VulvodyniaPhase 3NCT04057755
Botulinum Toxin A as a Treatment for Provoked Vestibulodynia
A Phase 3 study testing botulinum toxin A and NaCl in people with Vulvodynia, run by Karolinska Institutet at 1 site in Sweden.
Trial summary
Women with provoked vestibulodynia (PVD) suffer from severe dyspareuni and often present a hyperactivity of the pelvic floor muscles (PFM) which maintain the dyspareunia. The rationale for the study is that for women with PVD who don't succeed to restore the function of the PFM by physiotherapy, Botulinum Toxin A… Read more about this study
- Who is it for?
- Ages 18 to 40 years, women only
- Conditions the study is for
- Vulvodynia
- What is being tested?
- botulinum toxin A and NaCl
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 90 participants
- Where
- 1 siteSweden
- Study dates
- 15 May 2016to 15 Jun 2019
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 40 years
- Women only
- No healthy volunteers
- Adult
Who may be able to join
- Age 18-40 years
- PVD defined as significant pain at vestibular contact and vaginal entry
- VAS ≥ 6 at the tampon test or severe pain, preventing intercourse
- Duration of symptoms of ≥ 3 months
- 0-para
- Patients who are willing to participate in the study after it has been explained orally and in writing will be included.
Who cannot join
- Vulvo-vaginal infection
- Dermatological conditions or other causes to dyspareunia
- Regular medication with analgesics
- Major psychiatric or medical disease
- Known allergy to any components of the active drug (Botox®)
- Medical disease contradictory to treatment with the active drug (Botox®)
Show all 10Show fewer
- Peripheral motor neurological disease such as myasthenia gravis, amyotrophic lateral sclerosis or Lambert-Eaton syndrome
- Diabetes)
- Pregnancy
- Pelvic floor deficiency with urine and or flatulence incontinence.
Trial locations and contacts
1 site in 1 country.
Sweden1 site
Danderyd Hospital, Dep. of Obstetrics and Gynecology
Stockholm, 182 88
About this study
Official title: Double Blind Placebo-controlled RCT of the Efficacy and Safety of Intramuscular Injections of Botulinum Toxin A as a Treatment for Provoked Vestibulodynia
Women with provoked vestibulodynia (PVD) suffer from severe dyspareuni and often present a hyperactivity of the pelvic floor muscles (PFM) which maintain the dyspareunia. The rationale for the study is that for women with PVD who don't succeed to restore the function of the PFM by physiotherapy, Botulinum Toxin A (BTA) could be an optional treatment by decreasing the high muscle tonus and thus possibly reduce the coital pain.
Objectives and outcome Women with PVD will be recruited for a double blind RCT of 2 injection of 50 Allergan-units BTA (3 months apart) or placebo in the bulbocavernous muscles (situated adjacent to the lower part of the vagina).
Read more
Primary outcome: The reduction of patient self-reported dyspareunia measured by VAS 0 (no pain) to 100 (worst pain imaginable).
Secondary outcome: Pain at tampon insertion measured by VAS 0-100, functional measurement of dyspareunia (see below), the reduction of pelvic floor hyperactivity/tonus, measured with a vaginal manometer, safety aspects and effect duration of BTA, influence on quality of life and psychosexual evaluation.
Read the detailed description
Rationale Dyspareunia is a common pain problem among women. The prevalence has been estimated to be 10-15%. The most common type of dyspareunia among premenopausal women is provoked vestibulodynia (PVD). PVD is characterized by pain upon touch, pressure and stretch of the vestibular tissue in spite of the absence of other gynecological or dermatological disease [4]. The pain and its associated sexual consequences have a severe negative impact on the quality of life of affected women. Currently the etiology, although still not completely clarified, is considered to be multi-factorial involving biomedical and psychosexual causes. Two sub-categories of PVD has been identified; primary PVD, where pain occurs at the first attempt of vaginal entry (intercourse or tampon use) and secondary PVD, where pain occurs after a period of normal functioning. There is evidence of patho-physiological changes in three interdependent systems; the vestibular tissue, the pelvic floor muscles and the pain regulatory pathways of the central nervous system. Signs of a neurogenic inflammation in the vestibular mucosa, with neural hyperplasia of CGRP and Substance P positive C-fibers have been found. Furthermore, recent evidence supports the importance of a pelvic floor muscle (PFM) dysfunction to the etiology of PVD. Women with PVD have been shown to have elevated resting activity, lower maximal strength and poorer control of the PFM compared to healthy controls. Evidence suggests that this hyperactivity, although possibly originating as a protective defense mechanism provoked by pain, is chronic and thus contributes to maintaining and exacerbating the neurogenic inflammation and pain. A circular model has been suggested in which pain during intercourse and fear of pain may decrease sexual arousal and increase PFM tonus, whereby the PFM hyperactivity might act as an initiator of vestibular sensory changes and inflammation. However there is a lack of longitudinal studies to answer the question whether the PFM dysfunction is antecedent to the pain or a result of the pain.
Gentilcore-Saulnier et al. proposed that superficial and deep layers of the PFM may differ in their involvement in PVD as assessed with EMG external surface electrodes and an intravaginal probe, respectively. They found that women with PVD have significantly higher resting activity in the superficial muscle (bulbocaverneous) in comparison with controls. The difference was not significant for the deep layer (puborectalis, pubococcygeus, ileococcygeus and ischiococcygeus muscles).
The treatment guidelines today recommend a multi-modal treatment including topical anesthetic agents, cognitive behavioral therapy and PFM rehabilitation based on physiotherapy. As a second line treatment injections with botulinum toxin A (BTA) in the bulbocavernous muscles bilaterally has been suggested and to a limited extent tested. The main target for BTA is a transient paretic effect on skeletal muscular fibers and it also blocks the release of neuropeptides and neurotransmitters involved in the neuropathic pain and could therefore have additional effect in the treatment of PVD. Previously published reports on the effects of BTA for PVD are few and the methods of injection (different injection sites, use or non-use of an EMG needle for direction of injection sites) and doses used (20, 35, 100 IU) differ as well as methods of measuring treatment outcome. Only one double blind RCT has been published so far where no additional effect of BTA compared to saline could be detected, however the BTA dose used was low (20 IU) and only one treatment was performed. Using BTA in the PFM seems to be safe and only tenderness at the injection site and mild influenza like symptoms have been reported side effects so far.
Hypothesis Our hypothesis is that two treatments (three months apart) of injections with 50 Allergan-units of BTA in the bulbocavernosus muscles in women with PVD will reduce the hyperactivity in the PFM and thus significantly decrease the pain during intercourse.
- Conditions
- Vulvodynia
- Being tested
- botulinum toxin A, NaCl
- Keywords
- botulinum toxin A, treatment
- Medical subject headings
- Vulvodynia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
botulinum toxin ADrug
Also called Botox
NaClDrug
Also called Saline
| Study group | Role | What they receive |
|---|---|---|
| Botulinum toxin A vs placebo | Experimental | 50 Allergan units (diluted in 0,5 ml sterile NaCl) or 0,5 ml of sterile NaCl will be injected in the bulbocavernosus muscle just inside the hymenal ring. The injection with the same substance, dose and volume will be repeated after 3 months. Receives: Drug: botulinum toxin A; Drug: NaCl |
| Botulinum toxin A | Active comparator | 50 Allergan units (diluted in 0,5 ml sterile NaCl) will be injected in the bulbocavernosus muscle just inside the hymenal ring. The injection with the same substance, dose and volume will be repeated after 3 months. Receives: Drug: botulinum toxin A |
| NaCl | Placebo comparator | 0,5 ml of sterile NaCl will be injected in the bulbocavernosus muscle just inside the hymenal ring. The injection with the same substance and volume will be repeated after 3 months. Receives: Drug: NaCl |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in self-reported dyspareunia last month measured by VAS 0-100
VAS 0 (no pain) to 100 (worst pain imaginable).
Measured: At baseline up to 6 months
Secondary outcomes (9)
Change in pain at tampon insertion last week, measured by VAS 0-100
VAS 0 (no pain) to 100 (worst pain imaginable).
Measured: At baseline and up to 12 months
Change in pelvic floor hyperactivity/tonus,
Measured with a vaginal manometer in mmHg
Measured: At baseline and up to 12 months
Safety aspects regarding adverse events of BTA
Monitoring possible adverse events
Measured: The complete study, 12 months
Change in quality of Life (WHOQOL-BREF)
The validated questionnaires WHO Quality of Life-BREF (WHOQOL-BREF) will be used
Measured: At baseline and up to 12 months
Change in quality of Life (EQ5D)
Health-related quality of life as assessed using the EuroQOL five dimensions
Measured: At baseline and up to 12 months
Change in sexual function
The validated questionnaire Female Sexual Function Index (FSFI) will be used
Measured: At baseline and up to 12 months
Change in sexual distress
The validated questionnaire Female Sexual Distress Scale (FSDS) will be used
Measured: At baseline and up to 12 months
Change in level of stress
The validated questionnaire PSS (Percieved stress scale) will be used
Measured: At baseline and up to 12 months
Change in level of anxiety
A validated questionnaire Adult Anxiety Scale will be used,
Measured: At baseline and up to 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Aug 2019
- Study start15 May 2016
- Primary completion15 Jun 2019
- Study completion15 Jun 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for VulvodyniaStudies of the same condition, open and under way.
- More trials run by Karolinska InstitutetThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 90 participants (actual)
Sponsor and investigators
- Lead sponsor
- Karolinska Institutet
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Nina Bohm-Starke, Karolinska Institutet
- Investigators
- Nina Bohm-Starke, MD, Principal investigator, Karolinska Institutet
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04057755
- Sponsor's protocol ID
- 2016-000375-25
- Other IDs
- 2016
- Status last verified
- Aug 2019
- First submitted
- 6 Mar 2018
- Last updated
- 15 Aug 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Aug 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Double Blind Placebo-controlled RCT of the Efficacy and Safety of Intramuscular Injections of Botulinum Toxin A as a Treatment for Provoked Vestibulodynia
- ClinicalTrials.gov ID
- NCT04057755
- Acronym / sponsor ID
- 2016-000375-25
- Conditions
- Vulvodynia
- Keywords
- botulinum toxin A, treatment
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 90 participants (actual)
- Lead sponsor
- Karolinska InstitutetUniversity, hospital or other
- Study start
- 15 May 2016 (actual)
- Primary completion
- 15 Jun 2019 (actual)
- Study completion
- 15 Jun 2019 (actual)
- First posted
- 15 Aug 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Women with provoked vestibulodynia (PVD) suffer from severe dyspareuni and often present a hyperactivity of the pelvic floor muscles (PFM) which maintain the dyspareunia. The rationale for the study is that for women with PVD who don't succeed to restore the function of the PFM by physiotherapy, Botulinum Toxin A (BTA) could be an optional treatment by decreasing the high muscle tonus and thus possibly reduce the coital pain.
Objectives and outcome Women with PVD will be recruited for a double blind RCT of 2 injection of 50 Allergan-units BTA (3 months apart) or placebo in the bulbocavernous muscles (situated adjacent to the lower part of the vagina).
Primary outcome: The reduction of patient self-reported dyspareunia measured by VAS 0 (no pain) to 100 (worst pain imaginable).
Secondary outcome: Pain at tampon insertion measured by VAS 0-100, functional measurement of dyspareunia (see below), the reduction of pelvic floor hyperactivity/tonus, measured with a vaginal manometer, safety aspects and effect duration of BTA, influence on quality of life and psychosexual evaluation.
Detailed description
Rationale Dyspareunia is a common pain problem among women. The prevalence has been estimated to be 10-15%. The most common type of dyspareunia among premenopausal women is provoked vestibulodynia (PVD). PVD is characterized by pain upon touch, pressure and stretch of the vestibular tissue in spite of the absence of other gynecological or dermatological disease [4]. The pain and its associated sexual consequences have a severe negative impact on the quality of life of affected women. Currently the etiology, although still not completely clarified, is considered to be multi-factorial involving biomedical and psychosexual causes. Two sub-categories of PVD has been identified; primary PVD, where pain occurs at the first attempt of vaginal entry (intercourse or tampon use) and secondary PVD, where pain occurs after a period of normal functioning. There is evidence of patho-physiological changes in three interdependent systems; the vestibular tissue, the pelvic floor muscles and the pain regulatory pathways of the central nervous system. Signs of a neurogenic inflammation in the vestibular mucosa, with neural hyperplasia of CGRP and Substance P positive C-fibers have been found. Furthermore, recent evidence supports the importance of a pelvic floor muscle (PFM) dysfunction to the etiology of PVD. Women with PVD have been shown to have elevated resting activity, lower maximal strength and poorer control of the PFM compared to healthy controls. Evidence suggests that this hyperactivity, although possibly originating as a protective defense mechanism provoked by pain, is chronic and thus contributes to maintaining and exacerbating the neurogenic inflammation and pain. A circular model has been suggested in which pain during intercourse and fear of pain may decrease sexual arousal and increase PFM tonus, whereby the PFM hyperactivity might act as an initiator of vestibular sensory changes and inflammation. However there is a lack of longitudinal studies to answer the question whether the PFM dysfunction is antecedent to the pain or a result of the pain.
Gentilcore-Saulnier et al. proposed that superficial and deep layers of the PFM may differ in their involvement in PVD as assessed with EMG external surface electrodes and an intravaginal probe, respectively. They found that women with PVD have significantly higher resting activity in the superficial muscle (bulbocaverneous) in comparison with controls. The difference was not significant for the deep layer (puborectalis, pubococcygeus, ileococcygeus and ischiococcygeus muscles).
The treatment guidelines today recommend a multi-modal treatment including topical anesthetic agents, cognitive behavioral therapy and PFM rehabilitation based on physiotherapy. As a second line treatment injections with botulinum toxin A (BTA) in the bulbocavernous muscles bilaterally has been suggested and to a limited extent tested. The main target for BTA is a transient paretic effect on skeletal muscular fibers and it also blocks the release of neuropeptides and neurotransmitters involved in the neuropathic pain and could therefore have additional effect in the treatment of PVD. Previously published reports on the effects of BTA for PVD are few and the methods of injection (different injection sites, use or non-use of an EMG needle for direction of injection sites) and doses used (20, 35, 100 IU) differ as well as methods of measuring treatment outcome. Only one double blind RCT has been published so far where no additional effect of BTA compared to saline could be detected, however the BTA dose used was low (20 IU) and only one treatment was performed. Using BTA in the PFM seems to be safe and only tenderness at the injection site and mild influenza like symptoms have been reported side effects so far.
Hypothesis Our hypothesis is that two treatments (three months apart) of injections with 50 Allergan-units of BTA in the bulbocavernosus muscles in women with PVD will reduce the hyperactivity in the PFM and thus significantly decrease the pain during intercourse.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Botulinum toxin A vs placebo | Experimental | Drug: botulinum toxin A, Drug: NaCl50 Allergan units (diluted in 0,5 ml sterile NaCl) or 0,5 ml of sterile NaCl will be injected in the bulbocavernosus muscle just inside the hymenal ring. The injection with the same substance, dose and volume will be repeated after 3 months. |
| Botulinum toxin A | Active comparator | Drug: botulinum toxin A50 Allergan units (diluted in 0,5 ml sterile NaCl) will be injected in the bulbocavernosus muscle just inside the hymenal ring. The injection with the same substance, dose and volume will be repeated after 3 months. |
| NaCl | Placebo comparator | Drug: NaCl0,5 ml of sterile NaCl will be injected in the bulbocavernosus muscle just inside the hymenal ring. The injection with the same substance and volume will be repeated after 3 months. |
Who may qualify
- Ages
- 18 to 40 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (6 listed)
- Age 18-40 years
- PVD defined as significant pain at vestibular contact and vaginal entry
- VAS ≥ 6 at the tampon test or severe pain, preventing intercourse
- Duration of symptoms of ≥ 3 months
- 0-para
Key exclusion criteria (10 listed)
- Vulvo-vaginal infection
- Dermatological conditions or other causes to dyspareunia
- Regular medication with analgesics
- Major psychiatric or medical disease
- Known allergy to any components of the active drug (Botox®)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| botulinum toxin AAlso: Botox | Drug | Botulinum toxin A, Botulinum toxin A vs placebo |
| NaClAlso: Saline | Drug | Botulinum toxin A vs placebo, NaCl |
Primary outcome
Change in self-reported dyspareunia last month measured by VAS 0-100
Time frame: At baseline up to 6 months
VAS 0 (no pain) to 100 (worst pain imaginable).
9 secondary outcomes
Change in pain at tampon insertion last week, measured by VAS 0-100
Time frame: At baseline and up to 12 months
Change in pelvic floor hyperactivity/tonus,
Time frame: At baseline and up to 12 months
Safety aspects regarding adverse events of BTA
Time frame: The complete study, 12 months
Change in quality of Life (WHOQOL-BREF)
Time frame: At baseline and up to 12 months
Change in quality of Life (EQ5D)
Time frame: At baseline and up to 12 months
Change in sexual function
Time frame: At baseline and up to 12 months
Change in sexual distress
Time frame: At baseline and up to 12 months
Change in level of stress
Time frame: At baseline and up to 12 months
Change in level of anxiety
Time frame: At baseline and up to 12 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Aug 2019.
- Individual participant dataSharing planned: No
Related trials for Vulvodynia
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Transcutaneous Genital Nerves Stimulation for the Treatment of Refractory Chronic Pelvic and Genital Pain in Women.
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A Pilot Study on the Efficacy of Oral Low Dose Naltrexone for Vulvodynia
Vulvodynia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Nina Bohm-Starke, MD principal investigator
Karolinska Institutet
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing botulinum toxin A and NaCl in people with Vulvodynia, run by Karolinska Institutet at 1 site in Sweden.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 15 Aug 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Sweden. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in self-reported dyspareunia last month measured by VAS 0-100 (time frame: At baseline up to 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Aug 2019.