Trial Outcomes & Findings for Safety, Tolerability and Efficacy Study of LiRIS® 400 mg in Women With Interstitial Cystitis (NCT NCT01824303)
NCT ID: NCT01824303
Last Updated: 2016-01-26
Results Overview
Participants rated their bladder pain over the previous 24 hours in an electronic diary using a horizontal line NRS by putting a mark on the line where: 0 (the far left of the line)=no pain to 10 (far right of the line)=worst pain imaginable. Bladder pain was averaged over a period of 3 days. A negative change from Baseline indicates improvement.
TERMINATED
PHASE2
31 participants
Baseline, Day 12
2016-01-26
Participant Flow
31 participants signed the informed consent form and were enrolled in the study, 30 participants had the investigational product inserted and are included the Intent-to-treat Population.
Participant milestones
| Measure |
LiRIS 400 mg
LiRIS 400 mg: investigational drug-delivery system which contains Lidocaine; the system remains in the bladder for 14 days.
|
LiRIS Placebo
LiRIS Placebo: investigational drug-delivery system which contains Lactose and no Lidocaine; the system remains in the bladder for 14 days.
|
|---|---|---|
|
Randomized Study
STARTED
|
16
|
14
|
|
Randomized Study
COMPLETED
|
14
|
13
|
|
Randomized Study
NOT COMPLETED
|
2
|
1
|
|
Open Label Extension
STARTED
|
12
|
11
|
|
Open Label Extension
COMPLETED
|
12
|
11
|
|
Open Label Extension
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
| Measure |
LiRIS 400 mg
LiRIS 400 mg: investigational drug-delivery system which contains Lidocaine; the system remains in the bladder for 14 days.
|
LiRIS Placebo
LiRIS Placebo: investigational drug-delivery system which contains Lactose and no Lidocaine; the system remains in the bladder for 14 days.
|
|---|---|---|
|
Randomized Study
Withdrawal by Subject
|
1
|
1
|
|
Randomized Study
Other Miscellaneous Reasons
|
1
|
0
|
Baseline Characteristics
Safety, Tolerability and Efficacy Study of LiRIS® 400 mg in Women With Interstitial Cystitis
Baseline characteristics by cohort
| Measure |
LiRIS 400 mg
n=16 Participants
LiRIS 400 mg: investigational drug-delivery system which contains Lidocaine; the system remains in the bladder for 14 days.
|
LiRIS Placebo
n=14 Participants
LiRIS Placebo: investigational drug-delivery system which contains Lactose and no Lidocaine; the system remains in the bladder for 14 days.
|
Total
n=30 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
41.7 years
STANDARD_DEVIATION 14.13 • n=5 Participants
|
42.1 years
STANDARD_DEVIATION 10.54 • n=7 Participants
|
41.9 years
STANDARD_DEVIATION 12.37 • n=5 Participants
|
|
Sex: Female, Male
Female
|
16 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
30 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline, Day 12Population: Intent-to-treat (ITT) population included all randomized participants.
Participants rated their bladder pain over the previous 24 hours in an electronic diary using a horizontal line NRS by putting a mark on the line where: 0 (the far left of the line)=no pain to 10 (far right of the line)=worst pain imaginable. Bladder pain was averaged over a period of 3 days. A negative change from Baseline indicates improvement.
Outcome measures
| Measure |
LiRIS 400 mg
n=16 Participants
LiRIS 400 mg: investigational drug-delivery system which contains Lidocaine; the system remains in the bladder for 14 days.
|
LiRIS Placebo
n=14 Participants
LiRIS Placebo: investigational drug-delivery system which contains Lactose and no Lidocaine; the system remains in the bladder for 14 days.
|
|---|---|---|
|
Change From Baseline in Participant Reported Average Bladder Pain on a Numerical Rating Scale (NRS)
Baseline
|
7.04 unit on a scale
Standard Deviation 0.750
|
7.67 unit on a scale
Standard Deviation 1.067
|
|
Change From Baseline in Participant Reported Average Bladder Pain on a Numerical Rating Scale (NRS)
Change from Baseline at Day 12
|
-1.44 unit on a scale
Standard Deviation 1.584
|
-2.50 unit on a scale
Standard Deviation 2.723
|
SECONDARY outcome
Timeframe: Baseline, Day 27Population: Participants from the ITT population, all randomized participants, with data available for analyses.
Participants recorded number of daily voids (day-time and night-time daily voids) in a 3 day Voiding Frequency electronic diary. The total number of daily voids was averaged over a period of 3 full days. A negative change from Baseline indicates improvement.
Outcome measures
| Measure |
LiRIS 400 mg
n=16 Participants
LiRIS 400 mg: investigational drug-delivery system which contains Lidocaine; the system remains in the bladder for 14 days.
|
LiRIS Placebo
n=14 Participants
LiRIS Placebo: investigational drug-delivery system which contains Lactose and no Lidocaine; the system remains in the bladder for 14 days.
|
|---|---|---|
|
Change From Baseline in Total Daily Voids
Baseline
|
13.4 voids
Standard Deviation 6.52
|
14.9 voids
Standard Deviation 7.81
|
|
Change From Baseline in Total Daily Voids
Change from Baseline at Day 27 (n=15,14)
|
-2.9 voids
Standard Deviation 4.64
|
-3.3 voids
Standard Deviation 4.93
|
SECONDARY outcome
Timeframe: Baseline, Day 27Population: Participants from the ITT population, all randomized participants, with data available for analyses.
Participants recorded number of night-time daily voids in a 3 day Voiding Frequency electronic diary. The total number of night-time daily voids was averaged over a period of 3 days. A negative change from Baseline indicates improvement.
Outcome measures
| Measure |
LiRIS 400 mg
n=16 Participants
LiRIS 400 mg: investigational drug-delivery system which contains Lidocaine; the system remains in the bladder for 14 days.
|
LiRIS Placebo
n=14 Participants
LiRIS Placebo: investigational drug-delivery system which contains Lactose and no Lidocaine; the system remains in the bladder for 14 days.
|
|---|---|---|
|
Change From Baseline in Night-Time Daily Voids
Baseline
|
2.4 voids
Standard Deviation 2.56
|
2.1 voids
Standard Deviation 2.37
|
|
Change From Baseline in Night-Time Daily Voids
Change from Baseline at Day 27 (n=15,14)
|
-0.5 voids
Standard Deviation 2.09
|
-0.9 voids
Standard Deviation 2.00
|
SECONDARY outcome
Timeframe: Baseline, Day 27Population: Participants from the ITT population, all randomized participants, with data available for analyses.
Participants recorded the amount of each void in millimeters (mL) in an electronic diary. The total amount of void per micturition (each void) was averaged over a period of 24 days. A negative change from Baseline indicates improvement.
Outcome measures
| Measure |
LiRIS 400 mg
n=16 Participants
LiRIS 400 mg: investigational drug-delivery system which contains Lidocaine; the system remains in the bladder for 14 days.
|
LiRIS Placebo
n=14 Participants
LiRIS Placebo: investigational drug-delivery system which contains Lactose and no Lidocaine; the system remains in the bladder for 14 days.
|
|---|---|---|
|
Change From Baseline in Average Void Volume Per Micturition
Baseline
|
141.1 mL
Standard Deviation 80.26
|
120.3 mL
Standard Deviation 73.06
|
|
Change From Baseline in Average Void Volume Per Micturition
Change from Baseline at Day 27 (n=14,12)
|
-2.2 mL
Standard Deviation 73.31
|
-0.5 mL
Standard Deviation 43.86
|
SECONDARY outcome
Timeframe: Baseline, Day 27Population: Participants from the ITT population, all randomized participants, with data available for analyses.
Participants rated their bladder pain immediately completing voiding in an electronic diary using a horizontal line NRS by putting a mark on the line where: 0 (the far left of the line)=no pain to 10 (far right of the line)=worst pain imaginable. The average of the data from the first 5 voids prior to Baseline and the 3 days prior to Day 12 were averaged for analyses. A negative change from Baseline indicates improvement.
Outcome measures
| Measure |
LiRIS 400 mg
n=16 Participants
LiRIS 400 mg: investigational drug-delivery system which contains Lidocaine; the system remains in the bladder for 14 days.
|
LiRIS Placebo
n=14 Participants
LiRIS Placebo: investigational drug-delivery system which contains Lactose and no Lidocaine; the system remains in the bladder for 14 days.
|
|---|---|---|
|
Change From Baseline in Post-Void Bladder Pain
Baseline (n=13,11)
|
4.86 units on a scale
Standard Deviation 1.857
|
6.17 units on a scale
Standard Deviation 1.925
|
|
Change From Baseline in Post-Void Bladder Pain
Change from Baseline at Day 27 (n=12,10)
|
-1.05 units on a scale
Standard Deviation 1.805
|
-1.69 units on a scale
Standard Deviation 1.997
|
SECONDARY outcome
Timeframe: Baseline, Day 27Population: Participants from the ITT population, all randomized participants, with data available for analyses.
The ICSI is a participant reported questionnaire to rate their symptoms by answering 4 questions. Question (Q) 1 and 2 (urine urgency) using a 6-point scale where: 0=Not at All to 5=Almost Always. Q3 (night-time voids) using a 6-point scale where: 0=None to 5=5 or More Times. Q4 (pain and burning) using a 6-point scale where: 0=Not at All to 5=Usually. The total score is the sum of the individual scores for a total possible score of 0 (best) to 20 (worst). A negative change from Baseline indicates improvement.
Outcome measures
| Measure |
LiRIS 400 mg
n=16 Participants
LiRIS 400 mg: investigational drug-delivery system which contains Lidocaine; the system remains in the bladder for 14 days.
|
LiRIS Placebo
n=14 Participants
LiRIS Placebo: investigational drug-delivery system which contains Lactose and no Lidocaine; the system remains in the bladder for 14 days.
|
|---|---|---|
|
Change From Baseline in O'Leary-Sant Interstitial Cystitis Symptom Index (ICSI) Score
Baseline
|
14.1 score on a scale
Standard Deviation 3.70
|
14.2 score on a scale
Standard Deviation 2.73
|
|
Change From Baseline in O'Leary-Sant Interstitial Cystitis Symptom Index (ICSI) Score
Change from Baseline at Day 27 (n=14,12)
|
-1.4 score on a scale
Standard Deviation 3.79
|
-2.8 score on a scale
Standard Deviation 3.04
|
SECONDARY outcome
Timeframe: Baseline, Day 27Population: Participants from the ITT population, all randomized participants, with data available for analyses.
The ICPI is a participant reported questionnaire to rate their problems in the following 4 areas: Frequent urination; Getting up at Night to Urinate: Urination with little warning; Burning pain and discomfort. Questions are answered on a 5-point scale where: 0=No Problem to 4=Big Problem. The total score is the sum of the individual scores for a total possible score of 0 (best) to 20 (worst). A negative change from Baseline indicates improvement.
Outcome measures
| Measure |
LiRIS 400 mg
n=16 Participants
LiRIS 400 mg: investigational drug-delivery system which contains Lidocaine; the system remains in the bladder for 14 days.
|
LiRIS Placebo
n=14 Participants
LiRIS Placebo: investigational drug-delivery system which contains Lactose and no Lidocaine; the system remains in the bladder for 14 days.
|
|---|---|---|
|
Change From Baseline in O'Leary-Sant Interstitial Cystitis Problem Index (ICPI) Score
Baseline
|
12.4 score on a scale
Standard Deviation 2.87
|
13.9 score on a scale
Standard Deviation 1.66
|
|
Change From Baseline in O'Leary-Sant Interstitial Cystitis Problem Index (ICPI) Score
Change from Baseline at Day 27 (n=14,12)
|
-1.3 score on a scale
Standard Deviation 3.52
|
-3.2 score on a scale
Standard Deviation 3.46
|
SECONDARY outcome
Timeframe: Baseline, Day 27Population: Participants from the ITT population, all randomized participants, with data available for analyses.
The BPIC-SS is a participant reported questionnaire consisting of 8 items. Questions (Q) 1-5 (How often urination because of pain; Need to urinate after just urinating; How often urination to avoid worse pain; How often feeling of pressure in bladder; How often pain in bladder) answered on a 5-point scale where: 0=Never to 4=Always. Q 6-7 (Bothered by frequent Day-time urination; Bothered by Night-time urination) answered on a 5-point scale where: 0=Not At All to 4=A Great Deal. Q8 (pain) rated on a NRS by putting a mark on the line where: 0 (the far left of the line)=no pain to 10 (far right of the line)=worst pain imaginable. The Total Score is the sum of the individual questionnaire of all 8 items for a total possible score of 0 (best) to 38 (worst). A negative change indicates improvement.
Outcome measures
| Measure |
LiRIS 400 mg
n=16 Participants
LiRIS 400 mg: investigational drug-delivery system which contains Lidocaine; the system remains in the bladder for 14 days.
|
LiRIS Placebo
n=14 Participants
LiRIS Placebo: investigational drug-delivery system which contains Lactose and no Lidocaine; the system remains in the bladder for 14 days.
|
|---|---|---|
|
Change From Baseline in Bladder Pain/Interstitial Cystitis Symptom Score (BPIC-SS)
Baseline (n=16,13)
|
28.8 score on a scale
Standard Deviation 4.16
|
31.2 score on a scale
Standard Deviation 3.00
|
|
Change From Baseline in Bladder Pain/Interstitial Cystitis Symptom Score (BPIC-SS)
Change from Baseline at Day 27 (n=14,12)
|
-4.0 score on a scale
Standard Deviation 5.75
|
-7.1 score on a scale
Standard Deviation 7.81
|
SECONDARY outcome
Timeframe: Baseline, Day 27Population: Participants from the ITT population, all randomized participants, with data available for analyses.
The BPI is a 7-item participant completed questionnaire. The participant answered questions as to how pain interfered with: General Activity, Mood, Walking Ability, Normal work, Relations with other people, Sleep and Enjoyment of life. Questions were answered on an 11-point scale where: 0=Does not interfere to 10=Completely interferes. The total score is the average of the individual questionnaire items for a total possible score of 0 (best) to 10 (worst). A negative change from Baseline indicates improvement.
Outcome measures
| Measure |
LiRIS 400 mg
n=16 Participants
LiRIS 400 mg: investigational drug-delivery system which contains Lidocaine; the system remains in the bladder for 14 days.
|
LiRIS Placebo
n=14 Participants
LiRIS Placebo: investigational drug-delivery system which contains Lactose and no Lidocaine; the system remains in the bladder for 14 days.
|
|---|---|---|
|
Change From Baseline in Brief Pain Inventory (BPI)
Baseline (n=16,13)
|
6.5 score on a scale
Standard Deviation 1.10
|
7.2 score on a scale
Standard Deviation 1.99
|
|
Change From Baseline in Brief Pain Inventory (BPI)
Change from Baseline at Day 27 (n=14,12)
|
-1.2 score on a scale
Standard Deviation 2.52
|
-2.3 score on a scale
Standard Deviation 2.21
|
Adverse Events
LiRIS 400 mg_Randomized Study
LiRIS Placebo_Randomized Study
LiRIS 400 mg_Open Label Extension
LiRIS Placebo/LiRIS 400 mg _Open Label Extension
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
LiRIS 400 mg_Randomized Study
n=16 participants at risk
LiRIS 400 mg: investigational drug-delivery system which contains Lidocaine; the system remains in the bladder for 14 days in the randomized study.
|
LiRIS Placebo_Randomized Study
n=14 participants at risk
LiRIS Placebo: investigational drug-delivery system which contains Lactose and no Lidocaine; the system remains in the bladder for 14 days in the randomized study.
|
LiRIS 400 mg_Open Label Extension
n=12 participants at risk
LiRIS 400 mg: investigational drug-delivery system which contains Lidocaine; the system remains in the bladder for 14 days in the randomized study then LiRIS 400 mg in the Open Label Extension.
|
LiRIS Placebo/LiRIS 400 mg _Open Label Extension
n=11 participants at risk
LiRIS Placebo: investigational drug-delivery system which contains Lactose and no Lidocaine; the system remains in the bladder for 14 days in the randomized study then LiRIS 400 mg in the Open Label Extension.
|
|---|---|---|---|---|
|
Renal and urinary disorders
Dysuria
|
25.0%
4/16
|
28.6%
4/14
|
0.00%
0/12
|
18.2%
2/11
|
|
Renal and urinary disorders
Bladder pain
|
12.5%
2/16
|
21.4%
3/14
|
0.00%
0/12
|
9.1%
1/11
|
|
Renal and urinary disorders
Bladder discomfort
|
12.5%
2/16
|
14.3%
2/14
|
8.3%
1/12
|
0.00%
0/11
|
|
Renal and urinary disorders
Pollakiuria
|
6.2%
1/16
|
14.3%
2/14
|
0.00%
0/12
|
9.1%
1/11
|
|
Renal and urinary disorders
Haematuria
|
0.00%
0/16
|
14.3%
2/14
|
0.00%
0/12
|
0.00%
0/11
|
|
Renal and urinary disorders
Urethral pain
|
12.5%
2/16
|
0.00%
0/14
|
8.3%
1/12
|
9.1%
1/11
|
|
Renal and urinary disorders
Bladder spasm
|
0.00%
0/16
|
7.1%
1/14
|
0.00%
0/12
|
0.00%
0/11
|
|
Renal and urinary disorders
Cystitis interstitial
|
6.2%
1/16
|
0.00%
0/14
|
8.3%
1/12
|
18.2%
2/11
|
|
Renal and urinary disorders
Micturition urgency
|
6.2%
1/16
|
0.00%
0/14
|
0.00%
0/12
|
0.00%
0/11
|
|
Renal and urinary disorders
Nocturia
|
0.00%
0/16
|
7.1%
1/14
|
0.00%
0/12
|
0.00%
0/11
|
|
Renal and urinary disorders
Urinary incontinence
|
0.00%
0/16
|
7.1%
1/14
|
0.00%
0/12
|
9.1%
1/11
|
|
Infections and infestations
Urinary tract infection
|
6.2%
1/16
|
14.3%
2/14
|
16.7%
2/12
|
9.1%
1/11
|
|
Infections and infestations
Nasopharyngitis
|
6.2%
1/16
|
7.1%
1/14
|
0.00%
0/12
|
0.00%
0/11
|
|
Infections and infestations
Cystitis
|
6.2%
1/16
|
0.00%
0/14
|
0.00%
0/12
|
0.00%
0/11
|
|
Infections and infestations
Genital herpes
|
0.00%
0/16
|
7.1%
1/14
|
0.00%
0/12
|
0.00%
0/11
|
|
Infections and infestations
Sinusitis
|
6.2%
1/16
|
0.00%
0/14
|
0.00%
0/12
|
18.2%
2/11
|
|
Infections and infestations
Tooth abscess
|
6.2%
1/16
|
0.00%
0/14
|
0.00%
0/12
|
0.00%
0/11
|
|
Nervous system disorders
Dizziness
|
0.00%
0/16
|
7.1%
1/14
|
0.00%
0/12
|
0.00%
0/11
|
|
Nervous system disorders
Headache
|
0.00%
0/16
|
7.1%
1/14
|
0.00%
0/12
|
0.00%
0/11
|
|
Nervous system disorders
Migraine
|
0.00%
0/16
|
7.1%
1/14
|
0.00%
0/12
|
0.00%
0/11
|
|
Vascular disorders
Hypertension
|
6.2%
1/16
|
7.1%
1/14
|
0.00%
0/12
|
0.00%
0/11
|
|
Vascular disorders
Pallor
|
0.00%
0/16
|
7.1%
1/14
|
0.00%
0/12
|
0.00%
0/11
|
|
Immune system disorders
Allergy to arthropod bite
|
0.00%
0/16
|
7.1%
1/14
|
0.00%
0/12
|
0.00%
0/11
|
|
Immune system disorders
Allergy to arthropod sting
|
0.00%
0/16
|
7.1%
1/14
|
0.00%
0/12
|
0.00%
0/11
|
|
Investigations
Blood creatine phosphokinase increased
|
0.00%
0/16
|
7.1%
1/14
|
0.00%
0/12
|
0.00%
0/11
|
|
Investigations
Residual urine volume increased
|
6.2%
1/16
|
0.00%
0/14
|
0.00%
0/12
|
0.00%
0/11
|
|
Psychiatric disorders
Anxiety
|
6.2%
1/16
|
0.00%
0/14
|
0.00%
0/12
|
0.00%
0/11
|
|
Psychiatric disorders
Depression
|
0.00%
0/16
|
7.1%
1/14
|
0.00%
0/12
|
9.1%
1/11
|
|
Reproductive system and breast disorders
Dysfunctional uterine bleeding
|
6.2%
1/16
|
0.00%
0/14
|
0.00%
0/12
|
0.00%
0/11
|
|
Reproductive system and breast disorders
Pelvic discomfort
|
6.2%
1/16
|
0.00%
0/14
|
0.00%
0/12
|
0.00%
0/11
|
|
Reproductive system and breast disorders
Vaginal haemorrhage
|
0.00%
0/16
|
7.1%
1/14
|
0.00%
0/12
|
0.00%
0/11
|
|
Skin and subcutaneous tissue disorders
Dermatitis contact
|
6.2%
1/16
|
0.00%
0/14
|
0.00%
0/12
|
0.00%
0/11
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
0.00%
0/16
|
7.1%
1/14
|
0.00%
0/12
|
0.00%
0/11
|
|
Eye disorders
Conjunctivitis
|
6.2%
1/16
|
0.00%
0/14
|
0.00%
0/12
|
0.00%
0/11
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/16
|
7.1%
1/14
|
0.00%
0/12
|
0.00%
0/11
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/16
|
7.1%
1/14
|
0.00%
0/12
|
0.00%
0/11
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/16
|
7.1%
1/14
|
0.00%
0/12
|
0.00%
0/11
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
6.2%
1/16
|
0.00%
0/14
|
0.00%
0/12
|
0.00%
0/11
|
|
Infections and infestations
Fungal infection
|
0.00%
0/16
|
0.00%
0/14
|
8.3%
1/12
|
0.00%
0/11
|
|
Infections and infestations
Lower respiratory tract infection
|
0.00%
0/16
|
0.00%
0/14
|
8.3%
1/12
|
0.00%
0/11
|
|
Infections and infestations
Tonsillitis
|
0.00%
0/16
|
0.00%
0/14
|
0.00%
0/12
|
9.1%
1/11
|
|
Infections and infestations
Vulvovaginal mycotic infection
|
0.00%
0/16
|
0.00%
0/14
|
8.3%
1/12
|
0.00%
0/11
|
|
Renal and urinary disorders
Urethritis noninfective
|
0.00%
0/16
|
0.00%
0/14
|
0.00%
0/12
|
9.1%
1/11
|
|
Gastrointestinal disorders
Abdominal pain lower
|
0.00%
0/16
|
0.00%
0/14
|
0.00%
0/12
|
9.1%
1/11
|
|
Investigations
Vitamin D decreased
|
0.00%
0/16
|
0.00%
0/14
|
0.00%
0/12
|
9.1%
1/11
|
|
Nervous system disorders
Burning sensation
|
0.00%
0/16
|
0.00%
0/14
|
0.00%
0/12
|
9.1%
1/11
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/16
|
0.00%
0/14
|
8.3%
1/12
|
0.00%
0/11
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee A disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 90 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot extend the embargo.
- Publication restrictions are in place
Restriction type: OTHER