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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
256
NT 201 placebo matching injection.
NT 201 injection.
Merz Investigational Site #9950003
K'obulet'i, Georgia
Merz Investigational Site #9950001
Tbilisi, Georgia
Merz Investigational Site #9950002
Tbilisi, Georgia
Merz Investigational Site #0360017
Balassagyarmat, Hungary
Merz Investigational Site #0360013
Budapest, Hungary
Merz Investigational Site # 0360014
Change From Baseline in Unstimulated Salivary Flow Rate (uSFR) at Week 4
This endpoint was planned to be analyzed in double-blind, MP, 6 to 17 years participants only. uSFR was assessed by weighing of absorbent swabs with safety threads soaked with saliva over 5 minutes and the procedure was repeated after 30 minutes. Salivary flow rate was equal to weight increase of swabs/time of collection. The average of the 2 results for flow rate was calculated. The reduction of measured weight over the study relates to improvement of sialorrhea.
Time frame: Baseline and Week 4
Global Impression of Change Scale (GICS) at Week 4 Assessed by the Carer/Parent(s)
This endpoint was analyzed in double-blind, MP, 6 to 17 years participants. The GICS was used to measure the carer's/parent's impression of change due to treatment. The response option was a common 7-point Likert scale, with the following values: +3 (very much improved); +2 (much improved); +1 (minimally improved); 0 (no change); -1 (minimally worse); -2 (much worse); -3 (very much worse).
Time frame: Week 4
Occurrence of Treatment Emergent Adverse Events (TEAEs) Overall and Per Injection Cycle
Time frame: Baseline up to Week 64
Change From Baseline in uSFR at Weeks 8 and 12
This endpoint was analyzed in double-blind, MP, 6 to 17 years participants. uSFR was assessed by weighing of absorbent swabs with safety threads soaked with saliva over 5 minutes and then procedure was repeated after 30 minutes. Salivary flow rate was equal to weight increase of swabs/time of collection. The average of the 2 results for flow rate was calculated. The reduction of measured weight over the study relates to improvement of sialorrhea.
Time frame: Baseline and Weeks 8 and 12
GICS at Weeks 8 and 12
This endpoint was analyzed in double-blind, MP, 6 to 17 years participants. The GICS was used to measure the carer's/parent's impression of change due to treatment. The response option was a common 7-point Likert scale with the following values: +3 (very much improved); +2 (much improved); +1 (minimally improved); 0 (no change); -1 (minimally worse); -2 (much worse); -3 (very much worse).
Time frame: Weeks 8 and 12
Occurrence of Treatment Emergent Adverse Events of Special Interest (AESI) Overall and by Injection Cycle
Time frame: Baseline up to Week 64
Occurrence of Treatment Emergent Serious Adverse Events (TESAEs) Overall and by Injection Cycle
Time frame: Baseline up to Week 64
Occurrence of TEAEs Related to Treatment as Assessed by the Investigator Overall and by Injection Cycle
Time frame: Baseline up to Week 64
Occurrence of TEAEs Leading to Discontinuation Overall and by Injection Cycle
Time frame: Baseline up to Week 64
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Budapest, Hungary
Merz Investigational Site # 0360015
Budapest, Hungary
Merz Investigational Site #0360018
Budapest, Hungary
Merz Investigational Site #0360016
Szombathely, Hungary
Merz Investigational Site #0480092
Bialystok, Poland
...and 21 more locations