Safety in Hemolytic PNH Patients Treated with Eculizumab: A Multi-center Open-label Research Design Study - SHEPHERD
- Conditions
- paroxysmal nocturnal hemoglobinuriaMedDRA version: 7.0Level: PTClassification code 10034042
- Registration Number
- EUCTR2004-002795-42-GB
- Lead Sponsor
- ALEXION PHARMACEUTICALS INCORPORATED
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 97
1. Individuals at least 18 years of age
2. Patients with a diagnosis of PNH > 6 months
3. Presence of a GPI deficient red blood cell clone (type III cells) by flow cytometry equal to or greater than 10%
4. Patients must have
- at least one transfusion in the past two years for anemia or anemia-related symptoms
- or
- personal beliefs that preclude the use of transfusion, with severe hemolytic PNH
5. Documented LDH level greater than or equal to 1.5 times upper limit of normal within 12 weeks of Visit 1 or during the screening period
6. Patients must avoid conception during the trial using a method that is most appropriate for their physical state and culture
7. Patients must be willing and able to give written informed consent
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
1. Platelet count of < 30,000 per cubic millimetre
2. Absolute neutrophil count of less than or equal to 500 per microlitre
3. Presence or suspicion of active bacterial infection, in the opinion of the investigator, at Visit 2 or recurrent bacterial infections
4. Known or suspected hereditary complement deficiency
5. History of bone marrow transplantation
6. Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedure within 30 days prior to screening
7. Pregnant, breast-feeding or intending to conceive during the course of the study, including the Safety Follow-up Visits
8. History of meningococcal disease
9. Patients who are not vaccinated against Neisseria meningitidis at least 14 days prior to Visit 2
10. 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
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: The primary objective is to evaluate the safety of eculizumab in patients with transfusion-dependent hemolytic paroxysmal nocturnal hemoglobinuria (PNH) ;Secondary Objective: The secondary objectives are to assess hemolysis as measured by lactate dehydrogenase (LDH) change from baseline and Quality of Life (QoL) as measured by the FACIT-F SCALE. ;Primary end point(s): Primary endpoints are treatment-emergent adverse events (AEs), clinical laboratory and electrocardiogram data, and vital signs. These data will be summarised for safety-evaluable patients. Adverse events will be assigned MedDRA preferred terms and tabulated as incidence rates. <br>Primary surrogate of efficacy endpoint is hemolysis as measured by lactate dehydrogenase (LDH) area under the curve (AUC). <br>
- Secondary Outcome Measures
Name Time Method