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.
- Diseases
- Parkinson disease
- 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
Sites (18)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Montreal Neurological Institute and Hospital Coordinator of this site? Confirm its status | Montreal | Canada | Unknown status |
| - Coordinator of this site? Confirm its status | Toronto | Canada | Unknown status |
| Laboratorios de Investigación Biocruces 3., Hospital de Cruces Coordinator of this site? Confirm its status | Barakaldo | Spain | Unknown status |
| Hospital General de Catalunya Coordinator of this site? Confirm its status | Barcelona | Spain | Unknown status |
| Hospital Clinic de Barcelona Coordinator of this site? Confirm its status | Barcelona | Spain | Unknown status |
| Hospital General Universitario Gregorio Marañón Coordinator of this site? Confirm its status | Madrid | Spain | Unknown status |
| - Coordinator of this site? Confirm its status | Seville | Spain | Unknown status |
| Queen Square (Neurology) CRF Site Institute of Neurology & the National Hospital for Neurology and Neurosurgery UCLH Coordinator of this site? Confirm its status | London | United Kingdom | Unknown status |
| Sourasky Medical Center Coordinator of this site? Confirm its status | Tel Aviv | Israel | Unknown status |
| Neuro-SysMed Center Coordinator of this site? Confirm its status | Bergen | Norway | Unknown status |
| St. Olav University Hospital Coordinator of this site? Confirm its status | Trondheim | Norway | Unknown status |
| Northwestern University PD and Movement Disorders Center Coordinator of this site? Confirm its status | Chicago | United States | Unknown status |
| The Cleveland Clinic Foundation Coordinator of this site? Confirm its status | Cleveland | United States | Unknown status |
| QUEST Research Institute Coordinator of this site? Confirm its status | Farmington Hills | United States | Unknown status |
| Alliance for Multispecialty Research Coordinator of this site? Confirm its status | Knoxville | United States | Unknown status |
| University of Pennsylvania Coordinator of this site? Confirm its status | Philadelphia | United States | Unknown status |
| Oregon Health and Science University Coordinator of this site? Confirm its status | Portland | United States | Unknown status |
| Inland Northwest Research Coordinator of this site? Confirm its status | Spokane | United States | Unknown status |
Related publications
- LRRK2-targeting antisense oligonucleotide in Parkinson's disease: a phase 1 randomized controlled trial.Nat Med · 2026 · PMID 41876647
This page is information, not medical advice. Discuss any trial with your doctor.