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

NCT06226064 | Completed Phase 1

• 1 other identifier: Ph1/VES001
• Study Type: interventional
• Target: 78
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Dementia, Frontotemporal

Outcome Measures

Primary Outcomes (13)

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

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

• Incidence of clinically significant abnormalities in safety laboratory values.

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Secondary Outcomes (14)

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

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

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

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

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

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

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

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

• Plasma PK parameter: Maximum concentration (Cmax).

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

Study Arms (2)

VES001 (Healthy Participants)

EXPERIMENTAL

Part A: Ascending single doses and Part B: Multiple ascending dose (seven days of treatment), for healthy volunteers.

Drug: VES001

Placebo (Healthy Participants)

PLACEBO COMPARATOR

Part A: Ascending single doses and Part B: Multiple ascending dose (seven days of treatment), for healthy volunteers.

Drug: Placebo

Interventions

VES001 DRUG

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

VES001 (Healthy Participants)

Placebo DRUG

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

Placebo (Healthy Participants)

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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).

Sponsors & Collaborators

• Vesper Biotechnologies ApS: lead

Study Sites (1)

Center for Human Drug Research

Leiden, 2333, Netherlands

Location

MeSH Terms

Conditions

Frontotemporal Dementia

Condition Hierarchy (Ancestors)

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

Study Officials

• Mads Kjolby, MD, PhD

  Vesper Bio

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 7, 2023
• First Posted: January 26, 2024
• Study Start: October 11, 2023
• Primary Completion: July 26, 2024
• Study Completion: July 26, 2024
• Last Updated: August 22, 2024

Record last verified: 2024-08

Locations

NL (1)