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

Primary Purpose

Blepharospasm, Dystonia

Status
Recruiting
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Dipraglurant
Placebo
Sponsored by
Addex Pharma S.A.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Blepharospasm focused on measuring Blepharospasm, Dystonia

Eligibility Criteria

18 Years - 85 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Emory UniversityRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Placebo Comparator

Arm Label

Dipraglurant 50 mg

Dipraglurant 100 mg

Placebo

Arm Description

Outcomes

Primary Outcome Measures

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.
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.
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.

Secondary Outcome Measures

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.
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.
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.
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.
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.
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.
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.
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.

Full Information

First Posted
July 29, 2021
Last Updated
November 1, 2021
Sponsor
Addex Pharma S.A.
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1. Study Identification

Unique Protocol Identification Number
NCT05027997
Brief Title
Exploratory Study of Dipraglurant (ADX48621) for the Treatment of Patients With Blepharospasm
Official Title
Exploratory Phase 2a Randomized, Double-blind, Placebo-controlled Study of Dipraglurant (ADX48621) Immediate Release Tablets in Patients With Blepharospasm
Study Type
Interventional

2. Study Status

Record Verification Date
November 2021
Overall Recruitment Status
Recruiting
Study Start Date
October 6, 2021 (Actual)
Primary Completion Date
December 2021 (Anticipated)
Study Completion Date
December 2021 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Addex Pharma S.A.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Blepharospasm, Dystonia
Keywords
Blepharospasm, Dystonia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
15 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Dipraglurant 50 mg
Arm Type
Experimental
Arm Title
Dipraglurant 100 mg
Arm Type
Experimental
Arm Title
Placebo
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
Dipraglurant
Other Intervention Name(s)
ADX48621
Intervention Description
Oral tablet
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Oral matching placebo tablet
Primary Outcome Measure Information:
Title
Safety and tolerability of dipraglurant as measured by incidence of adverse events
Description
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.
Time Frame
Baseline to Day 2
Title
Computerized Motor Objective Rater (CMOR) analysis of blinking activity
Description
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.
Time Frame
Baseline to Day 2
Title
Skintronics wearable analysis of blinking activity
Description
Analysis of blinking activity as recorded on the Skintronics wearable device. Decreased blink activity indicates improvement of blepharospasm symptoms.
Time Frame
Baseline to Day 2
Secondary Outcome Measure Information:
Title
Jankovic Rating Scale (JRS) severity score
Description
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.
Time Frame
Baseline to Day 2
Title
Jankovic Rating Scale (JRS) frequency score
Description
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.
Time Frame
Baseline to Day 2
Title
Global Dystonia Severity Rating Scale (GDS)
Description
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.
Time Frame
Baseline to Day 2
Title
Blepharospasm Phenotyping Tool (BPT)
Description
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.
Time Frame
Baseline to Day 2
Title
Blepharospasm Severity Rating Scale (BSRS)
Description
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.
Time Frame
Baseline to Day 2
Title
Clinician's Global Impression of Severity (CGI-S) score
Description
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.
Time Frame
Baseline to Day 2
Title
Blepharospasm Disability Index (BSDI)
Description
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.
Time Frame
Baseline to Day 2
Title
Patient Global Impression of Change (PGI-C) score
Description
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.
Time Frame
Baseline to Day 2

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
85 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Study Director
Phone
877-409-1775
Email
Clinical.Team@addexpharma.com
Facility Information:
Facility Name
Emory University
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30329
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Hyder Jinnah
Phone
404-712-9107
Email
hjinnah@emory.edu

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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