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

N/AActive Not RecruitingNCT07413679

Alexion Pharmaceuticals, Inc.50 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Paroxysmal Nocturnal Hemoglobinuria PNH

Interventions

DanicopanDRUG

Participants will receive Danicopan as an add-on therapy.

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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.

Locations (1)

Alexion Pharmaceuticals, Inc. (Sponsor)

Boston, Massachusetts, United States

Outcomes

Primary Outcomes

Number of Participants with Serious Adverse Events (SAEs)

Time frame: Up to approximately 5 years

Number of Participants with Serious Infections Caused by Encapsulated Bacteria

Time frame: Up to approximately 5 years

Secondary Outcomes

Number of Participants Who Discontinue Study Treatment

Time frame: Up to approximately 5 years

Frequency of Reasons for Discontinuation

Time frame: Up to approximately 5 years

Data from ClinicalTrials.gov

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