BlepharospasmPhase 2NCT05027997
Exploratory Study of Dipraglurant (ADX48621) for the Treatment of Patients With Blepharospasm
A Phase 2 study testing Dipraglurant in people with Blepharospasm and Dystonia, run by Addex Pharma S.A. at 1 site in United States.
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… Read more about this study
- Who is it for?
- Ages 18 to 85 years
- Conditions the study is for
- Blepharospasm, Dystonia
- What is being tested?
- Dipraglurant
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- 6 Oct 2021to 27 Nov 2022
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Exploratory Phase 2a Randomized, Double-blind, Placebo-controlled Study of Dipraglurant (ADX48621) Immediate Release Tablets in Patients With Blepharospasm
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
- Being tested
- Dipraglurant, Placebo
- Keywords
- Blepharospasm, Dystonia
- Medical subject headings
- Blepharospasm, Dystonia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DipraglurantDrug
Also called ADX48621
Oral tablet
PlaceboDrug
Oral matching placebo tablet
| Study group | Role | What they receive |
|---|---|---|
| Dipraglurant 50 mg | Experimental | Not described Receives: Drug: Dipraglurant |
| Dipraglurant 100 mg | Experimental | Not described Receives: Drug: Dipraglurant |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: Baseline to Day 2
Secondary outcomes (8)
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.
Measured: Baseline to Day 2
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.
Measured: Baseline to Day 2
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.
Measured: Baseline to Day 2
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.
Measured: Baseline to Day 2
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.
Measured: Baseline to Day 2
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.
Measured: Baseline to Day 2
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.
Measured: Baseline to Day 2
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.
Measured: Baseline to Day 2
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Aug 2021
- Study start6 Oct 2021
- Primary completion27 Oct 2022
- Study completion27 Nov 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for BlepharospasmStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Addex Pharma S.A.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05027997
- Sponsor's protocol ID
- ADX48621-203
- Status last verified
- Nov 2021
- First submitted
- 29 Jul 2021
- Last updated
- 3 Oct 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Kilic-Berkmen G, Kim H, Chen D, Yeo CI, Dinasarapu AR, Scorr LM, Yeo WH, Peterson DA, Williams H, Ruby A, Mills R, Jinnah HA. An Exploratory, Randomized, Double-Blind Clinical Trial of Dipraglurant for Blepharospasm. Mov Disord. 2024 Apr;39(4):738-745. doi: 10.1002/mds.29734. Epub 2024 Feb 3.
PubMed 38310362
Study at a glance
From the registry record, last updated by the sponsor on 3 Oct 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Exploratory Phase 2a Randomized, Double-blind, Placebo-controlled Study of Dipraglurant (ADX48621) Immediate Release Tablets in Patients With Blepharospasm
- ClinicalTrials.gov ID
- NCT05027997
- Acronym / sponsor ID
- ADX48621-203
- Conditions
- Blepharospasm, Dystonia
- Keywords
- Blepharospasm, Dystonia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Addex Pharma S.A.Industry (pharma, biotech, devices)
- Study start
- 6 Oct 2021 (actual)
- Primary completion
- 27 Oct 2022 (actual)
- Study completion
- 27 Nov 2022 (actual)
- First posted
- 31 Aug 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Dipraglurant 50 mg | Experimental | Drug: Dipraglurant |
| Dipraglurant 100 mg | Experimental | Drug: Dipraglurant |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DipraglurantAlso: ADX48621Oral tablet | Drug | Dipraglurant 100 mg, Dipraglurant 50 mg |
| PlaceboOral matching placebo tablet | Drug | Placebo |
Primary outcome
Safety and tolerability of dipraglurant as measured by incidence of adverse events
Time frame: Baseline to Day 2
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
Time frame: Baseline to Day 2
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
Time frame: Baseline to Day 2
Analysis of blinking activity as recorded on the Skintronics wearable device. Decreased blink activity indicates improvement of blepharospasm symptoms.
8 secondary outcomes
Jankovic Rating Scale (JRS) severity score
Time frame: Baseline to Day 2
Jankovic Rating Scale (JRS) frequency score
Time frame: Baseline to Day 2
Global Dystonia Severity Rating Scale (GDS)
Time frame: Baseline to Day 2
Blepharospasm Phenotyping Tool (BPT)
Time frame: Baseline to Day 2
Blepharospasm Severity Rating Scale (BSRS)
Time frame: Baseline to Day 2
Clinician's Global Impression of Severity (CGI-S) score
Time frame: Baseline to Day 2
Blepharospasm Disability Index (BSDI)
Time frame: Baseline to Day 2
Patient Global Impression of Change (PGI-C) score
Time frame: Baseline to Day 2
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Oct 2025.
- Derived from this study: PubMed 38310362Kilic-Berkmen G, Kim H, Chen D, Yeo CI, Dinasarapu AR, Scorr LM, Yeo WH, Peterson DA, Williams H, Ruby A, Mills R, Jinnah HA. An Exploratory, Randomized, Double-Blind Clinical Trial of Dipraglurant for Blepharospasm. Mov Disord. 2024 Apr;39(4):738-745. doi: 10.1002/mds.29734. Epub 2024 Feb 3.
- Individual participant dataSharing planned: No
Related trials for Blepharospasm
RecruitingPhase 2
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Blepharospasm of Both Eyelids, Blepharospasm of Left Eyelid
RecruitingPhase 4
Effects of DaxibotulinumtoxinA for Blepharospasm and Hemifacial Spasm
Benign Essential Blepharospasm, Hemifacial Spasm
RecruitingObservational study
Brain Networks in Dystonia
Spasmodic Dysphonia, Singer's Dystonia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Dipraglurant in people with Blepharospasm and Dystonia, run by Addex Pharma S.A. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Oct 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and tolerability of dipraglurant as measured by incidence of adverse events (time frame: Baseline to Day 2). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05027997, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Oct 2025.