Hereditary Angioedema Clinical Trial
— RAPIDe-3Official title:
A Phase 3, Randomized, Double-blind, Placebo-controlled, Cross-over Study of Oral Deucrictibant Soft Capsule for On-Demand Treatment of Attacks in Adolescents and Adults With Hereditary Angioedema
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.
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. |
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 |
Lead Sponsor | Collaborator |
---|---|
Pharvaris Netherlands B.V. |
United States, Puerto Rico,
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. |
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