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

Part of paid clinical trials in Boston, Massachusetts.

Sponsor
Alexion Pharmaceuticals, Inc.
Study ID
NCT07413679
Status
Active Not Recruiting

Conditions

  • PNH
  • Paroxysmal Nocturnal Hemoglobinuria

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • Danicopan — DRUG
    Participants will receive Danicopan as an add-on therapy.

Study Details

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.

Key Dates

Start date
Aug 18, 2025
Status verified
Feb 2026
Primary completion
Jul 1, 2029
Completion
Jul 1, 2029

Study Design

Enrollment
50 participants (estimated)

Arms

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

Primary Outcome Measure

Number of Participants with Serious Adverse Events (SAEs) [ Time Frame: Up to approximately 5 years ]

Locations (1)

FacilityCityStateZIPSite coordinators
Alexion Pharmaceuticals, Inc. (Sponsor)BostonMassachusetts02210-

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Alexion Pharmaceuticals, Inc. (Sponsor)· Boston, MA

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