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Danicopan Early Access Program

Primary Purpose

Paroxysmal Nocturnal Hemoglobinuria, PNH, Extravascular Hemolysis

Status
Available
Phase
Locations
Study Type
Expanded Access
Intervention
Danicopan
Sponsored by
Alexion
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for Paroxysmal Nocturnal Hemoglobinuria focused on measuring paroxysmal nocturnal hemoglobinuria, PNH, component 5 inhibitors, C5i, intravascular hemolysis, IVH, anemia, EVH, extravascular hemolysis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Male or female, aged 18 years and older at the time of providing informed consent. Diagnosed with PNH with clinically significant EVH as defined by anemia (Hgb ≤ 9.5 g/dL) with absolute reticulocyte count > ULN).≥ 120 × 109/L. Currently receiving treatment with SOLIRIS® or ULTOMIRIS®.® for at least 6 months prior to initiation of danicopan treatment. Platelet count ≥ 30,000/µL without the need for platelet transfusions. Absolute neutrophil counts ≥ 500/µL. Have failed, not tolerated, or are unable to receive other approved treatments. In the Treating Physician's opinion, the potential benefits of treatment with danicopan outweigh the potential risks for the participant. Vaccinated against N meningitidis. Participants who initiate danicopan less than 2 weeks after receiving a meningococcal vaccine must receive treatment with appropriate prophylactic antibiotics from the first day of danicopan treatment until 2 weeks after vaccination. Female participants of childbearing potential agree to use effective or highly effective contraception methods during treatment with danicopan and until 3 days after discontinuation. Exclusion Criteria: In the opinion of the Treating Physician, the participant is eligible for and able to participate in an ongoing clinical trial of danicopan or a comparable treatment. Taking of disallowed medications. Participant is unwilling or unable to comply with the Treating Physician's treatment plan related to this EAP for any reason. Known hypersensitivity to danicopan or any of its excipients, or any of the ingredients contained in danicopan. Participants who are nursing or pregnant (or women who are planning to become pregnant during treatment with danicopan). Laboratory abnormalities at screening, as follows: Alanine aminotransferase (ALT) > 2 × upper limit of normal (ULN) Direct bilirubin > 2 × ULN, with the exception of: participants with increased bilirubin due to EVH in the opinion of the Treating Physician or participants with documented Gilbert's syndrome Active bacterial or viral infection, a body temperature > 38°C on 2 consecutive daily measures, evidence of other infection, or history of any febrile illness within 14 days prior to initiating danicopan treatment.

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    August 1, 2023
    Last Updated
    August 1, 2023
    Sponsor
    Alexion
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    1. Study Identification

    Unique Protocol Identification Number
    NCT05982938
    Brief Title
    Danicopan Early Access Program
    Official Title
    Early Access Program (EAP) for Danicopan as Add-on Treatment to SOLIRIS® or ULTOMIRIS® in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Clinically Significant Extravascular Hemolysis (csEVH)
    Study Type
    Expanded Access

    2. Study Status

    Record Verification Date
    July 2023
    Overall Recruitment Status
    Available
    Study Start Date
    undefined (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    undefined (undefined)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Alexion

    4. Oversight

    5. Study Description

    Brief Summary
    This is an EAP, designed to provide early access to danicopan for participants with PNH experiencing clinically significant EVH who have failed, not tolerated, or are unable to receive other approved treatments; in the Treating Physician's opinion, the participant is not eligible for or is not able to participate in an ongoing clinical trial of danicopan or a comparable treatment; and according to the Treating Physician, the benefit of danicopan treatment outweighs the potential risks.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Paroxysmal Nocturnal Hemoglobinuria, PNH, Extravascular Hemolysis
    Keywords
    paroxysmal nocturnal hemoglobinuria, PNH, component 5 inhibitors, C5i, intravascular hemolysis, IVH, anemia, EVH, extravascular hemolysis

    7. Study Design

    8. Arms, Groups, and Interventions

    Intervention Type
    Drug
    Intervention Name(s)
    Danicopan
    Other Intervention Name(s)
    ALXN2040
    Intervention Description
    Participants will receive 150 mg danicopan three times per day (TID) orally as an add-on to a background C5i therapy (SOLIRIS® or ULTOMIRIS®). Participants may be dose-escalated to 200 mg TID based on safety and clinical effect.

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    18 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    Inclusion Criteria: Male or female, aged 18 years and older at the time of providing informed consent. Diagnosed with PNH with clinically significant EVH as defined by anemia (Hgb ≤ 9.5 g/dL) with absolute reticulocyte count > ULN).≥ 120 × 109/L. Currently receiving treatment with SOLIRIS® or ULTOMIRIS®.® for at least 6 months prior to initiation of danicopan treatment. Platelet count ≥ 30,000/µL without the need for platelet transfusions. Absolute neutrophil counts ≥ 500/µL. Have failed, not tolerated, or are unable to receive other approved treatments. In the Treating Physician's opinion, the potential benefits of treatment with danicopan outweigh the potential risks for the participant. Vaccinated against N meningitidis. Participants who initiate danicopan less than 2 weeks after receiving a meningococcal vaccine must receive treatment with appropriate prophylactic antibiotics from the first day of danicopan treatment until 2 weeks after vaccination. Female participants of childbearing potential agree to use effective or highly effective contraception methods during treatment with danicopan and until 3 days after discontinuation. Exclusion Criteria: In the opinion of the Treating Physician, the participant is eligible for and able to participate in an ongoing clinical trial of danicopan or a comparable treatment. Taking of disallowed medications. Participant is unwilling or unable to comply with the Treating Physician's treatment plan related to this EAP for any reason. Known hypersensitivity to danicopan or any of its excipients, or any of the ingredients contained in danicopan. Participants who are nursing or pregnant (or women who are planning to become pregnant during treatment with danicopan). Laboratory abnormalities at screening, as follows: Alanine aminotransferase (ALT) > 2 × upper limit of normal (ULN) Direct bilirubin > 2 × ULN, with the exception of: participants with increased bilirubin due to EVH in the opinion of the Treating Physician or participants with documented Gilbert's syndrome Active bacterial or viral infection, a body temperature > 38°C on 2 consecutive daily measures, evidence of other infection, or history of any febrile illness within 14 days prior to initiating danicopan treatment.
    Central Contact Person:
    First Name & Middle Initial & Last Name or Official Title & Degree
    AstraZeneca Clinical Study Information Center
    Phone
    1-877-240-9479
    Email
    information.center@astrazeneca.com

    12. IPD Sharing Statement

    Learn more about this trial

    Danicopan Early Access Program

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