Study of VSA001 Injection in Chinese Healthy Adult Volunteers

NCT ID: NCT05757596

Last Updated: 2025-03-20

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 24 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-05-17
• Study Completion Date: 2023-12-31

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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

VSA001 injection

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

Group Type: ACTIVE_COMPARATOR

VSA001 injection

Intervention Type: 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.

Placebo

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

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

0.9% Saline, volume matched

Interventions

VSA001 injection

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

Intervention Type: DRUG

Placebo

0.9% Saline, volume matched

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. 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.

2. Healthy male and female subjects aged between 18 and 55 years (both inclusive) at the time of informed consent.

3. 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.

4. 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.

5. 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).

6. 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.

7. Fasting serum TGs >80 mg/dL (>0.903 mmol/L) at screening.

Exclusion Criteria

1. 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.

2. A family history of congenital long QT syndrome, Brugada syndrome or unexplained sudden cardiac death.

3. Subjects who are with acute exacerbation of any significant acute or chronic disease as judged by the investigator.

4. AST and ALT >2×upper limit of normal (ULN) , or total bilirubin >ULN at screening.

5. Serum creatinine estimated eGFR < 60 ml/min/1.73 m2 per MDRD formula.

6. Cardiac troponin (troponin I) above ULN at Screening.

7. Fasting serum TGs >300 mg/dL (>3.38 mmol/L) at screening.

8. 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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 55 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

Arrowhead Pharmaceuticals

INDUSTRY

Sponsor Role: collaborator

Visirna Therapeutics HK Limited

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Peking University Third Hospital

Beijing, Beijing Municipality, China

Countries

China

References

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.

Reference Type: DERIVED

PMID: 41094680 (View on PubMed)

Other Identifiers

VSA001-1001

Identifier Type: -

Identifier Source: org_study_id