Hemotrial Un proyecto de SEHH

Ensayo clínico

A Phase II single blind, randomized, placebo controlled trial to study the efficacy and safety of anti-von Willebrand factor Nanobody administered as adjunctive treatment to patients with acquired thrombotic thrombocytopenic purpura.Estudio en fase II, ciego-simple, randomizado, controlado con placebo, para estudiar la eficacia y la seguridad del Nanobody Anti- Factor Von- Willebrand administrado como tratamiento adyuvante en pacientes con Púrpura Trombocitopénica Trombótica adquirida

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A Phase II single blind, randomized, placebo controlled trial to study the efficacy and safety of anti-von Willebrand factor Nanobody administered as adjunctive treatment to patients with acquired thrombotic thrombocytopenic purpura.Estudio en fase II, ciego-simple, randomizado, controlado con placebo, para estudiar la eficacia y la seguridad del Nanobody Anti- Factor Von- Willebrand administrado como tratamiento adyuvante en pacientes con Púrpura Trombocitopénica Trombótica adquirida


Resumen

2017-03-15 04:14:54
2010-019375-30
ALX-0681-2.1/10
A Phase II single blind, randomized, placebo controlled trial to study the efficacy and safety of anti-von Willebrand factor Nanobody administered as adjunctive treatment to patients with acquired thrombotic thrombocytopenic purpura.Estudio en fase II, ciego-simple, randomizado, controlado con placebo, para estudiar la eficacia y la seguridad del Nanobody Anti- Factor Von- Willebrand administrado como tratamiento adyuvante en pacientes con Púrpura Trombocitopénica Trombótica adquirida
ALX-0681-2.1/10

PROMOTORES DEL ENSAYO
Nombre del promotor Organización Persona de contacto País
Ablynx Belgium

Fármacos

  INFORMACIÓN DE FÁRMACOS USADOS:
  FÁRMACO 1:
Test
Anti-von Willebrand Factor Nanobody
ALX-0081 Solution for injection
Intravenous useSubcutaneous use

Detalles del Fármaco (Principio Activo):

ALX-0081 Nanobo Anti-von Willeb

Concentración del fármaco:

mg/ml milligram equal

5.0

Contenido del fármaco


No
Si

No
Information no disponible en EudraCT

No
No

No
No

Information no disponible en EudraCT
No

No
No

No
  INFORMACIÓN DE PLACEBOS USADOS:
  PLACEBO 1:

Si
Solution for injection

Subcutaneous use

Información General



Trombopenia

Acquired thrombotic thrombocytopenic purpura (TTP)Púrpura Trombocitopénica Trombótica adquirida (PTT)


Reduction of time-to-recovery, defined by the achievement of laboratory blood marker response, confirmed at 48 hours after the initial reporting of this response

? Improvement in number of patients responding to therapy? Reduction in, plasma exchange (PE) procedure- related items ? Reduction of time to resolution or improvement of symptoms typical of TTP, including blood markers? Reduction of number of exacerbations and relapses? Improvement of cognitive level at steady state post acute phase? Improvement of clinical symptoms and organ function ? Reduction in mortality at 30 days following PE.? Reduction of concomitant treatment-related complications? Evaluation of safety and immunogenicity of adjunctive treatment with ALX-0081? Determination of pharmacokinetic (PK) and pharmacodynamic (PD) characteristics of ALX 0081 in patients with acquired TTP

No


1. 18 years of age or older2. Men or women willing to accept an acceptable contraceptive regimen3. Patients with clinical diagnosis of TTP4. Necessitating plasma exchange 5. Patient accessible to FU6. Obtained, signed and dated informed consent

1. Platelet count greater or equal to 100,000/µL 2. Severe active infection indicated by sepsis (requirement for pressors with or without postive blood cultures)3. Clinical evidence of enteric infection with E. coli 0157 or related organism4. Anti-phospolipid syndrome5. Diagnosis of disseminated intravascular coagulation (DIC) 6. Pregnancy or breast-feeding7. Haematopoietic stem cell or bone marrow transplantation-associated thrombotic microangiopathy8. Known congenital TTP 9. Active bleeding or high risk of bleeding10. Uncontrolled arterial hypertension11. Known chronic treatment with anticoagulant treatment that can not be stopped safely, including but not limited to: - vitamin K antagonists - heparin or LMWH - non-acetyl salicylic acid non-steroidal anti-inflammatory molecules12. Severe or life threatening clinical condition other than TTP that would impair participation in the trial13. Subjects with malignancies resulting in a life expectation of less than 3 months 14. Subjects with bone marrow carcinosis15. Subjects who cannot comply with study protocol requirements and procedures. 16. Known hypersensitivity to the active substance or to excipients of the study drug17. Severe liver impairment

Time to response, based on the following criteria: - Recovery of platelets equal to or above 150,000/µL- This response must be confirmed at 48 hours after the initial reporting of platelet recovery equal to or above 150,000/ µL by a de novo measure of platelets equal to or above 150,000/µL and LDH equal to or less 2 X ULN

Fase II
  DISEÑO DEL ENSAYO:

Si
Si

No
Si

No
Si

No
No
  COMPARADOR DEL ENSAYO CONTROLADO:

No
Si

No
Si

No

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:


3

Para estudios internacionales:


65
110

Investigadores

  INVESTIGADORES QUE PARTICIPAN EN EL ESTUDIO

Estado actual


EC Finalizado



EC Finalizado