Study of Safety in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Treated With Eculizumab

Alexion Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: Hemoglobinuria, Paroxysmal

Treatments: Drug: eculizumab

Study type: Interventional

Funder types: Industry

Identifiers

NCT00122304

SHEPHERD

C04-002

Details and patient eligibility

About

The primary objective is to evaluate the safety of eculizumab in patients with transfusion-dependent hemolytic PNH

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• PNH > 6 months
• Type III PNH red blood cell (RBC) clone by flow cytometry >10%
• At least one transfusion in the past 2 years but no more than 3 transfusions in the past 12 months; or personal beliefs that preclude the use of transfusion with severe hemolytic PNH
• Lactate dehydrogenase (LDH) >1.5 x upper limit of normal
• Must avoid conception
• Willing and able to give written informed consent

Exclusion criteria

• Platelet count of <30,000/mm3
• Absolute neutrophil count <500/ul
• Active bacterial infection
• Hereditary complement deficiency
• History of bone marrow transplantation
• Participation in any other investigational drug trial or exposure to other investigational agent, device or procedure within 30 days
• Pregnant, breast-feeding, or intending to conceive
• History of meningococcal disease

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)