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

JRCT ID: jRCT2031230711

Registered date:15/03/2024

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Blepharospasm
• Date of first enrollment: 23/04/2024
• Target sample size: 25
• Study type: Interventional
• Intervention(s): NT 201 is injected into the affected muscles. Dosing period should be 42 weeks.

Outcome(s)

• Primary Outcome: Change from baseline in JRS severity score at 6 weeks after the first injection

Secondary Outcome

(1) JRS (severity score, frequency score, total score)
(2) Blepharospasm Disability Index (BSDI)
(3) Patient Evaluation of Global Response (PEGR)
(4) EQ-5D-5L
(5) Blink test
(6) Time to onset of treatment effect after injection
(7) Time to waning of treatment effect after injection
(8) Duration of treatment interval
(9) Investigator's Global Assessment of Efficacy

Key inclusion & exclusion criteria

• Age minimum: >= 18age old
• Age maximum: <= 80age old
• Gender: Both

Include criteria

Subjects must meet all of the following inclusion criteria.
(1) Written Informed consent obtained from subject before participating
(2) Age >= 18 years and =< 80 years at the time of informed consent
(3) A clinical diagnosis of blepharospasm
(4) Jankovic Rating Scale (JRS) severity score >= 2 at the screening visit
(5) Only for pretreated patients:
At least 10 weeks must have passed between the last injection with botulinum toxin for blepharospasm and the screening visit
(6) Only for pretreated patients:
Patients presenting with symptoms of blepharospasm and need for injection of botulinum toxin.

Exclude criteria

Subjects shall be excluded from this study if any of the following exclusion criteria.
(1) Psychotropic drugs induced blepharospasm
(2) The main symptom is apraxia of the eyelid opening
(3) Only for pretreated patients: No therapeutic response was observed with botulinum treatment for blepharospasm
(4) Treatment with botulism toxins for any injection other than blepharospasm (hemifacial spasm, cervical dystonia, cosmetics, etc.) within 16 weeks prior to the screening visit
(5) History of surgery for the treatment of dystonia including blepharospasm and myotomy surgery in the affected muscles
(6) Patients who have initiated, discontinued, or changed pharmaceuticals for the treatment of dystonia including blepharospasm within 12 weeks prior to screening visit
(7) Use any other investigational drugs/medical devices/products within 12 weeks prior to screening visit
(8) Patients who are planning to receive the prohibited concomitant medications from screening visit until the final visit
(9) Patients who are planning to start, discontinue or change the restricted concomitant medications by the end of the primary outcome measure from screening visit
(10) Myasthenia gravis, Lambert-Eaton syndrome, amyotrophic lateral sclerosis, or any other significant neuromuscular disease
(11) Infection in the area of the planned injection points or concerns about worsening systemic infection due to the local injection into the affected muscles
(12) Hypersensitivity to human serum albumin, sucrose, or Botulinum toxin A
(13) Pregnant women, patients who may be pregnant, patients who wish to become pregnant during the study period, or nursing mothers
(14) Women of childbearing potential who are unwilling or unable to use highly effective contraception from the date of informed consent until 16 weeks after the last injection of NT 201. [e.g., intrauterine device (IUD), intrauterine contraceptive system (IUS), oral contraceptives (low-dose pills), contraceptive surgery, or double barrier methods (e.g. condoms with spermicide, or condoms and pessaries) ]
(15) Severe or uncontrolled systemic diseases (e.g., cardiac, renal, pulmonary, hepatic, or gastrointestinal), malignant tumors, or known HIV infection in medical history
(16) Known or history of alcoholism or substance abuse
(17) Other than the above, patients judged by the investigator to be unsuitable for this study

Related Information

• Primary Sponsor: Hashizuka Masahiro