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CompletedNCT02270736SIPEXIUpdated Aug 10, 2021Results posted

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

A Phase 3 interventional study of NT 201 Placebo and NT 201 in Chronic Troublesome Sialorrhea, Cerebral Palsy and Stroke, sponsored by Merz Pharmaceuticals GmbH. Completed at 31 sites in 6 countries. Open to participants aged 2 Years to 17 Years. Per ClinicalTrials.gov, last updated 2021-08-10.

Sponsored by Merz Pharmaceuticals GmbH (part of Merz Pharma) · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
256
Allocation
Randomized
Ages
2 Years to 17 Years
Sex
All
01

Study summary

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.

02

Conditions studied

  • Chronic Troublesome Sialorrhea
  • Cerebral Palsy
  • Stroke
  • Traumatic Brain Injury
  • Intellectual Disability
03

In context

Cerebral Palsy

1,936 studies on the registry are indexed under Cerebral Palsy; 458 are open to participants now.

This study's enrollment of 256 is above the median of 34 across 1,431 interventional studies indexed under Cerebral Palsy.

Browse Cerebral Palsy studies →

Lead sponsor

Merz Pharmaceuticals GmbH is the lead sponsor of 61 studies on the registry; none are open to participants now.

Browse Merz Pharmaceuticals GmbH studies →All Merz Pharma studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
2 Years to 17 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  • Male or female child/adolescent age 2-17 years.
  • Any neurological disorder (e.g. cerebral palsy or traumatic brain injury) and/or intellectual disability associated with chronic troublesome sialorrhea for at least 3 months up to the screening. In subjects with intellectual disability (ID) without neurological disorders, a diagnosis of ID by a specialist, e.g. pediatrician or by a center for developmental medicine is required for inclusion.
  • Severe drooling (modified Teacher´s Drooling Scale [mTDS] ≥ 6; clothing occasionally becomes damp) as rated by the investigator.
  • Parental consent and the subject's oral or written assent as the subject is able to provide.

Exclusion criteria

Exclusion Criteria:

  • Chronic troublesome sialorrhea not related to neurological disorders and/or intellectual disability.
  • Body weight \< 12 kg.
  • Pharmacological treatment for sialorrhea or concomitant medication known to influence sialorrhea strongly (e.g. anticholinergics with exception of locally applied or short acting drugs used under general anesthesia) within 45 days before baseline and during the entire study period.
  • Any previous known or suspected to Botulinum toxin.
  • Aspiration pneumonia within 6 month before screening.
  • Any previous treatment with Botulinum toxin for any body region during the year before screening or within the screening period
  • Prior, concomitant or planned surgery or irradiation to head and neck to control sialorrhea (including salivary gland surgery or salivary gland irradiation) within one year before screening or planned for any part of the entire study period.
  • Concurrent diseases, including hematological, hepatic, renal, gastrointestinal, endocrine, pulmonary, musculoskeletal, or psychiatric diseases or conditions, which in the judgment of the investigator would put the subject at risk while in the study, could influence the results of the study, or negatively impact the subject's ability to participate in the study.
  • Extremely poor dental and/or oral condition that might preclude safe study participation by the judgment of the investigator.
  • Nursing mother or pregnant female subject.
Terms in these criteria, in plain language(1)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Hypersensitivity

An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.

05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Double (Participant, Investigator)
Enrollment
256 participants (actual)

Study arms

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

    Participants will receive 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 will be matched to the volumes of NT 201 (incobotulinumtoxinA; Xeomin) injected in the experimental arm.

    Drug: NT 201 Placebo

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

    Participants will receive 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.

    Drug: NT 201

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

    Participants will receive 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.

    Drug: NT 201

  • Experimental
    OLEX: NT 201 (Age 6 to 17 Years)

    Participants will receive 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 will consist of participants who will participate in MP arms "Double-blind, MP: placebo (age 6 to 17 years)" and "Double-blind, MP: NT 201 (age 6 to 17 years)".

    Drug: NT 201

  • Experimental
    OLEX: NT 201 (Age 2 to 5 Years)

    Participants will receive 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).

    Drug: NT 201

Interventions

  • DrugNT 201 Placebo

    NT 201 placebo matching injection.

  • DrugNT 201

    NT 201 injection.

    Also known as: IncobotulinumtoxinA, Xeomin, Botulinum toxin type A (150 kiloDalton), free from complexing proteins

06

What researchers measure

Primary outcomes

  1. 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

  2. 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

  3. Occurrence of Treatment Emergent Adverse Events (TEAEs) Overall and Per Injection Cycle

    Time frame: Baseline up to Week 64

Secondary outcomes

  1. 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

  2. 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

  3. Occurrence of Treatment Emergent Adverse Events of Special Interest (AESI) Overall and by Injection Cycle

    Time frame: Baseline up to Week 64

  4. Occurrence of Treatment Emergent Serious Adverse Events (TESAEs) Overall and by Injection Cycle

    Time frame: Baseline up to Week 64

  5. Occurrence of TEAEs Related to Treatment as Assessed by the Investigator Overall and by Injection Cycle

    Time frame: Baseline up to Week 64

  6. Occurrence of TEAEs Leading to Discontinuation Overall and by Injection Cycle

    Time frame: Baseline up to Week 64

07

Results

Posted Jan 20, 2021

Participant flow

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

Main Period (MP) (up to 16 Weeks)
Participant flow — Main Period (MP) (up to 16 Weeks)
MilestoneDouble-blind MP: Placebo (Age 6 to 17 Years)Double-blind, MP: NT 201 (Age 6 to 17 Years)Open-label, MP: NT 201 (Age 2 to 5 Years)OLEX: NT 201 (Age 6 to 17 Years)OLEX: NT 201 (Age 2 to 5 Years)
Started721483600
Treated721483500
Completed701463400
Not completed22200
Withdrew: Adverse event01000
Withdrew: Withdrawal by subject20100
Withdrew: Lost to follow-up01000
Withdrew: Randomized, not treated00100
OLEX Period (up to 48 Weeks)
Participant flow — OLEX Period (up to 48 Weeks)
MilestoneDouble-blind MP: Placebo (Age 6 to 17 Years)Double-blind, MP: NT 201 (Age 6 to 17 Years)Open-label, MP: NT 201 (Age 2 to 5 Years)OLEX: NT 201 (Age 6 to 17 Years)OLEX: NT 201 (Age 2 to 5 Years)
Started00021433
Completed00018933
Not completed000250
Withdrew: Physician decision00010
Withdrew: Lack of efficacy00020
Withdrew: Adverse event00040
Withdrew: Withdrawal by subject000160
Withdrew: Lost to follow-up00020

Outcome measures

PrimaryChange 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
Reported as:
Least squares mean · gram per minute (g/min)
Change From Baseline in Unstimulated Salivary Flow Rate (uSFR) at Week 4
gram per minute (g/min)Double-blind MP: Placebo (Age 6 to 17 Years)Double-blind, MP: NT 201 (Age 6 to 17 Years)
Change From Baseline in Unstimulated Salivary Flow Rate (uSFR) at Week 4-0.07 ± 0.015-0.14 ± 0.012
Statistical analysis
  • Double-blind MP: Placebo (Age 6 to 17 Years) vs Double-blind, MP: NT 201 (Age 6 to 17 Years) · MMRM · p = = 0.0012 · Least square mean (ls-mean) difference: -0.06 · 95% CI -0.1 to -0.03
PrimaryGlobal 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
Reported as:
Least squares mean · units on a scale
Global Impression of Change Scale (GICS) at Week 4 Assessed by the Carer/Parent(s)
units on a scaleDouble-blind MP: Placebo (Age 6 to 17 Years)Double-blind, MP: NT 201 (Age 6 to 17 Years)
Global Impression of Change Scale (GICS) at Week 4 Assessed by the Carer/Parent(s)0.63 ± 0.1040.91 ± 0.075
Statistical analysis
  • Double-blind MP: Placebo (Age 6 to 17 Years) vs Double-blind, MP: NT 201 (Age 6 to 17 Years) · MMRM · p = = 0.032 · Least square mean (ls-mean) difference: 0.28 · 95% CI 0.02 to 0.53
PrimaryOccurrence of Treatment Emergent Adverse Events (TEAEs) Overall and Per Injection Cycle
Time frame:
Baseline up to Week 64
Reported as:
Count of participants · Participants
Occurrence of Treatment Emergent Adverse Events (TEAEs) Overall and Per Injection Cycle
ParticipantsDouble-blind MP: Placebo (Age 6 to 17 Years)Double-blind, MP: NT 201 (Age 6 to 17 Years)Open-label, MP: NT 201 (Age 2 to 5 Years)OLEX: NT 201 (Age 6 to 17 Years)OLEX: NT 201 (Age 2 to 5 Years)
Overall112759215
First injection cycle (MP)11275——
Second injection cycle (OLEX)———447
Third injection cycle (OLEX)———355
Fourth injection cycle (OLEX)———4011
SecondaryChange 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
Reported as:
Least squares mean · g/min
Change From Baseline in uSFR at Weeks 8 and 12
g/minDouble-blind MP: Placebo (Age 6 to 17 Years)Double-blind, MP: NT 201 (Age 6 to 17 Years)
Change at Week 8-0.07 ± 0.015-0.16 ± 0.012
Change at Week 12-0.06 ± 0.016-0.16 ± 0.013
Statistical analysis
  • Double-blind MP: Placebo (Age 6 to 17 Years) vs Double-blind, MP: NT 201 (Age 6 to 17 Years) · MMRM · p = <0.0001 · Least square mean (ls-mean) difference: -0.09 · 95% CI -0.12 to -0.05
  • Double-blind MP: Placebo (Age 6 to 17 Years) vs Double-blind, MP: NT 201 (Age 6 to 17 Years) · MMRM · p = <0.0001 · Least square mean (ls-mean) difference: -0.1 · 95% CI -0.14 to -0.06
SecondaryGICS 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
Reported as:
Least squares mean · units on a scale
GICS at Weeks 8 and 12
units on a scaleDouble-blind MP: Placebo (Age 6 to 17 Years)Double-blind, MP: NT 201 (Age 6 to 17 Years)
Week 80.54 ± 0.0960.94 ± 0.068
Week 120.47 ± 0.1110.87 ± 0.073
Statistical analysis
  • Double-blind MP: Placebo (Age 6 to 17 Years) vs Double-blind, MP: NT 201 (Age 6 to 17 Years) · MMRM · p = =0.0008 · Least square mean (ls-mean) difference: 0.4 · 95% CI 0.17 to 0.63
  • Double-blind MP: Placebo (Age 6 to 17 Years) vs Double-blind, MP: NT 201 (Age 6 to 17 Years) · MMRM · p = =0.0026 · Least square mean (ls-mean) difference: 0.4 · 95% CI 0.14 to 0.66
SecondaryOccurrence of Treatment Emergent Adverse Events of Special Interest (AESI) Overall and by Injection Cycle
Time frame:
Baseline up to Week 64
Reported as:
Count of participants · Participants
Occurrence of Treatment Emergent Adverse Events of Special Interest (AESI) Overall and by Injection Cycle
ParticipantsDouble-blind MP: Placebo (Age 6 to 17 Years)Double-blind, MP: NT 201 (Age 6 to 17 Years)Open-label, MP: NT 201 (Age 2 to 5 Years)OLEX: NT 201 (Age 6 to 17 Years)OLEX: NT 201 (Age 2 to 5 Years)
Overall01040
First injection cycle (MP)010——
Second injection cycle (OLEX)———30
Third injection cycle (OLEX)———10
Fourth injection cycle (OLEX)———00
SecondaryOccurrence of Treatment Emergent Serious Adverse Events (TESAEs) Overall and by Injection Cycle
Time frame:
Baseline up to Week 64
Reported as:
Count of participants · Participants
Occurrence of Treatment Emergent Serious Adverse Events (TESAEs) Overall and by Injection Cycle
ParticipantsDouble-blind MP: Placebo (Age 6 to 17 Years)Double-blind, MP: NT 201 (Age 6 to 17 Years)Open-label, MP: NT 201 (Age 2 to 5 Years)OLEX: NT 201 (Age 6 to 17 Years)OLEX: NT 201 (Age 2 to 5 Years)
Overall10180
First injection cycle (MP)101——
Second injection cycle (OLEX)———30
Third injection cycle (OLEX)———50
Fourth injection cycle (OLEX)———00
SecondaryOccurrence of TEAEs Related to Treatment as Assessed by the Investigator Overall and by Injection Cycle
Time frame:
Baseline up to Week 64
Reported as:
Count of participants · Participants
Occurrence of TEAEs Related to Treatment as Assessed by the Investigator Overall and by Injection Cycle
ParticipantsDouble-blind MP: Placebo (Age 6 to 17 Years)Double-blind, MP: NT 201 (Age 6 to 17 Years)Open-label, MP: NT 201 (Age 2 to 5 Years)OLEX: NT 201 (Age 6 to 17 Years)OLEX: NT 201 (Age 2 to 5 Years)
Overall021100
First injection cycle (MP)021——
Second injection cycle (OLEX)———50
Third injection cycle (OLEX)———50
Fourth injection cycle (OLEX)———10
SecondaryOccurrence of TEAEs Leading to Discontinuation Overall and by Injection Cycle
Time frame:
Baseline up to Week 64
Reported as:
Count of participants · Participants
Occurrence of TEAEs Leading to Discontinuation Overall and by Injection Cycle
ParticipantsDouble-blind MP: Placebo (Age 6 to 17 Years)Double-blind, MP: NT 201 (Age 6 to 17 Years)Open-label, MP: NT 201 (Age 2 to 5 Years)OLEX: NT 201 (Age 6 to 17 Years)OLEX: NT 201 (Age 2 to 5 Years)
Overall11140
First injection cycle (MP)111——
Second injection cycle (OLEX)———20
Third injection cycle (OLEX)———20
Fourth injection cycle (OLEX)———00

Adverse events

Collected over Baseline up to Week 64. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Double-blind MP: Placebo (Age 6 to 17 Years)0/72 (0%)1/72 (1.4%)3/72 (4.2%)
Double-blind, MP: NT 201 (Age 6 to 17 Years)0/148 (0%)0/148 (0%)3/148 (2%)
Open-label, MP: NT 201 (Age 2 to 5 Years)0/35 (0%)1/35 (2.9%)2/35 (5.7%)
OLEX: NT 201 (Age 6 to 17 Years)0/214 (0%)8/214 (3.7%)34/214 (15.9%)
OLEX: NT 201 (Age 2 to 5 Years)0/33 (0%)0/33 (0%)12/33 (36.4%)
Most frequent serious events
Showing 10 of 16
Most frequent serious events
EventDouble-blind MP: Placebo (Age 6 to 17 Years)Double-blind, MP: NT 201 (Age 6 to 17 Years)Open-label, MP: NT 201 (Age 2 to 5 Years)OLEX: NT 201 (Age 6 to 17 Years)OLEX: NT 201 (Age 2 to 5 Years)
Staphylococcal bacteraemiaInfections and infestations0/720/1481/350/2140/33
Generalised tonic-clonic seizureNervous system disorders0/720/1481/350/2140/33
EpilepsyNervous system disorders1/720/1480/350/2140/33
Functional gastrointestinal disorderGastrointestinal disorders0/720/1480/352/2140/33
Gastrointestinal haemorrhageGastrointestinal disorders0/720/1480/351/2140/33
HaematemesisGastrointestinal disorders0/720/1480/351/2140/33
Gastroenteritis rotavirusInfections and infestations0/720/1480/351/2140/33
InfluenzaInfections and infestations0/720/1480/351/2140/33
PneumoniaInfections and infestations0/720/1480/351/2140/33
Pneumonia bacterialInfections and infestations0/720/1480/351/2140/33
Most frequent other events
Most frequent other events
EventDouble-blind MP: Placebo (Age 6 to 17 Years)Double-blind, MP: NT 201 (Age 6 to 17 Years)Open-label, MP: NT 201 (Age 2 to 5 Years)OLEX: NT 201 (Age 6 to 17 Years)OLEX: NT 201 (Age 2 to 5 Years)
Respiratory tract infection viralInfections and infestations0/720/1480/351/2144/33
PharyngitisInfections and infestations0/720/1480/3512/2143/33
Respiratory tract infectionInfections and infestations0/720/1480/356/2143/33
RhinitisInfections and infestations0/720/1480/352/2143/33
NasopharyngitisInfections and infestations3/723/1482/3513/2141/33
Viral infectionInfections and infestations0/720/1480/354/2142/33

Baseline characteristics

The safety evaluable set (SES) (MP) is the subset of all participants who received study medication (NT 201 or placebo) during the MP of the study.

Age, Customized
Age, Customized(Participants)Double-blind MP: Placebo (Age 6 to 17 Years)Double-blind, MP: NT 201 (Age 6 to 17 Years)Open-label, MP: NT 201 (Age 2 to 5 Years)Total
In utero0000
Preterm newborn infants (gestational age < 37 wks)0000
Newborns (0-27 days)0000
Infants and toddlers (28 days-23 months)0000
Children (2-11 years)489635179
Adolescents (12-17 years)2452076
Adults (18-64 years)0000
From 65-84 years0000
85 years and over0000
Sex: Female, Male
Sex: Female, Male(Participants)Double-blind MP: Placebo (Age 6 to 17 Years)Double-blind, MP: NT 201 (Age 6 to 17 Years)Open-label, MP: NT 201 (Age 2 to 5 Years)Total
Female27551395
Male459322160
Race/Ethnicity, Customized
Race/Ethnicity, Customized(Participants)Double-blind MP: Placebo (Age 6 to 17 Years)Double-blind, MP: NT 201 (Age 6 to 17 Years)Open-label, MP: NT 201 (Age 2 to 5 Years)Total
White7214835255
Black or African American0000
Height
Height(centimeter (cm))Double-blind MP: Placebo (Age 6 to 17 Years)Double-blind, MP: NT 201 (Age 6 to 17 Years)Open-label, MP: NT 201 (Age 2 to 5 Years)Total
Mean135.3 ± 16.92132.8 ± 17.15101.1 ± 8.09129.1 ± 19.64
Weight
Weight(kilogram (kg))Double-blind MP: Placebo (Age 6 to 17 Years)Double-blind, MP: NT 201 (Age 6 to 17 Years)Open-label, MP: NT 201 (Age 2 to 5 Years)Total
Mean30.8 ± 11.6728.8 ± 11.4815.7 ± 3.0027.6 ± 11.78
Body Mass Index (BMI)
Body Mass Index (BMI)(kilogram per square meter (kg/m^2))Double-blind MP: Placebo (Age 6 to 17 Years)Double-blind, MP: NT 201 (Age 6 to 17 Years)Open-label, MP: NT 201 (Age 2 to 5 Years)Total
Mean16.4 ± 3.6515.8 ± 3.2515.3 ± 1.8515.9 ± 3.23
08

Study locations

31 sites
  • Merz Investigational Site #9950003
    Kobuleti, 6200, Georgia
  • Merz Investigational Site #9950001
    Tbilisi, 0159, Georgia
  • Merz Investigational Site #9950002
    Tbilisi, 0159, Georgia
  • Merz Investigational Site #0360017
    Balassagyarmat, 2660, Hungary
  • Merz Investigational Site #0360013
    Budapest, 1083, Hungary
  • Merz Investigational Site # 0360014
    Budapest, 1125, Hungary
  • Merz Investigational Site # 0360015
    Budapest, 1125, Hungary
  • Merz Investigational Site #0360018
    Budapest, 1146, Hungary
  • Merz Investigational Site #0360016
    Szombathely, 9700, Hungary
  • Merz Investigational Site #0480092
    Bialystok, 15-274, Poland
  • Merz Investigational Site #0480090
    Gdansk, 80-952, Poland
  • Merz Investigational Site #0480076
    Katowice, 40-954, Poland
  • Merz Investigational Site #0480059
    Krakow, 30-359, Poland
  • Merz Investigational Site #0480060
    Wiazowna, 05-462, Poland
  • Merz Investigational Site #0070016
    Kazan, 420012, Russian Federation
  • Merz Investigational Site # 0070288
    Kemerovo, 650066, Russian Federation
  • Merz Investigational Site #0070290
    Khabarovsk, 680038, Russian Federation
  • Merz Investigational # 0070017
    Saint Petersburg, 194100, Russian Federation
  • Merz Investigational Site #0070013
    Smolensk, 214018, Russian Federation
  • Merz Investigational Site # 070019
    Stavropol, 355029, Russian Federation
  • Merz Investigational Site #0070300
    Tomsk, 634052, Russian Federation
  • Merz Investigational Site #0070301
    Yekaterinburg, 620149, Russian Federation
  • Merz Investigational Site #3810001
    Belgrade, 11040, Serbia
  • Merz Investigational Site #3800001
    Dnipropetrovsk, 49027, Ukraine
  • Merz Investigational Site #3800012
    Ivano-Frankivsk, 76014, Ukraine
  • Merz Investigational Site #3800005
    Kharkiv, 61068, Ukraine
  • Merz Investigational Site #3800007
    Kharkiv, 61153, Ukraine
  • Merz Investigational Site #3800013
    Kherson, 73010, Ukraine
  • Merz Investigational site #3800003
    Odesa, 65012, Ukraine
  • Merz Investigational Site #3800009
    Ternopil, 46020, Ukraine
  • Merz Investigational Site #3800011
    Zaporizhzhya, 69063, Ukraine
09

References and documents

Publications

  • Berweck S, Bonikowski M, Kim H, Althaus M, Flatau-Baque B, Mueller D, Banach MD. Placebo-Controlled Clinical Trial of IncobotulinumtoxinA for Sialorrhea in Children: SIPEXI. Neurology. 2021 Oct 4;97(14):e1425-e1436. doi: 10.1212/WNL.0000000000012573. PubMed 34341153 ↗
  • Berweck S, Banach M, Gaebler-Spira D, Chambers HG, Schroeder AS, Geister TL, Althaus M, Hanschmann A, Vacchelli M, Bonfert MV, Heinen F, Dabrowski E. Safety Profile and Lack of Immunogenicity of IncobotulinumtoxinA in Pediatric Spasticity and Sialorrhea: A Pooled Analysis. Toxins (Basel). 2022 Aug 25;14(9):585. doi: 10.3390/toxins14090585. PubMed 36136523 ↗

Study documents

PDFs hosted by the registry, ClinicalTrials.gov.

Individual participant data

Plan to share: No

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Aug 10, 2021, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT02270736
Lead sponsor
Merz Pharmaceuticals GmbH
Responsible party
Sponsor
First posted
Oct 21, 2014
Start date
Feb 9, 2015
Primary completion
Feb 23, 2018
Completion
May 7, 2019
Results posted
Jan 20, 2021
Last update
Aug 10, 2021

Study contacts

Merz Medical Expert
study director · Merz Pharmaceuticals GmbH

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

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Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Studies of the drug tested here: incobotulinumtoxinA

Other studies in this condition

Cerebral Palsy →

Other studies from this sponsor

Merz Pharmaceuticals GmbH →

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