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NCT05982938 Clinical Trial

Danicopan Early Access Program

Paroxysmal Nocturnal Hemoglobinuria Clinical Trial

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)

Clinical Trial Details — Status: Available

Administrative data
NCT number NCT05982938
Other study ID # D7332R00001
Secondary ID
Status Available
Phase
First received
Last updated

Study information
Verified date December 2023
Source Alexion Pharmaceuticals, Inc.
Contact Alexion Pharmaceuticals, Inc.
Phone 1-855-752-2356
Email clinicaltrials@alexion.com
Is FDA regulated No
Health authority
Study type Expanded Access

Clinical Trial 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.

Recruitment information / eligibility
Status Available
Enrollment 0
Est. completion date
Est. primary completion date
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility Key Inclusion Criteria - Male or female, aged 18 years and older - Diagnosed with PNH with clinically significant EVH as defined by anemia (Hgb = 9.5 g/dL) with absolute reticulocyte count = 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. - Vaccinated against N meningitidis. Key Exclusion Criteria - 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.

Study Design

Related Conditions & MeSH terms

Intervention

Drug:
• Danicopan
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.

Locations
Country Name City State
n/a

Sponsors (1)
Lead Sponsor Collaborator
Alexion Pharmaceuticals, Inc.
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