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

NCT05027997 | Completed Phase 2 FDA Drug

• 1 other identifier: ADX48621-203
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

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.

Conditions

Blepharospasm; Dystonia

Keywords

Blepharospasm; Dystonia

Outcome Measures

Primary Outcomes (3)

• 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

• 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

• 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 Outcomes (8)

• Jankovic Rating Scale (JRS) severity score

  Baseline to Day 2

• Jankovic Rating Scale (JRS) frequency score

  Baseline to Day 2

• Global Dystonia Severity Rating Scale (GDS)

  Baseline to Day 2

• Blepharospasm Phenotyping Tool (BPT)

  Baseline to Day 2

• Blepharospasm Severity Rating Scale (BSRS)

  Baseline to Day 2

Study Arms (3)

Dipraglurant 50 mg

EXPERIMENTAL

Drug: Dipraglurant

Dipraglurant 100 mg

EXPERIMENTAL

Drug: Dipraglurant

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

Dipraglurant DRUG

Oral tablet

Also known as: ADX48621

Dipraglurant 100 mg; Dipraglurant 50 mg

Placebo DRUG

Oral matching placebo tablet

Placebo

Eligibility Criteria

• Age: 18 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Addex Pharma S.A.: lead

Study Sites (1)

Emory University

Atlanta, Georgia, 30329, United States

Location

MeSH Terms

Conditions

Blepharospasm; Dystonia

Interventions

6-fluoro-2-(4-(pyridin-2-yl)but-3-yn-1-yl)imidazo(1,2-a)pyridine

Condition Hierarchy (Ancestors)

Eyelid Diseases; Eye Diseases; Dyskinesias; Neurologic Manifestations; Nervous System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 29, 2021
• First Posted: August 31, 2021
• Study Start: October 6, 2021
• Primary Completion: October 27, 2022
• Study Completion: November 27, 2022
• Last Updated: October 3, 2025

Record last verified: 2021-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)