Safety and Pharmacodynamic Effects of BIIB122 in Participants With LRRK2-Associated Parkinson's Disease (LRRK2-PD)

Phase 2

Recruiting

• Conditions: Parkinson Disease
• Interventions: Drug: BIIB122 225 mg; Other: BIIB122-Matching Placebo

• Registration Number: NCT06602193
• Lead Sponsor: Denali Therapeutics Inc.

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-09-16
• Study First Submitted QC: 2024-09-16
• Study First Posted: 2024-09-19
• Study Start: 2024-10-24
• Status Verified: 2024-12
• Last Update Submitted: 2024-12-17
• Last Update Posted: 2024-12-18
• Primary Completion: 2026-04-30
• Study Completion: 2028-02-28

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 50

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.

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
BIIB122 225 mg	BIIB122 225 mg	Oral 225 mg dose, once daily (QD)
BIIB122 Matching Placebo	BIIB122-Matching Placebo	Oral BIIB122 matching placebo, once daily (QD)

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (3)

Evergreen Health Laboratory; 🇺🇸 Kirkland, Washington, United States

Parkinson's Disease and Movement Disorders Center; 🇺🇸 Boca Raton, Florida, United States

Inland Northwest Research; 🇺🇸 Spokane, Washington, United States