Dysport in Vulvodynia Phase II Study

NCT03598777 | Terminated Phase 2 Results DMC FDA Drug

• 1 other identifier: D-FR-52120-236
• Study Type: interventional
• Target: 60
• Locations: 2 countries
• Sites: 9

Brief Summary

This study is designed to define optimal doses of Dysport and evaluate its efficacy and safety compared with placebo for the treatment of vulvodynia. The study will consist of a dose escalation stage (Stage 1) and a dose expansion stage (Stage 2). Both Stage 1 and Stage 2 will consist of a double-blind period (with treatment cycle 1; Dysport or placebo) followed by an open label treatment period. One or two optimally safe and effective doses of Dysport selected from Stage 1 will be further investigated in the Stage 2.

Conditions

Vulvodynia

Outcome Measures

Primary Outcomes (2)

• Number of Participants With Treatment-emergent Adverse Events (TEAEs) During the DB Treatment Period (Stage 1)

  For Stage 1, the primary endpoint was safety during the DB treatment period as assessed by the incidence of adverse events (AEs). An AE was the development of an undesirable medical condition or the deterioration of a pre-existing medical condition following or during exposure to a pharmaceutical product, whether or not considered causally related to the product. An undesirable medical condition could have been symptoms, signs or abnormal results of an investigation. A TEAE was an event with start date on or after the date of the first investigational medicinal product (IMP). Relatedness to treatment was assessed by the investigator. TEAEs of special interest included events suggesting a possible remote spread of effect of the toxin, events related to urinary incontinence or faecal incontinence and events assessed as a potential hypersensitivity reaction. Results for this outcome are reported as the number of participants experiencing at least one TEAE in each specified category.

  From Baseline (Cycle 1 Day 1) to Cycle 1 Week 12 (DB treatment period, Stage 1)

• Mean Change From Baseline in Vaginal Dilator Induced Pain During the DB Treatment Period at Week 6 (Stage 2)

  Baseline (Cycle 1 Day 1) and Cycle 1 Week 6 (DB treatment period, Stage 2)

Secondary Outcomes (13)

• Mean Change From Baseline in Vaginal Dilator Induced Pain as Reported on an 11-point Numeric Rating Scale (NRS) During the DB Treatment Period at Week 6 and Week 12 (Stage 1)

  Baseline (Cycle 1 Day 1) and Cycle 1 Week 6 and Week 12 (DB treatment period, Stage 1)

• Number of Participants Who Reported at Least a 50% Decrease From Baseline in Vaginal Dilator Induced Pain as Reported on an 11-point NRS During the DB Treatment Period at Week 6 and Week 12 (Stage 1)

  Baseline (Cycle 1 Day 1) and Cycle 1 Week 6 and Week 12 (DB treatment period, Stage 1)

• Number of Participants Who Reported at Least a 30% Decrease From Baseline in Vaginal Dilator Induced Pain as Reported on an 11-point NRS During the DB Treatment Period at Week 6 and Week 12 (Stage 1)

  Baseline (Cycle 1 Day 1) and Cycle 1 Week 6 and Week 12 (DB treatment period, Stage 1)

• Number of Participants Who Reported at Least a 2-point Decrease From Baseline in Vaginal Dilator Induced Pain as Reported on an 11-point NRS During the DB Treatment Period at Week 6 and Week 12 (Stage 1)

  Baseline (Cycle 1 Day 1) and Cycle 1 Week 6 and Week 12 (DB treatment period, Stage 1)

• Mean Change From Baseline in the DTMS During the DB Treatment Period at Week 6 and Week 12 (Stage 1)

  Baseline (Cycle 1 Day 1) and Cycle 1 Week 6 and Week 12 (DB treatment period, Stage 1)

Study Arms (3)

Dysport - Dose Escalation stage 1

EXPERIMENTAL

Intramuscular injection of Dysport on day 1 of each cycle.

Biological: Botulinum toxin type A

Placebo - Dose Escalation stage 1 and Dose Expansion stage 2

PLACEBO COMPARATOR

Intramuscular injection on day 1 of cycle 1.

Drug: Placebo

Dysport - Dose Expansion stage 2

ACTIVE COMPARATOR

Depending upon the results from Stage 1 one or two doses of Dysport will be selected. Intramuscular injection of Dysport on day 1 of each cycle.

Biological: Botulinum toxin type A

Interventions

Botulinum toxin type A BIOLOGICAL

Botulinum Toxin Type A (Dysport) using a vial of 500 U will be injected intramuscularly across pelvic floor muscles.

Also known as: AbobotulinumtoxinA (Dysport®)

Dysport - Dose Escalation stage 1; Dysport - Dose Expansion stage 2

Placebo DRUG

The reconstituted solution will be injected intramuscularly across pelvic floor muscles.

Placebo - Dose Escalation stage 1 and Dose Expansion stage 2

Eligibility Criteria

• Age: 18 Years+
• Sex: female
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Premenopausal
• Have vulvodynia for at least 6 months and for no more than 15 years
• Have provoked pain at the vestibule on a Q tip test

You may not qualify if:

• Deep pain during intercourse
• Have genitourinary or gastrointestinal conditions which may interfere with the study
• Previous surgery that according to investigator's judgement may impact on study outcome (including but not limited to hysterectomy, vestibulectomy, urologic surgery, perianal surgery) or genital trauma or mutilation/cutting

Sponsors & Collaborators

• Ipsen: lead

Study Sites (9)

James A. Simon, MD, PC

Washington D.C., District of Columbia, 20036, United States

Location

The Center for Vulvovaginal Disorders

Washington D.C., District of Columbia, 20037, United States

Location

New Age Medical Research Corporation

Miami, Florida, 33186, United States

Location

University of Kansas Medical Center

Kansas City, Missouri, 66160, United States

Location

Omaha OB-GYN Associates, PC

Omaha, Nebraska, 68130, United States

Location

The Center for Vulvovaginal Disorders

New York, New York, 10036, United States

Location

Women's Institute for Sexual Health (WISH)

Nashville, Tennessee, 37209, United States

Location

Seattle Women's: Health, Research, Gynecology®

Seattle, Washington, 98105, United States

Location

Clinique de Santé des Femmes

Québec, G15 2L6, Canada

Location

Related Publications (1)

• Goldstein A, Rubin R, Dahir M, Goldstein I, Faught BM, Bohm-Starke N, Krapf J, Caetano P, Volteau M, Silva R. Phase 2 randomized study of abobotulinumtoxinA in patients with provoked vestibulodynia: dose-finding results. J Sex Med. 2025 Apr 15;22(4):588-596. doi: 10.1093/jsxmed/qdaf022.

  PMID: 39953376 DERIVED

MeSH Terms

Conditions

Vulvodynia

Interventions

Botulinum Toxins, Type A; abobotulinumtoxinA

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

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

Limitations and Caveats

After all participants completed Week 12, a Data Review Committee (DRC) recommended: "No safety concerns noted in review of all available data. Efficacy cannot be assessed due to the small sample size of cohorts. The DRC would recommend that Stage 1 is not used to make an efficacy assessment; therefore, there is no justification to increase the dose, nor to move to Stage 2." Thus, sponsor terminated the study early; the decision was not related to any safety/tolerability concerns with Dysport.

Results Point of Contact

• Title: Medical Director
• Organization: Ipsen

clinical.trials@ipsen.com

see email

Study Officials

• Ipsen Medical Director

  Ipsen

  STUDY DIRECTOR

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 8, 2018
• First Posted: July 26, 2018
• Study Start: June 11, 2018
• Primary Completion: January 21, 2021
• Study Completion: January 21, 2021
• Last Updated: December 9, 2021
• Results First Posted: December 9, 2021

Record last verified: 2021-10

Locations

US (8); CA (1)