Brief Title
A Study of Pemigatinib in Non-muscle Invasive Bladder Cancer Patients With Recurrent Low- or Intermediate-Risk Tumors
Official Title
Phase 2 Window of Opportunity Study of Pemigatinib in Non-muscle Invasive Bladder Cancer Patients With Recurrent Low- or Intermediate-Risk Tumors
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).
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.
Study Phase
Phase 2
Study Type
Interventional
Primary Outcome
Complete response rate of pemigatinib therapy
Secondary Outcome
Characterize the safety profile of pemigatinib therapy
Condition
Bladder Cancer
Intervention
Pemigatinib
Study Arms / Comparison Groups
Treatment: Pemigatinib
Description: Patients will receive pemigatinib for 4 to 6 weeks prior to standard of care transurethral resection of bladder tumor (TURBT).
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
43
Start Date
October 2, 2020
Completion Date
May 2024
Primary Completion Date
May 2023
Eligibility 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
Gender
All
Ages
18 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Noah M Hahn, MD, 410-502-4910, [email protected]
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT03914794
Organization ID
J18158
Secondary IDs
IRB00194271
Responsible Party
Sponsor
Study Sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Collaborators
Incyte Corporation
Study Sponsor
Noah M Hahn, MD, Principal Investigator, Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Verification Date
October 2022