Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH)

Alexion Pharmaceuticals

Status and phase

Completed

Phase 3

Conditions

Paroxysmal Hemoglobinuria, Nocturnal

Treatments

Drug: eculizumab

Study type

Interventional

Funder types

Industry

Identifiers

NCT00122317

E05-001

Details and patient eligibility

About

The purpose of this study is to evaluate the long-term safety of eculizumab in patients with transfusion dependent hemolytic PNH.

Full description

An open-label extension study to evaluate long-term safety of eculizumab in PNH patients who had completed the TRIUMPH (C04-001), SHEPHERD (C04-002), and X03-001 studies.

Enrollment

187 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Patients who have fully completed the TRIUMPH, SHEPHERD, or X03-001 studies
TRIUMPH patients who have discontinued receiving investigational drug prior to the last visit of the study due to lack of efficacy or exacerbation of symptoms of PNH and have completed all monthly safety and efficacy procedures
Patient must be willing and able to give written informed consent
Patient must avoid conception during the trial

Exclusion criteria

Patients who have terminated early from the SHEPHERD or X03-001 studies
Patients who have terminated early from the TRIUMPH study due to an adverse event
Female who is pregnant, breast feeding, or intending to conceive during the course of the study
Any condition that could increase the patient's risk by participating in the study or could confound the outcome of the study