Active — Not RecruitingNot Applicable

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

United States50 participantsStarted 2025-08-18

Plain-language 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.

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 danicopan treatment initiation (baseline) as add-on therapy to ravulizumab/eculizumab. * Initiated treatment with danicopan on or after registry enrollment Exclusion Criteria: * Participants without known year of birth, sex, informed consent date, or treatment status of danicopan and ravulizumab and/or eculizumab treatment status.

What they're measuring

Number of Participants with Serious Adverse Events (SAEs)

Timeframe: Up to approximately 5 years

Number of Participants with Serious Infections Caused by Encapsulated Bacteria

Timeframe: Up to approximately 5 years

Trial details

NCT IDNCT07413679

SponsorAlexion Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2029-07-01

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