Phase 3
N=256
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
Chronic Troublesome Sialorrhea · Cerebral Palsy · Stroke · Traumatic Brain Injury · Intellectual Disability
Bottom Line
View on ClinicalTrials.gov: NCT02270736 ↗Enrolled (actual)
256
Serious AEs
2.0%
Results posted
Jan 2021
Primary outcome: Primary: Change From Baseline in Unstimulated Salivary Flow Rate (uSFR) at Week 4 — -0.07; -0.14 gram per minute (g/min) — p== 0.0012
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- NT 201 Placebo (Drug); NT 201 (Drug)
- Age
- Pediatric · 2+ yrs
- Sex
- All
- Sponsor
- Merz Pharmaceuticals GmbH
- Primary completion
- Feb 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in Unstimulated Salivary Flow Rate (uSFR) at Week 4 |
-0.07; -0.14 | = 0.0012 sig |
| PRIMARY Global Impression of Change Scale (GICS) at Week 4 Assessed by the Carer/Parent(s) |
0.63; 0.91 | = 0.032 sig |
| PRIMARY Occurrence of Treatment Emergent Adverse Events (TEAEs) Overall and Per Injection Cycle |
11; 27; 5; 92; 15; 11 | — |
| SECONDARY Change From Baseline in uSFR at Weeks 8 and 12 |
-0.07; -0.16; -0.06; -0.16 | <0.0001 sig |
| SECONDARY GICS at Weeks 8 and 12 |
0.54; 0.94; 0.47; 0.87 | =0.0008 sig |
| SECONDARY Occurrence of Treatment Emergent Adverse Events of Special Interest (AESI) Overall and by Injection Cycle |
0; 1; 0; 4; 0; 0 | — |
| SECONDARY Occurrence of Treatment Emergent Serious Adverse Events (TESAEs) Overall and by Injection Cycle |
1; 0; 1; 8; 0; 1 | — |
| SECONDARY Occurrence of TEAEs Related to Treatment as Assessed by the Investigator Overall and by Injection Cycle |
0; 2; 1; 10; 0; 0 | — |
| SECONDARY Occurrence of TEAEs Leading to Discontinuation Overall and by Injection Cycle |
1; 1; 1; 4; 0; 1 | — |
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.
Eligibility Criteria
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.
Data sourced from ClinicalTrials.gov (NCT02270736). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.