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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05389449
Recruitment Status : Not yet recruiting
First Posted : May 25, 2022
Last Update Posted : May 25, 2022
Sponsor:
Information provided by (Responsible Party):
Alexion Pharmaceuticals

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.

Condition or disease Intervention/treatment Phase
Paroxysmal Nocturnal Hemoglobinuria Drug: Danicopan Phase 3

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 100 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Estimated Study Start Date : May 16, 2022
Estimated Primary Completion Date : December 25, 2025
Estimated Study Completion Date : December 25, 2025


Arm Intervention/treatment
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 Name: ALXN2040, ACH-0144471




Primary Outcome Measures :
  1. Participants Experiencing Treatment-emergent Adverse Events (TEAEs) And Serious TEAEs [ Time Frame: Up to 3 years ]

Secondary Outcome Measures :
  1. Change In Hemoglobin Values [ Time Frame: Up to 3 years ]
  2. Change In Absolute Reticulocyte Count [ Time Frame: Up to 3 years ]
  3. Change In Lactate Dehydrogenase (LDH) [ Time Frame: Up to 3 years ]
  4. Proportion Of Participants With LDH ≤ 1.5 × Upper Limit Of Normal [ Time Frame: Up to 3 years ]
  5. Proportion Of Participants With Transfusion Avoidance [ Time Frame: Up to 3 years ]
  6. 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.

  7. 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).

  8. Participants Experiencing TEAEs Leading To Discontinuation [ Time Frame: Up to 3 years ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05389449


Contacts
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Contact: Alexion Pharmaceuticals, Inc. +1 855-752-2356 clinicaltrials@alexion.com

Sponsors and Collaborators
Alexion Pharmaceuticals
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Responsible Party: Alexion Pharmaceuticals
ClinicalTrials.gov Identifier: NCT05389449    
Other Study ID Numbers: ALXN2040-PNH-303
2021-004253-22 ( EudraCT Number )
First Posted: May 25, 2022    Key Record Dates
Last Update Posted: May 25, 2022
Last Verified: May 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
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.
Supporting Materials: Study Protocol
Clinical Study Report (CSR)

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Alexion Pharmaceuticals:
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Extravascular Hemolysis (EVH)
Factor D inhibitor
Complement
Danicopan
ALXN2040
C5 inhibitor
Additional relevant MeSH terms:
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Hemoglobinuria
Hemoglobinuria, Paroxysmal
Proteinuria
Urination Disorders
Urologic Diseases
Urological Manifestations
Anemia, Hemolytic
Anemia
Hematologic Diseases
Myelodysplastic Syndromes
Bone Marrow Diseases