Clinical trial NCT05389449: A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH
Official title: A Long-term Extension (LTE) Study to Characterize the Safety and Efficacy of Danicopan as an Add-on Therapy to a Complement Component 5 Inhibitor (C5i) in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Previously Treated With Danicopan in an Alexion-sponsored Clinical Study
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Paroxysmal Nocturnal Hemoglobinuria
Keywords: Paroxysmal Nocturnal Hemoglobinuria (PNH)
Extravascular Hemolysis (EVH)
Factor D inhibitor
Complement
Danicopan
ALXN2040
C5 inhibitor
Lead sponsor: Alexion Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2022-05-25
Enrollment: 80
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility 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.

Primary outcomes:
measure: Participants Experiencing Treatment-emergent Adverse Events (TEAEs) And Serious TEAEs
timeFrame: Up to 3 years
Secondary outcomes:
measure: Change In Hemoglobin Values
timeFrame: Up to 3 years
measure: Proportion of patients with Hgb increase ≥ 2g/dL in the absence of transfusion over time
timeFrame: Up to 3 years
measure: Change In Absolute Reticulocyte Count
timeFrame: Up to 3 years
measure: Change In Lactate Dehydrogenase (LDH)
timeFrame: Up to 3 years
measure: Proportion Of Participants With LDH ≤ 1.5 × Upper Limit Of Normal
timeFrame: Up to 3 years
measure: Proportion Of Participants With Transfusion Avoidance
timeFrame: Up to 3 years
measure: Change In Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scores
timeFrame: Up to 3 years
measure: Change In The European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire Core 30 Scale (QLQ-C30) Scores
timeFrame: Up to 3 years
measure: Participants Experiencing TEAEs Leading To Discontinuation
timeFrame: Up to 3 years
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Research Site
state: California
city: Chicago
country: United States
facility: Research Site
state: Illinois
city: Baltimore
country: United States
facility: Research Site
state: Maryland
city: Kansas City
country: United States
facility: Research Site
state: Missouri
city: Long Island City
country: United States
facility: Research Site
state: New York
city: Cleveland
country: United States
facility: Research Site
state: Ohio
city: Dallas
country: United States
facility: Research Site
state: Texas
city: Porto Alegre
country: Brazil
facility: Research Site
city: Rio de Janeiro
country: Brazil
facility: Research Site
city: Toronto
country: Canada
facility: Research Site
state: Ontario
city: Brno
country: Czechia
facility: Research Site
city: Lille
country: France
facility: Research Site
city: Paris
country: France
facility: Research Site
city: Pessac
country: France
facility: Research Site
city: Pierre-Benite
country: France
facility: Research Site
city: Athens
country: Greece
facility: Research Site
city: Thessaloniki
country: Greece
facility: Research Site
city: Haifa
country: Israel
facility: Research Site
city: Jerusalem
country: Israel
facility: Research Site
city: Avellino
country: Italy
facility: Research Site
city: Bassano del Grappa
country: Italy
facility: Research Site
city: Firenze
country: Italy
facility: Research Site
city: Milano
country: Italy
facility: Research Site
city: Reggio Calabria
country: Italy
facility: Research Site
city: Roma
country: Italy
facility: Research Site
city: Bunkyo-Ku
country: Japan
facility: Research Site
city: Kyoto-shi
country: Japan
facility: Research Site
city: Osaka-shi
country: Japan
facility: Research Site
city: Shibuya-ku
country: Japan
facility: Research Site
city: Tsukuba-shi
country: Japan
facility: Research Site
city: Seoul
country: Korea, Republic of
facility: Research Site
city: Seoul
country: Korea, Republic of
facility: Research Site
city: Suwon
country: Korea, Republic of
facility: Research Site
city: Kota Kinabalu
country: Malaysia
facility: Research Site
city: Kuching
country: Malaysia
facility: Research Site
city: Miri
country: Malaysia
facility: Research Site
city: Gdańsk
country: Poland
facility: Research Site
city: Badalona
country: Spain
facility: Research Site
city: Barcelona
country: Spain
facility: Research Site
city: Majadahonda
country: Spain
facility: Research Site
city: Sevilla
country: Spain
facility: Research Site
city: Bangkok
country: Thailand
facility: Research Site
city: Airdrie
country: United Kingdom
facility: Research Site
city: Leeds
country: United Kingdom
facility: Research Site
city: London
country: United Kingdom
facility: Research Site
Study officials:
References: