Botulinumtoxin type A for treatment of Chronic Pelvic Pain Syndrome (CPPS): a phase 3 randomized, double blind, placebo-controlled, multi-center study

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Ospedale San Raffaele S.r.l.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Phenomena and Processes [G] - Reproductive and Urinary Physiological Phenomena [G08], Analytical, Diagnostic and Therapeutic Techniques and Equipment [E] - Therapeutics [E02]

Decision date (initial)

2025-06-17

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Therapy, Efficacy

This is a phase 3, randomized, double blind, placebo-controlled, multi-center study aimed to establish the efficacy of botulinumtoxin-A (BoNT/A) injections in pelvic floor muscles in reducing pain in patients with chronic pelvic pain syndrome (CPPS).

Secondary objectives 1

1. The study aims to assess the treatment safety and the effects of treatment on urinary, bowel and sexual function, as well as on the quality of life.

Conditions and MedDRA coding

Adult patients, male or female, with chronic pelvic pain syndrome (CPPS).

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. age ≥ 18 years
2. Subject able to express consent to participate in the study
3. CPPS diagnosis according to the European Association of Urology (EAU) guidelines
4. mean of the worst daily pain intensity ≥4 in a PI-NRS for a period of one week within the month preceding V1
5. Failure of at least one intervention for chronic pelvic pain (CPP) according to the current best clinical practice

Exclusion criteria 13

1. Pregnant or breast-feeding women
2. Concomitant oral drugs that could interfere with botulinum toxin action, such as aminoglycosides, baclofen or diazepam
3. Presence of ongoing pelvic pathology
4. Presence of neurological disorders
5. Previous or actual major depressive disorder
6. Bleeding disorders or current anticoagulant medications
7. Previous surgical procedures or trauma on pelvic organs
8. Previous or current chemotherapy or radiotherapy on pelvic organs
9. Urinary or fecal incontinence within 3 months prior to screening
10. Raised post void residual >150 ml at any time in the 6 months prior to screening
11. History of botulinumtoxin injections in the previous 6 months
12. Recognized hypersensitivity to botulinumtoxin or to any component of toxin formulation or known botulinumtoxin resistance
13. Infection at the proposed injection sites

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Proportion of patients considered to be responders (defined as ≥ 30% reduction in pain intensity compared to the baseline) at week 4 in the BoNT/A-treated group versus the placebo group. Pain intensity is rated with Pain Intensity Numeric Rating Scale (PI-NRS) on a 7-day pain diary (mean of the worst daily pain intensity over the 7 preceding days) compared to a 7-day baseline period

Secondary endpoints 7

1. Proportion of responders in the BoNT/A-treated group versus the placebo group at other scheduled assessments (i.e. week 2, 6, 8, 10, 12, 14, 16, 18, 20, 22, 24)
2. Change (%) in amount of rescue medication taken, evaluated on a 7-day diary filled-out the week before each scheduled visits (i.e. week 4, 8, 12, 16, 20, 24) compared to the baseline
3. Change in DN4 questionnaire and in Pelvic Floor Muscles Hyperalgesia (PFMH) score at each scheduled visits (i.e. week 4, 8, 12, 16, 20, 24) compared to the baseline
4. Incidence of adverse events (AEs) (total AEs, new/or worsened urinary incontinence, new/or worsened urinary retention, new/or worsened fecal incontinence, new/or worsened constipation, other AEs) (at each follow-up visit and phone interview)
5. Severity of AEs (mild, moderate, severe) (at each follow-up visit and phone interview)
6. Change in International Prostate Symptom Score (IPSS), Wexner score, Female Sexual Distress Scale (FSDS) and International Index of Erectile Function (IIEF) score at scheduled visits (i.e. week 4, 8, 12, 16, 20, 24) compared to the baseline
7. Change in Short Form 12 Health Survey (SF-12) score at scheduled visits (i.e. week 4, 8, 12, 16, 20, 24) compared to the baseline

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Ospedale San Raffaele S.r.l.

Sponsor organisation

Ospedale San Raffaele S.r.l.

Address

Via Olgettina 60

City

Milan

Postcode

20132

Country

Italy

Scientific contact point

Organisation

Ospedale San Raffaele S.r.l.

Contact name

Ubaldo Del Carro

Public contact point

Organisation

Ospedale San Raffaele S.r.l.

Contact name

Ubaldo Del Carro

Locations

1 EU/EEA country · 11 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 9 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications