Objective: The SIPEXI (Sialorrhea Pediatric Xeomin Investigation) study investigated efficacy and safety of intraglandular incobotulinumtoxinA (incoBoNT/A) injections in children/adolescents with sialorrhea associated with neurological disorders and/or intellectual disability.

Background: Sialorrhea is a common issue in children with neurological disorders like cerebral palsy. SIPEXI was the pivotal trial for approval of incoBoNT/A treatment of pediatric sialorrhea in the USA (2020).

Method: SIPEXI was a prospective, multicenter, phase 3 study (NCT02270736) with a randomized, double-blind, parallel-group, placebo-controlled main period (MP, 1 injection cycle [IC]) and an open-label extension (OLEX, 3 ICs). Subjects aged 6‑17 yrs were randomized to receive a body weight-dependent dose of incoBoNT/A according to weight classes of ~2 U/kg (total 75 U for subjects ≥30 kg) or placebo in the MP. In the OLEX, all received up to 3 further incoBoNT/A ICs. The follow-up was 16 weeks per IC. Primary endpoints were the change in unstimulated salivary flow rate (uSFR) from baseline to MP week 4 and the carers’ global impression of change scale (GICS) rating at MP week 4. Further endpoints included changes in these parameters at later visits. The statistical analysis of the primary endpoints was a mixed model repeated measures (MMRM) approach with comparison of least squares (LS) means between incoBoNT/A and placebo. Adverse events were recorded.

Results: We present the efficacy results for 6-17-year-olds (mean age 10 yrs; ~60% with cerebral palsy; >85% with intellectual disability). In the MP, 148 subjects received incoBoNT/A and 72 received placebo. 216 subjects completed the MP. Of these, 214 subjects entered and 189 completed the OLEX. At MP week 4, significant improvements were seen for incoBoNT/A compared to placebo in uSFR (LS-Mean difference: -0.06 [95% confidence interval (CI): -0.10; -0.03] g/min; p = 0.0012) and carers’ GICS ratings (LS-Mean difference: 0.28 [95% CI: 0.02; 0.53]; p = 0.032). Other endpoints consistently supported these results. During the OLEX, prolonged and sustained treatment effects were seen. No unexpected safety concerns arose.

Conclusion: IncoBoNT/A is effective for treatment of chronic sialorrhea in children/adolescents.
This abstract was presented in similar form at the TOXINS 2021 conference.

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MDS Abstracts - https://www.mdsabstracts.org/abstract/efficacy-of-incobotulinumtoxin-a-treatment-for-children-adolescents-with-sialorrhea-associated-with-neurological-disorders/