Open-label, Single-arm Study of NT 201 in Patients With Chronic Sialorrhea

JRCT ID: jRCT2051210082

Registered date:13/09/2021

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: chronic sialorrhea
• Date of first enrollment: 04/11/2021
• Target sample size: 80
• Study type: Interventional
• Intervention(s): NT 201(100U) is administered intraglandally once every 16 weeks for a total three times.

Outcome(s)

• Primary Outcome: Change in Unstimulated Salivary Flow Rate (uSFR) from pre-first study treatment (baseline) to 4 weeks post-treatment in Group A

Key inclusion & exclusion criteria

• Age minimum: >= 20age old
• Age maximum: <= 80age old
• Gender: Both

Include criteria

(1) Patients for whom written informed consent has been obtained from the patient or the legally acceptable representative before participating in the clinical trial.
(2) Men and women aged >=20 and <=80 years at the time of informed consent
(3) Patients who meet all of the following 1) to 3) at the pre-enrollment examination
1) Drooling severity and frequency scale (DSFS) with a total score of 6 or more.
2) DSFS scores of 2 or more for each item (severity and frequency)
3) Modified Radboud Oral Motor Inventory for Parkinson's Disease (mROMP) Drooling A) score of 3 or more
(4) Patients continuously meeting the inclusion criteria (3) from 12 weeks or more before the day of pre-enrollment examination.
(5) Patients who have any disease presumed as a causes of sialorrhea [e.g., Parkinson's disease, atypical parkinsonism, stroke, traumatic brain injury, cerebral palsy, amyotrophic lateral sclerosis (ALS), muscular dystrophy, etc.]

[Group A]:
Patients who meet both 1) and 2) below
1) Patients who have been diagnosed with any of the following diseases i) to iv) and have had the disease for at least 24 weeks since the onset of the disease by the day of the pre-enrollment examination.
i) Isolated Parkinson's disease or hereditary Parkinson's disease diagnosed according to criteria of UK Parkinson's Disease Brain Bank (UKPDSBB)
ii) Clinically diagnosed atypical parkinsonism following a) to c)
a) Multiple system atrophy
b) Progressive supranuclear palsy
c) Corticobasal degeneration
iii) Stroke
iv) Traumatic brain injury
2) Individuals who are able to comply with the schedules of observation and examination specified in Group A, including uSFR measurements

[Group B]:
Patients not applicable to [Group A]. However, after the sponsor has completed enrollment in Group A, patients who fall into Group A can also be enrolled in Group B.

Exclude criteria

(1) Patients with a score of 3 or more on mROMP Swallowing symptoms A) or a score of 4 or more on the C) regarding swallowing function at the pre-enrollment examination. However, patients who use a tube feeding is used for nutritional support do not fall under mROMP Swallowing symptoms C) 5 "I had to use a feeding tube".
(2) Patients who have experienced aspiration pneumonitis twice or more, patients who had the experience once and are judged by the investigator to be at increased risk of the recurrence, or patients who have not had gastrostomy after TPPV induction.
(3) Patients with generalized neuromuscular junction disorders (such as myasthenia gravis and Lambert-Eaton syndrome)
(4) Patients with a history (or complication) of infection or tumor in the salivary glands or at the intended injection site. However, infections in childhood such as parotitis are permissible.
(5) Patients who are judged by the investigator to be at risk of preventing a safe participation in the clinical trial due to extremely poor dental oral health status.
(6) Patients who have received other investigational or unapproved drugs within 14 weeks prior to the date of the pre-registration examination.
(7) Patients who have undergone functional neurosurgery (deep brain stimulation, stereotactic disruption) or focused ultrasound within 24 weeks prior to the date of the pre-registration examination
(8) Patients who have previously undergone head and neck surgery or radiotherapy (e.g., salivary gland surgery or salivary gland radiotherapy) for the treatment of sialorrhea.
(9) Patients who are receiving muscle relaxants/drugs with muscle relaxant effects and anticoagulants. However, aspirin and antiplatelet agents are permissible.
(10) Patients who need to use (implementation) a prohibited concomitant medication or change in dosage and administration of a restricted concomitant medication after the date of the pre-registration examination

[Exclusion Criteria Applied Only to Group A].
(11) Patients who are unable to place the saliva collection swabs in their mouth and keep resting for 5 minutes
(12) Patients who are unable to open their mouth spontaneously
(13) Patients with Alzheimer's disease or other dementias who are judged by the investigator to have an impact on the evaluation of the study.

[Exclusion Criteria Applied to Group B Only].
(14) [Exclusion criteria only applicable to patients with ALS].
Patients who have a progressive decline in respiratory function and may receive noninvasive positive pressure ventilation (NPPV) or tracheostomy-guided positive pressure ventilation (TPPV) in 1 yr. However, those who have already undergone TPPV are not eligible for this exclusion criterion.
(15) [Exclusion criteria only applicable to patients with ALS].
Patients whose percent forced vital capacity (%FVC) is <80% at the pre-enrollment examination. However, those who have already undergone TPPV are not eligible for this exclusion criterion.

Related Information

• Primary Sponsor: Ishikawa Tetsuro