A Phase 2 in Adult Subjects With Hereditary Angioedema

Launched by SHANGHAI ARGO BIOPHARMACEUTICAL CO., LTD. · Feb 25, 2025

Keywords

ClinConnect Summary

This clinical trial is looking at a new treatment for adults with Hereditary Angioedema (HAE), a condition that causes sudden and severe swelling in various parts of the body. The study is currently not recruiting participants but aims to enroll adults aged 18 to 70 who have been diagnosed with HAE-1 or HAE-2 and have experienced at least two HAE attacks in the past two months. To participate, individuals must be able to use at least one medication for acute HAE attacks and provide written consent to join the study.

Participants in this trial can expect to undergo regular check-ups and monitoring throughout the study. It's important to note that not everyone will be eligible; for instance, individuals with certain other medical conditions or those who have had recent surgeries may not qualify. Additionally, women who are pregnant or breastfeeding and men with partners who could become pregnant will need to follow specific contraceptive measures. This trial aims to find a safe and effective new option for managing HAE, which could significantly improve the quality of life for those affected by this condition.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Must have given written informed consent and be able to comply with all study requirements.
• • Males or females 18 to 70 years of age at the time of informed consent.
• • Documented diagnosis of HAE-1/HAE-2.
• • At least 2 HAE attacks within the 8-week run-in period, as confirmed by an investigator based on the protocol-specified definition
• • Access to and ability to use ≥ 1 acute medication(s)
• • Female subjects must be non-pregnant;non-lactating, and either surgically sterile
• • Male subjects with WOCBP partners, dual contraception is required if no surgically sterile
• Exclusion Criteria:
• • Any clinically significant medical or psychiatric condition or medical history that, in the opinion of the Investigator, makes the subject unsuitable for participation in the study
• • Any concomitant diagnosis of another form of chronic angioedema, such as acquired angioedema, HAE with normal C1-INH, idiopathic angioedema, or recurrent angioedema associated with urticaria
• • History or presence of carcinoma within 5 years prior to screening, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated.
• • Have undergone major surgery within 3 months prior to screening.
• • History of clinically significant arterial or venous thrombosis, or current clinically significant prothrombotic or bleeding risk
• • History of allergic reaction to an oligonucleotide or N-acetylgalactosamine
• • Prior treatment with any oligonucleotides within 6 months if single dose or 12 months if multiple doses prior to screening.
• • Exposure to another investigational drug or biological agent within 30 days or within at least 5 half-lives prior to screening.
• • With ANY of the abnormalities in clinical laboratory tests at screening and run-in period
• • Clinically significant findings on 12-lead electrocardiogram that would place the patient at risk or interfere with participation in the study at screening.
• • Positive for Human immunodeficiency virus (HIV), HBsAg, Hepatitis C or syphilis infection
• • Active infection requiring systemic antiviral or antimicrobial therapy that will not be completed prior to dosing.