What side effects are associated with this type of intervention?

Common side effects of treatments for bladder cancer, particularly those involving FGFR inhibitors like Pemigatinib, include hyperphosphatemia, dry mouth, fatigue, alopecia, and gastrointestinal issues such as diarrhea and nausea. Ocular toxicities, including dry eye and retinal detachment, have also been reported.

What prior FDA approvals exist?

The FDA has approved the pan-FGFR inhibitor erdafitinib for the treatment of urothelial carcinoma, a type of bladder cancer. Erdafitinib targets FGFR1, FGFR2, and FGFR3, similar to Pemigatinib, which is currently being studied for non-muscle invasive bladder cancer (NMIBC) with recurrent low- or intermediate-risk tumors. Other FGFR-targeted drugs are also undergoing clinical trials, highlighting the potential of FGFR inhibition in bladder cancer treatment.

What other types of research exist?

Promising alternatives to Pemigatinib for bladder cancer treatment include Erdafitinib, another FGFR inhibitor recently approved for urothelial carcinoma. Additionally, immunotherapies such as Atezolizumab and Pembrolizumab have shown efficacy in advanced urothelial carcinoma. Enfortumab Vedotin, an antibody-drug conjugate targeting Nectin-4, is also a novel option showing promise in clinical trials.

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Inhibitor

Pemigatinib for Bladder Cancer

Phase 2

Recruiting

Led By Noah M Hahn, MD

Research Sponsored by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Age ≥ 18 years old

Prior histologically confirmed low- or intermediate-risk non-muscle invasive urothelial carcinoma of the bladder (NMIBC) defined according to specific characteristics

Must not have

Patients who have received the last administration of an anti-cancer therapy including chemotherapy, immunotherapy, and monoclonal antibodies ≤ 4 weeks prior to starting study drug, or who have not recovered from the side effects of such therapy

Patients who have received prior selective fibroblast growth factor receptor targeting agents (i.e. pemigatinib, dovitinib, BGJ398, AZD4547, JNJ-42756493, etc.)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 4 years

Awards & highlights

No Placebo-Only Group

All Individual Drugs Already Approved

Summary

This trial tests how well Pemigatinib, a pill that blocks proteins helping cancer grow, works in patients with bladder cancer that has come back after previous treatment. The drug aims to stop specific proteins needed for cancer growth.

Who is the study for?

This trial is for adults over 18 with recurrent non-muscle invasive bladder cancer that was previously low- or intermediate-risk. They must be able to perform daily activities with little to no assistance (ECOG status 0-2) and have certain lab values within specific ranges. People can't join if they've had recent radiotherapy, surgery, or other cancer treatments, have high-grade urothelial carcinoma in their latest urine test, another active cancer besides some exceptions, or previous treatment with similar drugs.

What is being tested?

The study tests Pemigatinib's effectiveness on patients with recurring bladder tumors who've had prior low- or intermediate-risk cancers. It's taken orally for 4-6 weeks before the standard tumor removal surgery (TURBT).

What are the potential side effects?

While not explicitly listed here, side effects of Pemigatinib may include diarrhea, dry mouth, changes in hair color, nail problems and fatigue based on its action as a fibroblast growth factor receptor inhibitor.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am 18 years old or older.

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My bladder cancer is confirmed to be at a low or intermediate risk and has not invaded the muscle.

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I can take care of myself and perform daily activities.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I received my last cancer treatment less than 4 weeks ago or am still experiencing side effects.

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I have been treated with specific drugs targeting fibroblast growth factor receptors.

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I have a non-invasive cancer in my kidney or ureter.

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I have another cancer besides non-melanoma skin cancer or prostate cancer that is not actively growing.

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My latest urine test shows high grade bladder cancer.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 4 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~4 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and 4 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Complete response rate of pemigatinib therapy

Secondary study objectives

Characterize the safety profile of pemigatinib therapy

Number of Participants with Non-Muscle Invasive Bladder Cancer (NMIBC) and Complete Response

Relapse Free Survival (RFS) at 12 months

+2 more

Side effects data

From 2022 Phase 2 trial • 147 Patients • NCT02924376

59%

Alopecia

56%

Hyperphosphataemia

54%

Diarrhoea

46%

Fatigue

43%

Stomatitis

43%

Constipation

42%

Nausea

42%

Dysgeusia

39%

Dry mouth

35%

Dry eye

34%

Arthralgia

32%

Vomiting

31%

Decreased appetite

28%

Dry skin

26%

Hypophosphataemia

25%

Back pain

24%

Pain in extremity

21%

Palmar-plantar erythrodysaesthesia syndrome

20%

Abdominal pain

19%

Headache

19%

Urinary tract infection

19%

Weight decreased

18%

Dizziness

18%

Epistaxis

15%

Oedema peripheral

15%

Hypercalcaemia

15%

Anaemia

15%

Dehydration

14%

Myalgia

14%

Asthenia

13%

Dyspepsia

12%

Gastrooesophageal reflux disease

12%

Insomnia

12%

Nasal dryness

12%

Pruritus

12%

Onychomadesis

11%

Blood alkaline phosphatase increased

11%

Rash

11%

Nail discolouration

11%

Alanine aminotransferase increased

10%

Muscle spasms

10%

Pyrexia

10%

Abdominal pain upper

10%

Nail dystrophy

10%

Oropharyngeal pain

10%

Trichiasis

Dyspnoea

Vitamin D deficiency

Onycholysis

Cough

Abdominal distension

Hyperbilirubinaemia

Hypertension

Hypokalaemia

Paronychia

Onychoclasis

Blood creatinine increased

Aspartate aminotransferase increased

Growth of eyelashes

Fall

Punctate keratitis

Erythema

Nasal congestion

Platelet count decreased

Conjunctivitis

Lacrimation increased

Nail disorder

Nasopharyngitis

Neuropathy peripheral

Skin exfoliation

Taste disorder

Upper respiratory tract infection

Cataract

Eye pain

Chills

Blood bilirubin increased

Depression

Hyponatraemia

Ocular hyperaemia

Influenza like illness

Dysphagia

Vitreous floaters

Cystitis

Cholangitis

Flank pain

Hypotension

Acute kidney injury

Muscular weakness

Neck pain

Oral candidiasis

Hyperuricaemia

Pain

Weight increased

Ascites

Skin fissures

Lymphocyte count decreased

Keratitis

Activated partial thromboplastin time prolonged

Breast pain

Dyspnoea exertional

Tinnitus

Blood parathyroid hormone decreased

Pollakiuria

Bronchitis

Cholangitis infective

Non-cardiac chest pain

Decubitus ulcer

Sepsis

Blood 1,25-dihydroxycholecalciferol increased

Electrocardiogram QT prolonged

Hypoalbuminaemia

Failure to thrive

Bacteraemia

Hypocalcaemia

Palpitations

Pharyngitis

Rash maculo-papular

Tachycardia

Trichomegaly

Dysuria

Hyperglycaemia

Dysphonia

Device occlusion

Small intestinal obstruction

Blood 1,25-dihydroxycholecalciferol decreased

Chronic kidney disease

Biliary obstruction

Pleural effusion

Pneumonia

Hypercholesterolaemia

Prostate cancer

Skin infection

Retinal detachment

Septic shock

Thrombosis

Biliary tract infection

Complication associated with device

Enterobacter bacteraemia

Intestinal obstruction

Hyperkalaemia

Jaundice

Kidney infection

Oesophageal varices haemorrhage

Micturition urgency

Seizure

Pseudomonal bacteraemia

Varices oesophageal

Oral herpes

Clostridium difficile infection

Device leakage

Gynaecomastia

Somnolence

Catheter site infection

Gastrointestinal haemorrhage

Haematemesis

Hydronephrosis

Optic ischaemic neuropathy

Pneumonitis

Transaminases increased

C-reactive protein increased

Cancer pain

Candida infection

Confusional state

Herpes zoster

Musculoskeletal pain

Psoriasis

Blood chloride decreased

Cerebrovascular accident

Malignant biliary obstruction

Melaena

Paraplegia

Pneumonia aspiration

Pneumonia pneumococcal

Syncope

Haemorrhoids

Sinus pain

Urinary tract pain

100%

80%

60%

40%

20%

Study treatment Arm

Cohort A: FGFR2 Rearrangements or Fusions

Cohort B: FGF/FGFR Alterations Other Than FGFR2 Rearrangements or Fusions

Cohort C: Negative for FGF/FGFR Alterations

Other

Total

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Treatment: PemigatinibExperimental Treatment1 Intervention

Patients will receive pemigatinib for 4 to 6 weeks prior to standard of care transurethral resection of bladder tumor (TURBT).

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Pemigatinib

FDA approved

0 / 8Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Bladder cancer treatments often target specific pathways to inhibit tumor growth and progression. Pemigatinib, an FGFR inhibitor, works by blocking fibroblast growth factor receptors 1, 2, and 3, which are often overexpressed in bladder cancer cells, thereby reducing cell proliferation and survival. This targeted approach is crucial for patients with FGFR alterations, offering a more personalized and potentially effective treatment. Other common treatments include chemotherapy, which uses cytotoxic drugs to kill rapidly dividing cancer cells, and immunotherapy, which enhances the body's immune response to target and destroy cancer cells. Understanding these mechanisms helps patients and doctors choose the most appropriate and effective treatment strategy based on the cancer's specific characteristics.

[The role of immunotherapy in the modern treatment of urothelial carcinoma].Fibroblast growth factor receptor 3 is overexpressed in urinary tract carcinomas and modulates the neoplastic cell growth.