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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00122304
• Date of registration: 19/07/2005
• Prospective Registration: No
• Primary sponsor: Alexion Pharmaceuticals
• Public title: Study of Safety in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Treated With Eculizumab
• Scientific title: Safety in Hemolytic PNH Patients Treated With Eculizumab: a Multi-Center Open-Label Research Design Study
• Date of first enrolment: December 2004
• Target sample size: 85
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00122304
• Study type: Interventional
• Study design: Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 3

Countries of recruitment

Australia, Belgium, Canada, France, Germany, Ireland, Italy, Netherlands, Spain, Sweden, Switzerland, United Kingdom, United States

Key inclusion & exclusion criteria

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



Age minimum: 18 Years
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

Hemoglobinuria, Paroxysmal

Intervention(s)

Drug: eculizumab

Primary Outcome(s)

Primary surrogate of efficacy endpoint is hemolysis measured by LDH area under the curve.

Acceptable safety (adverse events [AEs], labs, electrocardiograms [ECGs], vital signs)

Secondary Outcome(s)

Hemolysis measured by the change of LDH from baseline;

Quality of Life

Secondary ID(s)

C04-002

SHEPHERD

Source(s) of Monetary Support

Please refer to primary and secondary sponsors