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

June 17, 2026 updated by: Alexion Pharmaceuticals, Inc.

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

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
80

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Brazil
      • Porto Alegre, Brazil, 90110-270
        • Research Site
      • Rio de Janeiro, Brazil, 20211-080
        • Research Site
  • Canada
    • Ontario
      • Toronto, Ontario, Canada, M5G 2C4
        • Research Site
  • Czechia
      • Brno, Czechia, 625 00
        • Research Site
  • France
      • Lille, France, 59037
        • Research Site
      • Paris, France, 75010
        • Research Site
      • Pessac, France, 33604
        • Research Site
      • Pierre-Bénite, France, 69495
        • Research Site
  • Greece
      • Athens, Greece, 11527
        • Research Site
      • Thessaloniki, Greece, 57010
        • Research Site
  • Israel
      • Haifa, Israel, 31048
        • Research Site
      • Jerusalem, Israel, 91120
        • Research Site
  • Italy
      • Avellino, Italy, 83100
        • Research Site
      • Bassano del Grappa, Italy, 36061
        • Research Site
      • Florence, Italy, 50134
        • Research Site
      • Milan, Italy, 20122
        • Research Site
      • Reggio Calabria, Italy, 89131
        • Research Site
      • Roma, Italy, 161
        • Research Site
  • Japan
      • Bunkyō City, Japan, 113 8603
        • Research Site
      • Kyoto, Japan, 605-0981
        • Research Site
      • Osaka, Japan, 530-8480
        • Research Site
      • Shibuya-ku, Japan, 150-8935
        • Research Site
      • Tsukuba, Japan, 305-8576
        • Research Site
  • Malaysia
      • Kota Kinabalu, Malaysia, 88586
        • Research Site
      • Kuching, Malaysia, 93200
        • Research Site
      • Miri, Malaysia, 98000
        • Research Site
  • Poland
      • Gdansk, Poland, 80-952
        • Research Site
  • South Korea
      • Seoul, South Korea, 03722
        • Research Site
      • Seoul, South Korea, 06591
        • Research Site
      • Suwon, South Korea, 16247
        • Research Site
  • Spain
      • Badalona, Spain, 8916
        • Research Site
      • Barcelona, Spain, 08036
        • Research Site
      • Majadahonda, Spain, 28222
        • Research Site
      • Seville, Spain, 41013
        • Research Site
  • Thailand
      • Bangkok, Thailand, 10330
        • Research Site
  • United Kingdom
      • Airdrie, United Kingdom, ML6 0JS
        • Research Site
      • Leeds, United Kingdom, LS9 7TF
        • Research Site
      • London, United Kingdom, SE5 9RS
        • Research Site
  • United States
    • California
      • Los Angeles, California, United States, 90089
        • Research Site
    • Illinois
      • Chicago, Illinois, United States, 60612
        • Research Site
    • Maryland
      • Baltimore, Maryland, United States, 21231
        • Research Site
    • Missouri
      • Kansas City, Missouri, United States, 64111
        • Research Site
    • New York
      • Long Island City, New York, United States, 11101
        • Research Site
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Research Site
    • Texas
      • Dallas, Texas, United States, 75390-9047
        • Research Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Danicopan
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.
Drug: Danicopan
Oral tablet
Other Names:
  • ALXN2040, ACH-0144471

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Participants Experiencing Treatment-emergent Adverse Events (TEAEs) And Serious TEAEs
Time Frame: Up to 3 years
Up to 3 years

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Change In Hemoglobin Values
Time Frame: Up to 3 years
Up to 3 years
Change In Absolute Reticulocyte Count
Time Frame: Up to 3 years
Up to 3 years
Change In Lactate Dehydrogenase (LDH)
Time Frame: Up to 3 years
Up to 3 years
Proportion Of Participants With LDH ≤ 1.5 × Upper Limit Of Normal
Time Frame: Up to 3 years
Up to 3 years
Proportion Of Participants With Transfusion Avoidance
Time Frame: Up to 3 years
Up to 3 years
Change In Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scores
Time Frame: Up to 3 years
Total scores range from 0 to 52, with higher scores indicating better QoL.
Up to 3 years
Change In The European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire Core 30 Scale (QLQ-C30) Scores
Time Frame: 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).
Up to 3 years
Participants Experiencing TEAEs Leading To Discontinuation
Time Frame: Up to 3 years
Up to 3 years
Proportion of patients with Hgb increase ≥ 2g/dL in the absence of transfusion over time
Time Frame: Up to 3 years
Up to 3 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Alexion Pharmaceuticals, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
October 28, 2022

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
August 24, 2026

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
August 24, 2026

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
May 20, 2022

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 20, 2022

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
May 25, 2022

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
June 18, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 17, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
June 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • ALXN2040-PNH-303
  • 2021-004253-22 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD 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.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • CSR

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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