Parkinson´s disease
Conditions
Interventions
Product Code: NLX-112
Pharmaceutical Form: Tablet
INN or Proposed INN: BEFIRADOL
Current Sponsor code: NLX-112
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 0.25-
Sponsors
Neurolixis SAS
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Patient is 30 – 85 years old (inclusive) with a diagnosis of idiopathic PD according to the UK Parkinson’s Disease Society Brain Bank Clinical Diagnosis criteria. 2. PD patient is stably and optimally treated with L-DOPA; other anti-PD treatments are allowed if used for at least 4 weeks of previous continuous treatment. 3. Patient agrees to be challenged with 150% of their normal L-DOPA dose (maximum L-DOPA dose 250 mg) 30 minutes prior to efficacy assessments at baseline (Visit 2) and at the 2 efficacy clinic visits (Visits 6 and 7). 4. PD patient exhibits troublesome peak-dose LID, confirmed by a score of at least 1 on part IV, item 33 (disability) of the UPDRS at screening (Visit 1) and at Day 1 (baseline, Visit 2). 5. At least two 30-minute time periods from 9 am to 4 pm and at least 90 minutes total of each 24-hour period are indicated as “ON with troublesome dyskinesia” (according to the PD Home Dyskinesia Diary) prior to Day 1 (baseline, Visit 2). 6. Patient (and/or caregiver) demonstrates ability to accurately complete the PD Home Dyskinesia Diary entries during the screening visit. 7. Patient can read well enough to understand the informed consent document and other subject materials. 8. Female patients of child-bearing potential must have a negative urine pregnancy test at screening (Visit 1) and on Day 1 (Visit 2), must agree to avoid pregnancy during the study, and must practice abstinence (only allowed when this is the preferred and usual lifestyle of the subject) or must agree to use a highly effective method of contraception with a failure rate of =65 years) yes F.1.3.1 Number of subjects for this age range 8
Exclusion criteria
Exclusion criteria: 1. Patient has severe PD with a Hoehn and Yahr stage = 5. 2. Patient has unstable medical status, prior brain surgery (excluding deep brain stimulation [DBS], i.e., DBS patients will be allowed to be enrolled) or is scheduled to receive surgery during the trial period. 3. Patient has orthostatic hypotension: a decrease in systolic blood pressure (at least 20 mm Hg) or diastolic blood pressure (at least 10 mm Hg) within 2 minutes of the patient standing up, compared to pressures obtained while in a supine position for at least 5 minutes. At screening and baseline visits (Visit 1 and Visit 2), vital signs to assess orthostatic hypotension will be conducted in triplicate, 15-20 minutes apart, with the average of the 3 assessments used for exclusion. 4. Patient has dementia (MMSE <20). 5. Patient has clinically significant renal or liver disorder. 6. Patient currently exhibits generalized obsessive-compulsive disorder, panic disorder, bipolar disorder, post-traumatic stress syndrome (PTSD), clinically significant parasomnias or any other psychotic disorder as established by structured clinical interview for DSM disorders (SCID). Visual hallucinations are allowed. 7. Any suicidal actions in the past 2 years (per investigator judgement i.e. actual attempt, interrupted attempt, aborted attempt, or preparatory acts or behavior). 8. Any suicidal ideation of type 4 or 5 in the C-SSRS in the past 3 months (i.e. active suicidal thought with intent but without specific plan, or active suicidal thought with plan and intent). 9. Patient has taken an anti-convulsant, an anti-psychotic (except quetiapine), pindolol, tertatolol or buspirone within 4 weeks of baseline (Day 1, Visit 2). 10. Patient has taken any medication, within 4 weeks of baseline (Day 1, Visit 2) that inhibits or up-regulates CYP4503A4. 11. Patient is concurrently participating in another investigational drug trial or has participated in another investigational drug trial within the past 3 months. 12. Patient is at high risk of non-compliance in the Investigator’s opinion.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety and tolerability of NLX-112, titrated up to a maximum of 2 mg/day, compared to placebo during eight weeks of daily treatment in PD patients with LID.;Secondary Objective: To assess the potential efficacy of NLX-112 treatment in reducing troublesome LID.;Primary end point(s): • Frequency, intensity and seriousness of AEs • Clinically significant changes from baseline in: o ECG o Vital signs o Safety laboratory parameters o Physical examinations • Suicidal ideation/behavior as assessed by the Columbia Suicide Severity Rating Scale (C-SSRS). ;Timepoint(s) of evaluation of this end point: According to study flow-chart | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • • Change from baseline at the final efficacy clinic visit (Day 42) in the Unified Dyskinesia Rating Scale (UDysRS) total score [Timeframe: Baseline to Day 42]. Patients will be challenged with 150% of their standard L-DOPA dose (maximum L DOPA dose 250 mg) 30 minutes prior to the first UDysRS assessment. • Change from baseline in UDysRS total score at Day 28, after a 150% L DOPA dose challenge. • Change from baseline in total objective score (Parts III, IV) of the UDysRS at Day 28 and Day 42, after a 150% L-DOPA dose challenge. • Change from baseline in ON Time Without Troublesome Dyskinesia (ON Without Dyskinesia plus ON With Non-troublesome Dyskinesia) based on a PD Home Dyskinesia Diary. • Change from baseline in Unified Parkinson's Disease Rating Scale (UPDRS) scores (Part III, motor examination). • Change from baseline in UPDRS combined scores (Parts I, II, III and IV). • Clinical Global Impression of Change (CGI-C) in overall PD symptoms • Change from baseline in dyskinesia scores measured by the Kinesia 360 (Great Lakes Neurotechnologies, Inc) wearable dyskinesia assessment system.;Timepoint(s) of evaluation of this end point: On days 28 and 42 after start of treatment | — |
Countries
Sweden
Contacts
Public ContactCEO
Neurolixis SAS
Outcome results
None listed