This is a multi-center, randomized, double-blinded, placebo-controlled, parallel design, prospective phase 3 study in participants with moderate to severe AD. The study will consist of a screening visit (up to 14 days prior to first dose), 24-week double-blind treatment phase, another 24-week open extension phase, and end-of-study (EOS) visits 4 weeks after an end-of-treatment [EOT] visit at each phase. Eligible participants will be randomized in a 1:1:1 ratio to receive GV1001 0.56 mg, GV1001 1.12 mg, or placebo (normal saline) every week for 4 weeks beginning on Day 1 (of Week 1) followed by every 2 weeks for 20 weeks during the double-blind treatment phase. Participants who give consents to enroll in the open extension phase, will receive GV1001 1.12mg every week for 4 weeks beginning on Day 1 (of Week 26) followed by every 2 weeks through Week 49. Yet, the group of participants who received GV1001 in the double-blind treatment phase (either 0.56 mg or 1.12 mg) will receive GV1001 1.12mg and placebo (normal saline) alternately first 4 weeks of the open extension phase.~Prior to randomization, eligibility of potential participants will be confirmed through an adjudication process in which screening data (eg, Korea Mini-Mental State Examination [K-MMSE], Global Deterioration Scale [GDS], MRI/CT scans) obtained to evaluate AD status. Results from CT or MRI performed within 1 year prior to screening will also be used to confirm eligibility.~If a participant discontinues the study prematurely, the participant will be asked to come for a drop-out [DO] visit for efficacy evaluation are scheduled at an end-of-study [EOS] visit at Week 28 or Week 53.~For an individual participant, the maximum duration of study participation is approximately 14 months, including an up to 14-day screening period.~Efficacy evaluations will be performed at baseline, 1 Month, 3 Month, 6 Month, 9 Month and 12 Month using the Severe Impairment Battery [SIB] and the Clinical Dementia Rating Scale Sum of Boxes [CDR-SOB], K-MMSE, Neuropsychiatric Inventory Questionnaire [NPI-Q], GDS, and Alzheimer's Disease Cooperative Study Activities of Daily Living Inventory for Severe Alzheimer's Disease [ADCS-ADL-severe]. At the visits where several efficacy assessments are administered, every effort should be made to perform the efficacy evaluations in the same order at each visit.~Safety will be assessed throughout the study by monitoring for AEs, laboratory evaluations, vital signs measurements, and physical assessments.
The study will be conducted by the Sponsor to evaluate the efficacy and safety of GV1001 (0.56 mg and 1.12 mg) administered subcutaneously as a treatment for moderate to severe Alzheimer's disease (AD). Studies using in vivo and in vitro AD models have shown that GV1001 inhibits neurotoxicity, apoptosis, and the production of reactive oxygen species induced by amyloid beta (Aβ) in neural stem cells by mimicking the extra-telomeric functions of human telomerase reverse transcriptase (hTERT). In nonclinical studies, using both mild (early stage) and severe (late stage) AD mouse models, GV1001 was shown to improve cognitive function and memory, as well as significantly reduce the amount of Aβ and tau proteins. The multifunctional effect of GV1001 makes it a promising therapeutic option for the treatment for AD. In a completed Phase 2 study conducted in Korea, GV1001 showed significant improvement in change from baseline of Severe Impairment Battery score at Week 24 and demonstrated a clinically acceptable safety profile in patients with moderate to severe AD.