Efficacy and Safety of IncobotulinumtoxinA in Children/Adolescents with Chronic Sialorrhea in Neurological Disorders and/or Intellectual Disability

Background/Purpose: Analysis of incobotulinumtoxinA treatment for sialorrhea in children/adolescents.

Methods: SIPEXI (NCT02270736) was a phase III, randomized, double-blind, placebo-controlled, parallel-group trial with open-label extension (OLEX) including children/adolescents (2–17 years) with chronic sialorrhea with neurological disorders and/or intellectual disability, and severe drooling (investigator's Modified Teacher's Drooling Scale rating ≥6). Six- to 17-year-olds were randomized 2:1 to receive ~2 U/kg body weight (BW) of incobotulinumtoxinA (fixed total dose 75 U for subjects ≥30 kg BW) or placebo during main phase (MP). Subjects aged 2 to 5 years received only incobotulinumtoxinA. All subjects received three further incobotulinumtoxinA injection cycles (IC). The co-primary efficacy endpoints were unstimulated salivary flow rate (uSFR) change from baseline to week 4 and carer's Global Impression of Change Scale (GICS) score at week 4 in 6- to 17-year-olds. The change in uSFR from study/cycle baseline to weeks 4 and 16; GICS score at weeks 4, 8, 12, and 16 of each OLEX cycle; and the occurrence of treatment-emergent adverse events (TEAEs) overall and by injection cycle were also assessed.

Results: A total of 256 subjects were randomized, 250 (97.7%) completed the MP. A total of 247 subjects entered OLEX, and 222 (89.9%) completed all three ICs. IncobotulinumtoxinA demonstrated statistically significant superiority over placebo in both co-primary endpoints at week 4. Improvement in uSFR and carer's GICS score was also observed in OLEX with sustained effects after repeated treatment. Both MP and OLEX had good overall safety profiles.

Conclusion: IncobotulinumtoxinA is effective and well tolerated for the treatment of children / adolescents with chronic troublesome sialorrhea.

Publication History

Article published online:
28 October 2021