An open-label, uncontrolled, single-arm study of NT 201 in patients with cervical dystonia

JRCT ID: jRCT2031230690

Registered date:12/03/2024

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: Cervical Dystonia
• Date of first enrollment: 10/04/2024
• Target sample size: 22
• Study type: Interventional
• Intervention(s): The investigator administers NT 201 to the patient's affected muscle. Dosing period should be 42 weeks.

Outcome(s)

• Primary Outcome: Change from baseline in the TWSTRS- Total score at 4 weeks after the first injection

Secondary Outcome

(1) TWSTRS-total score
(Excluding change from baseline to 4 week)
(2) TWSTRS Severity Score, TWSTRS Disability Score, TWSTRS Pain Score
(3) Cervical Dystonia Impact Profile (CDIP-58)
(4) Patient Evaluation of Global Response (PEGR)
(5) Global Assessment of Efficacy by Investigator (GAEI)
(6) Modified Tsui scale sub scale D (head tremor evaluation)
(7) Pain Visual Analogue Scale (VAS)
(8) EQ-5D-5L
(9) Duration of treatment
(10) Time to Onset of Treatment Effect After Injection
(11) Time to Waning of Treatment Effect After Injection

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: <= 75age old
• Gender: Both

Include criteria

The investigator will select subjects for Group A who meet all of the following criteria (1) to (10).
(1) Patients whose written informed consent has been obtained before participating in this study
(2) Patients aged 18 to 75 years at the time of informed consent
(3) A clinical diagnosed of cervical dystonia
(4) TWSTRS - total score >= 20 at the screening test
(5) TWSTRS - severity score >= 10 at the screening test
(6) TWSTRS - disability score >= 3 at the screening test
(7) TWSTRS - pain score >= 1 at the screening test
(8)For patients treated with a botulinum toxin therapy for cervical dystonia: At least 10 weeks must have been passed between the last injection with botulinum toxin for cervical dystonia and the screening test (The screening test can be conducted on the same day of the next 10 weeks or later after the last injection)
(9)For patients treated with a botulinum toxin therapy for cervical dystonia: The effect of the most recent botulinum treatment has disappeared in terms of the TWSTRS score at the time of the screening test, and the investigator has determined that the next botulinum toxin injection is required
(10)For patients treated with a botulinum toxin therapy for cervical dystonia: The investigator has determined that the therapeutic effect of the botulinum toxin preparation has been obtained with the most recent injection

The investigator will select subjects for Group B who meet all of (1), (2), (4) to (10), and the following criteria (11).
(11) A clinical diagnosed of tardive torticollis

Exclude criteria

[Exclusion criteria applicable to Group A and Group B]
(1) Traumatic torticollis and congenital torticollis
(2) Patients who only have symptoms of anterocollis, or among patients with symptoms other than anterocollis, patients with a TWSTRS - severity score for anterocollis >= 2 at the screening test
(3) Patients with a history of deep brain stimulation therapy, intrathecal baclofen therapy, or surgical therapy for cervical dystonia (myotomy, selective peripheral nerve blockade, etc.)
(4) Marked limitation on passive range of cervical motion that suggests contractures or other malformations
(5) Patients with systemic neuromuscular junction disorders (myasthenia gravis, Lambert-Eaton syndrome, etc.), patients with amyotrophic lateral sclerosis
(6) Treatment with botulinum toxins for any injection other than cervical dystonia within 16 weeks prior to baseline
(The screening test can be conducted on the same day of the next 16 weeks or later after the last injection)
(7) Patients who have been judged by the investigator to have had primary or secondary non-responder due to previous injection of botulinum toxin preparations
(8) Patients for whom one or more weeks have not passed since the last administration of anticoagulants (heparin, warfarin, etc.) by the date of the screening test (antiplatelet drugs are permitted)
(The screening test can be conducted on the same day of the next week or later after the last administration)
(9) Dysphagia scale >= 3: sever, with swallowing difficulties and requiring a change in diet
(10) Patients who need to perform the applicable prohibited concomitant treatment after the date of the screening test and until the completion of main period (excluding observation period after main period) or the completion of the final termination visit
(11) Patients whose dosage or administration of the applicable concomitant restricted drug(s) was changed in the last one week before the screening test (the screening test can be conducted on the same day of the next week or later after changing the dosage or administration), or patients whose dosage or administration of the applicable concomitant restricted drug(s) needs to be changed until the completion of the main period (excluding observation period thereafter)

[Exclusion criteria applicable only to Group A]
(12) Patients with tardive torticollis
(13) Patients who only have symptoms of retrocollis, or among patients with symptoms other than retrocollis, patients with a TWSTRS - severity score for retrocollis >= 2 at the screening test

Related Information

• Primary Sponsor: Sasaki Yuzuru