EndometriosisPhase 1/2NCT01553201
Botulinum Toxin for Pelvic Pain in Women With Endometriosis
A Phase 1/2 study testing Botulinum toxin (BoNT) in people with Endometriosis and Chronic Pelvic Pain, run by National Institute of Neurological Disorders and Stroke (NINDS) at 1 site in United States.
Trial summary
Background: Some women with endometriosis have chronic pelvic pain. This pain may be caused by spasms of the pelvic floor muscles. These spasms can be detected by an examination. Studies suggest that botulinum toxin can help treat problems caused by muscle spasms. Read more about this study
- Who is it for?
- Ages 18 to 55 years, women only
- Conditions the study is for
- Endometriosis, Chronic Pelvic Pain, Pelvic Muscle Spasm
- What is being tested?
- Botulinum toxin (BoNT)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 30 participants
- Where
- 1 siteUnited States
- Study dates
- 30 Jul 2014to 24 Jul 2019
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 55 years
- Women only
- No healthy volunteers
- Adult
Who may be able to join
- Female gender
- Age between 18 and 55
- History of endometriosis at surgery
- Persistent pelvic pain for at least 3 months
- Pelvic floor spasm
- Negative pregnancy test (in women who have not had a hysterectomy)
Show all 9Show fewer
- Willing to use reliable method of contraception for the month after botulinum toxin injection including oral contraceptives, IUD, and barrier with spermicide.
- Willing and able to give informed consent
- Willing and able to comply with study requirements
Who cannot join
- Women with other causes of chronic pelvic pain including infectious, gastrointestinal, psychological disorders, fibromyalgia and chronic fatigue syndrome based on review of medical history within 1 year of first study visit,
- Untreated severe cervical dysplasia or other gynecologic condition clinically significant abnormalities on physical or laboratory examinations that require evaluation or treatment or that would make participation unsafe
- Hysterectomy and bilateral salpingo-oophorectomy
- Pregnancy
- Lactation
- Allergy to albumen or botulinum toxin
Show all 11Show fewer
- Presence of antibodies to botulinum toxin or loss of response to previous injections for any indication
- A known neuromuscular junction disorder such as myasthenia gravis or Eaton-Lambert syndrome
- History of urinary or fecal incontinence
- Known pelvic prolapse
- Eligibility will be based on a physical exam at NIH at the first study visit, as well as history and available medical records within 1 year before the study visit. Negative test results will be documented to confirm study eligibility. If the Pap and the GC and chlamydia tests were not done outside of the NIH within the previous year, or if documentation of the negative test results is not available, we will await the results of the screening tests done at the NIH before administering the study medication.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, 20892
About this study
Official title: The Effectiveness of Botulinum Toxin on Persistent Pelvic Pain in Women With Endometriosis
Background:
- Some women with endometriosis have chronic pelvic pain. This pain may be caused by spasms of the pelvic floor muscles. These spasms can be detected by an examination. Studies suggest that botulinum toxin can help treat problems caused by muscle spasms. Researchers want to see if botulinum toxin injections into the pelvic floor muscles can decrease pain and spasms in women with pelvic pain.
Read more
Objectives:
- To see if botulinum toxin can relieve pain from pelvic floor spasm in women with pelvic pain.
Eligibility:
- Women between 18 and 50 years of age with pain associated with pelvic muscle spasm and endometriosis. Pain must be persistent (lasting for at least 3 months).
Design:
- Participants will keep a pain diary and record their pain medication use for a month before the first visit.
- Participants will be screened with a physical exam and medical history. Blood and urine samples will be collected. Participants will also answer questions about their pain levels and quality of life.
- Participants will receive either botulinum toxin or a placebo (salt water) injection. The injection will be given into the pelvic floor muscles through the vaginal wall. Participants will take a muscle relaxant like Valium and have anesthetic cream put on the vaginal wall before the injection.
- After the injection, participants will keep a pain diary for another month.
- At a 1-month followup visit, participants will answer questions about their pain. If the pain has not improved, all participants may have a botulinum toxin injection (no placebo) into the pelvic floor muscles as before.
- Participants will have followup visits for up to a year after the initial 1-month followup visit.
Read the detailed description
Chronic pelvic pain associated with endometriosis is poorly understood. Some women with chronic pelvic pain have muscle spasm of their pelvic muscles. Muscle spasm may be a significant part of pain in women with endometriosis and other types of chronic pelvic pain. Botulinum toxin injection is widely used to treat conditions associated with excessive muscle activity and spasm. Studies of botulinum toxin injected into pelvic muscles of women with pelvic pain have shown a decrease pain and spasm, but too few women have been studied to conclude its effectiveness. We expect to show that botulinum toxin injection in women with pelvic pain will relieve some of their pelvic pain.
Eligible subjects will be otherwise healthy women who have chronic pelvic pain and a history of endometriosis. Subjects will be randomized to either botulinum toxin injection or placebo (salt water) injection. After one month, we will evaluate the presence of the pain and all women will be offered botulinum toxin injection. We will also evaluate the need for reinjection.
- Being tested
- Botulinum toxin (BoNT), Placebo
- Keywords
- Quality of Life, Endometriosis, Botulinum Toxin A, Pelvic Floor Muscle Spasm, Chronic Pelvic Pain
- Medical subject headings
- Endometriosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Botulinum toxin (BoNT)Drug
OnabotulinumtoxinA 100 Units diluted in 4cc saline, one time intramuscular administration
PlaceboDrug
Saline, 4cc, one time intramuscular administration
| Study group | Role | What they receive |
|---|---|---|
| Botulinum toxin (BoNT) | Experimental | OnabotulinumtoxinA 100 Units diluted in 4cc saline, one time intramuscular administration Receives: Drug: Botulinum toxin (BoNT) |
| Placebo | Placebo comparator | Saline, 4cc, one time intramuscular administration Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Improvement in Pain
Self reported improvement in pain symptoms - a binary measurement of improvement/no improvement
Measured: 1 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Mar 2012 (estimated)
- Study start30 Jul 2014
- Primary completion31 Jul 2018
- Study completion24 Jul 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (6)Papers the sponsor has linked to this study.
- Other trials for EndometriosisStudies of the same condition, open and under way.
- More trials run by National Institute of Neurological Disorders and Stroke (NINDS)The 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Barbara I Karp, M.D., Principal investigator, National Institute of Neurological Disorders and Stroke (NINDS)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01553201
- Sponsor's protocol ID
- 120083
- Other IDs
- 12-N-0083
- Status last verified
- Sep 2018
- First submitted
- 10 Mar 2012
- Last updated
- 28 Jul 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Abbott JA, Jarvis SK, Lyons SD, Thomson A, Vancaille TG. Botulinum toxin type A for chronic pain and pelvic floor spasm in women: a randomized controlled trial. Obstet Gynecol. 2006 Oct;108(4):915-23. doi: 10.1097/01.AOG.0000237100.29870.cc.
PubMed 17012454Bhidayasiri R, Truong DD. Expanding use of botulinum toxin. J Neurol Sci. 2005 Aug 15;235(1-2):1-9. doi: 10.1016/j.jns.2005.04.017.
PubMed 15990116Jankovic J. Botulinum toxin in clinical practice. J Neurol Neurosurg Psychiatry. 2004 Jul;75(7):951-7. doi: 10.1136/jnnp.2003.034702.
PubMed 15201348Karp BI, Tandon HK, Sinaii N, Aredo JV, Phan VT, Salmeri N, Shah JP, Merideth MA, Stratton P. Botulinum toxin for endometriosis-associated chronic pelvic pain: a randomised, double-masked, parallel, phase 2 trial. EClinicalMedicine. 2026 Jan 5;91:103740. doi: 10.1016/j.eclinm.2025.103740. eCollection 2026 Jan.
PubMed 41551998Phan VT, Stratton P, Tandon HK, Sinaii N, Aredo JV, Karp BI, Merideth MA, Shah JP. Widespread myofascial dysfunction and sensitisation in women with endometriosis-associated chronic pelvic pain: A cross-sectional study. Eur J Pain. 2021 Apr;25(4):831-840. doi: 10.1002/ejp.1713. Epub 2021 Jan 8.
PubMed 33326662Tandon HK, Stratton P, Sinaii N, Shah J, Karp BI. Botulinum toxin for chronic pelvic pain in women with endometriosis: a cohort study of a pain-focused treatment. Reg Anesth Pain Med. 2019 Jul 8:rapm-2019-100529. doi: 10.1136/rapm-2019-100529. Online ahead of print.
PubMed 31289238
Study at a glance
From the registry record, last updated by the sponsor on 28 Jul 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Effectiveness of Botulinum Toxin on Persistent Pelvic Pain in Women With Endometriosis
- ClinicalTrials.gov ID
- NCT01553201
- Acronym / sponsor ID
- 120083
- Keywords
- Quality of Life, Endometriosis, Botulinum Toxin A, Pelvic Floor Muscle Spasm, Chronic Pelvic Pain
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)US National Institutes of Health
- Study start
- 30 Jul 2014 (actual)
- Primary completion
- 31 Jul 2018 (actual)
- Study completion
- 24 Jul 2019 (actual)
- First posted
- 14 Mar 2012 (estimated)
- Results
- Posted 8 Jul 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Background:
- Some women with endometriosis have chronic pelvic pain. This pain may be caused by spasms of the pelvic floor muscles. These spasms can be detected by an examination. Studies suggest that botulinum toxin can help treat problems caused by muscle spasms. Researchers want to see if botulinum toxin injections into the pelvic floor muscles can decrease pain and spasms in women with pelvic pain.
Objectives:
- To see if botulinum toxin can relieve pain from pelvic floor spasm in women with pelvic pain.
Eligibility:
- Women between 18 and 50 years of age with pain associated with pelvic muscle spasm and endometriosis. Pain must be persistent (lasting for at least 3 months).
Design:
- Participants will keep a pain diary and record their pain medication use for a month before the first visit.
- Participants will be screened with a physical exam and medical history. Blood and urine samples will be collected. Participants will also answer questions about their pain levels and quality of life.
- Participants will receive either botulinum toxin or a placebo (salt water) injection. The injection will be given into the pelvic floor muscles through the vaginal wall. Participants will take a muscle relaxant like Valium and have anesthetic cream put on the vaginal wall before the injection.
- After the injection, participants will keep a pain diary for another month.
- At a 1-month followup visit, participants will answer questions about their pain. If the pain has not improved, all participants may have a botulinum toxin injection (no placebo) into the pelvic floor muscles as before.
- Participants will have followup visits for up to a year after the initial 1-month followup visit.
Detailed description
Chronic pelvic pain associated with endometriosis is poorly understood. Some women with chronic pelvic pain have muscle spasm of their pelvic muscles. Muscle spasm may be a significant part of pain in women with endometriosis and other types of chronic pelvic pain. Botulinum toxin injection is widely used to treat conditions associated with excessive muscle activity and spasm. Studies of botulinum toxin injected into pelvic muscles of women with pelvic pain have shown a decrease pain and spasm, but too few women have been studied to conclude its effectiveness. We expect to show that botulinum toxin injection in women with pelvic pain will relieve some of their pelvic pain.
Eligible subjects will be otherwise healthy women who have chronic pelvic pain and a history of endometriosis. Subjects will be randomized to either botulinum toxin injection or placebo (salt water) injection. After one month, we will evaluate the presence of the pain and all women will be offered botulinum toxin injection. We will also evaluate the need for reinjection.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Botulinum toxin (BoNT) | Experimental | Drug: Botulinum toxin (BoNT)OnabotulinumtoxinA 100 Units diluted in 4cc saline, one time intramuscular administration |
| Placebo | Placebo comparator | Drug: PlaceboSaline, 4cc, one time intramuscular administration |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (9 listed)
- Female gender
- Age between 18 and 55
- History of endometriosis at surgery
- Persistent pelvic pain for at least 3 months
- Pelvic floor spasm
Key exclusion criteria (11 listed)
- Women with other causes of chronic pelvic pain including infectious, gastrointestinal, psychological disorders, fibromyalgia and chronic fatigue syndrome based on review of medical history within 1 year of first study visit,
- Untreated severe cervical dysplasia or other gynecologic condition clinically significant abnormalities on physical or laboratory examinations that require evaluation or treatment or that would make participation unsafe
- Hysterectomy and bilateral salpingo-oophorectomy
- Pregnancy
- Lactation
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 (BoNT)OnabotulinumtoxinA 100 Units diluted in 4cc saline, one time intramuscular administration | Drug | Botulinum toxin (BoNT) |
| PlaceboSaline, 4cc, one time intramuscular administration | Drug | Placebo |
Primary outcome
Number of Participants With Improvement in Pain
Time frame: 1 month
Self reported improvement in pain symptoms - a binary measurement of improvement/no improvement
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Jul 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 17012454Abbott JA, Jarvis SK, Lyons SD, Thomson A, Vancaille TG. Botulinum toxin type A for chronic pain and pelvic floor spasm in women: a randomized controlled trial. Obstet Gynecol. 2006 Oct;108(4):915-23. doi: 10.1097/01.AOG.0000237100.29870.cc.
- Background: PubMed 15990116Bhidayasiri R, Truong DD. Expanding use of botulinum toxin. J Neurol Sci. 2005 Aug 15;235(1-2):1-9. doi: 10.1016/j.jns.2005.04.017.
- Background: PubMed 15201348Jankovic J. Botulinum toxin in clinical practice. J Neurol Neurosurg Psychiatry. 2004 Jul;75(7):951-7. doi: 10.1136/jnnp.2003.034702.
- Derived from this study: PubMed 41551998Karp BI, Tandon HK, Sinaii N, Aredo JV, Phan VT, Salmeri N, Shah JP, Merideth MA, Stratton P. Botulinum toxin for endometriosis-associated chronic pelvic pain: a randomised, double-masked, parallel, phase 2 trial. EClinicalMedicine. 2026 Jan 5;91:103740. doi: 10.1016/j.eclinm.2025.103740. eCollection 2026 Jan.
- Derived from this study: PubMed 33326662Phan VT, Stratton P, Tandon HK, Sinaii N, Aredo JV, Karp BI, Merideth MA, Shah JP. Widespread myofascial dysfunction and sensitisation in women with endometriosis-associated chronic pelvic pain: A cross-sectional study. Eur J Pain. 2021 Apr;25(4):831-840. doi: 10.1002/ejp.1713. Epub 2021 Jan 8.
- Derived from this study: PubMed 31289238Tandon HK, Stratton P, Sinaii N, Shah J, Karp BI. Botulinum toxin for chronic pelvic pain in women with endometriosis: a cohort study of a pain-focused treatment. Reg Anesth Pain Med. 2019 Jul 8:rapm-2019-100529. doi: 10.1136/rapm-2019-100529. Online ahead of print.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
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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.
Barbara I Karp, M.D. principal investigator
National Institute of Neurological Disorders and Stroke (NINDS)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Botulinum toxin (BoNT) in people with Endometriosis and Chronic Pelvic Pain, run by National Institute of Neurological Disorders and Stroke (NINDS) at 1 site in United States.
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 9 inclusion and 11 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 28 Jul 2020. 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 United States. 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 8 Jul 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Number of Participants With Improvement in Pain (time frame: 1 month). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01553201, which also lists 6 linked publications.
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 28 Jul 2020.