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SIAXI: Placebo-controlled, randomized, double-blind study of incobotulinumtoxinA for sialorrhea.

ABSTRACT: OBJECTIVE:This pivotal phase III study, SIAXI, investigated the efficacy and safety of incobotulinumtoxinA for the treatment of chronic sialorrhea due to Parkinson disease (PD), atypical parkinsonism, stroke, or traumatic brain injury (TBI). METHODS:Adult patients with PD (70.7%), atypical parkinsonism (8.7%), stroke (19.0%), or TBI (2.7%) were randomized (2:2:1) to double-blind treatment with placebo (n = 36), or total doses of incobotulinumtoxinA 75 U (n = 74) or 100 U (n = 74), in a single treatment cycle. The coprimary endpoints were change in unstimulated salivary flow rate from baseline to week 4, and patients' Global Impression of Change Scale score at week 4. Adverse events were recorded throughout. RESULTS:A total of 184 patients were randomized. Both incobotulinumtoxinA dose groups showed reductions in mean unstimulated salivary flow rate at week 4, with a significant difference vs placebo in the incobotulinumtoxinA 100 U group (p = 0.004). Patients' Global Impression of Change Scale scores also improved at week 4, with a significant difference vs placebo in the incobotulinumtoxinA 100 U group (p = 0.002). A lasting effect was observed at week 16 post injection. The most frequent treatment-related adverse events in the incobotulinumtoxinA 75 U and 100 U groups were dry mouth (5.4% and 2.7% of patients) and dysphagia (2.7% and 0.0% of patients). CONCLUSIONS:IncobotulinumtoxinA 100 U is an effective and well-tolerated treatment of chronic sialorrhea in adults. CLINICALTRIALSGOV IDENTIFIER:NCT02091739. CLASSIFICATION OF EVIDENCE:This study provides Class I evidence that incobotulinumtoxinA reduces salivary flow rates in patients with chronic sialorrhea due to PD, atypical parkinsonism, stroke, or TBI.

SUBMITTER: Jost WH

PROVIDER: S-EPMC6511076 | biostudies-literature |

REPOSITORIES: biostudies-literature

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Project description:Background and objectivesThis prospective phase III study (SIPEXI) investigated efficacy and safety of repeated injections of incobotulinumtoxinA (incoBoNT/A) for treatment of chronic sialorrhea (drooling) associated with neurological disorders (e.g., cerebral palsy, traumatic brain injury) and/or intellectual disability in children/adolescents.MethodsThe study enrolled 2-17-year-olds with sialorrhea due to neurological disorders and/or intellectual disability. Patients received body weight-dependent doses of incoBoNT/A (20 U to 75 U). A main period with 1 injection cycle (placebo-controlled, double-blind, 6-17-year-olds) was followed by an open-label extension with up to 3 further cycles. An additional cohort of 2-5-year-olds received active treatment throughout the study. Co-primary endpoints were the change in unstimulated salivary flow rate (uSFR) from baseline to week 4, and the carers' global impression of change scale (GICS) rating at week 4. Adverse events were recorded.ResultsIn the main period, 220 patients aged 6-17 years were randomized and treated (148 patients in incoBoNT/A group, 72 patients in placebo group). 35 patients aged 2-5 years received incoBoNT/A (no placebo). 214 patients aged 6-17 years and 33 patients aged 2-5 years continued treatment in the open-label extension period. For the 6-17-year-olds, a significant difference between incoBoNT/A and placebo was seen in the mean uSFR decrease (difference: -0.06 g/min; p = 0.0012) and the carers' GICS rating (difference: 0.28 points; p = 0.032) at week 4, in favor of active treatment. The secondary endpoints consistently supported these results. A sustained benefit was observed during the extension. Incidences of adverse events were comparable between incoBoNT/A and placebo and did not increase notably with repeated injections. The most common adverse events were respiratory infections. Efficacy and safety were also favorable in the uncontrolled cohort of 2-5-year-olds.DiscussionBoth co-primary efficacy endpoints were reached and superiority of incoBoNT/A over placebo was confirmed. IncoBoNT/A (up to 75 U, up to 4 cycles) is an effective and well-tolerated treatment for sialorrhea associated with neurological disorders in children.Study registrationsClinicaltrials.gov: NCT02270736 (www.clinicaltrials.gov/ct2/show/results/NCT02270736); EU Clinical Trials Register: 2013-004532-30 (www.clinicaltrialsregister.eu/ctr-search/search?query=2013-004532-30).Classification of evidenceThis study provides Class I evidence that injection of incobotulinumtoxinA decreases drooling in children aged 6-17 years with neurological disorders.

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SIAXI: Placebo-controlled, randomized, double-blind study of incobotulinumtoxinA for sialorrhea.

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