This trial is active, not recruiting.

Condition alzheimer's disease
Treatment aadvac1
Phase phase 1
Sponsor Axon Neuroscience SE
Start date January 2014
End date August 2016
Trial size 25 participants
Trial identifier NCT02031198, 2013-004499-36, AC-AD-002


This follow-up study continues to observe patients who have completed the phase 1 trial of AADvac1, for another 18 months.

Long-term safety and behavior of the immune response to AADvac1 over time are the main points of interest.

AADvac1 is a vaccine directed against pathologically modified Alzheimer tau protein that is the main constituent of neurofibrillary tangles (NFTs), and is intended to be a disease-modifying treatment for Alzheimer's disease, i.e. to halt its progress.

As this study is a Phase I study focused on tolerability and safety, efficacy will be assessed in an exploratory manner.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Patients who have received 6 doses in the previous trial will be administered 1-2 booster doses of AADvac1 (2 if their antibody titers decline below those achieved in the previous trial). Patients who have received 3 doses in the previous trial will be administered another 3 doses, then vaccinated with booster doses as above.
aadvac1 Axon peptide 108 (coupled to KLH), 40ug/0.3mL
Active immunization against pathological Alzheimer's disease tau protein

Primary Outcomes

Tolerability and safety profile of AADvac1 in patients with mild-to-moderate Alzheimer's disease
time frame: Tolerability & safety are assessed over a period of 18+ months

Secondary Outcomes

Immunogenicity of AADvac1
time frame: Immune response to the vaccine will be assessed over 18 month
Patient cognition
time frame: 18+ months

Eligibility Criteria

Male or female participants from 50 years up to 86 years old.

Inclusion Criteria: 1. Completion of visit V8 of the AADvac1 phase I study AXON CO 18700 (EUDRACT 2012-003916-29). 2. Informed consent capability (as determined by an independent neurologist/psychiatrist). 3. Written informed consent signed and dated by the patient and the caregiver. 4. Availability of a partner/caregiver knowing the patient and being able to accompany the patient to the visits 5. Adequate visual and auditory abilities and language skills to allow neuropsychological testing. 6. Female patients are only eligible for the study if they are either surgically sterile or at least 2 years postmenopausal. 7. Sexually active males must be using reliable contraception methods (i.e. condoms) or be surgically sterile. Exclusion Criteria: 1. Pregnant women. 2. Participation in another clinical trial during the course of this study. 3. Contraindication for MRI imaging such as MRI-incompatible metallic endoprosthesis or MRI-incompatible stent implantation 4. History and/or presence of autoimmune disease, if considered relevant by the investigator. 5. Significant systemic illness (e.g., chronic renal failure, chronic liver disease, poorly controlled diabetes, poorly controlled congestive heart failure, congenital long QT syndrome, other deficiencies), if considered relevant by the investigator. 6. Current treatment with immunosuppressive drugs.

Additional Information

Official title An 18-months Open Label Phase I Follow-up Study on Patients With Alzheimer's Disease Who Have Completed the AADvac1 Phase I Study "AXON CO 18700"
Principal investigator Reinhold Schmidt, Professor
Description AADvac1 is a candidate therapeutic vaccine for Alzheimer's disease that targets misfolded tau protein, a common denominator of neurofibrillary pathology. Based on preclinical results, the intervention is expected to reduce the number of neurofibrillary tangles, remove hyperphosphorylated tau protein and reduce the amount of oligomerized and insoluble pathological tau in the brain, to halt the spread of neurofibrillary pathology through the brain, and thus prevent associated cognitive decline. The vaccine's antigenic determinant is a synthetic peptide derived from a tau protein sequence, which is coupled to keyhole limpet hemocyanin (KLH) and uses aluminum hydroxide (Alhydrogel) as an adjuvant. At present AADvac1 is intended as an active immunotherapy for patients with diagnosed Alzheimer's disease (AD). According to need, patients will receive additional immunization doses beyond those administered in the preceding pase 1 trial; the raised titers of therapeutic antibodies and possible benefits of the treatment can extend beyond the duration of the study. Because of the central role of pathological misfolded tau protein in the etiology of AD, the vaccine is expected to be more effective than active or passive immunotherapies aiming to eliminate the amyloid β plaques that have been clinically investigated so far.
Trial information was received from ClinicalTrials.gov and was last updated in July 2016.
Information provided to ClinicalTrials.gov by Axon Neuroscience SE.