A Study to Assess Tolerability, Safety, Pharmacokinetics and Effect of AZP2006 in Patients With PSP

Study Overview
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Brief Summary
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A phase 2 study to assess tolerability, safety, pharmacokinetics and effect of AZP2006 at different doses versus placebo on cerebrospinal fluid biomarkers in 36 patients with progressive supranuclear palsy. The patient study duration is 29 weeks including a washout period.

Official Title
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A Multi-center, Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Assess Tolerability, Safety, Pharmacokinetics and Effect of AZP2006 on Cerebrospinal Fluid Biomarkers in 36 Patients With Progressive Supranuclear Palsy

Conditions
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Progressive Supranuclear Palsy

Intervention / Treatment
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* Drug: AZP2006 oral solution
* Drug: Placebo oral solution


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male and female patients with probable or possible PSP Patients must be stable with their medication for at least 30 days prior to the inclusion visit. Exclusion Criteria: Any history of clinically significant head trauma or cerebrovascular disease or recent history of substance abuse or alcohol abuse and deemed to be clinically significant by the Investigator. History of deep brain stimulator (DBS) surgery other than sham surgery for DBS clinical study.

Ages Eligible for Study
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Minimum Age: 40 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 60mg/day/84 days<br>Patients randomized in this arm will receive 60 mg of study investigational drug AZP2006 once daily during 84 days. | Drug: AZP2006 oral solution<br>* Once daily intake in the morning<br>|
| Experimental: 80mg/day/10 days followed by 50mg/day/74 days<br>Patients randomized in this arm will receive 80 mg of study investigational drug AZP2006 once daily during 10 days followed by 50 mg of study investigational drug AZP2006 once daily during the next 74 days. | Drug: AZP2006 oral solution<br>* Once daily intake in the morning<br>|
| Placebo Comparator: Placebo/84 days<br>Patients randomized in this arm will receive placebo solution once daily during 84 days. | Drug: Placebo oral solution<br>* Once daily intake in the morning<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number and percentage of patients who prematurely discontinue from the study due to adverse events (AEs) | Incidence in pourcentage of treatment-emergent adverse events observed directly by investigator and adverse event spontaneously reported by the patient using concise medical terminology | From Day 1 to Day 180 | 
| The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF | To determine the Cmax of AZP2006 in plasma, blood, and CSF after single and multiple dose administration, once daily for 12 weeks | From Day 1 of Day 84 (12 weeks) | 
| The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF | To determine the tmax of AZP2006 in plasma, blood, and CSF after single and multiple dose administration, once daily for 12 weeks | From Day 1 of Day 84 (12 weeks) | 
| The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF | To determine the kel of AZP2006 in plasma, blood, and CSF after single and multiple dose administration, once daily for 12 weeks | From Day 1 of Day 84 (12 weeks) | 
| The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF | To determine the Clast of AZP2006 in plasma, blood, and CSF after single and multiple dose administration, once daily for 12 weeks | From Day 1 of Day 84 (12 weeks) | 
| The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF | To determine the AUC 0-10 of AZP2006 in plasma, blood, and CSF after single and multiple dose administration, once daily for 12 weeks | From Day 1 of Day 84 (12 weeks) | 
| The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF | To determine the AUC 0-24 of AZP2006 in plasma, blood, and CSF after single and multiple dose administration, once daily for 12 weeks | From Day 1 of Day 84 (12 weeks) | 
| The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF | To determine the AUC 0-t of AZP2006 in plasma, blood, and CSF after single and multiple dose administration, once daily for 12 weeks | From Day 1 of Day 84 (12 weeks) | 
| The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF | To determine the t1/2 of AZP2006 in plasma, blood, and CSF after single and multiple dose administration, once daily for 12 weeks | From Day 1 of Day 84 (12 weeks) | 
| The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF | To determine the tlast of AZP2006 in plasma, blood, and CSF after single and multiple dose administration, once daily for 12 weeks | From Day 1 of Day 84 (12 weeks) | 
| The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF | To determine the %AUCextra of AZP2006 in plasma, blood, and CSF after single and multiple dose administration, once daily for 12 weeks | From Day 1 of Day 84 (12 weeks) | 
| The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF | To determine the CL/F of AZP2006 in plasma, blood, and CSF after single and multiple dose administration, once daily for 12 weeks | From Day 1 of Day 84 (12 weeks) | 
| The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF | To determine the Vd/F of AZP2006 in plasma, blood, and CSF after single and multiple dose administration, once daily for 12 weeks | From Day 1 of Day 84 (12 weeks) | 
| The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF | To determine the Ctrough of AZP2006 in plasma, blood, and CSF after single and multiple dose administration, once daily for 12 weeks | From Day 1 of Day 84 (12 weeks) |