A Study of ORN252 in Healthy Participants

April 13, 2026 updated by: Orna Therapeutics, Inc.

A Phase 1 Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ORN252 in Healthy Participants

The purpose of this study is to assess the safety and tolerability of ORN252 in healthy adult participants.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

20

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Australia
    • Queensland
      • Herston, Queensland, Australia, 4006

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult

Accepts Healthy Volunteers

Yes

Description

Inclusion Criteria:

  • Male and female participants who are healthy as determined by the Investigator based on review of medical history, physical examination, and laboratory tests
  • Participant is willing and able to comply with study visits and other protocol requirements
  • Other protocol specific inclusion criteria may apply

Exclusion Criteria:

  • Clinically significant history or presence of organ dysfunction
  • Use of any investigational product within 3 months, or 5 half-lives, whichever longer
  • Other protocol specific exclusion criteria may apply

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Basic Science
  • Allocation: N/A
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Single Ascending Dose
Ascending single dose of ORN252 on a specified day
Biological: ORN252
ORN252 is a lipid nanoparticle encapsulating a circular RNA encoding an anti-CD19 CAR protein.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
The incidence and severity of safety, clinical, and laboratory abnormalities in healthy adult participants.
Time Frame: 12 weeks
To evaluate the safety and tolerability of ORN252 administered to healthy adult participants.
12 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Pharmacokinetic (PK) parameters of ORN252 components
Time Frame: 29 days
Area under the concentration versus time curve from time zero to the time of the last quantifiable concentration [AUClast]
29 days
Pharmacokinetic (PK) parameters of ORN252 components
Time Frame: 29 days
Maximum concentration [Cmax]
29 days
Pharmacokinetic (PK) parameters of ORN252 components
Time Frame: 29 days
Time of maximum concentration [Tmax]
29 days
Pharmacokinetic (PK) parameters of ORN252 components
Time Frame: 29 days
Terminal elimination half-life [t1/2]
29 days
Pharmacokinetic (PK) parameters of ORN252 components
Time Frame: 29 days
Volume of distribution [Vdss]
29 days
Pharmacokinetic (PK) parameters of ORN252 components
Time Frame: 29 days
Clearance [CL]
29 days
Pharmacokinetic (PK) parameters of ORN252 components
Time Frame: 29 days
Area under the concentration versus time curve from time zero to infinity (AUCinf)
29 days
Pharmacodynamic (PD) effects of of ORN252
Time Frame: 12 weeks
Changes in circulating B cell counts and lymphocytes
12 weeks
Immunogenicity of ORN252
Time Frame: 12 weeks
To assess the presence of anti-drug antibodies in healthy adult participants
12 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Orna Therapeutics, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 16, 2026

Primary Completion (Estimated)

December 1, 2026

Study Completion (Estimated)

March 1, 2027

Study Registration Dates

First Submitted

February 10, 2026

First Submitted That Met QC Criteria

February 23, 2026

First Posted (Actual)

February 27, 2026

Study Record Updates

Last Update Posted (Actual)

April 16, 2026

Last Update Submitted That Met QC Criteria

April 13, 2026

Last Verified

April 1, 2026

More Information

Terms related to this study

Other Study ID Numbers

  • AD25201

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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