Patient case: 64-year-old male with bladder cancer; genetic feature: FGFR3 (S249C)

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

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).


Eligibility: 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

Relevance to patient case: possibly relevant