Research Into the Safety of a New Agent (VT-5006) in People With and Without Parkinson's Disease

A Phase I, Randomized, Single Ascending Dose, Multiple Ascending Dose, Double-Blind Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of AX-5006 (Aka VT-5006) in Healthy Participants and Participants With Parkinson's Disease.

This is a first-in-human (FIH) study of orally administered VT-5006 (also known as AX-5006) in healthy adult volunteers (HVs) and adult participants with Parkinson's disease (PD). The goal of this clinical trial is to learn if VT-5006 is safe and tolerable in healthy volunteers and in participants with PD. It has three Parts (A, B, and C).

Part A: Healthy volunteers aged 18-54 will attend a screening visit, take a single dose of VT-5006 or matching placebo after an overnight fast, stay in the clinic for three nights, and complete a follow-up visit. One group of participants in Part A will be asked to return to the clinic after approximately two weeks, take a single dose of VT-5006 or matching placebo after consuming a high-fat meal and stay in the clinic for another three nights.

Part B: Healthy volunteers aged 18-54 will attend a screening visit, take one dose of VT-5006 or matching placebo each day for seven days after fasting overnight, stay in the clinic for 10 nights, and complete a follow up visit.

Part C: Participants with PD aged 40-80 will attend a screening visit, take one dose of VT-5006 (high dose), VT-5006 (low dose), or matching placebo each day for 28 days, complete two overnight stays in the clinic, attend three clinic visits, one phone call and a follow up visit.

Study Overview

• Status: Recruiting
• Conditions: Parkinson's Disease (PD); Healthy Volunteers (HV)
• Intervention / Treatment: Drug: VT-5006; Drug: Placebo

Detailed Description

Part A will consist of five single ascending dose (SAD) cohorts, with 8 participants in each cohort. Participants will be randomized to receive either VT-5006 or placebo in a 6:2 ratio. One cohort of 8 participants will complete an additional clinic stay for the purpose of evaluating food effect (FE).

Part B will consist of two multiple ascending dose (MAD) cohorts, with 10 participants in each cohort. Participants will be randomized to receive either VT-5006 or placebo in an 8:2 ratio.

Part C will consist of a single cohort of approximately 24 (or up to 32) participants with PD, randomized to receive either VT-5006 (high dose), VT-5006 (low dose) or placebo over 28 days in a 9:9:6 ratio.

• Study Type: Interventional
• Enrollment (Estimated): 84
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: P.H.C. Kremer; Phone Number: +31 0715246400; Email: clintrials@chdr.nl

Study Locations

• Netherlands
  • Leiden, Netherlands
    • Recruiting
    • Center for Human Drug Research
    • Contact: P.H.C. Kremer; Phone Number: +31 0715246400; Email: clintrials@chdr.nl

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: Yes

Description

Parts A and B enroll healthy volunteers; only key entry criteria for Part C are described below.

Inclusion Criteria:

• Diagnosed with PD confirmed by a neurologist within a maximum of 10 years (based of year of diagnosis) prior to screening
• Has current or history of GI motility dysfunction or persistent constipation
• Score of < 3 on the Modified Hoehn and Yahr Scale
• Is able to swallow multiple and large capsules without assistance or difficulty, in the opinion of the investigator
• Participants should be on a stable regimen of any prescribed (except levodopa/carbidopa, levodopa/benserazide or anticholinergic agents) or over-the-counter medications or supplements for at least 60 days prior to enrolment in the study. Participants should not change the dosage or frequency of these medications or supplements while in the study. If changes to medications or supplements are contemplated during the study, the Investigator should be contacted prior to any change.
• Has suitable venous access for blood sampling

Exclusion Criteria:

• Has a known allergy or hypersensitivity to any component of the formulation of VT-5006 or matching placebo, or history of severe allergy or anaphylaxis to a drug, food, or other exposure
• Participants taking levodopa/carbidopa or levodopa/benserazide must remain on a stable dose and regimen from at least 21 days prior to Day 1 visit to end of study (EOS) visit. Other treatments for PD symptoms may be allowed at the discretion of the medical monitor
• Has any clinically significant arrhythmia(s) on ECG; specifically, the participant's corrected QT interval (QTcf) (Fridericia's correction) is >450 ms for males or >470 ms for females
• Has clinically significant abnormalities, as judged by the investigator, in laboratory test results (including hepatic and renal panels, complete blood count, chemistry panel and urinalysis). In the case of uncertain or questionable results, tests performed during screening may be repeated before randomization to confirm eligibility or judged to be clinically irrelevant for healthy participants
• Has any of the following test results: a serum total bilirubin value >1.5 x upper limit of normal (ULN); a serum alanine aminotransferase (ALT) or aspartate aminotransferase (AST) value 2x ULN. As an exception, participants that present with elevated bilirubin in the absence of elevations in ALT or AST that fits the pattern of Gilbert's syndrome may be enrolled after discussion with the medical monitor if their conjugated bilirubin is not or slightly elevated and the level is considered to be not clinically significant.
• Any history of lumbar surgery for any reason (e.g. herniated disc) that in the opinion of the Investigator would interfere with or pose risks to a lumbar puncture procedure
• Other contraindications to having a lumbar puncture

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

7

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Part A SAD Active; Single Ascending Doses of VT-5006	Drug: VT-5006; Oral Capsules, Active; Other Names: AX-5006
Placebo Comparator: Part A SAD Placebo; Matched Placebo	Drug: Placebo; Oral Capsules, Matched Placebo
Experimental: Part B MAD Active; Multiple ascending doses of VT-5006	Drug: VT-5006; Oral Capsules, Active; Other Names: AX-5006
Placebo Comparator: Part B MAD Placebo; Matched Placebo	Drug: Placebo; Oral Capsules, Matched Placebo
Experimental: Part C Active Low Dose; Low dose VT-5006	Drug: VT-5006; Oral Capsules, Active; Other Names: AX-5006
Experimental: Part C Active High Dose; High dose VT-5006	Drug: VT-5006; Oral Capsules, Active; Other Names: AX-5006
Placebo Comparator: Part C Placebo; Matched placebo	Drug: Placebo; Oral Capsules, Matched Placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants with treatment-related adverse events (AEs)	Incidence of treatment-emergent AEs (e.g. clinically significant electrocardiogram, vital signs and physical examination abnormalities; clinically significant changes on the Columbia-Suicide Severity Rating Scale [C-SSRS]).	Part A: Up to 8 days; Part B: Up to 15 days; Part C: Up to 35 days;

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Plasma pharmacokinetic (PK) parameter: Cmax	Maximum peak plasma concentration (Cmax)	Part A: predose, and up to 72 hours postdose; Part B: predose, and up to 37 hours postdose on Days 1 and 7; Part C: predose, and up to 10 hours postdose on Days 1 and 28
Plasma PK parameter: Tmax	Time to Maximum Observed Concentration (Tmax)	Part A: predose, and up to 72 hours postdose; Part B: predose, and up to 37 hours postdose on Days 1 and 7; Part C: predose, and up to 10 hours postdose on Days 1 and 28
Plasma PK parameter: AUClast	Area under the plasma concentration-time curve from time zero to the time of last quantifiable concentration (AUClast)	Part A: predose, and up to 72 hours postdose; Part B: predose, and up to 37 hours postdose on Days 1 and 7; Part C: predose, and up to 10 hours postdose on Days 1 and 28
Plasma PK parameters: AUCinf	Area under the concentration-time curve extrapolated to infinity (AUCinf)	Part A: predose, and up to 72 hours postdose; Part B: predose, and up to 37 hours postdose on Days 1 and 7; Part C: predose, and up to 10 hours postdose on Days 1 and 28
Plasma PK parameter: t1/2	Terminal-phase elimination half-life (t1/2)	Part A: predose, and up to 72 hours postdose; Part B: predose, and up to 37 hours postdose on Days 1 and 7; Part C: predose, and up to 10 hours postdose on Days 1 and 28
Food effect on plasma PK: Cmax	Cmax following a high fat meal	Part A: predose and up to 72 hours postdose
Food effect on plasma PK: Tmax	Tmax following a high fat meal	Part A: predose and up to 72 hours postdose
Food effect on plasma PK: AUC	AUC following a high fat meal	Part A: predose and up to 72 hours postdose
Urine PK parameter: CLr	Renal clearance rate (CLr)	Part A: Up to 72 hours postdose; Part B: Up to 24 hours postdose on Days 1 and 7; Part C: Up to 8 hours postdose on Days 1 and 28
Plasma PK parameter: Apparent CL/F	Apparent clearance (CL/F)	Part A: predose, and up to 72 hours postdose; Part B: predose, and up to 37 hours postdose on Days 1 and 7; Part C: predose, and up to 10 hours postdose on Days 1 and 28
Plasma PK parameter: Apparent Vd/F	Apparent volume of distribution (Vd/F)	Part A: predose, and up to 72 hours postdose; Part B: predose, and up to 37 hours postdose on Days 1 and 7; Part C: predose, and up to 10 hours postdose on Days 1 and 28
Urine PK parameter: Cumulative amount excreted (urine)	Cumulative amount excreted in urine	Part A: Up to 72 hours postdose; Part B: Up to 24 hours postdose on Days 1 and 7; Part C: Up to 8 hours postdose on Days 1 and 28
Urine PK parameter: %Cumulative amount excreted	Percent cumulative amount excreted in urine	Part A: Up to 72 hours postdose; Part B: Up to 24 hours postdose on Days 1 and 7; Part C: Up to 8 hours postdose on Days 1 and 28
Feces PK parameter: Observed concentration	Observed concentration in fecal samples (ng/mL)	Part A: 0-72 hours postdose; Part B: 0-12 hours and 12-24 hours postdose on Days 1 and 7, and anytime postdose on Day 4; Part C: Anytime postdose on Days 8, 15, 22 and 28

Collaborators and Investigators

• Sponsor: Vertero Therapeutics

Study record dates

Study Major Dates

• Study Start (Actual): October 30, 2025
• Primary Completion (Estimated): September 28, 2026
• Study Completion (Estimated): September 28, 2026

Study Registration Dates

• First Submitted: November 17, 2025
• First Submitted That Met QC Criteria: December 15, 2025
• First Posted (Actual): December 30, 2025

Study Record Updates

• Last Update Posted (Actual): March 20, 2026
• Last Update Submitted That Met QC Criteria: March 17, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Synucleinopathies; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurodegenerative Diseases; Movement Disorders; Parkinsonian Disorders; Basal Ganglia Diseases; Parkinson Disease
• Other Study ID Numbers: VT-5006-101 (Vertero Therapeutics); AXL-5006-101 (Other Identifier: Vertero Therapeutics); 2025-523254-14-00 (Ctis)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No