Urothelial Carcinoma Ureteral Location Clinical Trial
Official title:
Individual Patient Expanded Access - MitoGel for Upper Urinary Tract Urothelial Carcinoma
The aim of this study is to evaluate the feasibility, safety, and efficacy of MitoGel in the treatment of UTUC in a human subject with low-grade UTUC which is endoscopically unresectable or rapidly recurring, and in whom nephrectomy would likely result in the need for permanent hemodialysis. The study drug would be obtained under the single patient access program approved by the FDA. The patient would then undergo instillation of MitoGel into the affected kidney. The catheter would be left indwelling in the ureter and would be externalized. The ureteral and urethral catheters would remain indwelling for the duration of the 6 treatments, which would occur twice weekly for 3 weeks or once weekly for 6 weeks. Following the final instillation, the catheters would be removed. The patient would then undergo ureteroscopic evaluation at 3 months following the final instillation of MitoGel. The total duration of study would be 3 months. The total number of study patients is 1.
Upper urinary tract urothelial carcinoma (UTUC) is a rare malignant neoplasm arising from the
urothelial lining of the renal collecting system and ureters. The current treatment paradigm
for UTUC involves endoscopic resection, when technically feasible and oncologically sound, or
complete resection of the kidney and ureter (nephroureterectomy) in cases in which
organ-sparing treatment is not feasible.
Both organ-sparing and complete resection options present significant technical and clinical
challenges. For patients with bilateral disease, an anatomic or functionally solitary kidney,
or significant underlying medicorenal disease, treatment with nephroureterectomy would result
dialysis dependence, a state which carries with it a significant risk of 5-year mortality in
the often elderly and co-morbid patient population affected by UTUC.
MitoGel is a novel investigational product composed of RTGel, a unique hydrogel polymer which
exists as liquid at cold temperatures and a viscous gel at body temperatures, and Mitomycin
C, a clinically proven safe and effective treatment for urothelial carcinoma in the bladder.
MitoGel can be instilled into the upper urinary tract as a cold liquid, where it solidifies
to a gel upon warming to body temperature. The agent remains in the upper tract for a period
of 4-6 hours, resulting in sustained contact between the tissues and Mitomycin C. Preclinical
studies of MitoGel in the Yorkshire swine have demonstrated that both single and serial
instillations into the pelvicalyceal system via both retrograde and antegrade approaches are
safe, with no observable adverse clinical, laboratory, or histological effects. For these
reasons, MitoGel has generated interest as a treatment for UTUC.
For patients with UTUC in whom nephroureterectomy would result in dialysis dependence, and in
whom the disease is difficult to manage endoscopically, there is no currently available means
to effectively deliver adjuvant chemotherapy to the upper tracts. These patients are thus
either condemned to treatment with complete resection of their kidney and resultant
hemodialysis, or risk progression of disease to an incurable and unmanageable state. MitoGel
represents a promising treatment option for patients who have no other therapeutic options.
For these reasons, MitoGel has been granted Orphan Designation Status by the FDA. While
clinical trials of MitoGel are currently being planned, no trials are open to date.
The aim of this study is to evaluate the feasibility, safety, and efficacy of MitoGel in the
treatment of UTUC in a human subject with low-grade UTUC which is endoscopically unresectable
or rapidly recurring, and in whom nephrectomy would likely result in the need for permanent
hemodialysis. The study drug would be obtained under the single patient access program
approved by the FDA. The patient would then undergo instillation of MitoGel into the affected
kidney. The catheter would be left indwelling in the ureter and would be externalized through
a urethral catheter. The ureteral and urethral catheters would remain indwelling for the
duration of the 6 treatments, which would occur twice weekly for 3 weeks. Following the final
instillation, the catheters would be removed. The patient would then undergo ureteroscopic
evaluation at 3 months following the final instillation of MitoGel. The total duration of
study would be 3 months. The total number of study patients is 1.
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