Oral EX039 for treating mild Alzheimer's disease

A Phase 2, Randomized, Double Blind, Placebo-Controlled Study of Oral EX039 As Add-on to Acetylcholine Esterase Inhibitors in Subjects with Mild Alzheimer's Disease

This study is testing if adding a new oral medication called EX039 can help people with mild Alzheimer's disease feel better when taken alongside their usual treatment.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 120 (estimated)
• Ages: 50 Years to 80 Years
• Sex: All
• Sponsor: Excelsior (Industry-sponsored)
• Locations: 1 site (Taipei)
• Trial ID: NCT05413655 on ClinicalTrials.gov

What this trial studies

This phase 2 clinical trial evaluates the efficacy of oral EX039 as an add-on treatment to Acetylcholine Esterase Inhibitors in patients diagnosed with mild Alzheimer's disease. The study is designed as a randomized, double-blind, placebo-controlled trial, ensuring that neither the participants nor the researchers know who receives the treatment versus the placebo. Participants will be monitored for cognitive improvements and overall health during the trial period.

Who should consider this trial

Good fit: Ideal candidates are individuals aged 50-80 with a clinical diagnosis of mild Alzheimer's disease and specific cognitive assessment scores.

Not a fit: Patients with severe cognitive impairment or those not meeting the eligibility criteria may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could enhance cognitive function and quality of life for patients with mild Alzheimer's disease.

How similar studies have performed: While this approach is being tested in this specific context, similar studies have shown promise in exploring new treatments for Alzheimer's disease.

Eligibility criteria

Inclusion Criteria:

* Aged 50-80 years.
* Clinical diagnosis of probable mild Alzheimer disease dementia based on National Institute of Neurological and Communicative Disorders and Stroke and the Alzheimer's Disease and Related Disorders Association (NINCDS/ADRDA criteria, 2011)
* Last time (which must be within 3 months ) of Mini Mental State Exam (MMSE) between 10-26.
* Last time (which must be within 3 months) of Clinical Dementia Rating (CDR) score of 1.
* Physically healthy and all laboratory assessments (including hematology, chemistry, urinalysis and electrocardiograph) are within normal range or meet the following criteria:

  * AST, ALT level ≦ upper limit of normal
  * Creatine Kinase (CK) concentration more than 3 times upper limit of normal
  * Serum creatinine level ≦ upper limit of normal
  * HbA1c more than 8.0
* Complaints of subjective memory impairment and cognitive disturbances by patients themselves or caregivers, including memory loss and at least one of the following cognitive disturbances: language, perceptual skills, attention, constructive abilities, orientation, problem solving, functional abilities.
* Cognitive deficits caused impairment in social or occupational function.
* Disease progression with gradual and continued decline from a previous level of functioning.
* Female subjects must be of non-childbearing potential (greater than 1 year without menstrual period in the absence of hormone replacement therapy) or surgically sterile. If pre-menopausal or menopausal for 1 year or less, must have a negative pregnancy test and must not be lactating at screening visit. Female subjects of childbearing potential and who are sexually active are required to practice adequate methods of birth control. Male subjects who are sexually active will also be required to use an adequate form of birth control.
* Has sufficient education equivalent to elementary education to communicate effectively and were capable of completing the assessments of the study.

Exclusion Criteria:

* Having other causes of dementia.
* Having substantial concomitant cerebrovascular disease (defined by a history of a stroke

  / intracranial hemorrhage temporally related to the onset of worsening of cognitive impairment) per investigator judgement.
* Medical history of cancer (except for basal cell carcinoma) and/or treatment for cancer within the last 5 years.
* Medical history or diagnosis of any of the following symptomatic and unstable / uncontrolled conditions per investigator's judgement:

  * Uncontrolled cardiovascular illnesses such as chronic congestive heart failure (with or without edema), tachycardia, arrhythmias, uncontrolled hypertension.
  * Significant ischemic heart disease, myocardial infarction within the last two years and/or with residual angina, orthopnea, conduction defects (ECG), or any other clinical significant heart disease classified as New York Heart Association (NYHA) III or IV.
  * Significant gastrointestinal disorders (for example gastrointestinal bleeding within the last two years, malabsorption syndromes, post-gastrectomy, or active peptic ulcer disease).
  * Uncontrolled endocrine disease such as uncontrolled diabetes mellitus or hyperthyroidism.
  * Unstable/Uncontrolled major depression.
  * Has neurological disease (other than dementia of Alzheimer's type, such as: Lewy body dementia - primary diagnosis, Huntington's disease, Parkinson's Disease, encephalitis, epilepsy, vascular or multiinfarct dementia, stroke, congenital mental deficiency, multiple sclerosis).
  * Significant pulmonary disease predisposing to hypoxia.
  * Has major physical illnesses (e.g. brain tumor, craniocerebral trauma, thyroid disease)
* Any other psychiatric disorders such as schizophrenia, or mental retardation.
* Any suicidal actions in the past 2 years (per investigator judgement i.e. actual attempt, interrupted attempt, aborted attempt, or preparatory acts or behavior).
* Hachinski Ischemic Score \> 4
* Significant history of drug dependence or abuse (including alcohol, as defined in Diagnostic and Statistical Manual of Mental Disorders \[DSM-V\] or in the opinion of the investigator)
* Clinically significant uncompensated hearing loss in the judgment of the investigator. Use of hearing aids is allowed.
* Has other condition(s) that in the opinions of investigators to be ineligible to participate in this study, e.g. Subject who cannot swallow the capsule whole.

Where this trial is running

Taipei

• National Taiwan University Hospital — Taipei, Taiwan (Recruiting)

Study contacts

• Principal investigator: Ching-Kuan Liu, Dr. — Kaohsiung Medical University
• Study coordinator: Cliff Lin

Conditions Alzheimer Disease