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

NCT02975700 | Completed Results

• 1 other identifier: PLX108-13
• Study Type: interventional
• Target: 6
• Locations: 2 countries
• Sites: 5

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

Keywords

PLX3397; Kit-mutant Melanoma; Unresectable or Metastatic KIT-mutated Melanoma; Developmental Phase I/II

Outcome Measures

Primary Outcomes (2)

• Summary of Best Overall Response Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma

  For the assessment of best overall response, participants were classified by RECIST v1.1: complete response (CR), disappearance of all target lesions and normalization of tumor marker level; partial response (PR), at least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters; stable disease (SD); 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; progressive disease (PD), at least a 20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study and the sum must also demonstrate an absolute increase of at least 5 mm; or not evaluable (NE) for analysis.

  within 18 months postdose

• Objective Response Rate (ORR) Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma

  Objective response rate was defined as complete response (CR) or partial response (PR).

  within 18 months postdose

Secondary Outcomes (13)

• Duration of Response Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma

  within 18 months postdose

• Progression-free Survival Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma

  within 18 months postdose

• Overall Survival Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma

  within 18 months postdose

• Disease Control Rate Following Twice Daily Administration of PL3397 at 1000 mg/Day In Participants With Unresectable or Advanced KIT-mutated Melanoma

  within 18 months postdose

• Summary of Pharmacokinetic Parameter of Maximum Concentration (Cmax) of PL3397 at Days 1 and 15 Following Twice Daily Administration of PL3397 at 1000 mg/Day in Participants With Unresectable or Advanced KIT-mutated Melanoma

  Cycle 1, Day 1 and Day 15 at predose, 0.5 h, 1h, 2h, 4h, and 6h postdose

Study Arms (1)

PLX3397

EXPERIMENTAL

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.

Drug: PLX3397

Interventions

PLX3397 DRUG

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

Also known as: Pexidartinib

PLX3397

Eligibility Criteria

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

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Daiichi Sankyo Co., Ltd.: lead
• Daiichi Sankyo: collaborator

Study Sites (5)

Beijing Cancer Hospital

Beijing, Beijing Municipality, 100142, China

Location

Sun Yat-sen Hospital

Guangzhou, Guangdong, China

Location

Samsung Medical Center

Seoul, Gangnam-Gu, 06351, South Korea

Location

Severance Hospital, Yonsei University Health System

Seoul, Seodaemun-gu, 03722, South Korea

Location

Seoul National University Hospital

Seoul, South Korea

Location

MeSH Terms

Conditions

Melanoma

Interventions

pexidartinib

Condition Hierarchy (Ancestors)

Neuroendocrine Tumors; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Nerve Tissue; Nevi and Melanomas; Skin Neoplasms; Neoplasms by Site; Skin Diseases; Skin and Connective Tissue Diseases

Results Point of Contact

• Title: Contact for Clinical Trial Information
• Organization: Daiichi Sankyo, Inc.

CTRinfo@dsi.com

908-992-6400

Study Officials

• Global Clinical Leader

  Daiichi Sankyo

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 21, 2016
• First Posted: November 29, 2016
• Study Start: January 31, 2017
• Primary Completion: August 31, 2018
• Study Completion: October 11, 2024
• Last Updated: January 13, 2026
• Results First Posted: April 9, 2020

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

CN (2); KR (3)