Pemigatinib for the Treatment of Metastatic or Unresectable Colorectal Cancer Harboring FGFR Alterations

NCT04096417 | Unknown Phase 2 Results DMC FDA Drug

• 3 other identifiers: ACCRU-GI-1701NCI-2019-05877P30CA015083
• Study Type: interventional
• Target: 14
• Locations: 1 country
• Sites: 6

Brief Summary

This phase II trial studies how well pemigatinib works in treating patients with colorectal cancer with mutations (alterations) in a FGFR gene and that has spread to other places in the body (metastatic) or cannot be removed by surgery (unresectable). Pemigatinib may stop the growth of tumor cells by blocking FGFR, which is needed for cell growth.

Conditions

FGFR1 Gene Amplification; FGFR1 Gene Mutation; FGFR1 Gene Translocation; FGFR2 Gene Amplification; FGFR2 Gene Mutation; FGFR2 Gene Translocation; FGFR3 Gene Amplification; FGFR3 Gene Mutation; FGFR3 Gene Translocation; Metastatic Colorectal Carcinoma; Stage III Colorectal Cancer AJCC v8; Stage IIIA Colorectal Cancer AJCC v8; Stage IIIB Colorectal Cancer AJCC v8; Stage IIIC Colorectal Cancer AJCC v8; Stage IV Colorectal Cancer AJCC v8; Stage IVA Colorectal Cancer AJCC v8; Stage IVB Colorectal Cancer AJCC v8; Stage IVC Colorectal Cancer AJCC v8; Unresectable Colorectal Carcinoma

Outcome Measures

Primary Outcomes (1)

• Overall Response Rate (ORR)

  Defined as the rate of patients, among evaluable patients, who experience an objective response per RECIST 1.1. The proportion of successes will be estimated by the number of successes divided by the total number of evaluable patients. Confidence intervals for the true success proportion will be calculated according to the approach of Clopper and Pearson.

  4.4 Months

Secondary Outcomes (5)

• Clinical Benefit Rate

  4.4 Months

• Progression-free Survival (PFS)

  4.4 Months

• Overall Survival (OS)

  29.4 Months

• Quality of Life (QOL) as Measured by the LASA [Item 1: Overall QOL]

  9 Months

• Incidence of Adverse Events

  5.4 Months

Study Arms (1)

Treatment (pemigatinib)

EXPERIMENTAL

Patients receive pemigatinib PO QD on days 1-21. Treatment repeats every 21 days for up to 35 cycles in the absence of disease progression or unacceptable toxicity.

Drug: Pemigatinib; Other: Quality-of-Life Assessment

Interventions

Pemigatinib DRUG

Given PO

Also known as: INCB054828

Treatment (pemigatinib)

Quality-of-Life Assessment OTHER

Ancillary studies

Also known as: Quality of Life Assessment

Treatment (pemigatinib)

Eligibility Criteria

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

You may qualify if:

• Registered to Colorectal and Liquid Biopsy Molecularly Assigned Therapy (COLOMATE) Academic and Community Cancer Research United (ACCRU)-GI-1611 and:
• COLOMATE Companion Trial Recommendation Form indicates patient qualifies to be screened for a COLOMATE companion trial
• COLOMATE Companion Trial Recommendation Form date of completion is =\< 30 days prior to registration
• Histologically or cytologically confirmed diagnosis of metastatic or unresectable colorectal cancer (mCRC), based on documentation from local or outside review of pathology according to each site?s established institutional procedure
• Documentation of an activating genomic alteration(s) in FGFR1-3 (gain of function mutations, translocations, and amplifications allowed)
• Provide informed written consent
• Patient must have received and progressed on, or be intolerant to, each of the following treatments for mCRC (or have contraindication to these treatments):
• Fluoropyrimidine
• Oxaliplatin
• Irinotecan
• Anti-VEGF (vascular endothelial growth factor) monoclonal antibody, if eligible for this therapy
• Anti-EGFR (epidermal growth factor receptor) monoclonal antibody, if eligible for this therapy
• Measurable disease
• Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0, 1, or 2
• Absolute neutrophil count (ANC) \>= 1500/mm\^3 (obtained =\< 28 days prior to registration)

You may not qualify if:

• Prior treatment with pemigatinib
• Prior treatment with a selective FGFR inhibitor =\< 180 days (6 months) prior to registration
• Known hypersensitivity or severe reaction to an FGFR inhibitor, or to the excipients of pemigatinib (i.e. microcrystalline cellulose, sodium starch glycolate, and magnesium stearate)
• Current evidence of clinically significant corneal or retinal disorder confirmed by ophthalmologic examination
• Treatment with other investigational study drug for any indication for any reason, or receipt of anticancer medications =\< 14 days prior to registration
• Major surgery =\< 28 days prior to registration
• External beam radiation therapy =\< 28 days prior to registration, or palliative radiation for non-central nervous system (CNS) disease =\< 14 days prior to registration
• Brain metastases, central nervous system (CNS) metastases, leptomeningeal disease, or spinal cord compression
• NOTE: Patients who are asymptomatic or previously treated and stable, without evidence of progression for \>= 28 days prior to registration are eligible
• NOTE: Patients taking concomitant corticosteroids and/or anticonvulsants are allowed if patient is on a stable or decreasing dose of such treatment for \>= 28 days prior to registration
• History or presence of significant cardiovascular disease or condition including:
• Uncontrolled angina pectoris (Canadian Cardiovascular Society grade II-IV despite medical therapy)
• Congestive heart failure (New York Heart Association class III or IV)
• Uncontrolled arrhythmia requiring therapy. Note: Patients with a pacemaker and well-controlled rhythm for \>= 28 days prior to registration are not excluded
• Any of the following occurring =\< 6 months prior to registration: myocardial infarction, angioplasty, cardiac stenting, coronary/peripheral artery bypass graft, cerebrovascular accident or transient ischemic attack

Sponsors & Collaborators

• Academic and Community Cancer Research United: lead
• National Cancer Institute (NCI): collaborator

Study Sites (6)

Mayo Clinic in Arizona

Scottsdale, Arizona, 85259, United States

Location

Emory University Hospital/Winship Cancer Institute

Atlanta, Georgia, 30322, United States

Location

Mayo Clinic in Rochester

Rochester, Minnesota, 55905, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Vanderbilt University/Ingram Cancer Center

Nashville, Tennessee, 37232, United States

Location

Aurora Cancer Care-Milwaukee West

Wauwatosa, Wisconsin, 53226, United States

Location

Related Publications (1)

• Wheless MC, Zemla TJ, Hubbard JM, Strickler JH, Gbolahan OB, Wilson L, Waechter B, Ou FS, Nixon AB, Bekaii-Saab TS, Ciombor KK. A phase II, multicenter, single-arm study of pemigatinib in patients with metastatic or unresectable colorectal cancer harboring FGFR alterations. Oncologist. 2025 Jun 4;30(6):oyaf069. doi: 10.1093/oncolo/oyaf069.

  PMID: 40515475 DERIVED

MeSH Terms

Conditions

Colorectal Neoplasms

Interventions

pemigatinib

Condition Hierarchy (Ancestors)

Intestinal Neoplasms; Gastrointestinal Neoplasms; Digestive System Neoplasms; Neoplasms by Site; Neoplasms; Digestive System Diseases; Gastrointestinal Diseases; Colonic Diseases; Intestinal Diseases; Rectal Diseases

Results Point of Contact

• Title: Tanios Bekaii-Saab M.D.
• Organization: Mayo Clinic

Bekaii-Saab.Tanios@mayo.edu

480-342-2000

Study Officials

• Kristen K Ciombor

  Academic and Community Cancer Research United

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 12, 2019
• First Posted: September 19, 2019
• Study Start: August 31, 2020
• Primary Completion: May 13, 2022
• Study Completion: December 1, 2025
• Last Updated: February 21, 2024
• Results First Posted: February 21, 2024

Record last verified: 2023-11

Locations

US (6)