Deucrictibant for Hereditary Angioedema

(BK-AE-nC1INH Trial)

Overseen By Henry Li, MD

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Institute for Asthma and Allergy

Must not be taking: ACE inhibitors, Estrogens, CYP3A4 modulators

Disqualifiers: Pregnancy, Cardiovascular issues, Renal dysfunction, others

Compensation: Varies

Number of Visits: 1

10 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial examines the effectiveness of a new treatment, deucrictibant, for individuals with hereditary angioedema, a condition causing sudden swelling under the skin. The study will test deucrictibant in two ways: taking a tablet daily to prevent attacks and taking capsules during an attack to relieve symptoms. It targets individuals who have experienced at least two angioedema attacks in the last two months and have not found success with common allergy medications. As a Phase 1 and Phase 2 trial, this research aims to understand how deucrictibant works in people and to measure its effectiveness in an initial, smaller group.

Will I have to stop taking my current medications?

You may need to stop taking certain medications before joining the trial. Specifically, you cannot take ACE inhibitors, estrogen-containing medications, or certain other drugs that affect liver enzymes within a specified time before the trial. It's best to discuss your current medications with the trial team to see if any changes are needed.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that deucrictibant extended-release (XR) tablets effectively prevent angioedema attacks without major safety issues. In earlier studies, most patients tolerated these tablets well, experiencing no serious side effects.

For the on-demand deucrictibant capsules used during sudden angioedema attacks, long-term research indicates they were also generally well-tolerated. In a study of 465 attacks, the capsules proved safe and did not cause significant adverse events.

Overall, both the daily tablets and the as-needed capsules of deucrictibant have demonstrated promising safety in studies. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about deucrictibant for hereditary angioedema because it offers a novel approach compared to current treatments. Unlike traditional options that often involve injections or intravenous administration, deucrictibant is taken orally, making it more convenient for patients. Additionally, it features an extended-release (XR) formulation for prophylactic use, which means it could help prevent attacks with just one tablet a day, potentially improving quality of life and adherence. For on-demand situations, the unique capsule form of deucrictibant provides a targeted way to quickly address acute angioedema attacks, offering a promising alternative to existing therapies.

What evidence suggests that deucrictibant could be an effective treatment for hereditary angioedema?

Research has shown that deucrictibant extended-release (XR) tablets can effectively prevent angioedema attacks over an extended period. Evidence indicates that the number of attacks remained low for up to 34 months after starting treatment. In this trial, some participants will receive deucrictibant XR tablets for prophylaxis. For immediate relief, other participants will receive deucrictibant capsules, which studies have shown work quickly, easing symptoms in as little as 1.1 hours during sudden attacks. This suggests that both forms of deucrictibant could be promising options for managing hereditary angioedema (HAE) by either preventing attacks or treating them as they occur. ^{6 7 8 9 10}

Are You a Good Fit for This Trial?

This trial is for individuals with Hereditary Angioedema, a condition causing recurrent swelling episodes. Participants should be those seeking prevention of attacks or needing acute treatment during an attack. Specific inclusion and exclusion criteria details are not provided.

Inclusion Criteria

Provision of written informed consent

Documented history of at least 2 angioedema attacks in the previous 2 months

I am 18 years old or older.

Exclusion Criteria

Participation in a clinical study with any other investigational drug within the previous 30 days or within 5 half-lives of the investigational drug at Screening

Pregnant, planning to become pregnant, or currently breast-feeding females

History of alcohol or drug abuse within the previous year

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Treatment Part 1

Participants receive deucrictibant XR tablet or placebo for prophylaxis against angioedema attacks

24 weeks

Regular visits for monitoring

Treatment Part 2

Participants receive deucrictibant soft capsule or placebo for on-demand treatment of angioedema attacks

16 weeks

Self-administration at home

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

End-of-Treatment and End-of-Study visits

What Are the Treatments Tested in This Trial?

Interventions

• Deucrictibant

Trial Overview The study tests deucrictibant in two forms: as an extended-release tablet for preventing angioedema attacks, and as a soft capsule for quick relief during acute attacks. Both are compared to placebo counterparts in the trial.

How Is the Trial Designed?

Group I: On-demand deucrictibant 20 capsule, oral use Experimental Treatment 1 Intervention

Single 20 capsule orally for treatment of on-demand angioedema attack, for up to 16 weeks

Group II: Deucrictibant XR tablet, 40 mg, prophylaxis Experimental Treatment 1 Intervention

Deucrictibant XR tablet, 40 mg, QD, oral use for 12 weeks

Group III: Placebo to deucrictibant XR tablet, 40 mg, prophylaxis Placebo Group 1 Intervention

Placebo tablets, 40 mg equivalent, QD, oral use for 12 weeks

Group IV: Placebo comparator to on-demand deucrictibant 20 capsule, oral use Placebo Group 1 Intervention

Placebo capsules for treatment of on-demand angioedema attacks, for up to 16 weeks

Who Is Running the Clinical Trial?

Institute for Asthma and Allergy

Lead Sponsor

Citations

1. ir.pharvaris.com ir.pharvaris.com/news-releases/news-release-details/data-supporting-potentially-differentiated-pharvaris-portfolio

Data Supporting the Potentially Differentiated Pharvaris ... The attack rate reduction observed by participants within one week of deucrictibant treatment remained low for up to approximately 34 months: ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06679881

NCT06679881 | Long-Term, Open-label Study of Oral ... Long-Term, Open-label Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE (CHAPTER ...

3. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40882771/

Long-term prophylactic treatment with deucrictibant for ... Conclusion: Deucrictibant extended-release tablet effectively prevented angioedema attacks in patients with AAE-C1INH, with no safety concerns.

4. ir.pharvaris.com ir.pharvaris.com/static-files/fba3fb2f-190e-4e13-94fd-5761801f90f2

Long-Term Safety and Efficacy of Oral Deucrictibant for ... 15. EC85, effective concentration estimated to provide 85% maximal response (13.8 ng/mL); HAE, hereditary angioedema; IR, immediate-release; R, ...

5. jacionline.org jacionline.org/article/S0091-6749(24)01892-X/fulltext

Long-Term Safety and Efficacy of Oral Deucrictibant for ... Results of the ongoing CHAPTER-1 OLE provide evidence on the long-term safety and efficacy of deucrictibant for prevention of HAE attacks.

6. ir.pharvaris.com ir.pharvaris.com/news-releases/news-release-details/pharvaris-presents-data-supporting-ongoing-clinical-development

Pharvaris Presents Data Supporting Ongoing Clinical ... Deucrictibant data shows single-dose durability without symptom reoccurrence in the majority of HAE attacks treated; First-ever bradykinin ...

7. ir.pharvaris.com ir.pharvaris.com/static-files/ed6a506c-fa37-4aa1-92a2-75e34079fac6

Long-Term Safety and Efficacy of Oral Deucrictibant for ... 4. HAE, hereditary angioedema; IR, immediate-release; XR, extended-release. ... deucrictibant extended-release (XR) tablet is self-administered.

8. jacionline.org jacionline.org/article/S0091-6749(25)00889-9/fulltext

Long-term prophylactic treatment with deucrictibant for ... Deucrictibant extended-release tablet effectively prevented angioedema attacks in patients with AAE-C1INH, with no safety concerns. Key ...

9. clinicaltrials.gov clinicaltrials.gov/study/NCT06679881

NCT06679881 | Long-Term, Open-label Study of Oral ... Long-Term, Open-label Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE ...

10. sciencedirect.com sciencedirect.com/science/article/abs/pii/S1081120625005307

r087 sustained therapeutic exposure with once-daily oral ... A single oral deucrictibant XR tablet resulted in sustained exposure for at least 24 hours, which was not altered by repeat dosing, supporting once-daily dosing ...