Double-blind, Placebo-controlled, Randomized Clinical Trial Comparing the Efficacy and Safety of Sialanar Plus orAl rehabiLitation Against Placebo Plus Oral Rehabilitation for chIldren and Adolescents With seVere Sialorrhoea and Neurodisabilties,

NCT04873115 | Unknown Phase 4 DMC

• 1 other identifier: PRO-GLY-002 / E2013
• Study Type: interventional
• Target: 80
• Locations: 1 country
• Sites: 1

Brief Summary

Double blind, placebo-controlled, randomised trial, multicentre in France with open-label tolerability phase. The double-blind placebo-controlled study duration will be scheduled for 3 months with the final visit of the double-blind period at D84. After the D84 assessment, patients will be invited to continue into a 6-month openlabel study extension (OLSE) with ex-Sialanar® patients continuing the treatment and ex-placebo patients starting Sialanar®

Conditions

Sialorrhea; Neurodevelopmental Disorders

Keywords

Pediatric; sialorrhea; neurodisability

Outcome Measures

Primary Outcomes (1)

• The Change in Drooling Impact Scale (DIS) between baseline and D84.

  DIS possible range is 10-100. Lower score indicates a better outcome.

  84 days

Secondary Outcomes (6)

• Change in Drooling Impact Scale (DIS) between baseline and D28.

  28 days

• Proportion of responders at D84 (a response is defined as a DIS improvement ≥ 13.6 points).

  84 days

• Proportion of responders at D28 (a response is defined as a DIS improvement ≥ 13.6 points).

  28 days

• Proportion of good responders at D84 (a good response is defined as a DIS improvement ≥ 28 points).

  84 days

• Changes in the number of used bibs or clothing over 7 days (Item 3 of the DIS) at D84

  84 days

Other Outcomes (10)

• Change in the DIS 10-point item 9 from baseline. "To what extent did your child's drooling affect his or her life?" to D28 and D84.

  28 and 84 days

• Change in in the DIS 10-point item 10 from baseline. "To what extent did your child's dribbling affect you and your family's life?" to D28 and D84.

  28 and 84 days

• The change in DISABKIDS instrument from baseline to D84.

  84 days

Study Arms (2)

Sialanar

ACTIVE COMPARATOR

Sialanar administered as per the SmPC - titration over 4 weeks to reach a dose balancing efficacy with tolerability.

Drug: Sialanar; Other: Oral Rehabilitation

Placebo

PLACEBO COMPARATOR

Placebo administered as per the Sialanar SmPC - titration over 4 weeks to reach a dose balancing efficacy with tolerability.

Other: Placebo; Other: Oral Rehabilitation

Interventions

Placebo OTHER

Sialanar placebo

Placebo

Sialanar DRUG

Sialanar - a licensed glycopyrronium bromide product

Sialanar

Oral Rehabilitation OTHER

Standard oral rehabilitation for provided drooling

Placebo; Sialanar

Eligibility Criteria

• Age: 3 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Children aged ≥ 3 years old and \< 18 years old.
• Children with weight ≥ 13 kg
• Children with chronic neurological disorders (such as polyhandicap, cerebral palsy, Angelman syndrome, Rett's syndrome, epilepsy, amyotrophic lateral sclerosis and mental retardation)
• Diagnosis of severe sialorrhoea due to a chronic neurological disorder as assessed by a modified Teachers Drooling Scale (mTDS) ≥ 6.
• DIS Scale ≥ 50. Impact of drooling as assessed by drooling impact scale.
• Children who have completed at least 3 months of non-pharmacological standard of care treatment (i.e. rehabilitation e.g. intraoral stimulation and oral facial exercise).
• Children with stable drooling for the past 4 weeks.
• Written consent form signed by parents (or, when applicable, the subject's legally acceptable representative).
• Affiliated or beneficiary of a social security scheme.
• A nominated parent or carer who can commit to complete parent / carer questionnaires, with good ability to understand and speak French.

You may not qualify if:

• Children unwilling to provide assent to participate in the study. (children who are unable to provide assent should be considered eligible).
• Botulinum injection for sialorrhoea given within 6 months of enrolment.
• Any anticholinergic therapy used in the previous 4 weeks.
• Scopoderm patch used in the previous 4 weeks.
• History of surgery for drooling in the previous 12 months.
• Children prescribed non-permitted concomitant medication as defined in section 7.2.2
• Children in whom anticholinergics are contraindicated such as those with glaucoma, myasthenia gravis, urinary retention, severe renal impairment, history of intestinal obstruction, ulcerative colitis, paralytic ileus, pyloric stenosis or hypersensitivity to the active substance or the excipient.
• On-going or programmed orthodontic treatment over the study period.
• Untreated oro-mandibular dystonia (isolated lingual dystonia accepted), clinical gastro oesophageal reflux, dental inflammatory condition (dental caries, gingivitis…).
• Family and carers unable to commit to the schedule of the study protocol.
• Female patients who are lacting or pregnant
• Female patients who are planning a pregnancy within the study period
• Patients having participated in another clinical study within at least 30 days or within 5 half-lives of last dose of IMP (whichever is longer).
• Patients receiving systemic immunosuppressive treatment including cyclosporin, methotrexate, azathioprine cyclophosphamide, mycophenolic acid, anti TNFα, monoclonal antibodies or with congenital immunodeficiency

Sponsors & Collaborators

• Proveca Pharma Limited: lead

Study Sites (1)

CHU Lille

Lille, France

RECRUITING

MeSH Terms

Conditions

Sialorrhea; Neurodevelopmental Disorders

Interventions

Rehabilitation

Condition Hierarchy (Ancestors)

Salivary Gland Diseases; Mouth Diseases; Stomatognathic Diseases; Mental Disorders

Intervention Hierarchy (Ancestors)

Aftercare; Continuity of Patient Care; Patient Care; Therapeutics; Health Services; Health Care Facilities Workforce and Services

Study Officials

• Nick Probert

  Proveca Pharma Limited

  STUDY DIRECTOR

• Pierre Fayoux

  CHU Lille

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Nick Probert

CONTACT

07767630041; nickprobert@proveca.com

Helen Shaw

CONTACT

07775704497; helen@proveca.com

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Double-blinded randomised phase followed by open label treatment.

Study Record Dates

• First Submitted: April 27, 2021
• First Posted: May 5, 2021
• Study Start: May 10, 2021
• Primary Completion: December 10, 2022
• Study Completion: June 10, 2023
• Last Updated: July 8, 2022

Record last verified: 2022-07

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1)