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

Phase 1

Completed

Conditions: Parkinsonism
Parkinson's Disease

Interventions: Biological: UB-312
Biological: Placebo

Registration Number: NCT04075318

Lead Sponsor: United Neuroscience Ltd.

Brief Summary: 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: 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.

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 70

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

Study & Design

Study Type: INTERVENTIONAL

Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
UB-312 300 mcg	UB-312	UB-312 300 mcg by intramuscular injection at Weeks 1, 5 and 13
UB-312 40/1000 mcg	UB-312	UB-312 40 mcg at Week 1 and 1000 mcg at Weeks 5 and 13 by intramuscular injection
UB-312 40 mcg	UB-312	UB-312 40 mcg by intramuscular injection at Weeks 1, 5 and 13
UB-312 100 mcg	UB-312	UB-312 100 mcg by intramuscular injection at Weeks 1, 5 and 13
UB-312 300/100 mcg	UB-312	UB-312 300 mcg at Week 1 and 100 mcg at Weeks 5 and 13 by intramuscular injection
UB-312 40/300 mcg	UB-312	UB-312 40 mcg at Week 1 and 300 mcg at Weeks 5 and 13 by intramuscular injection
UB-312 2000 mcg	UB-312	UB-312 2000 mcg by intramuscular injection at Weeks 1, 5 and 13
Placebo	Placebo	Placebo by intramuscular injection at Weeks 1, 5 and 13
UB-312 1000 mcg	UB-312	UB-312 1000 mcg by intramuscular injection at Weeks 1, 5 and 13
Part A: UB-312 40 mcg	UB-312	UB-312 40 mcg by intramuscular injection at Weeks 1, 5 and 13
Part A: UB-312 100 mcg	UB-312	UB-312 100 mcg by intramuscular injection at Weeks 1, 5 and 13
Part A: UB-312 40/300 mcg	UB-312	UB-312 40 mcg at Week 1 and 300 mcg at Weeks 5 and 13 by intramuscular injection
Part A: UB-312 300 mcg	UB-312	UB-312 300 mcg by intramuscular injection at Weeks 1, 5 and 13
Part A: UB-312 40/1000 mcg	UB-312	UB-312 40 mcg at Week 1 and 1000 mcg at Weeks 5 and 13 by intramuscular injection
Part A: UB-312 1000 mcg	UB-312	UB-312 1000 mcg by intramuscular injection at Weeks 1, 5 and 13
Part B: UB-312 300/100 mcg	UB-312	UB-312 300 mcg at Week 1 and 100 mcg at Weeks 5 and 13 by intramuscular injection
Part B: UB-312 300 mcg	UB-312	UB-312 300 mcg at Weeks 1, 5 and 13 by intramuscular injection
Part A: UB-312 2000 mcg	UB-312	UB-312 2000 mcg by intramuscular injection at Weeks 1, 5 and 13
Part A: Placebo	Placebo	Placebo by intramuscular injection at Weeks 1, 5 and 13
Part B: Placebo	Placebo	Placebo by intramuscular injection at Weeks 1, 5 and 13

Primary Outcome Measures

Name	Time	Method
Frequency of Adverse Events	44 weeks	Number of AEs will be assessed
Immunogenicity of UB-312 as Determined by Anti-aSyn Antibodies in Blood	44 weeks	Number of Participants with Anti-aSyn Antibodies in Blood from Weeks 1 through 45.
Immunogenicity of UB-312 as Determined by Anti-aSyn Antibodies in CSF	44 weeks	Number of Participants with Anti-aSyn Antibodies in CSF from Weeks 1 through 45.