Clinical Trials Logo

Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT06343779
Other study ID # PHA022121-C306
Secondary ID
Status Recruiting
Phase Phase 3
First received
Last updated
Start date February 26, 2024
Est. completion date March 2026

Study information

Verified date April 2024
Source Pharvaris Netherlands B.V.
Contact Pharvaris Clinical Team
Phone +31 (71) 203-6410
Email clinicaltrials@pharvaris.com
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

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.


Description:

The study consists of a Screening Phase during which eligibility is confirmed, a Treatment Phase in which participants will be randomized and receive double blinded study drug to treat 2 qualifying HAE attacks (i.e., 2 Treatment Periods within the Treatment Phase), and an End-of-Study Follow-up Phase after the second attack treated with study drug. In addition, for adolescent participants (age ≥12 to <18 years), PK samples are collected after administration of deucrictibant at Day 1 in a non-attack state.


Recruitment information / eligibility

Status Recruiting
Enrollment 120
Est. completion date March 2026
Est. primary completion date March 2026
Accepts healthy volunteers No
Gender All
Age group 12 Years to 75 Years
Eligibility Inclusion Criteria: 1. Provision of written informed consent/assent. 2. Male or female, aged =12 to =75 years at the time of providing written informed consent/assent. 3. Diagnosis of HAE-1/2. 4. History of at least 2 HAE attacks in the last 3 months before screening. 5. Experience with using standard-of-care treatment to effectively manage on-demand treatment for HAE attacks. 6. Participants on long-term prophylactic therapy with plasma-derived C1-INH (danazol, anti-fibrinolytics, berotralstat, or lanadelumab) must be on a stable dose and regimen and intend to remain on the same dose for 6 months before screening and the duration of the study. 7. Capable of recording, without assistance, electronic HAE diary and ePRO data using an electronic device. 8. For adolescent participants aged =12 and <18 years of age: body weight >40 kg. 9. Female participants of childbearing potential must agree to the protocol specified pregnancy testing and contraception methods. Exclusion Criteria: 1. Any female who is pregnant, plans to become pregnant, or is breastfeeding. 2. Any diagnosis of angioedema other than HAE-1/2. 3. Any clinically significant comorbidity or systemic dysfunction that would interfere with the participant's safety or ability to participate in the study. 4. Use of attenuated androgens for short-term prophylaxis within the last 30 days before the time of randomization. 5. Abnormal hepatic function. 6. Abnormal renal function (eGFR <60 ml/min/1.73 m2). 7. History of alcohol or drug abuse within the previous year, or current evidence of substance dependence or abuse. 8. Has received prior on-demand HAE treatment with deucrictibant. 9. Currently participating in any other investigational drug study or receiving other investigational treatment within the last 30 days, or within 5 half-lives (whichever is longer) of the time of randomization. 10. Prior gene therapy for any indication at any time. 11. Use of concomitant medications that are strong inhibitors/inducers of CYP3A4 within the last 30 days, or within 5 half-lives (whichever is longer) of the time of randomization. 12. Known hypersensitivity to study drug or any of the excipients of study drug.

Study Design


Intervention

Drug:
Deucrictibant, Placebo
Deucrictibant Soft Capsules for Oral Use

Locations

Country Name City State
Puerto Rico Study Site San Juan
United States Study Site Birmingham Alabama
United States Study Site Chevy Chase Maryland
United States Study Site Colorado Springs Colorado
United States Study Site Dallas Texas
United States Study Site Little Rock Arkansas
United States Study Site Paradise Valley Arizona
United States Study Site Santa Monica California
United States Study Site Walnut Creek California

Sponsors (1)

Lead Sponsor Collaborator
Pharvaris Netherlands B.V.

Countries where clinical trial is conducted

United States,  Puerto Rico, 

Outcome

Type Measure Description Time frame Safety issue
Primary Time to onset of symptom relief, defined as Patient Global Impression of Change (PGI-C) rating of at least "a little better" for 2 consecutive timepoints within 12 hours post-treatment. The PGI-C (7-point scale) is used to evaluate the change in the HAE attack symptoms as compared to pre-treatment. Pre-treatment to 12 hours post-treatment.
Secondary Proportion of study drug-treated attacks achieving PGI-C rating of at least "a little better" at 4 hours post-treatment. The PGI-C (7-point scale) is used to evaluate the change in the HAE attack symptoms as compared to pre-treatment. Pre-treatment to 4 hours post-treatment.
Secondary Time to substantial symptom relief, defined as achieving PGI-C rating of at least "better" for 2 consecutive timepoints within 12 hours post-treatment. The PGI-C (7-point scale) is used to evaluate the change in the HAE attack symptoms as compared to pre-treatment. Pre-treatment to 12 hours post-treatment.
Secondary Time to substantial symptom relief by Patient Global Impression of Severity (PGI-S). Defined as achieving =1 point reduction in PGI-S (5-point scale) from pre-treatment for 2 consecutive timepoints within 12 hours post-treatment. Pre-treatment to 12 hours post-treatment.
Secondary Time to complete symptom resolution, defined as achieving PGI-S rating of "none" within 48 hours post-treatment. The PGI-S (5-point scale) is used to evaluate the severity of HAE attack symptoms. Pre-treatment to 48 hours post-treatment.
Secondary Time to End of Progression (EoP) in attack symptoms within 12 hours. EoP time defined as the earliest post-treatment timepoint after which all subsequent PGI-C ratings are stable or improved. Pre-treatment to 12 hours post-treatment.
Secondary Proportion of study drug-treated attacks requiring rescue medication within 24 hours post-treatment. Rescue medication is defined as the participant's usual acute on-demand HAE treatment taken if symptoms persist or progress after study drug administration. Pre-treatment to 24 hours post-treatment.
Secondary Proportion of attacks achieving symptom resolution. Defined as achieving PGI-S rating of "none" with one dose of study drug at 24 hours post-treatment. Pre-treatment to 24 hours post-treatment.
Secondary Time to substantial symptom relief by Angioedema Symptom Rating Scale (AMRA). Defined as a =50% reduction in AMRA composite score from pre-treatment for 2 consecutive timepoints within 12 hours post-treatment. Pre-treatment to 12 hours post-treatment.
Secondary Time to almost complete or complete symptom relief by AMRA. Defined as all item scores in AMRA having a value =10 for 2 consecutive timepoints within 24 hours post-treatment. Pre-treatment to 24 hours post-treatment.
Secondary Proportion of study drug-treated attacks reaching almost complete or complete symptom relief by AMRA. Defined as all item scores in AMRA having a value =10 at 24 hours post-treatment. Pre-treatment to 24 hours post-treatment.
Secondary Time to EoP in attack symptoms within 12 hours. Defined as the earliest post-treatment timepoint after which every individual AMRA item is stable or improved at all subsequent timepoints. Pre-treatment to 12 hours post-treatment.
See also
  Status Clinical Trial Phase
Enrolling by invitation NCT06007677 - A Long-term Study of STAR-0215 in Participants With Hereditary Angioedema Phase 2
Completed NCT00997204 - EASSI - Evaluation of the Safety of Self-Administration With Icatibant Phase 3
Completed NCT00438815 - Open-Label C1 Esterase Inhibitor (C1INH-nf) for the Treatment of Acute Hereditary Angioedema (HAE) Attacks Phase 3
Completed NCT00748202 - Berinert P Study of Subcutaneous Versus Intravenous Administration Phase 3
Completed NCT01426763 - A Study to Evaluate the Safety and Pharmacology of Subcutaneous Administration of CINRYZE With Recombinant Human Hyaluronidase Phase 2
Terminated NCT04091113 - Hereditary Angioedema Kininogen Assay
Completed NCT00432510 - Pharmacokinetics of C1 Esterase Inhibitor in Hereditary Angioedema Subjects Phase 1
Completed NCT03712228 - A Study to Investigate CSL312 in Subjects With Hereditary Angioedema (HAE) Phase 2
Active, not recruiting NCT05453968 - Berotralstat Treatment in Children With Hereditary Angioedema Phase 3
Recruiting NCT05505916 - An Open-label Extension Trial to Evaluate the Long-term Safety of KVD900 for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema (HAE) Phase 3
Recruiting NCT05511922 - PK Subtrial in Adolescent Patients With HAE Type I or II Participating in the KVD900-302 Trial Phase 3
Not yet recruiting NCT02159430 - Hereditary AngioEdema, Neurobiology and Psychopathology N/A
Completed NCT02303626 - 12-Week Safety and Efficacy Study of BCX4161 as an Oral Prophylaxis Against HAE Attacks Phase 2/Phase 3
Completed NCT01984788 - Safety and Efficacy of Oral BCX4161 as a Prophylactic Treatment for HAE Phase 2
Completed NCT04888650 - Assessment of the State of Health, Quality of Life and Expectations of Patients With Hereditary Angioedema
Completed NCT02448264 - First-in-Human Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of BCX7353 in Healthy Western and Japanese Volunteers Phase 1
Completed NCT05118958 - Phase 1 Crossover Study in Healthy Subjects to Evaluate the PK Profile of KVD824 Following Single and Multiple Doses of Modified Release (MR) Formulations Phase 1
Completed NCT06414252 - Social Evaluated Cold Pressor Test in Hereditary Angioedema Patients
Active, not recruiting NCT04739059 - Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema Attacks Phase 3
Completed NCT02819102 - An Open-label Drug-Drug Interaction Study to Evaluate the Effect of BCX7353 on Cytochrome P450 Enzyme Activity Using Probe Substrates Phase 1