Rare Diseases

All Conditions

Crouzon Syndrome

Jackson-Weiss Syndrome

Pfeiffer Syndrome

Pemigatinib will be treated according to the treatment plan of 2 weeks of administration/1 week of discontinuation, with oral administration of 1 capsule, 13.5mg, QD, and a cycle of 21 days. Meanwhile, the molecular testing results of the patient are analyzed and interpreted by the MTB team, and appropriate combination therapy(Pemigatinib+) strategies are proposed based on the patient's previous treatment history, physical condition, drug accessibility, and economic status.

Pemigatinib

HaiTao Wang, Ph.D

Jinhuan Wang, Ph.D

Haitao Wang, Ph.D

Haitao Wang

Jinhuan Wang

Lili Wang

Dingkun Hou

The purpose of this clinical trial is to demonstrate safety and efficacy of Pemigatinib in the treatment of advanced pan solid tumor patients with FGF/FGFR alterations.

This study is a prospective, single arm, phase II clinical study. Patients with advanced solid tumors who had previously failed standard treatment and had FGFR1-3 alterations were selected and included in this study after signing the informed consent form and meeting the inclusion criteria. The patient will receive oral treatment with pemitinib (13.5 mg QD, administered for 2 weeks/discontinued for 1 week). Before assessment for eligibility, patients were prescreened centrally for FGF/FGFR status using massively parallel DNA sequencing.Patients who already had an FGF/FGFR status report based on local assessment (Clinical Laboratory Improvement Amendments \[CLIA\]-certifed) or an existing MTB approved report were also included. The subjects will continue treatment until disease progression or intolerable toxicity occurs. During the treatment process, clinical tumor imaging evaluation was conducted according to RECIST v1.1, every 6 weeks (± 7 days), and every 9 weeks (± 7 days) after 48 weeks. Use NCI-CTCAE 5.0 for safety assessment.

Pemigatinib for FGF/FGFR Alterations Advanced Pan Solid Tumors

Safety and Efficacy of Pemigatinib in the Treatment of Advanced Pan Solid Tumors With FGF/FGFR Alterations: An Open, Single-arm, Prospective Phase II Trial

The progression free survival (PFS1) after the most recent treatment before enrollment is defined as the progression of the disease from the most recent treatment before enrollment.The progression free survival period (PFS2) after enrollment is defined as the time from matched targeted therapy or unmatched therapy to disease progression or death.

The proportion of patients whose tumor volume is reduced to 30% and can be maintained for more than 4 weeks,Based on RECIST criteria v1.1.

The time from the beginning of the patient's treatment to the disease progression or death for any reason.Based on RECIST criteria v1.1.

The DCR was defined as SD, PR or CR according to RECIST criteria v1.1.

The time from the patient receiving treatment to the death of the patient for any reason,OS evaluated according to RECIST v1.1.

Duration of Response,from the first time the evaluation results meet CR or PR criteria to the observation of PD or death.

Include Treatment emerge adverse events, treatment related adverse events and serious adverse events,AEs evaluated according to NCI-CTCAE v5.0.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Pemigatinib for FGF/FGFR Alterations Advanced Pan Solid Tumors	NCT06022289	Solid Tumor FGF Receptor Ge... FGF Amplificati... FGF Receptor Ge... FGF Receptor Ge...	Pemigatinib	18 Years -	Tianjin Medical University Second Hospital
Pemigatinib for FGF/FGFR Alterations Advanced Pan Solid Tumors	NCT06022289	Solid Tumor FGF Receptor Ge... FGF Amplificati... FGF Receptor Ge... FGF Receptor Ge...	Pemigatinib	18 Years -	Tianjin Medical University Second Hospital
Pemigatinib for FGF/FGFR Alterations Advanced Pan Solid Tumors	NCT06022289	Solid Tumor FGF Receptor Ge... FGF Amplificati... FGF Receptor Ge... FGF Receptor Ge...	Pemigatinib	18 Years -	Tianjin Medical University Second Hospital

Spots Global Cancer Trial Database for fgf receptor gene translocation