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NCT04096417

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

Status unknown Phase 2 Results posted Last updated 21 February 2024
What this trial tests

Phase 2 trial testing Pemigatinib in FGFR1 Gene Amplification in 14 participants. Status unknown.

Timeline
31 August 2020
Primary endpoint
13 May 2022
1 December 2025

Quick facts

Lead sponsorAcademic and Community Cancer Research United
PhasePhase 2
StatusStatus unknown
Study typeINTERVENTIONAL
Allocationna
Designsingle group
Maskingnone
Primary purposetreatment
Enrollment14
Start date31 August 2020
Primary completion13 May 2022
Estimated completion1 December 2025
Sites6 locations across United States

Drugs / interventions tested

Conditions studied

Sponsor

Academic and Community Cancer Research United — full company profile →

Who can join

18 and older, any sex, with FGFR1 Gene Amplification or FGFR1 Gene Mutation. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Overall Response Rate (ORR) Primary · 4.4 Months

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.

GroupValue95% CI
Treatment (Pemigatinib)0.00.0 – 0.232
Clinical Benefit Rate Secondary · 4.4 Months

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.

GroupValue95% CI
Treatment (Pemigatinib)0.00.0 – 0.232
Progression-free Survival (PFS) Secondary · 4.4 Months

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.

GroupValue95% CI
Treatment (Pemigatinib)9.17.9 – NA
Overall Survival (OS) Secondary · 29.4 Months

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.

GroupValue95% CI
Treatment (Pemigatinib)7.93.4 – NA
Quality of Life (QOL) as Measured by the LASA [Item 1: Overall QOL] Secondary · 9 Months

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.

GroupValue95% CI
Treatment (Pemigatinib)-0.1± 1.22
Incidence of Adverse Events Secondary · 5.4 Months

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.

GroupValue95% CI
Treatment (Pemigatinib)14

Adverse events — posted to ClinicalTrials.gov

Time frame: Adverse events were followed for 5.4 months and mortality was followed for 29.4 months. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Treatment (Pemigatinib)
Serious: 6/14 (43%)
Deaths: 11/14

Serious adverse events (9 terms)

ReactionSystemTreatment (Pemigatinib)
NauseaGastrointestinal disorders
Small intestinal obstructionGastrointestinal disorders
VomitingGastrointestinal disorders
FatigueGeneral disorders
PainGeneral disorders
Lung infectionInfections and infestations
Neoplasms benign, mal, uncpec - Oth specNeoplasms benign, malignant and unspecified (incl cysts and polyps)
DizzinessNervous system disorders
HydrocephalusNervous system disorders
Other adverse events (40 terms — click to expand)

ReactionSystemTreatment (Pemigatinib)
AnemiaBlood and lymphatic system disorders
HyperphosphatemiaMetabolism and nutrition disorders
Alkaline phosphatase increasedInvestigations
FatigueGeneral disorders
Aspartate aminotransferase increasedInvestigations
DiarrheaGastrointestinal disorders
Alanine aminotransferase increasedInvestigations
Blood bilirubin increasedInvestigations
AnorexiaMetabolism and nutrition disorders
Abdominal painGastrointestinal disorders
NauseaGastrointestinal disorders
Platelet count decreasedInvestigations
Blurred visionEye disorders
Mucositis oralGastrointestinal disorders
VomitingGastrointestinal disorders
DehydrationMetabolism and nutrition disorders
HypercalcemiaMetabolism and nutrition disorders
Dry eyeEye disorders
Eye painEye disorders
ConstipationGastrointestinal disorders
Dry mouthGastrointestinal disorders
DyspepsiaGastrointestinal disorders
Gastrointestinal fistulaGastrointestinal disorders
Oral painGastrointestinal disorders
ChillsGeneral disorders
Gait disturbanceGeneral disorders
Mucosal infectionInfections and infestations
ThrushInfections and infestations
Cholesterol highInvestigations
Creatinine increasedInvestigations
Lymphocyte count decreasedInvestigations
HyperuricemiaMetabolism and nutrition disorders
Muscle weakness lower limbMusculoskeletal and connective tissue disorders
DizzinessNervous system disorders
DysgeusiaNervous system disorders
HeadacheNervous system disorders
InsomniaPsychiatric disorders
AlopeciaSkin and subcutaneous tissue disorders
Palmar-plantar erythrodysesthesia syndrmSkin and subcutaneous tissue disorders
Skin and subcut tissue disord - Oth specSkin and subcutaneous tissue disorders

Most-reported serious reactions: Nausea, Small intestinal obstruction, Vomiting, Fatigue, Pain, Lung infection, Neoplasms benign, mal, uncpec - Oth spec, Dizziness.

Data from ClinicalTrials.gov NCT04096417 adverse events section.

Sponsor's own description

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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Angiogenic signaling pathways and anti-angiogenic therapy for cancer.
    Liu ZL, Chen HH, Zheng LL, Sun LP, et al · · 2023 · cited 808× · PMID 37169756 · DOI 10.1038/s41392-023-01460-1
  2. FGFR-TKI resistance in cancer: current status and perspectives.
    Yue S, Li Y, Chen X, Wang J, et al · · 2021 · cited 98× · PMID 33568192 · DOI 10.1186/s13045-021-01040-2
  3. FGFR Fusions in Cancer: From Diagnostic Approaches to Therapeutic Intervention.
    De Luca A, Esposito Abate R, Rachiglio AM, Maiello MR, et al · · 2020 · cited 87× · PMID 32962091 · DOI 10.3390/ijms21186856
  4. FGFR4: A promising therapeutic target for breast cancer and other solid tumors.
    Levine KM, Ding K, Chen L, Oesterreich S. · · 2020 · cited 68× · PMID 32492514 · DOI 10.1016/j.pharmthera.2020.107590
  5. Fibroblast growth factor receptor fusions in cancer: opportunities and challenges.
    Chen L, Zhang Y, Yin L, Cai B, et al · · 2021 · cited 50× · PMID 34732230 · DOI 10.1186/s13046-021-02156-6
  6. Targeting FGFR2 Positive Gastroesophageal Cancer: Current and Clinical Developments.
    Gordon A, Johnston E, Lau DK, Starling N. · · 2022 · cited 25× · PMID 36238135 · DOI 10.2147/ott.s282718
  7. Targeting FGFRs by pemigatinib induces G1 phase cell cycle arrest, cellular stress and upregulation of tumor suppressor microRNAs.
    Pace A, Scirocchi F, Napoletano C, Zizzari IG, et al · · 2023 · cited 12× · PMID 37715207 · DOI 10.1186/s12967-023-04450-7
  8. Co-Clinical Trials: An Innovative Drug Development Platform for Cholangiocarcinoma.
    Balasubramanian B, Venkatraman S, Myint KZ, Janvilisri T, et al · · 2021 · cited 7× · PMID 33440754 · DOI 10.3390/ph14010051

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