Trial Outcomes & Findings for Efficacy and Safety Study of Apaziquone vs. Placebo in Patients With Non-Muscle Invasive Bladder Cancer (NMIBC) (NCT NCT01469221)
NCT ID: NCT01469221
Last Updated: 2017-12-15
Results Overview
Time from randomization to the date of first histologically confirmed recurrence of bladder cancer
Recruitment status
TERMINATED
Study phase
PHASE3
Target enrollment
47 participants
Primary outcome timeframe
24 Months
Results posted on
2017-12-15
Participant Flow
Patients 1) meeting Open Label Phase and Double Blind Phase inclusion and exclusion criteria, including confirmed histology based on Central Pathology Review, and 2) has eligibility packet approved by Sponsor, are randomized in a 1:1 fashion to receive 6 weekly instillations of apaziquone or matching placebo.
Participant milestones
| Measure |
Apaziquone
6 weekly multi-instillation of Apaziquone 4 mg in 40 mL
|
Placebo
6 weekly multi-instillation of matching placebo in 40 mL
|
Open Label-Apaziquone
Single dose of Apaziquone 4 mg in 40 mL
|
|---|---|---|---|
|
Open Label Phase
STARTED
|
0
|
0
|
47
|
|
Open Label Phase
COMPLETED
|
0
|
0
|
44
|
|
Open Label Phase
NOT COMPLETED
|
0
|
0
|
3
|
|
Double Blind Phase
STARTED
|
14
|
17
|
0
|
|
Double Blind Phase
COMPLETED
|
0
|
0
|
0
|
|
Double Blind Phase
NOT COMPLETED
|
14
|
17
|
0
|
Reasons for withdrawal
| Measure |
Apaziquone
6 weekly multi-instillation of Apaziquone 4 mg in 40 mL
|
Placebo
6 weekly multi-instillation of matching placebo in 40 mL
|
Open Label-Apaziquone
Single dose of Apaziquone 4 mg in 40 mL
|
|---|---|---|---|
|
Double Blind Phase
Protocol Violation
|
1
|
0
|
0
|
|
Double Blind Phase
Physician Decision
|
1
|
1
|
0
|
|
Double Blind Phase
Sponsor Terminate Study
|
12
|
16
|
0
|
Baseline Characteristics
Efficacy and Safety Study of Apaziquone vs. Placebo in Patients With Non-Muscle Invasive Bladder Cancer (NMIBC)
Baseline characteristics by cohort
| Measure |
Apaziquone
n=14 Participants
Apaziquone (4 mg in 40 mL)
Apaziquone: 6 weekly multi-instillation of Apaziquone 4 mg in 40 mL
|
Placebo
n=17 Participants
Matching placebo (40 mL)
Placebo: 6 weekly multi-instillation of matching placebo in 40mL
|
Total
n=31 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
5 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
14 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
9 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
17 Participants
n=5 Participants
|
|
Age, Continuous
|
68.4 years
STANDARD_DEVIATION 9.9 • n=5 Participants
|
63.6 years
STANDARD_DEVIATION 9.2 • n=7 Participants
|
65.7 years
STANDARD_DEVIATION 9.7 • n=5 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
12 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
19 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
14 Participants
n=5 Participants
|
17 Participants
n=7 Participants
|
31 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
13 Participants
n=5 Participants
|
17 Participants
n=7 Participants
|
30 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 24 MonthsPopulation: Patients who were in the Double Blind Phase
Time from randomization to the date of first histologically confirmed recurrence of bladder cancer
Outcome measures
| Measure |
Apaziquone
n=14 Participants
Apaziquone (4 mg in 40 mL)
Apaziquone: 6 weekly multi-instillation of Apaziquone 4 mg in 40 mL
|
Placebo
n=17 Participants
Matching placebo (40 mL)
Placebo: 6 weekly multi-instillation of matching placebo in 40mL
|
|---|---|---|
|
Time to Recurrence
|
NA months
Interval 1.5 to 1.5
only 1 patient recurred, median time to recurrence not reached
|
8.5 months
Interval 2.5 to 8.5
|
SECONDARY outcome
Timeframe: 24 MonthsPopulation: Patients who were in the Double Blind Phase
Proportion of patients with recurrence at or before 24 months
Outcome measures
| Measure |
Apaziquone
n=14 Participants
Apaziquone (4 mg in 40 mL)
Apaziquone: 6 weekly multi-instillation of Apaziquone 4 mg in 40 mL
|
Placebo
n=17 Participants
Matching placebo (40 mL)
Placebo: 6 weekly multi-instillation of matching placebo in 40mL
|
|---|---|---|
|
2-Year Recurrence Rate
|
1 Participants
|
6 Participants
|
Adverse Events
Apaziquone
Serious events: 1 serious events
Other events: 6 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 11 other events
Deaths: 0 deaths
Open Label-Apaziquone
Serious events: 2 serious events
Other events: 23 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Apaziquone
n=14 participants at risk
6 weekly multi-instillation of Apaziquone 4 mg in 40 mL
|
Placebo
n=17 participants at risk
6 weekly multi-instillation of matching placebo in 40 mL
|
Open Label-Apaziquone
n=47 participants at risk
Single dose of Apaziquone 4 mg in 40 mL
|
|---|---|---|---|
|
Cardiac disorders
Cardiac failure congestive
|
7.1%
1/14
|
0.00%
0/17
|
2.1%
1/47
|
|
Renal and urinary disorders
Urinary retention
|
0.00%
0/14
|
0.00%
0/17
|
2.1%
1/47
|
Other adverse events
| Measure |
Apaziquone
n=14 participants at risk
6 weekly multi-instillation of Apaziquone 4 mg in 40 mL
|
Placebo
n=17 participants at risk
6 weekly multi-instillation of matching placebo in 40 mL
|
Open Label-Apaziquone
n=47 participants at risk
Single dose of Apaziquone 4 mg in 40 mL
|
|---|---|---|---|
|
Infections and infestations
Cystitis
|
0.00%
0/14
|
17.6%
3/17
|
6.4%
3/47
|
|
Metabolism and nutrition disorders
Hypercholesterolaemia
|
14.3%
2/14
|
0.00%
0/17
|
4.3%
2/47
|
|
Renal and urinary disorders
Haematuria
|
7.1%
1/14
|
11.8%
2/17
|
8.5%
4/47
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/14
|
5.9%
1/17
|
2.1%
1/47
|
|
Infections and infestations
Bronchitis
|
0.00%
0/14
|
5.9%
1/17
|
2.1%
1/47
|
|
Infections and infestations
Pharyngitis
|
0.00%
0/14
|
5.9%
1/17
|
2.1%
1/47
|
|
Renal and urinary disorders
Calculus bladder
|
0.00%
0/14
|
5.9%
1/17
|
2.1%
1/47
|
|
Renal and urinary disorders
Cystitis noninfective
|
0.00%
0/14
|
5.9%
1/17
|
4.3%
2/47
|
|
Renal and urinary disorders
Nephrolithiasis
|
7.1%
1/14
|
0.00%
0/17
|
2.1%
1/47
|
|
Renal and urinary disorders
Pollakiuria
|
7.1%
1/14
|
0.00%
0/17
|
4.3%
2/47
|
|
Renal and urinary disorders
Urinary retention
|
0.00%
0/14
|
5.9%
1/17
|
4.3%
2/47
|
|
Vascular disorders
Peripheral ischaemia
|
7.1%
1/14
|
0.00%
0/17
|
2.1%
1/47
|
|
Nervous system disorders
Paraesthesia
|
0.00%
0/14
|
5.9%
1/17
|
2.1%
1/47
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Abdominal neoplasm
|
0.00%
0/14
|
5.9%
1/17
|
2.1%
1/47
|
|
Hepatobiliary disorders
Hepatic cirrhosis
|
7.1%
1/14
|
0.00%
0/17
|
2.1%
1/47
|
|
Infections and infestations
Skin infection
|
0.00%
0/14
|
5.9%
1/17
|
2.1%
1/47
|
|
Infections and infestations
Tooth infection
|
0.00%
0/14
|
5.9%
1/17
|
2.1%
1/47
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/14
|
5.9%
1/17
|
2.1%
1/47
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place