Skip to main content
OpenTrials
Completed

NCT Number: NCT03976349

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

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.

Completed

Looking for future studies?

Notify Me

Key information

Age range

35 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Research Site, Toronto, Ontario, Canada

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Key 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.

Key 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.

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Treatment and study plan

BIIB094

Drug

Administered as specified in the treatment arm.

Placebo

Drug

Administered as specified in the treatment arm.

Primary outcomes

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

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

    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.

Secondary outcomes

  1. Serum Concentrations of BIIB094

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

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

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

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

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

  4. Maximum Concentration (Cmax) of BIIB094

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

  5. Time to Reach Maximum Concentration (Tmax) of BIIB094

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

  6. Terminal Elimination Half-Life (t1/2) of BIIB094

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

Sponsors and collaborators

Lead sponsor

Ionis Pharmaceuticals, Inc.

Industry

Registry information

Official study title

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

Acronym: REASON

Important dates

Study start
2019
Primary completion
2024
Study completion
2024
First posted
Jun 6, 2019
Registry last updated
Sep 23, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.