Hemotrial Un proyecto de SEHH

Ensayo clínico

Estudio de extensión abierto de terapia de sustitución enzimática con glucocerebrosidasa humana activada genéticamente® (GA-GCB) en pacientes con enfermedad de Gaucher de tipo IAn Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease

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Estudio de extensión abierto de terapia de sustitución enzimática con glucocerebrosidasa humana activada genéticamente® (GA-GCB) en pacientes con enfermedad de Gaucher de tipo IAn Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease


Resumen

2017-03-15 04:07:53
2008-001965-27
HGT-GCB-044
Estudio de extensión abierto de terapia de sustitución enzimática con glucocerebrosidasa humana activada genéticamente® (GA-GCB) en pacientes con enfermedad de Gaucher de tipo IAn Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease
HGT-GCB-044

PROMOTORES DEL ENSAYO
Nombre del promotor Organización Persona de contacto País
Shire Human Genetic Therapies, Inc. United States

Fármacos

  INFORMACIÓN DE FÁRMACOS USADOS:
  FÁRMACO 1:
Test
Gene-Activated Human Glucocerebrosidase
GA-GCB Powder for infusion*
Intravenous use

Detalles del Fármaco (Principio Activo):

GA-GCB Gene activated

Concentración del fármaco:

IU/ml internati equal

100

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 de tipo IType I Gaucher disease


The primary objective of this study is to evaluate the long-term safety of GA-GCB when administered every other week (EOW) intravenously in patients with type 1 gaucher disease.

The secondary objectives of this study are to evaluate the effects of GA-GCB administration on clinical parameters of Gaucher disease, including:-hemoglobin concentration-Platelet count-Liver volume by MRI-Spleen volume by MRI (in patients who have not undergone splenectomy)

No


Patients must meet all of the following criteria to be considered eligible for enrollment:1. The patient has completed through Week 53 of study TKT032 or TKT034, or through Week 41 of study HGT-GCB-039. 2. Female patients of child-bearing potential must agree to use a medically acceptable method of contraception at all times during the study. Pregnancy tests will be performed at the time of enrollment and as required throughout participation in the study. Male patients must agree to use a medically acceptable method of contraception at all times during the study and report a partner’s pregnancy to the investigator.3. The patient, the patient’s parent(s) or legal guardian(s) has provided written informed consent that has been approved by the Institutional Review Board/Independent Ethics Committee (IRB/IEC).4. The patient must be sufficiently cooperative to participate in this clinical study as judged by the Investigator.

Patients who meet any of the following criteria are not eligible for this study:1. The patient has received treatment with any investigational drug, other than GA-GCB, or device within the 30 days prior to study entry; such use during the study is not permitted. Treatment with imiglucerase, if provided as a part of participation in HGT-GCB-039, is allowed.2. The patient is pregnant or lactating.3. The patient, patient’s parent(s), or patient’s legal guardian(s) is/are unable to understand the nature, scope, and possible consequences of the study.4. The patient has a significant co-morbidity(ies) that might affect study data or confound the study results (e.g., malignancies, hepatic cirrhosis, autoimmune liver disease, significant infectious disease, etc.). 5. The patient is unable to comply with the protocol, e.g., has a clinically relevant medical condition making implementation of the protocol difficult, has an uncooperative attitude, is unable to return for safety evaluations, is otherwise unlikely to complete the study, or continued treatment with GA-GCB is not in the best interest of the patient, as determined by the Investigator.

The primary endpoint of this study is safety as assessed by adverse events (including infusion-related adverse events), concomitant medication use, vital sign measurement, physical examinations, and clinical laboratory tests (hematology, serum chemistry, urinalysis, and anti-GA-GCB antibody formation).

Fase III
  DISEÑO DEL ENSAYO:

Si
No

Si
No

No
No

No
No
  COMPARADOR DEL ENSAYO CONTROLADO:

No
No

Si
No

No

Centros participantes:


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

3
3
  TIEMPO ESTIMADO DURACIÓN DEL ENSAYO EN ESPAÑA:


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

Rango de edad:


1
1

1

Sexo:


1
1

Número planeado de pacientes a incluir:


12

Para estudios internacionales:


35
102

Investigadores

  INVESTIGADORES QUE PARTICIPAN EN EL ESTUDIO

Estado actual


EC Finalizado



EC Finalizado