Hemotrial Un proyecto de SEHH

Ensayo clínico

A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gaucher DiseaseEstudio multicéntrico, aleatorizado, a doble ciego, en grupos paralelos sobre la terapia de reemplazo enzimático con Gene-Activated® glucocerebrosidasa humana (GA-GCB) en comparación con imiglucerasa en pacientes con enfermedad de Gaucher de tipo 1

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Resumen

2017-03-15 04:05:24
2007-002840-21
HGT-GCB-039
A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gaucher DiseaseEstudio multicéntrico, aleatorizado, a doble ciego, en grupos paralelos sobre la terapia de reemplazo enzimático con Gene-Activated® glucocerebrosidasa humana (GA-GCB) en comparación con imiglucerasa en pacientes con enfermedad de Gaucher de tipo 1
GCB039
HGT-GCB-039

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 Cerezyme
Genzyme Europe B.V. Gene Activated Human glucocerebrosidase
GA-GCB Powder for infusion*
Intravenous use

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:
Comparator
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

Information no disponible en EudraCT
  INFORMACIÓN DE PLACEBOS USADOS:

No hay placebos asignados al ensayo

Información General



Otros

Type I Gaucher diseaseEnfermedad de Gaucher tipo 1


To compare the effects of GA-GCB and imiglucerase on hemoglobin concentration in patients with type 1 Gaucher disease.

To compare the effects of GA-GCB and imiglucerase on platelet count To compare the effects of GA-GCB and imiglucerase on liver and spleen volumes (by MRI)To compare the effects of GA-GCB and imiglucerase on Gaucher disease-specific biomarkers (plasma chitotriosidase and CCL18 levels)To evaluate the safety of GA-GCB and imiglucerase in patients with type 1 Gaucher disease, as measured by standard clinical laboratory assessments (including rates of antibody formation and enzyme neutralizing antibody activity) and safety evaluations (including rates of infusion-related adverse events and the proportion of patients requiring premedication use to manage infusion-related adverse events) for each treatment groupTo compare the effects of GA-GCB and imiglucerase on the earliest time to response for hemoglobin (defined as a greater than or equal to 1 g/dL improvement in hemoglobin levels relative to Baseline)

No


The patient has a documented diagnosis of type 1 Gaucher disease, as determined by deficient glucocerebrosidase (GCB) activity relative to normal as measured in leukocytes or by genotype analysis. The patient is at least 2 years of age.The patient has Gaucher disease-related anemia, defined as hemoglobin concentration of at least 0.5 g/dL below the lower limit of normal for age and gender (based on the results obtained during Screening and at Baseline). AND ONE OR MORE OF THE FOLLOWING THREE CRITERIA:The patient has at least moderate splenomegaly (2 to 3 cm below the left costal margin), by palpation. (Patients who have undergone splenectomy must satisfy either inclusion criterion 3c or inclusion criterion 3d to be eligible for this study.)ORThe patient has Gaucher disease-related thrombocytopenia (defined as a platelet count less than or equal to 120 x 10000/cu mm).ORThe patient has a Gaucher disease-related readily palpable enlarged liver.The patient has not received treatment for Gaucher disease (investigational products, miglustat, or imiglucerase) within 12 months prior to study entry, as documented in the patient’s medical history.Female patients of child-bearing potential must agree to use a medically acceptable method of contraception at all times during the study and must have negative results to a pregnancy test performed at the time of enrollment and as required throughout their participation in the study.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).The patient must be sufficiently cooperative to participate in this clinical study as judged by the Investigator.

The patient has type 2 or 3 Gaucher disease or is suspected of having type 3 Gaucher disease. The patient is antibody-positive to imiglucerase or GA-GCB at Screening or the patient has experienced an anaphylactic reaction to imiglucerase or GA-GCB or the patient has required premedication use to manage infusion reactions to imiglucerase or GA-GCB.The patient has received treatment with any non-Gaucher disease-related investigational drug or device within the 30 days prior to study entry; such use during the study is not permitted.The patient is currently receiving red blood cell growth factor, chronic systemic corticosteroids, or has been on such treatment within the last 6 months.The patient is known to be positive for human immunodeficiency virus (HIV), i.e., has a documented positive result. Patients who do not have a documented positive result will be tested for HIV at screening.The patient is known to be positive for hepatitis B and/or C, i.e., has a documented positive result. Patients who do not have a documented positive result will be tested for hepatitis at screening.The patient presents with exacerbated anemia at Screening (e.g., due to folic acid and/or vitamin B12 deficiency).The patient presents with serum transferrin saturation <20 micrograms/dL and serum ferritin < 50 micrograms/dLThe 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.The patient has a significant comorbidity(ies) that might affect study data or confound the study results (e.g., malignancies, primary biliary cirrhosis, autoimmune liver disease, etc.).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, or is otherwise unlikely to complete the study, as determined by the Investigator.

The primary endpoint of this study is to compare the mean change from Baseline in hemoglobin concentration between the two treatment groups.

Fase III
  DISEÑO DEL ENSAYO:

Si
Si

Information no disponible en EudraCT
Information no disponible en EudraCT

Si
Si

Information no disponible en EudraCT
Information no disponible en EudraCT
  COMPARADOR DEL ENSAYO CONTROLADO:

Si
Information no disponible en EudraCT

Information no disponible en EudraCT
No

No

Centros participantes:


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

1
0
  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:


3

Para estudios internacionales:


9
32

Investigadores

  INVESTIGADORES QUE PARTICIPAN EN EL ESTUDIO

Estado actual


Por Determinar



En Marcha