Hemotrial Un proyecto de SEHH

Ensayo clínico

A phase IV, open-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch from Enzyme Replacement Therapy (ERT) in adult patients with stable Type 1 Gaucher Disease

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Resumen

2017-03-15 03:57:12
2005-001651-37
OGT 918-011
A phase IV, open-label, non comparative, multi-center study to evaluate the long term efficacy and safety and tolerability of oral miglustat as a maintenance after a switch from Enzyme Replacement Therapy (ERT) in adult patients with stable Type 1 Gaucher Disease
OGT 918-011

PROMOTORES DEL ENSAYO
Nombre del promotor Organización Persona de contacto País
Actelion Pharmaceuticals Ltd Switzerland

Fármacos

  INFORMACIÓN DE FÁRMACOS USADOS:
  FÁRMACO 1:
Test Zavesca
Actelion Registration Ltd Miglustat
OGT918 Capsule, hard
Oral use

Detalles del Fármaco (Principio Activo):
OGT918

Concentración del fármaco:
mg milligram(s) equal

100

Contenido del fármaco

Si
No

No
Information no disponible en EudraCT

No
No

Information no disponible en EudraCT
Information no disponible en EudraCT

Information no disponible en EudraCT
No

No
No

No
  INFORMACIÓN DE PLACEBOS USADOS:

No hay placebos asignados al ensayo

Información General

Otros

Gaucher disease is the most common of the glycosphingolipid storage diseases and has autosomal recessive inheritance. For a more precise description, please refer to page 16 of the protocol:1 BACKGROUND AND RATIONALE1.1 Disease

To evaluate the long-term (i.e. over a 24 month period) effect on liver volume of miglustat (100mg TID) as maintenance therapy in adult patients with stable mild to moderate Type 1 Gaucher disease after a switch from Enzyme Replacement Therapy (ERT)

To evaluate the long-term (i.e. over a 24 month period) effects of miglustat (100mg TID) on spleen volume and other markers of Type 1 Gaucher diseaseTo evaluate the long-term safety and tolerability with particular reference to any changes in the neurological assessments (including cognitive function)

Information no disponible en EudraCT


Eligible patients must meet all of the following inclusion criteria:1. Patients aged 18 years or older at the study entry with a confirmed diagnosis of type 1 mild to moderate Gaucher disease at the time of enrollment. Diagnosis will be confirmed on glucocerebrosidase assay or molecular analysis of the glucocerebrosidase gene.2. Patients treated with ERT for at least 3 years and who are receiving their current dose for a minimum of last six months.3. Clinically and biologically stable for the previous 2 years, i.e. (with at least 3 timepoint assessments available for each parameters) a. Stable organomegaly, defined by <= 10% variation. b. Free of progressive symptomatic bone disease (no proven bone disease progression for the last 2 years) c. Controlled hemoglobin level above 11 g/dl , d. Controlled platelets counts with a mean of at least 100x10 Power 9 /l e. Stable chitotriosidase activity, defined by <= 20% variation. 4. Patients who provide written informed consent to participate in the study

Eligible patients must meet none of the following exclusion criteria:1. Patients (males and females) who do not agree to use reliable contraception throughout the study and for three months after cessation of miglustat treatment.2. Female patients who are pregnant or breast feeding, or will not undergo a pregnancy test prior to enrollment into the study.3. Patients with a history of significant lactose intolerance.4. Patients with a confirmed neuropathy.5. Patients suffering from clinically significant diarrhea (>3 liquid stools per day for >7 days) without definable cause within 6 months of Visit 1, or who have a history of significant gastrointestinal disorders (e.g., Irritable Bowel Syndrome).6. Patients with history of cataracts or known increased risk of cataract formation.7. Patients with severe renal impairment i.e., with a creatinine clearance <30 ml/min/1.73mPower28. Patients who are not ambulant, or have severe bone disease according to the bone history and symptom list (see appendix 9)9. Patients splenectomised before 18 years old, because of massive splenomegaly or severe cytopenia.10. Patients with an intercurrent medical condition that would render them unsuitable for study.11. Patients currently undergoing therapy with other investigational product, or who have received an investigational product within 3 months prior to study start.12. Patients with an intercurrent active medical condition such as HIV or Hepatitis B/C that would render them unsuitable for study.13. Patients who for whatever reason are, in the opinion of the Investigator, thought to be unsuitable for the study.14. Patients who have previously received treatment with miglustat.15. Patients with a history of evidence of oculomotor gaze palsies, ataxia or other manifestations typically associated with type 3 (“neuronopatic”) Gaucher disease.16. Predicted hypersensitivity to miglustat or any excipients.17. Patients with current alcohol or drug abuse or dependence.

Percent change from baseline to Month 24 in liver volume For the primary endpoint, the clinically acceptable difference is a mean percentage increase from Baseline to Month 24 of no greater than 10% in liver volume.

Fase IV
  DISEÑO DEL ENSAYO:

No
No

No
No

No
No

No
No
  COMPARADOR DEL ENSAYO CONTROLADO:

No
No

No
Si

No

Centros participantes:

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


30
  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:

40
50

Investigadores

  INVESTIGADORES QUE PARTICIPAN EN EL ESTUDIO

Estado actual


 EC Finalizado



EC Finalizado