A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB094 in Adults With Parkinson's Disease

NCT ID: NCT03976349

Last Updated: 2025-09-23

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

62 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-08-12

Study Completion Date

2024-08-12

Brief Summary

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The primary objective of this study is to evaluate the safety and tolerability of single and multiple doses of BIIB094 administered via intrathecal (IT) injection to participants with Parkinson's Disease (PD). The secondary objective of this study is to evaluate the pharmacokinetic (PK) profile of BIIB094.The study is open for PD patients with verified presence or absence of variations in the leucine-rich repeated kinase 2 (LRRK2) gene, but also for patients without any verified PD-related genetic variant.

Detailed Description

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Conditions

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Parkinson's Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Investigators Outcome Assessors

Study Groups

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Part A (SAD): BIIB094 Dose 1

Participants will receive a single IT injection of BIIB094 during Part A \[Single Ascending Dose (SAD)\].

Group Type EXPERIMENTAL

BIIB094

Intervention Type DRUG

Administered as specified in the treatment arm.

Part A (SAD): BIIB094 Dose 2

Participants will receive a single IT injection of BIIB094 during Part A (SAD).

Group Type EXPERIMENTAL

BIIB094

Intervention Type DRUG

Administered as specified in the treatment arm.

Part A (SAD): BIIB094 Dose 3

Participants will receive a single IT injection of BIIB094 during Part A (SAD).

Group Type EXPERIMENTAL

BIIB094

Intervention Type DRUG

Administered as specified in the treatment arm.

Part A (SAD): BIIB094 Dose 4

Participants will receive a single IT injection of BIIB094 during Part A (SAD).

Group Type EXPERIMENTAL

BIIB094

Intervention Type DRUG

Administered as specified in the treatment arm.

Part A (SAD): BIIB094 Dose 5

Participants will receive a single IT injection of BIIB094 during Part A (SAD).

Group Type EXPERIMENTAL

BIIB094

Intervention Type DRUG

Administered as specified in the treatment arm.

Part A (SAD): BIIB094 Dose 6

Participants will receive a single IT injection of BIIB094 during Part A (SAD).

Group Type EXPERIMENTAL

BIIB094

Intervention Type DRUG

Administered as specified in the treatment arm.

Part B (MAD): BIIB094 Dose 1

Participants will receive a single IT injection of BIIB094 on multiple days during Part B \[Multiple Ascending Dose (MAD)\].

Group Type EXPERIMENTAL

BIIB094

Intervention Type DRUG

Administered as specified in the treatment arm.

Part B (MAD): BIIB094 (Non LRRK2) Dose 2

Participants \[Non leucine-rich repeat kinase 2 (Non LRRK2)\] will receive a single IT injection of BIIB094 on multiple days during Part B (MAD).

Group Type EXPERIMENTAL

BIIB094

Intervention Type DRUG

Administered as specified in the treatment arm.

Part B (MAD): BIIB094 (LRRK2) Dose 2

Participants (LRRK2) will receive a single IT injection of BIIB094 on multiple days during Part B (MAD).

Group Type EXPERIMENTAL

BIIB094

Intervention Type DRUG

Administered as specified in the treatment arm.

Part B (MAD): BIIB094 (Non LRRK2) Dose 3

Participants (Non LRRK2) will receive a single IT injection of BIIB094 on multiple days during Part B (MAD).

Group Type EXPERIMENTAL

BIIB094

Intervention Type DRUG

Administered as specified in the treatment arm.

Part B (MAD): BIIB094 (LRRK2) Dose 3

Participants (LRRK2) will receive a single IT injection of BIIB094 on multiple days during Part B (MAD).

Group Type EXPERIMENTAL

BIIB094

Intervention Type DRUG

Administered as specified in the treatment arm.

Part A (SAD): Matching Placebo

Participants will receive matching placebo during Part A \[Single Ascending Dose (SAD)\].

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Administered as specified in the treatment arm.

Part B (MAD): Matching Placebo

Participants will receive matching placebo on multiple days during Part B (MAD).

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Administered as specified in the treatment arm.

Interventions

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BIIB094

Administered as specified in the treatment arm.

Intervention Type DRUG

Placebo

Administered as specified in the treatment arm.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use confidential health information in accordance with national and local participant privacy regulations.
* Diagnosed with PD within 7 years at the time of initial enrollment (i.e., at time of SAD enrollment for rollover participants), without major motor fluctuations or dyskinesia that may interfere with study treatment and assessments in the opinion of the investigator after consultation with the Sponsor.
* Modified Hoehn and Yahr Stage ≤ 3.

Exclusion Criteria

* Montreal Cognitive Assessment (MoCA) score less than (\<) 23, dementia, or other significant cognitive impairment that, in the opinion of the Investigator, would interfere with study evaluation.
* History of any brain surgery for PD or a history of focused ultrasound treatment at any time; or history of neuromodulation procedures.
* Transient ischemic attack or stroke or any unexplained loss of consciousness within 1 year before Screening.
* History of unstable angina, myocardial infarction, chronic heart failure, or clinically significant conduction abnormalities within 1 year before Screening.
* Poorly controlled diabetes mellitus, as defined by having dosage adjustment of diabetic medication within 3 months before dosing (Day 1) or glycosylated hemoglobin value greater than or equal to (≥) 8 percent (%) at Screening.
* History or positive test result at Screening for human immunodeficiency virus.
* History or positive test result at Screening for hepatitis C virus antibody.
Minimum Eligible Age

35 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Ionis Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Northwestern University PD and Movement Disorders Center

Chicago, Illinois, United States

Site Status

Quest Research Institute

Farmington Hills, Michigan, United States

Site Status

The Cleveland Clinic Foundation

Cleveland, Ohio, United States

Site Status

Oregon Health and Science University

Portland, Oregon, United States

Site Status

University of Pennsylvania

Philadelphia, Pennsylvania, United States

Site Status

Alliance for Multispecialty Research

Knoxville, Tennessee, United States

Site Status

Inland Northwest Research

Spokane, Washington, United States

Site Status

Research Site

Toronto, Ontario, Canada

Site Status

Montreal Neurological Institute and Hospital

Montreal, Quebec, Canada

Site Status

Sourasky Medical Center

Tel Aviv, , Israel

Site Status

Neuro-SysMed Center

Bergen, , Norway

Site Status

St. Olav University Hospital

Trondheim, , Norway

Site Status

Laboratorios de Investigación Biocruces 3., Hospital de Cruces

Barakaldo, Bizkaia, Spain

Site Status

Hospital General de Catalunya

Barcelona, Vizcaya, Spain

Site Status

Hospital Clinic de Barcelona

Barcelona, , Spain

Site Status

Hospital General Universitario Gregorio Marañón

Madrid, , Spain

Site Status

Research Site

Seville, , Spain

Site Status

Queen Square (Neurology) CRF Site Institute of Neurology & the National Hospital for Neurology and Neurosurgery UCLH

London, , United Kingdom

Site Status

Countries

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United States Canada Israel Norway Spain United Kingdom

Other Identifiers

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2018-002995-42

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

254PD101

Identifier Type: -

Identifier Source: org_study_id

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