GV1001 Subcutaneous(SC) for the Treatment of Mild to Moderate Alzheimer's Disease (AD)

NCT05189210 | Completed Phase 2 DMC FDA Drug

• 1 other identifier: GV1001-AD-CL2-007
• Study Type: interventional
• Target: 199
• Locations: 8 countries
• Sites: 35

Brief Summary

The current study is being 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 mild to moderate 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.

Conditions

Mild to Moderate Alzheimer's Disease

Keywords

Alzheimer's Disease; GV1001

Outcome Measures

Primary Outcomes (1)

• Change from baseline in ADAS-Cog11 score at Week 52

  The Alzheimer's Disease Assessment Scale (ADAS) consists of 2 parts, a cognitive subscale and a behavioral subscale. The 11-item cognitive subscale (ADAS-cog) of the ADAS instrument will be used in this study. The ADAS-cog11 is a widely accepted performance-based assessment of cognition used in clinical studies for the treatment of participants with AD. The ADAS-cog11 (score range) consists of word recall (0-10) and word recognition memory tests (0-12), object and finger naming (0-5), commands (0-5), constructional praxis (0-5), ideational praxis (0-5), orientation (0-8), remembering test instructions (0-5), spoken language ability (0-5), comprehension of spoken language (0-5) and word finding difficulty (0-5). The maximum possible total score is 70, with a higher ADAS-cog11 score indicating worse cognitive function.

  Baseline, Week 12, Week 26, Week 38, and Week 52

Secondary Outcomes (7)

• Change from baseline in A-IADL-Q score at Week 12, Week 26, Week 38, and Week 52

  Baseline, Week 12, Week 26, Week 38, and Week 52

• Change from baseline in CDR-SB score at Week 12, Week 26, Week 38, and Week 52

  Baseline, Week 12, Week 26, Week 38, and Week 52

• Clinical worsening, defined as ≥4 points change from baseline in the ADAS-cog11 score at Week 12, Week 26, Week 38, and Week 52

  Baseline, Week 12, Week 26, Week 38, and Week 52

• Change from baseline in NPI score at Week 12, Week 26, Week 38, and Week 52

  Baseline, Week 12, Week 26, Week 38, and Week 52

• Change from baseline in MMSE score at Week 12, Week 26, Week 38, and Week 52

  Baseline, Week 12, Week 26, Week 38, and Week 52

Study Arms (3)

Placebo

PLACEBO COMPARATOR

Placebo SC injection administered once weekly for 4 weeks then every 2 weeks through Week 50

Drug: GV1001 Placebo

GV1001 0.56 mg

EXPERIMENTAL

GV1001 0.56 mg SC injection administered once weekly for 4 weeks then every 2 weeks through Week 50

Drug: GV1001 0.56mg

GV1001 1.12 mg

EXPERIMENTAL

GV1001 1.12 mg SC injection administered once weekly for 4 weeks then every 2 weeks through Week 50

Drug: GV1001 1.12mg

Interventions

GV1001 Placebo DRUG

0.9% normal saline

Also known as: Normal saline

Placebo

GV1001 0.56mg DRUG

Lyophilized peptide from hTERT

Also known as: Tertomotide 0.84mg

GV1001 0.56 mg

GV1001 1.12mg DRUG

Lyophilized peptide from hTERT

Also known as: Tertomotide 1.68mg

GV1001 1.12 mg

Eligibility Criteria

• Age: 55 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female participants 55 to 85 years of age (both inclusive) at the time of signing the informed consent.
• Diagnosis of probable AD based on NINCDS-ADRDA criteria (a and b) as determined by a neurologist, geriatrician, psychiatrist, or clinician approved by the Sponsor or designee.
• a. Presence of an early and significant episodic memory impairment that includes the following features: i. Gradual and progressive change in memory function reported by patients or informants over \>6months.
• ii. Objective evidence of significantly impaired episodic memory on testing: this generally consists of recall deficit that does not improve significantly or does not normalize with cueing or recognition testing and after effective encoding of information has been previously controlled.
• iii. The episodic memory impairment can be isolated or associated with other cognitive changes at the onset of AD or as AD advances.
• b. One or more findings for probable AD by either MRI, Aβ PET scan, historical CSF results, or a historical genetic test in the 2 years before screening, or an MRI or Aβ PET scan at screening. The MRI must have findings consistent with AD and without any other disease that may cause dementia. The Aβ PET scan and historical CSF results must be consistent with the presence of amyloid pathology.
• Mild or moderate dementia as evidenced by MMSE score ≥13 to ≤24 at screening (Visit 1).
• Not applicable.
• Not applicable.
• If receiving an approved medication for AD (ie, donepezil, galantamine, rivastigmine, memantine, or memantine/donepezil combination product), must be on the medication with a stable dose for at least 12 weeks before the screening visit (dosing should remain stable throughout the study).
• If receiving an OTC supplement for cognition (eg, gingko biloba, omega-3 polyunsaturated fatty acid, vitamin E, curcumin), must not be exceeding the recommended dose for at least 12 weeks prior to screening visit.
• Able to visit the study center and undergo cognitive, functional, and other tests specified in the protocol.
• Has a caregiver who:
• Agrees to accompany the participant to all study visits and able to supervise the participant's compliance with the study procedures and provide detailed information about the participant.
• Either lives with the participant or sees the participant on average for ≥1 hour/day ≥3 days/week, or in the Investigator's opinion, the extent of contact is sufficient to provide meaningful assessment of changes in participant behavior and function over time and provide information on safety and tolerability.

You may not qualify if:

• Any other cause of dementia shown by MRI/CT findings within 2 years of screening and neurological examination at screening and Day 1.
• Possible, probable, or definite vascular dementia according to the National Institute of Neurological Disorders and Stroke and Association Internationale pour la Recherché et l'Enseignement en Neurosciences (NINDS-AIREN) criteria.
• Evidence of significant abnormality that would suggest another potential etiology for dementia (eg, evidence of cerebral contusion, encephalomalacia, aneurysm, vascular malformation, \>5 microhemorrhages, macrohemorrhage, single infarct \>1 cm3).
• Other central nervous system diseases that may cause cognitive impairment (eg, cerebrovascular disease including cerebrovascular dementia, Parkinsonism, Huntington's disease, subdural hematoma, normal pressure hydrocephalus, brain tumor, Creutzfeldt-Jakob disease).
• Concurrent or history of schizophrenia or bipolar disorder; OR any other clinically significant psychiatric conditions that in the Investigator's opinion prevents the participant from participating, or is likely to confound interpretation of drug effect or affect cognitive assessments or participant safety; OR the presence or history of suicidal attempts or suicidal ideation evidenced by endorsing Items 4 or 5 of the C-SSRS at screening or Day 1, endorsing any suicidal behavior item on the C-SSRS Since Last Visit form on Day 1, or any suicide attempt within 2 years prior to screening.
• Vitamin B12, folic acid, syphilis serology, and thyroid stimulating hormone (TSH) results that are thought to contribute to the severity of dementia or cause dementia. Participants may be enrolled if in the Investigator's medical judgment, the abnormal laboratory values are not the cause of the cognitive symptoms.
• History of known or suspected seizures including febrile seizures (excluding self-limited childhood febrile seizures), a history of significant head trauma with loss of consciousness or recent unconsciousness that is not explained.
• Acute or unstable cardiovascular disease, active peptic ulcer, uncontrolled hypertension, uncontrolled diabetes or insulin dependent patients or any medical condition that may interfere with the completion of the clinical study.
• Known allergies, hypersensitivity, or intolerance to GV1001 or similar products or excipients.
• History of alcohol, substance abuse or dependence as per DSM-V criteria (except nicotine dependence) within the last 2 years.
• Concurrent malignancies or invasive cancers diagnosed within the past 5 years except for non-metastatic basal cell carcinoma or squamous cell carcinoma of skin, in situ carcinoma of the uterine cervix or non-metastatic prostate cancer.
• Sexually-active WOCBP or man capable of fathering a child who do not consent to using medicinally acceptable contraception (such as surgical sterilization, intrauterine contraceptive device, condom or diaphragm, an injectable or inserted contraceptive) during the study and for 3 months after the last dose of study treatment.
• Pregnant, breast feeding, or planning a pregnancy or fathering a child while enrolled in the study or for 3 months after the last dose of study treatment.
• Use of anxiolytics, narcotics, or sleep aids in a manner that would interfere with cognitive testing, in the opinion of the Investigator. Atypical antipsychotics may be used at the discretion of the Investigator. Tricyclic antidepressants and monoamine oxidase (MAO) inhibitors are prohibited
• Previous treatment with GV1001.

Sponsors & Collaborators

• GemVax & Kael: lead

Study Sites (35)

First Choice Neurology - Aventura Neurologic Associates

Adventura, Florida, 33180, United States

Location

Finlay Medical Research Corporation - Greenacres

Greenacres City, Florida, 33467, United States

Location

Outpatient Psy Care

Miami, Florida, 33125, United States

Location

Finlay Medical Research Corporation - Miami Dade

Miami, Florida, 33126, United States

Location

Aqualane Clinical Research

Naples, Florida, 34105, United States

Location

Palm Beach Neurological Center

Palm Beach Gardens, Florida, 33410, United States

Location

Neurostudies - Port Charlotte

Port Charlotte, Florida, 33952, United States

Location

Palm Beach Neurology and Premiere Research Institute

West Palm Beach, Florida, 33407, United States

Location

Accel Research Sites (ARS) - NeuroStudies

Decatur, Georgia, 30033, United States

Location

Global Medical Institutes - Princeton Medical Institute

Princeton, New Jersey, 08540, United States

Location

Flourish Research - Alzheimer's Memory Center/AMC Research, LLC

Charlotte, North Carolina, 28270, United States

Location

Neurology Clinic - Cordova

Cordova, Tennessee, 38018, United States

Location

Terveystalo Oulu

Oulu, 90100, Finland

Location

Hôpital La Grave

Toulouse, Midi-Pyrenees, 31300, France

Location

Hôpital Pierre Wertheimer

Bron, Rhône, 69677, France

Location

Casa di Cura iGEA SpA c/o Casa di Cura Privata del Policlinico

Milan, 20144, Italy

Location

Brain Research Center - Amsterdam

Amsterdam, North Holland, 1081 GN, Netherlands

Location

Brain Research Center - Zwolle

Zwolle, Overijssel, 8025 AZ, Netherlands

Location

Brain Research Centre - Den Bosch

Amsterdam, Netherlands

Location

Centrum Medyczne Neuromed - Ośrodek Badań Klinicznych

Bydgoszcz, Kuyavian-Pomeranian Voivodeship, 85-163, Poland

Location

Wroctawskie Centrum Alzheimerowskie (WCA)

Wroclaw, Lower Silesian Voivodeship, 53-659, Poland

Location

Galen Clinic

Lublin, Lublin Voivodeship, 20-064, Poland

Location

Centrum Medyczne Neuroprotect

Warsaw, Masovian Voivodeship, 01-684, Poland

Location

Niepubliczny Zakład Opieki Zdrowotnej NOVO-MED

Katowice, Silesian Voivodeship, 40-584, Poland

Location

Clinhouse Centrum Medyczne

Zabrze, Silesian Voivodeship, 41-807, Poland

Location

Unidade Local de Saúde de Santa Maria, E. P. E. - Hospital de Santa Maria

Lisbon, Lisbon District, 1649-035, Portugal

Location

Hospital Pedro Hispano

Senhora da Hora, Porto District, 4464-513, Portugal

Location

Unidade Local de Saúde de Braga, E. P. E (Hospital de Braga)

Braga, 4710-243, Portugal

Location

Hospital Universitario Quirónsalud Madrid

Pozuelo de Alarcón, Madrid, 28223, Spain

Location

Hospital Universitari Vall d'Hebrón

Barcelona, 08035, Spain

Location

Hospital Clinic de Barcelona

Barcelona, 08036, Spain

Location

Fundacio ACE

Barcelona, Spain

Location

Hospital Victoria Eugenia

Seville, 41009, Spain

Location

Hospital Universitario Virgen del Rocío

Seville, 41013, Spain

Location

Hospital Universitari i Politècnic La Fe

Valencia, 46026, Spain

Location

MeSH Terms

Conditions

Alzheimer Disease

Interventions

Saline Solution

Condition Hierarchy (Ancestors)

Dementia; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Tauopathies; Neurodegenerative Diseases; Neurocognitive Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Crystalloid Solutions; Isotonic Solutions; Solutions; Pharmaceutical Preparations

Study Officials

• Jeongmi Kim

  GemVax & KAEL Co., Ltd.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 29, 2021
• First Posted: January 12, 2022
• Study Start: October 5, 2022
• Primary Completion: April 3, 2025
• Study Completion: April 16, 2025
• Last Updated: September 4, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

FR (2); NL (3); ES (7); PT (3); FI (1); US (12); PL (6); IT (1)