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Danicopan LTE in PNH
NCT05389449Phase 3
Key facts
- Status
- Active, not recruiting
- Start
- Oct 2022
- Primary completion
- Aug 2026
- Countries
- 15
- Sponsor
- Alexion Pharmaceuticals
- Modality
- Small Molecules
- Drugs
- danicopan
- Conditions
- paroxysmal nocturnal hemoglobinuria (PNH)
01
Overview
Phase 3, open-label, single-arm trial of danicopan in paroxysmal nocturnal hemoglobinuria (PNH), sponsored by Alexion Pharmaceuticals. Primary endpoint: Treatment-emergent Adverse Events and Serious TEAEs up to 3 years. Started Oct 2022; status: active, not recruiting.
02
Design
1 arm- Study design
- Non-randomizedOpen-labelSingle group
Danicopan
Participants will receive daily treatment with danicopan together with their background C5i therapy.
03
Who it enrols
Participants with PNH who completed an Alexion-sponsored clinical study with danicopan as an add-on to a C5i, are vaccinated for Neisseria meningitidis, and are not pregnant or breastfeeding.
04
Endpoints
Primary
Treatment-emergent Adverse Events and Serious TEAEs
Secondary
Change In Hemoglobin Values
Proportion of patients with Hgb increase ≥ 2g/dL in the absence of transfusion over time
Change In Absolute Reticulocyte Count
Change In Lactate Dehydrogenase
Proportion Of Participants With LDH ≤ 1.5 × Upper Limit Of Normal
Proportion Of Participants With Transfusion Avoidance
Change In Functional Assessment of Chronic Illness Therapy-Fatigue Scores
Change In The European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 Scale Scores
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