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A Study to Investigate The Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of RO7486967 in Participants With Early Idiopathic Parkinson's Disease.

A Phase 1b, Adaptive, Multi-Center, Randomized, Double Blind, Placebo-Controlled, Parallel Design Study to Investigate The Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of RO7486967 in Participants With Early Idiopathic Parkinson's Disease

Status
Completed
Phases
Phase 1
Study type
Interventional
Source
ClinicalTrials.gov
Registry ID
NCT05924243
Enrollment
60
Registered
2023-06-29
Start date
2022-09-22
Completion date
2024-07-18
Last updated
2024-09-19

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Parkinson Disease

Brief summary

This is a multi-center, randomized, double blind, adaptive, parallel-group, placebo controlled Phase 1b study to evaluate the safety, tolerability, pharmacokinetic (PK) and pharmacodynamics of RO7486967 in participants with idiopathic PD at the early stage of the disease (modified H&Y stage ≤2.5) who are either treatment-naïve or on stable treatment with symptomatic therapy (levodopa and/or pramipexole, ropinirole, rotigotine).

Interventions

DRUGRO7486967

For up to approximately 28 days

DRUGPlacebo

For up to approximately 28 days

Sponsors

Hoffmann-La Roche
Lead SponsorINDUSTRY

Study design

Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)

Eligibility

Sex/Gender
ALL
Age
40 Years to 85 Years
Healthy volunteers
No

Inclusion criteria

Inclusion Key Criteria: * Male or post-menopausal female * Diagnosis of clinically probable idiopathic PD based on MDS criteria with bradykinesia plus one of the other cardinal signs of PD (resting tremor, rigidity) * A time from diagnosis of PD of at least 3 to maximum 60 months (5 years) at screening * Modified H&Y Stage ≤2.5 (in ON state) * Dopaminergic imaging consistent with dopamine transporter deficit * High-affinity binder or mixed-affinity binder genotype for TSPO * Either treatment naïve or treatment with symptomatic PD therapy (levodopa and/or pramipexole, ropinirole, rotigotine) given for at least 90 days, with stable doses for at least 30 days prior to the first dose * No anticipated changes in PD therapy throughout the study duration * SARS-CoV-2 vaccination completed at least 60 days prior to the first dose. Exclusion Key Criteria: * Medical history indicating a Parkinsonian syndrome other than idiopathic PD * CNS or psychiatric disorders other than idiopathic PD (mild depression or anxiety arising in the context of PD is not exclusionary) * History of brain surgery for PD * Use of any of symptomatic drug for PD other than levodopa pramipexole, ropinirole, or rotigotine within 60 days prior to the first dose * Known carriers for mutations in the following genes: alpha-synuclein, LRRK2, GBA, PRKN, PINK1, or DJ1 * Unstable or clinically significant cardiovascular disease within the last year prior to screening * Uncontrolled hypertension * Use of oral anticoagulants, low-molecular-weight heparin, warfarin (Coumadin), acenocoumarol, and phenprocoumon is not allowed within 10 days before the first Lumbar Puncture and during the study (low dose aspirin is permitted as monotherapy) * Concomitant disease or unstable medical condition within 6 months of screening that could interfere with the study or treatment that might interfere with the conduct of the study, including but not limited to autoimmune disease, immunodeficiency diseases, any active infectious disease * History of immunodeficiency diseases * Presence of hepatitis B surface antigen (HBsAg) or positive for total hepatitis B core antibody (HBcAb), or positive hepatitis C (HCV) at screening * Vaccine(s) other than SARS-CoV2 vaccine within 28 days prior to the first dose, or plans to receive vaccines during the study or within 28 days of the last dose * History of chronic liver disease * Clinically significant abnormalities in laboratory test results at screening, including hepatic and renal panels, complete blood count, chemistry panel and urinalysis * Any previous administration of RO7486967 or other compound targeting NLRP3 * Enrollment in another investigational study * Use of any of other investigational therapy (other than protocol-mandated study treatment) within 90 days or 5 drug elimination half-lives (whichever is longer) prior to the first dose

Design outcomes

Primary

MeasureTime frame
Percentage of Participants with adverse events (AEs)Up to 45 Days
The change in Columbia-Suicide Severity Rating Scale (C-SSRS) Scores from baselineFrom Baseline to Up to 45 Days

Secondary

MeasureTime frame
Time to maximum concentration of RO7486967 in PlasmaDay 1, Day 15, and Day 28
Maximum concentration (Cmax) of RO7486967 in PlasmaDay 1, Day 15, and Day 28
Area under the curve (AUC) RO7486967 in PlasmaDay 1, Day 15, and Day 28
Change from baseline in parametric bindings of [18F]-DPA-714 in different brain areas at Day 25 PETFrom Baseline to Approximately Day 25

Countries

Netherlands, United Kingdom, United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026