Evaluating ADX-324 for safety and effectiveness in healthy volunteers and patients with Hereditary Angioedema

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

This study is testing a new drug called ADX-324 to see if it's safe and effective for healthy people and those with Hereditary Angioedema.

Quick facts

• Phase: PHASE1; PHASE2
• Study type: Interventional
• Enrollment: 53 (estimated)
• Ages: 18 Years to 55 Years
• Sex: All
• Sponsor: ADARx Pharmaceuticals, Inc. (industry)
• Locations: 1 site (Adelaide, South Australia)
• Trial ID: NCT05691361 on ClinicalTrials.gov

What this trial studies

This clinical trial is a Phase 1 study designed to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of a new drug, ADX-324, in both healthy volunteers and patients diagnosed with Hereditary Angioedema (HAE). The study is divided into two parts: the first part involves a randomized, double-blind, placebo-controlled approach with healthy volunteers receiving single ascending doses of the drug, while the second part includes an open-label expansion cohort for HAE patients using a selected dose from the first part. The goal is to gather comprehensive data on how the drug behaves in the body and its potential therapeutic effects.

Who should consider this trial

Good fit: Ideal candidates for this study include healthy adults aged 18 to 55 and patients diagnosed with Hereditary Angioedema.

Not a fit: Patients with significant medical histories, active malignancies, or other serious health conditions may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to a new treatment option for patients suffering from Hereditary Angioedema.

How similar studies have performed: Other studies evaluating similar pharmacological approaches have shown promise, but this specific drug and its application in HAE is novel.

Eligibility criteria

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.

Where this trial is running

Adelaide, South Australia

• CMAX Clinical Research — Adelaide, South Australia, Australia (RECRUITING)

Study contacts

• Principal investigator: Nicholas Farinola, MD — CMAX Clinical Research Pty Ltd
• Study coordinator: CMAX Reception

Conditions: Hereditary Angioedema