The Objective of This Study is to Evaluate the Efficacy and Safety of Botulax® in Patients With Essential Blepharospasm.

Multi-center, Single Arm, Open-label, Phase IV Clinical Trial to Evaluate the Efficacy and Safety of Botulax® in Patients With Essential Blepharospasm

The purpose of this study is evaluate the efficacy and safety of Botulax® in the treatment of Essential blepharospasm.

Study Overview

• Status: Completed
• Conditions: Essential Blepharospasm
• Intervention / Treatment: Drug: Botulinum toxin type A

Detailed Description

Multi-center, single arm, open-label, Phase IV clinical trial to evaluate the efficacy and safety of Botulax® in Subjects with essential blepharospasm

• Study Type: Interventional
• Enrollment (Actual): 249
• Phase: Phase 4

Contacts and Locations

Study Locations

• Korea, Republic of
  • Korea
    • Seoul, Korea, Korea, Republic of, 06162
      • Hugel

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Men and women at the age of 18 or older
2. Subjects diagnosed with essential blepharospasm with Jankovic Rating Scale (JRS) frequency and severity sum score ≥2 at screening
3. Individuals who agree to participate in the clinical study and voluntarily sign the written informed consent form
4. Subjects who are cooperative, have a good understanding of the clinical study, and can comply with study procedures until the end of the study

Exclusion Criteria:

1. Subjects who had received surgery such as orbicularis oculi resection or facial nerve block for the treatment of the study indication
2. Subjects with a history of hypersensitivity reactions to any of the components of the investigational product (botulinum toxins, serum albumin, etc.)
3. Subjects with secondary blepharospasm
4. Subjects with the hemifacial spasm
5. Subjects on treatment with muscle relaxants, benzodiazepines, anticholinergics, or benzamides who had changes in medication within 4 weeks prior to screening or have been on stable medication but are expected to have changes in medication while participating in the study
6. Subjects who administered any of the following drugs within 7 days prior to screening: drugs with muscle relaxation activity such as aminoglycoside antibiotics or other antibiotics (spectinomycin HCl, polypeptide antibiotics, tetracycline antibiotics, lincomycin antibiotics)
7. Subjects who administered medications similar to the investigational product (botulinum toxin type A) within 12 weeks
8. Subjects with systemic neuromuscular junction disorders: myasthenia gravis, Lambert-Eaton syndrome, or amyotrophic lateral sclerosis
9. Pregnant and lactating women
10. Women of childbearing potential who are planning to become pregnant within the next 3 months or are not using an appropriate method of contraception (contraceptive methods with an annual failure rate of less than 1% if used properly and continuously, including implant, injection, oral contraceptive, at least one barrier method such as intrauterine device [IUD], hormone IUD, abstinence, or vasectomized partner)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Botulinum Toxin Type A (Botulax)	Drug: Botulinum toxin type A; Recommended initial dose to be intra-muscularly injected to the medial and contralateral pretarsal orbicularis oculi of the upper eyelid and the contralateral pretarsal orbicularis oculi of the lower eyelid is 1.25-2.5U (0.05ml ~ 0.1ml per site).; Other Names: Botulax

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Total score change from baseline at 4 weeks post-injection of Jankovic Rating scale	To evaluate the change of JRS (Jankovic Rating scale) score at 4 weeks post treatment based on baseline(0 week). JRS total score will be calculated as the sum (0~8) of two sub-scores of severity and frequency (0~4 each for severity and frequency) of blepharospasm.	At 4 weeks post-injection

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Total score change from baseline at 16 weeks post-injection of Jankovic Rating scale	To evaluate the change of JRS (Jankovic Rating scale) score at 16 weeks post treatment based on baseline(0 week)	At 0 weeks and 16 weeks post-injection
Change from baseline at 4 weeks and 16 weeks of Disability Index	To evaluate the change of Blepharospasm Disability Index at 4 weeks and 16 weeks post treatment. Functional disability assessment scale will be assessed as functional disability score. Functional disability assessment scale consists of 6 daily activities (reading, watching television, household activities (cleaning, etc.), mobility, driving, work).	At 4 weeks and 16 weeks post-injection
Global Response evaluated by investigator at 4 weeks and 16 weeks after post-injection	To evaluate the Investigator's assessment of global response at Weeks 4 and 16 after treatment. At Weeks 4 and 16 Visits after treatment, the investigator will assess global response on a 9-point scale from -4 (marked worsening) to +4 (Complete abolition of all signs and symptoms).	At 4 weeks and 16 weeks post-injection
Change from baseline at 4 weeks and 16 weeks of WHO-Quality of Life(QOL)	To evaluate the change in WHO-Quality of Life (QoL) at Weeks 4 and 16 after treatment from baseline WHOQOL will be assessed by the brief Korean version of WHOQOL (WHOQOL-BREF). The Korean WHOQOL-BREF consists of a total of 26 subscales in 4 domains: physical health (7 items), psychological health (6 items), social relationships (3 items), and environment (8 items). Items in each subscale are in a range from 1 (not at all or very dissatisfied) to 5 (very much or very satisfied).	At 4 weeks and 16 weeks post-injection

Collaborators and Investigators

• Sponsor: Hugel

Study record dates

Study Major Dates

• Study Start (Actual): April 30, 2015
• Primary Completion (Actual): May 1, 2015
• Study Completion (Actual): January 7, 2016

Study Registration Dates

• First Submitted: August 19, 2018
• First Submitted That Met QC Criteria: August 20, 2018
• First Posted (Actual): August 22, 2018

Study Record Updates

• Last Update Posted (Actual): August 22, 2018
• Last Update Submitted That Met QC Criteria: August 20, 2018
• Last Verified: March 1, 2016

More Information

Terms related to this study

• Keywords: Essential Blepharospasm
• Additional Relevant MeSH Terms: Eye Diseases; Eyelid Diseases; Blepharospasm; Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Peripheral Nervous System Agents; Cholinergic Agents; Membrane Transport Modulators; Acetylcholine Release Inhibitors; Neuromuscular Agents; Botulinum Toxins; Botulinum Toxins, Type A; abobotulinumtoxinA
• Other Study ID Numbers: HG-BOT-IV3