paroxysmal nocturnal hemoglobinuria MedDRA version: 7.0 Level: PT Classification code 10034042
Conditions
Interventions
Product Name: eculizumab
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: eculizumab
Current Sponsor code: h5G1.1-mAb
Oth
Sponsors
ALEXION PHARMACEUTICALS INCORPORATED
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Patients have fully completed the TRIUMPH (C04-001), SHEPHERD (C04-002), or X03-001 studies, or, TRIUMPH patients who have discontinued receiving investigational product prior to the Visit 18 due to lack of efficacy or exacerbation of symptoms of PNH and have completed, at minimum, all efficacy and safety procedures at Visits 4, 5, 6, 7, 9, 11, 13, 15, 17 and 18. 2. Patient must be willing and able to give written informed consent. 3. Patient must avoid conception during the trial using a method that is most appropriate for their physical state and culture. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Patients who have early terminated from the SHEPHERD (C04-002) or X03- 001 studies; 2. Patients who have withdrawn from the TRIUMPH study (C04-001) due to an adverse event; 3. Females: pregnant, breast-feeding, or intending to conceive during the course of the study, including the Safety Follow-up Visits; 4. Any condition that, in the opinion of the Investigator, could increase the patient’s risk by participating in the study or confound the outcome of the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective is to evaluate the long-term safety of eculizumab in patients with transfusion-dependent hemolytic PNH. ;Secondary Objective: ;Primary end point(s): Primary endpoints are treatment-emergent adverse events (AEs), clinical laboratory and electrocardiogram data, and vital signs. These data will be summarized for safety-evaluable patients. Adverse events will be assigned MedDRA? preferred terms and tabulated as incidence rates. | — |
Countries
Germany, Ireland, Italy, Spain, Sweden, United Kingdom
Outcome results
None listed