Skip to main content
OpenTrials
Completed

NCT Number: NCT02270736

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

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Chronic Troublesome Sialorrhea Brain Damage, Chronic Brain Diseases Brain Injuries Brain Injuries, Traumatic Cardiovascular Diseases Central Nervous System Diseases Cerebral Palsy Cerebrovascular Disorders Craniocerebral Trauma Intellectual Disability Mental Disorders Nervous System Diseases Neurobehavioral Manifestations Neurodevelopmental Disorders Neurologic Manifestations Pathological Conditions, Signs and Symptoms Signs and Symptoms Stroke Trauma, Nervous System Traumatic Brain Injury Vascular Diseases Wounds and Injuries

Age range

2 year–17 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Merz Investigational Site #9950003, K'obulet'i, Georgia

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

  • 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 hypersensitivity 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.

Treatment and study plan

NT 201 Placebo

Drug

NT 201 placebo matching injection.

NT 201

Drug

NT 201 injection.

Other names: IncobotulinumtoxinA, Xeomin, Botulinum toxin type A (150 kiloDalton), free from complexing proteins

Primary outcomes

  1. Change From Baseline in Unstimulated Salivary Flow Rate (uSFR) at Week 4

    Time frame: Baseline and 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.

  2. Global Impression of Change Scale (GICS) at Week 4 Assessed by the Carer/Parent(s)

    Time frame: Week 4

    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).

  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

    Time frame: Baseline and 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.

  2. GICS at Weeks 8 and 12

    Time frame: 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).

  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

Sponsors and collaborators

Lead sponsor

Merz Pharmaceuticals GmbH

Industry

Registry information

Official study title

Prospective, Randomized, Double-blind, Placebo-controlled, Parallel-group, Multicenter Study With an Open-label Extension Period to Investigate the Efficacy and Safety of NT 201 in the Treatment of Children and Adolescents (2-17 Years) With Chronic Troublesome Sialorrhea Associated With Neurological Disorders, and/or Intellectual Disability

Acronym: SIPEXI

Important dates

Study start
2015
Primary completion
2018
Study completion
2019
First posted
Oct 21, 2014
Registry last updated
Aug 10, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.