Danicopan Early Access Program

NCT05982938 | Unknown

• 1 other identifier: D7332R00001
• Study Type: expanded_access
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Paroxysmal Nocturnal Hemoglobinuria; PNH; Extravascular Hemolysis

Keywords

paroxysmal nocturnal hemoglobinuria; PNH; component 5 inhibitors; C5i; intravascular hemolysis; IVH; anemia; EVH; extravascular hemolysis

Interventions

Danicopan DRUG

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.

Also known as: ALXN2040

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal; Hemolysis; Anemia

Interventions

danicopan

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Central Study Contacts

Alexion Pharmaceuticals, Inc. (Sponsor)

CONTACT

1-855-752-2356; clinicaltrials@alexion.com

Study Design

• Study Type: expanded access
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 1, 2023
• First Posted: August 9, 2023
• Last Updated: March 6, 2026

Record last verified: 2026-03