Exploratory Study of Dipraglurant (ADX48621) for the Treatment of Patients With Blepharospasm

Dystonia Clinical Trial

Official title:

Exploratory Phase 2a Randomized, Double-blind, Placebo-controlled Study of Dipraglurant (ADX48621) Immediate Release Tablets in Patients With Blepharospasm

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05027997
• Other study ID #: ADX48621-203
• Status: Recruiting
• Phase: Phase 2
• Start date: October 6, 2021
• Est. completion date: December 2021

Study information

• Verified date: November 2021
• Source: Addex Pharma S.A.
• Contact: Study Director
• Phone: 877-409-1775
• Email: Clinical.Team[@]addexpharma.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This study is designed to assess the safety and tolerability of dipraglurant in patients with blepharospasm (BSP) (randomized 1:1:1 to receive dipraglurant 50 mg, 100 mg or placebo) and explore the efficacy of 50 mg and 100 mg immediate release tablets (versus placebo) on the severity and frequency of BSP signs and symptoms using objective measures, clinical ratings and patient reported outcomes.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 15
• Est. completion date: December 2021
• Est. primary completion date: December 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 85 Years

Eligibility

Inclusion Criteria:

• Patients with an established diagnosis of idiopathic benign essential blepharospasm

• Must have had prior response to to botulinum toxin (BoNT) reported on last 2 consecutive injection cycles

• Last injection of BoNT at least 8 weeks prior to Screening assessments

• Patient is experiencing features of Blepharospasm (BSP) of moderate severity/frequency at study entry.

Exclusion Criteria:

• BSP that is known or suspected to be associated with a known cause such as neuroleptic exposure, brain injury or lesion, stroke, Parkinson's disease, or related Parkinsonisms

• History of surgical intervention (e.g., deep brain stimulation) or orbital myectomy for dystonia

• Disabling eyelid opening apraxia

• Other neurological disease (including psychiatric disease and/or cognitive impairment) that, in the opinion of the investigator, would affect the patient's ability to complete study assessments.

• Other significant medical condition that may affect the safety of the patient or preclude adequate participation in the study.

Other protocol-defined inclusion and exclusion criteria may apply

Related Conditions & MeSH terms

• Blepharospasm
• Dystonia
• Dystonic Disorders

Intervention

Drug:
• Dipraglurant
Oral tablet
• Placebo
Oral matching placebo tablet

Locations

Country	Name	City	State
United States	Emory University	Atlanta	Georgia

Sponsors (1)

Lead Sponsor	Collaborator
Addex Pharma S.A.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety and tolerability of dipraglurant as measured by incidence of adverse events	The primary outcome will be testing the safety and tolerability of dipraglurant in patients with blepharospasm based on the incidence of adverse events reported by patients and/or as identified by the Investigator based on clinical assessments conducted during the study.	Baseline to Day 2
Primary	Computerized Motor Objective Rater (CMOR) analysis of blinking activity	Video analysis, by an independent rater, of the mean proportion of eye closure time, severity of periocular spasms, and blink rate. Decreased eye closure time, severity of periocular spasms and blink rate indicates improvement of blepharospasm symptoms.	Baseline to Day 2
Primary	Skintronics wearable analysis of blinking activity	Analysis of blinking activity as recorded on the Skintronics wearable device. Decreased blink activity indicates improvement of blepharospasm symptoms.	Baseline to Day 2
Secondary	Jankovic Rating Scale (JRS) severity score	The JRS is a clinician rated scale for blepharospasm. The intensity of eyelid spasms is measured based on a 5 point grading system where 0 indicates no symptoms and 4 indicates the most severe symptoms.	Baseline to Day 2
Secondary	Jankovic Rating Scale (JRS) frequency score	The JRS is a clinician rated scale for blepharospasm. The frequency of eyelid spasms is measured based on a 5 point grading system where 0 indicates no symptoms and 4 indicates highest frequency symptoms.	Baseline to Day 2
Secondary	Global Dystonia Severity Rating Scale (GDS)	The GDS is a clinician rated scale for the assessment of dystonia severity across multiple body regions. Each body region is rated on a scale from 0-10 where 0 represents no dystonia and 10 represents severe dystonia.	Baseline to Day 2
Secondary	Blepharospasm Phenotyping Tool (BPT)	The BPT is a clinician rated scale assessing 4 major phenotypic elements of blepharospasm using a 4 point scale for severity and frequency, where 0 indicates no symptoms and 4 indicates the highest frequency and/or most severe symptoms.	Baseline to Day 2
Secondary	Blepharospasm Severity Rating Scale (BSRS)	The BSRS is a clinician rated scale for rating severity of blepharospasm based on 6 clinical features of the condition. A total score is generated based on the combined score for severity and frequency and where a higher value indicates more severe symptoms.	Baseline to Day 2
Secondary	Clinician's Global Impression of Severity (CGI-S) score	The CGI-S score is used by the Investigator to determine, in a global sense, how severe the patients blepharospasm is in the context of other blepharospasm patients. Increased scores indicate more severe symptoms.	Baseline to Day 2
Secondary	Blepharospasm Disability Index (BSDI)	The BSDI is a 6 item patient rated disability scale that measures how blepharospasm effects activities of daily living. Each item is scored on a 5 point scale to produce a total score of up to 30, where a higher individual item score and a higher total score indicate greater disability to the patient.	Baseline to Day 2
Secondary	Patient Global Impression of Change (PGI-C) score	The PGI-C score is a patient reported outcome that assesses how much the blepharospasm symptoms have improved or worsened. A score of 0 indicates no change, negative scores down to -3 indicate worsening, and positive scores of up to 3 indicate improvement.	Baseline to Day 2