Clinical trial NCT02361424: Pilot Study Assessing the Effects of PXT00864 in Patients With Mild Alzheimer Disease (AD)
Official title: First Single-blind Sequential Placebo-controlled Prospective Phase IIA Pilot Study Assessing the Effects of PXT00864 in Mild AD Patients
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Alzheimer Disease
Keywords: PXT00864
mild AD
phase 2a
Lead sponsor: Pharnext S.C.A.
Sponsor class: OTHER
Collaborators: name: Ascopharm Groupe Novasco
First posted: 2015-02-11
Enrollment: 47
Sex: ALL
Minimum age (years): 60
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to assess the safety and efficacy on cognitive impairment and functioning of several doses of PXT00864 (new fixed combination of acamprosate and baclofen at low dose) in patients with mild Alzheimer Disease.


Eligibility criteria: Inclusion Criteria:

* Male or female patients aged ≥ 60 years.
* Patient with a diagnosis of probable AD
* Progressive decline in cognition for more than six months which story is documented in patient medical records
* A Mini-Mental State Examination (MMSE) score of 20-26
* With a minimum of educational background
* Naïve to anti-dementia treatment
* MRI assessment which corroborates the clinical diagnosis (hippocampal atrophy) and excludes other potential causes of dementia especially cerebrovascular lesions
* If available, Cerebral Spinal Fluid (CSF) classical biomarkers should be at levels which corroborate the clinical diagnosis
* Ambulatory patient living at home with a caregiver available and living in the same household or interacting with the patient daily and available if necessary to ensure administration of the investigational product
* Absence of major or severe depressive disease
* Patient with a willingness to participate in this study and who have signed an informed consent form

Exclusion Criteria:

* Early onset of dementia, i.e. before 60 years old to avoid hereditary AD forms
* Significant neurological disease other than AD
* Major psychiatric disorder or syndrome (schizophrenia or bipolar disorder)
* Seizure disorders
* Other infectious, metabolic or systemic diseases affecting central nervous system
* Other active clinically significant illness
* Hospitalization or change of chronic concomitant medications one month prior to screening
* Patients with severe respiratory, hepatic or renal failure or with any other significantly potentially disabling abnormality detected during screening
* Known hypersensitivity to the tested treatment including active substance and excipients.
* Patients participating in another study and exposed to any investigational therapy within the 30 days prior to the entry in this study.
* Patient without medical care insurance

Primary outcomes:
measure: Change From Baseline in the total score of the 11-item Alzheimer´s Disease Assessment Scale- Cognitive Subscale (ADAS-Cog)
timeFrame: Visit V0 (train), V1 (baseline), and every 4 weeks (visits V2, V3 and V4)
measure: Number of Treatment Emergent Adverse Events (TEAEs)
timeFrame: throughout the 12-week study period.
Secondary outcomes:
measure: Change From Baseline in the score of the Digit Symbol Substitution Test (DSST)
timeFrame: Visit V0 (train), V1 (baseline), and every 4 weeks (visits V2, V3 and V4)
measure: Change From Baseline in the speed to perform the Zazzo's Cancellation Test
timeFrame: Visit V0 (train), V1 (baseline), and every 4 weeks (visits V2, V3 and V4)
measure: Change From Baseline in the score of the Zazzo's Cancellation Test
timeFrame: Visit V0 (train), V1 (baseline), and every 4 weeks (visits V2, V3 and V4)
measure: Change From Baseline in the score of the 15-second Isaacs Set Test
timeFrame: Visit V0 (train), V1 (baseline), and every 4 weeks (visits V2, V3 and V4)
measure: Change From Baseline in the time score of the Trail Making Test - part A
timeFrame: Visit V0 (train), V1 (baseline), and every 4 weeks (visits V2, V3 and V4)
measure: Change From Baseline in the time score of the Trail Making Test - part B
timeFrame: Visit V0 (train), V1 (baseline), and every 4 weeks (visits V2, V3 and V4)
measure: Change From Baseline in the score of the Free and Cued Selective Reminding Test (FCSRT)
timeFrame: 1 (baseline) and V4 (end of study, after 12 weeks of treatment)
measure: Change From Baseline in the score of the 4-item Instrumental Activities of Daily Living scale (IADL-PAQUID)
timeFrame: V0 (train), V1 (baseline), and every 4 weeks (V2, V3 and V4)
measure: Change From Baseline in the score of the Clinical Dementia Rating (CDR) scale
timeFrame: 1 (baseline) and V4 (end of study, after 12 weeks of treatment)
measure: Change From Baseline in the score of the Apathy Inventory (AI) scale
timeFrame: 1 (baseline) and V4 (end of study, after 12 weeks of treatment)
Other outcomes:
measure: Change From Baseline in the cognitive Event Related Potential parameters (optional)
timeFrame: V1 (baseline), and every 4 weeks (V2, V3 and V4)
Locations:
city: Bordeaux
country: France
facility: CMRR
Study officials:
affiliation: Hospital Pellegrin, Bordeaux, France
name: Jean-Marc Orgogozo, MD
role: PRINCIPAL_INVESTIGATOR
References: