Clinical Study to Investigate the Efficacy and Safety of NT 201 Compared to Placebo in the Treatment of Chronic Troublesome Drooling Associated With Neurological Disorders and/or Intellectual Disability "SIPEXI"

Recruiting

Phase 3 Results N/A

Trial Description

The objective of this study is to investigate the efficacy and safety of NT 201 compared with placebo for the treatment of chronic troublesome sialorrhea associated with neurological disorders (e.g. cerebral palsy, traumatic brain injury) and/or intellectual disability in children and adolescents naïve to Botulinum neurotoxin treatment and aged 2-17 years.

Conditions

Interventions

  • Placebo Drug
    Intervention Desc: Solution for injection prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl).
    ARM 1: Kind: Experimental
    Label: Placebo
    Description: For subjects aged 6-17 years only. Main period (1 treatment cycle): Subjects to receive placebo injection. Extension period (3 treatment cycles): Subjects to receive on average 2 Units IncobotulinumtoxinA per kg body weight per treatment cycle (subjects with a body weight ≥ 30 kg to receive a fixed total dose of 75 U per cycle). Mode of administration: Four injections at the beginning of each treatment cycle (parotid and submandibular glands, bilateral)
  • IncobotulinumtoxinA Drug
    Intervention Desc: Active ingredient: Clostridium Botulinum neurotoxin Type A free from complexing proteins. Solution for injection prepared by reconstitution of powder with 0.9% Sodium Chloride (NaCl).
    ARM 1: Kind: Experimental
    Label: IncobotulinumtoxinA (Xeomin)
    Description: Main and extension period: subjects to receive on average 2 units incobotulinumtoxinA per kg body weight per treatment cycle (subjects with a body weight ≥ 30 kg to receive a fixed total dose of 75 U per cycle). Mode of administration: Four injections at the beginning of each treatment cycle (parotid and submandibular glands, bilateral)
    ARM 2: Kind: Experimental
    Label: Placebo
    Description: For subjects aged 6-17 years only. Main period (1 treatment cycle): Subjects to receive placebo injection. Extension period (3 treatment cycles): Subjects to receive on average 2 Units IncobotulinumtoxinA per kg body weight per treatment cycle (subjects with a body weight ≥ 30 kg to receive a fixed total dose of 75 U per cycle). Mode of administration: Four injections at the beginning of each treatment cycle (parotid and submandibular glands, bilateral)

Trial Design

  • Allocation: Randomized
  • Masking: Double Blind (Subject, Investigator)
  • Purpose: Treatment
  • Endpoint: Efficacy Study
  • Intervention: Parallel Assignment

Outcomes

Type Measure Time Frame Safety Issue
Primary Change in unstimulated salivary flow rate [uSFR] from baseline to Week 4 Baseline to week 4 No
Primary Global Impression of Change Scale [GICS] at Week 4 representing the functional improvement in drooling since baseline as assessed by the carer/parent(s). Week 4 No
Primary Occurrence of treatment emergent AEs [TEAEs] overall and by injection cycle Baseline up to week 64 No
Secondary Change in uSFR from baseline to Week 8 and 12. Baseline up to week 12 No
Secondary GICS at Week 8 and 12. Up to week 12 No
Secondary Occurrence of treatment emergent AESIs [TEAESIs] overall and by injection cycle Baseline up to week 64 No
Secondary Occurrence of treatment emergent SAEs [TESAEs] overall and by injection cycle Baseline up to week 64 No
Secondary Occurrence of TEAEs related to treatment as assessed by the investigator overall and by injection cycle Baseline up to week 64 No
Secondary Occurrence of TEAEs leading to discontinuation overall and by injection cycle Baseline up to week 64 No

Sponsors