A Single Ascending Doses Phase I Study to Evaluate the Safety and Pharmacokinetics of RBD4059 in Healthy Subjects

NCT ID: NCT05653037

Last Updated: 2024-12-03

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE1
• Total Enrollment: 32 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-03-21
• Study Completion Date: 2025-12-31

Brief Summary

This is a Randomized, Single-blind, Placebo-Controlled Phase I Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single Ascending Doses of Subcutaneously Administered RBD4059 in Healthy Subjects. The study will be performed in single ascending dose (SAD) phase in healthy subjects. The decision to escalate to subsequent dose levels will be made by the SRC based on the review of all available safety information and PK/PD data in each cohort.

RBD4059, a GalNAc conjugated siRNA, is an FXI-targeted new molecular entity independently developed by Ribo using its proprietary siRNA delivery system - RIBO-GalSTARTM.

Detailed Description

RBD4059, a GalNAc conjugated siRNA, is an FXI-targeted new molecular entity independently developed by Ribo using its proprietary siRNA delivery system - RIBO-GalSTARTM. As a GalNAc-based liver targeting delivery system, RIBO-GalSTARTM incorporates a unique technology for the delivery of oligonucleotide therapeutics for various targets and indications associated with the liver, thus enabling highly specific and highly durable enrichment of the siRNA in hepatocytes, the primary source of FXI. RBD4059 is, therefore, proposed to be a novel effective and durable antithrombotic treatment, with low bleeding risk via inhibition of FXI activity and prolongation of APTT.

Conditions

Healthy Volunteers

Study Design

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

Study Groups

RBD4059 SAD experimental group

Subjects in SAD experimental groups will receive a single subcutaneous injection of RBD4059 on Day 1.

Group Type: EXPERIMENTAL

RBD4059

Intervention Type: DRUG

Subcutaneously Administered RBD4059 in Healthy Subjects.

Placebo SAD group

Subjects in SAD placebo groups will receive a single subcutaneous injection of placebo on Day 1.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Subcutaneously Administered Placebo in Healthys Subject.

Interventions

RBD4059

Subcutaneously Administered RBD4059 in Healthy Subjects.

Intervention Type: DRUG

Placebo

Subcutaneously Administered Placebo in Healthys Subject.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Male and female (non-childbearing potential only), aged 18 to 65 years, inclusive.
• Body mass index (BMI) between 18 and 32 kg/m2, inclusive.
• APTT, Prothrombin time(PT), INR, thrombin time (TT)，FXI activity must be within the normal reference range (as per the local laboratory reference range) at screening .
• Adequate complete blood counts (CBCs) and platelet are within the normal reference range (If CBCs are outside the reference range, unless not clinically significant determined by the Investigator ).
• Healthy as determined by pre-study medical history, vital signs, physical examination, clinical laboratory assessments, and 12-lead electrocardiogram (ECG).
• Subjects who are willing to cooperate with the study staff, comply with the study requirements, complete the study according to the relevant procedures specified in the protocol, and correctly understand and sign the informed consent form in writing.

Exclusion Criteria

• Any uncontrolled or serious disease that may interfere with participation in the clinical study and/or put the subject at significant risk (according to Investigator's judgment) if he/she participates in the clinical study.
• History or presence of cardiovascular disease (including peripheral artery and cerebrovascular disease).
• Systolic blood pressure (SBP) is less than 90 or greater than 140 mmHg and/or diastolic blood pressure (DBP) is less than 40 or greater than 90 mmHg after 10 minutes of supine rest, unless determined by the Investigator to be not clinically significant.
• Diagnosis of diabetes mellitus, history of gestational diabetes that is fully resolved is not permitted.
• Any conditions which, in the opinion of the Investigator, would make the subject unsuitable for enrollment or could interfere with the subject's participation in or completion of the study.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

Suzhou Ribo Life Science Co. Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Q-Pharm Pty Limited

Brisbane, , Australia

Countries

Australia

Other Identifiers

RBFI1101

Identifier Type: -

Identifier Source: org_study_id