A Randomized, Placebo-controlled, Parallel Group, Patient-blind, Phase I Study Assessing the Safety and Exploring the Immunogenicity/Therapeutic Activity of AFFITOPE® PD01A and PD03A in Patients With Early Multiple System Atrophy
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- Multiple System Atrophy
- Sponsor
- Affiris AG
- Enrollment
- 30
- Locations
- 2
- Primary Endpoint
- Number of patients who withdraw due to Adverse Events (AEs)
- Status
- Completed
- Last Updated
- 8 years ago
Overview
Brief Summary
This is a randomized controlled parallel Group phase I study to investigate the safety and immunological/ therapeutic activity of two new vaccines, AFFITOPE® PD01A and AFFITOPE® PD03A, given to patients with early Multiple System Atrophy (MSA).
In total 30 patients are planned to be enrolled in the study: 12 patients in each treatment arm who will receive either 75µg AFFITOPE® PD01A (with adjuvant) or 75µg AFFITOPE® PD03A (with adjuvant) and 6 patients in the control group who will receive the reference substance (Placebo). Over a study duration of 52 weeks, the study participants will receive 4 injections as basic immunization in a 4-weekly interval and 1 boost immunization 36 weeks after the first injection. Male and female patients aged 30 to 75 years can participate in the trial. 2 study sites in France (Bordeaux and Toulouse) will be involved.
AFF009 is part of the project SYMPATH funded by the European Commission (FP7-HEALTH-2013-INNOVATION-1 project; N° HEALTH-F4-2013-602999).
Investigators
Eligibility Criteria
Inclusion Criteria
- •Possible or probably MSA diagnosis (MSA-P or MSA-C) according to Gilman 2008 consensus criteria
- •Onset of MSA symptoms less than 4 years
- •Participants with an anticipated survival of at least 3 years in the opinion of the PI
- •Written informed consent obtained prior to study entry
- •MSA patient \> 30 and \< 75 years of age at time of study entry
- •Female patients of childbearing potential using a medically accepted contraceptive method
- •Stable medication for MSA symptoms (Levodopa, Dopamine agonists, Midodrine, Fludrocortisone, monoamine oxidase-B and Catechol-O-methyltransferase inhibitors; Antidepressants, Laxatives, NSAIDs or paracetamol as basic medication for pain in the musculoskeletal system)
Exclusion Criteria
- •Pregnant or lactating women
- •Sexually active women of childbearing potential not using a medically accepted birth control method
- •Patients with dementia (MOCA at Screening \< 21)
- •Speech impairment as assessed by a score of ≥ 3 on UMSARS question 1
- •Swallowing impairment as assessed by a score of ≥ 3 on UMSARS question 2
- •Impairment in ambulation as assessed by a score of ≥ 3 on UMSARS question 7
- •History or evidence of any other central nervous system disorder like stroke, angioma and other relevant neurological diseases
- •History of malignancy other than skin cancer during the last 5 years (if considered to be cured, patient might be included)
- •Active or passive vaccination 4 weeks before the first vaccination on Day 0 and during the main study period ending on Day
- •Emergency vaccinations are acceptable
Outcomes
Primary Outcomes
Number of patients who withdraw due to Adverse Events (AEs)
Time Frame: 12 months
Physical Examination
Time Frame: 12 months
New findings or change in pre-existing findings assessed in physical examinations over time (study period)
Body mass
Time Frame: 12 months
Change of Body mass over time (study period)
Occurrence of Adverse Events and Serious Adverse Events
Time Frame: 12 months
Evaluation of Adverse Events and Serious Adverse Events in regards to autoimmune reactions
Vital signs
Time Frame: 12 months
Change in vital signs. The Evaluation includes the changes in blood pressure, heart rate, respiratory rate and Body temperature over time (measured at each visit).
Clinical significance/ changes in laboratory parameters over time (study period)
Time Frame: 12 months
Laboratory assessment includes hematology, biochemistry, coagulation, serology and urinanalysis
Safety related evaluation of MRI results of patients' brain after visit 5 and visit 8 compared to baseline
Time Frame: 12 months
Safety measures will e.g. include the occurrence of inflammatory reactions (meningoencephalitis), new/changed hemorrhages and lacunar infarcts.
Neurological Examination
Time Frame: 12 months
New findings or change in pre-existing findings assessed in neurological examinations over time (study period)
Secondary Outcomes
- Immunological activity of AFFITOPE® vaccines PD01A and PD03A.(12 months)
- Change in motor symptoms at Visit 5 and Visit 8 compared to baseline(12 months)
- Change in non-motor symptoms at Visit 5 and Visit 8 compared to baseline(12 months)