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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.
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):
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 60mg/day/84 days | Experimental | Patients 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 days | Experimental | 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. |
|
| Placebo/84 days | Placebo Comparator | Patients randomized in this arm will receive placebo solution once daily during 84 days. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| AZP2006 oral solution | Drug | Once daily intake in the morning |
|
| 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 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Philippe Verwaerde, PhD | AlzProtect SAS | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hôpital Salengro | Lille | Hauts-de-France | 59037 | France | ||
| Hôpital de la Fondation Adolphe de Rothschild |
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| ID | Term |
|---|---|
| D013494 | Supranuclear Palsy, Progressive |
| ID | Term |
|---|---|
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
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| Placebo oral solution | Drug | Once daily intake in the morning |
|
| 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) |
| Paris |
| 75019 |
| France |
| Hôpitaux Universitaires Pitié Salpêtrière - Charles Foix | Paris | Île-de-France Region | 75013 | France |
| D009069 | Movement Disorders |
| D009886 | Ophthalmoplegia |
| D015835 | Ocular Motility Disorders |
| D003389 | Cranial Nerve Diseases |
| D024801 | Tauopathies |
| D019636 | Neurodegenerative Diseases |
| D010243 | Paralysis |
| D009461 | Neurologic Manifestations |
| D005128 | Eye Diseases |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |