A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB094 in Adults With Parkinson's Disease
- Registration Number
- NCT03976349
- Lead Sponsor
- Biogen
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 62
- 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
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Part A (SAD): BIIB094 Dose 5 BIIB094 Participants will receive a single IT injection of BIIB094 during Part A (SAD). Part A (SAD): BIIB094 Dose 6 BIIB094 Participants will receive a single IT injection of BIIB094 during Part A (SAD). Part A (SAD): BIIB094 Dose 3 BIIB094 Participants will receive a single IT injection of BIIB094 during Part A (SAD). Part A (SAD): BIIB094 Dose 4 BIIB094 Participants will receive a single IT injection of BIIB094 during Part A (SAD). Part B (MAD): Matching Placebo Placebo Participants will receive matching placebo on multiple days during Part B (MAD). Part A (SAD): BIIB094 Dose 2 BIIB094 Participants will receive a single IT injection of BIIB094 during Part A (SAD). Part B (MAD): BIIB094 Dose 1 BIIB094 Participants will receive a single IT injection of BIIB094 on multiple days during Part B \[Multiple Ascending Dose (MAD)\]. Part B (MAD): BIIB094 (LRRK2) Dose 2 BIIB094 Participants (LRRK2) will receive a single IT injection of BIIB094 on multiple days during Part B (MAD). Part A (SAD): Matching Placebo Placebo Participants will receive matching placebo during Part A \[Single Ascending Dose (SAD)\]. Part B (MAD): BIIB094 (Non LRRK2) Dose 2 BIIB094 Participants \[Non leucine-rich repeat kinase 2 (Non LRRK2)\] will receive a single IT injection of BIIB094 on multiple days during Part B (MAD). Part B (MAD): BIIB094 (Non LRRK2) Dose 3 BIIB094 Participants (Non LRRK2) will receive a single IT injection of BIIB094 on multiple days during Part B (MAD). Part B (MAD): BIIB094 (LRRK2) Dose 3 BIIB094 Participants (LRRK2) will receive a single IT injection of BIIB094 on multiple days during Part B (MAD). Part A (SAD): BIIB094 Dose 1 BIIB094 Participants will receive a single IT injection of BIIB094 during Part A \[Single Ascending Dose (SAD)\].
- Primary Outcome Measures
Name Time Method Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) 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 Outcome Measures
Name Time Method 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 Terminal Elimination Half-Life (t1/2) of BIIB094 Part A: pre-dose through Day 57, Part B: pre-dose through Day 169 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 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
Trial Locations
- Locations (17)
Laboratorios de Investigación Biocruces 3., Hospital de Cruces
🇪🇸Barakaldo, Bizkaia, Spain
Northwestern University PD and Movement Disorders Center
🇺🇸Chicago, Illinois, United States
The Cleveland Clinic Foundation
🇺🇸Cleveland, Ohio, United States
University of Pennsylvania
🇺🇸Philadelphia, Pennsylvania, United States
Research Site
🇪🇸Sevilla, Spain
Inland Northwest Research
🇺🇸Spokane, Washington, United States
Hospital General de Catalunya
🇪🇸Barcelona, Vizcaya, Spain
Queen Square (Neurology) CRF Site Institute of Neurology & the National Hospital for Neurology and Neurosurgery UCLH
🇬🇧London, United Kingdom
Hospital General Universitario Gregorio Marañón
🇪🇸Madrid, Spain
Hospital Clinic de Barcelona
🇪🇸Barcelona, Spain
Quest Research Institute
🇺🇸Farmington Hills, Michigan, United States
Oregon Health and Science University
🇺🇸Portland, Oregon, United States
Montreal Neurological Institute and Hospital
🇨🇦Montreal, Quebec, Canada
Sourasky Medical Center
🇮🇱Tel-Aviv, Israel
St. Olav University Hospital
🇳🇴Trondheim, Norway
Neuro-SysMed Center
🇳🇴Bergen, Norway
Alliance for Multispecialty Research
🇺🇸Knoxville, Tennessee, United States