Pimicotinib for Giant Cell Tumor

Recruiting at 39 trial locations

Overseen By YUAN LU

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Abbisko Therapeutics Co, Ltd

Must not be taking: Strong CYP inhibitors

Disqualifiers: Metastatic TGCT, Active infections, 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

90 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot use strong CYP inhibitors or inducers during the study. It's best to discuss your current medications with the study team.

What makes the drug Pimicotinib unique for treating Giant Cell Tumor?

Pimicotinib (ABSK021) is unique because it targets specific pathways involved in tumor growth, potentially offering a novel approach compared to existing treatments. While other treatments may not specifically target these pathways, Pimicotinib's mechanism could provide a new option for patients with Giant Cell Tumor. ^{1 2 3 4 5}

Eligibility Criteria

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.

Inclusion Criteria

Participants should complete stiffness and pain scales during the screening period, and symptomatic disease because of active TGCT should meet minimum requirements as outlined in study protocol

I am fully active or restricted in physically strenuous activity but can do light work.

I am 18 years old or older.

Exclusion Criteria

Known allergy or hypersensitivity to any components of the investigational drug product

Any other clinically significant comorbidities, which in the judgment of the Investigator, could compromise compliance with the protocol, interfere with the interpretation of study results, or predispose the patient to safety risks

I have been treated with specific inhibitors but not those targeting CSF-1/CSF-1R, except imatinib and nilotinib are okay.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Part 1

Participants receive the blinded treatment of Pimicotinib(ABSK021) or placebo for 24 weeks

24 weeks

Regular visits as per protocol

Treatment Part 2

Participants receive open-label Pimicotinib(ABSK021) for 24 weeks

24 weeks

Regular visits as per protocol

Open-label Extension

Participants receive open-label Pimicotinib(ABSK021) until study end or withdrawal

Indefinite until study end

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Pimicotinib(ABSK021)
• Placebo

Trial Overview 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.

Participant Groups

Group I: Part 1/Part 2/Part 3- Pimicotinib(ABSK021)/ Pimicotinib(ABSK021) Experimental Treatment 1 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 Group 2 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.

Who Is Running the Clinical Trial?

Abbisko Therapeutics Co, Ltd

Lead Sponsor

Trials

26

Recruited

1,700+

Findings from Research

In a Phase 1b study involving 110 patients with advanced solid tumors, gedatolisib combined with cisplatin showed promising clinical activity in triple-negative breast cancer (TNBC), with an overall response rate of 40% in first-line treatment and 33.3% in second/third-line treatment.

The combination therapy was generally well-tolerated, with only 10% of evaluable patients experiencing dose-limiting toxicities, primarily grade 3 oral mucositis, indicating a manageable safety profile for further investigation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A Phase 1B open-label study of gedatolisib (PF-05212384) in combination with other anti-tumour agents for patients with advanced solid tumours and triple-negative breast cancer. Curigliano, G., Shapiro, GI., Kristeleit, RS., et al.[2023]

NVP-BKM120, a selective PI3K inhibitor, effectively inhibits growth in glioblastoma cells and blocks the PI3K/Akt signaling pathway, leading to different forms of cell death depending on the p53 status of the cells.

In vivo studies using an intracranial U87 glioma model showed that NVP-BKM120 significantly increased median survival from 26 days in the control group to 38 and 48 days in treated groups, indicating its potential as a glioma treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Antitumor activity of NVP-BKM120--a selective pan class I PI3 kinase inhibitor showed differential forms of cell death based on p53 status of glioma cells. Koul, D., Fu, J., Shen, R., et al.[2022]

References

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

A Phase 1B open-label study of gedatolisib (PF-05212384) in combination with other anti-tumour agents for patients with advanced solid tumours and triple-negative breast cancer. [2023]

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

Antitumor activity of NVP-BKM120--a selective pan class I PI3 kinase inhibitor showed differential forms of cell death based on p53 status of glioma cells. [2022]

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

Targeting of glioblastoma cell lines and glioma stem cells by combined PIM kinase and PI3K-p110α inhibition. [2019]

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

New Mechanisms of mTOR Pathway Activation in KIT-mutant Malignant GISTs. [2021]

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

Inhibition of PI3K/mTOR pathways in glioblastoma and implications for combination therapy with temozolomide. [2022]