Active — Not RecruitingNot Applicable

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

United States50 participantsStarted 2026-01-14

Plain-language summary

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.

Who can participate

Age range18 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * 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. Exclusion Criteria: * Participants without known year of birth, sex, informed consent date, or treatment status of danicopan and Ultomiris and/or Soliris.

What they're measuring

Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)

Timeframe: From initiation of danicopan until discontinuation + 7 days, or end of follow-up, up to 5 years

Event Rate of Meningococcal Infections

Timeframe: From initiation of danicopan until discontinuation + 7 days, or end of follow-up, up to 5 years

Event Rate of Serious Infections

Timeframe: Up to approximately 5 years

Event Rate of Malignancies and Hematologic Abnormalities

Timeframe: Up to approximately 5 years

Trial details

NCT IDNCT07413250

SponsorAlexion Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2030-01-15

View on ClinicalTrials.gov More Paroxysmal Nocturnal Hemoglobinuria trials