Safety, Tolerability, PK, PD of ADX-324 in Healthy Volunteers and Hereditary Angioedema Patients

June 12, 2026 updated by: ADARx Pharmaceuticals, Inc.

A Phase 1, Randomized, Placebo-Controlled, Double-Blind Study in Healthy Volunteers and a Phase 2a, Open-Label Study in Patients With Hereditary Angioedema to Evaluate the Safety, Tolerability, PK and PD of ADX-324

The first-in-human Phase 1 study will evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ADX-324 in healthy volunteers (HV) and in patients with Hereditary Angioedema (HAE).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

The clinical study described in this protocol is a Phase 1, single-center study evaluating safety, tolerability, PK, and PD of ADX-324.

The study consists of 2 parts:

  • Randomized, double-blind, placebo-controlled, parallel group, single ascending dose (SAD) in HV with up to 6 dose cohorts. For SAD cohorts and planned dosing; and,
  • Expansion cohort in participants with Hereditary Angioedema (HAE) at selected dose from Part A and will be open label.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
53

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2
  • 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 Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Australia
    • South Australia
      • Adelaide, South Australia, Australia, 5000
        • CMAX Clinical Research

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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years to 55 years (Adult)

Accepts Healthy Volunteers

Yes

Description

Part A - HV

Inclusion Criteria:

  1. Male and female adults 18 to 55 years old
  2. Body mass index (BMI) between 18 and 30 kg/m2
  3. Contraception use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies
  4. Willing and able to provide informed consent and comply with all study visits

Exclusion Criteria:

  1. Any significant medical history
  2. Active malignancy and/or history of malignancy in the past 5 years
  3. History of liver disease, Gilbert's syndrome, or abnormal liver function test
  4. Estimated creatinine clearance <60 mL/min or serum creatinine > 1.5-fold upper limit of normal.
  5. Any active infection or acute illness
  6. Major surgery or significant traumatic injury occurring within 3 months
  7. Have any other conditions that, in the opinion of the Investigator or Sponsor, would make the participant unsuitable for inclusion, or could interfere with the participant participating in or completing the study.
  8. Positive serology tests (HepB, Hep C, HIV)
  9. Use of any prescription, vaccines, supplements/vitamins, or over-the counter medication
  10. Treatment with another investigational product within 30 days prior to the first study drug administration
  11. Known any clinically significant allergic reactions which, in the opinion of the Investigator, would interfere with the volunteer's ability to participate in the study
  12. Known hypersensitivity to any of the study drug ingredients.
  13. Pregnancy, intent to become pregnant during the course of the study, or lactating women

Part B - HAE

Inclusion Criteria:

  1. Male and female ≥18 years old, inclusive, at the time of signing the PICF
  2. Confirmed diagnosis of HAE Types I or II
  3. Evidence of an average of (at least) one HAE attack per month
  4. Participants must have access to, and the ability to use, acute medication(s) to treat angioedema attacks.
  5. Body mass index (BMI) between 18 and 30 kg/m2
  6. Contraception use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies
  7. Willing and able to provide informed consent and comply with all study visits

Exclusion Criteria:

  1. Concurrent diagnosis of any other type of chronic angioedema
  2. History of clinically significant arterial or venous thrombosis, or current history of a clinically significant prothrombotic risk.
  3. Any significant medical history
  4. Active malignancy and/or history of malignancy in the past 5 years
  5. Any active infection or acute illness, inclusive of cold/flu or COVID-19, within 30 days prior to the first study drug administration.
  6. Major surgery or significant traumatic injury occurring within 3 months prior to signature of the PICF
  7. Have any other conditions that, in the opinion of the Investigator or Sponsor, would make the participant unsuitable for inclusion, or could interfere with the participant participating in or completing the study.
  8. Positive serology tests for human immunodeficiency virus (HIV), hepatitis B surface antigen (HBsAg), or hepatitis C virus (HCV).
  9. Use of C1-INH products, androgens, antifibrinolytics or other small molecule medications for routine prophylaxis within four half-lives prior to screening
  10. Must have documented evidence of medical history of HAE attacks
  11. Use of any prescription, vaccines, supplements/vitamins, or over-the counter medication (with the exception of oral contraceptives) within 7 days prior to the first study drug administration.
  12. Treatment with another investigational product or biologic agent within 30 days prior to the study drug administration
  13. History or presence of alcohol abuse or drug use within 30 days prior to the first study drug administration and throughout the study.
  14. Blood donation of 50 to 499 mL within 30 days prior to the first study drug administration or of >499 mL within 60 days prior to the first study drug administration.
  15. Pregnancy, intent to become pregnant during the course of the study, or lactating women.

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: Treatment
  • Allocation: Randomized
  • Interventional Model: Sequential Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: PART A - Active ADX-324 administered to HV
For each cohort in Part A (SAD), 8 participants will be randomized in a 3:1 ratio; 6 participants to active (ADX-324): 2 participants to control (matched placebo). Randomization will be on Day 1. Initially, 2 sentinel participants (1 active and 1 placebo) will be randomized and dosed. The sentinel participants will be evaluated for safety. The investigator's assessment and the independent medical monitor will decide upon the randomization and dosing of the 6 remaining participants (5 active and 1 placebo) according to the randomization schedule.
Drug: ADX-324
siRNA duplex oligonucleotide
Other Names:
  • siRNA
Placebo Comparator: PART A- Placebo administered to HV
For each cohort in Part A (SAD), 8 participants will be randomized in a 3:1 ratio; 6 participants to active (ADX-324): 2 participants to control (matched placebo). Randomization will be on Day 1. Initially, 2 sentinel participants (1 active and 1 placebo) will be randomized and dosed. The sentinel participants will be evaluated for safety. The investigator's assessment and the independent medical monitor will decide upon the randomization and dosing of the 6 remaining participants (5 active and 1 placebo) according to the randomization schedule.
Drug: Placebo
saline
Other Names:
  • Saline
Experimental: PART B - ADX-324 administered to HAE participants
This will be initiated at the dose level determined by the Safety Review Committee from SAD in HVs. The treatment of HAE participants is an open-label study.
Drug: ADX-324
siRNA duplex oligonucleotide
Other Names:
  • siRNA

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Safety in Healthy Volunteers
Time Frame: 365 days
To evaluate the safety and tolerability of ADX-324 in HVs by incidence, relationship, and severity of adverse events and serious adverse events
365 days
Safety in Healthy Volunteers
Time Frame: 365 days
To evaluate the safety and tolerability of ADX-324 in HVs by change in baseline electrocardiogram (ECG) parameters (PR, QRS, QT, and QTcF intervals)
365 days
Safety in Hereditary Angioedema
Time Frame: 365 days
To evaluate the safety and tolerability of ADX-324 in HAE by incidence, relationship, and severity of adverse events and serious adverse events
365 days

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Pharmacokinetics in Healthy Volunteers
Time Frame: 8 days
To characterize the Pharmacokinetics of ADX-324 in HVs by measuring the Maximum observed concentration (Cmax)
8 days
Pharmacokinetics in Healthy Volunteers
Time Frame: 8 days
To characterize the Pharmacokinetics of ADX-324 in HVs by measuring the Time to Cmax (Tmax)
8 days
Pharmacokinetics in Healthy Volunteers
Time Frame: 8 days
To characterize the Pharmacokinetics of ADX-324 in HVs by measuring the Area under the concentration-time curve from 0 to time of last quantifiable concentration (AUC0-last)
8 days
Pharmacokinetics in Healthy Volunteers
Time Frame: 8 days
To characterize the Pharmacokinetics of ADX-324 in HVs by measuring the Area under the concentration-time curve from 0 to infinity (AUC0-∞)
8 days
Pharmacokinetics in Healthy Volunteers
Time Frame: 8 days
To characterize the Pharmacokinetics of ADX-324 in HVs by measuring the Apparent terminal half-life (t½)
8 days
Pharmacokinetics in Healthy Volunteers
Time Frame: 8 days
To characterize the Pharmacokinetics of ADX-324 in HVs by measuring the Terminal elimination rate constant (λz)
8 days
Pharmacokinetics in Healthy Volunteers
Time Frame: 8 days
To characterize the Pharmacokinetics of ADX-324 in HVs by measuring the Total apparent body clearance (CL/F)
8 days
Pharmacokinetics in Healthy Volunteers
Time Frame: 8 days
To characterize the Pharmacokinetics of ADX-324 in HVs by measuring the Apparent volume of distribution (Vz/F)
8 days
Pharmacodynamics in Healthy Volunteers
Time Frame: 365 days
To characterize the PD of ADX-324 in HVs by the Change from base in plasma concentrations over time of pre Kallikrein (PKK)
365 days
Pharmacodynamics in Healthy Volunteers
Time Frame: 365 days
To characterize the PD of ADX-324 in HVs by the Change from base in plasma concentrations over time of Kallikrein (KK)
365 days
Pharmacokinetics in Hereditary Angioedema
Time Frame: 365 days
To characterize the PD of ADX-324 in HAE by Maximum observed concentration (Cmax) of ADX-324
365 days
Pharmacokinetics in Hereditary Angioedema
Time Frame: 8 days
To characterize the PD of ADX-324 in HAE by Time to Cmax (Tmax) of ADX-324
8 days
Pharmacokinetics in Hereditary Angioedema
Time Frame: 8 days
To characterize the PD of ADX-324 in HAE by Area under the concentration-time curve from 0 to time of last quantifiable concentration (AUC0-last) of ADX-324
8 days
Pharmacokinetics in Hereditary Angioedema
Time Frame: 8 days
To characterize the PD of ADX-324 in HAE by Area under the concentration-time curve from 0 to infinity (AUC0-∞) of ADX-324
8 days
Pharmacokinetics in Hereditary Angioedema
Time Frame: 8 days
To characterize the PD of ADX-324 in HAE by Apparent terminal half-life (t½)
8 days
Pharmacokinetics in Hereditary Angioedema
Time Frame: 8 days
To characterize the PD of ADX-324 in HAE by Terminal elimination rate constant (λz)
8 days
Pharmacokinetics in Hereditary Angioedema
Time Frame: 8 days
To characterize the PD of ADX-324 in HAE by Total apparent body clearance (CL/F)
8 days
Pharmacokinetics in Hereditary Angioedema
Time Frame: 8 days
To characterize the PD of ADX-324 in HAE by Apparent volume of distribution (Vz/F)
8 days
Pharmacodynamics in Hereditary Angioedema
Time Frame: 365 days
To characterize the PD of ADX-324 in HV by Change from base in plasma concentrations over time pre-kallikrein (PKK)
365 days
Pharmacodynamics in Hereditary Angioedema
Time Frame: 365 days
To characterize the PD of ADX-324 in HAE by Change from base in plasma concentrations over time kallikren (KK)
365 days

Collaborators and Investigators

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

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
ADARx Pharmaceuticals, Inc.

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Avance Clinical Pty Ltd.

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Lauge Farnaes, MD, ADARx Pharmaceuticals

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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
December 14, 2022

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 31, 2027

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 31, 2027

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
December 16, 2022

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 10, 2023

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
January 20, 2023

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
June 16, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 12, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
June 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • ADX-324-101

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

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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