Single-Ascending Dose Study of BIIB054 in Healthy Participants and Early Parkinson's Disease

Study Overview
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Brief Summary
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The primary objective of the study is to evaluate the safety and tolerability of a range of single BIIB054 doses, administered as a single intravenous (IV) infusion, in healthy participants and participants with early Parkinson's disease (PD). Secondary objectives of the study are to assess the serum pharmacokinetics (PK) profile of BIIB054 after single-dose administration and to evaluate the immunogenicity of BIIB054 after single-dose administration.

Detailed Description
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Only healthy participants will be enrolled in Cohorts 1 through 6. Only participants with early PD will be enrolled in Cohort 7

Official Title
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A Phase 1 Randomized, Double-Blinded, Placebo-Controlled Single-Ascending Dose Study of the Safety, Tolerability, and Pharmacokinetics of BIIB054 in Healthy Subjects and Subjects With Early Parkinson's Disease

Conditions
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Parkinson's Disease, Healthy

Intervention / Treatment
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* Drug: BIIB054
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Key Inclusion Criteria: All women of childbearing potential and all men must practice effective contraception during the study and for 6 months after their last dose of study treatment. Must have a body mass index from 19 to 32 kg/m2, inclusive. Must be in good health as determined by the Investigator, based on medical history, physical examination, and 12-lead ECG. For cohort 7 only: Diagnosis of idiopathic PD Key Exclusion Criteria: History of cardiovascular disease. Subject smokes more than 5 cigarettes or the equivalent in tobacco daily. Surgery within 3 months prior to Day -1 (other than minor cosmetic surgery and minor dental surgery, as determined by the Investigator). History or positive test result for hepatitis C, hepatitis B, or human immunodeficiency virus (HIV). Female subjects who are pregnant, currently breastfeeding, or attempting to conceive during the study. NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Ages Eligible for Study
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Minimum Age: 40 Years
Maximum Age: 80 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: Cohort 1<br>Single intravenous (IV) low dose infusion with staggered participant dosing | Drug: BIIB054<br>* IV infusion<br>Drug: Placebo<br>* IV infusion<br>|
| Experimental: Cohort 2<br>Single IV ascending dose infusion with staggered participant dosing | Drug: BIIB054<br>* IV infusion<br>Drug: Placebo<br>* IV infusion<br>|
| Experimental: Cohort 3<br>Single IV ascending dose infusion with staggered participant dosing | Drug: BIIB054<br>* IV infusion<br>Drug: Placebo<br>* IV infusion<br>|
| Experimental: Cohort 4<br>Single IV ascending dose infusion with staggered participant dosing | Drug: BIIB054<br>* IV infusion<br>Drug: Placebo<br>* IV infusion<br>|
| Experimental: Cohort 5<br>Single IV ascending dose infusion with staggered participant dosing | Drug: BIIB054<br>* IV infusion<br>Drug: Placebo<br>* IV infusion<br>|
| Experimental: Cohort 6<br>Single IV ascending dose infusion with staggered participant dosing | Drug: BIIB054<br>* IV infusion<br>Drug: Placebo<br>* IV infusion<br>|
| Experimental: Cohort 7<br>Single IV ascending dose infusion with staggered participant dosing | Drug: BIIB054<br>* IV infusion<br>Drug: Placebo<br>* IV infusion<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Adverse events/serious adverse events (AEs/SAEs), | After a range of single BIIB054 doses administered as a single IV infusion in healthy participants and participants with early Parkinson's Disease, clinical laboratory test data, vital signs, neurological and physical examination findings, 12-lead electrocardiogram (ECG) data, and brain magnetic resonance imaging (MRI) findings will be used in the evaluation of AEs/SAEs. | 20 Weeks | 
| Columbia Suicide Severity Rating Scale (C-SSRS) | The Columbia Suicide Severity Rating Scale, or C-SSRS, is a suicidal ideation rating scale created by researchers at Columbia University to evaluate suicidality. | 20 Weeks | 
| Montreal Cognitive Assessment (MoCA) | The Montreal Cognitive Assessment MoCA is a brief cognitive screening tool for Mild Cognitive Impairment. | 20 Weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Evaluate serum BIIB054 concentrations and PK parameters | Area under the concentration-time curve (AUC) from time zero extrapolated to infinity (0-∞), AUC from time 0 to time of the last measurable concentration (AUC0-tlast), maximum concentration (Cmax), time to Cmax (Tmax), elimination half-life (t1/2), and clearance (Cl), will be used to assess the serum PK profile of BIIB054 after single-dose administration. | 20 Weeks | 
| evaluation of anti-BIIB054 antibodies in serum | evaluate the immunogenicity of BIIB054 after single-dose administration | 20 Weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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PD, alpha synuclein