NCT03976349

Brief Summary

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.

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
62

participants targeted

Target at P75+ for phase_1

Timeline
Completed

Started Aug 2019

Longer than P75 for phase_1

Geographic Reach
6 countries

18 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

June 4, 2019

Completed
2 days until next milestone

First Posted

Study publicly available on registry

June 6, 2019

Completed
2 months until next milestone

Study Start

First participant enrolled

August 12, 2019

Completed
5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

August 12, 2024

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

August 12, 2024

Completed
Last Updated

September 23, 2025

Status Verified

September 1, 2025

Enrollment Period

5 years

First QC Date

June 4, 2019

Last Update Submit

September 22, 2025

Conditions

Parkinson's Disease

Outcome Measures

Primary Outcomes (1)

  • Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

    An AE is any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An SAE is any untoward medical occurrence that at any dose: results in death; in the view of the Investigator, places the participant at immediate risk of death (a life-threatening event); however, this does not include an event that, had it occurred in a more severe form, might have caused death; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; results in a congenital anomaly/birth defect; is a medically important event.

    Part A: Screening (Day -42) up to Day 85, Part B: Screening (Day -77) up to Day 253

Secondary Outcomes (6)

  • Serum Concentrations of BIIB094

    Part A: pre-dose through Day 57, Part B: pre-dose through Day 169

  • Area Under the Concentration-Time Curve From Time Zero Extrapolated to Infinity (AUCinf) of BIIB094

    Part A: pre-dose through Day 57, Part B: pre-dose through Day 169

  • Area Under the Concentration-Time Curve From Time Zero to Last Quantifiable Concentration (AUClast) of BIIB094

    Part A: pre-dose through Day 57, Part B: pre-dose through Day 169

  • Maximum Concentration (Cmax) of BIIB094

    Part A: pre-dose through Day 57, Part B: pre-dose through Day 169

  • Time to Reach Maximum Concentration (Tmax) of BIIB094

    Part A: pre-dose through Day 57, Part B: pre-dose through Day 169

  • +1 more secondary outcomes

Study Arms (13)

Part A (SAD): BIIB094 Dose 1

EXPERIMENTAL

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

Drug: BIIB094

Part A (SAD): BIIB094 Dose 2

EXPERIMENTAL

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

Drug: BIIB094

Part A (SAD): BIIB094 Dose 3

EXPERIMENTAL

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

Drug: BIIB094

Part A (SAD): BIIB094 Dose 4

EXPERIMENTAL

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

Drug: BIIB094

Part A (SAD): BIIB094 Dose 5

EXPERIMENTAL

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

Drug: BIIB094

Part A (SAD): BIIB094 Dose 6

EXPERIMENTAL

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

Drug: BIIB094

Part B (MAD): BIIB094 Dose 1

EXPERIMENTAL

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

Drug: BIIB094

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

EXPERIMENTAL

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

Drug: BIIB094

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

EXPERIMENTAL

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

Drug: BIIB094

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

EXPERIMENTAL

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

Drug: BIIB094

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

EXPERIMENTAL

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

Drug: BIIB094

Part A (SAD): Matching Placebo

PLACEBO COMPARATOR

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

Drug: Placebo

Part B (MAD): Matching Placebo

PLACEBO COMPARATOR

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

Drug: Placebo

Interventions

BIIB094DRUG

Administered as specified in the treatment arm.

Part A (SAD): BIIB094 Dose 1Part A (SAD): BIIB094 Dose 2Part A (SAD): BIIB094 Dose 3Part A (SAD): BIIB094 Dose 4Part A (SAD): BIIB094 Dose 5Part A (SAD): BIIB094 Dose 6Part B (MAD): BIIB094 (LRRK2) Dose 2Part B (MAD): BIIB094 (LRRK2) Dose 3Part B (MAD): BIIB094 (Non LRRK2) Dose 2Part B (MAD): BIIB094 (Non LRRK2) Dose 3Part B (MAD): BIIB094 Dose 1
PlaceboDRUG

Administered as specified in the treatment arm.

Part A (SAD): Matching PlaceboPart B (MAD): Matching Placebo

Eligibility Criteria

Age35 Years - 80 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (18)

Northwestern University PD and Movement Disorders Center

Chicago, Illinois, 60611, United States

Location

Quest Research Institute

Farmington Hills, Michigan, 48334, United States

Location

The Cleveland Clinic Foundation

Cleveland, Ohio, 44195, United States

Location

Oregon Health and Science University

Portland, Oregon, 97239, United States

Location

University of Pennsylvania

Philadelphia, Pennsylvania, 19107, United States

Location

Alliance for Multispecialty Research

Knoxville, Tennessee, 37920, United States

Location

Inland Northwest Research

Spokane, Washington, 99202, United States

Location

Research Site

Toronto, Ontario, Canada

Location

Montreal Neurological Institute and Hospital

Montreal, Quebec, QC H3A 2B4, Canada

Location

Sourasky Medical Center

Tel Aviv, 64239, Israel

Location

Neuro-SysMed Center

Bergen, 5053, Norway

Location

St. Olav University Hospital

Trondheim, 7030, Norway

Location

Laboratorios de Investigación Biocruces 3., Hospital de Cruces

Barakaldo, Bizkaia, 48903, Spain

Location

Hospital General de Catalunya

Barcelona, Vizcaya, 08195, Spain

Location

Hospital Clinic de Barcelona

Barcelona, 08036, Spain

Location

Hospital General Universitario Gregorio Marañón

Madrid, 28007, Spain

Location

Research Site

Seville, Spain

Location

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

London, WC1N 3BG, United Kingdom

Location

MeSH Terms

Conditions

Parkinson Disease

Interventions

BIIB094

Condition Hierarchy (Ancestors)

Parkinsonian DisordersBasal Ganglia DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesMovement DisordersSynucleinopathiesNeurodegenerative Diseases

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
TRIPLE
Who Masked
PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 4, 2019

First Posted

June 6, 2019

Study Start

August 12, 2019

Primary Completion

August 12, 2024

Study Completion

August 12, 2024

Last Updated

September 23, 2025

Record last verified: 2025-09

Data Sharing

IPD Sharing
Will not share

Locations

US(7)IL(1)NO(2)ES(5)GB(1)CA(2)