Vimseltinib for Tenosynovial Giant Cell Tumor

(MOTION Trial)

Recruiting at 37 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

123 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing a new medicine called vimseltinib for patients with a type of tumor called TGCT who can't have surgery. The medicine aims to block proteins that help the tumor grow, potentially shrinking it or stopping its growth. Vimseltinib has shown promising results in early studies for TGCT.

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.

What data supports the effectiveness of the drug vimseltinib for treating tenosynovial giant cell tumor?

Vimseltinib is designed to target the CSF1 receptor, which is involved in the growth of tenosynovial giant cell tumors. Similar drugs, like emactuzumab and nilotinib, which also target the CSF1 receptor, have shown effectiveness in treating this condition, suggesting that vimseltinib may also be effective. ^{1 2 3 4 5}

Is vimseltinib safe for humans?

The safety of vimseltinib, a treatment for tenosynovial giant cell tumor, is being evaluated in a Phase III trial. While specific safety data for vimseltinib is not detailed, similar treatments targeting the same pathway have shown potential side effects that may lead to discontinuation due to an unfavorable risk-benefit ratio. ^{1 2 3 4 6}

What makes the drug Vimseltinib unique for treating Tenosynovial Giant Cell Tumor?

Vimseltinib is unique because it specifically targets the CSF1R (colony-stimulating factor 1 receptor), which is involved in the growth of Tenosynovial Giant Cell Tumors, offering a targeted approach compared to other treatments that may not specifically address this pathway. ^{7 8 9 10 11}

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

Findings from Research

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]

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]

References

1. England pubmed.ncbi.nlm.nih.gov

The MOTION study: a randomized, Phase III study of vimseltinib for the treatment of tenosynovial giant cell tumor. [2023]

2. England 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. [2021]

3. England pubmed.ncbi.nlm.nih.gov

Long-term follow-up of nilotinib in patients with advanced tenosynovial giant cell tumours: Long-term follow-up of nilotinib in TGCT. [2022]

4. United States pubmed.ncbi.nlm.nih.gov

Long term term follow-up of tyrosine kinase inhibitors treatments in inoperable or relapsing diffuse type tenosynovial giant cell tumors (dTGCT). [2020]

5. England pubmed.ncbi.nlm.nih.gov

Localized tenosynovial giant cell tumor in children. [2023]

6. England pubmed.ncbi.nlm.nih.gov

Tenosynovial giant cell tumors (TGCT): molecular biology, drug targets and non-surgical pharmacological approaches. [2022]

7. United States pubmed.ncbi.nlm.nih.gov

[18F]-FDG-PET/CT Correlates With the Response of Radiorefractory Thyroid Cancer to Lenvatinib and Patient Survival. [2021]

8. United States pubmed.ncbi.nlm.nih.gov

Phase II results of Dovitinib (TKI258) in patients with metastatic renal cell cancer. [2021]

9. United States pubmed.ncbi.nlm.nih.gov

Vandetanib for the treatment of medullary thyroid cancer. [2022]

10. United States pubmed.ncbi.nlm.nih.gov

Antitumor activity of lenvatinib (e7080): an angiogenesis inhibitor that targets multiple receptor tyrosine kinases in preclinical human thyroid cancer models. [2022]

11. United States pubmed.ncbi.nlm.nih.gov

Lenvatinib in Advanced, Radioactive Iodine-Refractory, Differentiated Thyroid Carcinoma. [2015]