Hemotrial Un proyecto de SEHH

Ensayo clínico

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

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Resumen

2017-03-15 03:56:06
2004-002795-42
C04-002
Safety in Hemolytic PNH Patients Treated with Eculizumab: A Multi-center Open-label Research Design Study
SHEPHERD
C04-002

PROMOTORES DEL ENSAYO
Nombre del promotor Organización Persona de contacto País
ALEXION PHARMACEUTICALS INCORPORATED United States

Fármacos

  INFORMACIÓN DE FÁRMACOS USADOS:
  FÁRMACO 1:
Test
eculizumab
Solution for infusion
Intravenous use

Detalles del Fármaco (Principio Activo):

h5G1.1-mAb Anti-C5 antibod

Concentración del fármaco:

mg/ml milligram equal

10

Contenido del fármaco


No
Si

No
Information no disponible en EudraCT

No
Si

Information no disponible en EudraCT
Information no disponible en EudraCT

Information no disponible en EudraCT
No

No
No

No
  INFORMACIÓN DE PLACEBOS USADOS:

No hay placebos asignados al ensayo

Información General



Anemias, Insuficiencias Medulares

paroxysmal nocturnal hemoglobinuria


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

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.

Information no disponible en EudraCT


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

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

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).

Fase III
  DISEÑO DEL ENSAYO:

No
No

No
No

No
No

No
No
  COMPARADOR DEL ENSAYO CONTROLADO:

No
No

No
No

Si

Centros participantes:


Si
Information no disponible en EudraCT
  TIEMPO ESTIMADO DURACIÓN DEL ENSAYO:

2
0
  TIEMPO ESTIMADO DURACIÓN DEL ENSAYO EN ESPAÑA:


  POBLACIÓN DE PACIENTES EN EL ESTUDIO:
  Población de pacientes: 1

Rango de edad:


0
1

1

Sexo:


1
1

Número planeado de pacientes a incluir:


10

Para estudios internacionales:


45
85

Investigadores

  INVESTIGADORES QUE PARTICIPAN EN EL ESTUDIO

Estado actual


Por Determinar



En Marcha