Clinical trial NCT05027997: 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
Overall status: UNKNOWN
Phase: PHASE2
Conditions: Blepharospasm
Dystonia
Keywords: Blepharospasm
Dystonia
Lead sponsor: Addex Pharma S.A.
Sponsor class: INDUSTRY
First posted: 2021-08-31
Enrollment: 15
Sex: ALL
Minimum age (years): 18
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Safety and tolerability of dipraglurant as measured by incidence of adverse events
timeFrame: Baseline to Day 2
measure: Computerized Motor Objective Rater (CMOR) analysis of blinking activity
timeFrame: Baseline to Day 2
measure: Skintronics wearable analysis of blinking activity
timeFrame: Baseline to Day 2
Secondary outcomes:
measure: Jankovic Rating Scale (JRS) severity score
timeFrame: Baseline to Day 2
measure: Jankovic Rating Scale (JRS) frequency score
timeFrame: Baseline to Day 2
measure: Global Dystonia Severity Rating Scale (GDS)
timeFrame: Baseline to Day 2
measure: Blepharospasm Phenotyping Tool (BPT)
timeFrame: Baseline to Day 2
measure: Blepharospasm Severity Rating Scale (BSRS)
timeFrame: Baseline to Day 2
measure: Clinician's Global Impression of Severity (CGI-S) score
timeFrame: Baseline to Day 2
measure: Blepharospasm Disability Index (BSDI)
timeFrame: Baseline to Day 2
measure: Patient Global Impression of Change (PGI-C) score
timeFrame: Baseline to Day 2
Other outcomes:
Locations:
city: Atlanta
country: United States
facility: Emory University
state: Georgia
Study officials:
References: