Hemotrial Un proyecto de SEHH

Ensayo clínico

A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase.Estudio abierto multicéntrico sobre la terapia de sustitución enzimática con glucocerebrosidasa humana genéticamente activada (Gene-Activated®, GA-GCB) en pacientes con enfermedad de Gaucher de tipo 1 previamente tratados con imiglucerasa

  • Guardar

  • Imprimir
  • << Volver

Resumen

2017-03-15 04:02:44
2006-006304-11
TKT034
A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase.Estudio abierto multicéntrico sobre la terapia de sustitución enzimática con glucocerebrosidasa humana genéticamente activada (Gene-Activated®, GA-GCB) en pacientes con enfermedad de Gaucher de tipo 1 previamente tratados con imiglucerasa
TKT034
TKT034

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 solution 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

Terapia Celular

Type I Gaucher DiseaseEnfermedad de Gaucher de Tipo I

To evaluate the safety of every other week dosing of GA-GCB in patients with type 1 Gaucher disease who were previously treated with imiglucerase.

To evaluate changes from Baseline in hemoglobin concentration after every otherweek dosing of GA-GCB.To evaluate changes from Baseline in platelet count after every other week dosing of GA-GCB.To evaluate changes from Baseline in liver and spleen volumes by abdominalmagnetic resonance imaging (MRI) after every other week dosing of GA-GCB.

No


The patient has a documented diagnosis of type 1 Gaucher disease, as documented by deficient glucocerebrosidase (GCB) activity in leukocytes or by genotype analysis.The patient has received imiglucerase at a dose ? 60 U/kg and ? 15 U/kg every otherweek for a minimum of 30 consecutive months. Patients must have received thesame dose and dose regimen during the 6 months prior to study enrollment. (Note:Patients who are anti-imiglucerase antibody positive will be allowed to enter thisstudy).The patient is at least 2 years of age.Female patients of child bearing potential must agree to use a medically acceptablemethod of contraception at all times during the study and must have negative resultsto a pregnancy test performed at Screening and as required throughout theirparticipation 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 asjudged by the Investigator.

The patient has a hemoglobin concentration ? 10 g/dL and a platelet count ? 80,000 platelets/cu mm.The difference between the patient’s highest and lowest hemoglobin concentrationcollected over 3 consecutive evaluations is > 1 g/dL, AND/OR, the differencebetween the patient’s highest and lowest platelet count collected over 3 onsecutive evaluations is > 20%. (Note: At least 1 of these values must have been collected atleast 6 months prior to the Screening evaluation and 1 value must be collected during Screening).The patient has type 2 or 3 Gaucher disease or is suspected of having type 3 Gaucher disease.The patient has received treatment with any investigational drug or device within the30 days prior to study entry; such use during the study is not permitted.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 positiveresult will be tested for HIV during Screening.The patient is known to be positive for hepatitis B and/or C, i.e., has a documentedpositive result. Patients who do not have a documented positive result will be testedfor hepatitis during Screening.The patient presents with sustained iron, folic acid and/or vitamin B12 deficiencyrelated anemia during Screening (i.e., non-Gaucher disease-related anemia).The patient, patient’s parent(s), or patient’s legal guardian(s) is/are unable tounderstand the nature, scope, and possible consequences of the study.The patient has a significant comorbidity that might affect study data or confound thestudy 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 relevantmedical condition making implementation of the protocol difficult, has anuncooperative attitude, is unable to return for safety evaluations, or is otherwiseunlikely to complete the study, as determined by the Investigator.The patient has had inconsistent treatment with imiglucerase in the last 6 months such as a drug holiday.The patient has experienced an anaphylactic reaction during treatment withimiglucerase.The patient has received miglustat during the 6 months prior to study enrollment.The patient is currently receiving erythropoietin or chronic systemic corticosteroids inthe last 6 months. (NOTE: Use of intermittent corticosteroids as premedication toprevent infusion reactions is allowed.)The patient has experienced spleen infarction.The patient has bone necrosis.

To evaluate the safety of transitioning patients previously receiving enzyme replacement therapy with imiglucerase to GA-GCB therapy administered as the same number of units as their imiglucerase therapy. Safety assessments include adverse events (including infusion-related adverse events), concomitant medication use, vital signs, 12-lead ECG, physical examinations, and clinical laboratory tests (hematology, serum chemistry, urinalysis, and anti-GA-GCB antibody testing).

Fase IIFase III
  DISEÑO DEL ENSAYO:

No
Information no disponible en EudraCT

Information no disponible en EudraCT
Information no disponible en EudraCT

Information no disponible en EudraCT
Information no disponible en EudraCT

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

Information no disponible en EudraCT
Information no disponible en EudraCT

Information no disponible en EudraCT
Si

No

Centros participantes:

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

1
8
  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:

5

Para estudios internacionales:

27
40

Investigadores

  INVESTIGADORES QUE PARTICIPAN EN EL ESTUDIO

Estado actual


 Por Determinar



En Marcha