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

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ClinicalTrials.gov Identifier: NCT03914794
Recruitment Status : Not yet recruiting
First Posted : April 15, 2019
Last Update Posted : September 17, 2019
Sponsor:
Collaborator:
Incyte Corporation
Information provided by (Responsible Party):
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

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).

Condition or disease Intervention/treatment Phase
Bladder Cancer NMIBC Non-Muscle Invasive Bladder Cancer Urothelial Carcinoma Recurrent Drug: Pemigatinib Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 43 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase 2 Window of Opportunity Study of Pemigatinib in Non-muscle Invasive Bladder Cancer Patients With Recurrent Low- or Intermediate-Risk Tumors
Estimated Study Start Date : October 2019
Estimated Primary Completion Date : May 2022
Estimated Study Completion Date : May 2023

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Bladder Cancer

Arm Intervention/treatment
Experimental: Treatment: Pemigatinib
Patients will receive pemigatinib for 4 to 6 weeks prior to standard of care transurethral resection of bladder tumor (TURBT).
Drug: Pemigatinib
Participants will take Pemigatinib once daily on days 1 through 28 of each cycle prior to standard of care TURBT.
Other Names:
  • INCB054828
  • FGFR inhibitor INCB054828




Primary Outcome Measures :
  1. Complete response rate of pemigatinib therapy [ Time Frame: 6 weeks ]
    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.


Secondary Outcome Measures :
  1. Characterize the safety profile of pemigatinib therapy [ Time Frame: 4 years ]
    Number of Common Terminology Criteria for Adverse Events (CTCAE) grade ≥ 3 toxicities

  2. Number of Participants with Complete Response and FGFR3 Mutational Status [ Time Frame: Up to 4 weeks ]
  3. Number of Participants with Non-Muscle Invasive Bladder Cancer (NMIBC) and Complete Response [ Time Frame: Up to 4 years ]
    Number of participants with complete response rate and baseline NMIBC risk group (low- vs. intermediate-risk)

  4. Maximal concentration (Cmax, nmol/L) of pemigatinib in urothelial tissue at post-treatment TURBT [ Time Frame: Up to 4 weeks ]
  5. Relapse Free Survival (RFS) at 6 months [ Time Frame: 6 months ]
    Number of months from achieving a complete response at initial post-treatment TURBT until relapse.

  6. Relapse Free Survival (RFS) at 12 months [ Time Frame: 12 months ]
    Number of months from achieving a complete response at initial post-treatment TURBT until relapse.

  7. Relapse Free Survival (RFS) at 24 months [ Time Frame: 24 months ]
    Number of months from achieving a complete response at initial post-treatment TURBT until relapse.



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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)
  • 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

Exclusion Criteria:

  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03914794


Contacts
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Contact: Abby Asiama 410-955-1168 aasiama1@jhmi.edu
Contact: Rana Sullivan 410-614-6337 tomalra@jhmi.edu

Locations
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United States, Maryland
Johns Hopkins School of Medicine - Sidney Kimmel Comprehensive Cancer Center Not yet recruiting
Baltimore, Maryland, United States, 21205
Sponsors and Collaborators
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Incyte Corporation
Investigators
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Principal Investigator: Noah M Hahn, MD Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

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Responsible Party: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
ClinicalTrials.gov Identifier: NCT03914794     History of Changes
Other Study ID Numbers: J18158
IRB00194271 ( Other Identifier: JHM IRB )
First Posted: April 15, 2019    Key Record Dates
Last Update Posted: September 17, 2019
Last Verified: September 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins:
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
Additional relevant MeSH terms:
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Urinary Bladder Neoplasms
Carcinoma, Transitional Cell
Carcinoma
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Neoplasms
Urologic Neoplasms
Urogenital Neoplasms
Neoplasms by Site
Urinary Bladder Diseases
Urologic Diseases