VT7208 safety, dosing, and effects in healthy adults and people with multiple sclerosis

A Phase 1/2, Randomized, Double-Blind, Placebo-Controlled, Single and Multiple Dose Escalation Study in Healthy Participants and an Expansion Cohort in Adult Patients With Multiple Sclerosis to Characterize the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VT7208

This trial will try VT7208 to see if it's safe, how the body processes it, and its effects in healthy adults and people with multiple sclerosis.

Quick facts

• Phase: PHASE1; PHASE2
• Study type: Interventional
• Enrollment: 100 (estimated)
• Ages: 18 Years to 65 Years
• Sex: All
• Sponsor: Vidya Therapeutics Inc (industry)
• Locations: 1 site (Adelaide, South Australia)
• Trial ID: NCT07085507 on ClinicalTrials.gov

What this trial studies

This Phase 1/2 randomized, double-blind, placebo-controlled program uses single-ascending-dose (SAD) and multiple-ascending-dose (MAD) cohorts in healthy adults to define safety, tolerability, pharmacokinetics, and pharmacodynamics of VT7208. After SAD and MAD, a randomized open-label crossover compares VT7208 pharmacokinetics in fasted versus fed states. Part 3 enrolls adults with multiple sclerosis to receive VT7208 at doses chosen from Parts 1–2 with weekly clinic visits for dosing and monitoring. Procedures include lumbar punctures for CSF collection and standard safety and PK/PD measurements, with placebo control in Parts 1–2 and open-label dosing in Parts 2 and 3.

Who should consider this trial

Good fit: Healthy adults 18–65 without significant medical problems are eligible for Parts 1–2, and adults 18–60 with a confirmed MS diagnosis (2017 McDonald criteria), able to attend weekly visits and undergo lumbar punctures, are eligible for Part 3.

Not a fit: People with clinically significant comorbidities, recent illicit drug or heavy alcohol use, recent nicotine use, or any condition that makes lumbar puncture or study participation unsafe are unlikely to be eligible or to benefit.

Why it matters

Potential benefit: If successful, VT7208 could identify safe, brain-penetrant dosing that may become a new treatment option for people with MS.

How similar studies have performed: This is an early-phase PK/PD program; prior first-in-human CNS small-molecule studies have sometimes identified safe dosing and brain exposure but have not consistently predicted later clinical efficacy, so the approach remains exploratory.

Eligibility criteria

Inclusion Criteria:

Parts 1 and 2

* Age 18-65
* Must be in good health with no significant medical history
* Willing and able to attend all study visits and comply with study requirements, including lumbar puncture for CSF collection
* Able and willing to provide written informed consent

Part 3

* Age 18-60
* Must be in good health with no significant medical history
* MS diagnosis prior to Day 1 in accordance with 2017 McDonald criteria.
* Willing and able to attend all study visits and comply with study requirements, including lumbar puncture for CSF collection
* Able and willing to provide written informed consent

Exclusion Criteria:

* Evidence of clinically significant condition or disease
* Any physical or psychological condition that prohibits study completion
* Known history of illicit drug use or drug abuse, harmful alcohol use (at the -Investigator's discretion), alcoholism, and/or smoking or nicotine-containing product use within 7 days prior to the first dose of study agent
* History of severe allergic reactions or hypersensitivity
* Donation or loss of ≥ 1 unit of whole blood or plasma within 4 weeks prior to dosing

Where this trial is running

Adelaide, South Australia

• CMAX — Adelaide, South Australia, Australia (RECRUITING)

Study contacts

• Study coordinator: MIchele DeSciscio, MBSS

Conditions: Multiple Sclerosis, pharmacokinetics, healthy volunteers, pharmacodynamics, central nervous system