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Double-blind comparative study followed by open-label extension study to evaluate the efficacy and safety of TW-012R in Alzheimer's disease with presenilin 1 mutations(Phase 2/3 Study)

Basic Information

Recruitment status	Recruiting
Health condition(s) or Problem(s) studied	Presenilin1 gene mutation in Alzheimer's disease patients
Date of first enrollment	20/06/2025
Target sample size	24
Countries of recruitment
Study type	Interventional
Intervention(s)	[Double-blind period] 1) Low-dose maintenance period Initiate oral administration of one tablet of the investigational drug (TW-012R or placebo) once a day immediately after breakfast on the next day of Visit 2, and increase the daily dose by one tablet every week, up to a maximum of four tablets per day as the maintenance dose. 2) High-dose maintenance period Initiate administration of two to five tablets of the investigational drug (TW-012R or placebo) per day (one tablet more per day than the maintenance dose in the low-dose maintenance period), and increase the daily dose by one tablet every week, up to a maximum of nine tablets per day as the maintenance dose. [Continuation Administration Period] 3) Continuation Administration Period From the day after Week 37, TW-012R will be administered orally at one tablet (2.5 mg) once a day, increasing the daily dose by one tablet each week, with a maintenance dose of four tablets (10 mg) per day, with a maximum of nine tablets (22.5 mg) per day, administered orally in divided doses immediately after each meal. Administer until Week 48, then gradually reduce over 1-2 weeks until administration is complete.

Outcome(s)

Primary Outcome	SIB-J
Secondary Outcome

Key inclusion & exclusion criteria

Age minimum	>= 18age old
Age maximum	Not applicable
Gender	Both
Include criteria	1) AD patients with PSEN1 mutations identified by genetic testing 2) Patients diagnosed with "probable Alzheimer's disease" according to the diagnostic criteria for AD in DSM-5 3) Patients with a clear decline in cognitive function and daily living function based on medical records or information from informants who know the patient well 4) Patients with an MMSE-J score of 2-19 at Week 0 (Visit 2) 5) Patients with a caregiver who can provide the subject information necessary for this clinical trial, properly manage medication, and accompany the patient on scheduled visits 6) Patients aged 18 years or older at the time of obtaining consent 7) Patients or their legal representatives have given written consent to participate in this clinical trial
Exclude criteria	1) Those who have difficulty taking tablets orally. 2) Patients with the following diseases that the investigator judges to affect cognitive function or cognitive function evaluation (nutritional deficiency, metabolic disease, infectious encephalopathy, diabetes, hypertension, thyroid/endocrine disease, VB1/VB12 deficiency, folic acid deficiency) 3) Patients with a history of alcoholism, drug dependence, or drug abuse within 5 years prior to obtaining consent 4) Patients taking anti-dementia medication, whose dosage or administration has changed within 2 months prior to obtaining consent 5) Patients whose SIB-J score has worsened by -1.85/week or more (number of weeks rounded up to the nearest whole number) from the start of screening (Visit 1) to 0 weeks (Visit 2) 6) Patients with dementia due to a condition other than AD (vascular dementia, frontotemporal dementia, dementia with Lewy bodies, progressive supranuclear palsy, corticobasal degeneration, Huntington's disease, prion disease, etc.)