A Phase 3 Open-Label Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Significant Extravascular Hemolysis

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Brief summary

Change from Baseline in Hgb at Week 12

Detailed description

• PK parameters (Ctrough, Cmax, and accumulation ratio), • Transfusion avoidance, defined as participants who remain transfusion-free and do not require a transfusion as per protocol-specified guidelines through Weeks 12 and 24, • Change from Baseline in absolute reticulocyte count at Weeks 12 and 24, • Change from Baseline in PedsQL Generic Core Scales score at Weeks 12 and 24, • Acceptability and palatability questionnaire scores, • Change from Baseline in pediatric FACIT-Fatigue scores at Weeks 12 and 24, • Change from Baseline in Hgb at Week 24, • Change from Baseline in PD parameters (such as AP activity, Bb) during Treatment Period and LTE, • Incidence of TEAEs, SAEs, laboratory abnormalities, and events leading to discontinuation

Related papers

No related papers found yet.

Interventions

DRUGALXN 2040

Eligibility

Sex/Gender

All

Age

0 Years to 17 Years

Design outcomes

Primary

Measure	Time frame
Change from Baseline in Hgb at Week 12	—

Secondary

Measure	Time frame
• PK parameters (Ctrough, Cmax, and accumulation ratio), • Transfusion avoidance, defined as participants who remain transfusion-free and do not require a transfusion as per protocol-specified guidelines through Weeks 12 and 24, • Change from Baseline in absolute reticulocyte count at Weeks 12 and 24, • Change from Baseline in PedsQL Generic Core Scales score at Weeks 12 and 24, • Acceptability and palatability questionnaire scores, • Change from Baseline in pediatric FACIT-Fatigue scores at Weeks 12 and 24, • Change from Baseline in Hgb at Week 24, • Change from Baseline in PD parameters (such as AP activity, Bb) during Treatment Period and LTE, • Incidence of TEAEs, SAEs, laboratory abnormalities, and events leading to discontinuation	—

Measure

Time frame

• PK parameters (Ctrough, Cmax, and accumulation ratio), • Transfusion avoidance, defined as participants who remain transfusion-free and do not require a transfusion as per protocol-specified guidelines through Weeks 12 and 24, • Change from Baseline in absolute reticulocyte count at Weeks 12 and 24, • Change from Baseline in PedsQL Generic Core Scales score at Weeks 12 and 24, • Acceptability and palatability questionnaire scores, • Change from Baseline in pediatric FACIT-Fatigue scores at Weeks 12 and 24, • Change from Baseline in Hgb at Week 24, • Change from Baseline in PD parameters (such as AP activity, Bb) during Treatment Period and LTE, • Incidence of TEAEs, SAEs, laboratory abnormalities, and events leading to discontinuation

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Countries

France

Outcome results

None listed