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)

Denali Therapeutics Inc.27 sites in 4 countries50 target enrollmentOctober 24, 2024

ConditionsParkinson Disease

InterventionsBIIB122 225 mg BIIB122-Matching Placebo

DrugsBIIB122 225 mg

Overview

Phase: Phase 2
Intervention: BIIB122 225 mg
Conditions: Parkinson Disease
Sponsor: Denali Therapeutics Inc.
Enrollment: 50
Locations: 27
Primary Endpoint: Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) with BIIB122 compared with placebo over the 12-week double-blind period
Status: Recruiting
Last Updated: 2 months ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

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.

Registry

clinicaltrials.gov

Start Date

October 24, 2024

End Date

February 28, 2028

Last Updated

2 months ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Denali Therapeutics Inc.

Responsible Party

Sponsor

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

Arms & Interventions

BIIB122 225 mg

Oral 225 mg dose, once daily (QD)

Intervention: BIIB122 225 mg

BIIB122 Matching Placebo

Oral BIIB122 matching placebo, once daily (QD)

Intervention: BIIB122-Matching Placebo

Outcomes

Primary Outcomes

Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) with BIIB122 compared with placebo over the 12-week double-blind period

Time Frame: 12 weeks

Secondary Outcomes

Change from baseline in whole-blood pS935 LRRK2 with BIIB122 compared with placebo at Week 12(12 weeks)
Change from baseline in urine BMP with BIIB122 compared with placebo at Week 12(12 weeks)