Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry Data

Part of paid clinical trials in Boston, Massachusetts.

Sponsor
Alexion Pharmaceuticals, Inc.
Study ID
NCT07413250
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 treated with danicopan as an add-on therapy.
  • Soliris/Ultomiris — DRUG
    Participants treated with Soliris/Ultomiris monotherapy.

Study Details

This is a noninterventional registry-based cohort study utilizing data collected on danicopan-treated patients with paroxysmal nocturnal hemoglobinuria (PNH) through the International PNH Interest Group (IPIG) PNH registry. The primary objectives seek to characterize the long-term safety profile of danicopan as add-on therapy to ravulizumab/eculizumab in participants with PNH.

Key Dates

Start date
Jan 14, 2026
Status verified
Jan 2026
Primary completion
Jan 15, 2030
Completion
Jan 15, 2030

Study Design

Enrollment
50 participants (estimated)

Arms

  • Arm: Cohort 1
    Participants ever-treated with danicopan as add-on therapy to ravulizumab/eculizumab on or after IPIG PNH Registry enrollment.
  • Arm: Cohort 2
    Participants initiating treatment with Ultomiris or Soliris on or after IPIG or Alexion International PNH Registry enrollment and without any danicopan treatment experience during follow-up.

Primary Outcome Measure

Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: From initiation of danicopan until discontinuation + 7 days, or end of follow-up, up to 5 years ]

Locations (1)

FacilityCityStateZIPSite coordinators
Clinical Research SiteBostonMassachusetts02210-

Find similar trials in Boston, MA

By research site
Clinical Research Site· Boston, MA

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