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

N/AActive Not RecruitingNCT07413250

Alexion Pharmaceuticals, Inc.50 enrolled

Overview

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.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Paroxysmal Nocturnal Hemoglobinuria PNH

Interventions

DanicopanDRUG

Participants treated with danicopan as an add-on therapy.

Soliris/UltomirisDRUG

Participants treated with Soliris/Ultomiris monotherapy.

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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.

Locations (1)

Clinical Research Site

Boston, Massachusetts, United States

Outcomes

Primary Outcomes

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

Event Rate of Meningococcal Infections

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

Event Rate of Serious Infections

Time frame: Up to approximately 5 years

Event Rate of Malignancies and Hematologic Abnormalities

Time frame: Up to approximately 5 years

Secondary Outcomes

Number of Participants with AEs and SAEs in Pregnant Participants, Pregnant Partners of Participants and Partners Who are Breastfeeding

Time frame: Up to approximately 5 years

Number of Infant Health Abnormalities in Pregnant Participants, Pregnant Partners of Participants and Partners Who are Breastfeeding

Time frame: Up to 12 months

Number of Participants with PNH symptoms at Diagnosis

Time frame: Baseline (Day 1)

Number of Participants with a History Bone Marrow Transplant

Time frame: Baseline (Day 1)

Number of Participants with a history of Major Adverse Vascular Events, including Thrombosis

Time frame: Baseline (Day 1)

Number of Participants with Ongoing Severe Hepatic Impairment as Defined by Child-Pugh Class C at Enrollment

Data from ClinicalTrials.gov

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