A Study of PLX3397 in Patients With Unresectable or Metastatic KIT-mutated Melanoma

NCT ID: NCT02975700

Last Updated: 2026-01-13

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 6 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2017-01-31
• Study Completion Date: 2024-10-11

Brief Summary

The purpose of this Phase I/II study is to evaluate safety, pharmacokinetics, and preliminary efficacy of the investigational drug PLX3397 in subjects with unresectable or metastatic KIT-mutated melanoma.

Conditions

Melanoma

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

PLX3397

Part 1: Open label, multicenter study includes a dose evaluation portion in which the safety profile of PLX3397 as a single oral agent will be evaluated

Part 2: An expansion cohort in which the efficacy and safety of PLX3397 administered at the recommended Phase 2 dose will be evaluated in patients with unresectable stage III or stage IV KIT-mutated melanoma.

Group Type: EXPERIMENTAL

PLX3397

Intervention Type: DRUG

1000 mg/day (400 mg in the morning and 600 mg in the evening)

Interventions

PLX3397

1000 mg/day (400 mg in the morning and 600 mg in the evening)

Intervention Type: DRUG

Other Intervention Names

Pexidartinib

Eligibility Criteria

Inclusion Criteria

• Age ≥ 18 years
• Unresectable stage III or stage IV melanoma which is histologically confirmed at the treating institution with KIT mutation(s) not known to be resistant to PLX3397
• Presence of measurable lesions by Response Evaluation Criteria in Solid Tumors
• Eastern Cooperative Oncology Group (ECOG) performance Status (PS) 0-2
• Life expectancy ≥ 3 months
• Adequate organ and bone marrow function
• Women of child-bearing potential must have a negative serum pregnancy test at Screening and must agree to use an effective form of contraception from the time of the negative pregnancy test up to 3 months after the last dose of study drug. Women of non-child-bearing potential must have been postmenopausal for ≥ 1 year or surgically sterile.
• Fertile men must agree to use an effective method of birth control during the study and for up to 3 months after the last dose of study drug.
• Willingness and ability to provide written informed consent prior to any study-related procedures and to comply with all study requirements

Exclusion Criteria

• Prior treatment with a KIT inhibitor for melanoma
• Presence of NRAS or BRAF mutation
• Exposure to any investigational drug within 28 days or unresolved adverse effects from previous therapy
• Symptomatic brain metastases.
• Active secondary malignancy unless the malignancy is not expected to interfere with the evaluation of safety and is approved by the Sponsor
• Concomitant treatment with other anti-neoplastic agents (hormonal therapy acceptable)
• Uncontrolled intercurrent or infectious illness
• Major surgical procedure or significant traumatic injury within 14 days of initiating study drug or anticipation of the need for major surgery during the study
• Previous radiotherapy to 25% or more of the bone marrow and/or radiation therapy within 28 days prior to study entry
• Inability to swallow capsules, or refractory nausea and vomiting, malabsorption, an external biliary shunt, or significant bowel resection that would preclude adequate absorption
• Congestive heart failure (CHF) New York (NY) Heart Association class III or IV; unstable coronary artery disease [myocardial infarction (MI) more than 6 months prior to study entry is permitted] or serious cardiac arrhythmia
• Baseline QT interval corrected using Fridericia equation (QTcF) ≥ 450 msec (for males) or ≥ 470 msec (for females) at Screening
• Active or chronic infection with human immunodeficiency virus (HIV), hepatitis C virus (HCV), or hepatitis B virus (HBV)
• Known chronic liver disease
• Women who are breast-feeding or pregnant

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Daiichi Sankyo

INDUSTRY

Sponsor Role: collaborator

Daiichi Sankyo Co., Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Global Clinical Leader

Role: STUDY_DIRECTOR

Daiichi Sankyo

Locations

Beijing Cancer Hospital

Beijing, Beijing Municipality, China

Sun Yat-sen Hospital

Guangzhou, Guangdong, China

Samsung Medical Center

Seoul, Gangnam-Gu, South Korea

Severance Hospital, Yonsei University Health System

Seoul, Seodaemun-gu, South Korea

Seoul National University Hospital

Seoul, , South Korea

Countries

China; South Korea

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

PLX108-13

Identifier Type: -

Identifier Source: org_study_id