Japan Ph2 Study of Pexidartinib in TGCT

JRCT ID: jRCT2041200074

Registered date:15/12/2020

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Tenosynovial giant cell tumor
• Date of first enrollment: 15/03/2021
• Target sample size: 21
• Study type: Interventional
• Intervention(s): pexidartinib 400 mg twice a day, oral administration

Outcome(s)

Primary Outcome

Part 1: Dose-limiting toxicity, pharmacokinetics parameters
Part 2: Objective response rate

Secondary Outcome

Part 1, Part 2: Treatment-emergent adverse events (TEAEs) and other safety parameters during the study
Part 2: Tumor volume score, Range of motion, Patient-Reported Outcomes Measurement Information System (PROMIS) physical function scale,Brief Pain Inventory (BPI)

Key inclusion & exclusion criteria

• Age minimum: >= 20age old
• Age maximum: Not applicable
• Gender: Both

Include 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 1.1 (except that a minimal size of 2 cm is required), assessed from MRI scan by a central radiologist.

Exclude criteria

- Known metastatic TGCT.
- Pre-existing increased serum transaminases; total bilirubin or direct bilirubin (>ULN); or active liver or biliary tract disease, including increased ALP.
- 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 subject`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.

Related Information

• Primary Sponsor: Inoguchi Akihiro