Study of Pimicotinib in Japanese Participants With Tenosynovial Giant Cell Tumor (TGCT) (J-MANEUVER)

Phase 2RecruitingNCT07499362

Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany20 enrolled

Overview

The primary purpose of this study is to assess the tolerability, pharmacokinetics, and efficacy of pimicotinib in Japanese participants with TGCT

The purpose of this study is to assess the tolerability, pharmacokinetics (PK), efficacy, and safety of pimicotinib in Japanese participants with TGCT in two cohorts: Safety Run-in and Expansion.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Tenosynovial Giant Cell Tumor

Interventions

PimicotinibDRUG

In the Safety Run-in Cohort, participants will receive 50 milligrams (mg) of pimicotinib once daily (QD), orally in 28-day cycles until disease progression (DP), death, discontinuation, or any other reason. The Safety Monitoring Committee (SMC) will monitor and assess tolerability of pimicotinib 50 mg QD during the safety run-in cohort. After making a recommendation about opening the Expansion Cohort based on the assessment in safety run-in cohort, participants will receive 50 mg of pimicotinib monotherapy QD, orally in 28-day cycles until disease progression (DP), death, discontinuation, or any other reason.

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Japanese participants with a diagnosis of TGCT that has been histologically confirmed at the local laboratory and is unresectable (that is \[i.e.\] it is located in a complex anatomical site, extensively invasive, and cannot be completely resected; or a surgical operation may cause dysfunction or serious complications). Symptomatic disease because of active TGCT, defined as a worst pain of greater than or equal to \[\>=\] 4 within 2 weeks prior to enrollment (based on scale of 0 to 10, with 10 representing "pain as bad as you can imagine"), and/or a worst stiffness of \>= 4 within 2 weeks prior to first dose of pimicotinib (based on a scale of 0 to 10, with 10 representing "stiffness as bad as you can imagine") * Eastern Cooperative Oncology Group (ECOG) Performance Status less than or equal to \[\<=\] 1 * Participants with adequate hepatic, renal hematologic functions * Other protocol defined inclusion criteria may apply Exclusion Criteria: * Known additional malignancy that required active treatment and may affect the participant's participation in the study or affect the outcome of the study as assessed by the Investigator. Exceptions include cured basal cell carcinoma of skin, squamous cell carcinoma of skin, and other carcinoma in situ * Serious gastrointestinal bleeding within 3 months of first dose of pimicotinib or factors that significantly affected the absorption of oral drug, such as inability to take oral medication or significant nausea and vomiting, malabsorption, external bile duct drainage, massive small-bowel resection, etc. * Impaired cardiac function or clinically significant cardiac disease, including any one of the following: New York Heart Association (NYHA) class III or IV heart disease, active ischemia or any other uncontrolled cardiac condition such as angina pectoris, clinically significant cardiac arrhythmia requiring therapy, uncontrolled hypertension or congestive heart failure; prolongation of the rate corrected QT interval based on repeated demonstration of QT interval corrected using Fridericia's formula (QTcF) greater than (\>) 480 milliseconds (ms), or history of long QT interval corrected (QTc) syndrome; Left ventricular ejection fraction (LVEF) less than (\<) 50 percent (%) or below the lower limit of normal, whichever is higher * Cerebrovascular accident/stroke (within 6 months of first dose of pimicotinib) * Other protocol defined exclusion criteria may apply

Locations (1)

Kyushu University Hospital - 300173484

Fukuoka, Japan

RECRUITING

Outcomes

Primary Outcomes

Safety Run-In Cohort: Number of Participants with Dose Limiting Toxicity (DLT)-like events

Time frame: Day 1 up to Day 28 of Cycle 1 (each cycle is of 28 days)

Safety Run-In Cohort: Pharmacokinetic (PK) Plasma Concentration of Pimicotinib

Time frame: Pre-dose up to 24 hours post-dose on Cycle 1 Day 1 and Cycle 1 Day 15 (each cycle is of 28 days)

Objective Response (OR) According to Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST v1.1) as Assessed by Independent Review Committee

Time frame: Time from first study treatment until progressive disease or death, assessed up to approximately 2 years

Secondary Outcomes

Objective Response (OR) According to Tumor Volume Score (TVS) as Assessed by Independent Review Committee (IRC)

Time frame: Time from first study treatment until progressive disease or death, assessed up to approximately 2 years

Objective Response (OR) According to Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST v1.1) as assessed by Investigator

Time frame: Time from first study treatment until progressive disease or death, assessed up to approximately 2 years

Mean Change from Baseline in Range of Motion (ROM) at Week 25

The ROM of the affected joint or tumor site will be assessed using a goniometer. Measurements will be recorded in degrees.

Time frame: Baseline, Week 25

Mean Change from Baseline in Worst-Stiffness-Numeric Rating Scale (NRS) Score at Week 25

Time frame: Baseline, Week 25

Mean Change from Baseline in Brief Pain Inventory (BPI)- Worst-Stiffness-Numeric Rating Scale (NRS) Score at Week 25

Central Contacts

Communication Center

CONTACT

+49 6151 72 5200 service@emdgroup.com

Data from ClinicalTrials.gov

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