Pimicotinib for Giant Cell Tumor

Not currently recruiting at 42 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called Pimicotinib (also known as ABSK021) to evaluate its effectiveness and safety for individuals with Tenosynovial Giant Cell Tumor (TGCT), a type of tumor not easily removed by surgery. Participants will receive either Pimicotinib or a placebo (a substance with no active drug) for 24 weeks and may then continue with Pimicotinib for a longer period. Only those with TGCT confirmed by a doctor, who cannot undergo surgical removal, and who require regular pain medication, may qualify for this study. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to the potential availability of a new treatment.

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.

Is there any evidence suggesting that Pimicotinib(ABSK021) is likely to be safe for humans?

Research has shown that Pimicotinib, also known as ABSK021, is safe for treating Tenosynovial Giant Cell Tumor (TGCT). Studies have found it effective and well-tolerated. Pimicotinib demonstrated strong tumor-fighting abilities without harming the liver, indicating a positive safety profile. Tests at daily doses of 50 mg and 25 mg both proved safe. Early trials reported no major safety issues, suggesting it could be a reliable option for patients with TGCT. ^{1 2 3 4 5}

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

Researchers are excited about Pimicotinib (ABSK021) for treating Giant Cell Tumor because it offers a new approach that targets specific pathways involved in tumor growth. Unlike current treatments, which often involve surgery or radiation, Pimicotinib is designed to work by inhibiting the CSF1R pathway, a key player in the development of these tumors. This targeted action could potentially lead to more effective management of the disease with fewer side effects, offering hope for improved patient outcomes.

What evidence suggests that Pimicotinib might be an effective treatment for TGCT?

Research shows that Pimicotinib holds promise for treating Tenosynovial Giant Cell Tumor (TGCT). Studies have found that it improves physical function and reduces symptoms. One study highlighted that Pimicotinib led to significant and meaningful results for TGCT patients. It blocks a protein called CSF-1, which aids tumor growth. Previous research also shows that Pimicotinib is effective and well-tolerated, making it a strong option for people with TGCT. Participants in this trial may receive Pimicotinib or a placebo in Part 1, with the option to continue on open-label Pimicotinib in subsequent parts of the study. ^{1 2 3 5 6}

Are You a Good Fit for This Trial?

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.

Patients should understand the study procedures and sign the informed consent form prior to screening

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

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]

Citations

1. ascopubs.org ascopubs.org/doi/10.1200/JCO.2025.43.16_suppl.11500

Pimicotinib in tenosynovial giant cell tumor (TGCT) Pimi produced statistically significant and clinically meaningful improvements in physical function and symptoms, representing an effective, well-tolerated and ...

2. emdserono.com emdserono.com/us-en/company/news/press-releases/phase-3-maneuver-pt-1-pimicotinib-data-28-05-2025.html

2025-05-28 Phase 3 MANEUVER Pt 1 Pimicotinib Data Pimicotinib demonstrates best-in-class potential with significant efficacy and clinically meaningful improvements in patients with tenosynovial giant cell ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT05804045

Study of Pimicotinib (ABSK021) for Tenosynovial Giant Cell ... The goal of this clinical trial is to assess the efficacy and safety of Pimicotinib (ABSK021) in patients with Tenosynovial Giant Cell Tumor (TGCT).

4. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39287124/

MANEUVER: A Phase III study of pimicotinib to assess ... Pimicotinib is a medicine which blocks CSF-1, and researches have shown that it is safe and effective for treating TGCT in smaller, early study.

5. onclive.com onclive.com/view/long-term-maneuver-trial-data-spotlight-durable-responses-with-pimicotinib-in-tgct

Long-Term MANEUVER Trial Data Spotlight Durable ... Abstract 2690. Pimicotinib significantly improved outcomes for patients with tenosynovial giant cell tumor in a global phase III trial. News ...

6. asco.org asco.org/abstracts-presentations/ABSTRACT407670

Efficacy and safety profile of pimicotinib (ABSK021) in ... Pimicotinib has demonstrated a significant antitumor activity, favorable safety, and PK profiles at both 50 mg QD and 25 mg QD with no apparent hepatotoxicity.