Botulinum Toxin for Pelvic Pain in Women With Endometriosis

NCT01553201 | Completed Phase 1 Results DMC FDA Drug

• 2 other identifiers: 12008312-N-0083
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

Endometriosis; Chronic Pelvic Pain; Pelvic Muscle Spasm; Quality of Life

Keywords

Quality of Life; Endometriosis; Botulinum Toxin A; Pelvic Floor Muscle Spasm; Chronic Pelvic Pain

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Improvement in Pain

  Self reported improvement in pain symptoms - a binary measurement of improvement/no improvement

  1 month

Study Arms (2)

Botulinum toxin (BoNT)

EXPERIMENTAL

OnabotulinumtoxinA 100 Units diluted in 4cc saline, one time intramuscular administration

Drug: Botulinum toxin (BoNT)

Placebo

PLACEBO COMPARATOR

Saline, 4cc, one time intramuscular administration

Drug: Placebo

Interventions

Botulinum toxin (BoNT) DRUG

OnabotulinumtoxinA 100 Units diluted in 4cc saline, one time intramuscular administration

Botulinum toxin (BoNT)

Placebo DRUG

Saline, 4cc, one time intramuscular administration

Placebo

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: female
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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)
• 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

You may not qualify if:

• 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
• 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.

Sponsors & Collaborators

• National Institute of Neurological Disorders and Stroke (NINDS): lead

Study Sites (1)

National Institutes of Health Clinical Center, 9000 Rockville Pike

Bethesda, Maryland, 20892, United States

Location

Related Publications (6)

• 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.

  PMID: 17012454 BACKGROUND

• Bhidayasiri 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.

  PMID: 15990116 BACKGROUND

• Jankovic J. Botulinum toxin in clinical practice. J Neurol Neurosurg Psychiatry. 2004 Jul;75(7):951-7. doi: 10.1136/jnnp.2003.034702.

  PMID: 15201348 BACKGROUND

• Karp 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.

  PMID: 41551998 DERIVED

• Phan 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.

  PMID: 33326662 DERIVED

• Tandon 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.

  PMID: 31289238 DERIVED

Related Links

• NIH Clinical Center Detailed Web Page

MeSH Terms

Conditions

Endometriosis

Interventions

Botulinum Toxins

Condition Hierarchy (Ancestors)

Genital Diseases, Female; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Genital Diseases

Intervention Hierarchy (Ancestors)

Metalloendopeptidases; Endopeptidases; Peptide Hydrolases; Hydrolases; Enzymes; Enzymes and Coenzymes; Metalloproteases; Bacterial Proteins; Proteins; Amino Acids, Peptides, and Proteins; Bacterial Toxins; Toxins, Biological; Biological Factors

Results Point of Contact

• Title: Barbara Karp, MD
• Organization: National Institute of Neurological Disorders & Stroke

karpb@ninds.nih.gov

301-496-0150

Study Officials

• Barbara I Karp, M.D.

  National Institute of Neurological Disorders and Stroke (NINDS)

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 10, 2012
• First Posted: March 14, 2012
• Study Start: July 30, 2014
• Primary Completion: July 31, 2018
• Study Completion: July 24, 2019
• Last Updated: July 28, 2020
• Results First Posted: July 8, 2020

Record last verified: 2018-09

Locations

US (1)