Clinical trial NCT06602193: Safety and Pharmacodynamic Effects of BIIB122 in Participants With LRRK2-Associated Parkinson's Disease (LRRK2-PD)
Official title: A Phase 2a, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Pharmacodynamic Effects of BIIB122 in Participants With LRRK2-Associated Parkinson's Disease (LRRK2-PD)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Parkinson Disease
Keywords: LRRK2
Movement Disorders
Lead sponsor: Denali Therapeutics Inc.
Sponsor class: INDUSTRY
Collaborators: name: Biogen
First posted: 2024-09-19
Enrollment: 50
Sex: ALL
Minimum age (years): 30
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This Phase 2a, multicenter, randomized, 12-week double-blind, placebo-controlled, parallel-group study, followed by an OLE, is designed to evaluate the safety, tolerability, and pharmacodynamic effects of BIIB122 in participants with LRRK2-PD. LRRK2-PD is defined as Parkinson's Disease (PD) in individuals who are heterozygous or homozygous carriers of a pathogenic LRRK2 variant that increases LRRK2 kinase activity.


Eligibility criteria: Inclusion Criteria:

* For heterozygous pathogenic LRRK2 mutation carriers: ≥ 30 to ≤ 80 years
* For homozygous pathogenic LRRK2 mutation carriers: ≥ 30 years
* Have screening genetic test results verifying the presence of a pathogenic LRRK2 variant.
* Have a clinical diagnosis of PD meeting the Movement Disorder Society Clinical Diagnostic Criteria.

Exclusion Criteria:

* Have a history of any clinically significant neurological disorder other than PD, including, but not limited to, stroke and dementia, in the opinion of the investigator, within 5 years of the screening visit.
* Have clinical evidence of atypical parkinsonism (eg, multiple-system atrophy or progressive supranuclear palsy) or evidence of drug-induced parkinsonism.
* Have previously participated or are currently participating in the BIIB122 LUMA study (Study 283PD201).
* Have previously participated or are currently participating in a gene therapy study for PD.
* Have a history of brain surgical intervention for PD (eg, deep-brain stimulation, pallidotomy).
* Have any physical condition that may confound the motor assessment (MDS-UPDRS) over time (eg, severe arthritis, severe dyskinesias, traumatic injuries with permanent physical disability).
* Abnormal vitals including Blood Pressure, Heart Rate, or Body Temperature
* Have abnormal PFT results at screening

Note: Other protocol defined Inclusion/Exclusion criteria may apply

Primary outcomes:
measure: Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) with BIIB122 compared with placebo over the 12-week double-blind period
timeFrame: 12 weeks
Secondary outcomes:
measure: Change from baseline in whole-blood pS935 LRRK2 with BIIB122 compared with placebo at Week 12
timeFrame: 12 weeks
measure: Change from baseline in urine BMP with BIIB122 compared with placebo at Week 12
timeFrame: 12 weeks
Other outcomes:
measure: Incidence of TEAEs and SAEs during the OLE period
timeFrame: 2.5 years
Locations:
city: San Francisco
country: United States
facility: University of California San Francisco
state: California
city: Boca Raton
country: United States
facility: Parkinson's Disease and Movement Disorders Center
state: Florida
city: Boston
country: United States
facility: Beth Israel Deaconess Medical Center
state: Massachusetts
city: New York
country: United States
facility: Ichan School of Medicine at Mount Sinai/Beth Israel Downtown-Movement Disorder Center
state: New York
city: Kirkland
country: United States
facility: Evergreen Health Laboratory
state: Washington
city: Spokane
country: United States
facility: Inland Northwest Research
state: Washington
city: Dresden
country: Germany
facility: Technische Universität Dresden
city: Lübeck
country: Germany
facility: University of Lübeck
city: Tübingen
country: Germany
facility: University Hospital Tübingen
city: Barcelona
country: Spain
facility: Hospital Universitari General de Catalunya
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d'Hebron
city: Donostia
country: Spain
facility: Hospital Universitario Donostia
city: Madrid
country: Spain
facility: Universitary Hospital La Princesa
city: Santander
country: Spain
facility: IDIVAL/University Hospital Marques de Valdecilla
city: Sevilla
country: Spain
facility: Hospital Universitario Virgen del Rocio
Study officials:
affiliation: Denali Therapeutics
name: Danna Jennings, MD
role: STUDY_DIRECTOR
References: