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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05059262




Registration number
NCT05059262
Ethics application status
Date submitted
17/09/2021
Date registered
28/09/2021
Date last updated
9/08/2024

Titles & IDs
Public title
Study of Vimseltinib for Tenosynovial Giant Cell Tumor
Scientific 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)
Secondary ID [1] 0 0
DCC-3014-03-001
Universal Trial Number (UTN)
Trial acronym
MOTION
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Tenosynovial Giant Cell Tumor 0 0
Pigmented Villonodular Synovitis 0 0
Giant Cell Tumor of Tendon Sheath 0 0
Tenosynovial Giant Cell Tumor, Diffuse 0 0
Tenosynovial Giant Cell Tumor, Localized 0 0
Condition category
Condition code
Cancer 0 0 0 0
Bone
Cancer 0 0 0 0
Other cancer types
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - vimseltinib
Treatment: Drugs - Placebo

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

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


Treatment: Drugs: vimseltinib
CSF1R inhibitor

Treatment: Drugs: Placebo
Placebo
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Objective Response Rate (ORR= complete response [CR]+partial response [PR]) per RECIST Version 1,1
Timepoint [1] 0 0
Baseline to Week 25 (Cycle 7, Day 1)
Secondary outcome [1] 0 0
ORR per Tumor Volume Score (TVS)
Timepoint [1] 0 0
Baseline to Week 25 (Cycle 7, Day 1)
Secondary outcome [2] 0 0
Range of motion (ROM)
Timepoint [2] 0 0
Baseline to Week 25 (Cycle 7, Day 1)
Secondary outcome [3] 0 0
Physical function
Timepoint [3] 0 0
Baseline to Week 25 (Cycle 7, Day 1)
Secondary outcome [4] 0 0
Worst stiffness
Timepoint [4] 0 0
Baseline to Week 25 (Cycle 7, Day 1)
Secondary outcome [5] 0 0
Quality of life (QoL)
Timepoint [5] 0 0
Baseline to Week 25 (Cycle 7, Day 1)
Secondary outcome [6] 0 0
Worst pain
Timepoint [6] 0 0
Baseline to Week 25 (Cycle 7, Day 1)

Eligibility
Key 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
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
14/10/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/07/2026
Actual
Sample size
Target
120
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Chris O'Brien Lifehouse - Camperdown
Recruitment postcode(s) [1] 0 0
- Camperdown
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Kansas
Country [4] 0 0
United States of America
State/province [4] 0 0
Massachusetts
Country [5] 0 0
United States of America
State/province [5] 0 0
Minnesota
Country [6] 0 0
United States of America
State/province [6] 0 0
New York
Country [7] 0 0
United States of America
State/province [7] 0 0
North Carolina
Country [8] 0 0
United States of America
State/province [8] 0 0
Ohio
Country [9] 0 0
United States of America
State/province [9] 0 0
Texas
Country [10] 0 0
United States of America
State/province [10] 0 0
Washington
Country [11] 0 0
Canada
State/province [11] 0 0
Montréal
Country [12] 0 0
Canada
State/province [12] 0 0
Toronto
Country [13] 0 0
France
State/province [13] 0 0
Bordeaux
Country [14] 0 0
France
State/province [14] 0 0
Lyon
Country [15] 0 0
France
State/province [15] 0 0
Villejuif
Country [16] 0 0
Germany
State/province [16] 0 0
Berlin
Country [17] 0 0
Germany
State/province [17] 0 0
Essen
Country [18] 0 0
Hong Kong
State/province [18] 0 0
Hong Kong
Country [19] 0 0
Italy
State/province [19] 0 0
Bologna
Country [20] 0 0
Italy
State/province [20] 0 0
Milan
Country [21] 0 0
Italy
State/province [21] 0 0
Naples
Country [22] 0 0
Italy
State/province [22] 0 0
Padua
Country [23] 0 0
Italy
State/province [23] 0 0
Rome
Country [24] 0 0
Netherlands
State/province [24] 0 0
Leiden
Country [25] 0 0
Norway
State/province [25] 0 0
Oslo
Country [26] 0 0
Poland
State/province [26] 0 0
Warsaw
Country [27] 0 0
Spain
State/province [27] 0 0
Barcelona
Country [28] 0 0
Spain
State/province [28] 0 0
Madrid
Country [29] 0 0
Switzerland
State/province [29] 0 0
Basel
Country [30] 0 0
United Kingdom
State/province [30] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Deciphera Pharmaceuticals, LLC
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/study/NCT05059262
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT05059262