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FGFR1 Gene AmplificationPhase 2NCT04096417

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

A Phase 2 study testing Pemigatinib and Quality-of-Life Assessment in people with FGFR1 Gene Amplification and FGFR1 Gene Mutation, run by Academic and Community Cancer Research United at 6 sites in United States.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial 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). Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Pemigatinib and Quality-of-Life Assessment
Could I receive a placebo?
No, everyone receives an active treatment
How many people
14 participants
Where
6 sitesUnited States
Study dates
31 Aug 2020to 1 Dec 2025
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
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  • 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)
  • Platelet count >= 100,000/mm^3 (obtained =< 28 days prior to registration)
  • Hemoglobin >= 9.0 g/dL (obtained =< 28 days prior to registration)
  • Total bilirubin =< 1.5x upper limit of normal (ULN), or =< 2.5x ULN if patient has Gilbert syndrome or disease involving the liver (obtained =< 28 days prior to registration)
  • Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) =< 2.5x ULN (or =< 5x ULN in presence of suspected liver metastases) (obtained =< 28 days prior to registration)
  • Serum phosphate < institutional ULN (obtained =< 28 days prior to registration)
  • Serum calcium within institutional normal range, or serum albumin-corrected calcium within institutional normal range (if serum albumin is outside of the institutional normal range) (obtained =< 28 days prior to registration)
  • Potassium levels > institutional lower limit of normal (supplementation can be used to correct potassium level during screening) (obtained =< 28 days prior to registration)
  • Serum creatinine =< 1.5x ULN, or calculated creatinine clearance > 30 mL/min using the Cockcroft-Gault formula or 24-hours urine collection analysis (obtained =< 28 days prior to registration)
  • Corrected QT interval (QTc) by Fridericia?s method (QTcF) assessed by electrocardiogram (ECG) completed =< 28 days prior to registration, and resulted as:
  • QTcF =< 450 msec in men, or
  • QTcF =< 470 msec in women
  • Negative serum pregnancy test completed =< 7 days prior to registration, for women of childbearing potential only
  • Willing to provide tissue and blood samples for correlative research purposes
  • Willing to allow transfer of tissue and blood samples, clinical information, and outcome data collected from this trial for future research

Who cannot join

  • 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
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  • 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
  • Failure to adequately recover (i.e. to =< grade 1 [according to Common Terminology Criteria for Adverse Events (CTCAE) version (v.)5] or to pre-treatment baseline) from adverse events (AEs) deemed by the investigator as clinically significant and attributed to prior therapy. Exception: alopecia
  • Current use of prohibited medication
  • Use of any potent CYP3A4 inhibitors or inducers or moderate CYP3A4 inducers =< 14 days or 5 half-lives (whichever is shorter) prior to registration. Note: topical ketoconazole will be allowed
  • History of hypovitaminosis D requiring supraphysiologic doses to replenish the deficiency. Note: patients receiving vitamin D food supplements are allowed
  • History and/or current evidence of ectopic mineralization/calcification, including but not limited to soft tissue, kidneys, intestine, myocardia, or lung; with the exception of calcified lymph nodes and asymptomatic arterial or cartilage/tendon calcification
  • Unable or unwilling to swallow pemigatinib and keep a medication diary, or significant gastrointestinal disorder(s) that could interfere with absorption, metabolism or excretion of pemigatinib per the discretion of the investigator
  • Any of the following because this study involves an investigational agent whose genotoxic, mutagenic and teratogenic effects on the developing fetus and newborn are unknown:
  • Pregnant women
  • Nursing women
  • Women of childbearing potential or men able to father children who have a female partner of childbearing potential, who are unwilling to employ acceptable contraception
  • Known history of human immunodeficiency (HIV) infection or positivity on immunoassay confirmed per local standards
  • Note: HIV test is not required for screening, but patients with a known history of HIV infection will be excluded
  • Evidence of active hepatitis B virus (HBV) or hepatitis C virus (HCV) infection
  • Other known active malignancy =< 5 years prior to registration
  • EXCEPTIONS: Non-melanotic skin cancer or carcinoma in situ of the cervix, provided there is no known active disease and no additional therapy for the condition is ongoing or required during the trial period
  • NOTE: anti-estrogen/androgen therapy or bisphosphonates allowed
  • Co-morbid systemic illness, other severe concurrent disease, or psychiatric illness/social situation which, in the judgment of the investigator, would make the patient inappropriate for entry into this study, limit compliance with study requirements, or interfere significantly with the proper assessment of safety and toxicity of the prescribed regimen

Trial locations and contacts

6 sites in 1 country.

6 sites in 1 country

United States6 sites

  • Emory University Hospital/Winship Cancer Institute

    Atlanta, Georgia, 30322

  • Duke University Medical Center

    Durham, North Carolina, 27710

  • Vanderbilt University/Ingram Cancer Center

    Nashville, Tennessee, 37232

  • Mayo Clinic in Rochester

    Rochester, Minnesota, 55905

  • Mayo Clinic in Arizona

    Scottsdale, Arizona, 85259

  • Aurora Cancer Care-Milwaukee West

    Wauwatosa, Wisconsin, 53226

Other trials in United States

About this study

Official title: A Phase II, Multicenter, Single-Arm Study of Pemigatinib in Patients With Metastatic or Unresectable Colorectal Cancer Harboring FGFR Alterations

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.

Read the detailed description

PRIMARY OBJECTIVE:

  1. To assess overall response rate (ORR) of pemigatinib in patients with metastatic or unresectable colorectal cancer harboring activating FGFR alterations.

SECONDARY OBJECTIVES:

  1. To assess the clinical benefit rate (complete response + partial response + stable disease) with pemigatinib.

II. To assess progression free survival (PFS) and overall survival (OS) with pemigatinib.

III. Assess changes in patient quality of life (QOL) as measured by the linear analogue self-assessment (LASA) questionnaire.

IV. Assess the frequency and severity of adverse events.

CORRELATIVE RESEARCH OBJECTIVES:

  1. To assess plasma pharmacodynamic biomarkers of response and resistance to therapy.

II. To explore any correlation between tissue and blood based biomarkers and clinical outcomes.

OUTLINE:

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

After completion of study treatment, patients are followed up every 3 months for 3 years after registration.

Being tested
Pemigatinib, Quality-of-Life Assessment
Medical subject headings
Colorectal Neoplasms

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • PemigatinibDrug

    Also called INCB054828

    Given PO

  • Quality-of-Life AssessmentOther

    Also called Quality of Life Assessment

    Ancillary studies

Study groups in NCT04096417
Study groupRoleWhat they receive
Treatment (pemigatinib)ExperimentalPatients 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.
Receives: Drug: Pemigatinib; Other: Quality-of-Life Assessment

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

    Measured: 4.4 Months

Secondary outcomes (5)

  1. Clinical Benefit Rate

    Clinical Benefit Rate is defined as the number of patients that experience a complete or partial response, or have stable disease, divided by the number of evaluable patients. Analysis of this endpoint will mirror that of the primary objective.

    Measured: 4.4 Months

  2. Progression-free Survival (PFS)

    Progression-free survival (PFS) is defined as the time from study entry to the first of either disease progression or death from any cause, where disease progression is determined based on RECIST 1.1 criteria. PFS will be estimated using the Kaplan-Meier method. The median PFS and corresponding 95% confidence interval will be reported. Patients who do not experience disease progression or death while on protocol will be censored at the last disease assessment date.

    Measured: 4.4 Months

  3. Overall Survival (OS)

    Overall survival (OS) is defined as the time from study entry to death from any cause. Will be estimated using the Kaplan-Meier method. The median OS and corresponding 95% confidence interval will be reported.

    Measured: 29.4 Months

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

    Quality of Life (QOL) was measured using item 1: Overall QOL of the Linear Analogue Self-Assessment (LASA) Questionnaire on a 0-10 scale, with 0=as bad as it can be and 10=as good as it can be. The QOL scores was converted to a 100-point scale, with 0=Low QOL and 100=Best QOL. Change from baseline to Week 36 will be calculated by subtracting the baseline scores from the scores at Week 36. Negative change indicates the QOL decrease and positive change indicates the QOL improvement.

    Measured: 9 Months

  5. Incidence of Adverse Events

    Adverse events will be summarized by frequency and severity using Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 in the adverse event section of this report. The number of patients evaluated for adverse events is reported below.

    Measured: 5.4 Months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted19 Sep 2019
  2. Study start31 Aug 2020
  3. Primary completion13 May 2022
  4. Study completion1 Dec 2025 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
14 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04096417
Sponsor's protocol ID
ACCRU-GI-1701
Other IDs
NCI-2019-05877, ACCRU-GI-1701, P30CA015083
Status last verified
Nov 2023
First submitted
12 Sep 2019
Last updated
21 Feb 2024
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • 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.

    PubMed 40515475

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Pemigatinib and Quality-of-Life Assessment in people with FGFR1 Gene Amplification and FGFR1 Gene Mutation, run by Academic and Community Cancer Research United at 6 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 29 inclusion and 32 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: status not confirmed, as of the sponsor's last update on 21 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 6 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 21 Feb 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Overall Response Rate (ORR) (time frame: 4.4 Months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04096417, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Feb 2024.

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