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

NCT05653037 | Active Not Recruiting Phase 1

• 1 other identifier: RBFI1101
• Study Type: interventional
• Target: 32
• Locations: 1 country
• Sites: 1

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.

Conditions

Healthy Volunteers

Outcome Measures

Primary Outcomes (1)

• Number of Participants with Treatment Related Adverse Events as Assessed by CTCAE v5.0

  SAD: Up to Day 169 ;

Secondary Outcomes (12)

• To characterize the pharmacokinetics (PK) of RBD4059 in healthy subjects:Cmax

  Up to 48 hours post-dose

• To characterize the pharmacokinetics (PK) of RBD4059 in healthy subjects:Tmax

  Up to 48 hours post-dose

• To characterize the pharmacokinetics (PK) of RBD4059 in healthy subjects:AUC0-t

  Up to 48 hours post-dose

• To characterize the pharmacokinetics (PK) of RBD4059 in healthy subjects:AUC0-inf

  Up to 48 hours post-dose

• To characterize the pharmacokinetics (PK) of RBD4059 in healthy subjects:t1/2

  Up to 48 hours post-dose

Study Arms (2)

RBD4059 SAD experimental group

EXPERIMENTAL

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

Drug: RBD4059

Placebo SAD group

PLACEBO COMPARATOR

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

Drug: Placebo

Interventions

RBD4059 DRUG

Subcutaneously Administered RBD4059 in Healthy Subjects.

RBD4059 SAD experimental group

Placebo DRUG

Subcutaneously Administered Placebo in Healthys Subject.

Placebo SAD group

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Suzhou Ribo Life Science Co. Ltd.: lead

Study Sites (1)

Q-Pharm Pty Limited

Brisbane, Australia

Location

Study Design

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

Study Record Dates

• First Submitted: November 21, 2022
• First Posted: December 16, 2022
• Study Start: March 21, 2023
• Primary Completion: December 1, 2025
• Study Completion: December 1, 2025
• Last Updated: December 3, 2024

Record last verified: 2024-12

Locations

AU (1)