DEROTOX : DEgenerative ROtator cuff disease and botulinum TOXin

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Assistance Publique Hopitaux De Paris

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Musculoskeletal Diseases [C05]

Trial duration

20 Jan 2023 → ongoing

Decision date (initial)

2024-07-22

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Funding sources

MERZ THERAPEUTICS GMBH

External identifiers

EU CT number

2024-514074-40-00

EudraCT number

2021-003315-25

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy

To evaluate the effectiveness of botulinum toxin in the treatment of degenerative tendinopathy of the rotator cuff of the shoulder, at one month, based on the Shoulder Pain And Disability Index score (SPADI total)

Secondary objectives 6

1. To evaluate the effectiveness of botulinum toxin in the treatment of degenerative tendinopathy of the rotator cuff of the shoulder, based on the total SPADI score at 3 months
2. To evaluate the effectiveness of botulinum toxin in the treatment of degenerative tendinopathy of the rotator cuff of the shoulder, based on SPADI sub-scores (pain and functional limitation) at 3 months
3. To evaluate the effectiveness of botulinum toxin in the treatment of degenerative tendinopathy of the rotator cuff of the shoulder, based on perceived overall improvement, by EVA (17.4.2) from 0 to 100 at 1 and 3 months
4. To evaluate the effectiveness of botulinum toxin in the treatment of degenerative tendinopathy of the rotator cuff of the shoulder, based on painkiller medication consumption over the 3 months of follow-up
5. To evaluate the effectiveness of botulinum toxin in the treatment of degenerative tendinopathy of the rotator cuff of the shoulder, based on the acceptability of treatment by VAS from 0 to 100 at 1 month and 3 months
6. To evaluate the tolerance of botulinum toxin at 1 week by collecting adverse effects: weakness in the arm muscles; muscle weakness distant from the injection site or generalized weakness; difficulty swallowing, speaking; dyspnea; allergic reaction after injection

Conditions and MedDRA coding

Degenerative shoulder rotator cuff tendinopathy

Study design 1 period

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 12

1. Age > 40 years
2. Duration of pain > 1 month
3. Pain intensity ≥ 40/100 on the visual analog scale
4. SPADI score ≥ 30/100
5. Pain medication(s) unchanged within 30 days prior to enrollment
6. Pain with or without weakness during the Jobe maneuver
7. Ultrasound within 30 days, showing supraspinatus tendinopathy, with or without rupture
8. Affiliation to a social security scheme
9. Ability to give consent, complete the weekly notebook (collection of medicinal treatments taken for pain)
10. Availability for visits planned by the protocol
11. Use of an effective method of contraception in women of childbearing potential that began at least 1 month before and continues for at least 1 month after administration of study treatment
12. Negative urine test for women of childbearing age

Exclusion criteria 24

1. Reduced passive range of motion
2. Skin infection at the intended injection site
3. Glenohumeral instability
4. Tendon calcification
5. Ultrasound showing a concomitant rupture of the infraspinatus or subscapularis
6. Infiltration of corticosteroids into the shoulder in the 30 days preceding the inclusion visit
7. Shoulder surgery
8. Humeral fracture, inflammatory rheumatism or neoplastic disease
9. Contraindication to XEOMIN® (hypersensitivity to XEOMIN® or to one of the excipients or to any other botulinum toxin product, generalized disorder of muscle activity, in the event of infection or inflammation at the injection site concerned)
10. Concomitant use of aminoglycosides, cyclosporine, aminoquinolines or spectinomycin
11. Participation in other interventional research (RIPH) during the 3-month follow-up period of DEROTOX Research. Participation in another RIPH will be possible beyond these 3 months.
12. Patients with a history of inhalation pneumonitis and dysphagia
13. Patients on anticoagulant therapy or on therapy likely to have an anticoagulant effect
14. Patients with bleeding disorders
15. Patients suffering from amyotrophic lateral sclerosis, myasthenia gravis or Lambert-Eaton syndrome.
16. Patients with other disorders leading to peripheral neuromuscular dysfunction
17. Weakness or pronounced atrophy of the supraspinatus muscle
18. Women of childbearing age who have not taken a urine pregnancy test or who have taken a positive urine pregnancy test
19. Pregnancy, breast-feeding
20. Botulinum toxin injections within the last 6 months
21. Vulnerable persons (under legal protection, guardianship or curatorship).
22. Glenohumeral arthropathy
23. Painful acromioclavicular arthropathy
24. Neurogenic upper limb pain

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Total SPADI score at 1 month.

Secondary endpoints 5

1. Total SPADI score at 3 months
2. SPADI sub-scores at 3 months
3. Perceived overall improvement, by VAS at 1 and 3 months
4. Painkiller medication consumption over the 3 months of follow-up
5. Acceptability of treatment by VAS from 0 to 100 at 1 month and 3 months

Investigational products

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

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Assistance Publique Hopitaux De Paris

Sponsor organisation

Assistance Publique Hopitaux De Paris

Address

Porte 23, 1 Avenue Claude Vellefaux 1 Avenue Claude Vellefaux

City

Paris Cedex 10

Postcode

75475

Country

France

Scientific contact point

Organisation

Assistance Publique Hopitaux De Paris

Contact name

Dr Louis JACOB

Public contact point

Organisation

Assistance Publique Hopitaux De Paris

Contact name

Dr Louis JACOB

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 5 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