MOTION: Study of Vimseltinib for Tenosynovial Giant Cell Tumor

• Sponsor: Deciphera Pharmaceuticals LLC (Industry)
• Overall Status: Recruiting
• CT.gov ID: NCT05059262
• Collaborator: (none)
• Enrollment: 120
• Locations: 25
• Arms: 2
• Anticipated Duration (Months): 56.5
• Patients Per Site: 4.8
• Patients Per Site Per Month: 0.1

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
Tenosynovial Giant Cell Tumor; Pigmented Villonodular Synovitis; Giant Cell Tumor of Tendon Sheath; Tenosynovial Giant Cell Tumor, Diffuse; Tenosynovial Giant Cell Tumor, Localized	Drug: vimseltinib; Drug: Placebo	Phase 3

Study Design

• Study Type: Interventional
• Anticipated Enrollment: 120 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• 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)
• Actual Study Start Date: Oct 14, 2021
• Anticipated Primary Completion Date: Mar 1, 2024
• Anticipated Study Completion Date: Jul 1, 2026

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Part 1/Part 2 - vimseltinib/vimseltinib 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	Drug: vimseltinib; CSF1R inhibitor; Other Names: DCC-3014
Placebo Comparator: Part 1/Part 2 - placebo/vimseltinib 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	Drug: vimseltinib; CSF1R inhibitor; Other Names: DCC-3014 ; Drug: Placebo; Placebo

Outcome Measures

Primary Outcome Measures

1. Objective Response Rate (ORR= complete response [CR]+partial response [PR]) per RECIST Version 1,1 [Baseline to Week 25 (Cycle 7, Day 1)]

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

Secondary Outcome Measures

1. ORR per Tumor Volume Score (TVS) [Baseline to Week 25 (Cycle 7, Day 1)]

   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.

2. Range of motion (ROM) [Baseline to Week 25 (Cycle 7, Day 1)]

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

3. Physical function [Baseline to Week 25 (Cycle 7, Day 1)]

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

4. Worst stiffness [Baseline to Week 25 (Cycle 7, Day 1)]

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

5. Quality of life (QoL) [Baseline to Week 25 (Cycle 7, Day 1)]

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

6. Worst pain [Baseline to Week 25 (Cycle 7, Day 1)]

   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

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

1. Patients ≥18 years of age

2. TGCT for which surgical resection is not an option (tumor biopsy to confirm diagnosis required if no histology/pathology available at screening)

3. 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

4. Participants should complete 14 consecutive days of questionnaires during the screening period and must meet minimum requirements as outlined in study protocol

5. Must have stable analgesic regimen, as judged by the investigator, for at least 2 weeks prior to first dose of study drug

6. Must have measurable disease, as per RECIST Version 1.1, with at least one lesion having a minimum size of 2cm

7. Adequate organ and bone marrow function

8. If a female of childbearing potential, must have a negative pregnancy test prior to enrollment and agree to follow the contraception requirements

9. Must provide signed consent to participate in the study and is willing to comply with study-specific procedures

10. Willing and able to complete the patient-reported outcome (PRO) assessments on an electronic device

Exclusion Criteria:

1. 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

2. Received therapy for TGCT, including investigational therapy during the screening period. Participated in a non-TGCT investigational drug study within 30 days of screening.

3. Known metastatic TGCT or other active cancer that requires concurrent treatment (exceptions will be considered on a case-by-case basis)

4. QT interval corrected by Fridericia's formula (QTcF) >450 ms in males or >470 ms in females or history of long QT syndrome

5. Concurrent treatment with any study-prohibited medications

6. Major surgery within 14 days of the first dose of study drug

7. Any clinically significant comorbidities

8. Active liver or biliary disease including nonalcoholic steatohepatitis (NASH) or cirrhosis

9. Malabsorption syndrome or other illness that could affect oral absorption

10. Known active human immunodeficiency virus (HIV), acute or chronic hepatitis B, acute or chronic hepatitis C, or known active mycobacterium tuberculosis infection

11. If female, the participant is pregnant or breastfeeding

12. Known allergy or hypersensitivity to any component of the study drug

13. Contraindication to MRI

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	City of Hope	Duarte	California	United States	91010
2	UC Davis Comprehensive Cancer Center	Sacramento	California	United States	95817
3	University of Colorado	Aurora	Colorado	United States	80045
4	Dana Farber Cancer Institute	Boston	Massachusetts	United States	02215
5	Memorial Sloan-Kettering Cancer Center	New York	New York	United States	10016
6	Duke Sarcoma Research	Durham	North Carolina	United States	27710
7	University of Texas MD Anderson Cancer Center	Houston	Texas	United States	77030
8	Seattle Cancer Care Alliance	Seattle	Washington	United States	98109
9	Chris O'Brien Lifehouse	Camperdown		Australia
10	McGill University	Montréal		Canada
11	Princess Margaret Hospital	Toronto		Canada
12	Institut Bergonié	Bordeaux		France
13	Centre Léon Bérard	Lyon		France
14	Institut Gustave Roussy	Villejuif		France
15	University Hospital Essen (Universitätsklinikum Essen)	Essen		Germany
16	Fondazione IRCCS Istituto Nazionale dei Tumori	Milan		Italy
17	Leiden University Medical Center	Leiden		Netherlands
18	Klinika Nowotworów Tkanek Miękkich, Kości i Czerniaków Narodowy Instytut Onkologii im. Marii Skłodowskiej-Curie - Państwowy Instytut Badawczy	Warsaw		Poland
19	Hospital de la Santa Creu i Sant Pau	Barcelona		Spain
20	Hospital Universitario Vall d'Hebron	Barcelona		Spain
21	Fundacion Jimenez Diaz	Madrid		Spain
22	Hospital Clinico San Carlos	Madrid		Spain
23	Universitäts-Kinderspital beider Basel (UKBB)	Basel		Switzerland
24	Cancer & Haematology Centre, The Churchill Hospital - Oxford University Hospitals NHS Foundation Trust	London		United Kingdom
25	University College London Hospitals	London		United Kingdom

Sponsors and Collaborators

• Deciphera Pharmaceuticals LLC

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Deciphera Pharmaceuticals LLC
• ClinicalTrials.gov Identifier: NCT05059262
• Other Study ID Numbers: DCC-3014-03-001
• First Posted: Sep 28, 2021
• Last Update Posted: Jul 5, 2022
• Last Verified: Jun 1, 2022

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Keywords provided by Deciphera Pharmaceuticals LLC: TGCT; PVNS
• Additional relevant MeSH terms: Neoplasms; Giant Cell Tumors; Giant Cell Tumor of Tendon Sheath; Synovitis, Pigmented Villonodular; Synovitis

Study Results

No Results Posted as of Jul 5, 2022