biogen/studies/attachments/NCT03976349-LLS.pdf

NCT03976349-LLS

biogen/studies/attachments/NCT03976349-LLS_1.pdf

biogen/studies/attachments/NCT03976349-LLS_2.pdf

biogen/studies/attachments/NCT03976349-LLS_3.pdf

biogen/studies/attachments/NCT03976349-LLS_4.pdf

biogen/studies/attachments/NCT03976349-LLS_5.pdf

biogen/studies/attachments/NCT03976349-Synopsis.pdf

NCT03976349-Synopsis

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.

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

Parkinson's Disease

Neurological Disorders

Placebo

BIIB094

A Phase 1 Single- and Multiple-Ascending-Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB094 Administered Intrathecally to Adults With Parkinson's Disease

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.

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

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

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

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

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

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

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

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

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.

Northwestern University PD and Movement Disorders Center

Quest Research Institute

The Cleveland Clinic Foundation

Oregon Health and Science University

University of Pennsylvania

Alliance for Multispecialty Research

Inland Northwest Research

Research Site

Montreal Neurological Institute and Hospital

Sourasky Medical Center

Neuro-SysMed Center

St. Olav University Hospital

Laboratorios de Investigación Biocruces 3., Hospital de Cruces

Hospital General de Catalunya

Hospital Clinic de Barcelona

Hospital General Universitario Gregorio Marañón

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

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.

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

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

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

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

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