Long-term Safety of Danicopan: IPIG Registry-based Cohort Study

NCT07413679 | Active Not Recruiting FDA Drug

• 2 other identifiers: D7332R00004ALX-PNH-503
• Study Type: observational
• Target: 50
• Locations: 1 country
• Sites: 1

Brief Summary

This is a noninterventional cohort study using primary and secondary data from the International PNH Interest Group (IPIG) PNH registry. It is designed to characterize the long-term safety and tolerability of danicopan as add-on therapy to eculizumab or ravulizumab in adult participants with PNH.

Conditions

PNH; Paroxysmal Nocturnal Hemoglobinuria

Keywords

Paroxysmal Nocturnal Hemoglobinuria; PNH; Danicopan

Outcome Measures

Primary Outcomes (2)

• Number of Participants with Serious Adverse Events (SAEs)

  Up to approximately 5 years

• Number of Participants with Serious Infections Caused by Encapsulated Bacteria

  Up to approximately 5 years

Secondary Outcomes (2)

• Number of Participants Who Discontinue Study Treatment

  Up to approximately 5 years

• Frequency of Reasons for Discontinuation

  Up to approximately 5 years

Study Arms (1)

Danicopan

Participants with PNH treated with Danicopan as an add-on therapy to eculizumab or ravulizumab.

Drug: Danicopan

Interventions

Danicopan DRUG

Participants will receive Danicopan as an add-on therapy.

Danicopan

Eligibility Criteria

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

Study Population

The study population will consist of adult participants ≥ 18 years at danicopan treatment initiation (baseline) as add-on therapy to ravulizumab/eculizumab. Participants will have initiated treatment with danicopan on or after registry enrollment.

You may qualify if:

• Adult participants aged ≥ 18 years at danicopan treatment initiation (baseline) as add-on therapy to ravulizumab/eculizumab.
• Initiated treatment with danicopan on or after registry enrollment

You may not qualify if:

• Participants without known year of birth, sex, informed consent date, or treatment status of danicopan and ravulizumab and/or eculizumab treatment status.

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

Study Sites (1)

Alexion Pharmaceuticals, Inc. (Sponsor)

Boston, Massachusetts, 02210, United States

Location

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Interventions

danicopan

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 5 Years
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 10, 2026
• First Posted: February 17, 2026
• Study Start: August 18, 2025
• Primary Completion (Estimated): July 1, 2029
• Study Completion (Estimated): July 1, 2029
• Last Updated: February 17, 2026

Record last verified: 2026-02

Locations

US (1)