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NCT03976349CompletedPhase 1

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

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

Source: ClinicalTrials.gov · Last updated 23 Sept 2025

Brief summary (registry, in English)

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.

Status (registry)
Completed
Phase (registry)
Phase 1
Study type
Interventional
Enrollment
62 (actual)
First submitted
4 Jun 2019
Start date
12 Aug 2019
Primary completion date
12 Aug 2024
Lead sponsor
Ionis Pharmaceuticals, Inc.
NCT number
NCT03976349
EudraCT number
2018-002995-42

What you commit to

  • Study type: Interventional · Phase 1
  • Participants enrolled: 62
  • From 12 Aug 2019 to the primary completion date, 12 Aug 2024
  • 18 sites in 6 countries
  • Visits and tests: ask the trial team, the registry does not list them

Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.

Who cannot take part

Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.

Other illness

  • 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.
  • History or positive test result at Screening for hepatitis C virus antibody.
    • NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Lab values

  • 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.

Other

  • History or positive test result at Screening for human immunodeficiency virus.

Who the study is looking for

  • 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.

Age in the registry: 35 years to 80 years · Sex: All · Does not accept healthy volunteers

Full criteria on ClinicalTrials.gov

Sites (18)

Sites in registry order. Pick your city on the disease page to sort them by distance.

SiteCityCountrySite status
Montreal Neurological Institute and Hospital
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MontrealCanadaUnknown status
-
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TorontoCanadaUnknown status
Laboratorios de Investigación Biocruces 3., Hospital de Cruces
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BarakaldoSpainUnknown status
Hospital General de Catalunya
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BarcelonaSpainUnknown status
Hospital Clinic de Barcelona
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BarcelonaSpainUnknown status
Hospital General Universitario Gregorio Marañón
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MadridSpainUnknown status
-
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SevilleSpainUnknown status
Queen Square (Neurology) CRF Site Institute of Neurology & the National Hospital for Neurology and Neurosurgery UCLH
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LondonUnited KingdomUnknown status
Sourasky Medical Center
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Tel AvivIsraelUnknown status
Neuro-SysMed Center
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BergenNorwayUnknown status
St. Olav University Hospital
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TrondheimNorwayUnknown status
Northwestern University PD and Movement Disorders Center
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ChicagoUnited StatesUnknown status
The Cleveland Clinic Foundation
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ClevelandUnited StatesUnknown status
QUEST Research Institute
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Farmington HillsUnited StatesUnknown status
Alliance for Multispecialty Research
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KnoxvilleUnited StatesUnknown status
University of Pennsylvania
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PhiladelphiaUnited StatesUnknown status
Oregon Health and Science University
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PortlandUnited StatesUnknown status
Inland Northwest Research
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SpokaneUnited StatesUnknown status

Related publications

This page is information, not medical advice. Discuss any trial with your doctor.