MedPath

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

Phase 2
Completed
Conditions
Progressive Supranuclear Palsy
Interventions
Drug: Placebo oral solution
Drug: AZP2006 oral solution
Registration Number
NCT04008355
Lead Sponsor
AlzProtect SAS
Brief Summary

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.

Detailed Description

This is a randomized, double blind, placebo controlled, parallel group study comparing 2 doses of AZP2006 (60 mg Once daily \[QD\] during the 12-week treatment period or 80 mg for 10 days followed by 50 mg QD) with placebo in 36 men and women aged ≥40 years and ≤80 years and diagnosed with probable or possible PSP according to Movement Disorder Society PSP (MDS-PSP) criteria.

Patients were assessed for eligibility at baseline. Eligible patients were randomized on

Day -1 in a 1:1:1 ratio into one of the following 3 study intervention groups based on a randomization scheme with blocks stratified by center using an interactive web response system (IWRS):

1. 60 mg AZP2006/day during the 12-week treatment period;

2. 80 mg AZP2006/day for 10 days followed by 50 mg AZP2006/day;

3. Placebo.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
36
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.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Placebo/84 daysPlacebo oral solutionPatients randomized in this arm will receive placebo solution once daily during 84 days.
60mg/day/84 daysAZP2006 oral solutionPatients randomized in this arm will receive 60 mg of study investigational drug AZP2006 once daily during 84 days.
80mg/day/10 days followed by 50mg/day/74 daysAZP2006 oral solutionPatients 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.
Primary Outcome Measures
NameTimeMethod
The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSFFrom Day 1 of Day 84 (12 weeks)

To determine the Ctrough of AZP2006 in plasma, blood, and CSF after single and multiple dose administration, once daily for 12 weeks

Number and percentage of patients who prematurely discontinue from the study due to adverse events (AEs)From Day 1 to Day 180

Incidence in pourcentage of treatment-emergent adverse events observed directly by investigator and adverse event spontaneously reported by the patient using concise medical terminology

Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (3)

Hôpital Salengro

🇫🇷

Lille, Hauts De France, France

Hôpitaux Universitaires Pitié Salpêtrière - Charles Foix

🇫🇷

Paris, Ile-de-France, France

Hôpital de la Fondation Adolphe de Rothschild

🇫🇷

Paris, France

© Copyright 2025. All Rights Reserved by MedPath