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Completed

NCT Number: NCT06226064

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VES001 in Healthy Participants

This is a Phase 1, randomized, placebo-controlled, double-blind study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single and multiple doses of VES001 in a two part followed by a multicenter, open-label Phase 1b study in asymptomatic GRN mutation carriers.

Part A will evaluate the safety, tolerability, PK, and PD of single doses of VES001 in healthy volunteers.

Part B will evaluate the safety, tolerability, PK, and PD of multiple doses of VES001 in healthy volunteers.

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Key information

Conditions

Dementia, Frontotemporal Brain Diseases Central Nervous System Diseases Dementia Frontotemporal Dementia Frontotemporal Lobar Degeneration Mental Disorders Metabolic Diseases Nervous System Diseases Neurocognitive Disorders Neurodegenerative Diseases Nutritional and Metabolic Diseases Proteostasis Deficiencies TDP-43 Proteinopathies

Age range

18 year–55 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Center for Human Drug Research

Leiden, 2333, Netherlands

About this study

Part A will include six cohorts, with eight participants per cohort. Participants in each cohort will be randomised in a 6:2 ratio (VES001 vs. placebo).

Part B will include three cohorts, with ten participants per cohort. Participants in each cohort will be randomised in a 8:2 ratio (VES001 vs. placebo).

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Part A & B:

  • Healthy men or women aged 18 to 55 years.
  • Body Mass Index between 18 and 32 kg/m2, with a minimum weight of 50 kg.
  • Effective contraception required during the study and for at least 90 days after their last dose.
  • Participants in group 3, where the food effect is being investigated, must be able to eat a high-fat meal within 30 minutes for breakfast.

Exclusion criteria

Part A & B:

  • Medical conditions or treatments that could interfere with the study.
  • History of any known neurologic disease, cognitive impairment, or a history of seizure, (significant) head trauma, or loss of consciousness.
  • History of active malignancy (active cancer cells or tumors) within the last 5 years.
  • Abnormal laboratory test results or infectious diseases (Hepatitis B, Hepatitis C, and/or HIV).
  • Recent medication or supplement use, unless allowed by the investigator.
  • Participation in other research studies involving study treatment or devices.
  • Positive tests for illegal drugs or alcohol at screening.
  • Heavy smoking or inability to abstain from smoking during the study.
  • Excessive consumption of caffeine (more than 8 cups per day).
  • History of severe allergic reactions to medication
  • Recent blood donation or significant blood loss.
  • Pregnancy, breastfeeding, or plans to become pregnant (for women).

Treatment and study plan

VES001

Drug

VES001 is an oral, blood brain barrier penetrating ligand of sortilin.

Placebo

Drug

A matching dosage form, indistinguishable from the active treatment will be used as the placebo treatment.

Primary outcomes

  1. Incidence, severity, and seriousness of treatment-emergent adverse events (TEAEs).

    Time frame: Part A: 21 weeks. Part B: 13 weeks.

  2. Incidence of clinically significant abnormalities in safety laboratory values.

    Time frame: Part A: 21 weeks. Part B: 13 weeks.

  3. Change from baseline in vital sign measurement: Pulse Rate (bpm).

    Time frame: Part A: 21 weeks. Part B: 13 weeks.

  4. Change from baseline in vital sign measurement: Systolic blood pressure (mmHg) and Diastolic blood pressure (mmHg).

    Time frame: Part A: 21 weeks. Part B: 13 weeks.

  5. Change from baseline in vital sign measurement: Electrocardiogram parameter Heart Rate (HR).

    Time frame: Part A: 21 weeks. Part B: 13 weeks.

  6. Change from baseline in vital sign measurement: Electrocardiogram parameter beats per minute (bpm)

    Time frame: Part A: 21 weeks. Part B: 13 weeks.

  7. Change from baseline in vital sign measurement: Electrocardiogram parameter PR Interval

    Time frame: Part A: 21 weeks. Part B: 13 weeks.

  8. Change from baseline in vital sign measurement: Electrocardiogram parameter QRS Interval

    Time frame: Part A: 21 weeks. Part B: 13 weeks.

  9. Change from baseline in vital sign measurement: Electrocardiogram parameter QT Interval.

    Time frame: Part A: 21 weeks. Part B: 13 weeks.

  10. Change from baseline in vital sign measurement: Electrocardiogram parameter QTcB (calculated using Bazzet method).

    Time frame: Part A: 21 weeks. Part B: 13 weeks.

  11. Change from baseline in vital sign measurement: Electrocardiogram parameter QTcF, (calculated using Fredericia's method).

    Time frame: Part A: 21 weeks. Part B: 13 weeks.

  12. Incidence of clinically significant abnormalities in physical/neurological examination findings.

    Time frame: Part A: 21 weeks. Part B: 13 weeks.

  13. Change from baseline in Columbia-Suicide Severity Rating Scale (C-SSRS; Parts B).

    Time frame: Part A: 21 weeks. Part B: 13 weeks.

Secondary outcomes

  1. Plasma PK parameter: Area under the concentration-time curve from time zero to infinity (AUCinf).

    Time frame: Part A: 21 weeks. Part B: 13 weeks.

  2. Plasma PK parameter: Area under the concentration-time curve from time zero to infinity AUCinf(%extrapolated).

    Time frame: Part A: 21 weeks. Part B: 13 weeks.

  3. Plasma PK parameter: Area under the concentration-time from time zero to time of last measurable concentration (AUClast).

    Time frame: Part A: 21 weeks. Part B: 13 weeks.

  4. Plasma PK parameter: Apparent total clearance following extravascular administration (CL/F).

    Time frame: Part A: 21 weeks. Part B: 13 weeks.

  5. Plasma PK parameter: Maximum concentration (Cmax).

    Time frame: Part A: 21 weeks. Part B: 13 weeks.

  6. Plasma PK parameter: Absorption lag time (tlag).

    Time frame: Part A: 21 weeks. Part B: 13 weeks.

  7. Plasma PK parameter: Time to reach maximum concentration (tmax).

    Time frame: Part A: 21 weeks. Part B: 13 weeks.

  8. Plasma PK parameter: Terminal elimination half-life (t1/2).

    Time frame: Part A: 21 weeks. Part B: 13 weeks.

  9. Plasma PK parameter: Apparent volume of distribution during the terminal elimination phase after extravascular administration (Vz/F).

    Time frame: Part A: 21 weeks. Part B: 13 weeks.

  10. Concentration of VES001 in CSF in the highest two dose level cohorts in Part A.

    Time frame: Part A: 21 weeks. Part B: 13 weeks.

  11. Concentration of VES001 in CSF in all dose level cohorts in Part B.

    Time frame: Part A: 21 weeks. Part B: 13 weeks.

  12. Concentration of VES001 in plasma/CSF ratio in the highest two dose level cohorts in Part A.

    Time frame: Part A: 21 weeks. Part B: 13 weeks.

  13. Concentration of VES001 in plasma/CSF ratio in all dose level cohorts in Part B.

    Time frame: Part A: 21 weeks. Part B: 13 weeks.

  14. Comparison of the plasma PK of VES001 following a single oral dose in the fed and fasted state in Part A.

    Time frame: Part A: 21 weeks. Part B: 13 weeks.

    Refer to the PK parameters listed above.

Sponsors and collaborators

Lead sponsor

Vesper Biotechnologies ApS

Industry

Registry information

Official study title

A Randomised, Double-blind, Placebo-controlled Single and Multiple Ascending Dose Study in Healthy Volunteers and Asymptomatic GRN Mutation Carriers to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VES001

Acronym: SORT-IN-1

Important dates

Study start
2023
Primary completion
2024
Study completion
2024
First posted
Jan 26, 2024
Registry last updated
Aug 22, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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