Clinical trial NCT04008355: A Study to Assess Tolerability, Safety, Pharmacokinetics and Effect of AZP2006 in Patients With PSP
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Progressive Supranuclear Palsy
Lead sponsor: AlzProtect SAS
Sponsor class: INDUSTRY
First posted: 2019-07-05
Enrollment: 36
Sex: ALL
Minimum age (years): 40
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Number and percentage of patients who prematurely discontinue from the study due to adverse events (AEs)
timeFrame: From Day 1 to Day 180
measure: The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF
timeFrame: From Day 1 of Day 84 (12 weeks)
measure: The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF
timeFrame: From Day 1 of Day 84 (12 weeks)
measure: The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF
timeFrame: From Day 1 of Day 84 (12 weeks)
measure: The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF
timeFrame: From Day 1 of Day 84 (12 weeks)
measure: The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF
timeFrame: From Day 1 of Day 84 (12 weeks)
measure: The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF
timeFrame: From Day 1 of Day 84 (12 weeks)
measure: The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF
timeFrame: From Day 1 of Day 84 (12 weeks)
measure: The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF
timeFrame: From Day 1 of Day 84 (12 weeks)
measure: The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF
timeFrame: From Day 1 of Day 84 (12 weeks)
measure: The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF
timeFrame: From Day 1 of Day 84 (12 weeks)
measure: The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF
timeFrame: From Day 1 of Day 84 (12 weeks)
measure: The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF
timeFrame: From Day 1 of Day 84 (12 weeks)
measure: The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF
timeFrame: From Day 1 of Day 84 (12 weeks)
Secondary outcomes:
Other outcomes:
Locations:
city: Lille
country: France
facility: Hôpital Salengro
state: Hauts De France
city: Paris
country: France
facility: Hôpitaux Universitaires Pitié Salpêtrière - Charles Foix
state: Ile-de-France
city: Paris
country: France
facility: Hôpital de la Fondation Adolphe de Rothschild
Study officials:
affiliation: AlzProtect SAS
name: Philippe Verwaerde, PhD
role: STUDY_DIRECTOR
References: