Trial Outcomes & Findings for 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 (NCT NCT02270736)

Last Updated: 2021-08-10

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

256 participants

Primary outcome timeframe

Baseline and Week 4

Results posted on

2021-08-10

Participant Flow

The study was conducted at 28 investigational sites in Georgia, Hungary, Poland, Russia, Serbia, and Ukraine.

A total of 281 participants were screened, out of which 256 participants were enrolled/randomized into the study. Of these 256 participants, 255 participants received the study treatment. A total of 247 participants who completed the Main Period (MP) entered the Open-label Extension Period (OLEX) of the study.

Participant milestones

Participant milestones
Measure	Double-blind MP: Placebo (Age 6 to 17 Years) Participants received placebo via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks. Volumes were matched to the volumes of NT 201 (incobotulinumtoxinA; Xeomin) injected in the experimental arm.	Double-blind, MP: NT 201 (Age 6 to 17 Years) Participants received NT 201 (up to 2.5 Units per kilogram \[U/kg\] body weight) via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks.	Open-label, MP: NT 201 (Age 2 to 5 Years) Participants received NT 201 (about 1.5-2 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks.	OLEX: NT 201 (Age 6 to 17 Years) Participants received NT 201 (up to 2.5 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands on Day 1 of second (Visit 6), third (Visit 10), and fourth (Visit 14) injection cycle of the OLEX, followed by an observation period of 16 weeks each (48 weeks in total). This arm consisted of participants who participated in MP arms "Double-blind, MP: placebo (age 6 to 17 years)" and "Double-blind, MP: NT 201 (age 6 to 17 years)".	OLEX: NT 201 (Age 2 to 5 Years) Participants received NT 201 (about 1.5-2 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands on Day 1 of second (Visit 6), third (Visit 10), and fourth (Visit 14) injection cycle of the OLEX, followed by an observation period of 16 weeks each (48 weeks in total).
Main Period (MP) (up to 16 Weeks) STARTED	72	148	36	0	0
Main Period (MP) (up to 16 Weeks) Treated	72	148	35	0	0
Main Period (MP) (up to 16 Weeks) COMPLETED	70	146	34	0	0
Main Period (MP) (up to 16 Weeks) NOT COMPLETED	2	2	2	0	0
OLEX Period (up to 48 Weeks) STARTED	0	0	0	214	33
OLEX Period (up to 48 Weeks) COMPLETED	0	0	0	189	33
OLEX Period (up to 48 Weeks) NOT COMPLETED	0	0	0	25	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Double-blind MP: Placebo (Age 6 to 17 Years) Participants received placebo via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks. Volumes were matched to the volumes of NT 201 (incobotulinumtoxinA; Xeomin) injected in the experimental arm.	Double-blind, MP: NT 201 (Age 6 to 17 Years) Participants received NT 201 (up to 2.5 Units per kilogram \[U/kg\] body weight) via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks.	Open-label, MP: NT 201 (Age 2 to 5 Years) Participants received NT 201 (about 1.5-2 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks.	OLEX: NT 201 (Age 6 to 17 Years) Participants received NT 201 (up to 2.5 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands on Day 1 of second (Visit 6), third (Visit 10), and fourth (Visit 14) injection cycle of the OLEX, followed by an observation period of 16 weeks each (48 weeks in total). This arm consisted of participants who participated in MP arms "Double-blind, MP: placebo (age 6 to 17 years)" and "Double-blind, MP: NT 201 (age 6 to 17 years)".
Main Period (MP) (up to 16 Weeks) Adverse Event	0	1	0	0
Main Period (MP) (up to 16 Weeks) Withdrawal by Subject	2	0	1	0
Main Period (MP) (up to 16 Weeks) Lost to Follow-up	0	1	0	0
Main Period (MP) (up to 16 Weeks) Randomized, not Treated	0	0	1	0
OLEX Period (up to 48 Weeks) Physician Decision	0	0	0	1
OLEX Period (up to 48 Weeks) Lack of Efficacy	0	0	0	2
OLEX Period (up to 48 Weeks) Adverse Event	0	0	0	4
OLEX Period (up to 48 Weeks) Withdrawal by Subject	0	0	0	16
OLEX Period (up to 48 Weeks) Lost to Follow-up	0	0	0	2

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Double-blind MP: Placebo (Age 6 to 17 Years) n=72 Participants Participants received placebo via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks. Volumes were matched to the volumes of NT 201 (incobotulinumtoxinA; Xeomin) injected in the experimental arm.	Double-blind, MP: NT 201 (Age 6 to 17 Years) n=148 Participants Participants received NT 201 (up to 2.5 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks.	Open-label, MP: NT 201 (Age 2 to 5 Years) n=35 Participants Participants received NT 201 (about 1.5-2 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks.	Total n=255 Participants Total of all reporting groups
Age, Customized In utero	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Customized Preterm newborn infants (gestational age < 37 wks)	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Customized Newborns (0-27 days)	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Customized Infants and toddlers (28 days-23 months)	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Customized Children (2-11 years)	48 Participants n=5 Participants	96 Participants n=7 Participants	35 Participants n=5 Participants	179 Participants n=4 Participants
Age, Customized Adolescents (12-17 years)	24 Participants n=5 Participants	52 Participants n=7 Participants	0 Participants n=5 Participants	76 Participants n=4 Participants
Age, Customized Adults (18-64 years)	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Customized From 65-84 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Age, Customized 85 years and over	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Sex: Female, Male Female	27 Participants n=5 Participants	55 Participants n=7 Participants	13 Participants n=5 Participants	95 Participants n=4 Participants
Sex: Female, Male Male	45 Participants n=5 Participants	93 Participants n=7 Participants	22 Participants n=5 Participants	160 Participants n=4 Participants
Race/Ethnicity, Customized White	72 Participants n=5 Participants	148 Participants n=7 Participants	35 Participants n=5 Participants	255 Participants n=4 Participants
Race/Ethnicity, Customized Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants	0 Participants n=4 Participants
Height	135.3 centimeter (cm) STANDARD_DEVIATION 16.92 • n=5 Participants	132.8 centimeter (cm) STANDARD_DEVIATION 17.15 • n=7 Participants	101.1 centimeter (cm) STANDARD_DEVIATION 8.09 • n=5 Participants	129.1 centimeter (cm) STANDARD_DEVIATION 19.64 • n=4 Participants
Weight	30.8 kilogram (kg) STANDARD_DEVIATION 11.67 • n=5 Participants	28.8 kilogram (kg) STANDARD_DEVIATION 11.48 • n=7 Participants	15.7 kilogram (kg) STANDARD_DEVIATION 3.00 • n=5 Participants	27.6 kilogram (kg) STANDARD_DEVIATION 11.78 • n=4 Participants
Body Mass Index (BMI)	16.4 kilogram per square meter (kg/m^2) STANDARD_DEVIATION 3.65 • n=5 Participants	15.8 kilogram per square meter (kg/m^2) STANDARD_DEVIATION 3.25 • n=7 Participants	15.3 kilogram per square meter (kg/m^2) STANDARD_DEVIATION 1.85 • n=5 Participants	15.9 kilogram per square meter (kg/m^2) STANDARD_DEVIATION 3.23 • n=4 Participants

PRIMARY outcome

Timeframe: Baseline and Week 4

Population: The full analysis set (FAS) is identical to the subset of participants in the SES (MP) where subset of all participants received study medication (NT 201 or placebo) during the MP of the study.

Outcome measures

Outcome measures
Measure	Double-blind MP: Placebo (Age 6 to 17 Years) n=72 Participants Participants received placebo via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks. Volumes were matched to the volumes of NT 201 (incobotulinumtoxinA; Xeomin) injected in the experimental arm.	Double-blind, MP: NT 201 (Age 6 to 17 Years) n=148 Participants Participants received NT 201 (up to 2.5 Units per kilogram \[U/kg\] body weight) via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks.	Open-label, MP: NT 201 (Age 2 to 5 Years) Participants received NT 201 (about 1.5-2 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks.	OLEX: NT 201 (Age 6 to 17 Years) Participants received NT 201 (up to 2.5 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands on Day 1 of second (Visit 6), third (Visit 10), and fourth (Visit 14) injection cycle of the OLEX, followed by an observation period of 16 weeks each (48 weeks in total). This arm consisted of participants who participated in MP arms "Double-blind, MP: placebo (age 6 to 17 years)" and "Double-blind, MP: NT 201 (age 6 to 17 years)".	OLEX: NT 201 (Age 2 to 5 Years) Participants received NT 201 (about 1.5-2 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands on Day 1 of second (Visit 6), third (Visit 10), and fourth (Visit 14) injection cycle of the OLEX, followed by an observation period of 16 weeks each (48 weeks in total).
Change From Baseline in Unstimulated Salivary Flow Rate (uSFR) at Week 4	-0.07 gram per minute (g/min) Standard Error 0.015	-0.14 gram per minute (g/min) Standard Error 0.012	—	—	—

PRIMARY outcome

Timeframe: Week 4

Population: The FAS is identical to the subset of participants in the SES (MP) where subset of all participants received study medication (NT 201 or placebo) during the MP of the study.

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).

Outcome measures

Outcome measures
Measure	Double-blind MP: Placebo (Age 6 to 17 Years) n=72 Participants Participants received placebo via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks. Volumes were matched to the volumes of NT 201 (incobotulinumtoxinA; Xeomin) injected in the experimental arm.	Double-blind, MP: NT 201 (Age 6 to 17 Years) n=148 Participants Participants received NT 201 (up to 2.5 Units per kilogram \[U/kg\] body weight) via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks.	Open-label, MP: NT 201 (Age 2 to 5 Years) Participants received NT 201 (about 1.5-2 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks.	OLEX: NT 201 (Age 6 to 17 Years) Participants received NT 201 (up to 2.5 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands on Day 1 of second (Visit 6), third (Visit 10), and fourth (Visit 14) injection cycle of the OLEX, followed by an observation period of 16 weeks each (48 weeks in total). This arm consisted of participants who participated in MP arms "Double-blind, MP: placebo (age 6 to 17 years)" and "Double-blind, MP: NT 201 (age 6 to 17 years)".	OLEX: NT 201 (Age 2 to 5 Years) Participants received NT 201 (about 1.5-2 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands on Day 1 of second (Visit 6), third (Visit 10), and fourth (Visit 14) injection cycle of the OLEX, followed by an observation period of 16 weeks each (48 weeks in total).
Global Impression of Change Scale (GICS) at Week 4 Assessed by the Carer/Parent(s)	0.63 units on a scale Standard Error 0.104	0.91 units on a scale Standard Error 0.075	—	—	—

PRIMARY outcome

Timeframe: Baseline up to Week 64

Population: SES: subset of all participants who received study medication (NT 201 or placebo) during MP or (NT 201) during OLEX of study. "n" is number of participants evaluable for this measure at a given time period and who were included in the assessment.

Outcome measures

Outcome measures
Measure	Double-blind MP: Placebo (Age 6 to 17 Years) n=72 Participants Participants received placebo via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks. Volumes were matched to the volumes of NT 201 (incobotulinumtoxinA; Xeomin) injected in the experimental arm.	Double-blind, MP: NT 201 (Age 6 to 17 Years) n=148 Participants Participants received NT 201 (up to 2.5 Units per kilogram \[U/kg\] body weight) via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks.	Open-label, MP: NT 201 (Age 2 to 5 Years) n=35 Participants Participants received NT 201 (about 1.5-2 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks.	OLEX: NT 201 (Age 6 to 17 Years) n=214 Participants Participants received NT 201 (up to 2.5 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands on Day 1 of second (Visit 6), third (Visit 10), and fourth (Visit 14) injection cycle of the OLEX, followed by an observation period of 16 weeks each (48 weeks in total). This arm consisted of participants who participated in MP arms "Double-blind, MP: placebo (age 6 to 17 years)" and "Double-blind, MP: NT 201 (age 6 to 17 years)".	OLEX: NT 201 (Age 2 to 5 Years) n=33 Participants Participants received NT 201 (about 1.5-2 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands on Day 1 of second (Visit 6), third (Visit 10), and fourth (Visit 14) injection cycle of the OLEX, followed by an observation period of 16 weeks each (48 weeks in total).
Occurrence of Treatment Emergent Adverse Events (TEAEs) Overall and Per Injection Cycle Overall	11 Participants	27 Participants	5 Participants	92 Participants	15 Participants
Occurrence of Treatment Emergent Adverse Events (TEAEs) Overall and Per Injection Cycle First injection cycle (MP)	11 Participants	27 Participants	5 Participants	—	—
Occurrence of Treatment Emergent Adverse Events (TEAEs) Overall and Per Injection Cycle Second injection cycle (OLEX)	—	—	—	44 Participants	7 Participants
Occurrence of Treatment Emergent Adverse Events (TEAEs) Overall and Per Injection Cycle Third injection cycle (OLEX)	—	—	—	35 Participants	5 Participants
Occurrence of Treatment Emergent Adverse Events (TEAEs) Overall and Per Injection Cycle Fourth injection cycle (OLEX)	—	—	—	40 Participants	11 Participants

SECONDARY outcome

Timeframe: Baseline and Weeks 8 and 12

Population: The FAS is identical to the subset of participants in the SES (MP) where subset of all participants received study medication (NT 201 or placebo) during the MP of the study.

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.

Outcome measures

Outcome measures
Measure	Double-blind MP: Placebo (Age 6 to 17 Years) n=72 Participants Participants received placebo via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks. Volumes were matched to the volumes of NT 201 (incobotulinumtoxinA; Xeomin) injected in the experimental arm.	Double-blind, MP: NT 201 (Age 6 to 17 Years) n=148 Participants Participants received NT 201 (up to 2.5 Units per kilogram \[U/kg\] body weight) via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks.	Open-label, MP: NT 201 (Age 2 to 5 Years) Participants received NT 201 (about 1.5-2 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks.	OLEX: NT 201 (Age 6 to 17 Years) Participants received NT 201 (up to 2.5 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands on Day 1 of second (Visit 6), third (Visit 10), and fourth (Visit 14) injection cycle of the OLEX, followed by an observation period of 16 weeks each (48 weeks in total). This arm consisted of participants who participated in MP arms "Double-blind, MP: placebo (age 6 to 17 years)" and "Double-blind, MP: NT 201 (age 6 to 17 years)".	OLEX: NT 201 (Age 2 to 5 Years) Participants received NT 201 (about 1.5-2 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands on Day 1 of second (Visit 6), third (Visit 10), and fourth (Visit 14) injection cycle of the OLEX, followed by an observation period of 16 weeks each (48 weeks in total).
Change From Baseline in uSFR at Weeks 8 and 12 Change at Week 8	-0.07 g/min Standard Error 0.015	-0.16 g/min Standard Error 0.012	—	—	—
Change From Baseline in uSFR at Weeks 8 and 12 Change at Week 12	-0.06 g/min Standard Error 0.016	-0.16 g/min Standard Error 0.013	—	—	—

SECONDARY outcome

Timeframe: Weeks 8 and 12

Population: The FAS is identical to the subset of participants in the SES (MP) where subset of all participants received study medication (NT 201 or placebo) during the MP of the study.

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).

Outcome measures

Outcome measures
Measure	Double-blind MP: Placebo (Age 6 to 17 Years) n=72 Participants Participants received placebo via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks. Volumes were matched to the volumes of NT 201 (incobotulinumtoxinA; Xeomin) injected in the experimental arm.	Double-blind, MP: NT 201 (Age 6 to 17 Years) n=148 Participants Participants received NT 201 (up to 2.5 Units per kilogram \[U/kg\] body weight) via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks.	Open-label, MP: NT 201 (Age 2 to 5 Years) Participants received NT 201 (about 1.5-2 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks.	OLEX: NT 201 (Age 6 to 17 Years) Participants received NT 201 (up to 2.5 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands on Day 1 of second (Visit 6), third (Visit 10), and fourth (Visit 14) injection cycle of the OLEX, followed by an observation period of 16 weeks each (48 weeks in total). This arm consisted of participants who participated in MP arms "Double-blind, MP: placebo (age 6 to 17 years)" and "Double-blind, MP: NT 201 (age 6 to 17 years)".	OLEX: NT 201 (Age 2 to 5 Years) Participants received NT 201 (about 1.5-2 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands on Day 1 of second (Visit 6), third (Visit 10), and fourth (Visit 14) injection cycle of the OLEX, followed by an observation period of 16 weeks each (48 weeks in total).
GICS at Weeks 8 and 12 Week 8	0.54 units on a scale Standard Error 0.096	0.94 units on a scale Standard Error 0.068	—	—	—
GICS at Weeks 8 and 12 Week 12	0.47 units on a scale Standard Error 0.111	0.87 units on a scale Standard Error 0.073	—	—	—

SECONDARY outcome

Timeframe: Baseline up to Week 64

Outcome measures

Outcome measures
Measure	Double-blind MP: Placebo (Age 6 to 17 Years) n=72 Participants Participants received placebo via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks. Volumes were matched to the volumes of NT 201 (incobotulinumtoxinA; Xeomin) injected in the experimental arm.	Double-blind, MP: NT 201 (Age 6 to 17 Years) n=148 Participants Participants received NT 201 (up to 2.5 Units per kilogram \[U/kg\] body weight) via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks.	Open-label, MP: NT 201 (Age 2 to 5 Years) n=35 Participants Participants received NT 201 (about 1.5-2 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks.	OLEX: NT 201 (Age 6 to 17 Years) n=214 Participants Participants received NT 201 (up to 2.5 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands on Day 1 of second (Visit 6), third (Visit 10), and fourth (Visit 14) injection cycle of the OLEX, followed by an observation period of 16 weeks each (48 weeks in total). This arm consisted of participants who participated in MP arms "Double-blind, MP: placebo (age 6 to 17 years)" and "Double-blind, MP: NT 201 (age 6 to 17 years)".	OLEX: NT 201 (Age 2 to 5 Years) n=33 Participants Participants received NT 201 (about 1.5-2 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands on Day 1 of second (Visit 6), third (Visit 10), and fourth (Visit 14) injection cycle of the OLEX, followed by an observation period of 16 weeks each (48 weeks in total).
Occurrence of Treatment Emergent Adverse Events of Special Interest (AESI) Overall and by Injection Cycle Overall	0 Participants	1 Participants	0 Participants	4 Participants	0 Participants
Occurrence of Treatment Emergent Adverse Events of Special Interest (AESI) Overall and by Injection Cycle First injection cycle (MP)	0 Participants	1 Participants	0 Participants	—	—
Occurrence of Treatment Emergent Adverse Events of Special Interest (AESI) Overall and by Injection Cycle Second injection cycle (OLEX)	—	—	—	3 Participants	0 Participants
Occurrence of Treatment Emergent Adverse Events of Special Interest (AESI) Overall and by Injection Cycle Third injection cycle (OLEX)	—	—	—	1 Participants	0 Participants
Occurrence of Treatment Emergent Adverse Events of Special Interest (AESI) Overall and by Injection Cycle Fourth injection cycle (OLEX)	—	—	—	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Baseline up to Week 64

Outcome measures

Outcome measures
Measure	Double-blind MP: Placebo (Age 6 to 17 Years) n=72 Participants Participants received placebo via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks. Volumes were matched to the volumes of NT 201 (incobotulinumtoxinA; Xeomin) injected in the experimental arm.	Double-blind, MP: NT 201 (Age 6 to 17 Years) n=148 Participants Participants received NT 201 (up to 2.5 Units per kilogram \[U/kg\] body weight) via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks.	Open-label, MP: NT 201 (Age 2 to 5 Years) n=35 Participants Participants received NT 201 (about 1.5-2 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks.	OLEX: NT 201 (Age 6 to 17 Years) n=214 Participants Participants received NT 201 (up to 2.5 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands on Day 1 of second (Visit 6), third (Visit 10), and fourth (Visit 14) injection cycle of the OLEX, followed by an observation period of 16 weeks each (48 weeks in total). This arm consisted of participants who participated in MP arms "Double-blind, MP: placebo (age 6 to 17 years)" and "Double-blind, MP: NT 201 (age 6 to 17 years)".	OLEX: NT 201 (Age 2 to 5 Years) n=33 Participants Participants received NT 201 (about 1.5-2 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands on Day 1 of second (Visit 6), third (Visit 10), and fourth (Visit 14) injection cycle of the OLEX, followed by an observation period of 16 weeks each (48 weeks in total).
Occurrence of Treatment Emergent Serious Adverse Events (TESAEs) Overall and by Injection Cycle Overall	1 Participants	0 Participants	1 Participants	8 Participants	0 Participants
Occurrence of Treatment Emergent Serious Adverse Events (TESAEs) Overall and by Injection Cycle First injection cycle (MP)	1 Participants	0 Participants	1 Participants	—	—
Occurrence of Treatment Emergent Serious Adverse Events (TESAEs) Overall and by Injection Cycle Second injection cycle (OLEX)	—	—	—	3 Participants	0 Participants
Occurrence of Treatment Emergent Serious Adverse Events (TESAEs) Overall and by Injection Cycle Third injection cycle (OLEX)	—	—	—	5 Participants	0 Participants
Occurrence of Treatment Emergent Serious Adverse Events (TESAEs) Overall and by Injection Cycle Fourth injection cycle (OLEX)	—	—	—	0 Participants	0 Participants

SECONDARY outcome

Timeframe: Baseline up to Week 64

Outcome measures

Outcome measures
Measure	Double-blind MP: Placebo (Age 6 to 17 Years) n=72 Participants Participants received placebo via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks. Volumes were matched to the volumes of NT 201 (incobotulinumtoxinA; Xeomin) injected in the experimental arm.	Double-blind, MP: NT 201 (Age 6 to 17 Years) n=148 Participants Participants received NT 201 (up to 2.5 Units per kilogram \[U/kg\] body weight) via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks.	Open-label, MP: NT 201 (Age 2 to 5 Years) n=35 Participants Participants received NT 201 (about 1.5-2 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks.	OLEX: NT 201 (Age 6 to 17 Years) n=214 Participants Participants received NT 201 (up to 2.5 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands on Day 1 of second (Visit 6), third (Visit 10), and fourth (Visit 14) injection cycle of the OLEX, followed by an observation period of 16 weeks each (48 weeks in total). This arm consisted of participants who participated in MP arms "Double-blind, MP: placebo (age 6 to 17 years)" and "Double-blind, MP: NT 201 (age 6 to 17 years)".	OLEX: NT 201 (Age 2 to 5 Years) n=33 Participants Participants received NT 201 (about 1.5-2 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands on Day 1 of second (Visit 6), third (Visit 10), and fourth (Visit 14) injection cycle of the OLEX, followed by an observation period of 16 weeks each (48 weeks in total).
Occurrence of TEAEs Related to Treatment as Assessed by the Investigator Overall and by Injection Cycle Overall	0 Participants	2 Participants	1 Participants	10 Participants	0 Participants
Occurrence of TEAEs Related to Treatment as Assessed by the Investigator Overall and by Injection Cycle First injection cycle (MP)	0 Participants	2 Participants	1 Participants	—	—
Occurrence of TEAEs Related to Treatment as Assessed by the Investigator Overall and by Injection Cycle Second injection cycle (OLEX)	—	—	—	5 Participants	0 Participants
Occurrence of TEAEs Related to Treatment as Assessed by the Investigator Overall and by Injection Cycle Third injection cycle (OLEX)	—	—	—	5 Participants	0 Participants
Occurrence of TEAEs Related to Treatment as Assessed by the Investigator Overall and by Injection Cycle Fourth injection cycle (OLEX)	—	—	—	1 Participants	0 Participants

SECONDARY outcome

Timeframe: Baseline up to Week 64

Outcome measures

Outcome measures
Measure	Double-blind MP: Placebo (Age 6 to 17 Years) n=72 Participants Participants received placebo via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks. Volumes were matched to the volumes of NT 201 (incobotulinumtoxinA; Xeomin) injected in the experimental arm.	Double-blind, MP: NT 201 (Age 6 to 17 Years) n=148 Participants Participants received NT 201 (up to 2.5 Units per kilogram \[U/kg\] body weight) via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks.	Open-label, MP: NT 201 (Age 2 to 5 Years) n=35 Participants Participants received NT 201 (about 1.5-2 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks.	OLEX: NT 201 (Age 6 to 17 Years) n=214 Participants Participants received NT 201 (up to 2.5 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands on Day 1 of second (Visit 6), third (Visit 10), and fourth (Visit 14) injection cycle of the OLEX, followed by an observation period of 16 weeks each (48 weeks in total). This arm consisted of participants who participated in MP arms "Double-blind, MP: placebo (age 6 to 17 years)" and "Double-blind, MP: NT 201 (age 6 to 17 years)".	OLEX: NT 201 (Age 2 to 5 Years) n=33 Participants Participants received NT 201 (about 1.5-2 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands on Day 1 of second (Visit 6), third (Visit 10), and fourth (Visit 14) injection cycle of the OLEX, followed by an observation period of 16 weeks each (48 weeks in total).
Occurrence of TEAEs Leading to Discontinuation Overall and by Injection Cycle Overall	1 Participants	1 Participants	1 Participants	4 Participants	0 Participants
Occurrence of TEAEs Leading to Discontinuation Overall and by Injection Cycle First injection cycle (MP)	1 Participants	1 Participants	1 Participants	—	—
Occurrence of TEAEs Leading to Discontinuation Overall and by Injection Cycle Second injection cycle (OLEX)	—	—	—	2 Participants	0 Participants
Occurrence of TEAEs Leading to Discontinuation Overall and by Injection Cycle Third injection cycle (OLEX)	—	—	—	2 Participants	0 Participants
Occurrence of TEAEs Leading to Discontinuation Overall and by Injection Cycle Fourth injection cycle (OLEX)	—	—	—	0 Participants	0 Participants

Adverse Events

Double-blind MP: Placebo (Age 6 to 17 Years)

Serious events: 1 serious events

Other events: 3 other events

Deaths: 0 deaths

Double-blind, MP: NT 201 (Age 6 to 17 Years)

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Open-label, MP: NT 201 (Age 2 to 5 Years)

Serious events: 1 serious events

Other events: 2 other events

Deaths: 0 deaths

OLEX: NT 201 (Age 6 to 17 Years)

Serious events: 8 serious events

Other events: 34 other events

Deaths: 0 deaths

OLEX: NT 201 (Age 2 to 5 Years)

Serious events: 0 serious events

Other events: 12 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Double-blind MP: Placebo (Age 6 to 17 Years) n=72 participants at risk Participants received placebo via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks. Volumes were matched to the volumes of NT 201 (incobotulinumtoxinA; Xeomin) injected in the experimental arm.	Double-blind, MP: NT 201 (Age 6 to 17 Years) n=148 participants at risk Participants received NT 201 (up to 2.5 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks.	Open-label, MP: NT 201 (Age 2 to 5 Years) n=35 participants at risk Participants received NT 201 (about 1.5-2 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks.	OLEX: NT 201 (Age 6 to 17 Years) n=214 participants at risk Participants received NT 201 (up to 2.5 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands on Day 1 of second (Visit 6), third (Visit 10), and fourth (Visit 14) injection cycle of the OLEX, followed by an observation period of 16 weeks each (48 weeks in total). This arm consisted of participants who participated in MP arms "Double-blind, MP: placebo (age 6 to 17 years)" and "Double-blind, MP: NT 201 (age 6 to 17 years)".	OLEX: NT 201 (Age 2 to 5 Years) n=33 participants at risk Participants received NT 201 (about 1.5-2 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands on Day 1 of second (Visit 6), third (Visit 10), and fourth (Visit 14) injection cycle of the OLEX, followed by an observation period of 16 weeks each (48 weeks in total).
Infections and infestations Staphylococcal bacteraemia	0.00% 0/72 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/148 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	2.9% 1/35 • Number of events 1 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/214 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/33 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.
Nervous system disorders Generalised tonic-clonic seizure	0.00% 0/72 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/148 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	2.9% 1/35 • Number of events 1 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/214 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/33 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.
Nervous system disorders Epilepsy	1.4% 1/72 • Number of events 1 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/148 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/35 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/214 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/33 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.
Gastrointestinal disorders Functional gastrointestinal disorder	0.00% 0/72 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/148 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/35 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.93% 2/214 • Number of events 2 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/33 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.
Gastrointestinal disorders Gastrointestinal haemorrhage	0.00% 0/72 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/148 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/35 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.47% 1/214 • Number of events 1 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/33 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.
Gastrointestinal disorders Haematemesis	0.00% 0/72 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/148 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/35 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.47% 1/214 • Number of events 1 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/33 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.
Infections and infestations Gastroenteritis rotavirus	0.00% 0/72 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/148 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/35 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.47% 1/214 • Number of events 1 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/33 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.
Infections and infestations Influenza	0.00% 0/72 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/148 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/35 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.47% 1/214 • Number of events 2 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/33 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.
Infections and infestations Pneumonia	0.00% 0/72 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/148 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/35 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.47% 1/214 • Number of events 1 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/33 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.
Infections and infestations Pneumonia bacterial	0.00% 0/72 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/148 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/35 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.47% 1/214 • Number of events 2 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/33 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.
Infections and infestations Respiratory tract infection	0.00% 0/72 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/148 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/35 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.47% 1/214 • Number of events 1 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/33 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.
Injury, poisoning and procedural complications Foreign body in gastrointestinal tract	0.00% 0/72 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/148 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/35 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.47% 1/214 • Number of events 1 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/33 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.
Injury, poisoning and procedural complications Joint dislocation	0.00% 0/72 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/148 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/35 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.47% 1/214 • Number of events 1 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/33 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.
Blood and lymphatic system disorders Anaemia	0.00% 0/72 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/148 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/35 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.47% 1/214 • Number of events 1 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/33 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.
Musculoskeletal and connective tissue disorders Limb deformity	0.00% 0/72 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/148 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/35 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.47% 1/214 • Number of events 1 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/33 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.
Surgical and medical procedures Gastric operation	0.00% 0/72 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/148 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/35 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.47% 1/214 • Number of events 1 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/33 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.

Other adverse events

Other adverse events
Measure	Double-blind MP: Placebo (Age 6 to 17 Years) n=72 participants at risk Participants received placebo via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks. Volumes were matched to the volumes of NT 201 (incobotulinumtoxinA; Xeomin) injected in the experimental arm.	Double-blind, MP: NT 201 (Age 6 to 17 Years) n=148 participants at risk Participants received NT 201 (up to 2.5 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks.	Open-label, MP: NT 201 (Age 2 to 5 Years) n=35 participants at risk Participants received NT 201 (about 1.5-2 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands in one injection session on Day 1 (Visit 2) of the MP, followed by an observation period of 16 weeks.	OLEX: NT 201 (Age 6 to 17 Years) n=214 participants at risk Participants received NT 201 (up to 2.5 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands on Day 1 of second (Visit 6), third (Visit 10), and fourth (Visit 14) injection cycle of the OLEX, followed by an observation period of 16 weeks each (48 weeks in total). This arm consisted of participants who participated in MP arms "Double-blind, MP: placebo (age 6 to 17 years)" and "Double-blind, MP: NT 201 (age 6 to 17 years)".	OLEX: NT 201 (Age 2 to 5 Years) n=33 participants at risk Participants received NT 201 (about 1.5-2 U/kg body weight) via bilateral intraglandular injection into the parotid and submandibular glands on Day 1 of second (Visit 6), third (Visit 10), and fourth (Visit 14) injection cycle of the OLEX, followed by an observation period of 16 weeks each (48 weeks in total).
Infections and infestations Nasopharyngitis	4.2% 3/72 • Number of events 3 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	2.0% 3/148 • Number of events 3 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	5.7% 2/35 • Number of events 3 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	6.1% 13/214 • Number of events 16 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	3.0% 1/33 • Number of events 1 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.
Infections and infestations Pharyngitis	0.00% 0/72 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/148 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/35 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	5.6% 12/214 • Number of events 13 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	9.1% 3/33 • Number of events 3 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.
Infections and infestations Respiratory tract infection	0.00% 0/72 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/148 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/35 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	2.8% 6/214 • Number of events 6 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	9.1% 3/33 • Number of events 4 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.
Infections and infestations Viral infection	0.00% 0/72 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/148 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/35 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	1.9% 4/214 • Number of events 7 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	6.1% 2/33 • Number of events 3 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.
Infections and infestations Respiratory tract infection viral	0.00% 0/72 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/148 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/35 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.47% 1/214 • Number of events 2 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	12.1% 4/33 • Number of events 5 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.
Infections and infestations Rhinitis	0.00% 0/72 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/148 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.00% 0/35 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	0.93% 2/214 • Number of events 2 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.	9.1% 3/33 • Number of events 4 • Baseline up to Week 64 The investigator asked the participant for adverse events (AEs) systematically at each visit.

Additional Information

Public Disclosure Manager

Merz Pharmaceuticals GmbH

Phone: +49 69 1503 1

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Publication of study information usually requires agreement with the sponsor. In case of justified doubts by the sponsor, the INVESTIGATOR will consider these doubts in the publication as long as the scientific neutrality is not affected.
Publication restrictions are in place

Restriction type: OTHER