A Phase 3, Randomized, Placebo-controlled, Double-blind Study of Vimseltinib to Assess the Efficacy and Safety in Patients With Tenosynovial Giant Cell Tumor (MOTION)
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
- 入组人数
- 123
- 试验地点
- 35
- 主要终点
- Objective Response Rate (ORR) at Week 25 Per Response Evaluation Criteria In Solid Tumors (RECIST) Version 1.1
研究概览
简要总结
This is a multicenter Phase 3 clinical study, which aims to evaluate the effectiveness of an investigational drug called vimseltinib for the treatment of tenosynovial giant cell tumor (TGCT) in cases where surgical removal of the tumor is not an option.
The study consists of two parts. In Part 1, eligible study participants will be assigned to receive either vimseltinib or matching placebo for 24 weeks. A number of assessments will be carried out during the course of the study, including physical examinations, blood tests, imaging studies, electrocardiograms, and questionnaires. MRI scans will be used to evaluate the response of the tumors to the treatment. Participants assigned to placebo in Part 1 will have the option to receive vimseltinib for Part 2. Part 2 is a long-term treatment phase in which all participants receive open-label vimseltinib.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients ≥18 years of age
- •TGCT for which surgical resection is not an option (tumor biopsy to confirm diagnosis required if no histology/pathology available at screening)
- •Symptomatic disease as defined as at least moderate pain or at least moderate stiffness (defined as a score of 4 or more, with 10 describing the worst condition) within the screening period and documented in the medical record
- •Participants should complete 14 consecutive days of questionnaires during the screening period and must meet minimum requirements as outlined in study protocol
- •Must have stable analgesic regimen, as judged by the investigator, for at least 2 weeks prior to first dose of study drug
- •Must have measurable disease, as per RECIST Version 1.1, with at least one lesion having a minimum size of 2cm
- •Adequate organ and bone marrow function
- •If a female of childbearing potential, must have a negative pregnancy test prior to enrollment and agree to follow the contraception requirements
- •Must provide signed consent to participate in the study and is willing to comply with study-specific procedures
- •Willing and able to complete the patient-reported outcome (PRO) assessments on an electronic device
排除标准
- •Previous use of systemic therapy (investigational or approved) targeting colony-stimulating factor 1 (CSF1) or colony-stimulating factor 1 receptor (CSF1R); previous therapy with imatinib and nilotinib is allowed
- •Received therapy for TGCT, including investigational therapy during the screening period. Participated in a non-TGCT investigational drug study within 30 days of screening.
- •Known metastatic TGCT or other active cancer that requires concurrent treatment (exceptions will be considered on a case-by-case basis)
- •QT interval corrected by Fridericia's formula (QTcF) >450 ms in males or >470 ms in females or history of long QT syndrome
- •Concurrent treatment with any study-prohibited medications
- •Major surgery within 14 days of the first dose of study drug
- •Any clinically significant comorbidities
- •Active liver or biliary disease including nonalcoholic steatohepatitis (NASH) or cirrhosis
- •Malabsorption syndrome or other illness that could affect oral absorption
- •Known active human immunodeficiency virus (HIV), acute or chronic hepatitis B, acute or chronic hepatitis C, or known active mycobacterium tuberculosis infection
- •If female, the participant is pregnant or breastfeeding
- •Known allergy or hypersensitivity to any component of the study drug
- •Contraindication to MRI
研究组 & 干预措施
Part 1/Part 2 - Vimseltinib/Vimseltinib
Participants received blinded treatment of 30 mg twice a week (BIW) vimseltinib for 24 weeks in Part 1 and open-label 30 mg BIW vimseltinib in Part 2.
干预措施: Vimseltinib (Drug)
Part 1/Part 2: Placebo/Vimseltinib
Participants received blinded treatment of BIW matching placebo for 24 weeks in Part 1 and open-label 30 mg BIW vimseltinib in Part 2.
干预措施: Vimseltinib (Drug)
Part 1/Part 2: Placebo/Vimseltinib
Participants received blinded treatment of BIW matching placebo for 24 weeks in Part 1 and open-label 30 mg BIW vimseltinib in Part 2.
干预措施: Placebo (Drug)
结局指标
主要结局
Objective Response Rate (ORR) at Week 25 Per Response Evaluation Criteria In Solid Tumors (RECIST) Version 1.1
时间窗: Baseline to Week 25 (Cycle 7, Day 1)
ORR was assessed by blinded independent radiologic review (IRR) using RECIST Version 1.1. ORR was defined as the percentage of participants who achieved either complete response (CR) or partial response (PR). * CR: Disappearance of all target lesions. Any pathological lymph nodes must be \<10 millimeter (mm) in short axis. Non-nodal targets must be absent. * PR: At least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum diameters.
次要结局
- ORR at Week 25 Per Tumor Volume Score (TVS)(Baseline to Week 25 (Cycle 7, Day 1))
- Change From Baseline in Active Range of Motion (ROM) at Week 25(Baseline to Week 25 (Cycle 7, Day 1))
- Change From Baseline in the Patient-reported Outcomes Measurement Information System (PROMIS) Physical Function (PF) Score at Week 25(Baseline to Week 25 (Cycle 7, Day 1))
- Change From Baseline in the Worst Stiffness Numeric Rating Scale (NRS) Score at Week 25(Baseline to Week 25 (Cycle 7, Day 1))
- Change From Baseline in EuroQoL Visual Analogue Scale (EQ-VAS) at Week 25(Baseline to Week 25 (Cycle 7, Day 1))
- Percentage of Participants With Response at Week 25 Based on Brief Pain Inventory (BPI) Worst Pain NRS Score and Narcotic Analgesic Use(Baseline to Week 25 (Cycle 7, Day 1))