This is a randomized, placebo-controlled, parallel group, patient-blind, single-center phase I clinical trial of repeated once every 4 weeks administration by subcutaneous injection of AFFITOPE® PD01A, adsorbed to aluminium oxide in 30 patients with PD-GBA over a treatment period of 8 weeks. Patients will be randomized in a 2:1 ratio to two different treatment groups: A) 75 µg AFFITOPE® PD01A, adsorbed to aluminium oxide and B) placebo (= 1 mg aluminium oxide). Over a study duration of 52 weeks, each patient will receive 3 injections of AFFITOPE® PD01A or placebo during the participation in the clinical trial. Patients will either receive 75 µg AFFITOPE® PD01A adsorbed to 1 mg aluminium oxide or placebo (=1mg aluminium oxide). The treatment group consists of 20 PDGBA patients, the placebo group of 10 PDGBA patients. Male and female patients aged 40 to 80 years can participate in the trial. AFF010 is part of the project MULTISYN funded by the European Commission (FP7-HEALTH-2013-INNOVATION-1 project; N° HEALTH-F4-2013-602646).
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
s.c. injection
s.c. injection
University Hospital Tübingen
Tübingen, Germany
Number of patients who withdraw due to Adverse Events (AEs) and reason for withdrawal
Time frame: 52 weeks
Occurrence of any Serious Adverse Event (SAE) possibly, probably or definitely related to the study vaccine at any time during the study
Time frame: 52 weeks
Occurrence of any Grade 3 or higher AEs possibly, probably or definitely related to the study vaccine within 4 weeks after the vaccinations
Time frame: 12 weeks (week 0 to 12)
Occurrence of solicited local AEs
Injection site pain, erythema (redness), hyperthermia at injection site, itching, edema (swelling), induration \[hardening\], granuloma within 1 week (Day 1-7) after the vaccinations: Severity and duration
Time frame: up to 52 weeks
Occurrence of solicited systemic AEs
Headache, myalgia (muscle pain), fever, fatigue, nausea within 1 week (Day 1-7) after the vaccinations: Severity and duration.
Time frame: up to 52 weeks
Occurrence of unsolicited non-serious AEs within four weeks after the vaccinations
Severity, duration and relationship to vaccination
Time frame: 12 weeks (week 0 to 12)
Immunological activity of AFFITOPE® vaccine PD01A over time (study period)
Titer of immunoglobulin G (IgG) Abs specific for the immunizing peptide (PD01A), the carrier (KLH) and the target (targeted native α-Synuclein (aSyn) epitope coupled to bovine serum albumin (BSA) (mandatory) or presented in different forms - particularly monomers, pre-fibrils and fibrils (optional)) assessed by Enzyme-Linked Immunosorbent Assay (ELlSA) (or an equivalent method)
Time frame: 52 weeks
Imaging efficacy variables at visit 6 (or EDV) compared to baseline
11C-Pittsburgh Compound B (11C-PIB), 18F-Fluorodeoxyglucose-Positron Emission Tomography (18F-FDG-PET), resting-state functional magnetic resonance imaging (fMRI), diffusion-weighted/tensor magnetic resonance imaging (MRI), Magnetic Resonance-Spectroscopy
Time frame: 52 weeks
Biomarker data at visit 6 (or EDV) compared to baseline
β-Glucocerebrosidase (GCase) enzyme activity
Time frame: 52 weeks
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.