A Study of Vemurafenib in Pediatric Patients With Stage IIIC or Stage IV Melanoma Harboring BRAFV600 Mutations - Full Text View

Purpose

This open-label, multicenter. single arm Phase I dose-escalation study with efficacy tail extension will evaluate the maximum tolerated dose/recommended dose, the safety and efficacy of vemurafenib (RO5185426) in pediatric patients (aged 12 through 17) with newly diagnosed or recurrent surgically incurable and unresectable Stage IIIC or Stage IV melanoma harboring BRAFV600 mutations. Patients will receive vemurafenib orally twice daily until disease progression or unacceptable toxicity occurs.

Condition	Intervention	Phase
Malignant Melanoma	Drug: vemurafenib	Phase 1

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	An Open-label, Multicenter, Single-arm, Phase I Dose-escalation With Efficacy Tail Extension Study of RO5185426 in Pediatric Patients With Surgically Incurable and Unresectable Stage IIIC or Stage IV Melanoma Harboring BRAFV600 Mutations Mutations

Resource links provided by NLM:

MedlinePlus related topics: Melanoma

Drug Information available for: Vemurafenib

U.S. FDA Resources

Further study details as provided by Hoffmann-La Roche:

Primary Outcome Measures:

Maximum tolerated dose (MTD)/recommended dose [ Time Frame: approximately 4 years ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Pharmacokinetics: Area under the concentration-time curve [ Time Frame: Pre- and post-dose Days 1, 15 and 22 of Cycle 1, Day 1 in following Cycles ] [ Designated as safety issue: No ]
Safety: Incidence of adverse events [ Time Frame: approximately 4 years ] [ Designated as safety issue: No ]
Best overall response rate (BORR; tumor assessments according to RECIST criteria) [ Time Frame: approximately 4 years ] [ Designated as safety issue: No ]
Clinical benefit rate (CBR) [ Time Frame: approximately 4 years ] [ Designated as safety issue: No ]
Progression-free survival (PFS) [ Time Frame: approximately 4 years ] [ Designated as safety issue: No ]
Overall survival (OS) [ Time Frame: approximately 4 years ] [ Designated as safety issue: No ]

Estimated Enrollment:	27
Study Start Date:	January 2013
Estimated Study Completion Date:	September 2016
Estimated Primary Completion Date:	September 2016 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: Single Arm	Drug: vemurafenib Multiple doses

Eligibility

Ages Eligible for Study:	12 Years to 17 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Pediatric patients, 12 to 17 years of age inclusive
Histologically confirmed surgically incurable and unresectable Stage IIIC or Stage IV (AJCC) melanoma
Positive BRAF mutation result (Cobas 4800 BRAF V600 Mutation Test)
Measurable disease according to RECIST criteria
Performance status: Karnofsky (for patients >/= 16 years of age) or Lansky (for patients < 16 years of age) score of >/= 60
Adequate bone marrow, liver and renal function
Patients must have fully recovered from the acute toxic effects of all prior therapy prior to first administration of study drug

Exclusion Criteria:

Active or untreated central nervous system (CNS) lesions
History of or known spinal cord compression or carcinomatous meningitis
Anticipated or ongoing administration of anti-cancer therapies other than those administered in this study
Previous malignancy within the past 5 years except for basal or squamous cell carcinoma of the skin, melanoma in-situ, and carcinoma in-situ of the cervix
Previous treatment with selective/specific BRAF or MEK inhibitor (previous treatment with sorafenib is allowed)
Any previous treatment with study drug (RO5185426) or participation in a clinical trial that includes RO5185426
Pregnant or lactating females
Known HIV positivity or AIDS-related illness, active hepatitis B virus, or active hepatitis C virus

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT01519323

Contacts

Contact: Please reference Study ID Number: NO25390 www.roche.com/about_roche/roche_worldwide.htm

888-662-6728 (U.S. Only)

genentechclinicaltrials@druginfo.com

Locations

United States, California
	Recruiting
Los Angeles, California, United States, 90027
United States, Colorado
	Not yet recruiting
Aurora, Colorado, United States, 80045
United States, Florida
	Recruiting
St. Petersburgh, Florida, United States, 33701
United States, Maryland
	Recruiting
Bethesda, Maryland, United States, 20892
United States, Massachusetts
	Recruiting
Boston, Massachusetts, United States, 02115
United States, New York
	Recruiting
New York, New York, United States, 10021
United States, Tennessee
	Recruiting
Memphis, Tennessee, United States, 38105
United States, Texas
	Recruiting
Houston, Texas, United States, 77030
Australia
	Recruiting
Herston, Australia, 4029
	Recruiting
Westmead, Australia, 2145
Germany
	Recruiting
Kiel, Germany, 24105
	Recruiting
Mainz, Germany, 55131
	Recruiting
Tuebingen, Germany, 72076
Italy
	Recruiting
Genova, Italy, 16147
	Recruiting
Milano, Italy, 20133
	Recruiting
Roma, Italy, 00165
United Kingdom
	Recruiting
Newcastle Upon Tyne, United Kingdom, NE1 4LP
	Recruiting
Sutton, United Kingdom, SM2 5PT

Sponsors and Collaborators

Hoffmann-La Roche

Investigators

Study Director:

Clinical Trials

Hoffmann-La Roche

More Information

No publications provided

Responsible Party:	Hoffmann-La Roche
ClinicalTrials.gov Identifier:	NCT01519323 History of Changes
Other Study ID Numbers:	NO25390, 2011-000874-67
Study First Received:	January 16, 2012
Last Updated:	May 7, 2013
Health Authority:	United States: Food and Drug Administration

Additional relevant MeSH terms:

Melanoma
Neuroendocrine Tumors
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal

Neoplasms by Histologic Type
Neoplasms
Neoplasms, Nerve Tissue
Nevi and Melanomas

ClinicalTrials.gov processed this record on June 18, 2013