A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH

Paroxysmal Nocturnal Hemoglobinuria Clinical Trial

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05389449
• Other study ID #: ALXN2040-PNH-303
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: October 28, 2022
• Est. completion date: January 31, 2027

Study information

• Verified date: March 2024
• Source: Alexion Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 80
• Est. completion date: January 31, 2027
• Est. primary completion date: January 31, 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Hemoglobinuria
• Hemoglobinuria, Paroxysmal
• Paroxysmal Nocturnal Hemoglobinuria

Intervention

Drug:
• Danicopan
Oral tablet

Locations

Country	Name	City	State
Brazil	Research Site	Porto Alegre
Brazil	Research Site	Rio de Janeiro
Canada	Research Site	Toronto	Ontario
Czechia	Research Site	Brno
France	Research Site	Lille
France	Research Site	Paris
France	Research Site	Pessac
France	Research Site	Pierre Benite Cedex
Greece	Research Site	Athens
Greece	Research Site	Thessaloniki
Israel	Research Site	Haifa
Israel	Research Site	Jerusalem
Italy	Research Site	Avellino
Italy	Research Site	Bassano del Grappa
Italy	Research Site	Firenze
Italy	Research Site	Milano
Italy	Research Site	Reggio Calabria
Italy	Research Site	Roma
Japan	Research Site	Bunkyo-Ku
Japan	Research Site	Kyoto-shi
Japan	Research Site	Osaka-shi
Japan	Research Site	Shibuya-ku
Japan	Research Site	Tsukuba-shi
Korea, Republic of	Research Site	Seoul
Korea, Republic of	Research Site	Seoul
Korea, Republic of	Research Site	Suwon
Malaysia	Research Site	Kota Kinabalu
Malaysia	Research Site	Kuching
Malaysia	Research Site	Miri
Poland	Research Site	Gdansk
Spain	Research Site	Badalona
Spain	Research Site	Barcelona
Spain	Research Site	Majadahonda
Spain	Research Site	Sevilla
Thailand	Research Site	Bangkok
United Kingdom	Research Site	Airdrie
United Kingdom	Research Site	Leeds
United Kingdom	Research Site	London
United States	Research Site	Baltimore	Maryland
United States	Research Site	Chicago	Illinois
United States	Research Site	Cleveland	Ohio
United States	Research Site	Dallas	Texas
United States	Research Site	Kansas City	Missouri
United States	Research Site	Long Island City	New York
United States	Research Site	Los Angeles	California

Sponsors (1)

Lead Sponsor	Collaborator
Alexion Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  Brazil,  Canada,  Czechia,  France,  Greece,  Israel,  Italy,  Japan,  Korea, Republic of,  Malaysia,  Poland,  Spain,  Thailand,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Participants Experiencing Treatment-emergent Adverse Events (TEAEs) And Serious TEAEs		Up to 3 years
Secondary	Change In Hemoglobin Values		Up to 3 years
Secondary	Proportion of patients with Hgb increase = 2g/dL in the absence of transfusion over time		Up to 3 years
Secondary	Change In Absolute Reticulocyte Count		Up to 3 years
Secondary	Change In Lactate Dehydrogenase (LDH)		Up to 3 years
Secondary	Proportion Of Participants With LDH = 1.5 × Upper Limit Of Normal		Up to 3 years
Secondary	Proportion Of Participants With Transfusion Avoidance		Up to 3 years
Secondary	Change In Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scores	Total scores range from 0 to 52, with higher scores indicating better QoL.	Up to 3 years
Secondary	Change In The European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire Core 30 Scale (QLQ-C30) Scores	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).	Up to 3 years
Secondary	Participants Experiencing TEAEs Leading To Discontinuation		Up to 3 years