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Parkinson's DiseasePhase 1NCT02758730

Study Assessing Tolerability and Safety and Exploring the Immunogenicity and Therapeutic Activity of AFFITOPE® PD01A in PD-GBA Patients

A Phase 1 study testing AFFITOPE® PD01A + Adjuvant and Adjuvant without active component in people with Parkinson's Disease, run by Affiris AG at 1 site in Germany.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial summary

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. Read more about this study

Who is it for?
Ages 40 to 80 years
Conditions the study is for
Parkinson's Disease
What is being tested?
AFFITOPE® PD01A + Adjuvant and Adjuvant without active component
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
0 participants
Where
1 siteGermany
Study dates
Start not givento Oct 2017
Phase
Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 40 to 80 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Confirmed diagnosis of Parkinson's disease and confirmed carrier status for a heterozygous GBA mutation (PDGBA)
  • Individuals who present in Hoehn\&Yahr Stages I/II/III and fulfill the United Kingdom Parkinson's Disease Society Brain Bank Criteria
  • Confirmed carrier status for a heterozygous GBA mutation (PDGBA)-The result of the MRI scan of the patient's brain has to be consistent with the diagnosis of PD
  • Written informed consent signed and dated by the patient and, preferentially, the caregiver (caregiver is not mandatory)
  • Age between 40 and 80
  • In the investigator's opinion, does not have visual or auditory impairments that would reduce the patients' ability to complete study questionnaires or be unable to receive instructions for these
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  • Female patients of childbearing potential are eligible if they use a medically accepted contraceptive method
  • A potential participant treated with conventional PD therapies must be on stable doses for at least 3 months prior to Visit 1 and during the entire trial period
  • Accepted PD medications include the following: levodopa (alone or in combination with benserazide, carbidopa), catechol-O-methyltransferase (COMT) inhibitors (entacapone, tolcapone), amantadine, non-ergot dopamine agonists (pramipexol, ropinirol, rotigotine), monoamine oxidase-B (MAO-B) inhibitors (rasagiline, selegiline) and anticholinergic medication
  • A potential participant should be on stable doses of all medications he/she is taking because of consisting illnesses according to medical history (except PD therapies, these will be recorded separately) for at least 30 days prior to Visit 1 if considered relevant by the investigator
  • Able to communicate well with the investigator, to understand and comply with the requirements of the study

Who cannot join

  • Pregnant women
  • Sexually active women of childbearing potential who are not using a medically accepted birth control method
  • Participation in another clinical trial within 3 months before Visit 1
  • History of questionable compliance to visit schedule; patients not expected to complete the clinical trial
  • Presence or history of allergy to components of the vaccine if considered relevant by the investigator
  • Contraindication for MRI imaging such as metallic implants (e.g. endoprosthesis, stents, cardiac pacemakers) which are not MR compatible at 3.0 Tesla with the given MR protocol, other foreign metal bodies (e.g. bullets, metal splinters, e.g.) or claustrophobia
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  • Missing agreement to be informed about incident findings and consultation of a neuroradiologist
  • Contraindication for PET, that is, pregnancy and breast feeding.
  • Ongoing participation in other interventional studies or clinical trials using radiotracers
  • Dementia according to Diagnostic and Statistical Manual (DSM) IV criteria
  • History and/or presence of autoimmune disease, if considered relevant by the investigator
  • Recent (≤3 years since last specific treatment) history of cancer (Exceptions: non-melanoma skin cancer, intraepithelial cervical neoplasia)
  • Active infectious disease (e.g., Hepatitis B, C)
  • Presence and/or history of Immunodeficiency (e.g., HIV)
  • Significant systemic illness (e.g., chronic renal failure, chronic liver disease, poorly controlled diabetes, poorly controlled congestive heart failure, other deficiencies), if considered relevant by the investigator
  • History of significant psychiatric illness such as schizophrenia, bipolar affective disorder or psychotic depression
  • Parkinson-like disease secondary to drug therapy side effects (e.g., due to exposure to medications that deplete dopamine [reserpine, tetrabenazine] or block dopamine receptors [neuroleptics, antiemetics])
  • Parkinson-plus syndromes (e.g., multiple system atrophy (MSA), progressive supranuclear palsy (PSP)), Dementia with Lewy Bodies (DLB)
  • Heredodegenerative disorders other than PDGBA, evidence for other genetic forms of PD (e.g. LRRK2, Parkin gene mutations)
  • Alcoholism or substance abuse within the past year (alcohol or drug intoxication)
  • Prior and/or current treatment with experimental immunotherapeutics including Intravenous Immunoglobulin (IVIG)
  • Prior and/or current treatment with immune modulating drugs:
  • Interleukins, Interferons, Tumor Necrosis Factor (TNF) and analogues, colony stimulating factor compounds
  • Ciclosporin, Tacrolimus, Sirolimus and analogues
  • Cytostatic drugs and certain DMARD for rheumatoid arthritis (and similar autoimmune disorders) (e.g. Cyclophosphamid, Azathioprin, methotrexate, sulfasalazine, leflunomide, sodium aurothiomalate (Gold), cyclosporin) and analogues
  • Systemic (gluco)corticoid therapy
  • All antibody therapies (e.g., anti cluster of differentiation 3 (CD3), anti cluster of differentiation 25 (CD25) or also anti-lymphocyte globulins) that might modulate, enhance or weaken an immune response
  • Change in dose of standard treatments for PD within 3 months prior to Visit 1
  • Change in dose of previous and current medications which the patient is taking because of consisting illnesses according medical history (except PD therapies, these will be recorded separately) within the last 30 days prior to Visit 1, if clinically relevant
  • Treatment with deep brain stimulation
  • Venous status rendering it impossible to place an i.v. access

Trial locations and contacts

1 site in 1 country.

Germany1 site

  • University Hospital Tübingen

    Tübingen, 72076

Other trials in Germany

About this study

Official title: A Randomized, Placebo-controlled, Parallel Group, Patient-blind, Single-center Phase I Pilot Study Assessing Tolerability and Safety and Exploring the Immunogenicity and Therapeutic Activity of AFFITOPE® PD01A, a New Vaccine Against Alpha-synuclein, in Patients With PD-GBA

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).

Being tested
AFFITOPE® PD01A + Adjuvant, Adjuvant without active component
Keywords
GBA mutation
Medical subject headings
Parkinson Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • AFFITOPE® PD01A + AdjuvantBiological

    s.c. injection

  • Adjuvant without active componentBiological

    s.c. injection

Study groups in NCT02758730
Study groupRoleWhat they receive
AFFITOPE® PD01A + AdjuvantExperimental3 injections of 75µg AFFITOPE® PD01A/ adjuvanted, once every 4 weeks
Receives: Biological: AFFITOPE® PD01A + Adjuvant
Adjuvant without active componentPlacebo comparator3 injections of Placebo once every 4 weeks
Receives: Biological: Adjuvant without active component

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of patients who withdraw due to Adverse Events (AEs) and reason for withdrawal

    Measured: 52 weeks

  2. Occurrence of any Serious Adverse Event (SAE) possibly, probably or definitely related to the study vaccine at any time during the study

    Measured: 52 weeks

  3. Occurrence of any Grade 3 or higher AEs possibly, probably or definitely related to the study vaccine within 4 weeks after the vaccinations

    Measured: 12 weeks (week 0 to 12)

  4. 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

    Measured: up to 52 weeks

  5. Occurrence of solicited systemic AEs

    Headache, myalgia (muscle pain), fever, fatigue, nausea within 1 week (Day 1-7) after the vaccinations: Severity and duration.

    Measured: up to 52 weeks

  6. Occurrence of unsolicited non-serious AEs within four weeks after the vaccinations

    Severity, duration and relationship to vaccination

    Measured: 12 weeks (week 0 to 12)

Secondary outcomes (3)

  1. 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)

    Measured: 52 weeks

  2. 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

    Measured: 52 weeks

  3. Biomarker data at visit 6 (or EDV) compared to baseline

    β-Glucocerebrosidase (GCase) enzyme activity

    Measured: 52 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted2 May 2016 (estimated)
  2. Primary completionOct 2017 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Single blind
Who is masked
Participant
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02758730
Sponsor's protocol ID
AFF010
Other IDs
2016-001462-28, 2016
Status last verified
Aug 2016
First submitted
26 Apr 2016
Last updated
5 Aug 2016
Results posted
No
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing AFFITOPE® PD01A + Adjuvant and Adjuvant without active component in people with Parkinson's Disease, run by Affiris AG at 1 site in Germany.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 11 inclusion and 31 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: withdrawn, as of the sponsor's last update on 5 Aug 2016. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was withdrawn before enrolling participants, so there are no results.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of patients who withdraw due to Adverse Events (AEs) and reason for withdrawal (time frame: 52 weeks). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02758730.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Aug 2016.

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Clinical Trial NCT02758730 | AzTrials