Oral PHA-022121 for Hereditary Angioedema
(HAE CHAPTER-1 Trial)

Recruiting in Palo Alto (17 mi)

+35 other locations

Overseen byMarc Riedl, MD

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: Pharvaris Netherlands B.V.

Prior Safety Data

Trial Summary

What is the purpose of this trial?

This study evaluates the safety and efficacy of PHA-022121 administered orally for prophylaxis against angioedema attacks in patients with hereditary angioedema (HAE). The study consists of 2 parts, with patients completing participation in Part 1 prior to initiation of treatment in Part 2. Part 1 of the study has 3 parallel arms and approximately 30 patients will be equally randomized to one of two dose regimens of PHA-022121 or matching placebo. Patients will continue to the single open-label arm in Part 2 of the study after completion of Part 1. The screening period is up to 8 weeks and the treatment periods are 12 weeks (Part 1) and 30 months (Part 2) in duration.

Eligibility Criteria

This trial is for people with Hereditary Angioedema (HAE) types I or II who've had at least 3 attacks in the last 3 months, or a minimum of 2 during screening. They must be able to use standard acute attack meds and sign consent forms. It's not for pregnant/breastfeeding individuals, those with significant health issues that could affect safety/participation, recent drug abuse history, abnormal kidney/liver function, or if they've used certain HAE treatments/drugs recently.

Inclusion Criteria

I can access and know how to use emergency medications.

I have been diagnosed with Hereditary Angioedema type I or II.

Signed and dated informed consent form

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Exclusion Criteria

I do not have any health conditions that would make it unsafe for me to join the study.

Participation in any other investigational drug study within defined period

I haven't taken specific medications for swelling attacks before joining this study.

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Treatment Details

Interventions

PHA-022121 (Other)
Placebo (Placebo)

Trial OverviewThe study tests PHA-022121 taken orally to prevent angioedema attacks in HAE patients. It has two parts: Part 1 randomly assigns patients to low/high doses of the drug or placebo for 12 weeks; all move to Part 2 afterwards for an open-label extension lasting up to 30 months.

Participant Groups

4Treatment groups

Experimental Treatment

Placebo Group

Group I: Part 2: Open-labelExperimental Treatment1 Intervention

BID high dose of deucrictibant

Group II: Part 1: Low doseExperimental Treatment1 Intervention

BID low dose of deucrictibant

Group III: Part 1: High doseExperimental Treatment1 Intervention

BID high dose of deucrictibant

Group IV: Part 1: PlaceboPlacebo Group1 Intervention

BID placebo