Study of UB-312 in Healthy Participants and Parkinson's Disease Patients

Study Overview
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Brief Summary
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This is a 44-week, randomized, placebo-controlled, double-blind, single-center, phase 1 clinical trial consisting of a dose-escalation Part A study in healthy participants, followed by a Part B in participants with Parkinson's disease with a selected doses from Part A.

Detailed Description
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This is a first-in-human Phase 1 study to determine the safety, tolerability, and immunogenicity of UB-312 in healthy participants and in participants with Parkinson's disease (PD). UB-312 is a UBITh®-enhanced synthetic peptide-based vaccine and may provide an active immunotherapy option for treating synucleinopathies including the most prevalent form, PD. The study consists of two parts. Part A of the study with healthy participants will consist of dose escalation and cohort staggering for up to seven planned dose levels or placebo. Part B of the study will consist of two cohorts of participants with Parkinson's disease (PD). Dosing for Part B will be based on safety, tolerability and immunogenicity from Part A. All eligible participants will be enrolled in a 44-week study consisting of 20 weeks of treatment and 24 weeks of follow-up.

Official Title
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A Phase 1 Study to Evaluate the Safety, Tolerability, and Immunogenicity of UBITh® PD Immunotherapeutic Vaccine (UB-312) in Healthy Participants and Participants With Parkinson's Disease

Conditions
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Parkinson's Disease, Parkinsonism

Intervention / Treatment
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* Biological: UB-312
* Biological: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male or female aged 40 to 85 years old, inclusive at screening Expected to be able to undergo all study procedures Other inclusion criteria apply For Part B only: A diagnosis of PD, confirmed by a neurologist Hoehn &Yahr Stage ≤ III at Screening Stable treatment of permitted antiparkinsonian medications from 30 days prior to first study drug administration or 60 days for MAO-B inhibitors, and expected to remain stable throughout the study Exclusion Criteria: Clinically significant abnormalities, as judged by the investigator History of medical, neurological or psychiatric conditions which in the opinion of the investigator may compromise participant's safety or scientific value of the study Acute or chronic infection as judged by the investigator, for positive human immunodeficiency virus (HIV), hepatitis C virus (HCV) or hepatitis B virus (HBV) History or evidence of an autoimmune disorder History of anergy. Participated/participating in any clinical trial with monoclonal antibodies or vaccines directed at aSyn Other exclusion criteria apply For Part B only: Other known or suspected cause of Parkinsonism other than idiopathic PD History or evidence at Screening of PD-related freezing episodes, falls, or orthostatic hypotension Dopamine transporter single-photon emission computerized tomography scan (DaTscan) inconsistent with dopamine transporter deficit. Clinically significant neurological disease other than PD

Ages Eligible for Study
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Minimum Age: 40 Years
Maximum Age: 85 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: UB-312 40 mcg<br>UB-312 40 mcg by intramuscular injection at Weeks 1, 5 and 13 | Biological: UB-312<br>* A synthetic peptide-based vaccine<br>|
| Experimental: UB-312 100 mcg<br>UB-312 100 mcg by intramuscular injection at Weeks 1, 5 and 13 | Biological: UB-312<br>* A synthetic peptide-based vaccine<br>|
| Experimental: UB-312 40/300 mcg<br>UB-312 40 mcg at Week 1 and 300 mcg at Weeks 5 and 13 by intramuscular injection | Biological: UB-312<br>* A synthetic peptide-based vaccine<br>|
| Experimental: UB-312 300 mcg<br>UB-312 300 mcg by intramuscular injection at Weeks 1, 5 and 13 | Biological: UB-312<br>* A synthetic peptide-based vaccine<br>|
| Experimental: UB-312 40/1000 mcg<br>UB-312 40 mcg at Week 1 and 1000 mcg at Weeks 5 and 13 by intramuscular injection | Biological: UB-312<br>* A synthetic peptide-based vaccine<br>|
| Experimental: UB-312 1000 mcg<br>UB-312 1000 mcg by intramuscular injection at Weeks 1, 5 and 13 | Biological: UB-312<br>* A synthetic peptide-based vaccine<br>|
| Experimental: UB-312 2000 mcg<br>UB-312 2000 mcg by intramuscular injection at Weeks 1, 5 and 13 | Biological: UB-312<br>* A synthetic peptide-based vaccine<br>|
| Placebo Comparator: Placebo<br>Placebo by intramuscular injection at Weeks 1, 5 and 13 | Biological: Placebo<br>* Matching placebo<br>|
| Experimental: UB-312 300/100 mcg<br>UB-312 300 mcg at Week 1 and 100 mcg at Weeks 5 and 13 by intramuscular injection | Biological: UB-312<br>* A synthetic peptide-based vaccine<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Frequency of Adverse Events | Number of AEs will be assessed | 44 weeks | 
| Immunogenicity of UB-312 as determined by anti-aSyn antibodies in blood and CSF | Immunogenicity will be measured by change from baseline of blood anti-aSyn antibody titers. | Weeks 1, 2, 5, 6, 9, 13, 14, 17, 21, 29, 37 and 45 | 
| Immunogenicity of UB-312 as determined by anti-aSyn antibodies in blood and CSF | Immunogenicity will be measured by change from baseline of CSF anti-aSyn antibody titers | Weeks 1, 21 and 45 |