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

NCT03914794 | Completed Phase 2 DMC FDA Drug

• 2 other identifiers: J18158IRB00194271
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 6

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

Conditions

Bladder Cancer; NMIBC; Non-Muscle Invasive Bladder Cancer; Urothelial Carcinoma Recurrent

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

Outcome Measures

Primary Outcomes (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.

  6 weeks

Secondary Outcomes (7)

• Characterize the safety profile of pemigatinib therapy

  4 years

• Number of Participants with Complete Response and FGFR3 Mutational Status

  Up to 4 weeks

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

  Up to 4 years

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

  Up to 4 weeks

• Relapse Free Survival (RFS) at 6 months

  6 months

Study Arms (1)

Treatment: Pemigatinib

EXPERIMENTAL

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

Drug: Pemigatinib

Interventions

Pemigatinib DRUG

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

Also known as: INCB054828, FGFR inhibitor INCB054828

Treatment: Pemigatinib

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• \<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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins: lead
• Incyte Corporation: collaborator

Study Sites (6)

Sibley Memorial Hospital

Washington D.C., District of Columbia, 20016, United States

Location

Johns Hopkins School of Medicine - Sidney Kimmel Comprehensive Cancer Center

Baltimore, Maryland, 21205, United States

Location

Associated Medical Professionals Urology

Syracuse, New York, 13210, United States

Location

Midlantic Urology

Bala-Cynwyd, Pennsylvania, 19004, United States

Location

Keystone Urology

Lancaster, Pennsylvania, 17604, United States

Location

Carolina Urologic Research Center

Myrtle Beach, South Carolina, 29572, United States

Location

MeSH Terms

Conditions

Urinary Bladder Neoplasms; Non-Muscle Invasive Bladder Neoplasms; Carcinoma, Transitional Cell

Interventions

pemigatinib

Condition Hierarchy (Ancestors)

Urologic Neoplasms; Urogenital Neoplasms; Neoplasms by Site; Neoplasms; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Urinary Bladder Diseases; Urologic Diseases; Male Urogenital Diseases; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type

Study Officials

• Noah M Hahn, MD

  Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 11, 2019
• First Posted: April 16, 2019
• Study Start: October 2, 2020
• Primary Completion: September 30, 2025
• Study Completion: September 30, 2025
• Last Updated: October 3, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (6)