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A Study to Investigate The Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of RO7486967 in Participants With Early Idiopathic Parkinson's Disease
- Neurodegenerative Disorder
- Parkinson's Disease (PD)
Please note that the recruitment status of the trial at your site may differ from the overall study status because some study sites may recruit earlier than others.
Trial Status:
Completed
This trial runs in
Cities
- Amsterdam
- Birmingham
- Denver
- Exeter
- Farmington Hills
- London
- Los Angeles
- New York
- Newcastle upon Tyne
- Nijmegen
- Orlando
- Philadelphia
- Tulsa
- Washington
- Zwolle
Trial Identifier:
NCT05924243 BP43176
Trial 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).
Hoffmann-La Roche
Sponsor
Phase 1
Phase
NCT05924243, BP43176
Trial Identifier
All
Gender
≥40 Years & ≤ 85 Years
Age
No
Healthy Volunteers
Background and study aims:
Parkinson’s disease (PD) is a progressive and incurable disease that affects the nervous system. It is caused by the loss of brain cells that produce dopamine, a chemical that helps brain cells communicate and control movement. The main symptoms include decreased mobility, rigidity or stiffness of arms, legs, and trunk, and resting tremors (shaking that occurs at rest). Evidence suggests that people with PD may have increased inflammation (swelling) in the brain. A protein called NLRP3 is thought to play a key role in this inflammatory process. RO748967 is a new drug that has been developed to slow the progression of disease in people with early PD. RO748967 acts by blocking NLRP3, which might lower the inflammation in the brain. Health authorities have not yet approved RO748967 for the treatment of PD or any other disease. The main purpose of this study is:
-To determine how safe and tolerable RO748967 is when compared to a placebo (medicine without an active ingredient)
-To determine how RO748967 is absorbed, distributed and, eventually eliminated from the body
-To assess the effect of RO748967 on the inflammation in the brain using certain scans called the TSO-PET scans
Who can participate?
People aged between 50 to 85 years with PD.
What does the study involve?
Participants may be asked to be in the study for approximately 100 days. This includes: -
Screening Period of up to 60 days where tests will be done to check if the participants are eligible to take part in the study. Participants may have to visit the clinic approximately 3 times during the Screening Period.
The baseline period of up to 3 days before the start of the study wherein participants will be contacted over the phone to collect details about other medicines and changes to their health and life. MRI and TSPO PET scans will also be done at this time.
Treatment Period of approximately 28 days where participants will have to report 5 times to the clinic and 2 times to a dedicated external PET center for tests, assessments, and procedures in addition to taking the study medication twice a day.
Follow-up Period where participants will have a check-up 14 days after the last study treatment administration.
Participants will be placed in one of the following treatment groups:
Group 1 will receive RO7486967, given as two pills in the morning and two pills in the evening every day for about 4 weeks.
Group 2 will receive a placebo, given as two pills in the morning and two pills in the evening every day for about 4 weeks
What are the possible benefits and risks of participating?
Participants will not receive any benefit from participating in this study, but the information that is learned may help people with PD in the future.
Participants may have side effects from the drugs or procedures used in this study that are mild to severe and even life-threatening in nature, and they can vary from person to person.
Trial 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).
Hoffmann-La Roche
Sponsor
Phase 1
Phase
NCT05924243, BP43176
Trial Identifier
RO7486967, Placebo
Treatments
Parkinson Disease
Condition
Official Title
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
Eligibility Criteria
All
Gender
≥40 Years & ≤ 85 Years
Age
No
Healthy Volunteers
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 Criteria
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
For the latest version of this information please go to www.forpatients.roche.com