Danicopan Early Access Program

Sponsor

Alexion (Industry)

Overall Status

Available

CT.gov ID

NCT05982938

Collaborator

(none)

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
Paroxysmal Nocturnal Hemoglobinuria PNH Extravascular Hemolysis	Drug: Danicopan

Study Design

Study Type:

Expanded Access

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)

Outcome Measures

Primary Outcome Measures

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts 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.