Trial Outcomes & Findings for Efficacy and Safety Evaluation of EN3348 (Mycobacterial Cell Wall-DNA Complex [MCC]) as Compared With Mitomycin C in the Intravesical Treatment of Subjects With BCG Recurrent/Refractory Non-muscle Invasive Bladder Cancer (NCT NCT01200992)

NCT ID: NCT01200992

Last Updated: 2017-08-24

Results Overview

Primary efficacy endpoint will be event-free survival - the interval from randomization to an event. An event is defined as tumor recurrence, tumor progression to muscle invasive bladder cancer or death, whichever occurs first. Tumor recurrence or progression must be documented by bladder biopsy.

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

84 participants

Primary outcome timeframe

1 year

Results posted on

2017-08-24

Participant Flow

Participant milestones

Participant milestones
Measure	EN3348 8 mg EN3348 mixed with water for injection for a total volume of 50mL EN3348: Induction: 6 weekly instillations. Maintenance: Monthly instillations to Month 12	Mitomycin C 40 mg mitomycin C mixed with water for injection to a total volume of 40 mL Mitomycin C: Induction: 6 weekly instillations. Maintenance: Monthly instillations to Month 12
Overall Study STARTED	39	45
Overall Study COMPLETED	2	4
Overall Study NOT COMPLETED	37	41

Reasons for withdrawal

Reasons for withdrawal
Measure	EN3348 8 mg EN3348 mixed with water for injection for a total volume of 50mL EN3348: Induction: 6 weekly instillations. Maintenance: Monthly instillations to Month 12	Mitomycin C 40 mg mitomycin C mixed with water for injection to a total volume of 40 mL Mitomycin C: Induction: 6 weekly instillations. Maintenance: Monthly instillations to Month 12
Overall Study Study terminated early	35	41
Overall Study Randomized but not treated	2	0

Baseline Characteristics

Efficacy and Safety Evaluation of EN3348 (Mycobacterial Cell Wall-DNA Complex [MCC]) as Compared With Mitomycin C in the Intravesical Treatment of Subjects With BCG Recurrent/Refractory Non-muscle Invasive Bladder Cancer

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	EN3348 n=39 Participants 8 mg EN3348 mixed with water for injection for a total volume of 50mL EN3348: Induction: 6 weekly instillations. Maintenance: Monthly instillations to Month 12	Mitomycin C n=45 Participants 40 mg mitomycin C mixed with water for injection to a total volume of 40 mL Mitomycin C: Induction: 6 weekly instillations. Maintenance: Monthly instillations to Month 12	Total n=84 Participants Total of all reporting groups
Age, Continuous	73.0 years STANDARD_DEVIATION 7.99 • n=5 Participants	70.7 years STANDARD_DEVIATION 11.18 • n=7 Participants	71.8 years STANDARD_DEVIATION 9.84 • n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants	12 Participants n=7 Participants	17 Participants n=5 Participants
Sex: Female, Male Male	34 Participants n=5 Participants	33 Participants n=7 Participants	67 Participants n=5 Participants

PRIMARY outcome

Timeframe: 1 year

Population: The study was discontinued early. The number of subjects randomized at time of closure represented 18.7% of the planned enrollment of 450 subjects. Thus, the planned analysis as stated in the protocol was not performed. Only 2 subjects (5.1%) in the EN3348 arm and 4 subjects (8.9%) in the mitomycin C arm completed all planned doses.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Through study early termination, approximately 23 months from first subject enrolled.

Population: Relevant safety data are presented in the Adverse Events and Serious Adverse Events Modules. Any clinically significant findings pertaining to other secondary outcomes such as vital signs, physical exams and laboratory test results would appear in these Modules as well. No statistical comparisons have been performed between the 2 treatment arms.

Safety endpoint displayed includes adverse events (other than serious adverse events) with a frequency threshold of 5% or greater, for each treatment arm. No statistical comparisons have been performed between the 2 treatment arms.

Outcome measures

Outcome measures
Measure	EN3348 n=37 Participants 8 mg mixed with water for injection for a total volume of 50mL Treatment - Induction (6 weekly instillations) followed Maintenance (monthly instillations up to Month 12)	Mitomycin C n=45 Participants 40 mg mixed with water for injection to a total volume of 40 mL Treatment - Induction (6 weekly instillations) followed Maintenance (monthly instillations up to Month 12)
Comparison of Safety of EN3348 With Mitomycin C [Adverse Events (Other Than Serious Adverse Events) With Frequency Threshold of 5% or Greater]. Dysuria	9 participants	12 participants
Comparison of Safety of EN3348 With Mitomycin C [Adverse Events (Other Than Serious Adverse Events) With Frequency Threshold of 5% or Greater]. Hematuria	7 participants	3 participants
Comparison of Safety of EN3348 With Mitomycin C [Adverse Events (Other Than Serious Adverse Events) With Frequency Threshold of 5% or Greater]. Pollakiuria	6 participants	6 participants
Comparison of Safety of EN3348 With Mitomycin C [Adverse Events (Other Than Serious Adverse Events) With Frequency Threshold of 5% or Greater]. Fatigue	6 participants	3 participants
Comparison of Safety of EN3348 With Mitomycin C [Adverse Events (Other Than Serious Adverse Events) With Frequency Threshold of 5% or Greater]. Urinary tract infection	5 participants	5 participants
Comparison of Safety of EN3348 With Mitomycin C [Adverse Events (Other Than Serious Adverse Events) With Frequency Threshold of 5% or Greater]. Diarrhea	3 participants	2 participants
Comparison of Safety of EN3348 With Mitomycin C [Adverse Events (Other Than Serious Adverse Events) With Frequency Threshold of 5% or Greater]. Back pain	2 participants	4 participants
Comparison of Safety of EN3348 With Mitomycin C [Adverse Events (Other Than Serious Adverse Events) With Frequency Threshold of 5% or Greater]. Nausea	2 participants	3 participants

Adverse Events

EN3348

Serious events: 4 serious events

Other events: 32 other events

Deaths: 0 deaths

Mitomycin C

Serious events: 2 serious events

Other events: 35 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	EN3348 n=37 participants at risk 8 mg mixed with sterile water for injection for a total volume of 50mL Treatment - Induction (6 weekly instillations) followed by Maintenance (monthly instillations up to Month 12)	Mitomycin C n=45 participants at risk 40 mg mixed with sterile water for injection to a total volume of 40 mL Treatment - Induction (6 weekly instillations) followed by Maintenance (monthly instillations up to Month 12)
Respiratory, thoracic and mediastinal disorders Asthma	2.7% 1/37 • Number of events 1	0.00% 0/45
Renal and urinary disorders Bladder spasms	2.7% 1/37 • Number of events 1	0.00% 0/45
Renal and urinary disorders Bladder perforation	2.7% 1/37 • Number of events 1	0.00% 0/45
Injury, poisoning and procedural complications Post-procedural hemorrhage	2.7% 1/37 • Number of events 1	0.00% 0/45
Vascular disorders Phlebitis	0.00% 0/37	2.2% 1/45 • Number of events 1
Infections and infestations Sepsis	0.00% 0/37	2.2% 1/45 • Number of events 1

Other adverse events

Other adverse events
Measure	EN3348 n=37 participants at risk 8 mg mixed with sterile water for injection for a total volume of 50mL Treatment - Induction (6 weekly instillations) followed by Maintenance (monthly instillations up to Month 12)	Mitomycin C n=45 participants at risk 40 mg mixed with sterile water for injection to a total volume of 40 mL Treatment - Induction (6 weekly instillations) followed by Maintenance (monthly instillations up to Month 12)
Renal and urinary disorders Dysuria	24.3% 9/37	26.7% 12/45
Renal and urinary disorders Hematuria	18.9% 7/37	6.7% 3/45
Renal and urinary disorders Pollakiuria	16.2% 6/37	13.3% 6/45
Gastrointestinal disorders Diarrhea	8.1% 3/37	4.4% 2/45
Gastrointestinal disorders Nausea	5.4% 2/37	6.7% 3/45
General disorders Fatigue	16.2% 6/37	6.7% 3/45
Infections and infestations Urinary tract infection	13.5% 5/37	11.1% 5/45
Musculoskeletal and connective tissue disorders Back pain	5.4% 2/37	8.9% 4/45

Additional Information

Aline Hagerimana, MD, Senior Director Clinical Affairs

Prometic Biosciences Inc.

Phone: (514) 781-0115

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: OTHER