tumor with a BRAFV600 mutation at a CLIA certified laboratory.
• Patient must have received at least one prior treatment for recurrent, metastatic and /or locally advanced disease and for whom no standard therapy options are anticipated to result in a durable remission.
• Patient must have progressive and measurable disease per RECIST 1.1. or other appropriate hematological response criteria.
• Patient has an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 1

You may not qualify if:

• Patient has received prior treatment with LGX818.
• Patients with Central Nerve System (CNS) metastasis or leptomeningeal carcinomatosis.
• Patient has received chemotherapy or other anticancer therapy ≤ 4 weeks (6 weeks for nitrosourea, antibodies or mitomycin-C) prior to starting study drug.
• Patients with acute or chronic pancreatitis.
• Patients with impaired cardiac function or clinically significant cardiac diseases.
• Patients with another primary malignancy within 3 years prior to starting study treatment, with the exception of adequately treated basal cell carcinoma, squamous cell carcinoma or other non-melanomatous skin cancer, or in-situ carcinoma of the uterine cervix.

Sponsors & Collaborators

• Pfizer: lead

Study Sites (13)

Alabama Oncology St. Vincent's Birmingham

Birmingham, Alabama, 35211, United States

Location

Highlands Oncology Group Highlands Oncology Group (22)

Fayetteville, Arkansas, 72703, United States

Location

Yale University School of Medicine Smilow Cancer Hospital

New Haven, Connecticut, 06520, United States

Location

Whittingham Cancer Center Norwalk Hospital

Norwalk, Connecticut, 06856, United States

Location

Florida Cancer Specialists Florida Cancer Specialists (31

Fort Myers, Florida, 33901, United States

Location

Lurie Children's Hospital of Chicago Developmental Therapeutics

Chicago, Illinois, 60611, United States

Location

Comprehensive Cancer Centers of Nevada CCC of Nevada (1)

Las Vegas, Nevada, 89109, United States

Location

Genesis Cancer Services

Zanesville, Ohio, 43701, United States

Location

University of Pennsylvania Presbyterian Medical Center University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

Sanford Research Sanford Health

Sioux Falls, South Dakota, 57104, United States

Location

Oncology Consultants Oncology Group

Houston, Texas, 77024, United States

Location

Utah Cancer Specialists Utah Cancer Specialists (11)

Salt Lake City, Utah, 84106, United States

Location

Shenandoah Oncology Shenadoah Oncology (2)

Winchester, Virginia, 22601, United States

Location

MeSH Terms

Conditions

Hematologic Neoplasms

Interventions

encorafenib

Condition Hierarchy (Ancestors)

Neoplasms by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Limitations and Caveats

Due to slow enrollment and lack of response observed during the enrollment period, the Sponsor decided to close study enrollment early on 28 January 2015.

Results Point of Contact

• Title: Pfizer ClinicalTrials.gov Call Center
• Organization: Pfizer Inc.

ClinicalTrials.gov_Inquiries@pfizer.com

1-800-718-1021

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 5, 2013
• First Posted: November 11, 2013
• Study Start: January 14, 2014
• Primary Completion: September 1, 2015
• Study Completion: October 13, 2015
• Last Updated: March 26, 2021
• Results First Posted: March 26, 2021

Record last verified: 2021-03

Data Sharing

• IPD Sharing: Will share

Locations

US (13)