Back to resultsA Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH
Primary Purpose
Paroxysmal Nocturnal Hemoglobinuria
Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Danicopan
Sponsored by
About this trial
This is an interventional treatment trial for Paroxysmal Nocturnal Hemoglobinuria focused on measuring Paroxysmal Nocturnal Hemoglobinuria (PNH), Extravascular Hemolysis (EVH), Factor D inhibitor, Complement, Danicopan, ALXN2040, C5 inhibitor
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.
Sites / Locations
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Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Danicopan
Arm Description
Participants will receive their last dose of danicopan from the parent study the night prior to Day 1 of this LTE study and will continue daily treatment with danicopan together with their background C5i therapy.
Outcomes
Primary Outcome Measures
Participants Experiencing Treatment-emergent Adverse Events (TEAEs) And Serious TEAEs
Secondary Outcome Measures
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 (LDH)
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 (FACIT)-Fatigue Scores
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
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
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05389449
Brief Title
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
2. Study Status
Record Verification Date
October 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
October 28, 2022 (Actual)
Primary Completion Date
February 26, 2027 (Anticipated)
Study Completion Date
February 26, 2027 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Alexion
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Paroxysmal Nocturnal Hemoglobinuria
Keywords
Paroxysmal Nocturnal Hemoglobinuria (PNH), Extravascular Hemolysis (EVH), Factor D inhibitor, Complement, Danicopan, ALXN2040, C5 inhibitor
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
90 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Danicopan
Arm Type
Experimental
Arm Description
Participants will receive their last dose of danicopan from the parent study the night prior to Day 1 of this LTE study and will continue daily treatment with danicopan together with their background C5i therapy.
Intervention Type
Drug
Intervention Name(s)
Danicopan
Other Intervention Name(s)
ALXN2040, ACH-0144471
Intervention Description
Oral tablet
Primary Outcome Measure Information:
Title
Participants Experiencing Treatment-emergent Adverse Events (TEAEs) And Serious TEAEs
Time Frame
Up to 3 years
Secondary Outcome Measure Information:
Title
Change In Hemoglobin Values
Time Frame
Up to 3 years
Title
Proportion of patients with Hgb increase ≥ 2g/dL in the absence of transfusion over time
Time Frame
Up to 3 years
Title
Change In Absolute Reticulocyte Count
Time Frame
Up to 3 years
Title
Change In Lactate Dehydrogenase (LDH)
Time Frame
Up to 3 years
Title
Proportion Of Participants With LDH ≤ 1.5 × Upper Limit Of Normal
Time Frame
Up to 3 years
Title
Proportion Of Participants With Transfusion Avoidance
Time Frame
Up to 3 years
Title
Change In Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scores
Description
Total scores range from 0 to 52, with higher scores indicating better QoL.
Time Frame
Up to 3 years
Title
Change In The European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire Core 30 Scale (QLQ-C30) Scores
Description
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).
Time Frame
Up to 3 years
Title
Participants Experiencing TEAEs Leading To Discontinuation
Time Frame
Up to 3 years
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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.
Facility Information:
Facility Name
Clinical Trial Site
City
Los Angeles
State/Province
California
ZIP/Postal Code
90033
Country
United States
Facility Name
Clinical Trial Site
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
Clinical Trial Site
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Facility Name
Clinical Trial Site
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64111
Country
United States
Facility Name
Clinical Trial Site
City
New York
State/Province
New York
ZIP/Postal Code
10065
Country
United States
Facility Name
Clinical Trial Site
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Facility Name
Clinical Trial Site
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390-8896
Country
United States
Facility Name
Clinical Trial Site
City
Porto Alegre
State/Province
RS
ZIP/Postal Code
90110-270
Country
Brazil
Facility Name
Clinical Trial Site
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G2C4
Country
Canada
Facility Name
Clinical Trial Site
City
Lille
ZIP/Postal Code
59037
Country
France
Facility Name
Clinical Trial Site
City
Paris
ZIP/Postal Code
75475
Country
France
Facility Name
Clinical Trial Site
City
Pessac
ZIP/Postal Code
33604
Country
France
Facility Name
Clinical Trial Site
City
Pierre Benite
ZIP/Postal Code
69495
Country
France
Facility Name
Clinical Trial Site
City
Avellino
State/Province
AV
ZIP/Postal Code
83100
Country
Italy
Facility Name
Clinical Trial Site
City
Milano
State/Province
Lombardia
ZIP/Postal Code
20122
Country
Italy
Facility Name
Clinical Trial Site
City
Rome
State/Province
RM
ZIP/Postal Code
00161
Country
Italy
Facility Name
Clinical Trial Site
City
Bassano del Grappa
ZIP/Postal Code
36061
Country
Italy
Facility Name
Clinical Trial Site
City
Firenze
ZIP/Postal Code
50134
Country
Italy
Facility Name
Clinical Trial Site
City
Reggio Calabria
ZIP/Postal Code
89133
Country
Italy
Facility Name
Clinical Trial Site
City
Tsukuba-shi
State/Province
Ibaraki
ZIP/Postal Code
305-8576
Country
Japan
Facility Name
Clinical Trial Site
City
Kyoto-shi
State/Province
Kyoto
ZIP/Postal Code
605-0981
Country
Japan
Facility Name
Clinical Trial Site
City
Osaka-shi
State/Province
Osaka
ZIP/Postal Code
530-8480
Country
Japan
Facility Name
Clinical Trial Site
City
Bunkyo-ku
State/Province
Tokyo
ZIP/Postal Code
113-8603
Country
Japan
Facility Name
Clinical Trial Site
City
Shibuya-ku
State/Province
Tokyo
ZIP/Postal Code
150-8935
Country
Japan
Facility Name
Clinical Trial Site
City
Fukuoka-shi
ZIP/Postal Code
812-8582
Country
Japan
Facility Name
Clinical Trial Site
City
Seoul
ZIP/Postal Code
03722
Country
Korea, Republic of
Facility Name
Clinical Trial Site
City
Seoul
ZIP/Postal Code
06591
Country
Korea, Republic of
Facility Name
Clinical Trial Site
City
Suwon-si
ZIP/Postal Code
16247
Country
Korea, Republic of
Facility Name
Clinical Trial Site
City
Kota Kinabalu
State/Province
Sabah
ZIP/Postal Code
88586
Country
Malaysia
Facility Name
Clinical Trial Site
City
Kuching
State/Province
Sarawak
ZIP/Postal Code
93586
Country
Malaysia
Facility Name
Clinical Trial Site
City
Miri
State/Province
Sarawak
ZIP/Postal Code
98000
Country
Malaysia
Facility Name
Clinical Trial Site
City
Gdansk
State/Province
Pomorskie
ZIP/Postal Code
80-214
Country
Poland
Facility Name
Clinical Trial Site
City
Sevilla
State/Province
Andalucia
ZIP/Postal Code
41013
Country
Spain
Facility Name
Clinical Trial Site
City
Barcelona
State/Province
Cataluña
ZIP/Postal Code
08036
Country
Spain
Facility Name
Clinical Trial Site
City
Barcelona
State/Province
Cataluña
ZIP/Postal Code
08916
Country
Spain
Facility Name
Clinical Trial Site
City
Madrid
ZIP/Postal Code
28222
Country
Spain
Facility Name
Clinical Trial Site
City
Bangkok
ZIP/Postal Code
10330
Country
Thailand
Facility Name
Clinical Trial Site
City
Leeds
State/Province
West Yorkshire
ZIP/Postal Code
LS9 7TF
Country
United Kingdom
Facility Name
Clinical Trial Site
City
Airdrie
ZIP/Postal Code
ML6 0JS
Country
United Kingdom
Facility Name
Clinical Trial Site
City
London
ZIP/Postal Code
SE5 9RS
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing Plan Description
Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.
Learn more about this trial
A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH
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