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

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

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Progressive Supranuclear Palsy
• Intervention / Treatment: Drug: AZP2006 oral solution; Drug: Placebo oral solution

• Study Type: Interventional
• Enrollment (Actual): 36
• Phase: Phase 2

Contacts and Locations

Study Locations

• France
  • Paris, France, 75019
    • Hôpital de la Fondation Adolphe de Rothschild
  • Hauts De France
    • Lille, Hauts De France, France
      • Hôpital Salengro
  • Ile-de-France
    • Paris, Ile-de-France, France, 75013
      • Hôpitaux Universitaires Pitié Salpêtrière - Charles Foix

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 40 years to 80 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

3

Arms and Interventions

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

What is the study measuring?

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)

Collaborators and Investigators

• Sponsor: AlzProtect SAS
• Investigators: Study Director: Philippe Verwaerde, PhD, AlzProtect SAS

Study record dates

Study Major Dates

• Study Start (Actual): June 22, 2020
• Primary Completion (Actual): July 18, 2022
• Study Completion (Estimated): July 1, 2024

Study Registration Dates

• First Submitted: June 18, 2019
• First Submitted That Met QC Criteria: July 1, 2019
• First Posted (Actual): July 5, 2019

Study Record Updates

• Last Update Posted (Actual): August 7, 2023
• Last Update Submitted That Met QC Criteria: August 4, 2023
• Last Verified: August 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Eye Diseases; Neurologic Manifestations; Basal Ganglia Diseases; Movement Disorders; Neurodegenerative Diseases; Tauopathies; Cranial Nerve Diseases; Ocular Motility Disorders; Paralysis; Ophthalmoplegia; Supranuclear Palsy, Progressive; Pharmaceutical Solutions
• Other Study ID Numbers: AZP2006C04

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No