Help us take a closer look at managing HAE

Study purpose

The HAE CHAPTER-1 Study is assessing the safety and efficacy of PHVS416 in preventing hereditary angioedema (HAE) attacks in people with HAE type I or type II in Part 1 of the study.

Part 2 of the HAE CHAPTER-1 Study is assessing the safety of PHVS416 administered as long-term prophylactic HAE treatment.

Study periods

The total duration of participation can last approximately 36 months, including a screening period that can last up to 8 weeks, a 12-week double-blind treatment period, an open-label treatment period (30 months), and an end-of-study visit 4 weeks after the last study treatment administration. Participants will have some visits on-site at the study clinic, while other visits may be performed remotely (where applicable and permitted by local regulations).

About PHVS416

In Part 1 of this clinical research study (the double-blind treatment period), PHVS416 is a softgel capsule taken orally that is being compared to a placebo. Participants will be randomised to receive the low dose of PHVS416 (1 capsule of PHVS416 and 1 capsule of placebo), the high dose of PHVS416 (2 capsules of PHVS416), or the placebo (2 capsules of placebo). Two capsules of PHVS416 or placebo are taken by mouth twice daily after breakfast and dinner.

In Part 2 of this clinical research study (the open-label treatment period), 2 capsules of PHVS416 will be taken twice a day, after breakfast and dinner. No participants will receive placebo during this period.

PHVS416 attempts to address the increased amount of the protein bradykinin in people with HAE. In most cases, people with HAE have a genetic defect in the C1-inhibitor, causing a biochemical imbalance with increased amounts of bradykinin. This bradykinin increase leads to the painful swelling that occurs with HAE attacks. PHVS416 is designed to attempt to prevent some of the effects of bradykinin, which may prevent HAE swelling attacks.

Inclusion criteria

• Signed and dated informed consent form
• Diagnosis of HAE type I or II
• Documented history of at least 3 HAE attacks within the last 3 consecutive months prior to screening
  • If the patient has no documentation or has less than 3 attacks in the 3 months before the screening visit, or was on prophylactic treatment, patient must experience a minimum of 2 HAE attacks during the screening period (up to 8 weeks)
• Reliable access and experience to use standard-of-care acute attack medications

Other inclusion and exclusion criteria apply. For additional information, visit ClinicalTrials.gov.

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Exclusion criteria

• Pregnancy or breastfeeding
• Clinically significant abnormal electrocardiogram
• Any other systemic disease or significant disease or disorder that would interfere with the patient’s safety or ability to participate in the study
• Patient is receiving prophylactic treatment for HAE
  • Patients who have previously received prophylactic therapy but have stopped can participate in this study provided a sufficiently long washout period is observed before the patient is screened
• Use of C1-esterase inhibitor, oral kallikrein inhibitors, attenuated androgens, anti-fibrinolytics, or monoclonal HAE therapy within a defined period prior to enrolment
• Abnormal hepatic function
• Abnormal renal function
• History of alcohol or drug abuse within a defined period, or current evidence of substance dependence or abuse
• History of documented severe hypersensitivity to any medicinal product
• Participation in any other investigational drug study within a defined period

Other inclusion and exclusion criteria apply. For additional information, visit ClinicalTrials.gov.

Find a participating site