Study of VSA001 Injection in Chinese Healthy Adult Volunteers

Healthy Adult Volunteers Clinical Trial

Official title:

A Phase 1 Clinical Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Effects of a Single Dose of VSA001 Injection in Chinese Healthy Adult Volunteers

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT05757596
• Other study ID #: VSA001-1001
• Status: Not yet recruiting
• Phase: Phase 1
• Start date: June 1, 2023
• Est. completion date: December 31, 2023

Study information

• Verified date: February 2023
• Source: Visirna Therapeutics HK Limited
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 24
• Est. completion date: December 31, 2023
• Est. primary completion date: December 1, 2023
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years to 55 Years

Eligibility

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.

Related Conditions & MeSH terms

• Healthy Adult Volunteers

Intervention

Drug:
• 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.
• Placebo
0.9% Saline, volume matched

Locations

Country	Name	City	State
China	Peking University Third Hospital	Beijing	Beijing

Sponsors (2)

Lead Sponsor	Collaborator
Visirna Therapeutics HK Limited	Arrowhead Pharmaceuticals

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Pharmacokinetics parameter: Cmax	Maximum plasma concentration (Cmax)	48 hours
Secondary	Pharmacokinetics parameter: Tmax	Time to maximum plasma concentration (Tmax)	48 hours
Secondary	Pharmacokinetics parameter: AUC0-t	Area under the plasma concentration-time curve from the time 0 to the last quantifiable time point (AUC0-t).	48 hours
Secondary	Pharmacokinetics parameter: t1/2	Half-life (t1/2).	48 hours
Secondary	Pharmacokinetics parameter: CL/F	Apparent clearance (CL/F).	48 hours
Secondary	Pharmacokinetics parameter: Vz/F	Apparent volume of distribution (Vz/F).	48 hours
Secondary	Pharmacodynamic (PD) parameters	Change from baseline over time in fasting serum APOC3 and triglycerides (TGs).	85 days