A phase IIb, randomized, double-blind, multicenter study evaluating the efficacy of botulinum toxin versus placebo on pain and health related quality of life of patients with piriformis muscle syndrome

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Centre Hospitalier Universitaire De Nimes

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Musculoskeletal Diseases [C05]

Decision date (initial)

2025-08-14

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Funding sources

DGOS

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy

To evaluate on an exploratory basis, the efficacy of a single-dose injection of botulinum toxin (Toxin group), versus placebo (placebo group) performed under ultrasound (with electromyographic confirmation) or CT guidance in reducing sciatic pain intensity at 6 weeks in patients with piriformis muscle syndrome (PMS).

Secondary objectives 5

1. To demonstrate the efficacy of botulinum toxin injection versus placebo in reducing buttock pain intensity at 6 weeks.
2. To demonstrate the efficacy of botulinum toxin injection in improving HR-QoL as assessed by the EQ-5D self-report questionnaire at 6 weeks.
3. To analyze, over a 6-month follow-up period, the effect of botulinum toxin injection versus placebo on: o Variations from baseline in sciatic and buttock pain intensity o HR-QoL as assessed by the EQ-5D self-report questionnaire o Overall symptom measures, assessed using the Brief Pain Inventory Short Form (BPI) and Patient Global Impression of Improvement (PGI-I) o Anxiety and depression measured using the Hospital Anxiety and Depression Scale (HADS) o Disability, assessed using the Oswestry Disability Index (ODI) o Variations from baseline in tolerance of the sitting position o Consumption of analgesics, nonsteroidal anti-inflammatory drugs and muscle relaxants
4. To compare the number of patients requiring a second injection (botulinum toxin) at 12 weeks in both arms.
5. To evaluate safety and side effects in both arms.

Conditions and MedDRA coding

Piriformis muscle syndrome

Study design 1 period

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 8

1. Adult patients (age >or =18 years) with objective clinical diagnosis of piriformis muscle syndrome.
2. Objective clinical diagnosis of unilateral piriformis syndrome for at least 3 months (as assessed by Clinical Scoring System for the Diagnosis of Piriformis Muscle Syndrome: score of 8 or greater
3. Absence of herniated lumbar disc which can explain radiating pain (MRI or computed tomography (CT) of the lumbar spine)
4. Patients not responding to conventional care (physiotherapy, muscle relaxants, analgesics)
5. Baseline (at D0) sciatic pain intensity of at least 4 points on visual analog scale (VAS)
6. Adult patients aged over 18 and under 80 years
7. Provision of written informed consent.
8. Patients affiliated to social security system (health insurance coverage).

Exclusion criteria 14

1. Bilateral piriformis muscle syndrome.
2. History of piriformis syndrome surgery.
3. History of botulinum toxin administration.
4. Any treatment (general or local) likely to interfere with botulinum toxin or evaluation of the primary endpoint (Corticosteroids, aminoglycosides).
5. Corticosteroids in the past 3 weeks.
6. Signs of severe fibrosis (on MRI or CT) of the piriformis muscle.
7. Other causes of sciatic pain (lumbar root compression, inflammatory, infectious or neoplasic pelvic disease, particularly for inflammatory sacroiliac pain).
8. Hip prosthesis on the same side as piriformis syndrome; knee prosthesis is tolerated.
9. Contraindication to BT injection: History of intolerance, hypersensitivity or known allergy to any botulinum toxin product or excipients; Patients with myasthenia gravis or other diseases of the neuromuscular junction; Patients with Lambert-Eaton Syndrome; Patients with neurological disorders such as dysphagia, swallowing disorders or aspiration pneumonia; Current infection at the proposed injection site; Long-term anticoagulant therapy ; Antibiotics and vaccination are prohibited during a period of 15 days around BT injection (15 days before and 15 days after BT injection).
10. Pregnant or breastfeeding women. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant must use an effective method of contraception (oral contraceptives, contraceptive injections, intrauterine devices, method of double-barrier contraceptive patches). The contraception should be maintained throughout the study. NB: Women will receive a pregnancy test prior to inclusion in the study.
11. Patients unable to complete the patient diary.
12. Inability to understand the reasons for the study; psychiatric disorders judged by the investigator to be incompatible with inclusion in the study.
13. Patients with legal disability or limited legal capacity.
14. Patients judged as noncompliant.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. The change in sciatic pain intensity, from baseline, at 6 weeks after injection. The sciatic pain will be measured by VAS (100mm scale). The patient will answer the following question: “What was the average intensity of your pain over the last 24 hours?”.

Secondary endpoints 5

1. Change in buttock pain intensity (assessed on Visual Analog Scale value, 0 = no pain, 10 = worst pain), from baseline, at 6 weeks after injection.
2. Variations from baseline in Health-related quality of life (HRQoL) (EQ-5D) at 6 weeks after injection as well as over a period of 6 months. HRQoL will be assessed at each visit (at inclusion and at 6, 12, 18 and 24 weeks of follow-up after injection).
3. Variations from baseline, over the 6-month follow-up, in sciatic pain intensity (VAS value), in buttock pain intensity (on VAS), in quality of life (HRQoL, EQ-5D) at 6 weeks after injection as well as over a period of 6 months, in physical functioning (BPI and PGI-I), in anxiety and depression (HADS) over 6 months in disability (ODI), in the tolerance of the sitting position assessed at each visit, consumption of painkillers.
4. Number of patients requiring a second injection (botulinum toxin) at 12 weeks in each arm.
5. Side effects of treatment during follow-up in each arm.

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Centre Hospitalier Universitaire De Nimes

Sponsor organisation

Centre Hospitalier Universitaire De Nimes

Address

Place Du Professeur Robert Debre

City

Nimes

Postcode

30900

Country

France

Scientific contact point

Organisation

Centre Hospitalier Universitaire De Nimes

Contact name

Brigitte LAFONT

Public contact point

Organisation

Centre Hospitalier Universitaire De Nimes

Contact name

Brigitte LAFONT

Locations

1 EU/EEA country · 8 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 11 files

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

Application history

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