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Study of Vimseltinib for Tenosynovial Giant Cell Tumor (MOTION)

Primary Purpose

Tenosynovial Giant Cell Tumor, Pigmented Villonodular Synovitis, Giant Cell Tumor of Tendon Sheath

Status

Active

Phase

Phase 3

Locations

International

Study Type

Interventional

Intervention

vimseltinib

Placebo

About this trial

This is an interventional treatment trial for Tenosynovial Giant Cell Tumor focused on measuring TGCT, PVNS

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

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

Exclusion Criteria:

Previous use of systemic therapy (investigational or approved) targeting colony stimulating factor 1 (CSF1) or CSFR1 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

Sites / Locations

City of Hope
UC Davis Comprehensive Cancer Center
University of Colorado
University of Kansas
Dana Farber Cancer Institute
Mayo Clinic Rochester
Memorial Sloan-Kettering Cancer Center
Duke Sarcoma Research
Ohio State University
University of Texas MD Anderson Cancer Center
Seattle Cancer Care Alliance
Chris O'Brien Lifehouse
McGill University
Princess Margaret Hospital
Institut Bergonié
Centre Léon Bérard
Institut Gustave Roussy
Helios Klinikum Berlin-Buch
University Hospital Essen (Universitätsklinikum Essen)
Prince of Wales Hospital
Istituto Ortopedico Rizzoli
Fondazione IRCCS Istituto Nazionale dei Tumori
Istituto Nazionale Tumori IRCCS "Fondazione G. Pascale"
Istituto Oncologico Veneto
Istituto Nazionale Tumori Regina Elena
Leiden University Medical Center
Oslo University Hospital
Klinika Nowotworów Tkanek Miękkich, Kości i Czerniaków Narodowy Instytut Onkologii im. Marii Skłodowskiej-Curie - Państwowy Instytut Badawczy
Hospital de la Santa Creu i Sant Pau
Hospital Universitario Vall d'Hebron
Fundacion Jimenez Diaz
Hospital Clinico San Carlos
Universitäts-Kinderspital beider Basel (UKBB)
Cancer & Haematology Centre, The Churchill Hospital - Oxford University Hospitals NHS Foundation Trust
University College London Hospitals

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Part 1/Part 2 - vimseltinib/vimseltinib

Part 1/Part 2 - placebo/vimseltinib

Arm Description

Participants receive blinded treatment of 30 mg twice a week (biw) vimseltinib for 24 weeks in Part 1 and continue on 30 mg biw vimseltinib in Part 2

Participants receive blinded treatment of 30 mg twice a week (biw) matching placebo for 24 weeks in Part 1 and have option to receive 30 mg biw vimseltinib in Part 2

Outcomes

Primary Outcome Measures

Objective Response Rate (ORR= complete response [CR]+partial response [PR]) per RECIST Version 1,1

Assessed by central read using Response Evaluation Criteria In Solid Tumors (RECIST) Version 1.1

Secondary Outcome Measures

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.

Range of motion (ROM)

Mean change from baseline in active ROM of the affected joint, relative to a reference standard at Week 25

Physical function

Mean change from baseline in the Patient-reported Outcomes Measurement Information System (PROMIS) physical function score at Week 25

Worst stiffness

Mean change from baseline in the Worst Stiffness Numeric Rating Scale (NRS) score at Week 25

Quality of life (QoL)

Mean change from baseline in EuroQoL Visual Analogue Scale (EQ-VAS) at Week 25

Worst pain

Proportion of responders, with a response defined as at least a 30% improvement in the mean Brief Pain Inventory (BPI) Worst Pain NRS score without a 30% or greater increase in narcotic analgesic use at Week 25

Full Information

NCT ID

NCT05059262

First Posted

September 17, 2021

Last Updated

March 15, 2023

Sponsor

Deciphera Pharmaceuticals LLC

1. Study Identification

Unique Protocol Identification Number

NCT05059262

Brief Title

Study of Vimseltinib for Tenosynovial Giant Cell Tumor

Acronym

MOTION

Official Title

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)

Study Type

Interventional

2. Study Status

Record Verification Date

March 2023

Overall Recruitment Status

Active, not recruiting

Study Start Date

October 14, 2021 (Actual)

Primary Completion Date

March 2024 (Anticipated)

Study Completion Date

July 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Deciphera Pharmaceuticals LLC

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Tenosynovial Giant Cell Tumor, Pigmented Villonodular Synovitis, Giant Cell Tumor of Tendon Sheath, Tenosynovial Giant Cell Tumor, Diffuse, Tenosynovial Giant Cell Tumor, Localized

Keywords

TGCT, PVNS

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

120 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Part 1/Part 2 - vimseltinib/vimseltinib

Arm Type

Experimental

Arm Description

Participants receive blinded treatment of 30 mg twice a week (biw) vimseltinib for 24 weeks in Part 1 and continue on 30 mg biw vimseltinib in Part 2

Arm Title

Part 1/Part 2 - placebo/vimseltinib

Arm Type

Placebo Comparator

Arm Description

Participants receive blinded treatment of 30 mg twice a week (biw) matching placebo for 24 weeks in Part 1 and have option to receive 30 mg biw vimseltinib in Part 2

Intervention Type

Drug

Intervention Name(s)

vimseltinib

Other Intervention Name(s)

DCC-3014

Intervention Description

CSF1R inhibitor

Intervention Type

Drug

Intervention Name(s)

Placebo

Intervention Description

Placebo

Primary Outcome Measure Information:

Title

Objective Response Rate (ORR= complete response [CR]+partial response [PR]) per RECIST Version 1,1

Description

Assessed by central read using Response Evaluation Criteria In Solid Tumors (RECIST) Version 1.1

Time Frame