What side effects are associated with this type of intervention?

Common side effects of treatments for Giant Cell Tumor, particularly those involving CSF1R inhibitors like Pimicotinib, often include gastrointestinal symptoms such as nausea and diarrhea, as well as fatigue and potential skin reactions like rash. These treatments may also cause hematologic issues such as neutropenia. It is important for patients to discuss these potential side effects with their healthcare provider to manage and mitigate them effectively.

What prior FDA approvals exist?

The FDA has approved denosumab (Xgeva) for the treatment of Giant Cell Tumor of bone that is unresectable or where surgical resection is likely to result in severe morbidity. Denosumab is a monoclonal antibody that works by inhibiting RANKL, a protein involved in bone resorption. While not a CSF1R inhibitor like Pimicotinib, denosumab represents a targeted approach to treating GCT by interfering with specific molecular pathways involved in the disease.

What other types of research exist?

Promising alternatives to Pimicotinib for Giant Cell Tumor patients include: 1) Denosumab, a RANKL inhibitor that has shown efficacy in reducing tumor size and pain. 2) Nivolumab, a PD-1 inhibitor, which has demonstrated potential in treating various tumors by enhancing immune response. 3) Cabozantinib, a tyrosine kinase inhibitor, which has shown activity in bone-related tumors. These alternatives have been evaluated in clinical trials and may offer effective treatment options for GCT patients.

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Tyrosine Kinase Inhibitor

Pimicotinib for Giant Cell Tumor

Phase 3

Waitlist Available

Research Sponsored by Abbisko Therapeutics Co, Ltd

Eligibility Criteria Checklist

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

Must have

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up the time (months) from the first documentation of objective response to the first documentation of radiographic disease progression (pd) or death due to any cause, whichever occurs first, assessed up to 24 months.

Awards & highlights

Pivotal Trial

Summary

This trial tests Pimicotinib, a new drug, in patients with Tenosynovial Giant Cell Tumor (TGCT). The drug aims to stop the tumor from growing by blocking signals that help tumor cells grow. The study will check if the drug works well and is safe for these patients.

Who is the study for?

Adults over 18 with a confirmed diagnosis of Tenosynovial Giant Cell Tumor (TGCT) that can't be surgically removed and have at least one measurable lesion. They must understand the study, sign consent, have stable pain medication needs, adequate organ and bone marrow function, and an ECOG performance status of 0 or 1.

What is being tested?

The trial is testing Pimicotinib (ABSK021), comparing it to a placebo to see if it's effective and safe for TGCT patients. Part one lasts about 24 weeks with either Pimicotinib or placebo; part two continues with Pimicotinib for another 24 weeks.

What are the potential side effects?

Potential side effects may include allergic reactions to the drug components, issues related to oral absorption affecting digestion, changes in liver function tests indicating liver health concerns, as well as general risks associated with taking new medications.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ the time (months) from the first documentation of objective response to the first documentation of radiographic disease progression (pd) or death due to any cause, whichever occurs first, assessed up to 24 months.

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~the time (months) from the first documentation of objective response to the first documentation of radiographic disease progression (pd) or death due to any cause, whichever occurs first, assessed up to 24 months.

This trial's timeline: 3 weeks for screening, Varies for treatment, and the time (months) from the first documentation of objective response to the first documentation of radiographic disease progression (pd) or death due to any cause, whichever occurs first, assessed up to 24 months. 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)

Secondary study objectives

Duration of Response (DOR)

Objective Response Rate (ORR) per Tumor Volume Score (TVS)

Physical Function

+4 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/Part 3- Pimicotinib(ABSK021)/ Pimicotinib(ABSK021)Experimental Treatment1 Intervention

Participants receive the blinded treatment of ABSK021 for 24 weeks in Part 1 and continue on the open-label Pimicotinib(ABSK021) in Part 2 and Part 3.

Group II: Part 1- Placebo/ Pimicotinib(ABSK021)Placebo Group2 Interventions

Participants receive the blinded treatment of matching placebo for 24 weeks in Part 1 and have option to receive the open-label Pimicotinib(ABSK021) in Part 2.

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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

Pimicotinib (ABSK021) is a CSF1R inhibitor, which works by blocking the colony-stimulating factor 1 receptor (CSF1R) pathway. This pathway is crucial for the survival and proliferation of the macrophage-like cells that drive the growth of Giant Cell Tumors (GCT). By inhibiting CSF1R, Pimicotinib reduces the recruitment and survival of these cells, thereby potentially shrinking the tumor and alleviating symptoms. This mechanism is significant for GCT patients as it targets the root cause of the tumor's growth, offering a more focused and potentially effective treatment option compared to traditional therapies.

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