Study of VSA001 Injection in Chinese Healthy Adult Volunteers

NCT05757596 | Completed Phase 1 FDA Drug

• 1 other identifier: VSA001-1001
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

This is a phase 1, randomized, double-blind, placebo-controlled, parallel-group study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics effects of a single dose of VSA001 injection in Chinese healthy adult volunteers. Eligible enrolled participants will initially receive VSA001 injection at the assigned dose level.

Conditions

Healthy Adult Volunteers

Outcome Measures

Primary Outcomes (1)

• Safety and tolerability

  Incidence, frequency, and severity of adverse events (AEs) and serious adverse events (SAEs), and the relationship with VSA-01001. Clinically significant abnormalities in laboratory tests, vital signs, physical examination, 12-lead electrocardiograms (ECG).

  85 days

Secondary Outcomes (7)

• Pharmacokinetics parameter: Cmax

  48 hours

• Pharmacokinetics parameter: Tmax

  48 hours

• Pharmacokinetics parameter: AUC0-t

  48 hours

• Pharmacokinetics parameter: t1/2

  48 hours

• Pharmacokinetics parameter: CL/F

  48 hours

Study Arms (2)

VSA001 injection

ACTIVE COMPARATOR

A single dose of active drug (VSA001, low or high dose) will be administered by subcutaneous injection on Day 1.

Drug: VSA001 injection

Placebo

PLACEBO COMPARATOR

The placebo is normal saline (0.9%) administered subcutaneously; volume matched to the corresponding VSA001 dose volume.

Drug: Placebo

Interventions

VSA001 injection DRUG

The active drug is VSA001 injection. The active pharmaceutical ingredient (API) contained in VSA001 is a synthetic, double-stranded, hepatocyte targeted NAG-conjugated RNAi.

VSA001 injection

Placebo DRUG

0.9% Saline, volume matched

Placebo

Eligibility Criteria

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

You may qualify if:

• The subjects voluntarily participate in this study who are able to read, understand, and sign the ICF before participation; and have a full understanding of the content, process and possible adverse reactions of the study and are able to complete the study in accordance with the requirements of the protocol.
• Healthy male and female subjects aged between 18 and 55 years (both inclusive) at the time of informed consent.
• Body mass index (BMI) = weight (kg)/height (m)2, within the range of 19.0-30.0 kg/m2 (both inclusive), and body weight no less than 50 kg.
• In good general health and without clinically significant abnormalities as judged by the investigator, based on medical history, physical examination, vital signs, 12-lead ECG, and laboratory results.
• Negative serum pregnancy test within 72 h prior to initiation of study treatment in all premenopausal females and females who have been amenorrheic for less than 12 months. (Serum pregnancy test is not required for females who have undergone surgical sterilization, such as hysterectomy and/or bilateral oophorectomy, or those who have not experienced menses for 12 consecutive months and are judged to be postmenopausal based on factors such as age and castration therapy).
• Subjects and their partners must agree to use adequate contraceptive methods prior to initiation of study treatment, during the study, and for at least 3 months after discontinuation of study treatment.
• Fasting serum TGs \>80 mg/dL (\>0.903 mmol/L) at screening.

You may not qualify if:

• History or presence of significant or clinically significant diseases/abnormality, including but not limited to cardiac/cardiovascular, respiratory, endocrine, gastrointestinal, renal, hepatic, gallbladder, dermatological, hematological, immunological, neurologic, or psychiatric diseases/abnormality, or the disease that, in the judgement of the investigator, present a safety concern or affects the pharmacokinetic evaluation.
• A family history of congenital long QT syndrome, Brugada syndrome or unexplained sudden cardiac death.
• Subjects who are with acute exacerbation of any significant acute or chronic disease as judged by the investigator.
• AST and ALT \>2×upper limit of normal (ULN) , or total bilirubin \>ULN at screening.
• Serum creatinine estimated eGFR \< 60 ml/min/1.73 m2 per MDRD formula.
• Cardiac troponin (troponin I) above ULN at Screening.
• Fasting serum TGs \>300 mg/dL (\>3.38 mmol/L) at screening.
• Presence of other conditions or treatments that may affect the study results and interfere with the subject's participation in the study as assessed by the investigator.

Sponsors & Collaborators

• Visirna Therapeutics HK Limited: lead
• Arrowhead Pharmaceuticals: collaborator

Study Sites (1)

Peking University Third Hospital

Beijing, Beijing Municipality, China

Location

Related Publications (1)

• Wang F, You D, Niu X, Shi J, Li Y, Qi L, Li H. Safety, pharmacokinetics and pharmacodynamics of Plozasiran in Chinese healthy volunteers. Cardiovasc Diabetol. 2025 Oct 15;24(1):399. doi: 10.1186/s12933-025-02929-9.

  PMID: 41094680 DERIVED

Study Design

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

Study Record Dates

• First Submitted: February 14, 2023
• First Posted: March 7, 2023
• Study Start: May 17, 2023
• Primary Completion: September 25, 2023
• Study Completion: December 31, 2023
• Last Updated: March 20, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)