TOXSIALO-TRIAL : Evaluation of the long-term efficacy of the injection of botulinum toxin A into the salivary glands versus scopolamine patches in the treatment of drooling in children over 4 years old with cerebral palsy.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Hospices Civils De Lyon

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

12 Feb 2025 → 7 Oct 2025

Decision date (initial)

2024-07-26

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

French Ministry of Health

External identifiers

EU CT number

2024-512160-58-00

EudraCT number

2021-000095-10

ClinicalTrials.gov

NCT03616067

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Safety, Efficacy

To evaluate and compare the efficacy after 15 months of a “standardized rehabilitation guidance” treatment, associated with the injection of botulinum toxin (Botox®) in the salivary glands, compared to a “standardized rehabilitation guidance” treatment associated with the use of a scopolamine patch (Scopoderm®), on the degree and impact of drooling for children with cerebral palsy aged 4 years and over with pathological drooling.

Secondary objectives 9

1. Degree and impact of drooling for children after 1, 3, 6, 9 and 12 months.
2. Quantification of the drooling after 1, 3, 6, 9, 12 and 15 months
3. Clinical complications of the drooling after 1, 3, 6, 9, 12 and 15 months
4. Description of the adverse events (MedRA) that have occurred, theirs frequencies and theirs levels of intensity according to CTCAE classification
5. Quantification of the adverse events that have occurred, measured by the proportion of patients with at least one adverse event and the average number of adverse events per patient
6. Quantification of the adverse events having led to a discontinuation of the treatment, measured by the proportion of patients with at least one adverse event having led to a discontinuation of the treatment.
7. Description of the physical rehabilitation procedures.
8. Description of the therapeutic strategies used (dose modifications /reinjections and their justifications).
9. Description of the concomitant treatments used.

Conditions and MedDRA coding

Cerebral palsy with pathological drooling

Study design 1 period

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

1. Aged 4 to 17 years old
2. Cerebral palsy (with permanent tonus and posture disorder caused by a stable (non-progressive/non-degenerative) brain injury, congenital or acquired before the age of 2 years, regardless of the origin of the lesion) and with disabling drooling
3. Significant impact of drooling on the children (DIS score ≥40)
4. Affiliated or beneficiary of a social security scheme
5. At least one of the parents understanding and speaking French
6. Written consent form signed by both parents
7. Absence of known current pregnancy and breastfeeding

Exclusion criteria 10

1. Previous history of surgery for drooling
2. Epileptic encephalopathies and Rett syndromes
3. Injection of botulinum toxin (all locations) in the 3 months prior to the inclusion
4. Treatment by scopolamine patch (Scopoderm®) or other anticholinergic (Artane®, etc.) in the 7 days prior to inclusion
5. Contraindication to the anesthetic or sedation
6. Contraindication to one of the treatments studied (glaucoma, myasthenia, uretroprostatic disorders)
7. Injection site infection
8. Patients without gastrostomy, with a swallowing disorder (to saliva) or absence of spontaneous swallowing reflex not investigated by an ENT specialist (by nasoscopy or other)
9. On-going or programmed orthodontic treatment over the study period
10. Untreated severe or acute dental inflammatory condition (dental caries, gingivitis…)

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Change in score of the degree and impact of the drooling between baseline and after 15 months of treatment, measured by the DIS scale

Secondary endpoints 9

1. Scores of the degree and impact of the drooling, measured by the DIS scale at 1, 3, 6, 9 and 12 months of treatment (compared to the baseline measurement at the initiation of treatment at M0).
2. Severity of the drooling (quantity of saliva), measured by the average number of bibs used per day per patient (counted over 7 days) at 1, 3, 6, 9, 12 and 15 months (compared to the baseline measurement at treatment initiation at M0).
3. Clinical complications of the drooling, measured by the number of hospitalizations for pulmonary infections and the number of prescriptions for antibiotic treatment linked to bronchial secondary infection at 1, 3, 6, 9, 12 and 15 months.
4. Description of the nature of the adverse events that have occurred (by symptom), as well as their frequency and their intensity, measured using the AE intensity scale (see § 8.1.5 safety) at each time point.
5. Quantification of the adverse events, measured by the proportion of patients with at least one adverse event and the number of adverse events per patient at each time point.
6. Level of treatment discontinuation due to adverse events, measured by the proportion of patients with at least one adverse event having led to a discontinuation of the treatment at each time point.
7. Description of the nature and the frequency of the rehabilitation procedures used over 15 months.
8. Description of the experimented strategies used (dose modifications /reinjections and their justifications) at each time point.
9. Description of concomitant treatments used at each time point.

Investigational products

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

Test 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Hospices Civils De Lyon

Sponsor organisation

Hospices Civils De Lyon

Address

3 Quai Des Celestins, Bp 2251 Bp 2251

City

Lyon Cedex 02

Postcode

69229

Country

France

Scientific contact point

Organisation

Hospices Civils De Lyon

Contact name

Dr. Sandrine TOUZET

Public contact point

Organisation

Hospices Civils De Lyon

Contact name

Dr. Sandrine TOUZET

Locations

1 EU/EEA country · 13 investigational sites

By country

Investigational sites

Country notifications

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

Oversight and notifications

Regulatory notifications under CTR Articles 38, 52, 53, 54 and 77

Temporary halts 1 · Art. 38 CTR

Results and documents

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

Documents 12 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