Trial Outcomes & Findings for Sirolimus, Cisplatin, and Gemcitabine Hydrochloride in Treating Patients With Bladder Cancer (NCT NCT01938573)

NCT ID: NCT01938573

Last Updated: 2017-10-20

Results Overview

Safety will be assessed through summaries of adverse events, vital signs, physical examinations, and clinical laboratory test data (including change from baseline).

• Recruitment status: COMPLETED
• Study phase: PHASE1/PHASE2
• Target enrollment: 21 participants
• Primary outcome timeframe: Up to 28 days
• Results posted on: 2017-10-20

Participant Flow

Participant milestones

Measure	Phase 2 Sirolimus day -2, cisplatin 70 mg/m2 IV Day 1 and gemcitabine hydrochloride 1000 mg/m2 IV days 1 and 8 every 21 days for 4 cycles followed by cystectomy (surgery) Cisplatin: Given IV Gemcitabine Hydrochloride: Given IV Sirolimus: Given PO Cystectomy: Undergo cystectomy when appropriate	Phase I Sirolimus 35 mg PO day -2, cisplatin 70 mg/m2 day 1, gemcitabine 1000 mg/m2 days 1 and 8, every 21 days
Overall Study STARTED	15	6
Overall Study COMPLETED	13	6
Overall Study NOT COMPLETED	2	0

Reasons for withdrawal

Measure	Phase 2 Sirolimus day -2, cisplatin 70 mg/m2 IV Day 1 and gemcitabine hydrochloride 1000 mg/m2 IV days 1 and 8 every 21 days for 4 cycles followed by cystectomy (surgery) Cisplatin: Given IV Gemcitabine Hydrochloride: Given IV Sirolimus: Given PO Cystectomy: Undergo cystectomy when appropriate	Phase I Sirolimus 35 mg PO day -2, cisplatin 70 mg/m2 day 1, gemcitabine 1000 mg/m2 days 1 and 8, every 21 days
Overall Study Lack of Efficacy	1	0
Overall Study Operation at other hospital	1	0

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Measure	Sirolimus, Cisplatin, Gemcitabine n=21 Participants Sirolimus day -2, cisplatin 70 mg/m2 IV Day 1 and gemcitabine hydrochloride 1000 mg/m2 IV days 1 and 8 every 21 days for 4 cycles followed by cystectomy (surgery) Cisplatin: Given IV Gemcitabine Hydrochloride: Given IV Sirolimus: Given PO Cystectomy: Undergo cystectomy when appropriate
Age, Continuous	69 years n=21 Participants
Sex: Female, Male Female	1 Participants n=21 Participants
Sex: Female, Male Male	20 Participants n=21 Participants
Region of Enrollment United States	21 participants n=21 Participants

PRIMARY outcome

Timeframe: Up to 28 days

Safety will be assessed through summaries of adverse events, vital signs, physical examinations, and clinical laboratory test data (including change from baseline).

Outcome measures

Measure	Phase 1 - Sirolimus, Cisplatin, Gemcitabine n=6 Participants Sirolimus day -2, cisplatin 70 mg/m2 IV Day 1 and gemcitabine hydrochloride 1000 mg/m2 IV days 1 and 8 every 21 days Cisplatin: Given IV Gemcitabine Hydrochloride: Given IV Sirolimus: Given PO Cystectomy: Undergo cystectomy when appropriate
Patients With Dose Limiting Toxicity	0 Participants

PRIMARY outcome

Timeframe: 12 weeks

Population: Patients treated in phase 2

The study will follow an optimal two-stage Simon design based on pathologic complete response rate.

Outcome measures

Measure	Phase 1 - Sirolimus, Cisplatin, Gemcitabine n=15 Participants Sirolimus day -2, cisplatin 70 mg/m2 IV Day 1 and gemcitabine hydrochloride 1000 mg/m2 IV days 1 and 8 every 21 days Cisplatin: Given IV Gemcitabine Hydrochloride: Given IV Sirolimus: Given PO Cystectomy: Undergo cystectomy when appropriate
Percent of Patients With Pathologic Complete Response (Phase II)	4 Participants

SECONDARY outcome

Timeframe: Up to 28 days after completion of study treatment

Graded according to the NCI CTCAE version 4.0. Safety will be assessed through summaries of adverse events, vital signs, physical examinations, and clinical laboratory test data (including change from baseline). All adverse events resulting in discontinuation, dose modification, dosing interruption, and/or treatment delay of study drug will also be listed and tabulated by preferred term.

Outcome measures

Measure	Phase 1 - Sirolimus, Cisplatin, Gemcitabine n=21 Participants Sirolimus day -2, cisplatin 70 mg/m2 IV Day 1 and gemcitabine hydrochloride 1000 mg/m2 IV days 1 and 8 every 21 days Cisplatin: Given IV Gemcitabine Hydrochloride: Given IV Sirolimus: Given PO Cystectomy: Undergo cystectomy when appropriate
Incidence of Adverse Events Including Any Unfavorable and Unintended Sign, Symptom, Diagnosis, or Disease Temporally Associated With the Use of a Medicinal Product, Whether or Not Related to the Medicinal Product (Phase I and II)	45 Number of events

Adverse Events

Sirolimus, Cisplatin, Gemcitabine

Serious events: 0 serious events

Other events: 21 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	Sirolimus, Cisplatin, Gemcitabine n=21 participants at risk Sirolimus day -2, cisplatin 70 mg/m2 IV Day 1 and gemcitabine hydrochloride 1000 mg/m2 IV days 1 and 8 every 21 days for 4 cycles followed by cystectomy (surgery) Cisplatin: Given IV Gemcitabine Hydrochloride: Given IV Sirolimus: Given PO Cystectomy: Undergo cystectomy when appropriate
Blood and lymphatic system disorders Neutropenia	23.8% 5/21 • Number of events 5 • 4 months (neoadjuvant therapy (3 months) and 1 month after completion)
Blood and lymphatic system disorders Thrombocytopenia	38.1% 8/21 • Number of events 8 • 4 months (neoadjuvant therapy (3 months) and 1 month after completion)
Blood and lymphatic system disorders Anemia	19.0% 4/21 • Number of events 4 • 4 months (neoadjuvant therapy (3 months) and 1 month after completion)
General disorders Fatigue	47.6% 10/21 • Number of events 10 • 4 months (neoadjuvant therapy (3 months) and 1 month after completion)

Additional Information

Dr. Bruce Montgomery

University of Washington

Phone: 206-598-0860

Email: rbmontgo@uw.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place