Study to Evaluate the Efficacy and Safety of Pemigatinib in Participants With Relapsed or Refractory Advanced Non-Small Cell Lung Cancer With an FGFR Alteration

NCT05253807 | Completed Phase 2 Results FDA Drug

• 2 other identifiers: INCB 54828-2102021-004934-12
• Study Type: interventional
• Target: 8
• Locations: 5 countries
• Sites: 36

Brief Summary

This is an open-label, single arm study to study the safety, efficacy and tolerability of Pemigatinib when used on participants with squamous or nonsquamous NSCLC with a documented FGFR1-3 mutations or fusions/rearrangement who have progressed on prior therapies and have no available standard treatment options

Conditions

Non-Small Cell Lung Cancer (NSCLC)

Keywords

Advanced Non-Small Cell Lung Cancer (NSCLC); fibroblast growth factor receptor (FGFR); FGFR1-3 mutations; FGFR1-3 fusions; FGFR1-3 rearrangements; squamous NSCLC; nonsquamous NSCLC; relapsed or refractory

Outcome Measures

Primary Outcomes (1)

• Objective Response Rate (ORR) in Cohort A

  ORR was defined as the percentage of participants who achieved a complete response (CR) or a partial response (PR) based on Response Evaluation Criteria in Solid Tumors (RECIST) v1.1. Response was determined by an Independent Central Radiology (ICR) review. CR: disappearance of all target and non-target lesions and no appearance of any new lesions. Any pathological lymph nodes (whether target or non-target) must have a reduction in the short axis to \<10 millimeters (mm). PR: complete disappearance or at least a 30% decrease in the sum of the diameters of target lesions, taking as a reference the baseline sum diameters, no new lesions, and no progression of non-target lesions.

  up to 267 days

Secondary Outcomes (6)

• ORR in Cohort B

  up to 80 days

• Progression-free Survival (PFS) in Cohort A

  up to 267 days

• Duration of Response (DOR) in Cohort A

  up to 182 days

• Overall Survival (OS) in Cohort A

  up to 267 days

• Number of Participants With Any Treatment-emergent Adverse Event (TEAE)

  up to 302 days

Study Arms (2)

Cohort A: Squamous NSCLC

EXPERIMENTAL

Participants with squamous NSCLC with known or likely FGFR1-3 driver mutations outside the kinase domain or fusions/rearrangements will receive intermittent dosing.

Drug: Pemigatinib

Cohort B: Non-squamous NSCLC

EXPERIMENTAL

Participants with non-squamous NSCLC with known or likely FGFR1-3 driver mutations outside the kinase domain or fusions/rearrangements will receive intermittent dosing.

Drug: Pemigatinib

Interventions

Pemigatinib DRUG

13.5 mg tablet

Also known as: INCB 54828

Cohort A: Squamous NSCLC; Cohort B: Non-squamous NSCLC

Eligibility Criteria

• Age: 18 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Histologically or cytologically confirmed advanced or metastatic NSCLC (Stage IIIB/C or IV per the AJCC Cancer Staging Manual, 8th Edition). Both squamous and nonsquamous NSCLC are eligible.
• Radiographically measurable disease (per RECIST v1.1). Tumor lesions located in a previously irradiated area, or in an area subjected to other loco-regional therapy, are considered measurable if progression has been clearly demonstrated in the lesion.
• Documentation of known/likely actionable known or likely FGFR1-3 alterations.
• Must have objective documented progression after at least 1 prior therapy, and must have no therapy available that is likely to provide clinical benefit. Participants who are intolerant of or decline the approved therapy are eligible only if they have no therapy available that is likely to provide clinical benefit.
• ECOG performance status of 0 to 2.
• Baseline archival tumor specimen (if less than 24 months from date of screening) or willingness to undergo a pretreatment tumor biopsy to obtain the specimen. Must be a tumor block or approximately 15 unstained slides from biopsy or resection of primary tumor or metastasis.
• Willingness to avoid pregnancy or fathering a child.

You may not qualify if:

• Prior receipt of a selective FGFR inhibitor.
• Receipt of anticancer medications or investigational drugs for any indication or reason within 28 days before the first dose of pemigatinib. Participants must have recovered (≤ Grade 1 as per CTCAE v5.0 or at pretreatment baseline) from AEs from previously administered therapies (excluding alopecia).
• Concurrent anticancer therapy (eg, chemotherapy, immunotherapy, biologic therapy, hormonal therapy, or investigational therapy).
• Candidate for potentially curative surgery.
• Current evidence of clinically significant corneal (including but not limited to bullous/band keratopathy, corneal abrasion, inflammation/ulceration, and keratoconjunctivitis) or retinal disorder (including but not limited to macular/retinal degeneration, diabetic retinopathy, and retinal detachment) as confirmed by ophthalmologic examination.
• Radiation therapy administered for the treatment of cancer lesions within 2 weeks before enrollment/first dose of study drug. Participants must have recovered from all radiation related toxicities, not require corticosteroids, and not have had radiation pneumonitis. Evidence of fibrosis within a radiation field from prior radiotherapy is permitted with medical monitor approval. A 1-week washout is permitted for palliative radiation to non-CNS disease.
• Untreated brain or CNS metastases or brain or CNS metastases that have progressed (eg, evidence of new or enlarging brain metastasis or new neurological symptoms attributable to brain or CNS metastases). Participants who have previously treated and clinically stable brain or CNS metastases are eligible if there is no evidence of progression for at least 4 weeks after CNS-directed treatment, as ascertained by clinical examination and brain imaging (MRI or CT scan) during the screening period, and if they are on a stable or decreasing dose of corticosteroids for at least 1 week.
• Known additional malignancy that is progressing or requires active treatment. Exceptions include basal cell carcinoma of the skin, squamous cell carcinoma of the skin, or in situ cervical cancer that has undergone potentially curative therapy.
• Participants with defined laboratory values at screening.
• History of calcium and phosphate hemostasis disorder or systemic mineral imbalance with ectopic calcification of soft tissues (exception: commonly observed calcifications in soft tissues such as the skin, kidney tendon, or vessels due to injury, disease, or aging in the absence of systemic mineral imbalance).
• History of hypovitaminosis D requiring supraphysiologic doses (eg, 50,000 UI/weekly) to replenish the deficiency. Vitamin D supplements are allowed.

Sponsors & Collaborators

• Incyte Corporation: lead

Study Sites (36)

Valkyrie Clinical Trials

Los Angeles, California, 90067, United States

Location

Florida Cancer Specialists & Research Institute

Fort Myers, Florida, 33901, United States

Location

Miami Cancer Institute

Miami, Florida, 33176, United States

Location

Memorial Healthcare System

Pembroke Pines, Florida, 33028, United States

Location

University of Kentucky Hospital

Lexington, Kentucky, 40536, United States

Location

Spoknwrd Clinical Trials Inc.

Easton, Pennsylvania, 18045, United States

Location

Tennessee Oncology

Nashville, Tennessee, 37203, United States

Location

H�PITAL NORD - CHU MARSEILLE

Marseille, 13385, France

Location

Chu de Toulouse Hopital Larrey Centre de Reference Des Maladies Rares de La Peau Service de Dermatol

Toulouse, 31000, France

Location

Zentralklinik Bad Berka Gmbh

Bad Berka, 99437, Germany

Location

Lungenklinik Hemer

Hemer, 58675, Germany

Location

Lki Lungenfachklinik Immenhausen

Immenhausen, 34376, Germany

Location

University Hospital Mannheim

Mannheim, 68167, Germany

Location

Irccs Centro Di Riferimento Oncologico

Aviano, 33081, Italy

Location

Istituto Tumori Giovanni Paolo Ii Irccs Ospedale Oncologico Bari

Bari, 70124, Italy

Location

Istituto Romagnolo Per Lo Studio Dei Tumori Dino Amadori

Meldola, 47014, Italy

Location

Azienda Ospedaliera Universitaria San Luigi Gonzaga Orbassano

Orbassano, 10043, Italy

Location

Azienda Ospedaliera Di Perugia - Ospedale Santa Maria Della Misericordia

Perugia, 06132, Italy

Location

Azienda Ospedaliero Universitaria Pisana

Pisa, 56124, Italy

Location

Istituto Nazionale Tumori Regina Elena Irccs

Roma, 00144, Italy

Location

Irccs Istituto Clinico Humanitas

Rozzano, 20089, Italy

Location

Complejo Hospitalario Universitario A Coruna

A Coru?a, 15006, Spain

Location

Hospital General Universitario Vall D Hebron

Barcelona, 08035, Spain

Location

Hospital Clinic de Barcelona

Barcelona, 08036, Spain

Location

Hospital de La Santa Creu I Sant Pau

Barcelona, 08041, Spain

Location

Ico Girona Hospital Universitari de Girona Dr Josep Trueta

Girona, 17007, Spain

Location

Hospital Universitario Ciudad de Jaen

Jaén, 23007, Spain

Location

Ico Institut Catala D Oncologia

L'Hospitalet de Llobregat, 08908, Spain

Location

Hospital General Universitario Gregorio Maranon

Madrid, 28007, Spain

Location

Hospital Universitario Ramon Y Cajal

Madrid, 28034, Spain

Location

Hospital Universitario 12 de Octubre

Madrid, 28041, Spain

Location

Hospital Universitario de La Paz

Madrid, 28046, Spain

Location

Hospital Universitario Hm Sanchinarro

Madrid, 28050, Spain

Location

Hospital Regional Universitario de Malaga

Málaga, 29010, Spain

Location

Hospital Universitario Virgen Macarena

Seville, 41009, Spain

Location

Hospital Universitario Miguel Servet

Zaragoza, 50009, Spain

Location

MeSH Terms

Conditions

Carcinoma, Non-Small-Cell Lung; Acrocephalosyndactylia; Recurrence

Interventions

pemigatinib; INCB 54828-101

Condition Hierarchy (Ancestors)

Carcinoma, Bronchogenic; Bronchial Neoplasms; Lung Neoplasms; Respiratory Tract Neoplasms; Thoracic Neoplasms; Neoplasms by Site; Neoplasms; Lung Diseases; Respiratory Tract Diseases; Craniosynostoses; Synostosis; Dysostoses; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Syndactyly; Craniofacial Abnormalities; Musculoskeletal Abnormalities; Limb Deformities, Congenital; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Study Director
• Organization: Incyte Corporation

medinfo@incyte.com

1-855-463-3463

Study Officials

• Luisa Veronese, MD

  Incyte Corporation

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Participants enrolled in this study will be assigned to 1 of 2 cohorts: Cohort A (squamous NSCLC) will enroll approximately 100 participants, and Cohort B (nonsquamous NSCLC) will enroll approximately 25 participants.

Study Record Dates

• First Submitted: February 14, 2022
• First Posted: February 24, 2022
• Study Start: April 29, 2022
• Primary Completion: August 16, 2023
• Study Completion: August 16, 2023
• Last Updated: August 5, 2025
• Results First Posted: August 6, 2024

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• Time Frame: Data will be shared after the primary publication or 2 years after the study has ended for market authorized products and indications.
• Access Criteria: Data from eligible studies will be shared with qualified researchers according to the criteria and process described in the Data Sharing section of the www.incyteclinicaltrials.com website. For approved requests, the researchers will be granted access to anonymized data under the terms of a data sharing agreement.

Locations

FR (2); US (7); ES (15); IT (8); DE (4)