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NCT07413250Active, not recruiting

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

A Study to Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of International PNH Interest Group (IPIG)-Registry Data

Source: ClinicalTrials.gov · Last updated 17 Feb 2026

Brief summary (registry, in English)

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.

Status (registry)
Active, not recruiting
Study type
Observational
Enrollment
50 (estimated)
First submitted
10 Feb 2026
Start date
14 Jan 2026
Primary completion date
15 Jan 2030
Lead sponsor
Alexion Pharmaceuticals, Inc.
NCT number
NCT07413250

What you commit to

  • Study type: Observational
  • Planned participants: 50
  • From 14 Jan 2026 to the primary completion date, 15 Jan 2030
  • 1 site in 1 country
  • Visits and tests: ask the trial team, the registry does not list them

Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.

Who cannot take part

Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.

Other

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

Who the study is looking for

  • Adult participants aged ≥ 18 years at treatment initiation.
  • Initiated treatment with Ultomiris, Soliris, and/or danicopan on or after IPIG or Alexion International PNH Registry enrollment.

Age in the registry: 18 years to - · Sex: All · Does not accept healthy volunteers

Full criteria on ClinicalTrials.gov

Sites (1)

Sites in registry order. Pick your city on the disease page to sort them by distance.

SiteCityCountrySite status
Clinical Research Site
Coordinator of this site? Confirm its status
BostonUnited StatesUnknown status

This page is information, not medical advice. Discuss any trial with your doctor.