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
• Status: Available

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
• 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
• 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.

Related Conditions & MeSH terms

• Hemoglobinuria
• Hemoglobinuria, Paroxysmal
• Hemolysis
• Paroxysmal Nocturnal Hemoglobinuria
• PNH

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.