Extension Study of ADX-324 in Hereditary angioedema

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Adarx Pharmaceuticals Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

Decision date (initial)

2026-06-29

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

External identifiers

EU CT number

2025-521353-16-00

WHO UTN

U1111-1318-0231

ClinicalTrials.gov

NCT07428499

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacokinetic, Safety, Pharmacodynamic, Efficacy

To evaluate the long-term safety of repeat dosing of ADX-324 in participants with HAE

Secondary objectives 2

1. -To evaluate the efficacy of ADX-324 in preventing HAE attacks
2. -To evaluate the effects of ADX 324 on the quality and pattern of HAE attacks.

Conditions and MedDRA coding

Hereditary Angioedema

Regulatory references

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. 1. Received 2 doses of study drug (ADX-324 and/or placebo) in Study ADX-324-301 and completed the study (follow up through Week 25).
2. 2. 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.
3. 3. Have access to, and the ability to use, at least one acute HAE therapy to treat HAE attacks (eg, plasma-derived or recombinant C1-INH concentrate or a BK2-receptor antagonist) that has previously been shown to be effective for the participant.
4. 4. Women of childbearing potential must have a negative urine pregnancy test on Day 1 before study drug administration and must agree to use acceptable contraceptive methods if engaged in sexual activity of childbearing potential (refer to Section 13.2.2) from the time of signing the informed consent form (ICF) until the EOS Visit or 3 months after the last study drug administration, whichever is longer. Fertile male participants with female partners of childbearing potential should agree to use acceptable contraceptive methods from the time of signing the ICF until the EOS Visit or 3 months after the last study drug administration, whichever is longer.

Exclusion criteria 2

1. 1. Had unacceptable toxicity in Study ADX-324-301, as determined by the Investigator. This includes, but is not limited to, the following: a. Met Hy’s law criteria (ALT or AST ≥3 × ULN AND total bilirubin ≥2 × ULN AND ALP <2 × ULN and no other reason could be found to explain the findings). b. Grade ≥3 hypersensitivity reaction to study drug.
2. 2. Has any condition or abnormal laboratory value, ECG finding, vital signs, or physical examination finding that, in the Investigator’s opinion, would pose an unacceptable risk to the participant if they participate in the study.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Incidence and severity of TEAEs.

Secondary endpoints 4

1. The time-normalized number of Investigator-confirmed HAE attacks (per month)
2. The time-normalized number of Investigator-confirmed HAE attacks requiring acute HAE therapy (per month)
3. The -normalized number of moderate or severe Investigator confirmed HAE attacks (per month)
4. The proportion of participants who have not experienced an Investigator-confirmed HAE attack (HAE attack-free)

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Adarx Pharmaceuticals Inc.

Sponsor organisation

Adarx Pharmaceuticals Inc.

Address

5871 Oberlin Drive Suite 200

City

San Diego

Postcode

92121-3732

Country

United States

Scientific contact point

Organisation

Adarx Pharmaceuticals Inc.

Contact name

VP, Clinical Development

Public contact point

Organisation

Adarx Pharmaceuticals Inc.

Contact name

VP, Clinical Development

Third parties 6

Locations

11 EU/EEA countries · 22 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 231 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications