SeqUential GeMcitabine and MITomycin Treatment for Favorable High-Risk Upper Urinary Tract Urothelial Carcinoma

NCT06822010 | Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: 082411Pro2024002505
• Study Type: interventional
• Target: 28
• Locations: 1 country
• Sites: 1

Brief Summary

This Phase II clinical trial, titled "A Phase II Trial of Sequential Gemcitabine and Mitomycin Treatment for Favorable High-Risk Upper Urinary Tract Urothelial Carcinoma (SUMMIT)," aims to evaluate the safety and effectiveness of a combination chemotherapy treatment for patients with favorable papillary high-grade upper tract urothelial carcinoma (UTUC). The study focuses on sequential administration of two drugs, gemcitabine and Jelmyto (a gel-based form of mitomycin), to potentially preserve kidney function and avoid nephroureterectomy (kidney removal), which is the current standard of care for participants with non-endoscopically resectable tumors.

Conditions

Urinary Bladder Cancer; Upper Tract Urothelial Cancer; Urothelial Cancer

Keywords

upper tract urothelial cancer; urothelial cancer; gemcitabine; jelmyto; mitomycin

Outcome Measures

Primary Outcomes (1)

• Complete Response (CR) Rate

  The proportion of participants who achieve a complete response, defined as the absence of detectable high-grade disease via ureteroscopy, imaging (CTU/MRU), and urine cytology following the completion of the initial Gem/Jel treatment regimen. A complete response indicates no visible tumor during endoscopic evaluation and no evidence of cancer cells in collected samples

  At the end of the initial 6-week induction phase (up to 12 weeks if re-induction is necessary)

Secondary Outcomes (1)

• Safety and Incidence of Adverse Events

  Throughout the study period (up to 3 years)

Study Arms (1)

Sequential Gemcitabine and Jelmyto Treatment (Gem/Jel)

EXPERIMENTAL

Participants in this arm will receive six weekly doses of sequential gemcitabine and Jelmyto (mitomycin) directly administered into the affected kidney through a nephrostomy tube. Depending on the tumor's response, participants may receive an additional six weekly doses or transition to a standard of care maintenance regimen. The primary goal of this arm is to evaluate the complete response rate, safety, and long-term outcomes such as recurrence-free survival and kidney preservation. Interventions: Drug: Gemcitabine (endoluminal administration) Drug: Jelmyto (gel-based mitomycin, endoluminal administration)

Drug: Sequential Gemcitabine and Jelmyto Treatment (Gem/Jel)

Interventions

Sequential Gemcitabine and Jelmyto Treatment (Gem/Jel) DRUG

Drug: Gemcitabine (endoluminal administration) Drug: Jelmyto (gel-based mitomycin, endoluminal administration)

Also known as: Gemcitabine (endoluminal administration), Jelmyto (gel-based mitomycin, endoluminal administration)

Sequential Gemcitabine and Jelmyto Treatment (Gem/Jel)

Eligibility Criteria

• Age: 18 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patient has signed Informed Consent Form and is willing and able to comply with all requirements of the protocol.
• Patient is at least 18 years of age.
• Naive or recurrent patients with High Grade Ta +/- CIS, non-invasive UTUC in the upper tract (pyelocalyceal system or ureter).
• Histologic confirmation with biopsy is necessary.
• All patients must undergo ureteroscopy with biopsy.
• Non-Invasive Papillary Carcinoma (HGTa) lesions must be maximally ablated with ≤ 15 mm residual papillary disease in total.
• \. Favorable high-grade upper tract urothelial carcinoma (UTUC) as per American Urological Association (AUA) risk stratification below (except that multifocality, obstruction, ureteral involvement, bladder involvement will be allowed).
• If the bladder is involved with NMIBC it should be treated prior to or during Gem/Jel treatments.
• \. Patients with ECOG (Eastern Cooperative Oncology Group) performance status \<3 (with Karnofsky \>40).
• \. Patients with life expectancy greater than 24 months at time of screening. 7. Patient has no active urinary tract infection (UTI) as confirmed by urine culture or urinalysis\*.
• \. Female Patients of childbearing potential\*\*, must have a negative serum pregnancy test at screening and must agree to use two acceptable \& effective methods of contraception\*\*\*, until 6 months post treatment 9. All sexually active male patients must agree to use a condom during intercourse, for at least 48 hours post each instillation.
• \. Male patients who have a partner that is a female of childbearing potential must agree to use two acceptable \& effective methods of contraception until 6 months post treatment.
• \. Patients must not have any other medical condition(s) that make(s) their participation in the study unadvisable in the opinion of the investigator.

You may not qualify if:

• Invasive upper tract urothelial carcinoma (suspected cT2-4 on imaging) or lymphadenopathy (cN1-3) Bilateral disease Patient is actively being treated or intends to be treated with systemic chemotherapy during the duration of the trial.
• \. Any other malignancy diagnosed within 2 years of trial entry with the exception of:
• a. Basal or squamous cell skin cancers, or b. Noninvasive cancer of the cervix, or c. Any other cancer deemed to be of low-risk for progression or patient morbidity during trial period (basal or squamous cell skin cancers, noninvasive cancer of the cervix, prostate cancer under active surveillance, renal cancer under active surveillance).
• \. Administration not feasible via nephrostomy tube. 5. Inability to deliver the investigational drug to the pyelocalyceal system. 6. Patient has any other medical or mental condition(s) that make(s) his/her participation in the trial unadvisable in the opinion of the treating urologist.
• \. Patient has contraindication to mitomycin C (MMC )treatment, or known sensitivity to MMC.
• \. Patient has an intractable bleeding disorder (e.g., coagulation factors deficiencies, Von Willebrand Disease).
• \. Patient is currently receiving any other investigational agents or has participated in a research protocol involving administration of an investigational product within the past 30 days prior to Visit 1.
• \. Pregnant (positive serum pregnancy test), planning to become pregnant during the trial period, breast-feeding, or of childbearing potential and not practicing reliable contraception\*\*\*.
• \* In case of a symptomatic UTI the patient will be treated with a full course of antibiotics, and the instillation will be postponed until resolution. In the case of asymptomatic bacteriuria, the use of prophylactic antibiotics and postponement of the treatment is left to the discretion of the PI.
• \*\* Women of non-childbearing potential:
• At least 12 months since the last menses, or
• Without uterus and/or both ovaries, or
• Has been surgically sterile for at least 6 months prior to trial drug administration.
• Acceptable methods of birth control which are considered to have a low failure rate (i.e., less than 1% per year) when used consistently and correctly, such as implants, injectable, combined (estrogen/progesterone) oral contraceptives, intrauterine devices (IUDs-only hormonal), condoms with spermicide, sexual abstinence or vasectomized partner.

Sponsors & Collaborators

• Rutgers, The State University of New Jersey: lead

Study Sites (1)

Rutgers Cancer Institute

New Brunswick, New Jersey, 08901, United States

RECRUITING

MeSH Terms

Conditions

Urinary Bladder Neoplasms

Interventions

Gemcitabine

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

Intervention Hierarchy (Ancestors)

Heterocyclic Compounds; Deoxycytidine; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring

Central Study Contacts

Vignesh Packiam, MD

CONTACT

732-235-2465; VP624@cinj.rutgers.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor

Model Details: The study uses a Single Group Assignment model, where all participants enrolled in the trial will receive the same experimental intervention: sequential administration of gemcitabine and Jelmyto (mitomycin). The intervention is delivered directly into the kidney through a nephrostomy tube over six weekly treatments, with possible additional cycles based on the tumor's response. The objective of this model is to evaluate how this combination treatment affects tumor response, recurrence rates, and kidney preservation outcomes in participants with favorable high-grade upper tract urothelial carcinoma (UTUC). No control group or randomization is used, as the primary purpose is to determine the safety and efficacy of the sequential chemotherapy treatment in a focused patient population.

Study Record Dates

• First Submitted: February 6, 2025
• First Posted: February 12, 2025
• Study Start: March 6, 2026
• Primary Completion (Estimated): April 1, 2028
• Study Completion (Estimated): April 1, 2029
• Last Updated: May 6, 2026

Record last verified: 2026-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)