A Study to Assess Tolerability, Safety, Pharmacokinetics and Effect of AZP2006 in Patients With PSP
Primary Purpose
Progressive Supranuclear Palsy
Status
Active
Phase
Phase 2
Locations
France
Study Type
Interventional
Intervention
AZP2006 oral solution
Placebo oral solution
Sponsored by
About this trial
This is an interventional treatment trial for Progressive Supranuclear Palsy
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.
Sites / Locations
- Hôpital Salengro
- Hôpitaux Universitaires Pitié Salpêtrière - Charles Foix
- Hôpital de la Fondation Adolphe de Rothschild
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Experimental
Experimental
Placebo Comparator
Arm Label
60mg/day/84 days
80mg/day/10 days followed by 50mg/day/74 days
Placebo/84 days
Arm Description
Patients randomized in this arm will receive 60 mg of study investigational drug AZP2006 once daily during 84 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.
Patients randomized in this arm will receive placebo solution once daily during 84 days.
Outcomes
Primary Outcome Measures
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
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
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
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
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
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
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
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
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
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
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
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
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
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
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04008355
Brief Title
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
2. Study Status
Record Verification Date
August 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
June 22, 2020 (Actual)
Primary Completion Date
July 18, 2022 (Actual)
Study Completion Date
July 1, 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
AlzProtect SAS
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Progressive Supranuclear Palsy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
36 (Actual)
8. Arms, Groups, and Interventions
Arm Title
60mg/day/84 days
Arm Type
Experimental
Arm Description
Patients randomized in this arm will receive 60 mg of study investigational drug AZP2006 once daily during 84 days.
Arm Title
80mg/day/10 days followed by 50mg/day/74 days
Arm Type
Experimental
Arm Description
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.
Arm Title
Placebo/84 days
Arm Type
Placebo Comparator
Arm Description
Patients randomized in this arm will receive placebo solution once daily during 84 days.
Intervention Type
Drug
Intervention Name(s)
AZP2006 oral solution
Intervention Description
Once daily intake in the morning
Intervention Type
Drug
Intervention Name(s)
Placebo oral solution
Intervention Description
Once daily intake in the morning
Primary Outcome Measure Information:
Title
Number and percentage of patients who prematurely discontinue from the study due to adverse events (AEs)
Description
Incidence in pourcentage of treatment-emergent adverse events observed directly by investigator and adverse event spontaneously reported by the patient using concise medical terminology
Time Frame
From Day 1 to Day 180
Title
The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF
Description
To determine the Cmax of AZP2006 in plasma, blood, and CSF after single and multiple dose administration, once daily for 12 weeks
Time Frame
From Day 1 of Day 84 (12 weeks)
Title
The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF
Description
To determine the tmax of AZP2006 in plasma, blood, and CSF after single and multiple dose administration, once daily for 12 weeks
Time Frame
From Day 1 of Day 84 (12 weeks)
Title
The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF
Description
To determine the kel of AZP2006 in plasma, blood, and CSF after single and multiple dose administration, once daily for 12 weeks
Time Frame
From Day 1 of Day 84 (12 weeks)
Title
The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF
Description
To determine the Clast of AZP2006 in plasma, blood, and CSF after single and multiple dose administration, once daily for 12 weeks
Time Frame
From Day 1 of Day 84 (12 weeks)
Title
The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF
Description
To determine the AUC 0-10 of AZP2006 in plasma, blood, and CSF after single and multiple dose administration, once daily for 12 weeks
Time Frame
From Day 1 of Day 84 (12 weeks)
Title
The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF
Description
To determine the AUC 0-24 of AZP2006 in plasma, blood, and CSF after single and multiple dose administration, once daily for 12 weeks
Time Frame
From Day 1 of Day 84 (12 weeks)
Title
The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF
Description
To determine the AUC 0-t of AZP2006 in plasma, blood, and CSF after single and multiple dose administration, once daily for 12 weeks
Time Frame
From Day 1 of Day 84 (12 weeks)
Title
The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF
Description
To determine the t1/2 of AZP2006 in plasma, blood, and CSF after single and multiple dose administration, once daily for 12 weeks
Time Frame
From Day 1 of Day 84 (12 weeks)
Title
The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF
Description
To determine the tlast of AZP2006 in plasma, blood, and CSF after single and multiple dose administration, once daily for 12 weeks
Time Frame
From Day 1 of Day 84 (12 weeks)
Title
The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF
Description
To determine the %AUCextra of AZP2006 in plasma, blood, and CSF after single and multiple dose administration, once daily for 12 weeks
Time Frame
From Day 1 of Day 84 (12 weeks)
Title
The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF
Description
To determine the CL/F of AZP2006 in plasma, blood, and CSF after single and multiple dose administration, once daily for 12 weeks
Time Frame
From Day 1 of Day 84 (12 weeks)
Title
The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF
Description
To determine the Vd/F of AZP2006 in plasma, blood, and CSF after single and multiple dose administration, once daily for 12 weeks
Time Frame
From Day 1 of Day 84 (12 weeks)
Title
The pharmacokinetics of AZP2006 (12-week treatment period) in plasma, blood, and CSF
Description
To determine the Ctrough of AZP2006 in plasma, blood, and CSF after single and multiple dose administration, once daily for 12 weeks
Time Frame
From Day 1 of Day 84 (12 weeks)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
40 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Philippe Verwaerde, PhD
Organizational Affiliation
AlzProtect SAS
Official's Role
Study Director
Facility Information:
Facility Name
Hôpital Salengro
City
Lille
State/Province
Hauts De France
Country
France
Facility Name
Hôpitaux Universitaires Pitié Salpêtrière - Charles Foix
City
Paris
State/Province
Ile-de-France
ZIP/Postal Code
75013
Country
France
Facility Name
Hôpital de la Fondation Adolphe de Rothschild
City
Paris
ZIP/Postal Code
75019
Country
France
12. IPD Sharing Statement
Learn more about this trial
A Study to Assess Tolerability, Safety, Pharmacokinetics and Effect of AZP2006 in Patients With PSP
We'll reach out to this number within 24 hrs