ADX-324 for Hereditary Angioedema

(STOP-HAE Trial)

Overseen By Chelsea Ruller, MS

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: ADARx Pharmaceuticals, Inc.

Must not be taking: ACE inhibitors, Androgens

Disqualifiers: Other angioedema, Cardiovascular disease, Diabetes, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 25 weeks

90 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

Evaluate the efficacy safety, pharmacokinetics (PK), pharmacodynamics (PD), and health-related quality of life (HRQoL) measures in participants with Type I and Type II HAE.

Who Is on the Research Team?

Masako Murai, MD

Principal Investigator

ADARx Pharmaceuticals, Inc.

Are You a Good Fit for This Trial?

This trial is for people with Hereditary Angioedema (HAE), specifically those who have a certain level of C1-INH function and antigen, a pathogenic mutation in the SERPING1 gene, complement factor C4 below normal levels, onset of HAE by age 30, and family history of HAE. It's not open to individuals outside these specific criteria.

Inclusion Criteria

Provided written informed consent and any authorizations required by local law and be willing to comply with all study requirements for the duration of the study

Experienced ≥1 Investigator-confirmed HAE attack in the first 4 weeks of Screening or ≥2 Investigator-confirmed HAE attacks in 8 weeks of Screening

I am 18 years old or older.

Exclusion Criteria

Received treatment with another investigational product or device within specified timeframes

History of alcohol or drug abuse within the previous year prior to Screening, or current evidence of substance dependence or abuse and/or self-reported alcoholic intake averaging >3 drinks/day

I have significant kidney disease or a history of nephrotic syndrome.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive ADX-324 or placebo to evaluate efficacy in preventing HAE attacks

25 weeks

Monthly visits

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• ADX-324

Trial Overview The STOP-HAE study is testing ADX-324 against a placebo to see if it's effective and safe for treating Type I and II HAE. The trial will also look at how the body processes the drug, its effects on the body over time, and how it impacts patients' quality of life.

How Is the Trial Designed?

Group I: ADX-324 Dose Level 2 Experimental Treatment 2 Interventions

Participants who meet screening eligibility criteria will be randomized to 1 of 3 treatment groups in a 1:1:1 ratio. Randomization will be stratified by baseline HAE attack rate.

Group II: ADX-324 Dose Level 1 Experimental Treatment 1 Intervention

Participants who meet screening eligibility criteria will be randomized to 1 of 3 treatment groups in a 1:1:1 ratio. Randomization will be stratified by baseline HAE attack rate.

Group III: Placebo Placebo Group 1 Intervention

Participants who meet screening eligibility criteria will be randomized to 1 of 3 treatment groups in a 1:1:1 ratio. Randomization will be stratified by baseline HAE attack rate.

Who Is Running the Clinical Trial?

ADARx Pharmaceuticals, Inc.

Lead Sponsor

Trials

3

Recruited

220+