Vimseltinib for Tenosynovial Giant Cell Tumor

(MOTION Trial)

Not currently recruiting at 38 trial locations

Overseen By Clinical Team

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Deciphera Pharmaceuticals, LLC

Must not be taking: CSF1 inhibitors

Disqualifiers: Metastatic TGCT, Active cancer, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new drug, vimseltinib, for individuals with tenosynovial giant cell tumor (TGCT), a type of tumor that surgery cannot remove. In the first part of the study, some participants will receive vimseltinib, while others will receive a placebo. In the second part, all participants will receive vimseltinib to evaluate its long-term effectiveness. Suitable candidates for this trial have TGCT causing moderate pain or stiffness and cannot undergo surgical treatment. As a Phase 3 trial, this study represents the final step before FDA approval, offering access to a potentially groundbreaking treatment.

Will I have to stop taking my current medications?

The trial protocol does not specify if you need to stop taking your current medications, but it mentions that you must have a stable pain medication routine for at least 2 weeks before starting the study. Also, you cannot take any medications that the study prohibits.

Is there any evidence suggesting that vimseltinib is likely to be safe for humans?

Research shows that vimseltinib has been tested in people with tenosynovial giant cell tumor (TGCT) when surgery isn't possible. Studies found that patients usually tolerate vimseltinib well. Some experienced side effects, but these were generally manageable. Common side effects included nausea and fatigue. Importantly, vimseltinib effectively reduced tumor size compared to a placebo. While side effects exist, the potential benefits could be significant for those with TGCT. Always consult a healthcare provider to determine if joining a trial is appropriate. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for TGCT?

Vimseltinib is unique because it specifically targets the CSF1R pathway, which is crucial in the development of tenosynovial giant cell tumor (TGCT). Unlike traditional treatments like surgery or systemic therapies, which can be invasive or have broad effects, vimseltinib offers a more targeted approach. This precision can potentially lead to fewer side effects and improved outcomes for patients. Researchers are excited about vimseltinib because it represents a novel, non-surgical option that directly addresses the underlying mechanism of TGCT, offering hope for more effective and less disruptive treatment.

What evidence suggests that vimseltinib might be an effective treatment for tenosynovial giant cell tumor?

Research has shown that vimseltinib holds promise for treating tenosynovial giant cell tumor (TGCT). In this trial, participants will receive either vimseltinib or a placebo in Part 1. One study found that patients taking vimseltinib had better outcomes than those taking a placebo. After a year, these patients experienced a noticeable decrease in tumor size. Another study found that vimseltinib was effective and safe over two years. This drug works by blocking a protein that helps the tumor grow. Overall, the evidence suggests vimseltinib could be a helpful option for people with TGCT when surgery isn't possible. ^{1 2 4 5 6}

Are You a Good Fit for This Trial?

Adults with tenosynovial giant cell tumor (TGCT) that can't be surgically removed. They must have a confirmed diagnosis, measurable disease, stable pain management, and no allergies to the study drug. Women of childbearing age need a negative pregnancy test and must follow contraception guidelines.

Inclusion Criteria

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

I have moderate pain or stiffness noted in my medical records.

My TGCT cannot be removed surgically, and I may need a biopsy to confirm this if no previous tests are available.

Exclusion Criteria

Concurrent treatment with any study-prohibited medications

Known allergy or hypersensitivity to any component of the study drug

I am currently pregnant or breastfeeding.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Part 1

Participants receive either vimseltinib or placebo for 24 weeks with assessments including physical exams, blood tests, imaging studies, and questionnaires

24 weeks

Regular visits for assessments

Treatment Part 2

Participants receive open-label vimseltinib in a long-term treatment phase

Long-term

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Placebo
• Vimseltinib

Trial Overview The trial is testing vimseltinib against a placebo for TGCT treatment over 24 weeks. Participants will undergo physical exams, blood tests, imaging studies including MRI scans to monitor tumor response. Afterward, all participants can receive vimseltinib in an open-label extension phase.

How Is the Trial Designed?

Group I: Part 1/Part 2 - Vimseltinib/Vimseltinib Experimental Treatment 1 Intervention

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.

Group II: Part 1/Part 2: Placebo/Vimseltinib Placebo Group 2 Interventions

Participants received blinded treatment of BIW matching placebo for 24 weeks in Part 1 and open-label 30 mg BIW vimseltinib in Part 2.

Who Is Running the Clinical Trial?

Deciphera Pharmaceuticals, LLC

Lead Sponsor

Trials

19

Recruited

2,100+

Deciphera Pharmaceuticals LLC

Lead Sponsor

Trials

17

Recruited

1,900+

Published Research Related to This Trial

Lenvatinib has been approved as a second targeted therapy for patients with radioactive iodine refractory, locally advanced, or metastatic differentiated thyroid carcinoma, showing a median progression-free survival (PFS) of 18.3 months compared to just 3.6 months for placebo, indicating a significant improvement in treatment efficacy.

The overall response rate (ORR) for lenvatinib was 64.7%, vastly higher than the 1.5% seen in the placebo group, highlighting its effectiveness in managing this challenging cancer type.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Lenvatinib in Advanced, Radioactive Iodine-Refractory, Differentiated Thyroid Carcinoma. Yeung, KT., Cohen, EE.[2015]

In a study of 63 patients with diffuse-type tenosynovial giant-cell tumour (dTGCT), treatment with emactuzumab showed a high overall response rate of 71%, indicating significant clinical activity over a follow-up period of up to 2 years.

The treatment was associated with a manageable safety profile, with common side effects including pruritus, asthenia, and edema, while also leading to improvements in quality of life as measured by standard questionnaires.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-term clinical activity, safety and patient-reported quality of life for emactuzumab-treated patients with diffuse-type tenosynovial giant-cell tumour. Cassier, PA., Italiano, A., Gomez-Roca, C., et al.[2021]

Citations

1. ascopubs.org ascopubs.org/doi/10.1200/JCO.2024.42.16_suppl.11500

Results from the phase 3 MOTION trial. Long-term clinical outcome assessments in patients with tenosynovial giant cell tumor treated with vimseltinib: 1-year results from the MOTION ...

2. thelancet.com thelancet.com/journals/lancet/article/PIIS0140-6736(24)00885-7/abstract

Vimseltinib versus placebo for tenosynovial giant cell ... This study evaluated the efficacy and safety of vimseltinib, an oral, switch-control, CSF1R inhibitor, in patients with symptomatic TGCT not ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT03069469

NCT03069469 | Study of Vimseltinib (DCC-3014) in ... This is a multicenter, open-label Phase 1/2 study of vimseltinib in patients with malignant solid tumors and tenosynovial giant cell tumor (TGCT).

4. annalsofoncology.org annalsofoncology.org/article/S0923-7534(24)03901-2/fulltext

LBA78 Updated efficacy and safety of vimseltinib in ... Updated efficacy and safety of vimseltinib in patients (pts) with tenosynovial giant cell tumor (TGCT): One-year follow-up from the MOTION phase III trial

5. deciphera.com deciphera.com/news/deciphera-presents-2-year-efficacy-and-safety-results-motion-phase-3-study-romvimzatm

Deciphera Presents 2-Year Efficacy and Safety Results ... Vimseltinib demonstrated statistically significant and clinically meaningful benefit vs placebo in antitumor response –.

6. annalsofoncology.org annalsofoncology.org/article/S0923-7534(24)03380-5/fulltext

1770P Safety, efficacy, and patient-reported outcomes ... Pts with symptomatic TGCT not amenable to surgery who had prior anti-CSF1/CSF1R therapy received vimseltinib 30 mg twice weekly (recommended phase II dose).