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Bladder CancerPhase 2NCT03914794

A Study of Pemigatinib in Non-muscle Invasive Bladder Cancer Patients With Recurrent Low- or Intermediate-Risk Tumors

A Phase 2 study testing Pemigatinib in people with Bladder Cancer and NMIBC, run by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins at 6 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This phase II trial studies how well Pemigatinib (an orally administered inhibitor of fibroblast growth factor receptors 1, 2, and 3) works in non-muscle invasive bladder cancer (NMIBC) patients with recurrent tumors and a prior history of low- or intermediate-risk NMIBC tumors. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Pemigatinib
Could I receive a placebo?
No, everyone receives an active treatment
How many people
30 participants
Where
6 sitesUnited States
Study dates
2 Oct 2020to 30 Sep 2025
Phase
Phase 2
Are there results?
None posted in the registry

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

  • Prior histologically confirmed low- or intermediate-risk non-muscle invasive urothelial carcinoma of the bladder (NMIBC) defined according to the following characteristics:
  • Low Risk
  • Initial tumor with all of the following:
  • Solitary tumor
  • Ta tumor
  • Low-grade
Show all 29Show fewer
  • <3 cm
  • No CIS
  • Intermediate Risk
  • --- All tumors not defined in the two adjacent categories (between the category of low- and high-risk)
  • High Risk
  • T1 tumor
  • High-grade
  • CIS
  • Multiple and recurrent and large (>3 cm) Ta low-grade tumors (all conditions must be met for this point on Ta low-grade tumors)
  • Documented tumor recurrence as noted in standard of care follow up cystoscopy.
  • ECOG (WHO) performance status 0-2
  • Age ≥ 18 years old
  • Patients must have the following laboratory values:
  • White blood cell count (WBC) > 3.0 K/mm3
  • Absolute neutrophil count (ANC) ≥ 1.5 K/mm3
  • Platelets ≥ 100 K/mm3
  • Hemoglobin (Hgb) ≥ 9 g/dL
  • Serum total bilirubin: ≤ 1.5 x ULN
  • ALT and AST ≤ 3.0 x ULN
  • Serum calcium < ULN
  • Serum phosphate < ULN
  • Serum creatinine ≤ 1.5 x ULN or serum creatinine > 1.5 - 3 x ULN if calculated creatinine clearance (CrCl) is ≥ 30 mL/min using the modified Cockcroft-Gault equation
  • Patients who give a written informed consent obtained according to local guidelines

Who cannot join

  • Patients with concurrent upper urinary tract (i.e. ureter, renal pelvis) non-invasive urothelial carcinoma.
  • Patients with high grade urothelial carcinoma on their most recent urine cytology.
  • Patients with another active second malignancy other than non-melanoma skin cancers and biochemical relapsed prostate cancer. (Patients that have completed all necessary therapy and are considered to be at less than 30% risk of relapse are not considered to have an active second malignancy and are eligible for enrollment.)
  • 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.).
  • Patients who have had radiotherapy ≤ 4 weeks prior to starting study drug, or who have not recovered from radiotherapy toxicities
  • Patients who have undergone major surgery (e.g. intra-thoracic, intra-abdominal or intra-pelvic), open biopsy or significant traumatic injury ≤ 4 weeks prior to starting study drug, or patients who have had minor procedures (i.e. TURBT), percutaneous biopsies or placement of vascular access device ≤ 1 week prior to starting study drug, or who have not recovered from side effects of such procedure or injury

Trial locations and contacts

6 sites in 1 country.

6 sites in 1 country

United States6 sites

  • Midlantic Urology

    Bala-Cynwyd, Pennsylvania, 19004

  • Johns Hopkins School of Medicine - Sidney Kimmel Comprehensive Cancer Center

    Baltimore, Maryland, 21205

  • Keystone Urology

    Lancaster, Pennsylvania, 17604

  • Carolina Urologic Research Center

    Myrtle Beach, South Carolina, 29572

  • Associated Medical Professionals Urology

    Syracuse, New York, 13210

  • Sibley Memorial Hospital

    Washington D.C., District of Columbia, 20016

Other trials in United States

About this study

Official title: Phase 2 Window of Opportunity Study of Pemigatinib in Non-muscle Invasive Bladder Cancer Patients With Recurrent Low- or Intermediate-Risk Tumors

This phase II trial studies how well Pemigatinib (an orally administered inhibitor of fibroblast growth factor receptors 1, 2, and 3) works in non-muscle invasive bladder cancer (NMIBC) patients with recurrent tumors and a prior history of low- or intermediate-risk NMIBC tumors. Participants will receive pemigatinib for 4-6 weeks prior to standard of care transurethral resection of bladder tumor (TURBT).

Read the detailed description

It is a single-arm phase 2 window of opportunity study to assess the antineoplastic activity of pemigatinib in non-muscle invasive bladder cancer (NMIBC) patients with recurrent tumors and a prior history of low- or intermediate-risk NMIBC tumors. Enrolled patients will receive pemigatinib for 4-6 weeks prior to standard of care transurethral resection of bladder tumor (TURBT). The primary endpoint will be complete response rate as determined at TURBT. Secondary endpoints will include safety profile, associations between complete response rate and tumor mutation/fusion status (e.g. FGFR3 specifically, others will also be examined) and NMIBC risk group (low- vs. intermediate-risk), and assessment of pemigatinib post-treatment urothelial tissue concentrations. The study will be conducted at 4-5 high volume bladder cancer institutions within the US and managed through the Johns Hopkins Sidney Kimmel Comprehensive Cancer Center clinical research office.

Being tested
Pemigatinib
Keywords
Bladder Cancer, Non Muscle Invasive Bladder Cancer, Non-Muscle Invasive Bladder Cancer (NMIBC), NMIBC, Urothelial Carcinoma, Urothelial Cancer, Urinary Bladder Neoplasm, Bladder Neoplasm, Fibroblast inhibitors, Pemigatinib, FGFR inhibitor
Medical subject headings
Urinary Bladder Neoplasms, Non-Muscle Invasive Bladder Neoplasms, Carcinoma, Transitional Cell

What participants receive

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

  • PemigatinibDrug

    Also called INCB054828, FGFR inhibitor INCB054828

    Participants will take Pemigatinib once daily on days 1 through 28 of each cycle prior to standard of care TURBT.

Study groups in NCT03914794
Study groupRoleWhat they receive
Treatment: PemigatinibExperimentalPatients will receive pemigatinib for 4 to 6 weeks prior to standard of care transurethral resection of bladder tumor (TURBT).
Receives: Drug: Pemigatinib

What is being measured

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

  1. Complete response rate of pemigatinib therapy

    The number of participants with a complete response to pemigatinib therapy. Tumor complete response is defined as the complete absence of any stage bladder tumor on post-treatment Transurethral Resection of a Bladder Tumor (TURBT) and no evidence of recurrent urothelial carcinoma on post-treatment urine cytology.

    Measured: 6 weeks

Secondary outcomes (7)

  1. Characterize the safety profile of pemigatinib therapy

    Number of Common Terminology Criteria for Adverse Events (CTCAE) grade ≥ 3 toxicities

    Measured: 4 years

  2. Number of Participants with Complete Response and FGFR3 Mutational Status

    Measured: Up to 4 weeks

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

    Number of participants with complete response rate and baseline NMIBC risk group (low- vs. intermediate-risk)

    Measured: Up to 4 years

  4. Maximal concentration (Cmax, nmol/L) of pemigatinib in urothelial tissue at post-treatment TURBT

    Measured: Up to 4 weeks

  5. Relapse Free Survival (RFS) at 6 months

    Number of months from achieving a complete response at initial post-treatment TURBT until relapse.

    Measured: 6 months

  6. Relapse Free Survival (RFS) at 12 months

    Number of months from achieving a complete response at initial post-treatment TURBT until relapse.

    Measured: 12 months

  7. Relapse Free Survival (RFS) at 24 months

    Number of months from achieving a complete response at initial post-treatment TURBT until relapse.

    Measured: 24 months

Timeline

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

  1. First posted16 Apr 2019
  2. Study start2 Oct 2020
  3. Primary completion30 Sep 2025
  4. Study completion30 Sep 2025

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
30 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03914794
Sponsor's protocol ID
J18158
Other IDs
IRB00194271
Status last verified
Oct 2025
First submitted
11 Apr 2019
Last updated
3 Oct 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Pemigatinib in people with Bladder Cancer and NMIBC, run by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins 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 7 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: finished, as of the sponsor's last update on 3 Oct 2025. 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?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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?

Complete response rate of pemigatinib therapy (time frame: 6 weeks). 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 NCT03914794.

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 3 Oct 2025.

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