Deucrictibant for Hereditary Angioedema

(RAPIDe-3 Trial)

Recruiting in Palo Alto (17 mi)

+64 other locations

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Recruiting

Sponsor: Pharvaris Netherlands B.V.

Must not be taking: Attenuated androgens, CYP3A4 inhibitors

Disqualifiers: Pregnancy, Angioedema, Hepatic, Renal, others

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?

This is a Phase 3, multicenter, randomized, double-blind, placebo-controlled, 2-period, 2-treatment cross-over study to evaluate the efficacy and safety of orally administered deucrictibant compared to placebo for the on-demand treatment of HAE attacks, including non-severe laryngeal attacks, in participants ≥12 to ≤75 years of age with HAE type 1 or type 2 (HAE-1/2), a proportion of whom are using long-term prophylactic medication for HAE.

Do I have to stop taking my current medications for the trial?

If you are on long-term prophylactic medication for HAE, you must either stay on a stable dose or have stopped using certain medications like plasma-derived C1-INH at least 2 weeks or lanadelumab at least 10 weeks before screening. Other medications, especially those affecting CYP3A4, may need to be stopped 30 days before the trial.

What makes the drug Deucrictibant unique for treating hereditary angioedema?

Deucrictibant is unique because it is an oral treatment option for hereditary angioedema, unlike icatibant, which is administered subcutaneously (under the skin). This oral administration could offer more convenience and ease of use for patients.

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Eligibility Criteria

This trial is for adolescents and adults aged 12 to 75 with Hereditary Angioedema (HAE) types 1 or 2. It's suitable for those who experience HAE attacks, including non-severe throat swelling, and some may be on long-term prevention meds.

Inclusion Criteria

Provision of written informed consent/assent

I am between 12 and 75 years old.

Capable of recording, without assistance, electronic HAE diary and ePRO data using an electronic device

Exclusion Criteria

I have previously been treated with deucrictibant for HAE.

Known hypersensitivity to study drug or any of the excipients of study drug

Any female who is pregnant, plans to become pregnant, or is breastfeeding

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants are randomized and receive double-blinded study drug to treat 2 qualifying HAE attacks

Varies per attack

2 visits (in-person) for each attack treatment

Follow-up

Participants are monitored for safety and effectiveness after the second attack treated with study drug

4 weeks

Participant Groups

The study tests Deucrictibant soft capsules against a placebo when an HAE attack occurs. It's a Phase 3 trial where participants are randomly assigned to receive either the real drug or placebo in different periods without knowing which one they're taking.

Group I: Arm 2 Experimental Treatment 1 Intervention

Placebo administered for first HAE attack, deucrictibant administered for second HAE attack.

Group II: Arm 1 Experimental Treatment 1 Intervention

Deucrictibant administered for first HAE attack, placebo administered for second HAE attack.

Find a Clinic Near You

Research Locations Nearby Select from list below to view details:

Study Site Little Rock, AR

Study Site Chevy Chase, MD

Study Site Dallas, TX

Study Site Madison, MS

More Trial Locations

Who Is Running the Clinical Trial?

Pharvaris Netherlands B.V. Lead Sponsor

References

1. England pubmed.ncbi.nlm.nih.gov

Management of patients with hereditary angioedema in Germany: comparison with other countries in the Icatibant Outcome Survey. [2020] The Icatibant Outcome Survey (IOS; NCT01034969) is a Shire-sponsored, international, observational study monitoring the safety and effectiveness of icatibant, a bradykinin B2 receptor antagonist approved for the acute treatment of adults with hereditary angioedema with C1 inhibitor deficiency (HAE-C1-INH).

2. Germany pubmed.ncbi.nlm.nih.gov

Social costs of icatibant self-administration vs. health professional-administration in the treatment of hereditary angioedema in Spain. [2021] Icatibant is the only subcutaneous treatment for acute Type I and Type II hereditary angioedema with C1-esterase inhibitor deficiency (HAE-C1-INH) licensed for self-administration in Europe.

3. Switzerland pubmed.ncbi.nlm.nih.gov

[Allergy-immunology. New therapies for acute attacks in hereditary angioedema]. [2013] Hereditary angioedema is a disease which develops as a result of a deficiency or dysfonction of C1-inhibitor, a key regulator of the complement, coagulation and contact cascades, resulting among others in excessive release of bradykinin. This disease mortality rate is high in absence of immediate and effective treatment, in particular in presence of acute attacks of the upper respiratory tract (laryngeal edema). Until now only administration of a purified C1-inhibitor extract was effective against these symptoms. This paper aims to synthesise essentials knowledge concerning news drugs, in particular icatibant, a selective bradykinin B2- receptor antagonist whose use should be widened to the treatment of angioedema with ACE-inhibitors intolerance.

4. Switzerland pubmed.ncbi.nlm.nih.gov

Icatibant for Multiple Hereditary Angioedema Attacks across the Controlled and Open-Label Extension Phases of FAST-3. [2015] In randomized, controlled, double-blind, multicenter phase 3 studies, one icatibant injection was efficacious and generally well tolerated in patients with a single hereditary angioedema (HAE) attack. Here, the efficacy and safety of icatibant for multiple HAE attacks was evaluated across the controlled and open-label extension phases of the For Angioedema Subcutaneous Treatment (FAST)-3 study (NCT00912093).

5. United States pubmed.ncbi.nlm.nih.gov

Oral Plasma Kallikrein Inhibitor for Prophylaxis in Hereditary Angioedema. [2018] Hereditary angioedema is a life-threatening illness caused by mutations in the gene encoding C1 inhibitor (also called C1 esterase inhibitor) that lead to overactivation of the kallikrein-bradykinin cascade. BCX7353 is a potent oral small-molecule inhibitor of plasma kallikrein with a pharmacokinetic and pharmacodynamic profile that may help prevent angioedema attacks.