Tolerability and Safety of Subcutaneous Administration of Two Doses of AFFITOPE® PD01A in Early Parkinson's Disease

A Randomized, Controlled, Parallel Group, Patient-blinded, Single-center, Phase I Pilot Study to Assess Tolerability and Safety of Repeated Subcutaneous Administration of Two Doses of AFFITOPE® PD01A Formulated With Adjuvant to Patients With Parkinson's Disease

This is a phase I study to assess the tolerability and safety of 4 injections of two different doses of AFFITOPE® PD01A formulated with an adjuvant to patients with early Parkinson's disease in male and female patients aged 45 to 65 years (or age between 40 and 45 years if there is no evidence for genetic forms of the disease and the diagnosis of idiopathic Parkinson's disease was confirmed, after approval by Sponsor). One study site in Vienna (Austria) will be involved. Each patient's participation will last 1 year.

In addition, up to 8 patients will be offered participation within an untreated control group.

Study Overview

• Status: Completed
• Conditions: Parkinson's Disease
• Intervention / Treatment: Biological: AFFITOPE® PD01A; Other: Control

• Study Type: Interventional
• Enrollment (Actual): 32
• Phase: Phase 1

Contacts and Locations

Study Locations

• Austria
  • Vienna, Austria, 1080
    • Studienzentrum der PROSENEX, AmbulatoriumbetriebsgesmbH an der Confraternität-Privatklinik Josefstadt, Skodagasse 32, 1080 Vienna

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 40 years to 65 years (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Informed consent capability
• Early PD (≤ 4 years), Hoehn&Yahr Stages I/II and fulfill the UK Parkinson's Disease Society Brain Bank Criteria
• Brain magnetic resonance imaging (MRI) scan and DAT-SPECT scan are consistent with the diagnosis of PD
• Age between 45 and 65 years or age between 40 and 45 years if there is no evidence for genetic forms of the disease and the diagnosis of idiopathic PD was confirmed, after approval by Sponsor
• Caregiver able to attend all visits with patient
• Stable doses of medications (levodopa (+/- benserazide, carbidopa), COMT inhibitors (entacapone, tolcapone), non-ergot dopamine agonists (pramipexol, ropinirol, rotigotine), the MAO-B inhibitor rasagiline and amantadine are allowed)

Exclusion Criteria:

• Women of childbearing potential without birth control or pregnant women
• Participation in another clinical trial
• Autoimmune disease or allergy to components of the vaccine
• Contraindications for MRI, DAT-SPECT, colonoscopy including biopsy or lumbar puncture
• Dementia
• History of cancer (Exceptions: basal cell carcinoma, intraepithelial cervical neoplasia)
• Active infectious disease
• Immunodeficiency
• Significant systemic illness or psychiatric illness
• Parkinson-like disease secondary to drug therapy side effects
• Parkinson-plus syndromes
• Heredodegenerative disorders
• Alcoholism or substance abuse
• Prior treatment with experimental immunotherapeutics for PD including IVIG, with immunosuppressive drugs or treatment with deep brain stimulation
• Venous status rendering it impossible to place an i.v. access

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: RANDOMIZED
• Interventional Model: PARALLEL
• Masking: SINGLE

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: A: AFFITOPE® PD01A + Adjuvant; 4 injections of 15µg AFFITOPE® PD01A/ adjuvanted, once every 4 weeks	Biological: AFFITOPE® PD01A; s.c. injection
Experimental: B: AFFITOPE® PD01A + Adjuvant; 4 injections of 75µg AFFITOPE® PD01A/ adjuvanted, once every 4 weeks	Biological: AFFITOPE® PD01A; s.c. injection
Other: Control; Untreated control group	Other: Control; Untreated control

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Tolerability/Safety	Occurrence of any AE (including a clinical grading scale of AEs according to NCI-CTCAE Version 4.02 (2009) for assessments of AEs + toxicity and including a grading of local injection site reactions according to the FDA Guidance for Industry: Toxicity Scale for Healthy Adult and Adolescents Patients Enrolled in Preventive Vaccine Clinical Trials (2007)); Occurrence of any SAE; Withdrawal criteria (number of patients who withdraw due to AEs/ reason for withdrawal)	12 month

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Immunological	- Titer of vaccination induced antibodies directed towards vaccine components, alpha- and beta synuclein	12 month
Clinical Activity	Change in motor symptoms (MDS-UPDRS III); Change in non-motor PD symptoms (e.g.; MDS-UPDRS Ia, II, PDQ39, cognition); Change in biological and radiological markers (e.g. CSF alpha synuclein levels)	12 month

Collaborators and Investigators

• Sponsor: Affiris AG
• Investigators: Principal Investigator: Dieter Volc, Prim. Dr., Studienzentrum der PROSENEX, AmbulatoriumbetriebsgesmbH an der Confraternität-Privatklinik Josefstadt, Skodagasse 32, 1080 Vienna

Publications and helpful links

General Publications

• McFarthing K, Simuni T. Clinical Trial Highlights: Targetting Alpha-Synuclein. J Parkinsons Dis. 2019;9(1):5-16. doi: 10.3233/JPD-189004. No abstract available.

Study record dates

Study Major Dates

• Study Start: February 1, 2012
• Primary Completion (Actual): May 1, 2014
• Study Completion (Actual): May 1, 2014

Study Registration Dates

• First Submitted: March 16, 2012
• First Submitted That Met QC Criteria: March 29, 2012
• First Posted (Estimate): April 2, 2012

Study Record Updates

• Last Update Posted (Estimate): August 17, 2015
• Last Update Submitted That Met QC Criteria: August 14, 2015
• Last Verified: August 1, 2015

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Parkinsonian Disorders; Basal Ganglia Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases; Parkinson Disease
• Other Study ID Numbers: AFF008; 2011-002650-31 (EudraCT Number)