A Long-term, Open-label Study to Evaluate the Safety and Efficacy of Orally Administered Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults with Hereditary Angioedema

Conditions

Hereditary Angioedema

Brief summary

Treatment-emergent adverse events (TEAEs), including serious adverse events (SAEs), adverse events of special interest (AESIs), and TEAEs leading to study drug discontinuation, Clinical laboratory tests, Vital signs, Electrocardiogram (ECG) parameters

Detailed description

Time-normalized (per 4 weeks) number of Investigator-confirmed HAE attacks during the Treatment Period, Time-normalized number of Investigator-confirmed HAE attacks treated with on-demand medication during the Treatment Period, Time-normalized number of Investigator-confirmed moderate or severe HAE attacks during Treatment Period, Time-normalized number of Investigator-confirmed severe HAE attacks during the Treatment Period, Proportion of time without angioedema symptoms during the Treatment Period, Angioedema Quality of Life (AE-QoL) questionnaire, Patient Global Assessment of Change (PGA-Change), Angioedema Control Test 4-week version (AECT-4wk), Work Productivity and Activity Impairment Questionnaire: Specific Health Problem (WPAI-SHP), Abbreviated Treatment Satisfaction Questionnaire for Medication (TSQM-9), Deucrictibant plasma concentration pre-dose (Ctrough)

Related papers

No related papers found yet.

Interventions

DRUGDeucrictibant (PHA-022121)

Eligibility

Sex/Gender

All

Age

0 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Treatment-emergent adverse events (TEAEs), including serious adverse events (SAEs), adverse events of special interest (AESIs), and TEAEs leading to study drug discontinuation, Clinical laboratory tests, Vital signs, Electrocardiogram (ECG) parameters	—

Secondary

Measure	Time frame
Time-normalized (per 4 weeks) number of Investigator-confirmed HAE attacks during the Treatment Period, Time-normalized number of Investigator-confirmed HAE attacks treated with on-demand medication during the Treatment Period, Time-normalized number of Investigator-confirmed moderate or severe HAE attacks during Treatment Period, Time-normalized number of Investigator-confirmed severe HAE attacks during the Treatment Period, Proportion of time without angioedema symptoms during the Treatment Period, Angioedema Quality of Life (AE-QoL) questionnaire, Patient Global Assessment of Change (PGA-Change), Angioedema Control Test 4-week version (AECT-4wk), Work Productivity and Activity Impairment Questionnaire: Specific Health Problem (WPAI-SHP), Abbreviated Treatment Satisfaction Questionnaire for Medication (TSQM-9), Deucrictibant plasma concentration pre-dose (Ctrough)	—

Measure

Time frame

Time-normalized (per 4 weeks) number of Investigator-confirmed HAE attacks during the Treatment Period, Time-normalized number of Investigator-confirmed HAE attacks treated with on-demand medication during the Treatment Period, Time-normalized number of Investigator-confirmed moderate or severe HAE attacks during Treatment Period, Time-normalized number of Investigator-confirmed severe HAE attacks during the Treatment Period, Proportion of time without angioedema symptoms during the Treatment Period, Angioedema Quality of Life (AE-QoL) questionnaire, Patient Global Assessment of Change (PGA-Change), Angioedema Control Test 4-week version (AECT-4wk), Work Productivity and Activity Impairment Questionnaire: Specific Health Problem (WPAI-SHP), Abbreviated Treatment Satisfaction Questionnaire for Medication (TSQM-9), Deucrictibant plasma concentration pre-dose (Ctrough)

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Countries

Austria, Bulgaria, France, Germany, Hungary, Ireland, Italy, Poland, Romania, Slovakia, Spain

Outcome results

None listed