Safety in Hemolytic PNH Patients Treated with Eculizumab: A Multi-center Open-label Research Design Study - SHEPHERD

• Register: EUCTR
• Last refreshed on: 8 May 2012
• Main ID: EUCTR2004-002795-42-SE
• Date of registration: 09/12/2004
• Prospective Registration: Yes
• Primary sponsor: ALEXION PHARMACEUTICALS INCORPORATED
• Public title: Safety in Hemolytic PNH Patients Treated with Eculizumab: A Multi-center Open-label Research Design Study - SHEPHERD
• Scientific title: Safety in Hemolytic PNH Patients Treated with Eculizumab: A Multi-center Open-label Research Design Study - SHEPHERD
• Date of first enrolment: 16/02/2005
• Target sample size: 85
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2004-002795-42
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: no Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no

Countries of recruitment

Germany, Ireland, Italy, Spain, Sweden, United Kingdom

Key inclusion & exclusion criteria

Inclusion criteria:
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

Exclusion criteria:
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


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

paroxysmal nocturnal hemoglobinuria
MedDRA version: 7.0 Level: PT Classification code 10034042

Intervention(s)

Product Name: eculizumab
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: eculizumab
Current Sponsor code: h5G1.1-mAb
Other descriptive name: Anti-C5 antibody
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 10-

Primary Outcome(s)

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.

Main Objective: The primary objective is to evaluate the safety of eculizumab in patients with transfusion-dependent hemolytic paroxysmal nocturnal hemoglobinuria (PNH)

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.
Primary surrogate of efficacy endpoint is hemolysis as measured by lactate dehydrogenase (LDH) area under the curve (AUC).

Secondary ID(s)

C04-002