Paroxysmal Nocturnal Hemoglobinuria
Conditions
Keywords
Paroxysmal Nocturnal Hemoglobinuria (PNH), Extravascular Hemolysis (EVH), Factor D inhibitor, Complement, Danicopan, ALXN2040, C5 inhibitor
Brief summary
This is a single-arm long-term extension study that will enroll participants with PNH who have completed participation in Alexion-sponsored clinical studies with danicopan as an add on therapy to a C5i.
Detailed description
The total duration of the study will be up to 3 years. Eligible participants must complete all study assessments on the parent protocol before starting this study. All participants entering this study will receive danicopan as an add-on to a background C5i therapy. The only allowed C5i therapies are eculizumab and ravulizumab.
Interventions
DRUGDanicopan
Oral tablet
Sponsors
Alexion Pharmaceuticals, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * All participants who completed their participation in an Alexion sponsored clinical study with danicopan as an add on to a C5i treatment. * Patient is capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form and in this protocol. * Documentation of vaccination for Neisseria meningitidis: All participants must be revaccinated as per national vaccination guidelines or local practice for vaccination use with complement inhibitors. Key
Exclusion criteria
* Any medical condition (for example, cardiac, pulmonary, renal, oncologic, or psychiatric) that, in the opinion of the Investigator, might interfere with participation in the study, pose any added risk to the participant, or confound the assessment of the participant. * Patient has been permanently discontinued from danicopan in the parent study for any reason other than enrollment into this LTE study. * Female participants who are pregnant, breastfeeding, or intending to conceive during the course of the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Participants Experiencing Treatment-emergent Adverse Events (TEAEs) And Serious TEAEs | Up to 3 years |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change In Hemoglobin Values | Up to 3 years | — |
| Proportion of patients with Hgb increase ≥ 2g/dL in the absence of transfusion over time | Up to 3 years | — |
| Change In Absolute Reticulocyte Count | Up to 3 years | — |
| Change In Lactate Dehydrogenase (LDH) | Up to 3 years | — |
| Proportion Of Participants With LDH ≤ 1.5 × Upper Limit Of Normal | Up to 3 years | — |
| Proportion Of Participants With Transfusion Avoidance | Up to 3 years | — |
| Change In Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scores | Up to 3 years | Total scores range from 0 to 52, with higher scores indicating better QoL. |
| Change In The European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire Core 30 Scale (QLQ-C30) Scores | Up to 3 years | Thirty questions related to QoL, with the first 28 questions scored on a 4-point scale (1 = not at all to 4 = very much) and the final 2 questions that probe the participant's overall health and QoL scored on a scale of 1 (very poor) to 7 (excellent). |
| Participants Experiencing TEAEs Leading To Discontinuation | Up to 3 years | — |
Countries
Brazil, Canada, Czechia, France, Greece, Israel, Italy, Japan, Malaysia, Poland, South Korea, Spain, Thailand, United Kingdom, United States
Outcome results
None listed