Hemotrial Un proyecto de SEHH

Ensayo clínico

Ensayo clinico de fase III, multicéntrico, aleatorizado, doble ciego; para evaluar la seguridad y eficacia de dos grupos paralelos de dosis de glucocerebrosidasa humana recombinante expresada en células vegetales (prGCD) en pacientes con enfermedad de Gaucher.A phase III Multicenter, Randomized, Double-Blind Trial to Assess the Safety and Efficacy of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease.

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Resumen

2017-03-15 04:03:56
2007-000498-42
PB-06-001
Ensayo clinico de fase III, multicéntrico, aleatorizado, doble ciego; para evaluar la seguridad y eficacia de dos grupos paralelos de dosis de glucocerebrosidasa humana recombinante expresada en células vegetales (prGCD) en pacientes con enfermedad de Gaucher.A phase III Multicenter, Randomized, Double-Blind Trial to Assess the Safety and Efficacy of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients with Gaucher Disease.
NA
PB-06-001

PROMOTORES DEL ENSAYO
Nombre del promotor Organización Persona de contacto País
Protalix Biotherapeutics Israel

Fármacos

  INFORMACIÓN DE FÁRMACOS USADOS:
  FÁRMACO 1:
Test
recombinant human glucocerebrosidase
prGCD Powder for solution for infusion
Intravenous use

Detalles del Fármaco (Principio Activo):
prGCD recombinant hum

Concentración del fármaco:
U unit(s) equal

200

Contenido del fármaco

No
Si

No
Information no disponible en EudraCT

No
No

No
No

Information no disponible en EudraCT
No

No
No

Si
  FÁRMACO 2:
Test
recombinant human glucocerebrosidase
prGCD Powder for solution for infusion
Intravenous use

Detalles del Fármaco (Principio Activo):
prGCD recombinant hum

Concentración del fármaco:
U unit(s) equal

200

Contenido del fármaco

No
Si

No
Information no disponible en EudraCT

No
No

No
No

Information no disponible en EudraCT
No

No
No

Si
  INFORMACIÓN DE PLACEBOS USADOS:

No hay placebos asignados al ensayo

Información General

Otros

Enfermedad de Gaucher.Gaucher disease.

The change in spleen volume (in procent) from baseline at 9 months measured by MRI.

Change from baseline in: - Liver volume - Platelet count - Hemoglobin other secondary analysis parameter:-Biomarkers (chitotriosidase and pulmonary and activation-regulated chemokine (PARC/CCL18) - Proportion of patients with greater than 10% reduction in spleen volume at 9 months

No


- Males and females, 18 years or older. - Female patients of child-bearing potential or male patients with female partners of child-bearing potential must agree to use two methods of contraception, one of wich must be a barrier method. Acceptable methods of contraception include hormonal products, intrauterine device, or male or female condoms.- Diagnosis of Gaucher disease with leukocyte GCD activity level ?3 nmol/mg*hr (?30 % of the mean activity of the reference range) - Splenomegaly defined as greater than eight times the expected volume [measured volume divided by estimated volume (0.2% of body weight)] as determined by MRI volumetric analysis - Thrombocytopenia (defined as platelet counts <120,000 per mm3) with or without anemia (defined by hemoglobin at least 1 g/dL below normal range according to sex and age). ? Patients who have not received ERT in the past or patients who have not received ERT in the past 12 months and have a negative anti-glucocerebrosidase antibody ? Patients who have not received substrate reduction therapy (SRT) in the past 12 months.? Ability to provide a written informed consent.

- Currently taking another experimental drug for any condition- Presence of severe neurological signs and symptoms, defined as complete ocular paralysis, overt myoclonus or history of seizures, characteristic of neuronopathic Gaucher disease - Previous anaphylactoid reaction to Cerezyme® or Ceredase® - History of allergy to carrots- Pregnant or nursing- Presence of HIV and/or, HBsAg and/or hepatitis C infections- Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the patient’s compliance with the requirements of the study.

Primary efficacy endpoint: Percent change from baseline in spleen volume measured by MRI at 9 months

Fase III
  DISEÑO DEL ENSAYO:

Si
Si

No
No

Si
Si

No
No
  COMPARADOR DEL ENSAYO CONTROLADO:

No
No

Si
No

No

Centros participantes:

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

1
6
  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:

5

Para estudios internacionales:

15
30

Investigadores

  INVESTIGADORES QUE PARTICIPAN EN EL ESTUDIO

Estado actual


 EC Finalizado



EC Finalizado