Clinical trial NCT06556173: Phase 2a Study of VTX3232 in Parkinson's Disease
Official title: A Phase 2a, Single Site, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VTX3232 in Participants With Early-Stage Parkinson's Disease
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: Idiopathic Parkinson Disease
Keywords: VTX3232
Idiopathic Parkinson's Disease
NLRP3 Inhibitor
Zomagen
Lead sponsor: Zomagen Biosciences Ltd.
Sponsor class: INDUSTRY
First posted: 2024-08-15
Enrollment: 11
Sex: ALL
Minimum age (years): 40
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is a study to understand if taking VTX3232 is safe in participants diagnosed with early stage idiopathic Parkinson's Disease (PD). Approximately 10 patients will take VTX3232 Dose A.

The study consists of a 30-day Screening Period (to see if a participant qualifies for the study), a 7-day Pre-Baseline Period, a 28-day Open Label Treatment period (a participant receives active Dose A), and a 14-day Follow-Up Period.


Eligibility criteria: Inclusion Criteria:

* Participants must be ≥ 40 years up to 80 years of age, inclusive, at the time of signing the informed consent, with BMI \> 18.5 and \< 32.0 kg/m2 and body weight ≥ 50.0 kg for males and ≥ 45.0 kg for females.
* Diagnosis of idiopathic Parkinson's Disease between 0 and 60 months prior to screening.
* Score of 2 or less on Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Part IV at screening.
* Have not received prior treatment with deep brain stimulation (DBS).
* If receiving treatment with symptomatic PD therapies, treatment must be stable. Note: The Medical Monitor should be contacted with any questions regarding concomitant therapies.
* A female participant is eligible if they are of nonchildbearing potential
* A male participant sexually active with a woman of child bearing potential is eligible if they agree to use contraception/barrier and refrain from donating sperm during the study and for at least 90 days after the last dose

Exclusion Criteria:

* Diagnosis of a Parkinsonian syndrome other than idiopathic Parkinson's Disease.
* A diagnosis of a significant central nervous system (CNS) disease other than Parkinson's disease; history of repeated head injury or traumatic brain injury; history of epilepsy or seizure disorder other than febrile seizures as a child.
* History of brain surgery.

Primary outcomes:
measure: Adverse Event (AE) / Serious Adverse Event (SAE) Incidence Rate and Severity through study completion
timeFrame: Day 1 of treatment period through study completion, up to 6 weeks
Secondary outcomes:
measure: Pharmacokinetics (PK) of VTX3232 in plasma and Cerebrospinal Fluid (CSF) at Day 28
timeFrame: Day 1 of treatment period to Day 28 of treatment period
Other outcomes:
Locations:
city: New Haven
country: United States
facility: Local Site #840001
state: Connecticut
Study officials:
affiliation: Zomagen Biosciences Ltd.
name: Snehal Naik, PhD
role: STUDY_DIRECTOR
References: