Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema

JRCT ID: jRCT2061240060

Registered date:02/10/2024

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Hereditary Angioedema
• Date of first enrollment: 02/10/2024
• Target sample size: 7
• 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,Israel,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 will be administered orally during HAE attacks.

Outcome(s)

Primary Outcome

- Treatment-emergent adverse events (TEAEs), treatment-related TEAEs, treatment-emergent serious adverse events (TESAEs), treatment-related TESAEs, and TEAEs leading to deucrictibant discontinuation
- Clinical laboratory tests
- Physical examination
- Vital signs
- ECG

Key inclusion & exclusion criteria

• Age minimum: >= 12age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1. Provision of written informed consent. If the participant is a minor(i.e., <18 years of age or as determined by local law), consent will be obtained from the participant's parent/legal guardian and written assent will be obtained from theparticipant, per country regulations.
2. For participants from Study C306, participant was randomized (and for adolescent participants 12 to <18 years received a dose of study drug in a non-attack state at Visit 1) and completed Study C306, with 2 attacks treated, or after closure of that study by the Sponsor.
3. Female participants of childbearing potential (or who become of childbearing potential during the study) must agree to the protocol-specified pregnancy testand to be abstinent from heterosexual intercourse or to use an acceptable contraception method from enrollment until 30 days after the last study drug administration.
4. In the opinion of the Investigator, the participant (and parent/caregiver for adolescent participants) is willing and able to comply with the protocol.

Exclude criteria

1. Any female who is pregnant, plans to become pregnant, or is breast-feeding.
2. Any other systemic disease (e.g., cardiovascular, gastrointestinal, renal, respiratory, neurological) or significant disease or disorder that, in the opinion of the Investigator, would interfere with the participant's safety or ability to participate in the study.
3. Participants who have recently used short or long-term HAE prophylaxis or ondemand HAE treatment will not be excluded from the study provided the following washout period is observed (i.e., study screening or enrollment/rollover should be delayed allowing for washout):
- For Part B:
- If a participant is receiving long-term prophylactic therapy with one of the following medications indicated for HAE: plasma-derived C1-INH, danazol at less than or equal to 200 mg/day, anti-fibrinolytics, berotralstat, or lanadelumab, they must be on a stable dose and regimen for at least 3 months before screening and intends to remain on the same dose for the duration of the study.
4. History of alcohol or drug abuse within the previous year, or current evidence of substance dependence or abuse.
5. Participation in any other investigational drug study (except with deucrictibant) currently, within the last 30 days prior to the first deucrictibant dose or within 5 half-lives of study drug at enrollment, whichever is longer.
6. Discontinued from parent study after enrollment for any study drug-related safety reason or non-compliance including significant protocol deviation.
7. Use of concomitant medications that are strong CYP3A4 inhibitors (e.g., clarithromycin, erythromycin, itraconazole, ketoconazole, ritonavir) or potent CYP3A4 inducers (e.g., phenytoin, rifampicin, St. John's Wort).

Related Information

• Primary Sponsor: Sun Justin
• Secondary ID(s): NCT05396105