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

Parkinson's Disease Clinical Trial

— REASON  

Official 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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03976349
• Other study ID #: 254PD101
• Secondary ID: 2018-002995-42
• Status: Active, not recruiting
• Phase: Phase 1
• Start date: August 12, 2019
• Est. completion date: December 26, 2024

Study information

• Verified date: March 2024
• Source: Biogen
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 81
• Est. completion date: December 26, 2024
• Est. primary completion date: December 26, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 35 Years to 80 Years

Eligibility

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.

Related Conditions & MeSH terms

• Parkinson Disease
• Parkinson's Disease

Intervention

Drug:
• BIIB094
Administered as specified in the treatment arm.
• Placebo
Administered as specified in the treatment arm.

Locations

Country	Name	City	State
Canada	Montreal Neurological Institute and Hospital	Montreal	Quebec
Israel	Sourasky Medical Center	Tel-Aviv
Norway	Neuro-SysMed Center	Bergen
Spain	Laboratorios de Investigación Biocruces 3., Hospital de Cruces	Barakaldo	Bizkaia
Spain	Hospital Clinic de Barcelona	Barcelona
Spain	Hospital General de Catalunya	Barcelona	Vizcaya
Spain	Hospital General Universitario Gregorio Marañón	Madrid
Spain	Research Site	Sevilla
United Kingdom	Queen Square (Neurology) CRF Site Institute of Neurology & the National Hospital for Neurology and Neurosurgery UCLH	London
United States	Northwestern University PD and Movement Disorders Center	Chicago	Illinois
United States	The Cleveland Clinic Foundation	Cleveland	Ohio
United States	Quest Research Institute	Farmington Hills	Michigan
United States	Alliance for Multispecialty Research	Knoxville	Tennessee
United States	University of Pennsylvania	Philadelphia	Pennsylvania
United States	Oregon Health and Science University	Portland	Oregon
United States	Inland Northwest Research	Spokane	Washington

Sponsors (2)

Lead Sponsor	Collaborator
Biogen	Ionis Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  Canada,  Israel,  Norway,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)	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.	Part A: Screening (Day -42) up to Day 85, Part B: Screening (Day -77) up to Day 253
Secondary	Serum Concentrations of BIIB094		Part A: pre-dose through Day 57, Part B: pre-dose through Day 169
Secondary	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
Secondary	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
Secondary	Maximum Concentration (Cmax) of BIIB094		Part A: pre-dose through Day 57, Part B: pre-dose through Day 169
Secondary	Time to Reach Maximum Concentration (Tmax) of BIIB094		Part A: pre-dose through Day 57, Part B: pre-dose through Day 169
Secondary	Terminal Elimination Half-Life (t1/2) of BIIB094		Part A: pre-dose through Day 57, Part B: pre-dose through Day 169

External Links

•   Click here to learn more about this trial, visit our study website.