Study of Oral Deucrictibant Soft Capsule for On-Demand Treatment of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema

JRCT ID: jRCT2061240048

Registered date:31/08/2024

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Hereditary Angioedema
• Date of first enrollment: 31/08/2024
• Target sample size: 5
• Countries of recruitment: Argentina,Japan,Australia,Japan,Austria,Japan,Brazil,Japan,Bulgaria,Japan,Canada,Japan,Colombia,Japan,Czech Republic,Japan,France,Japan,Germany,Japan,Hong Kong,Japan,Hungary,Japan,Ireland,Japan,Italy,Japan,Netherlands,Japan,North Macedonia,Japan,Poland,Japan,Puerto Rico,Japan,Romania,Japan,Saudi Arabia,Japan,Singapore,Japan,Slovakia,Japan,South Africa,Japan,South Korea,Japan,Spain,Japan,Sweden,Japan,Turkey,Japan,United Kingdom,Japan,United States,Japan
• Study type: Interventional
• Intervention(s): Deucrictibant 20 mg or placebo will be administered orally during 2 HAE attacks as crossover treatment.

Outcome(s)

• Primary Outcome: 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.

Key inclusion & exclusion criteria

• Age minimum: >= 12age old
• Age maximum: <= 75age old
• Gender: Both

Include 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. OR, Participant has stopped using plasma-derived C1-INH (danazol, anti-fibrinolytics, berotralstat) at least 2 weeks or lanadelumab at least 10 weeks before screening.
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.

Exclude 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.

Related Information

• Primary Sponsor: Yu Ming
• Secondary ID(s): NCT06343779