Trial Outcomes & Findings for Pemigatinib for the Treatment of Metastatic or Unresectable Colorectal Cancer Harboring FGFR Alterations (NCT NCT04096417)

Last Updated: 2024-02-21

Results Overview

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.

Recruitment status

ACTIVE_NOT_RECRUITING

Study phase

PHASE2

Target enrollment

14 participants

Primary outcome timeframe

4.4 Months

Results posted on

2024-02-21

Participant Flow

Participant milestones

Participant milestones
Measure	Treatment (Pemigatinib) 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.
Overall Study STARTED	14
Overall Study COMPLETED	14
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment (Pemigatinib) n=14 Participants 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.
Age, Continuous	60.5 years n=5 Participants
Sex: Female, Male Female	4 Participants n=5 Participants
Sex: Female, Male Male	10 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	12 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	13 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants

PRIMARY outcome

Timeframe: 4.4 Months

Outcome measures

Outcome measures
Measure	Treatment (Pemigatinib) n=14 Participants 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.
Overall Response Rate (ORR)	0.0 proportion of participants Interval 0.0 to 0.232

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Treatment (Pemigatinib) n=14 Participants 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.
Clinical Benefit Rate	0.0 proportion of participants Interval 0.0 to 0.232

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Treatment (Pemigatinib) n=14 Participants 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.
Progression-free Survival (PFS)	9.1 Weeks Interval 7.9 to Not enough events occurred to calculate the upper limit

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Treatment (Pemigatinib) n=14 Participants 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.
Overall Survival (OS)	7.9 Months Interval 3.4 to Not enough event occurred to calculate the upper limit

SECONDARY outcome

Timeframe: 9 Months

Population: All patients that completed and submitted the baseline and week 36 Linear Analogue Self-Assessment.

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.

Outcome measures

Outcome measures
Measure	Treatment (Pemigatinib) n=11 Participants 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.
Quality of Life (QOL) as Measured by the LASA [Item 1: Overall QOL]	-0.1 score on a scale Standard Deviation 1.22

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Treatment (Pemigatinib) n=14 Participants 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.
Incidence of Adverse Events	14 Participants

Adverse Events

Treatment (Pemigatinib)

Serious events: 6 serious events

Other events: 13 other events

Deaths: 11 deaths

Serious adverse events

Serious adverse events
Measure	Treatment (Pemigatinib) n=14 participants at risk 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.
Gastrointestinal disorders Nausea	7.1% 1/14 • Number of events 1 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Gastrointestinal disorders Small intestinal obstruction	7.1% 1/14 • Number of events 1 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Gastrointestinal disorders Vomiting	7.1% 1/14 • Number of events 1 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
General disorders Fatigue	7.1% 1/14 • Number of events 1 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
General disorders Pain	7.1% 1/14 • Number of events 1 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Infections and infestations Lung infection	7.1% 1/14 • Number of events 1 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Neoplasms benign, mal, uncpec - Oth spec	7.1% 1/14 • Number of events 1 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Nervous system disorders Dizziness	7.1% 1/14 • Number of events 1 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Nervous system disorders Hydrocephalus	7.1% 1/14 • Number of events 1 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months

Other adverse events

Other adverse events
Measure	Treatment (Pemigatinib) n=14 participants at risk 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.
Blood and lymphatic system disorders Anemia	71.4% 10/14 • Number of events 22 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Eye disorders Blurred vision	14.3% 2/14 • Number of events 2 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Eye disorders Dry eye	7.1% 1/14 • Number of events 1 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Eye disorders Eye pain	7.1% 1/14 • Number of events 2 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Gastrointestinal disorders Abdominal pain	21.4% 3/14 • Number of events 4 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Gastrointestinal disorders Constipation	7.1% 1/14 • Number of events 1 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Gastrointestinal disorders Diarrhea	42.9% 6/14 • Number of events 16 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Gastrointestinal disorders Dry mouth	7.1% 1/14 • Number of events 1 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Gastrointestinal disorders Dyspepsia	7.1% 1/14 • Number of events 1 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Gastrointestinal disorders Gastrointestinal fistula	7.1% 1/14 • Number of events 1 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Gastrointestinal disorders Mucositis oral	14.3% 2/14 • Number of events 3 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Gastrointestinal disorders Nausea	21.4% 3/14 • Number of events 6 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Gastrointestinal disorders Oral pain	7.1% 1/14 • Number of events 1 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Gastrointestinal disorders Vomiting	14.3% 2/14 • Number of events 3 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
General disorders Chills	7.1% 1/14 • Number of events 1 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
General disorders Fatigue	57.1% 8/14 • Number of events 21 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
General disorders Gait disturbance	7.1% 1/14 • Number of events 1 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Infections and infestations Mucosal infection	7.1% 1/14 • Number of events 1 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Infections and infestations Thrush	7.1% 1/14 • Number of events 1 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Investigations Alanine aminotransferase increased	35.7% 5/14 • Number of events 9 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Investigations Alkaline phosphatase increased	64.3% 9/14 • Number of events 17 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Investigations Aspartate aminotransferase increased	57.1% 8/14 • Number of events 15 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Investigations Blood bilirubin increased	35.7% 5/14 • Number of events 7 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Investigations Cholesterol high	7.1% 1/14 • Number of events 1 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Investigations Creatinine increased	7.1% 1/14 • Number of events 2 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Investigations Lymphocyte count decreased	7.1% 1/14 • Number of events 1 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Investigations Platelet count decreased	21.4% 3/14 • Number of events 4 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Metabolism and nutrition disorders Anorexia	35.7% 5/14 • Number of events 9 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Metabolism and nutrition disorders Dehydration	14.3% 2/14 • Number of events 2 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Metabolism and nutrition disorders Hypercalcemia	14.3% 2/14 • Number of events 2 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Metabolism and nutrition disorders Hyperphosphatemia	71.4% 10/14 • Number of events 19 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Metabolism and nutrition disorders Hyperuricemia	7.1% 1/14 • Number of events 1 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Musculoskeletal and connective tissue disorders Muscle weakness lower limb	7.1% 1/14 • Number of events 1 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Nervous system disorders Dizziness	7.1% 1/14 • Number of events 1 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Nervous system disorders Dysgeusia	7.1% 1/14 • Number of events 2 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Nervous system disorders Headache	7.1% 1/14 • Number of events 2 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Psychiatric disorders Insomnia	7.1% 1/14 • Number of events 1 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Skin and subcutaneous tissue disorders Alopecia	7.1% 1/14 • Number of events 1 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Skin and subcutaneous tissue disorders Palmar-plantar erythrodysesthesia syndrm	7.1% 1/14 • Number of events 1 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months
Skin and subcutaneous tissue disorders Skin and subcut tissue disord - Oth spec	7.1% 1/14 • Number of events 2 • Adverse events were followed for 5.4 months and mortality was followed for 29.4 months

Additional Information

Tanios Bekaii-Saab M.D.

Mayo Clinic

Phone: 480-342-2000

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place