Trial Outcomes & Findings for Real-world Evidence Study on Cystistat (NCT NCT05544695)

Results Overview

For the primary analysis, a responder was defined as a participant who had an improvement on the PGA scale (i.e., PGA evaluated as markedly improved, moderately improved, or slightly improved. In case of slightly improved, participants were considered as responder only if the question "Did therapy have a positive effect on your life, and would you undergo this treatment again?" is answered with "Yes".).

Recruitment status

COMPLETED

Target enrollment

74 participants

Primary outcome timeframe

Up to week 12

Results posted on

2026-01-09

Participant Flow

One patient did not receive study treatment due to withdrawal of consent.

Participant milestones

Participant milestones
Measure	Single Arm: Cystistat® 40 mg/50 mL Solution All participants received treatment per standard of care
Overall Study STARTED	73
Overall Study COMPLETED	68
Overall Study NOT COMPLETED	5

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Full analysis set

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Single Arm: Cystistat® 40 mg/50 mL Solution n=73 Participants All participants received treatment per standard of care
Age, Continuous	51.9 Years STANDARD_DEVIATION 17.15 • n=73 Participants
Sex: Female, Male Female	72 Participants n=73 Participants
Sex: Female, Male Male	1 Participants n=73 Participants
Race/Ethnicity, Customized Ethnicity · Caucasian	67 Participants n=73 Participants
Race/Ethnicity, Customized Ethnicity · African	1 Participants n=73 Participants
Race/Ethnicity, Customized Ethnicity · Hispanic	5 Participants n=73 Participants
Region of Enrollment Europe	73 Participants n=73 Participants
Bladder Pain/ Interstitial Cystitis Symptom Score	29.3 score on a scale STANDARD_DEVIATION 4.58 • n=71 Participants • Full analysis set
Visual Analogue Scale (urinary urgency)	75.2 units on a scale STANDARD_DEVIATION 21.70 • n=71 Participants • Full analysis set
Visual Analogue Scale (bladder pain)	74.7 units on a scale STANDARD_DEVIATION 18.70 • n=71 Participants • Full analysis set
Visual Analogue Scale (quality of life)	53.1 units on a scale STANDARD_DEVIATION 28.90 • n=71 Participants • Full analysis set

PRIMARY outcome

Timeframe: Up to week 12

Population: Full analysis set

For the primary analysis, a responder was defined as a participant who had an improvement on the PGA scale (i.e., PGA evaluated as markedly improved, moderately improved, or slightly improved. In case of slightly improved, participants were considered as responder only if the question "Did therapy have a positive effect on your life, and would you undergo this treatment again?" is answered with "Yes".).

Outcome measures

Outcome measures
Measure	Single Arm: Cystistat® 40 mg/50 mL Solution n=71 Participants All participants received treatment per standard of care
Patient Global Assessment (PGA)	90.1 percentage of participants (%) Interval 82.28 to 95.28

SECONDARY outcome

Timeframe: Up to week 12 and additionally at Month 6

Population: Full analysis set

The Bladder Pain/ Interstitial Cystitis Symptom Score (BPIC-SS) is a validated questionnaire for the measurement of IC/BPS symptoms. It contains 8 questions and the total score ranges from 0 to 38, with higher scores indicating a worse situation

Outcome measures

Outcome measures
Measure	Single Arm: Cystistat® 40 mg/50 mL Solution n=71 Participants All participants received treatment per standard of care
Assessment of Bladder Pain/ Interstitial Cystitis Symptom Score End of treatment or latest week 12	-13.8 score on a scale (change from baseline) Standard Deviation 8.62
Assessment of Bladder Pain/ Interstitial Cystitis Symptom Score Month 6	-15.3 score on a scale (change from baseline) Standard Deviation 9.15

SECONDARY outcome

Timeframe: Up to week 12 and additionally at Month 6

Population: Full analysis set

Patients will be asked to assess their bladder pain on the VAS using the question "How was your bladder pain over the last 3 days?". The VAS will be an unmarked line ranging from 0 to 100 mm, where 0 = "no pain" and 100 = "worst possible pain".

Outcome measures

Outcome measures
Measure	Single Arm: Cystistat® 40 mg/50 mL Solution n=71 Participants All participants received treatment per standard of care
Assessment of Visual Analogue Scale (VAS) for Bladder Pain End of treatment or latest week 12	-42.0 units on a scale (change from baseline) Standard Deviation 27.49
Assessment of Visual Analogue Scale (VAS) for Bladder Pain Month 6	-49.2 units on a scale (change from baseline) Standard Deviation 29.69

SECONDARY outcome

Timeframe: Up to week 12 and additionally at Month 6

Population: Full analysis set

Patients will be asked to assess their urinary urgency on the VAS using the question "How intense was urinary urgency over the last 3 days?". The VAS will be an unmarked line ranging from 0 to 100 mm, where 0 = "no urgency" and 100 = "worst possible urgency".

Outcome measures

Outcome measures
Measure	Single Arm: Cystistat® 40 mg/50 mL Solution n=71 Participants All participants received treatment per standard of care
Assessment of Visual Analogue Scale (VAS) for Urinary Urgency. End of treatment or latest week 12	-39.3 units on a scale (change from baseline) Standard Deviation 28.90
Assessment of Visual Analogue Scale (VAS) for Urinary Urgency. Month 6	-49.2 units on a scale (change from baseline) Standard Deviation 30.48

SECONDARY outcome

Timeframe: Up to week 12 and additionally at Month 6

Population: Full anaylsis set

Patients will be asked to assess QoL on the VAS using the question "How good or bad would you rate your overall health condition throughout the last 3 days?". The VAS will be an unmarked line ranging from 0 to 100 mm, where 0 = "the worst health you can imagine" and 100 = "the best health you can imagine".

Outcome measures

Outcome measures
Measure	Single Arm: Cystistat® 40 mg/50 mL Solution n=71 Participants All participants received treatment per standard of care
Assessment of Visual Analogue Scale (VAS) for Quality of Life (QoL) End of treatment or latest week 12	17.1 units on a scale (change from baseline) Standard Deviation 35.84
Assessment of Visual Analogue Scale (VAS) for Quality of Life (QoL) Month 6	27.2 units on a scale (change from baseline) Standard Deviation 33.27

Adverse Events

Single Arm: Cystistat® 40 mg/50 mL Solution

Serious events: 1 serious events

Other events: 13 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Single Arm: Cystistat® 40 mg/50 mL Solution n=73 participants at risk Per standard of care
General disorders pyrexia	1.4% 1/73 • Number of events 1 • 6 months

Other adverse events

Other adverse events
Measure	Single Arm: Cystistat® 40 mg/50 mL Solution n=73 participants at risk Per standard of care
General disorders Catheter Site Pain	2.7% 2/73 • Number of events 2 • 6 months
Gastrointestinal disorders Abdominal Pain Lower	2.7% 2/73 • Number of events 3 • 6 months
Gastrointestinal disorders Diarrhoea	2.7% 2/73 • Number of events 2 • 6 months
General disorders Condition Agravated	4.1% 3/73 • Number of events 3 • 6 months
Infections and infestations Urinary Tract Infection	4.1% 3/73 • Number of events 3 • 6 months
Renal and urinary disorders Dysuria	2.7% 2/73 • Number of events 3 • 6 months
Renal and urinary disorders Renal Colic	2.7% 2/73 • Number of events 3 • 6 months

Additional Information

Christine Kolb

MEDA Pharma GmbH & Co. KG (A Viatris Company)

Phone: +49 6172 888

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60