What side effects are associated with this type of intervention?

CSF1R inhibitors, such as Vimseltinib, are being studied for the treatment of Giant Cell Tumor. Common side effects of CSF1R inhibitors can include fatigue, nausea, diarrhea, and liver enzyme abnormalities. Other potential side effects may involve hematologic issues such as anemia and thrombocytopenia. As with any targeted therapy, there is also a risk of more severe adverse effects, which should be monitored closely by healthcare providers.

What prior FDA approvals exist?

The FDA has approved denosumab, a RANKL inhibitor, for the treatment of Giant Cell Tumor of bone that is unresectable or where surgical resection is likely to result in severe morbidity. Denosumab works by inhibiting the RANK/RANKL pathway, which is crucial for the formation and function of osteoclasts, the cells responsible for bone resorption. While Vimseltinib is a CSF1R inhibitor targeting a different pathway, both drugs aim to reduce tumor growth by interfering with cellular mechanisms essential for tumor maintenance.

What other types of research exist?

Promising alternatives to Vimseltinib for Giant Cell Tumor patients include Trastuzumab, Gefitinib, and Sorafenib. These drugs are being explored for their potential in treating neoplastic conditions and may offer individualized therapy options for better outcomes.

← Back to Search

Kinase Inhibitor

Vimseltinib for Tenosynovial Giant Cell Tumor (MOTION Trial)

Phase 3

Waitlist Available

Research Sponsored by Deciphera Pharmaceuticals, LLC

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Symptomatic disease as defined as at least moderate pain or at least moderate stiffness within the screening period and documented in the medical record

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

Must not have

If female, the participant is pregnant or breastfeeding

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline to week 25 (cycle 7, day 1)

Awards & highlights

Pivotal Trial

Summary

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.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Specific side effects are not listed here but may include typical reactions related to oral medications such as digestive issues or allergic reactions; plus any potential unique effects of vimseltinib which would be monitored through assessments during the trial.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

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

Select...

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

Select...

I am 18 years old or older.

Select...

My pain medication has not changed in the last 2 weeks.

Select...

I have a tumor that can be measured and is at least 2cm big.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

I am currently pregnant or breastfeeding.

Select...

I have a liver condition like NASH or cirrhosis.

Select...

I do not have conditions that affect how my body absorbs medication.

Select...

I have not had major surgery within the last 2 weeks.

Select...

I do not have active HIV, hepatitis B, hepatitis C, or tuberculosis.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline to week 25 (cycle 7, day 1)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline to week 25 (cycle 7, day 1)

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline to week 25 (cycle 7, day 1) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

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

Secondary study objectives

ORR per Tumor Volume Score (TVS)

Physical function

Quality of life (QoL)

+3 more

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: Part 1/Part 2 - vimseltinib/vimseltinibExperimental Treatment1 Intervention

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

Group II: Part 1/Part 2 - placebo/vimseltinibPlacebo Group2 Interventions

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

0 / 10Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Giant Cell Tumor (GCT) treatments often target the CSF1R (colony-stimulating factor 1 receptor) pathway, which is crucial for the survival and proliferation of the tumor's osteoclast-like giant cells. Vimseltinib, a CSF1R inhibitor, works by blocking this receptor, thereby reducing the recruitment and activity of these giant cells, leading to tumor shrinkage and symptom relief. This mechanism is significant for GCT patients as it directly targets the cellular interactions driving tumor growth, offering a more focused and potentially effective treatment option compared to traditional therapies.

Emerging systemic treatment options in meningioma.Anti-angiogenic therapy for high-grade glioma.Rechallenge with mTOR inhibitors in metastatic renal cell carcinoma patients who progressed on previous mTOR inhibitor therapy.