A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VES001 in Healthy Participants (SORT-IN-1)

Vesper Biotechnologies

Status and phase: Enrolling; Phase 1

Conditions: Dementia, Frontotemporal

Treatments: Drug: Placebo; Drug: VES001

Study type: Interventional

Funder types: Industry

Identifiers

NCT06226064

Ph1/VES001

Details and patient eligibility

About

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.

Full description

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).

Enrollment: 78 estimated patients

Sex: All

Ages: 18 to 55 years old

Volunteers: Accepts Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria Part A & B:

1. Healthy men or women aged 18 to 55 years.
2. Body Mass Index between 18 and 32 kg/m2, with a minimum weight of 50 kg.
3. Effective contraception required during the study and for at least 90 days after their last dose.
4. 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:

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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78 participants in 2 patient groups, including a placebo group

VES001 (Healthy Participants)

Experimental group

Description:

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

Treatment:

Drug: VES001

Placebo (Healthy Participants)

Placebo Comparator group

Description:

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

Treatment:

Drug: Placebo