Study of Pimicotinib (ABSK021) for Tenosynovial Giant Cell Tumor (MANEUVER)

Tenosynovial Giant Cell Tumor Clinical Trial

Official title:

A Phase 3, Randomized, Double-blind, Placebo-Controlled, Multicenter Study of ABSK021 to Assess the Efficacy and Safety in Patients With Tenosynovial Giant Cell Tumor

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05804045
• Other study ID #: ABSK021-301
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: April 27, 2023
• Est. completion date: June 2028

Study information

• Verified date: April 2024
• Source: Abbisko Therapeutics Co, Ltd
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The goal of this clinical trial is to assess the efficacy and safety of Pimicotinib (ABSK021) in patients with Tenosynovial Giant Cell Tumor (TGCT). The main questions it aims to answer are: - Whether the Pimicotinib(ABSK021) works well in patients with TGCT. - Whether the Pimicotinib(ABSK021) is safe in patients with TGCT. Participants will be asked to complete the study procedures: - Receive the administration of Pimicotinib(ABSK021) or placebo (a placebo is a look-alike substance that contains no active drug) about 24 weeks in study part 1. - Receive the administration of Pimicotinib(ABSK021) about 24 weeks in study part 2. - Receive the administration of Pimicotinib(ABSK021) till study end in study part 3. - Complete the study procedures speficied in the protocol, which is guided by researchers.

Description:

This study consists of part 1 and part 2. Part 1 is a double-blind phase, eligible patients will be randomized to Pimicotinib(ABSK021) treatment group or matching placebo group and will receive Pimicotinib(ABSK021) or matching placebo until completion of Part 1. Part 2 is an open-label treatment phase, and all patients entering this phase will receive open-label Pimicotinib(ABSK021) until completion of 24 weeks of dosing or withdrawal from the study. Part 3 is an open-label extension treatment phase, and patients who completed the part 2 and continuted to be eligible, will go to the Part 3. Patients will receive the open-label Pimicotinib(ABSK021) until all patients withdraw from the study, or the sponsor decides to terminate the study, whichever occurs first.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 90
• Est. completion date: June 2028
• Est. primary completion date: May 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Patients should understand the study procedures and sign the informed consent form prior to screening.
• Age = 18 years.
• A histologically confirmed TGCT with unresectable.
• Measurable disease as defined by RECIST 1.1, and with at least one lesion of = 2 cm.
• Stable prescription of analgesic regimen for patients with an analgesic need.
• Participants should complete stiffness and pain scales during the screening period, and symptomatic disease because of active TGCT should meet minimum requirements as outlined in study protocol.
• ECOG PS (Eastern Cooperative Oncology Group Performance Status) of 0 or 1.
• Adequate organ function and bone marrow function.

Exclusion Criteria:

• Known allergy or hypersensitivity to any components of the investigational drug product.
• Previous treatment with highly selective inhibitors targeting CSF-1/CSF-1R. Previous therapy with imatinib and nilotinib is allowed.
• Known additional malignancy that required active treatment and may affect the patient's participation in the study or affect the outcome of the study as assessed by the Investigator.
• Known metastatic TGCT.
• Significant concomitant arthropathy in the affected joint, serious disease, uncontrolled infection.
• Known MRI contraindications.
• Has factors that significantly affected the absorption of oral drug.
• Major surgery or previous anti-tumor therapy for TGCT within 4 weeks prior to randomization.
• Concomitant use of strong CYP inhibitors or inducers as outlined in study protocol.
• Impaired cardiac function or clinically significant cardiac disease.
• Known active human immunodeficiency virus, active hepatitis B, active hepatitis C, or known active tuberculosis.
• Known active liver or biliary disease, or other diseases that may lead to abnormal liver function test results during the study.
• Pregnant or lactating women.
• Childbearing potential males or non-surgically sterilized female patients must agree to use effective methods of contraception during the study.
• Any other clinically significant comorbidities, which in the judgment of the Investigator, could compromise compliance with the protocol, interfere with the interpretation of study results, or predispose the patient to safety risks.

Related Conditions & MeSH terms

• Giant Cell Tumor of Tendon Sheath
• Giant Cell Tumors
• Neoplasms
• Pigmented Villonodular Synovitis
• Synovitis
• Synovitis, Pigmented Villonodular
• Tenosynovial Giant Cell Tumor

Intervention

Drug:
• Pimicotinib(ABSK021)
capsule
• Placebo
capsule

Locations

Country	Name	City	State
Canada	McGill University Health Center	Montréal
Canada	Princess Margaret Cancer Center	Toronto
China	Beijing Jishuitan Hospital	Beijing
China	Peking University People's Hospital	Beijing	Beijing
China	The First Affiliated Hospital of Bengbu Medical College	Bengbu	Anhui
China	Hunan Provincial People's Hospital	Changsha	Hunan
China	The Second Xiangya Hospital of Central South University	Changsha	Hunan
China	West China Hospital Sichuan University	Chengdu	Wuhan
China	The Central Hospital of Enshi Tujia and Miao Autonomous Prefecture	Enshi	Hubei
China	The First Affiliated Hospital of Fujian Medical University	Fuzhou	Fujian
China	Guangdong Provincial People's Hospital	Guangzhou	Guangdong
China	The First Affiliated Hospital of Jinan University	Guangzhou	Guangdong
China	The First Affiliated Hospital, Sun Yat-sen University	Guangzhou	Guangdong
China	The Affiliated Hospital of Guizhou Medical University	Guiyang	Guizhou
China	The Second Affiliated Hospital Zhejiang University School of Medicine	Hangzhou	Zhejiang
China	Zhejiang Cancer Hospital	Hangzhou	Zhejiang
China	Nanjing Drum Tower hospital	Nanjing	Jiangsu
China	Shanghai General Hospital	Shanghai	Shanghai
China	Liaoning Cancer Hospital&Institute	Shenyang	Liaoning
China	The Second Hospital of Shanxi Medical University	Taiyuan	Shanxi
China	The First Affiliated Hospital of Xinjiang Medical University	Ürümqi	Xinjiang
China	Weifang People's Hospital	Weifang	Shandong
China	Union Hospital Affiliated to Tongji Medical College, Huazhong University of Science and Technology	Wuhan	Hubei
China	Xi'an Honghui Hospital	Xi'an	Shanxi
China	Henan Cancer Hospital	Zhengzhou	Henan
Italy	IRCCS Istituto Ortopedico Rizzoli	Bologna
Italy	Fondazione IRCCS Istituto Nazionale dei Tumori	Milano
Italy	Ospedale di Prato	Prato
Netherlands	Leiden University Medical Center	Leiden
Poland	Maria Sklodowska-Curie National Research Institute of Oncology	Warsaw
Spain	Hospital Universitari Vall d'Hebron	Barcelona
Spain	Fundacion Jimenez Diaz	Madrid
United States	Precision NextGen Oncology	Beverly Hills	California
United States	The Cleveland Clinic Foundation	Cleveland	Ohio
United States	The Ohio State University	Columbus	Ohio
United States	Henry Ford Health System	Detroit	Michigan
United States	The University of Texas M.D. Anderson Cancer Center	Houston	Texas
United States	Fred Hutchinson Cancer Center	Seattle	Washington

Sponsors (1)

Lead Sponsor	Collaborator
Abbisko Therapeutics Co, Ltd

Countries where clinical trial is conducted

United States,  Canada,  China,  Italy,  Netherlands,  Poland,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Objective Response Rate (ORR)	Assessed by central read using Response Evaluation Criteria in Solid Tumors (RECIST) (Version 1.1)	Baseline to Week 25
Secondary	Objective Response Rate (ORR) per Tumor Volume Score (TVS)	Assessed by central read using Tumor Volume Score (TVS). TVS is a semi-quantitative MRI scoring system that describes tumor mass and is based on 10% increments of the estimated volume of the maximally distended synovial cavity or tendon sheath involved. A tumor that is equal in volume to that of a maximally distended synovial cavity or tendon sheath was scored 10; a score of 0 indicated no evidence of tumor.	Baseline to Week 25
Secondary	Range of Motion (ROM)	Mean change from baseline in ROM of the affected joint	Baseline to Week 25
Secondary	Worst Stiffness	Mean change from baseline in the Worst Stiffness Numeric Rating Scale (NRS) score at Week 25. The Worst Stiffness Numeric Rating Scale (NRS) was a 1-item, self-administered questionnaire assessing the "worst" stiffness in the last 24 hours. The NRS for this item ranged from 0 (no stiffness) to 10 (stiffness as bad as you can imagine). Higher scores mean a worse outcomes.	Baseline to Week 25
Secondary	Worst Pain	Mean change from baseline in the Worst Pain Numeric Rating Scale (NRS) score at Week 25. The Worst Pain Numeric Rating Scale Score (NRS) was a 1-item, self-administered questionnaire assessing the "worst" pain in the last 24 hours. The NRS for this item ranged from 0 (no pain) to 10 (pain as bad as you can imagine). Higher scores mean a worse outcomes.	Baseline to Week 25
Secondary	Physical Function	Mean change from baseline in the Patient-reported Outcomes Measurement Information System (PROMIS) Physical Function score at Week 25. The Patient-reported Outcomes Measurement Information System (PROMIS) physical function scale was used to assess physical function of the upper and lower limbs. The scale ranged from 1 defined as 'unable to do' or 'cannot do' to 5 defined as 'without any difficulty' or 'not at all', where higher scores represent better outcomes.	Baseline to Week 25
Secondary	Quality of life (QoL)	Mean change from baseline in EuroQol visual analogue scale (VAS) score at Week 25. The EuroQol visual analogue scale (VAS) is a Visual Analogue Scale on which the patient rates their current health, with 0 representing the "worst health you can imagine" and 100 representing the "best health you can imagine". Higher scores mean a better outcomes.	Baseline to Week 25
Secondary	Duration of Response (DOR)	Duration of Response as measured by RECIST Version 1.1 and Tumor Volume Score (TVS)	The time (months) from the first documentation of objective response to the first documentation of radiographic disease progression (PD) or death due to any cause, whichever occurs first, assessed up to 24 months.