A Study of Pexidartinib in Tenosynovial Giant Cell Tumor in Japan

NCT ID: NCT04703322

Last Updated: 2025-03-03

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 9 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-03-15
• Study Completion Date: 2026-05-31

Brief Summary

This phase 2, multicenter, two-part, open-label, single-arm study will be conducted in Japan and will evaluate the safety, tolerability, pharmacokinetics (PK), and efficacy of pexidartinib in adult participants with symptomatic tenosynovial giant cell tumor (TGCT) associated with severe morbidity or functional limitation and not amenable to improvement with surgery.

Detailed Description

This study will consist of 2 parts. In Part 1, pexidartinib 800 mg/day (400 mg twice a day [BID]) will be administered on an empty stomach and tolerability and PK of pexidartinib will be evaluated to determine the initiation of Part 2. In Part 2, pexidartinib 800 mg/day (400 mg BID) will be administered on an empty stomach and efficacy, safety, and PK of pexidartinib will be evaluated.

Conditions

Tenosynovial Giant Cell Tumor

Study Design

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

Study Groups

Pexidartinib

Participants with TGCT who will receive oral pexidartinib 800 mg (400 mg twice daily \[BID\]).

Group Type: EXPERIMENTAL

Pexidartinib

Intervention Type: DRUG

400 mg twice daily for a total daily dose of 800 mg (each capsule contains 200 mg of pexidartinib for oral administration)

Interventions

Pexidartinib

400 mg twice daily for a total daily dose of 800 mg (each capsule contains 200 mg of pexidartinib for oral administration)

Intervention Type: DRUG

Other Intervention Names

TURALIO™; PLX3397

Eligibility Criteria

Inclusion Criteria

• Age ≥20 years
• A diagnosis of TGCT (i) that has been histologically confirmed by a pathologist1 and (ii) associated with severe morbidity or functional limitations and not amenable to improvement with surgery determined consensually by qualified personnel (eg, 2 surgeons or a multi-disciplinary tumor board).
• Measurable disease as defined by RECIST version 1.1 (except that a minimal size of 2 cm is required), assessed from MRI scan by a central radiologist.

Exclusion Criteria

• Known metastatic TGCT.
• Pre-existing increased serum transaminases; total bilirubin or direct bilirubin (>upper limit of normal); or active liver or biliary tract disease, including increased alkaline phosphatase.
• Significant concomitant arthropathy in the affected joint, serious illness, uncontrolled infection, or a medical or psychiatric history that, in the Investigator's opinion, would likely interfere with a participant's study participation or the interpretation of his or her results.
• Use of strong cytochrome P450 3A inducers, including St John's wort, proton pump inhibitors and potassium-competitive acid blockers, or other products known to cause hepatotoxicity.

• Minimum Eligible Age: 20 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Daiichi Sankyo Co., Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Global Clinical Leader

Role: STUDY_DIRECTOR

Daiichi Sankyo

Locations

Nagoya University Hospital

Aichi, , Japan

Kyushu University Hospital

Fukuoka, , Japan

Kanazawa University Hospital

Ishikawa, , Japan

National Hospital Organization Osaka National Hospital

Osaka, , Japan

Osaka International Cancer Institute

Osaka, , Japan

National Cancer Center Hospital

Tokyo, , Japan

Countries

Japan

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

jRCT2041200074

Identifier Type: OTHER

Identifier Source: secondary_id

PL3397-A-J304

Identifier Type: -

Identifier Source: org_study_id